EBSIN 04/2014

Nº 4 | Volume 13 | 2014 | 10.00 € | ISSN 1618-8276 | A 60711 |

European Biotechnology News Science & Industry

CENTRAL EUROPE

AMD: Antitrust decision slams Roche and Novartis NORTHERN EUROPE

Denmark joins European life sciences data network Special

Big Data & IP – Linking life sciences and IT WESTERN EUROPE

Ferring makes grab at cell therapy against Crohn’s disease SOUTHERN EUROPE

Novamont leads EU effort to scale up bio-based production

Europa attracts Clinical Trials 01_EBSIN4_14_Titelseite_tg.indd 1

Eastern EUROPE

Polish researcher presents prediction tool for flu strains science & technology

Riboswitches can help improve control of viral vectors

03.04.2014 14:14:48 Uhr

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13.03.14 18:36 02.04.2014 15:59:10 Uhr

N –º 4 | Volume 13 | 2014

3

Euro|Biotech|News

Intro Editorial

Tech-transfer: time to send a wake-up call Pierre-Olivier Goineau, President, France Biotech, Paris

T

here’s no question that Europe’s biotech sector produces high-quality science.

We have world- class research organisations and start-ups, as witnessed by the number of alliances with Big Pharma and the number of products being registered. And let’s not forget that the European biotech industry has more pipeline products than the Big Five pharma companies (Novartis, Roche, Pfizer, Sanofi and Merck ) put together! Nevertheless, the pathway from invention to innovation – from scientific and/or technological progress to a marketed product – remains stubbornly blocked in Europe. Why? The answer is simply that following an inPierre-Olivier Goineau is the itial idea and proof-of-concept, developing co-founder, Vice President and CEO of Erytech Pharma. Before and marketing an invention (whether biotech­ setting up the company, he was a nological or anything else) requires signifiSenior Consultant in Strategy and cant financial resources. European innovation Development at KPMG in Lyons in general is hamstrung by one fundamental (France) where he was in charge of the Health Division. Goineau problem: a lack of access to adequate funding. was chosen as the new President The recent financial crisis broke an upward of the biotechnology association trend in European equity investment in highFrance Biotech in February. He tech, and the continent’s capital risk sector now has a DEA (post-graduate degree) in Management Science and a needs rebuilding. In addition, the stock marMaster’s degree in Pharmaceutical kets have broken down (with the notable excepIndustry Management from the IAE (School of Business Management) tion of France, which has become the European leader in terms of IPOs). In some countries, in Lyons. the ecosystem of analysts/brokers/lawyers/auditors has simply disappeared.

Some efforts, on the other hand, are beginning to pay off, as witnessed by the emergence of a whole ecosystem of maturing life science companies. France Biotech is continuing to help its recently incorporated members to improve their practices and benefit from their elders’ experience. We are also intensifying efforts to bring members into contact with Big Pharma companies in Europe and worldwide, and promote sustainable funding in our sector.

3_EBSIN4_14_Editorial_tg.indd 3

But a lack of funding is our Achilles’ heel. Unlike otherwise similar North American companies in this field, that makes us easy prey. Unless a European biotech company tries to gain a foothold in North America to raise funds, it soon becomes vulnerable. Its know-how and intellectual property can be bought by third parties, who go on to develop the latter in-house.

Asia has also entered the game, but Europe does not always play by the same rules there.

Japan and South Korea use their regulatory agencies as tools for business competitiveness (in the field of cell therapy, for example). The same can hardly be said of the European Medicines Agency. China is investing massively in infrastructure and know-how, but we still have very few links with our Chinese counterparts.

Even countries like Singapore and Taiwan are showing themselves to be very dynamic. And there are plenty of other examples.

The European Union has (at last!) become aware of this situation. However, its initiatives are not scaled to meet the challenges we face! Our innovative, value-added industry deserves much better. Access to European Union funding programmes is still laborious, and restricts inter-company collaborations. The key success factor in our industry is speed. So are we moving fast enough?

As entrepreneurs and representatives of industry bodies, it is up to us to put these arguments on the table and gear up for June’s European elections. I call on you all to send out the same, shared message: “Wake up, Europe!” B

02.04.2014 16:03:24 Uhr

4

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

CIRCUL ATION

European Biotechnology News is published in co-operation with the following organisations: European Biotechnology Net work

Europe: european-biotechnology.net

Germany: biodeutschland.org

Switzerland: swissbiotech.org

Belgium: edma-ivd.eu

Europe: eapb.org

CMYK

CouncilBlue: of European 100/15/0/35 BioRegions Orange: 0/75/90/0

Europe: europabio.org

Europe: cebr.net

Ireland: ibec.ie/ibia

Europe: ebe-biopharma.org

Denmark: danskbiotek.dk

Norway: biotekforum.no

The Netherlands: hollandbio.nl

Austria: lifescienceaustria.at

Sweden: swedenbio.com

Finland: finbio.net

Spain: asebio.com

France: france-biotech.org

Hungary: hungarianbiotech.org

Belgium: bio.be

EuroBiotechNews covers the biotechnology sector of the current 28 EU member states, Norway and Switzerland.

If you would like to subscribe, please refer to Portugal: www.apbio.pt

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Italy: assobiotec.it

www.eurobiotechnews.eu

02.04.2014 16:07:39 Uhr

N –º 4 | Volume 13 | 2014

5

Euro|Biotech|News

contents

Insight EMA debates transparency policy; Junk-food tax; Heard in Brussels

Cover Story 10

Study revives debate on unique names for biosimilars 11 Investment flows back to Europe

12

Agencies report on antibiotic resistance; DG Research update 13 Vote in favour of GM honey; Action against obesity; IP Flash

14

Regulatory Affairs Update on clinical trials

16

Europe’s new Clinical Trials Regulation

Breaking news from the EMA

18

Although the EU’s citizens and companies have profited in countless ways from a borderless Europe, some areas of regulation have not kept up with the times. Now a longoverdue overhaul of legislation involving multinational drug studies has been adopted by the bloc. Its aim is to help finally reduce the amount of bureaucracy involved in filing for permission to carry out a multinational trial in the EU, to streamline the process as a whole – and to cut in half the amount of time it takes to acquire approval.

Economic Focus on newsflow and dividends

19

Stock markets

20

Perspectives Device identification for med-tech products

Services

Special 44

Regional News Northern Europe

22

Central Europe

24

Western Europe

26

Southern Europe

28

Eastern Europe

30

6

Partners & Associations

Big Data & IP Is Big the same as relevant?

33

IP considerations in Companion Diagnostics

36

Leveraging Big Data for new medicines

38

Interview – Peter Langkafel, SAP

40

4

Biopeople News from Prothena Corp. plc, Roche AG, Canbex Therapeutics Ltd., Nov­immune SA, Merck KGaA and the European Food Safety Authority 43 Company index

47

Events What’s on in May-June 2014

49

Encore

50

Imprint European Biotechnology News is published monthly by: BIOCOM AG, Lützowstr. 33–36, D-10785 Berlin, Germany, Tel.: +49-30-264921-0, Fax: +49-30-264921-11, E-Mail: service@eurobiotechnews.eu; Internet: www.eurobiotechnews.eu, Publisher: Andreas Mietzsch, Editorial Team: Thomas Gabrielczyk (Managing Editor), Derrick Williams (Co-editor), Dr. Patrick Dieckhoff, Uta Mommert, Dr. Martin Laqua, Benjamin Stolzenberg; Advertising: Oliver Schnell, +49-30-2649-2145, Christian Böhm, +49-30-2649-2149; Distribution: Marcus Laschke, +49-30-2649-2148; Printed at: Druckhaus Humburg, Bremen; Graphic Design: Michaela Reblin. European Biotechnology Science & Industry News is only regularly available through subscription at BIOCOM AG. Annual subscription fees: € 100.00, Students € 50.00 (subject to proof of enrolment). Prices include VAT, postage & packaging. Ordered subscriptions can be cancelled within 2 weeks directly at BIOCOM AG. The subscription is initially valid for one year. Subscriptions will be renewed automatically for one more year, respectively, unless they are cancelled at least 6 weeks before the date of expiry. Failures of delivery which BIOCOM AG is not responsible for do not entitle the subscriber to delivery or reimbursement of pre-paid fees. Seat of court is Berlin, Germany. As regards contents: individually named articles are published within the sole responsibility of their respective authors. All material published is protected by copyright. No article or part thereof may be reproduced in any way or processed, copied and proliferated by electronic means without the prior written consent of the publisher.

Cover Photo: Part of a historical banknote from 1948 ® BIOCOM is a registered trademark of BIOCOM AG, Berlin, Germany.

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03.04.2014 14:15:55 Uhr

6

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

Cover Story professor of EU law at Italy’s University of Insubria. Speaking at the 17th International Conference on Pharmaceutical Medicine (ICPM) in Berlin, the former head of legal services at the European Medicines Agency (EMA) and senior counsel at Sidley Austin pointed out that companies in the future will only have to “submit and wait”.

© pix4U - Fotolia.com

Slicing through the red tape

Clinical studies

Streamlining trials in the European Union Cost and time pressures in the pharmaceutical industry have not encouraged choosing Europe as a site for late-stage multinational clinical trials in the last decade. According to figures released by the European Commission, companies conducted 25% fewer clinical trials in the bloc in 2011 than they had four years before – a trend that poses a long-term threat to both European competitiveness in the sector and patient access to novel treatments. In April, the bloc took steps to reverse it by adopting harmonised rules for the authorisation of multi­national studies. The new Clinical Trials Regulation, which will replace the current Clinical Trials Directive, is set to reduce the time to approval for a multi­-centre study in Europe from 152 to just 60 days. Even that is twice as long as the US FDA takes. But unlike the Clinical Trials Directive, the Clinical Trials Regulation looks set to reduce bureaucracy and – even more importantly – to provide more planning security for study sponsors.

The industry welcomed the adoption of the new legislation by the European Parliament at the beginning of April. “The regulation is set to reduce the bureaucratic burden for study sponsors, while increasing transparency of study results,” said Siegfried Throm from the German pharma industry association VFA. According to the new rules – which were aligned in previous “trilogue” talks between the European Commission, the European Parliament and EU Health

6-8_EBSIN4_14_cover_story_dw.indd 6

Ministers – companies will benefit from a harmonised authorisation procedure for clinical trials in all 28 Member States without establishing a new, central bureaucracy.

“Under the previous EU Clinical Trials Directive, we had 28 different Member States with 28 different national rules on how to assess and supervise an application. This will change now because the Regulation will create common rules for all Member States,” explained Vincenco Salvatore, a

After the regulation has come into force (by 2016 at the earliest), study sponsors will no longer need to send their application dossiers to regulatory authorities and ethics committees in every single Member State, contributing and adjusting along the way for very different national rules, with a range of different national deadlines. Instead, applications and a list of contributing countries will be filed through a new central IT gateway at the EMA. One “Reporting” Member State then will collect all comments from the contributing countries and votes from ethics committees and send them back within a defined deadline to the study sponsor for review. If there is no feedback from the Reporting Member State after 60 calender days, the application is automatically considered approved.

Although the Reporting Member State will coordinate the assessment, other involved Member States and national ethics committees will review on their own all aspects of the trial that are intrinsically ethical or national in nature – including issues such as compliance with informed consent, appropiate compensation schemes, recruitment, data protection, qualification of investigators, storage and future use of samples.

“Companies can propose but not determine their Reporting Member State of choice,” added Throm. If a contributing Member State vetoes a choice, the Reporting Member State will be determined by a vote.

Harmonisation speeds up time to approval An alliance of national authorities – dubbed the Clinical Trials Facilitation Group – has already proven in practice that better coordination of the authorisation procedure can help speed up the process. In 2009, the

03.04.2014 14:19:01 Uhr

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Our services for Life Sciences Transactions: > International Partnering Projects > Patent and Know-How Licenses > Research & Development Cooperations > M&A-Transactions incl. Corporate Finance

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02.04.2014 17:03:49 Uhr

8

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

Cover story CTFG established what’s known as the Vol­ untary Harmonisation Procedure (VHP), which enables applicants to process author­ isations of clinical trials in several Europe­ an countries simultaneously. For compa­ nies applying under the VHP scheme, the time required for obtaining authorisation for a multinational clinical trial has been reduced from 124 days (2009) to currently 53 days on average.

That’s another reason why the Commis­ sion is convinced the new Regulation will boost the EU’s attractiveness as a site for clinical studies, even if there have been doubts in the industry from the beginning about “emerging” countries challenging important EU study sites such as Germa­ ny, France or the UK. In fact, a search in clinicaltrials.gov does not even confirm the Commission’s claims of a negative trend in the number of study applications (see Ta­ ble) that was cited to encourage the move towards the Clinical Trials Regulation.

Some points of concern remain in the industry. According to Throm, the op­ tion to prolong processing time for bio­ pharmaceutical application dossiers by 50 additional calendar days would be a huge drawback, especially if it is applied not only to gene and cell therapy studies, but also to trials with antibody drugs.

Additionally, the EU pharma industry association EFPIA has voiced concerns about certain aspects of the Regulation

aimed at granting the public more trans­ parency in the area of study results.

Moving towards more transparency The most intense debates between EFPIA, the EMA and other stakeholders are not fo­ cused on the online publication of brief sum­ maries of study results for both health pro­ fessionals and the wider public. Nor are they about the additional publication of clinical study reports, which will become obligato­ ry as soon as the EMA has finished setting up a new database. Instead, study sponsors are concerned about the publication of raw data from studies. According to EFPIA’s Di­ rector General Richard Bergström, “greater transparency around clinical trials is need­ ed, but it must be a responsible transpar­ ency.” In other words, sponsors want the EMA to take the protection of private and commercially sensitive data more into ac­ count. The compromise solution that has been adopted by the European Parliament foresees measures for securing confidential business information and patient data in any given case from misuse.

Initiative to fast-track the approval of new therapies In addition to the Clinical Trials Regu­ lation, another ambitious EMA project kicked off in late March that is expected to

Country

2007

2008

2009

2010

2011

2012

2013*

A Belgium

365

494

432

498

500

523

467

A Denmark

278

333

335

359

403

432

390

A France

680

873 (561)

938 (525)

934 (532)

1,051 (584)

1,094 (535)

1,063

A Germany

893

1,074 (813)

1,079 (833)

1,120 (794)

1,149 (843)

1,075 (805)

900

A Italy

547

637

592

656

658

109

622

A Poland

318

389

352

390

410

380

269

A Spain

486

594

543

609

710

693

656

A Sweden

257

357

362

351

362

369

335

A UK

632

807

842

876

850

934

868

(3,755)

(3,152&)

(2,846)

(2,904)

(2,635)

3,805

4,038

4,273

4,518

4,830

A USA A Europe**

3,105

4,680

Number of applications for clinical trials according to www.clinicaltrials.gov. Search criteria: interventional study, country, all phases, all sponsors. In brackets: Number of industrysponsored trials in selected countries. *Possible further late registrations; **geographically

6-8_EBSIN4_14_cover_story_dw.indd 8

attract pharmaceutical developers to Eu­ rope. With “adaptive licensing”, the agen­ cy is inviting companies to participate in a pilot project that is aimed at speeding up the process of providing potentially lifesaving medicines to patients with critical illnesses. Under the programme, stakehold­ ers – including the EMA, the study spon­ sor and patient groups – would participate in informal discussions about the techni­ cal and scientific aspects of a product. The agency also says it wants to involve health technology assessors (HTAs) early on in the iterative assessment of promising treat­ ments for unmet medical needs that have gone through safety but not efficacy test­ ing. The goal is to start testing in a limited patient population, and then broaden ap­ plications step-by-step with increasing evi­ dence of a positive benefit/risk ratio.

According to the EMA’s senior medical of­ ficer Hans Georg Eichler, adaptive licens­ ing should “maximise the positive impact of new medicines on public health by balanc­ ing timely access for patients with the need to provide adequate evolving information on their benefits and risks.” Eichler also says the adaptive licensing pilot is designed for broader application than conditional mar­ keting approval, which is used only for cases that involve a life-threatening disease.

Although the British Industry Associa­ tion (BIA) supports the EMA’s pilot project, it ran into opposition from the EU Commis­ sion as well as German HTAs when it was first introduced back in 2012. Legal coun­ sel at the European Commission (DG SAN­ CO Unit D5) had doubts about the legality of adaptive licensing. The German G-BA, which decides on whether drugs should be reimbursed by state health insurers, said that adaptive licensing could be per­ ceived as lowering EMA standards. Attor­ neys from the FDA, the EMA, and Singa­ pore’s Health Sciences Agency (HSA) have confirmed that existing statutes provide authority for adaptive licensing. Howev­ er, the European Commission has not yet adopted an official position signalling sup­ port for the new approach. Taken together, however, the new Regulation and adaptive licensing have the potential to significant­ ly improve access to urgently needed treat­ ments in Europe.

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10

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

INSIGHT EUROPE EMA

Transparency takes time A London/Brussels – After delaying the finalisation of its new information poli­ cy on the publication of results from clini­ cal trials of approved medicines, the EMA announced in March it will complete ne­ gotiations in mid-June. Agency plans to proactively release clinical trial data faced stiff opposition from the pharmaceutical industry, which wants to keep trade se­ crets and distinct data under its control.

Originally, the EMA had been planning to present new rules governing how to grant researchers access to anonymised patientlevel data on 1 January 2014, but over 1,000 comments on its draft policy and litigation with Intermune and Abbvie have delayed publication of the hotly-debated rules. B

France/EU

Cola contribution A Paris/Brussels – A French proposal to put a tax on junk foods whose consumption is linked to cardiovascular disease has been added to next year’s EU Council agenda.

Senators Yves Daudigny (PS) and Cathe­ rine Deroche (UMP), who drafted the re­ port ”Taxation and public health: evalua­ tion of behavioural taxation”, are aiming specifically to slap a 20% tax on soft drinks in a legislative attempt to limit the risk of cardiovascular disease and cardiovascular events. The report concludes that the tax could partly help compensate for the public health costs that result from junk-food con­ sumption. According to recent research, se­ rious cardiac events can occur in genetical­ ly predisposed people who have consumed energy drinks. ”Our objective is to say that certain products are unhealthy,” says De­ roche, who will present the report to French Health Minister Marisol Touraine. EU min­ isters will discuss a health regulation in re­ sponse to the report next year.

B

10-11_EBSIN4_14_HIB_tg.indd 10

Heard in Brussels M

Throwing out the baby with the bathwater

Brussels – As we all know, the Europe­ an Commission is under intense pres­ sure to reduce costs and shed the image of being a gravy train. This is a favour­ ite topic at the national level in the on­ going fight against the reign of terror from Brussels.

The irony is that the era of eye­ watering salaries and mansions on leafy avenues actually ended some time ago.

While there are still plenty of Commis­ sion people who live very well ­– cour­ tesy of getting their feet under the ta­ ble back in the old days – many new staff face short contracts, lower wag­ es and substantially less sexy perks. I am all for the European Commission employing people on a realistic level (i.e. like the rest of us), but I am having problems with additional changes in structure that somebody thought was an ’efficiency’.

One cut too many?

The EC is a huge funding body, driving innovation and industrial development across Europe. To do that, it needs pas­ sionate and motivated staff who are in­ tegrated into the heart of the scientific process. Based on what I have seen my­ self and heard from plenty of others, the increased use of external agencies to ad­ minister project funding isn’t serving collaborative research well. Sure, the agencies come cheaper and let the EC talk about reducing costs, but the direct result is that you get people paid to ad­ minister – not to add value to – world­ class research collaborations.

Another irony is that there is a queue of people 10km long wanting to work for the Commission, and it includes talented scientists and project manag­ ers. So why make them just administer project reports? Managing collabora­ tive research is really hard, we all know

Claire Skentelbery, Secretary General of the European Biotechnology Network

that, so why not invest a bit more to en­ sure that it actually works? These guys would work on the same salaries as you pay external staff. In fact, I bet that those external staff would jump at the chance to stretch their brains beyond correctly completed personnel tables.

Investing in results The fact is that the European Commis­ sion will always take the heat for na­ tional issues. It is a useful whipping boy on any aspect of money because its budgets are so huge, and it always looks shifty because its answers are necessar­ ily complicated. The Commission is not going to win any debates at national lev­ el, regardless of how much it cuts budg­ ets, so it should stand up and say that it is proud to have skilled Project Officers working as partners with the projects that it funds. Then those projects have the maximum chance of delivering the results that they were funded to achieve, while the Directorates deliver­ ing the funding can also see the fruits of their labour and assess the effective­ ness of their policies.

B

02.04.2014 16:11:45 Uhr

N –º 4 | Volume 13 | 2014

INSIGHT EUROPE

Naming and tracking biosimilars

© Hospira

A Brussels – The World Health Organisation (WHO) is meeting in Geneva in April to discuss proposals on how to find a consistent naming process for bio­similars that assures they can be clearly distinguished from originator compounds. Before it kicked off, the European biotech association EuropaBio was already promoting results from a study suggesting that using the same generic name (International Non-Proprietary Name, INN) for an original biologic drug and its bio­similar can be misleading. As evidence, the association pointed to a non­ -representative survey conducted in November 2013 by Industry Standard Research for the Alliance for Safe Biologic Medicines (ASBM) among nephrologists, rheumatologists, dermatologists, neurologists, endocrinologists and oncologists. In the survey, 22% of the respondents considered themselves to be very familiar with biosimilars, but 20% were not able to properly define what a biosimilar is, while 4% had never heard of them before. EuropaBio concluded those results indicate that a false attribution of adverse events is possible ­– if not likely – due to mixups. Furthermore, says the organisation, it may also send the wrong impression that these medicines are structurally identical. According to EuropaBio, 54% of the physicians surveyed, most of whom worked in hospitals or clinical centres, thought that having the same INN meant

10-11_EBSIN4_14_HIB_tg.indd 11

the products were structurally identical, as is the case for generic drugs. Although ”the results showed that the doctors prefer to use brand names when prescribing biological medicines and reporting adverse events, the findings around the use of INN and its meaning in the context of bio­similar products, lead us to conclude that the use of distinguishable INN for all biologics, including biosimilars, is critical to further strengthen and facilitate patient safety through effective pharmacovigilance,” said the association in a press release.

© Fotolia.de

EuropaBio

Communication

and dissemination services for EU-funded consortia

Different proposals on the table Biosimilar producers, however, point out that today’s standard is to report the product’s brand name together with the batch number when adverse effects occur. ”That has worked well, and shows no problems with traceability,” says Hospira’s VP of Biologics Paul Greenland. He believes that among the proposals made by the WHO, ”the one attracting most interest is to add an additional identifier to the product that is separate to the INN.” Adding a specific identifier that identifies the manufacturer or the Marketing Authorisation Holder would leave the current INN naming convention intact, says Greenland, ”whilst adding a means to accurately trace biological medicines in regions where pharmaco­vigilance reporting systems may not be as advanced as those within the EU.” Biosimilar producers like Amgen favour clear naming. ”It would only be logical to express the nonidentity between innovator bio­logic and biosimilar in the drug naming or INN,” says Deputy Director Karl-Heinz Grajer. Europa­ Bio Secretary General Natalie Moll has announced the launch of an information campaign: ”The results of the survey indicate an understanding of biosimilars isn’t yet widespread among physicians. In the coming months, EuropaBio will commit to raising awareness through an open dialogue with physicians and regulators that will include workshops at the Member State level.” B

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03.04.2014 14:21:16 Uhr

12

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

Insight Europe ness model not based on a therapy, but Horizon is generating revenues with its technology platforms focusing on gene editing and human cell-line production. Despite those successes, many firms will still opt for the tech-focused Nasdaq rather than a European listing in order to access capital from investors experienced in the biotech industry. Britain’s Lombard Medical even decided in March to delist from London and switch to the Nasdaq, with a subsequent US$80m IPO there.

stock markets

Listing in Europe – is it now or never?

Dubbed by analysts the “biggest British IPO in a generation”, Circassia’s successful entry on the London stock exchange raises the question of whether its example is a one-off or the start of a sustainable new trend. The picture across the rest of the continent is mixed.

French companies are seizing the chance to leverage some money during the current IPO window, but companies from Switzerland and Germany continue to keep a low profile.

Circassia’s potential market is huge. The British biotech’s Phase III product is aimed at those who suffer from cat-associated allergies. Its follow-up products include new vaccines – all based on synthetic peptides – for dust mites, grass and ragweed. At the end of the public offering, Circassia’s share price had settled at the top end of the range, tying up a £200m (a240.8m) package for the company – far surpassing the hoped-for £175m. The IPO values the drug developer at £581m, and the first days of trading in late March were low-key. With the adrenalin still high, finance gurus have begun prophesying a wave of public debuts on European stock markets.

The NASDAQ addiction

© Linds :) / flickr.com

The biotech IPO boom in the US has been attracting money for several months now, while European markets watched with envy.

Many of the continent’s biotechs have chosen to cross the pond to go public. Oxford

Immunotec chose to join the Nasdaq last year, as did Dutch companies Prosensa and Uniqure (early 2014). Israel-based Galmed Pharmaceuticals also preferred the US to Europe. The liver disease and gallstone specialist filed on the Nasdaq in February and raised up to US$35m. Belgian Cardio3’s IPO in Brussels and Paris last summer looked slightly forlorn in comparison.

“Circassia is a British company with British technology,” CEO Steven Harris told EP Vantage when asked why he chose to stay in Europe. A risky attitude, as investors still remember some extremely painful biotech failures in Britain. Companies like Ark Therapeutics (£55m IPO in 2004) and Renovo (£50m IPO in 2006) disappeared long ago – along with all the capital that had been invested in them. In late March, Horizon Discovery Group followed in the footsteps of Circassia by raising £68.6m at the AIM on the London Stock Exchange.

Its original target was £25m. The massive oversubscription was unusual for a busi-

Circassia is trying to provide relief for people who are allergic to cats.

12_EBSIN4_14_insight_tg.indd 12

Who’s hot, who’s not If things get rolling in Europe, says news agency Reuters, then the UK, Switzerland and France will likely be at the forefront.

Swiss firms AC Immune, Novimmune, Polyphor and Molecular Partners are all said to be weighing up possible IPOs, but no tangible facts have surfaced so far. The situation is different in France, which according to Bio­PharmAnalyses is experiencing a “spring fanfare”. Dijon-based medtech firm Crossject raised a total of a17m in February, while biopharma Oncodesign – also from Dijon – followed up in late March with a a12.8m IPO. Both listed on the Alternext, and both yielded prices at the top of the proposed ranges. Cell therapy specialist Txcell from Valbonne in March announced plans to list on the Euronext, saying it wants to raise up to a34.9m. And vaccine developer Genticel from Toulouse (aiming at a39.7m) and diagnostics expert Genomic Vision from Paris (a26.4m in a best-case scenario) had their placements running at the time of the EuroBiotechNews editorial deadline.

“The market is receptive to good companies that have a well thought-out story,” Harris told Reuters. “I don’t know that there will be a lot, but I’m sure there are a number of other good-quality companies that will come to market in Europe.” Hanspeter Gehrer, head of corporate finance at Swiss bank Vontobel, is more cautious: “In the current environment, investors are not prepared to take too big a bet. That means that firms must be close to generating cash flows and profits.” Translated into bio­pharma speak: Without convincing Phase II data, floating an IPO in Europe will remain a shaky proposition. B

03.04.2014 14:22:19 Uhr

N –º 4 | Volume 13 | 2014

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INSIGHT EUROPE

DG Research update M

Mining for life sciences gold with European research

Brussels – This year’s slogan at the BIO International Convention (June 23-26, San Diego) “Connect, Partner, Innovate” perfectly fits to the European Commission’s (EC) goal to boost translation of research results into applications. In the 10th year of EC participation, DG Research[1] will increase visibility of a resource designed during FP6 and FP7 programmes to help projects and their results to flow in the exploitation pathway. The “Health Competence” database is more than an information resource on the outcomes and partners within the world’s largest research funding programme. It’s also a tool to drive innovation through the initiation of new cooperations and the synergy of public and private partners. A special panel session at BIO will provide insights on Health Competence as a tool for tech transfer and beyond.

All that glitters Exploiting projects funded by the taxpayer is a Holy Grail for governments, because it emphasises they are generating economic returns on the tax euro/

Torbjörn Ingemansson Principle Scientific Officer, DG Research and Innovation, European Commission [1] Short for: Directorate-General for Research and Innovation

13_EBSIN4_14_Letter from DG Research_tg.indd 13

dollar, creating jobs, building sectors and meeting unmet needs. Project completion is not the end of this process, but the beginning, and in a global economic crisis they need to effectively deliver technologies to end-users and markets. The panel “Mining for gold – creating business opportunities from publicly funded collaborative research” brings together key tools and platforms that help technologies progress beyond the project.

This is not just technology transfer; this is technology maturation and delivery – involving an evo­lution of research results and research partners.

The Health Competence database is a multi-million euro tool created by the European Commission with a global perspective. It brings together more than 23,000 collaboration opportunities from EC-funded projects taking place all over the world. The tool to underpin future research collaborations, business development and clustering technologies will be presented by Torbjörn Ingemansson from the European Commission.

Health Competence brings a global perspective through its Advisory Board, which includes Bonny Harbinger, Deputy Director at the NIH’s Technology Transfer Office, who will emphasise the importance of global exploitation and practical access to technologies. Industry uptake and exploitation of EC-funded projects – particularly among SMEs – is crucial, and the CSO of Collaborative Drug Discovery Inc. Sean Ekins will explain how projects drive their commercial development. Finally – it is all about the people. Project exploitation comes through collaboration, and Claire Skentelbery from the European Biotechnology Network will be sharing techniques and platforms where effective partnerships are created for the exploitation of research. B

Antibiotic Resistance

A situation that could be worse A Paris/Brussels – Food-borne bacterial pathogens like Salmonella and Campylobacter are showing significant resistance to common antimicrobials, but levels of multi-drug resistant strains have remained low. That is the central conclusion reached by the European Union Summary Report on antimicrobial resistance in zoonotic and indicator bacteria from humans, animals and food. The report was published at the end of March by EU food watchdog EFSA and epidemiologists at the European Centre of Disease Control (ECDC). It shows that clinical resistance in humans to commonly used anti­ microbials in Salmon­ella isolates was frequently detected at the EU level, with almost half of the tested isolates resistant to at least one antimicrobial, and 28.9% of them multidrug-resistant. However, levels of clinical resistance and co-resistance in Salmonella spp. isolates to critically important antimicrobials were low – less than 0.2% co-resistance occurred across the 12 Member States that submitted data. The same picture emerged when EFSA and the ECDC searched for resistant Campylobacter strains in clinical isolates.

MDR bugs not yet a major problem And the same proved true in animals, especially in chickens, pigs and turkeys. Coresistance of Salmonella, Campylobacter and E. coli strains to critically important anti­microbials ciprofloxacin and cefotaxime was either not detected or reported at very low levels in the 12 Member States.

In 2014, the ECDC is launching the EU protocol for harmonised monitoring of anti­microbial resistance in human isolates of Salmonella and Campylobacter. “We expect to get more accurate data from countries and as a result better comparability of data,” said Johan Giesecke, Chief Scientist at the ECDC. B

04.04.2014 11:52:25 Uhr

14

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

INSIGHT EUROPE EUROPEAN COMMISSION

IP FLASH

Battling obesity

Dr. Manja Epping, Partner Taylor Wessing, Munich

Dr. med. Christina Berchtold, Dipl.-Jur.

ditional nomenclature provisions are nec In December 2012, the European Comessary to clearly distinguish between inmission issued a directive in the context dividual products. of recognition of medical prescriptions Meanwhile, several proposals for unique among Member States that requires bioname identifiers are being discussed at logical products to be identified by brand WHO and EU levels. One approach to names and not by international non-proclear identification is to use a two-part prietary names (INNs). By stressing the name, with the first part being the INN of need to ensure correct identification of the reference prodbiologics due to the Biosimilars: uct and the second “special characterisidentifying the SBP. tics” of these prodNaming debate intensifies A comparable soluucts, the Commistion has been implemented in Australsion contributed to an ongoing debate ia, where SBP names are made up of the about the naming of biosimilars that has reference product INN and a biosimilar since grown in intensity. qualifier consisting of the prefix sim(a)The INN nomenclature scheme was initiated by the WHO in 1950 to assign nonand a three-letter code. In Japan, SBPs proprietary names to medicinal substancare identified by the name of the manues, meaning that each product could be facturer and a ‘BS’ suffix. globally recognised by a unique name.

On the other hand, advocates of the idea Although initially intended to facilitate that common INNs should be shared prescriptions, INNs now play an essenby SBPs and their reference product artial role in tracking and traceability by gue that the established INN system has attributing adverse events to the right worked effectively, and that new names product. or qualifiers could confuse both patients At present, biosimilars (similar biologiand healthcare professionals. They point cal products, SBPs) usually have the same out that unique identification is already INN as the reference biological product, achieved by brand names, or the INN with the only exception that glycosylatplus the manufacturer’s name. According ed SBPs are identified by adding a Greek to stakeholders, using SBP qualifiers like letter to the INN (e.g. Epoetin α, Epoetin “BS” in Japan could also have a negative ζ). However, Greek letter suffixes may impact on patient access to biosimilars. not be sufficient to ensure product trackWhile the majority of EU Member States ing and prevent accidental substitution. currently agree that biosimilars should Since even small molecular differences have the same INN as their reference can have a significant clinical impact – product, the outcome of the WHO INN in particular in immunogenic reactions programme debate remains open. Dis– stakeholders and experts claim that adcussions at the EU level are ongoing. 

14-15_EBSIN4_14_IP_tg.indd 14

 Brussels – Just under 10% of EU Member States’ accumulated health budgets are spent on diabetes. Speaking at the European Diabetes Leadership Forum, EU Health Commissioner Tonio Borg said in March that more than half of all Europeans are at an elevated risk of heart failure or stroke due to obesity. He called on Member States to fight the trend through prevention programmes.

At the meeting, Novo Nordisk’s EU head Jerzy Gruhn also said public-private partnerships could ensure that people with diabetes gain access to treatment. 

EUROPEAN PARLIAMENT

Vote in favour of GM honey  Brussels/Strasbourg – A European Parliament (EP) committee has backed rules drafted by the European Commission that allow beekeepers to circumvent GMO labeling of honey. A slim majority (28 in favour, 25 against) of the members of the EP’s ENVI committee voted in mid-March to define pollen as a natural constituent of honey, but not as an ingredient. The decision, which is in direct contrast to a 2011 judgement by the European Court of Justice defining pollen as an indredient, means that products must be only be labelled if GMO pollen makes up more than 0.9% of the honey. “In practice, that means never,” said Harald Ebner from Germany’s Green Party, “because honey never contains more than 0.5% pollen.” The parliamentary vote in mid-April now appears to be a question of form, as the new rules have already been informally agreed with the ministers of EU Member States.

“Scrapping labelling of genetically modified pollen means scrapping consumers’ right to choose between genetically modified and natural honey,” criticised Ilaria Passarani from the European Consumers’ Bureau (BEUC). 

03.04.2014 14:35:54 Uhr

C c a s e t c p c m

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

15

INSIGHT EUROPE REACH

FP7

Taking aim at endocrine disruptors Affair of the heart  Brussels/Strasbourg – EU Member States have increased pressure on EU Environment Commissioner Janez Potocˇnik to table a strategy on endocrine disruptors that harm health and the environment. In February, Swedish Environment Minister Lena Ek threatened to begin legal action against the Commission if Potocˇnik further delays his promise to provide a coherent definition of endocrine disruptors based on the hazards they cause for human health and the environment. The Environment Commissioner, who originally promised to do so by last December, now wants to assess the impact of chemical groups such as phthalates, brominated flame retardants and heavy metals. By March, French members of the European Parliament tabled a report urging the

Commissioner to take action. It states that endocrine disruptors add significant extra costs to health systems.

However, prohibiting some of the chemicals that are often found in cosmetics, food, pesticides, and drinking water is not a simple task, because doing so can impact on EU-US trade relations. According to a report published last November by the crop association CropLife Americas, EU regulations on crop protection products that are classified as endocrine disruptors would have detrimental effects on US exports. Estimates say that a ban would have the potential to negatively impact 40% – or more than US$4bn – of all agricultural commodity exports from the US. The lobbyists are asking for improved international coherence. 

 Brussels – Vascular damage can accumulate for years before it translates into cardiovascular disease (CVD). An EU consortium led by the University of Graz is now taking a systems biology approach to tackling early diagnosis by identifying underlying disease pathways, biomarkers and target molecules. Overall, the SYSVASC consortium will receive a6m over the next four years. The funding, awarded through the European Commission Seventh Framework Protocol (FP7), will go toward research to identify protein biomarkers that could be early indicators of incipient CVD. Although the project description calls for the inclusion of preclinical and clinical industry members, most of them named come from academia. 

Contract Manufacturing Excellence

Cobra provides a comprehensive biologics and pharmaceuticals service offering, with experienced project teams nurturing customers’ products from pre-clinical through to clinical and commercial manufacture.

Find out more at: www.cobrabio.com 14-15_EBSIN4_14_IP_tg.indd 15

03.04.2014 14:38:52 Uhr

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N –º 4 | Volume 13 | 2014

Euro|Biotech|News

regul atory Affair s

News M

Gene therapy expansion

Berlin – Mologen AG has received the green light from Belgian regulato­ ry authorities to begin the “Impulse” Phase II study in Small Cell Lung Can­ cer (SCLC). The study will compare the safety and efficacy of Mologen’s gene therapy MGN1703 as an addon treatment to the current standard­of-care. According to Mologen, over­ all survival will be determined in 100 patients who have responded to four cycles of chemo­therapy. By Febru­ ary, Mologen had raised an addition­ al a15.7m to finance a Phase III study with MGN1703 in metastatic colorec­ tal cancer.

M

Pulling the plug

Basel – Following a Phase III failure, Roche has halted development of its antibody drug onartuzumab for cmet positive tumours in non-small cell lung cancer.

M

Ambivalent success

London – Atopix’ asthma medicine OC459 has provided proof-of-concept in a Phase IIb study, but failed to dem­ onstrate dose-dependent response.

Administration of a 25mg once­­-daily dose of the CRTH2 blocker, which had been licensed from Oxagen Ltd., re­ sulted in increasing mean percent pre­ dicted force expiratory volume in one second (FEV1) from baseline to week 17 vs. placebo (162 vs. 57 mL, p=0.028).

However, higher doses (100mg and 200mg twice-daily) missed meeting the primary endpoint. In a subgroup of patients with uncontrolled, eosi­ nophilic asthma, each dose of OC459 significantly improved mean FEV1 from baseline to week 17 vs. placebo.

16-18_EBSIN4_14_Regulatory_tg.indd 16

Europe

Update of ongoing clinical trials A Adocia SA (Lyons) has reported positive Phase I/II clinical trials results for its Bio­ Chaperone formulation in patients with type I diabetes. In late March, the French firm announced that the formulation of Sanofi’s insulin analog glargine with Eli Lilly’s fast-acting insulin analog lispro im­ proved short- and long-term blood glucose control versus Humalog Mix, a premix for­ mulation of the insulin analog lispro and protamine from Eli Lilly. According to Acodia, BioChaperone Combo showed a faster onset of action (25±11 vs. 40±13min; p=0.002) and a higher early metabolic ef­ fect (AUC_GIR [0-2h] 504±210 vs. 325±183 mg/kg; p=0.001). Study results also sug­ gest a stronger late metabolic effect (AUC_ GIR [12-30h] 1480±900 vs. 961±553 mg/kg; p=0.026) and a longer duration of action.

30 hours after administration, 17 of the 19 patients treated with BioChaperone Com­ bo were still under glucose control vs. only 6 out of 20 with Humalog Mix (p=0.0002).

Adocia previously reported that it has kicked off a Phase IIa trial to determine the pharmaco­dynamic and pharmaco­ kinetic profiles of its BioChaperone for­ mulation.

The race to grab a significant share in the US$16.6bn market for asthma thera­ peutics is heating up. While Roche’s an­ ti-IgE antibody Xolair (omalizumab) was the first bio­logic drug to be approved as an asthma treatment in 2012, competitors in March reported new results from latestage trials of their candidates, which tar­ get treatment-resistant forms of the five subtypes of the auto­immune disorder. UKheadquartered Glaxo­SmithKline plc said it will submit global regulatory applications for its IL-5 blocker mepolizumab to treat severe eosinophilic asthma based on the results of two studies published in midMarch. In the MEA115588 trial, adminis­ tration of mepolizumab four times a week over 32 weeks met the primary endpoint of reducing the frequency of clinically sig­ nificant asthma exacerbations vs. placebo.

Subcutaneous injection of the antibody drug also reduced oral corticosteroid (OCS) use in subjects with severe refractory asth­ ma during GSK’s MEA115575 trial, while maintaining asthma control vs. placebo.

Another candidate in the asthma pipeline, Boehringer Ingelheim’s add-on treatment tiotropium met the primary endpoint in the Phase III Graziatina-trial – improv­ ing peak forced expiratory volume in one second (FEV1) vs. placebo. The broncho­ dilator that is administered by Boehring­ er’s inhaler Respimat is already approved to treat chronic obstructive pulmonary dis­ ease (COPD) in approximately 60 markets throughout the world. The growing com­ petition in the asthma market has induced Roche to extend the treatment scope of its asthma antibody Xolair and push de­ velopment of its IL-13 blocker lebrikizu­ mab. In mid-March, the pharma giant re­ ceived FDA approval for Xolair as a treat­ ment for the 50% of patients with chronic idiopathic urticaria who are treatment-re­ sistant to H1-antihistamines, the only au­ thorised treatment so far. In early March, the Swiss firm reported that lebrikizumab Phase IIb data showed a reduction in asth­ ma attack rates, as well as an improvement of lung function, in a subgroup of adult pa­ tients with severe uncontrolled asthma that express high levels of the biomarker peri­ ostin. Data showed FEV1 was increased by 6.8%, 10.7% and 10.1% in patients adminis­ tered with lebrikizumab doses of 37.5 mg, 125 mg and 250 mg respectively compared with placebo.

Orally available pills that mimic the posi­ tive effects of exercise and caloric restric­ tion on obese and metabolically challenged individuals are a Holy Grail for treating conditions such as fatty liver, non-alcoholic steatohepatitis (NASH) and type 2 diabe­ tes. Based on positive outcomes in animal tests, Swedish biotech company Betagenon AB launched a first-in man Phase I study in March with an AMPK agonist code-named O304. Activated Protein Kinase (AMPK)

03.04.2014 14:39:52 Uhr

N –º 4 | Volume 13 | 2014

17

Euro|Biotech|News

regul atory Affair s regulates fat metabolism and insulin sensitivity in liver, glucose disposal in muscle and glucose sensing in beta-cells, reducing fatty liver and peripheral insulin resistance as well as increasing glucose-stimulated insulin secretion in animal models.

Beta­genon AB and Baltic Bio AB will share profits from the sale of rights on the compound belonging to Baltic Bio AB, a subsidiary of investment company Fort Knox Förvaring AB (Umea).

The FDA has approved Berlin-based Piramal Imaging’s florbetaben 18F injection (Neuraceq™) for PET Imaging of betaamyloid neuritic plaques in the brain, which occur in late-stage Alzheimer’s disease (AD). The decision came on the heels of EU market authorisation granted four weeks before by the European Commission. A negative scan result reduces the likelihood that a patient’s cognitive impairment is due to AD.

French Neovacs SA has announced a positive review by an independent data monitoring committee of its Phase IIb study of TNF-kinoid in rheumatoid arthritis (RA).

The immunotherapy specialist said in midMarch that a review of the safety data re-

Dutch gene therapy specialist uniQure N.V. (Amsterdam) and its EU marketing partner Chiesi Farmaceutici S.p.A. have provided post-marketing data complementing the dataset for Glybera, its conditionally EUapproved gene therapy for lipo­protein lipase deficiency (LPLD). The analysis covers follow-up data for up to six years posttreatment of 13 patients, all of whom met indication requirements for the current labeling of Glybera in the EU. In the analysis, equal time periods of up to six years before and after Glybera treatment were compared to evaluate the number and severity of attacks of pancreatitis in each LPLD patient. The company says that although the data are not statistically relevant, the review suggests that treatment with Glybera provides long-term beneficial effects with regard to the risk of experiencing new pancreatitis events.

sulted in a positive assessment of the study.

140 RA patients are to be recruited within the programme. The Paris-based biotech expects to present results of the study in Q4/2014 The Innovative Medicines Initiative (IMI) has kicked off a a16.3m project to bridge the current information gap between health technology assessment and market approval conducted by different authorities. The GETREAL project is aimed at generating and measuring real-world efficacy data during clinical trials, and will seek to integrate the most appropriate comparators and most clinically meaningful patient subgroups to provide an appropriate database for assessment of the relative effectiveness of new medicines.

16-18_EBSIN4_14_Regulatory_tg.indd 17

At the American Association for Cancer Research (AACR) annual meeting in San Diego, Galapagos NV (Mechelen) presented favourable data on its pre-clinical candidate drug GLPG1790 in triple-negative breast cancer. In this indication, neither the absence of estrogen, progesterone or HER2 receptors affect prognosis for recovery. According to the company, the first-in-class oral selective blocker of the ephrin receptor kinase family showed remarkable in vivo efficacy in a xenograft model. Full tumour blockage was observed after 30 mg/ kg/d. The Dutch firm says GLPG1790 has good drug-like properties, and safety/tolerability studies with the candidate drug look favourable. Galapagos is completing preclinical studies, and is carefully considering a number of options before entering first clinical trials in humans.

B

News M

Proof of quality

Nijmegen – Synthon’s generic glatiramer acetate met the primary endpoint of equivalence with Teva’s originator Copaxone in a Phase III study in patients with relapsing remitting multiple sclerosis (RRMS). In the GATE study, 796 patients with active RRMS were randomised to daily injections of Synthon’s glatiramer acetate, Copaxone or matching placebo for a double-blind nine-month treatment period. The primary endpoint of the study was the number of T1 gadolinium enhancing brain lesions on MRI assessed after seven, eight and nine months of treatment, which were convincingly shown to be equally reduced in both treatment groups compared to placebo. Safety analyses showed a comparable incidence of reported adverse reactions in the treatment groups.

M

Testing continues

Lyon – Valneva announced in late March that it will continue its ongoing Phase II/III study of its Pseudomonas aeruginosa vaccine candidate IC43 according to a recommendation by an independent data monitoring board.

M

Turbo-fertiliser

Darmstadt – Merck KGaA started enrollment of 946 women classified as poor ovarian responders for its Espart Phase III study in March. The German pharma firm is testing Pergoveris for multifollicular development as part of an Assisted Reproductive Technology (ART) treatment cycle. Safety and efficacy of the company’s combination of recombinant follitropin alfa and lutropin alfa will be assessed versus Gonal-f (follitropin alfa).

04.04.2014 11:53:45 Uhr

18

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

REGUL ATORY AFFAIR S QUALITY BY DESIGN

FDA and EMA extend collaboration  London – Ten years after the publication of the first quality-by-design (QbD) guidelines set out by the US Food and Drug Administration (FDA), QbD is gaining momentum in the biopharmaceutical industry. The concept is a systematic approach for assuring a constant high level of quality in biologic products by defining and controlling all of the parameters that determine safety and efficacy throughout a drug’s entire life cycle. To remain in-line with the new paradigm in drug development, companies are employing statistical, analytical and riskmanagement methodology in the design, development and manufacturing of their medicines.

In early March, the European Medicines Agency (EMA) and the FDA announced

the extension of their pilot programme for parallel assessment of QbD applications for a further two years, beginning 1 April.

The agencies kicked off the collaboration in 2011 with the goal of creating a global standard for assessment of QbD applications by the industry. The task is no easy one, as the agencies are still in a steep learning curve on how much data they can demand from biopharmaceutical companies without affecting competitiveness.

Swiss Roche AG was the first company to file a market authorisation application at the FDA for a monoclonal antibody which was completely developed and produced according to QbD. Manufacturing of its glycooptimised CD20 blocker obinutuzumab, which received FDA approval in November

www.ema.europa.eu 2013 and is in line for approval in Europe, requires permanent control of more than 30 critical quality attributes. However, at the recent QbD & PAT Forum at Sartorius in Göttingen, most companies made it clear that their aim is to integrate QbD elements to control production processes rather than implementing full QbD. Parallel assessment of QbD elements and scientific advice given by the EMA and the FDA have proven extremely useful to drugmakers. According to the agencies, two guidance documents for the industry have already been published based on experience. Further guidance is being developed and is expected to be published in 2014. 

Outcomes of CHMP and COMP meetings Drug

Indication

Company

Status

 Entyvio (vedolizumab)

Treatment of ulcerative colitis and Crohn’s disease

Takeda Pharma A/S

MA recom.

 Folcepri (etarfolatide)

Single photon emission computed tomography (SPECT) imaging

Endocyte Europe B.V.

MA recom.

 Jardiance (empagliflozin)

Treatment of type II diabetes mellitus

Boehringer Ingelheim International GmbH

MA recom.

 Neocepri (folic acid)

Enhancement of 99mTc-etarfolatide single photon emission computed tomography (SPECT) image quality

Endocyte Europe B.V.

MA recom.

 Olysio (simeprevir)

Indicated in combination with other medicinal products for the treatment of chronic hepatitis C in adult patients

Janssen-Cilag International N.V.

MA recom.

 Vynfinit (vintafolide)

Treatment of platinum resistant ovarian cancer

Endocyte Europe B.V.

MA recom.

 Sylvant (siltuximab)

Treatment of multicentric Castleman’s disease

Janssen-Cilag International N.V.

MA recom.

 Autologous CD34+ hematopoietic stem cells transduced with lentiviral vector encoding the human beta A-T87Q-globin gene

Treatment of sickle cell disease

bluebird bio France

ODD recom.

 Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19

Treatment of B-lymphoblastic leukemia/lymphoma

Novartis Europharm Ltd

ODD recom.

 Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colonystimulating factor

Treatment of ovarian cancer

Oncos Therapeutics Oy

ODD recom.

 Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues

Treatment of ATTR amyloidosis

Voisin Consulting S.A.R.L.

ODD recom.

16-18_EBSIN4_14_Regulatory_tg.indd 18

02.04.2014 16:24:29 Uhr

Life Sciences in Austria 2014

N –º 4 | Volume 13 | 2014

Euro biotech stock s Market Sentiment

50.000 Euro

Biotech, Pharma and Medical devices

ement ary Manag aws Tempor ry complementa Financing of t expertise men age man maz www.awsg.at/

French biotech – a bubble or more?

Now available! The second edition of the unique directory offers a comprehensive overview of Austrian life sciences companies and manufacturers, distributors, ro 1.000.000 Eu suppliers and service providers, as well as research institutions and organisations. ancing aws Seedfin se start-up pha Financing the companies of life science ncing.at fina www.seed

tors, propelling some companies in a very short time to valuation levels never before achieved (Genfit and Innate Pharma). This interest has in turn had a twofold effect.

There has been a significant wave of IPOs/ rights issues in France – a positive development for patients because it strengthens R&D activities – and we’ve seen a changing of the guard in a hierarchy established in recent years. AB Science, Transgene and Frédéric Nicox are no longer the locomotives of the Gomez, French sector. New names have emerged.

Analyst 500 Euro ge t 3 × 1, bu question The that everyone is asking, of Pharmium s you a s Ide s es ta ines r Busin r teis swhether it will last. Stock market Securitities .000 Euro course, 50 d!

Phase 1 2014 2.6. – 27.11. bubbles have always existed. They are part 10,0 00 Euro Many of the financial market landscape.

In just one quarter, the market cap of ienna LISAvof investors believe that the rise the sector France’s biotech sector increased by 67% ar d Medtech Aw has probably been excessive recently, and or a1.1bn (10 names) while at the same not sufficiently linked to scientific (clinical time that of their Swedish colleagues fell and regulatory) underpinnings.

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1. 15,0 00 Eu gene and the exercise (or not) by Novartis 10,0 00 Euro of its option on TG4010. Bad news could 2.

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D eaustria .at heavily in some companies (Innate Pharww .lifescie ma, DBV and Erytech) to take advantage Companies used to calculate market cap of the attractive valuations. US funds have cash that the vast majority of European inCountry Companies vestors do not have, and they’ve played a A France AB Science, Adocia, BioAlliance, crucial role in this growth. Together with DBV Technologies, Erytech Pharma, Genfit, Innate Pharma, the creation in France of a new system for Neovacs, Nicox, Transgene channeling private savings towards the A Sweden Active Biotech, BioInvent, Karo Bio, biotech industry (PEA-PME), all of this Medivir, Neurovive, Orexo has awakened the appetite of retail inves-

The more than 1,900 entries in the directory ro stem from an OECD- and Eucomed200.000 Eu based survey carried out by BIOCOM AG and Life Science Austria – LISA. d aws PreSee Financing the se pha t star pret www.preseed.a

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d aws PreSee Financing the se pre-start pha t www.preseed.a

19_EBSIN4_14_analyst_bk.indd 19

46-2

978-3-928383-

Life Sciences in Austria 2014 € 29.80 ISBN 978-3-928383-46-2 Available at international booksellers! service@biocom.de www.biocom.de

02.04.2014 16:24:56 Uhr

20

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

Euro Biotech Stock s Company

Genefit

NASH: Stocks in the fast lane A Lille – Genfit’s shares soared in February when the US Food and Drug Administration (FDA) granted the GFT505 development programme in NASH (nonalcoholic steatohepatitis) a Fast Track designation. The French biopharma is currently conducting the Phase IIb study GFT505212-7; its primary objective is to demonstrate the efficacy of one year of treatment with GFT505 on the reversion of biopsydiagnosed NASH. In March, the company strengthened its scientific evidence by demonstrating the curative effects of GFT505 in an experimental mouse model of NASH associated with metabolic disorders. Genfit’s stock price has leapt from around a9 in December 2013 to about a28 in late March.

B

Valneva

Quack approval A Lyons – After the first veterinary vaccine using Valneva SE’s EB66 technology received market approval in 2012, the biotech company announced in late March that it had now received the first marketing authorisation for a human vaccine produced with its duck embryonic stem cell platform. In particular, the Chemo-Sero Therapeutic Research Institute (Kaketsuken) – a co-development partner to GlaxoSmithKline (GSK) – has received marketing authorisation in Japan for an H5N1 influenza vaccine. Kaketsuken will produce pandemic H5N1 vaccine for more than 40 million people within six months after the virus strain for vaccine production is decided. Valneva granted an exclusive commercial license to GSK to develop and market pandemic and seasonal human influenza vaccines worldwide using the EB66 technology. GSK’s agreement with Kaketsuken began back in 2009.

B

20-21_EBSIN4_14_StockMarket_ml.indd 20

Perform

4SC (D)

ê

2

AB Science (F)

3

Abcam (UK)

4

1

Quote

H/L

-13.3%

1.30

2.14/1.30

ê

-11.3%

12.90

20.93/10.86

ê

-18.4%

4.74

6.37/4.72

Ablynx (B)

é

5.3%

9.61

10.00/5.73

5

Acino Holding (CH)

è

0.0%

94.65

95.80/55.96

6

Actelion (CH)

ê

-5.8%

70.98

76.74/40.87

7

Active Biotech (S)

ê

-5.8%

3.87

8.95/3.11

8

Addex (CH)

é

4.2%

1.65

6.56/1.57 15.60/5.02

Adocia (F)

ê

-4.2%

13.79

10

Advanced Med. Sol. (UK)

é

15.7%

1.52

1.58/0.88

11

Algeta (N)

é

1.1%

43.46

43.58/22.51

12

Allergy Therapeutics (UK)

é

4.6%

0.24

0.32/0.08

13

Almirall (E)

ê

-3.2%

12.15

12.91/8.74 15.16/10.54

9

14

Analytik Jena (D)

ê

-0.2%

14.00

15

Ark Therapeutics (UK)

é

17.4%

0.01

0.01/0.00

16

Basilea (CH)

ê

-16.1%

83.95

106.41/40.86

17

Bavarian Nordic (D)

é

1.9%

13.20

14.72/7.53

18

Bayer (D)

ê

-2.3%

97.60

104.05/77.58

19

BB Biotech (D)

ê

-9.6%

132.05

147.80/82.16

20

BioAlliance (F)

ê

-1.0%

8.69

10.54/3.42

21

Biofrontera (D)

ê

-16.0%

3.31

4.90/3.20

22

Bioinvent (S)

ê

-8.5%

0.33

0.51/0.23

23

Bionor (N)

ê

-21.8%

0.29

0.52/0.26

24

Biotec Pharmacon (N)

é

80.5%

2.46

2.46/0.47

25

Biotest (D)

é

3.2%

88.75

93.80/53.93

26

BTG (UK)

ê

-6.1%

6.58

7.41/4.04

27

Cardio3 Biosciences (B)

ê

-1.8%

38.90

52.16/12.50

28

Cellectis (F)

é

46.0%

6.86

7.16/2.19

29

ChronTech (S)

é

5.6%

0.02

0.03/0.00

30

Clinigen (UK)

ê

-0.9%

6.58

8.19/2.86

31

Co.don (D)

é

104.5%

3.15

3.85/0.75

32

Cosmo (CH)

é

15.5%

88.06

88.06/32.92

33

Cytos (CH)

é

1.7%

2.47

3.54/2.24

34

DBV Techn. (F)

ê

-8.9%

18.53

23.40/7.7

35

Deinove (F)

ê

-2.4%

14.88

17.04/9.78

36

Diagenic (N)

ê

-14.9%

0.14

0.55/0.05

37

Diamyd Medical (S)

ê

-0.9%

0.38

0.48/0.25

38

Diaxonhit (F)

ê

-1.1%

0.91

1.05/0.57

39

DSM (NL)

é

7.9%

50.25

59.75/45.88

40

E-Therapeutics (UK)

ê

-4.3%

0.27

0.44/0.24

41

Epigenomics (D)

ê

-13.0%

5.87

8.25/1.44

42

Erytech (F)

ê

-3.9%

13.95

17.50/8.73

43

Eurofins Scientific (F)

é

4.2%

215.25

218.00/138.6

44

Evocutis (UK)

ê

-29.0%

0.00

0.04/0.00

45

Evolva (CH)

ê

-4.2%

1.12

1.29/0.52

46

Evonik (D)

ê

-1.5%

28.46

30.45/24.56

47

Evotec (D)

ê

-4.2%

3.88

4.84/2.09

48

Flamel Technologies (F)

é

34.0%

9.74

10.69/3.10

49

Formycon (D)

ê

-10.3%

7.25

8.65/3.36

50

Galapagos (B)

ê

-2.3%

16.00

20.45/13.41

51

Genfit (F)

é

5.6%

29.3

31.83/4.09

52

Genmab (DK)

ê

-4.9%

29.72

33.92/18.55

53

GW Pharmaceut. (UK)

ê

-13.7%

3.37

4.98/0.56

54

HBM (CH)

ê

-2.7%

63.21

65.84/40.53

55

HybriGenics (F)

é

17.1%

2.60

3.49/0.53

56

Immunicum (S)

é

14.4%

3.22

4.66/0.80

57

Immupharma (UK)

ê

-8.0%

0.63

0.92/0.42

58

Ipsen (F)

ê

-4.6%

30.15

34.65/25.27 1.50/0.08

59

Ixico (UK)

é

5.9%

0.87

60

Karo Bio (S)

é

8.3%

0.10

0.14/0.03

61

Karolinska (S)

ê

-5.5%

2.88

3.81/2.68

All quotes listed in euros. All data without guarantee. Effective Date April 1, 2014. Performance for 30 days. Highs and lows in 52 weeks.

02.04.2014 16:25:43 Uhr

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

21

Euro Biotech Stock s Company

Perform

62

KWS Saat (D)

é

Quote

H/L

0.3%

262.60

291.90/243.20 79.05/47.69

63

Lonza (CH)

ê

-1.5%

75.39

64

Magforce (D)

é

17.9%

6.60

6.64/2.17

65

MDX Health (B)

é

30.3%

4.56

4.96/1.97

66

Medigene (D)

é

37.8%

5.51

17.28/3.42

67

Medivir (S)

é

1.3%

13.06

13.26/7.15

68

Merck KGaA (D)

ê

-3.5%

121.05

135.45/111.00

69

Metabolic Explorer (F)

é

5.0%

3.34

3.71/2.45

70

Moberg Pharma (S)

ê

-3.3%

3.27

4.39/2.94

71

Molmed (I)

é

10.1%

0.76

0.85/0.40

72

Mologen (D)

ê

-6.6%

11.35

15.10/10.20

73

Morphosys (D)

é

5.2%

68.08

71.78/30.47

74

Nanobiotix (F)

ê

-17.3%

14.82

25.70/5.15

75

Neovacs (F)

ê

-5.6%

3.38

4.40/1.52

76

NeuroSearch (DK)

é

0.3%

0.43

0.59/0.41

77

Neurovive (S)

é

10.4%

3.10

3.37/1.76

78

Newron (CH)

ê

-9.9%

13.13

15.88/6.52 2.88/2.31

79

Nicox (F)

é

0.4%

2.57

80

Novacyt (F)

é

1.7%

6.10

8.11/5.40

81

Novozymes (DK)

ê

-3.5%

31.83

34.04/24.41

82

Orexo (S)

ê

-15.3%

15.60

20.49/5.48

83

Oxford Biomedica (UK)

ê

-18.7%

0.03

0.05/0.02

84

Paion (D)

ê

-16.0%

3.63

4.70/0.60

85

Pharming (NL)

é

2.0%

0.51

0.67/0.06

86

Photocure (N)

é

13.0%

3.03

5.00/2.68

87

Plethora (UK)

é

12.6%

0.13

0.22/0.02

88

Prosensa (NL)

ê

-14.3%

4.09

24.20/2.59

89

Prothena (IE)

é

6.4%

27.86

35.12/4.80

90

Qiagen (D)

ê

-4.8%

15.37

18.11/13.99 0.05/0.03

91

Reneuron Group (UK)

ê

-9.4%

0.04

92

Renovo (UK)

ê

-2.7%

0.22

0.24/0.19

93

Roche (CH)

é

1.3%

215.83

223.24/176.78

94

Santhera (CH)

é

6.6%

3.33

3.61/1.10

95

Sareum (UK)

ê

-3.8%

0.01

0.02/0.01

96

Sartorius (D)

é

3.6%

99.50

102.00/76.89

97

Scancell Holdings (UK)

é

1.5%

0.41

0.61/0.28

98

SerodusS (N)

ê

-5.0%

0.46

0.84/0.21 4.55/0.05

Silence Therapeutics (UK)

ê

-15.3%

3.75

100

SkyePharma (UK)

é

19.5%

2.83

2.91/0.53

101

Stallergenes (F)

é

5.9%

58.00

59.90/45.31

102

Stentys (F)

ê

-2.5%

10.33

11.70/8.93

103

Stratec Biomedical (D)

ê

-0.9%

33.19

35.25/29.8

99

104

Swed. Orphan Biovit. (S)

ê

-17.7%

8.05

9.78/4.04

105

Synairgen (UK)

é

5.2%

0.61

0.69/0.41 328.29/252.00

106

Syngenta (CH)

é

3.9%

274.55

107

Therametrics (CH)

ê

-9.1%

0.08

0.16/0.08

108

Thrombogenics (B)

é

1.3%

20.26

39.26/16.63

109

Tigenix (B)

è

0.0%

0.83

0.94/0.20

110

TopoTarget (DK)

é

6.9%

0.43

0.46/0.31

111

Transgene (F)

ê

-3.9%

11.89

13.80/8.18

112

UCB (B)

é

1.0%

58.19

59.20/38.45

113

Valneva (F)

ê

-14.1%

6.45

7.74/3.03

114

Vectura (UK)

ê

-4.4%

1.86

2.06/0.93 0.46/0.24

115

Vernalis (UK)

é

0.7%

0.43

116

Verona Pharma (UK)

ê

-36.3%

0.03

0.07/0.02

117

Virbac (F)

ê

-7.9%

161.00

184.10/138.60

118

Vita 34 (D)

é

10.6%

5.20

7.30/2.78

119

Wilex (D)

é

59.3%

0.94

1.80/0.52

121

Zealand Pharma (DK)

ê

-6.8%

9.25

10.72/7.57

122

Zeltia (E)

ê

-1.5%

2.68

3.00/1.26

All quotes listed in euros. All data without guarantee. Effective Date April 1, 2014. Performance for 30 days. Highs and lows in 52 weeks.

20-21_EBSIN4_14_StockMarket_ml.indd 21

Eurofins

Amsterdam add on A Luxembourg – Eurofins Scientific has grabbed the former Amsterdam water quality control board, now called Omegam Laboratoria BV. One of the leading independent laboratories in the Netherlands for environmental and water testing, it employs more than 100 staff and generates about a10m in revenues. Omegam is a specialist in the analysis of trace level components of pesticides, hormones and drug residues in water and soil. The acquisition reinforces Eurofins’ leadership in the Benelux region in the environmental testing market, and strengthens the Group’s service offering in water analysis. It also gives weight to its platform as the only privately-owned laboratory in the Netherlands’ domestic water-testing market. “This acquisition reflects Eurofins’ strategy of consolidating the markets that we are active in,” said Eurofins CEO Gilles Martin.

B

Novo Nordisk

Split in the works A Copenhagen – NNIT A/S, the information technology unit of Danish pharmaceuticals company Novo Nordisk, said in March it had appointed Morgan Stanley and Danske Bank to advise on the possibility of a listing on the NASDAQ OMX Copenhagen separate from the mother company. Novo Nordisk announced in January that it was looking into the option of spinning off NNIT, saying it wanted to concentrate on its core activities. NNIT published an operating profit (EBIT) of DKK246.5m for 2013, up 13.7% on 2012. “A strong position,” said CFO Krogsgaard Thomsen. “So this means the evaluation of a potential listing on the stock exchange continues as planned.” Thomsen only joined the company’s board – which is headed by CEO Per Kogut – back in January.

B

02.04.2014 16:25:48 Uhr

22

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

northern europe Medical Research

Denmark joins Big Data network

Fresh Danish IPO takes off Biotech Saniona AB has begun trading with a bang on the AktieTorget, an alternative trading platform for young companies. The Danish biotech’s initial public offering was oversubscribed more than six times, leading to a cash inflow of some a12.5m. The share issue comprised 3.4 million shares and brought 770 new shareholders. “We are proud that the total subscription amount exceeded SEK100m, and represents one of the largest subscription amounts ever in an IPO on the AktieTorget,” said Saniona CEO Jørgen Drejer.

The firm’s first official day of trading is slated for 22 April 2014, and its shares will be traded under the ticker SANION. The company was founded in 2011 through the buyout of R&D programmes as well as the transfer of employees from NeuroSearch A/S, including the rights and obligations of NeuroSearch under its agreement with Janssen Pharmaceuticals. Together with investor Atlas Venture, Saniona established a spin-off in the US called Ataxion in 2013. The AktieTorget market runs a so-called “multilateral trading facility” (MTF), which is oriented towards developing businesses. Running an MTF is a financial service according to Swedish financial law. That puts the exchange under the supervision of the Swedish Financial Supervisory Authority.

B

22-23_EBSIN4_14_Nörthern_pad.indd 22

cess to data that will enable us to shift up a gear in terms of driving the research and innovation that will ensure the growth and jobs that are needed to support Denmark’s welfare society,” says Sofie Carsten Nielsen, Denmark’s Minister for Higher Education and Science. The Danish Technical University (DTU) in Lyngby will head the initiative, while the Universities of Copenhagen, Aarhus and Southern Denmark will also participate. The consortium is additionally working with a wide range of Danish medical and biotech partners, including Lundbeck, Novo Nordisk, Novozymes and Exiqon.

D

© Dreaming Andy/fotolia

InDex Pharma

Denmark has ratified the Elixir agreement, and a participating consortium has been granted a3.5m from the country’s National Programme for Research Infrastructures, which secures Danish participation in the programme. Elixir is an intergovernmental organisation that is building on existing data resources and services within Europe. Its main office is located in EMBL-EBI in Hinxton (UK). The project’s primary aim is to orchestrate the collection, quality control and archiving of large amounts of biological data produced by life science experiments. Elixir’s stated goal is to create an infrastructure that integrates research data from all corners of Europe and ensures a seamless service provision that it is easily accessible to all, thereby facilitating discoveries that benefit humankind. Denmark is joining a group of Elixir countries that includes the Netherlands, the UK, Estonia, Switzerland, Sweden and the Czech Republic.

The Scandinavian country hopes that sharing research data could help significantly boost Danish research. “Danish research is renowned worldwide for its ability to apply data. Now we have greater ac-

Protein drugs

Cooperation with a long half-life Novozymes has sealed a collaboration with Johnson & Johnson research unit Janssen R&D LLC. The agreement allows Janssen to evaluate Novozymes’ biopharma platform Veltis to develop potential drug candidates. Novozymes thinks human albumin is suited as a half-life extension approach for protein drugs, as it is a natural, non-immunogenic plasma carrier protein that is stable, highly soluble and large enough to avoid renal clearance. Experts believe that albumin is recycled in the body due to its interaction with its receptor – the neonatal Fc receptor (FcRn). FcRn is present

on many cell types, and is thought to be responsible for rescuing the molecule from degradation in the lysosome.

The extended half-life of the albumins opens the door to reducing dosing frequency of drugs from daily to weekly or even monthly cycles. “The Veltis platform offers real potential to revolutionise the delivery of treatments. Working with partners across a wide-range of medical conditions, we are continuing to highlight the benefits of the solution in improving the lives of patients,” says Novozymes Senior Director Svend Licht.

B

02.04.2014 16:26:44 Uhr

N –º 4 | Volume 13 | 2014

23

Euro|Biotech|News

northern europe Successful bid

News

Landslide response to takeover

M

Bayer AG has finalised its takeover plans for Norwegian pharmaceutical company Algeta. The Leverkusen-based pharma giant has received acceptance notification for 97% of Algeta’s share capital in response to its takeover offer. Bayer paid NOK362 (a43.67), bringing the total value of the Oslo-based specialist for alpha particle­-emitting cancer pharmaceuticals to a1.9bn. Bayer is paying a premium of 37% on top of the closing price on the day before the bid became public. Bayer and Algeta cooperate on a licensing agree-

ment for Algeta’s lead drug (Alpharadin), which is already approved in the US. Algeta fits with Bayer CEO Marijn Dekkers’ strategy of driving growth by building up the company’s pharmaceuticals division, which now overshadows chemicals in importance. Bayer also sees high market potential for Xofigo, a radioactive agent that migrates to parts of the body with abnormal bone growth in prostate cancer patients. Although Xofigo sales only totalled US$17m in Q3/2013, they are expected to rise to US$1bn or more by 2018.

B

Cellular specialists unite

Lund – Two Swedish biotech specialists are joining forces to develop new therapies for mito­chondrial disorders. Neurovive Pharmaceuticals AB and A1M Pharma hope that the collaboration will give both parties access to a broader scientific platform. The two companies are located in the Medicon Valley biotech cluster.

“The collaboration will improve the potential for both parties to conduct leading research in mito­c hondrial medicine.

Innovation M

Put the pedal to the – wood? the parts on the car: UPM Formi is a recyclable biocomposite manufactured from cellulose fibre and plastics, while UPM Grada is a thermoformable wood material that enables ecological designs. The car is also designed to run on UPM BioVerno, a wood-based renewable diesel that can reduce emissions of greenhouse gases. The Biofore Concept Car premiered first at the Geneva Motor Show in early March. D

Full of biomaterials – the new Bioconcept Car from UPM.

22-23_EBSIN4_14_Nörthern_pad.indd 23

© UPM

At the International Day of Forests in March at the UN’s Palais des Nations in Geneva (Switzerland), UPM and the Helsinki Metropolia University of Applied Sciences introduced their jointly designed Biofore Concept Car. Wherever possible, the car is constructed from renewable bio­ materials that originate from responsibly managed forests. Two biomaterials from UPM in particular were used in most of

Novo expands R&D

Baegsvaerd – Danish global healthcare company Novo Nordisk is investing more than a 73m in drug development capacities at a new research and development facility in Baegsvaerd. Slated to be fully operational by late 2016, the pilot plant will be run by CMC Supply (Chemistry, Manufacturing and Control Supply) – a unit within Novo Nordisk R&D. CMC Supply develops, produces and formulates all new protein and peptide processes at the company. “The new pilot plant will significantly increase our capacity for early-phase diabetes projects. It will also create up to 35 new jobs over a two-year period, in addition to the 100 new employees CMC Supply will hire in Denmark in 2014,” said CMC Supply Senior VP Jesper Bøving. The project initially involves the establishment of the pilot plant with just one purification line. However, the facility can later be expanded to double that size to increase capacity. In total, five kilometres of steel pipes will be laid in the purification pilot plant, which is to have a total area of 2,700 m2.

02.04.2014 16:26:51 Uhr

24

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

Centr al europe Financing

Glycotope strikes biobetter bounty

Alzheimer’s

Pioneering Dx

© National Institute on Aging/National Institutes of Health

With the help of the validated Absoluteido p180 test kit from Biocrates Life Sciences AG, US researchers have developed the first blood test that can predict the risk of Alzheimer’s disease up to three years before the disease has become clinically manifest – with an accuracy of 90%. Innsbruck-based Biocrates develops meta­bolomics solutions for targeted, quantitative and quality-controlled metabolic pheno­t yping. Alzheimer’s is an irreversible, progressive brain disease that currently affects more than 35 million people worldwide. At the moment, there are no cure or disease-modifying therapies available to treat it. The study’s findings were published in Nature Medicine (doi:10.1038/nm.3466). 

In Alzheimer’s disease, connnections between neurons begin to break down

24-25_EBSIN4_14_Central_Europe_um.indd 24

By raisinga55m in additional equity, biobetters specialist Glycotope GmbH has brought in record additional financing for a German biotech firm. The Berlin-based company specialises in the glycooptimisation of proteins. With its proprietary technology GlycoExpress, the company can attach specific glycosylation patterns to protein therapeutics – thus improving efficacy and tolerability of the drug in addition to lowering manufacturing costs.

The ultimate goal is to produce biologics with superior properties.

Glycotope will use the money it raised for the development of its two most advanced new cancer drugs, which are currently both in Phase IIb trials. Antibody Pankomab-Gex targets the tumour­specific epitope TA-MUC1, while Cetugex is an anti-epidermal growth factor receptor monoclonal antibody. Both antibodies showed strong anti-tumour activity and in early-stage clinical studies.

The cash injection came from Munichbased Jossa Arznei GmbH (Strüngmann Group) and the ELSA GmbH (Eckert Life Science Accelerator) in Berlin. With the additional investor backing, CEO and founder of Glycotope Steffen Goletz is thinking big: “We are extremely encouraged by the confidence shown by our investors, and by the fact that – thanks to these funding commitments – Glycotope is becoming one of Germany’s largest independent bio­tech companies.”

The market for biobetters is hotly contested. There are currently around 20 glyco­ optimised antibodies in clinical development, and many companies are working on improving the biological activity and pharmacokinetics of recombinant proteins. But Glycotope isn’t afraid of the competition.

“We are the only company worldwide that has demonstrated that its technology can glycooptimise non-antibody molecules,” boasts COO Franzpeter Bracht.

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Diagnostics

Bet on blood poisoning Looking to expand, Swiss-based company Debiopharm has invested again in molecular diagnostic company Immunexpress for the development of sepsis diagnostic products. In a financing round led by the Swiss bio­pharma group, the sepsis expert secured US$6m in funding. The money it raised will be ploughed into Immunexpress’ latestage sepsis diagnostic product Septicyte. The test quantifies multiple specific molecular markers from the patient’s immune system for earlier diagnosis, detection, severity assessment and optimal targeting of drugs and other therapies. “Support and funding from Debiopharm will provide us with the resources necessary to advance and commercialise SeptiCyte technology,”

said Immunexpress President and CEO Roslyn Brandon.

The Debiopharm Group’s cooperation with Immunexpress goes back to 2012, when Debiopharm – together with Swiss Biocartis SA – forged a collaboration with the diagnostics firm and invested an initial US$2m. Immunexpress is a privatelyheld group of companies with locations in Brisbane (Australia) and Seattle (US).

The Debiopharm Group has also announced that it is making plans to expand activities at Debiopharm Research & Manufacturing S.A., its industrial development and production facility in Martigny (Switzerland), through the acquisition of industrial high-value added activities or companies in the pharmaceutical, biotech or medtech field.

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Centr al europe amd

Antitrust decision hits Swiss giants News M

Drugmakers Roche and Novartis have been fined a182.5m by the Italian anti­-trust authority for purportedly participating in an anticompetitive agreement in the market for ophthalmic drugs. The two companies are accused of colluding to exclude Roche’s anti-angiogentic therapeutic antibody Avastin, which is not approved as treatment for age-related macular degeneration (AMD) but often used off-label by physicians as alternative to the much more expensive drug Lucentis. Lucentis was jointly developed by Roche’s US subsidiary Genentech and Novartis.

According to the authority’s evidence, the two pharma giants colluded from 2011 on to create an artificial differentiation between the two products. To influence prescription choice among doctors and health services, Avastin was reported to be less

safe in intravitreal use than Lucentis. This point of view is shared by the Italian regulatory authority AIFA (see p. 42). Avastin has been approved since 2004 as cancer treatment, and has also been used offlabel to treat AMD. Lucentis is an Avastin especially formulated for intravitreal use. Recent studies give a mixed picture about the equivalence of the two products. A major difference between them, however, is the price. In Italy, an off-label injection of Avastin costs a81, while an injection of Lucentis costs more than ten times as much (around a900). According to the Italian antitrust authority, the illicit collusion caused additional expenses estimated at over a45m in 2012, with increased future costs possibly exceeding a600m per year. Both companies say they plan to appeal the decision.

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Antibiotics

EU Inducement

Target: Bacteria Vaccine prize Antibiotics are back in the limelight at Roche. UK biotech Discuva Ltd and the Swiss pharma goliath are joining forces to discover novel antibiotics against multi-drug resistant bacteria. The two companies announced that they have entered a collaboration and license agreement for the discovery and development of drugs used for the treatment of infections caused by multi-drug resistant Gram-negative bacteria. Discuva’s Selective Antibiotic Target Identification (SATIN) technology, a next-generation sequencing and bio­informatics platform applied to unique bacterial transposon libraries, will be used to identify bacterial targets and select promising drug candidates. Discuva will receive an upfront payment of a11.6m in addition to certain milestone payments: Each product may net up to a127m upon achievement of certain development, commercialisation or sales milestones.

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Biopharma firm Curevac GmbH has bagged the EU’s first innovation inducement prize, which is worth a2m. The German company received the award for its technology developed to store vaccines safely at high or low temperatures. The European Commission (EC) offered the prize to stimulate solutions for a major barrier to using vaccines in developing countries: the need to keep them stable at hot or cold temperatures. The Commission praised the winner, adding that “whilst Curevac GmbH was already working on the technology when applying, the prize opened their eyes to the potential of RNA-based vaccines for developing countries because of their high thermostability.“ Other such inducement prizes are “foreseen”, said the EC. Curevac is currently investigating their vaccines, which are based on mRNA molecules that stimulate the immune system, in a number of clinical trials.

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Hemophilia boost

Leverkusen – Bayer HealthCare AG is investing a record a500m in the production of hemophilia A medicines by establishing additional capacities for the manufacture of recombinant factor VIII hemophilia products that are currently under development. The move will create around 500 additional jobs at Bayer’s sites in Leverkusen and Wuppertal.

Currently, their approved hemophilia A drug – one of the company’s best-selling products – is only manufactured in the US.

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Animal Dx deal

Zurich – Swiss company Prionics AG, a privately held animal health diagnostics company, has been acquired by Thermo Fisher Scientific.

With the acquisition, the US giant aims to expand its portfolio of animal health testing products under the Life Technologies brand, which was created in early February when Thermo bought Life Technologies Co. Founded in 1997, Zurich-based Prionics has a major production facility in Lelystad (Netherlands).

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Big business advice

Karlsruhe – Big pharma and biotech veterans have come together to found Acteaventures GmbH. The new global advisory company will specialise in clients from the life science industry, and will offer services in the area of business development and licensing, M&A transactions, scientific evaluation, strategic advisory and assistance in financing rounds.

Acteaventures will have operations in Europe, Asia and the US.

04.04.2014 11:55:22 Uhr

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Western europe Private Placement

Stocking up the war-chest

GSK India heads back to its roots GlaxoSmithKline (GSK) has raised its stake in Indian pharma subsidiary GSK Pharmaceuticals Limited. The Mumbaibased company, which is one of the oldest pharmaceutical companies in India, announced the voluntary open offer last December, and in early March the parent firm confirmed it has increased its stake in the Indian company from 50.7% to 75%. The British pharma picked up 20,609,774 shares at a36.72/share from shareholders in its Mumbai-based affiliate, valuing the transaction at approximately a760m. The final payment for shares tendered and accepted was completed by 20 March, at which point the British drugmaker acquired full beneficial ownership for them.

GSK’s Chief Strategy Officer David Redfern said the company was “very pleased with the outcome of this transaction, which further increases our exposure to a strategically important market,” and called it “a significant vote of confidence in the future growth prospects of our pharmaceuticals business in India and...GSK’s long-standing commitment to the country.” The open offer, which was managed

by HSBC Securities and Capital Markets (India) Private Ltd, commenced on 18 February and closed on 5 March. GlaxoSmithKline Pharmaceuticals Limited will remain publicly-listed.

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man and CEO of Transgene. In concert with South Korean R&D company Sillajen and Chinese bio­pharma Lee’s Pharmaceutical, Transgene plans to initiate a global Phase III study in the first-line treatment of advanced hepatocellular carcinoma, as well as several additional Phase I/II trials in different cancers, both as a single agent and in combination with a variety of other treatments.

Transgene is also hoping for more cash from a lung cancer vaccine deal with Novartis. Stéphane Boissel, Executive Vice President and CFO of Transgene, explained that “with nearly a100m in cash, we now have the means to take the next steps in Transgene’s corporate development and implement our strategy with confidence. Should Novartis exercise its option on TG4010, our cash runway would be extended to at least the end of 2016.”

Further details were not disclosed.

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R&D cooperation

Sanofi and UCB go halves Sanofi SA launched a 50-50 research and development collaboration with Belgian biopharmaceutical company UCB SA in mid-March. The partnership will focus on the discovery and development of anti-inflammatory small molecules to be discovered by UCB that can potentially treat a range of immune-mediated diseases. The deal could be worth more than a100m to the Brussels-based company, which will be entitled to an upfront payment from Sanofi as well as preclinical and clinical development milestone payments. Overall, Sanofi and UCB will share costs and profits on an equal basis. President of Global R&D at Sanofi Elias Zerhouni called immune­ -mediated diseases “a significant public health burden”, and adding that by joining efforts with UCB, Sanofi “will address a scientific challenge in immun­ology and

increase the chances of accelerating the discovery and development of future therapies.” UCB is focussed on severe diseases of the immune system. Their research team has used a new approach to identify small molecule modulators of a biological pathway for which parenterally administered biologic therapies have proven highly efficacious in patients. 

© UCB

Voluntary open Offer

In a two-step capital increase, French bio­tech company Transgene SA (Strasbourg) raised a65.5m through a rights issue and a private placement at the end of March. At the same time, the company unveiled late-stage clinical development plans for its oncolytic immunotherapy Pexa-Vec with partners Lee’s Pharmaceutical Holdings Limited (Hong Kong) and Sillajen Inc. (Busan).

In the first step of the capital increase, a rights issue launched on 28 February, Transgene raised total gross proceeds of a45.5m through the issuance of 4.5 million new ordinary shares. The follow-up step, carried out on 24 March, was a private placement of a20m through the issuance of two million additional shares.

“Transgene is on the brink of an important transformation, with the coming launch of two clinical studies intended for registration,” said Philippe Archinard, Chair-

04.04.2014 11:56:34 Uhr

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Western europe Cooperations

News

French vaccines – Made in Korea

© Rawpixel, fotolia.com

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French giant Sanofi Pasteur SA and South Korean SK Chemicals Co. have entered into a collaboration to produce a vaccine against pneumococcal infections. Announced in mid-March, the agreement between the Paris-based vaccine division of Sanofi-Aventis SA and its new Busan-based partner to cooperate in the research, development and commercialisation of the pneumococcal conjugate vaccine targets a global market worth a2.9bn. As part of the agreement, Sanofi Pasteur will make an up-front payment of a16.5m to SK Chemicals, which is to produce the vaccine at its Andong­-based production facility. After the vaccine is registered, recently re­organised Sanofi Pasteur will launch the product with

shared profits outside of South Korea, while SK will retain exclusive rights in the country. The two companies are co-investing in the vaccine project. “With this agreement, Sanofi Pasteur will enlarge its unique portfolio of products, embracing the value of open innovation,” said Sanofi CEO Olivier Charmeil. “This is an important milestone for SK and for Korea,” added CEO of SK Chemicals In-Serk Lee. ”We are proud to be able to develop and manufacture in Korea a premium vaccine that has the potential to be distributed worldwide.” The WHO considers pneumococcus diseases like pneumonia and meningitis a major global public-health problem, and recommends the use of vaccines in all countries.

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Licensing agreement

A deal that TxCell can stomach

Chippenham – In mid-March, Britain’s Vectura Group plc purchased Munich-based respiratory disease specialist Activaero GmbH (Germany) for a total of a136m, to be funded through existing cash and equity payments. These in part consist of a95m payable at completion, made up of a45m in cash and a50m in new Vectura ordinary shares. Activaero’s proprietary smart nebuliser-based technology allows drug deposition into targeted areas of the lung, and is currently used in seven clinical and several preclinical stage programmes.

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of inflammatory bowel disease, including Crohn’s disease and ulcerative colitis.

TxCell wants to undertake an upcoming Phase IIb trial with Ovasave in refractory Crohn’s disease. If Ferring decides to proceed, the French firm will be responsible for further development and commercialisation. Ferring paid a1m upon signature, and a further a2m will be paid depending on the results of Phase IIb studies slated to start in the second half of 2014. TxCell will also be eligible for development, commercial and manufacturing milestones and payments totalling up to a73m.

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T-cell therapy honors

Ox ford – On 24 March, Adapt­ immune Limited (UK) was awarded a a2.5m grant from the Biomedical Catalyst Fund, which is managed by the UK’s innovation agency and the Medical Research Council.

The award is to help with the further development in the clinic of Adapt­ immune’s second engineered T-cell therapy programme in triple-negative breast cancer. Trials are planned for 2015.

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French biotech TxCell and Swiss bio­pharmaceuticals firm Ferring International Center SA have signed a development and license agreement potentially worth up to a76m, plus milestone royalities. The Valbonne­-based TxCell focuses on personalised cell-based immuno­ therapies using antigen-specific regulatory T-cells for auto­immune and chronic inflammatory diseases. Under the terms of the agreement, Ferring retains an option to acquire a global development, manufacturing and marketing license for TxCell’s lead candidate Ovasave for the treatment

Take a deep breath

Reading aid

Gosselies – Belgium-based OncoDNA SA, a company providing DNA nextgeneration sequencing tests for clinical use in cancer, announced a supplier agreement with Saudi Arabia’s NBCC (National Blood and Cancer Center) in mid-March. The aim of the agreement is to bring affordable, state-of-the-art DNA sequencing technologies to the GCC countries to help oncologists make the best decisions for treatment.

02.04.2014 16:29:31 Uhr

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Southern europe Orphan Diseases

Gene therapy for Sanfillipo syndrome

Fresh funds for fresh ideas Two Spanish venture capital initiatives made the news in late March. Caixa Capital Risc – the VC arm of Barcelona-based savings bank La Caixa – did so by setting up Caixa Innvierte BioMed II. In total the company is by now managing a103m.

Among its first funds was Caixa Capital BioMed from 2011, which has so far distributed a13m in total to 13 companies.

Now waiting in the wings, its successor (worth a35m) has been prepared in cooperation with the Spanish Ministry for Economy and Competitiveness’ Centre for Technological and Industrial Development (CDTI). The money is slated for investment in life sciences company Series A and B rounds. Supported companies must be specialised in either the development of new diagnostics, therapies or medical technologies. Caixa Innvierte BioMed II will invest between a0.5m-a4.5m per company.

Almost simultaneously, another Barcelona­-based VC firm – Ysios Capital – said it will start revving up its second life science fund. Ysios BioFund II will aim to gather a100m to finance up to 15 life sciences companies with “disruptive products, platforms or technologies in the pharmaceutical and medtech and diagnostics segments.” Its a69m predecessor, which started back in 2008, saw some successful exits among its 10 fosterlings.

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undisclosed upfront payment in the deal, and is eligible for milestones and royalties on net sales. In patients, the accumulation of non-degradable heparan sulfate glycosaminoglycans in the brain causes a range of neurological symptoms, whereas accumulation in the periphery can lead to enlargement of the liver and spleen. Most patients with MPSIIIA die as teenagers.

Généthon will be responsible for the development of the manufacturing process of the investigational gene therapeutic and its production for preclinical toxicology studies, the clinical trial and eventually for commercial use. Esteve plans to start clinical Phase I/II trials with the gene therapy programme in 2015. 

© Wikimedia Commons

Venture Capital

Esteve S.A. has pushed forward its Orphan Drug designated gene therapeutic for the treatment of Mucopolysaccharidosis type IIIA (MPSIIIA, or Sanfilippo A Syndrome). In March, the company from Barcelona signed two agreements, one with Regenx Biosciences LLC (US) and the other with French non-profit organisation Généthon. Regenx granted Esteve a non-exclusive, worldwide license to its viral vector NAV rAAV9 to treat MPSIIIA, which is caused by a mutation in N-sulfo­ glucosamine sulfohydrolase (SGSH), the gene encoding sulfamidase. Once inside the cells, the gene-containing vectors express the enzyme stably, hence compensating for its absence. Regenx will receive an

Sanfilippo syndrome is associated with unibrow (synophrys).

Cell Therapy

Minority Investment

Allied GvHD forces Milking the deal Cell therapy developer Cell2B from Cantanhede has entered an R&D collaboration for the development of the company’s lead product Immunesafe with the Portuguese Institute of Oncology (Porto).

The clinical centre will collaborate in the pharmaceutical development, including supplying bone marrow samples, and will also join the clinical trial network. The cell product targets acute steroid­-refractory Graft-versus-Host Disease (GvHD), a major complication of bone marrow transplantation. Immunesafe is in early clinical development in this indication.

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Ferrer S.A. plans to acquire an 18% stake in compatriot Venter Pharma S.L. for a3.5m, beginning with an initial a1.3m in cash. Approved by the boards of both companies, the deal includes the acquisition of existing shares from current shareholders, as well as a new equity offering. Ferrer will also nominate a director to Venter’s board.

The companies are working together in the field of lactose intolerance. Venter’s diagnostic LacTest is marketed by Ferrer in Spain and Germany, and the company owns the commercialisation rights in the rest of Europe and Latin America.

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02.04.2014 16:32:17 Uhr

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Southern europe Annual Report

News

Italian biotech hits the skids

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The new annual report on the Italian bio­tech sector was made public on the occasion of this year’s BioSpring Europe.

“Bio­technology in Italy 2014” kept track of the 422 life sciences firms active in the country in a field that generated more than a7bn in sales. According to the study, Italian R&D investments amounted to a0.5bn last year. The data was compiled by lobby group Assobiotec and analysts from EY Farmindustria in collaboration with ICE – the

agency responsible for the internationalisation of Italian companies and their promotion abroad. At the presentation, Assobiotec president Alessandro Sidoli commented: “After several years of growth at a fast pace, the biotechnology sector in Italy this year shows clear signs of difficulty. This is due to the chronic lack of measures to support R&D and protect innovative products.”

Sidoli also denounced the lack of an adequate tax credit on R&D expenditure. B

Bioeconomy

Biochemical Q.E.D. ticular has been at the focus of several European industry efforts so far. The research project is being sponsored by the EU within FP7, and will run for four years. It officially kicked off on 1 January 2014, and includes partners such as the Fraunhofer Institut, Nova Institut, Cargill, Lubrizol, Rina, TNO, Miplast, Patentopolis and Mater-Biotech.

Italian biochemicals company Novamont SpA, a long-time producer of biodegradable materials, is coordinating the efforts. “This project is of strategic relevance for Novamont’s research,” said Luigi Capuzzi, the company’s research director. “It will allow us to consolidate our partnerships with important stakeholders within the bioeconomy on an international level.”

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© Novamont

A major focus of the EU’s bioeconomy strategy is the readjustment of industrial production towards a more sustainable use of resources. A renewable resources initiative is now aiming to find ways to scale up the production of certain chemical building blocks. To achieve this, ten partners from six European countries – Italy, Germany, France, the Netherlands, Croatia and Spain – have launched BIO-QED (quod erat demonstrandum). The name says it all. The partners want to prove that demonstrationscale production of bio-based bulk chemicals, at reduced costs and with improved sustainability, is not magic. The focus is on the chemical building blocks 1,4 butanediol (BDO) and itaconic acid (IA). BDO in par-

Leukemia therapy lift-off

Milan – Molmed S.p.A. has submitted a Marketing Authorisation Application (MAA) seeking conditional approval of TK as adjunctive treatment for high-risk leukemia patients undergoing hematopoietic stem cell transplantation (HSCT). The cellbased gene therapy comprises donor lymphocytes engineered to express the human herpes simplex virus thymidine kinase suicide gene.

With TK application patients undergoing HSCT from a mismatched donor do not need post-transplant immuno­suppression.

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R&D incentive

Madrid – Spain’s research advancement agency CDTI has revamped its promotion scheme. From 1 February onwards, companies can receive non-reimbursable payments for their R&D efforts. Depending on the size of the company and type of project, this add-on can amount to between 5%-25% of the total sum.

Additionally, the repayment periods for “Línea Directa de Innovación” projects have been prolonged.

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Step into the clinic

Barcelona – Biopharma company Ability Pharmaceuticals SL announced it has received approval from the Spanish Medicines Agency to initiate the company’s first clinical trial, a Phase I/Ib study of the novel, orally-administered anticancer drug ABTL0812 in patients with lung and pancreatic cancer. This first-in-man study with the dual mTORC1/C2 inhibitor will be conducted at the Hospital Clínic i Provincial de Barcelona.

Preparing compost at Novamont SpA

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03.04.2014 14:44:11 Uhr

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E a stern europe Vaccine Research

Staying one step ahead of the flu

New target for antidepressants A team of European researchers has identified a new molecular pathway implicated in depression. Authored

by Gabriella Juhász from Semmelweis University in Budapest, the results were published in PNAS in late March (doi: 10.1073/ pnas.1403649111). The research focused on the brain peptide galanin, a small protein that was originally identified in neurons, which release it alongside the classical transmitter noradrenaline. Both molecules have long been linked to pain, stress, and depression. Examining a cohort of 2,361 subjects from Manchester (UK) and Budapest, Juhász and her colleagues from the UK and Sweden discovered that the genetic makeup of the galanin pathway modifies the stress response in a statistically reliable manner.

“We show that some versions of the galanin gene protect against the risk of depression and anxiety,” she said. “But only in people who have experienced early life neglect or trauma, or recent adverse events.” The three genes coding for galanin receptors also apparently influence the risk of depression.

The findings provide a strong argument for the development of drugs that modify the galanin network. A new class of anti­depressant drugs is badly needed, since almost all commonly-prescribed antidepressants act on serotonin – and are often not very effective.

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© Josef Muellek / fotolia

Neuroscience

Polish biologist Marta Łuksza – who works in New York – and Cologne-based German physicist Michael Lässig have jointly developed a computer programme that predicts which strains of the flu virus could dominate the upcoming season. As reported in Nature in late Fabruary (doi: 10.1038/ nature13087), their method gives developers more time for the production of suitable vaccines. The modelling approach relies on the Darwinian principle of “survival of the fittest”. Accordingly, only certain mutations are adapted to enable a sufficiently high influenza virus replication rate in hosts.

“Whenever a person gets the flu, they be-

Will flu vaccinations soon be more precise?

come immune as an individual against this strain for the rest of their lives,” Lässig says.

That would mean that every strain of the flu virus could infect each person only once. Viruses therefore tend to mutate rapidly. Successful mutants, however, must also be conservative in the sense that they have to retain certain important functions. In order to identify the likely successful mutations, the researchers examined nearly 4,000 codes for haemagglutinin gene segments of influenza pathogens from the years 1968-2012. These membrane protein mediates define the binding of virus and host cell. Łuksza and Lässig then compared this information with the incidence of influenza infections. In a simulation, they forecast the annual flu strains for the years from 1994 onwards. A comparison later with the dominant subtypes that actually occurred revealed that the prediction was quite accurate. The method could thus enable a new, systematic selection of vaccine strains. Whether this also leads to improved vaccines, however, remains to be seen. Up until now, experts at the World Health Organization have recommended three to four flu strains that should be targeted by vaccines each season.

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Manufacturing

Budapest lab changes hands Two US companies have agreed on a deal centered around a production laboratory in Budapest. According to a press release from mid-March, Codexis Inc. has sold its facility to Intrexon Corp. for US$1.5m. Codexis is a developer of biocatalysts for the pharmaceutical and fine chemical industries. The strategic move is thought to be part of its biofuels business phase-down. Intrexon, on the other hand, said the deal will expand its strain and protein development capabilities, and strengthen its fermentation process optimisation. Lab staff will not be made redundant, but no IP or Codexis ma-

terials were included in the deal. The lab is to be incorporated into Intrexon’s Industrial Products Division (IPD). It hopes the integration will allow it to better serve current collaborators and attract new opportunities for active pharmaceutical ingredients and industrial and consumer product collaborations. “The expansion affords Intrexon greater flexibility in existing collaborations, including those with Amneal, Johnson & Johnson, and Oragenics,” said Robert F. Walsh, President of Intrexon’s IPD. “It positions the company to meet demand from international collaborators.”

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02.04.2014 16:33:33 Uhr

European Biotechnology News Science & Industry

April 2014

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03.04.2014 14:45:38 Uhr

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BiG Data & IP Intro

Big Data & smart data There has been a huge rise in data collection related to medicine in the ten years since the Human Genome Project was declared finished. Big datasets that link electronic medical records with patient-specific diagnostic data or disease-associated genetic risk factors (biomarkers) are now promising to improve the efficacy of patient pre-selection for clinical trails and therapy. But the growing flood of information in the life sciences is also presenting huge challenges when it comes to secured storage, processing, data visualisation and analysis. In clinical medicine, especially oncology, “Big Data” is expected to play an increasingly important role in identifying causality of symptoms, predicting hazards of disease incidence or reoccurence, or in improving quality of care. But is the investment in projects like the “Big Data to Knowledge” (BD2K) initiative, the HANA Oncolyzer project or the free-access digital cancer treatment library “Eviti” worth the money? Or is the hype surrounding Big Data masking a field with big promise, but little real current value?

issued in March by the IMS Institute for Healthcare Informatics. “Riding the Information Technology Wave in Life Sciences: Priorities, Pitfalls and Promise” showed that cloud-based business intelligence applications and storage in non-relational parallel-processing databases, embedded analytics and systems integration has the potential to drive transformational change over the next three years – both in overall healthcare system efficiency and the efficacy of treatments. The study’s authors believe that the availability and adoption of secure, healthcare-specific tools and services are key to accelerating opportunity and deriving greater value from new, expanded sources of health information to optimise patient outcomes. Cost pressure is also driving solutions that promise to improve the efficiency of pharmaceutical development. According to IMS Health, Big Pharma will need to reduce combined operating costs by US$36bn annually through 2017 to maintain operating margins and current levels of R&D activities.

© IBM

Challenges and bottlenecks

IBM’s Watson Foundation presenting new visualisation tools for Big Data at CEBIT.

At BIOCOM’s recent 7th IP conference “Big Data – Big Drugs” in Berlin, Sachin Soni – the Director of Equity Research (Life Sciences) at Kempen & Co Merchant Bank – made it clear that integrating Big Data could have a massive impact on healthcare. He showed data suggesting that improvements in pharma­ceutical R&D productivity alone could create value of US$40-70bn annually. Evidence-based care could add another US$90-110bn – and that seems to be just the tip of the iceberg. Linking existing information on drug profiles with a growing body of omics data and patient records can for example identify new applications for drugs

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that have already been approved. Integrating sequencing and outcome data can help determine new drug targets and compounds, according to Dr. Michele Wales from InHouse Patent Counsel LLC. That’s shown by drugs such as Benlysta, Raxibacumab, Albiglutide or Darapladib, which were derived from Human Genome Sciences’ sequence databases. Other speakers at the conference saw further potential in areas like identifying drug responders, or finding reasons for noncompliance of therapies.

Industry is already aggressively embracing new Big Data approaches, according to a survey of 70 life sciences organisations

As competition among life sciences companies intensifies – and the mix of new medicines skews toward those with relatively small target patient populations – analytic systems that help bring medicines to the right patients and their physicians have become essential. The implementation of decision support algorithms can accelerate the improvement of health outcomes, while also bringing more efficiency to the entire health system. Nearly 60% of survey respondents additionally rated patient apps as extremely or very important to addressing commercial challenges, while 69% similarly rated investments in physician apps.

However, Big Data itself can be highly diverse and uninformative without preprocessing. Current limitations include selection bias, sample size, missing values, accuracy, completeness and the nature of reporting resources. Although a few first successes are surfacing, many challenges remain. “Data protection is key,” says Andrew Litt from Dell. “Confidence in genome research cannot undermined by inadequate data protection.”

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Euro|Biotech|News

Sponsored Article In Focus

Integrating Big Data The analysis and interpretation of massive amounts of biological data poses a significant bottleneck for researchers and clinicians seeking to understand and diagnose diseases. Prof. Dr. Reinhard Büttner explains how his laboratory addressed these challenges, and where he sees the value of pre-tailored commercial data processing systems. ?

Prof. Büttner, your lab was one of the first in Europe to implement next-generation sequencing (NGS) into routine clinical diagnostics. How do you use this technology today? ! Buettner: We use NGS in cancer diagnostics and initially started with lung cancer. Today, we have established additional tests for melanoma, chronic lymphomatic leukemia (CLL) and gastro­ intestinal (GI) tumors. However, with about 3,500 cases a year, lung cancer is still the most frequently performed test in our lab followed

by GI, melanoma and CLL. In total, we perform almost 5,000 NGS-based tests annually. ?

How long did it take to set it up? ! Buettner: The initial set-up of the entire workflow to its routine clinical implementation took us about a year. This is because we needed some time to gain experience with the NGS technology and validate our approach. In fact, we went through a very excessive validation phase to ensure the accuracy of our results. But still, we’re not there where we want to be. I think of this as a process during which we’re continuously implementing new technologies and also new gene sets.

It’s really about continuous development, and the current system we use is already our second version lung cancer test.

34-35_EBN_Advert-Qiagen_tg.indd 34

?

Are you looking into the entire genome, or a particular set of genes? ! Buettner: We’re working with gene panels that cover a set of around 20-40 genes, depending on the type of cancer. As of today, I don’t think that it makes sense to cover the exome or even entire genome in routine clinical diagnostics. This is because you have only a limited number of potential drugs at hand linked to biomarkers. In addition, it is still quite challenging in terms of sequencing costs and the amount of data you generate. ?

How much data do you have to process for one patient? ! Buettner: It’s usually one gigabyte of data per test.

This is because we sequence every gene in our panel at a very high coverage, in our case 5,000-fold. ?

This must be a significant challenge for your workflow? ! Buettner: I think that we have adapted quite well.

When we initially set-up the workflow, we developed our own proprietary data­ analysis pipeline, which first helps us to

Reinhard Buettner studied medicine at the Universities of Mainz, Munich, London, and Cologne, and received his MD at the Pettenkofer Institute for Virology in Munich. After postdoctoral fellowships at the Gene Center Munich and the MD Anderson Cancer Center in Houston, he became a staff pathologist at the University of Regensburg. From 1999–2001 he was a full Professor for Pathology at the RWTH Aachen. In 2001, Buettner was appointed Professor and Chairman for Pathology at the University Hospital Bonn, and since 2011 he has been a Professor and Chairman for Pathology at the University Hospital of Cologne’s Center for Integrated Oncology.

automatically filter the data to identify relevant mutations based upon different algorithms reflecting our quality standards – for instance, the quality of data or coverage depth. The subsequent clinical interpretation is still manual work. This is the most time-consuming part of the entire analysis process – about a day if you deal with 3-5 mutations – but we found the automatic algorithms we tried to implement weren’t reliable enough. ?

Is the entire pipeline based upon a proprietary system? ! Buettner: We ended up with a mix of analytical tools that are freely available, such as the Integrative Genomics Viewer and own tools developed in collaboration with our IT department.

04.04.2014 12:55:26 Uhr

Nº 4 | Volume 13 | 2014

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35

Sponsored Article ?

Was it a major cost factor for your lab, or is the investment in bioinformatics rather negligible? ! Buettner: It is a significant cost factor. For instance, we had to exchange all the switches and the connections to the existing IT system because the regular connection was too slow to transfer the amount of data we’re working with. So we had to invest in hardware.

But you also have to invest in people who generate your algorithms to make sense out of the data. However, I believe these are the typical starting costs associated with getting into NGS. ?

Do you think this approach would also be the way to go for other labs? ! Buettner: We have a very large discovery unit around lung cancer, so it made sense for us to invest in bioinformatics infrastructure and personnel. However, there are also vendors out there like QIAGEN that work on pre-tailored commercial bioinformatics solutions, and if you’re a pure diagnostic lab that wants to do NGS-based tests, I think it’s probably sounder to buy such a solution. In fact, I believe this is ideal for larger diagnostic labs that are focused on routine diagnostics and simply want to have a streamlined analysis pipeline with state-of-the-art technology. ?

But some argue that relying too much on integrated systems might harm professional standards … ! Buettner: There is little difference to other areas of modern medicine. Just think of an intensive care unit – you have no chance to control the technical credibility of all the para­ meters provided by various instruments. We are dependent on technology. This does not mean that you shouldn’t perform regular controls of your workflow. But the idea that someone can visually control the plausibility of gigabytes of data is simply not practicable; this would lead to many more er-

34-35_EBN_Advert-Qiagen_tg.indd 35

rors. So I rather see this as a purely psychological factor. ?

So where do you see the role of the pathologist in this setting? ! Buettner: Applied to cancer, I believe that you need a solution that delivers to the pathologist comprehensive and credible information about all validated mutations and genomic alternations in the sample. But then, the pathologist needs to integrate this information into a comprehensive diagnosis – a conclusive report – which connects the genomic information with data from microscopy and immunochemistry as well as other clinical information about the patient and the particular disease. This is something that you shouldn’t do automatically, in my view. ?

Speaking of integrating information, how do you handle the exchange with other institutions or external databases? ! Buettner: Other institutions aren’t directly connected to our genome data. The external institutions we work with – around 100 hospitals and pathologists in Germany – receive a standardized report containing a table that summarizes which genes have been sequenced and at which coverage, what is the allele frequency and the potential functionality of the mutation, and what would be the potential clinical interpretation in terms of treatment or inclusion in a study. ?

How do you ensure that your findings and recommendations are up-to-date? ! Buettner: This is a very critical point. When it comes to the clinical interpretation of mutations, we refer to guidelines that make statements about which genes should be tested for in what tumor entities. However, guidelines don’t include lists of drivable mutations, since this knowledge is continuously expanding at a very rapid pace. This can be quite challenging if you find novel or rare

mutations. You might have analyzed the tumor according to guidelines, but still don’t know what to tell the patient. So I think that over time, we need a database that connects even novel and rare mutations to clinical outcomes. This brings us to clinical studies. Today, we have a good overview of all studies performed here at the University of Cologne, and we know precisely the inclusion criteria and can direct patients to the studies if necessary. However, it is much more challenging when you start looking for external studies, especially when new compounds are in an early stage of development. This can be extremely time­-consuming. So I think that in both cases there is room for improvement. ?

Which other challenges do you see in the application of personalized approaches like these? ! Buettner: We are generating tremendous numbers of new biomarkers for all kinds of different cancers. These biomarkers literally flood clinical practice, but I’m still observing some skepticism towards targeted treatment approaches – in Germany and elsewhere. We need a broader understanding that we’re truly entering into a completely new age of medicine – an age of rational oncology that examines the molecular alterations driving a tumor and matches it with valid therapeutic concepts. However, cases in which healthcare professionals ask for a full mutation scan only to decide to stick with standard chemotherapy are still commonplace. This is the wrong path, and one which puts our entire healthcare system at risk. We need to invest in modern diagnostic technologies to determine which patients can benefit from particular treatments and act accordingly. If you try to save money in diagnostics, you will lose this money through imprecise therapies. There is still a lot of work that needs to be done here. ii

More lnformation QIAGEN QIAGEN-Str. 1 40724 Hilden Germany www.ingenuity.com; www.clcbio.com

04.04.2014 12:55:36 Uhr

European Biotechnology

Nº 4 | Volume 13 | 2014

Big Data & IP

Net work

Companion Diagnostics

IP considerations associated with CDx Ramin Ronny Amirsehhi, Amirsehhi Intellectual Property Law; Martinsried

Many pharmaceutical companies are considering a biomarker strategy for their drugs as advances in diagnostic technologies, the growth of biosimilars, a rising number of expiring patents, and concerns about profit margins drive them to adjust their approach.

Join the European Biotechnology Network!

The European Biotechnology Network is dedicated to facilitating co-operation between professionals in biotechnology and the life sciences all over Europe. This non-profit organisation brings research groups, universities, SMEs, large companies and indeed all actors in biotechnology together to build and deliver partnerships.

Do you want to know more about the advantages of a (free) membership?

Just have a look at our website: www.european-biotechnology.net

European Biotechnology Network Avenue de Tervueren 13 1040 Brusseles, Belgium Tel: +32 2 733 72 37 Fax +32 2 64 92 989 info@european-biotechnology.net www.european-biotechnology.net

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In the race to discover and develop com­ panion diagnostics successfully, many pharmaceutical companies are partner­ ing with biotech companies that are ex­ pert in the appropriate diagnostic or tech­ nology. Working with a diagnostic part­ ner to develop a companion diagnostic, which ultimately also requires obtaining market authorisation, complicates the al­ ready complex drug development and ap­ proval process. An intellectual property (IP) strategy is part of this complex proc­ ess, and plays a key role in the develop­ ment and commercialisation of compan­ ion diagnostics.

IP strategies for companion diagnos­ tics include a deep understanding of type of discovery, business objectives, patent eligibility and infringement considera­ tions. While it is beyond the scope of this article to examine all IP considerations for all major jurisdictions, it is important to briefly discuss how patent law is evolv­ ing in the US and Europe. In this first in­ stallment of a series of articles, we look at types of discovery and US patent eligi­ bility issues.

Type of discovery The strength of patent protection is most often correlated with the type of discov­ ery. Patent claims directed at compounds are the strongest type of protection. Such

protection could be used to exclude oth­ ers from using the compound or novel bio­ marker in any type of diagnostic for any type of drug.

In most cases, biomarkers are known proteins. A discovery involves their corre­ lation with a particular disease and drug that is under study. This type of discov­ ery may result in method-of-treatment claims, which can be very valuable – es­ pecially if such method claims read on a drug and/or diagnostic label.

Method-of-treatment claims need to be drafted with both the drug label and diagnostic label in mind. The labels can be in general terms or include a specif­ ic assay. A claim directed at a specific di­ agnostic assay or technology would in­ clude limitations associated with how the presence of the biomarker is determined.

Such claims can be vital in the long run, as they may extend the exclusivity of the drug against generics and biosimilars, since to meet FDA requirements, any ge­ neric or biosimilar equivalent would also be required to include reference to the di­ agnostic assay.

The discovery and patenting of new plat­ form technologies can also be valuable to a diagnostic company. It is important to con­ sider in advance how a potential competi­ tor may attempt to avoid patent infringe­ ment while at the same time demonstrat­ ing the equivalence of its device to a legal­

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Nº 4 | Volume 13 | 2014

Big Data & IP ly marketed device by submitting a 510(k) premarket submission to the FDA. Therefore, it is important to consider other feasible methods or techniques for measuring the bio­marker and include these in the specification and/or claims.

Discoveries that do not impact labels are less valuable, and are usually easier to get around. These include, for example, the discovery of specific assay reagents.

Patent eligibility By now, most people in the life science community have become aware of the US Supreme Court’s decision in Mayo Collaborative Services vs. Prometheus Laboratories, Inc. 132 S.Ct. 1289, (2012 –“Mayo” decision), as well as in Association for Molecular Pathology et al. vs.

Myriad Genetics, et al., 133 S.Ct. 2107, (2013 – “Myriad” decision). It is important to highlight the difference between these two rulings.

The Mayo decision is entirely independent of the “law of nature“; it is focused on process claims. The Myriad decision did not review process claims, and focused on the genes. There are numerous publications analysing the two decisions. This article will therefore propose strategies for obtaining claims that relate to diagnostic methods or biomarkers. It is important to remember that the following approach-

es are completely applicable only in the US. For other jurisdictions – such as Europe – different scope of claims may be obtained. When drafting applications, applicants should therefore consider these differences and include them in the specification and claims.

Congress and Exhibition

MEDIZIN INNOVATIV MedTech Pharma 2014

Proposed solutions According to the Mayo decision, correlations between a biomarker and efficacy are a natural law, and are therefore not patentable. The possible approaches in light of this can be claims 1) directed at a method of diagnosing a disease by detecting a novel biomarker, 2) use of a specific reagent (as mentioned above, these types of claims can be easy to circumvent for competitors and should be considered carefully), 3) adding a treatment step (the treatment step can be a particular drug or therapy), or 4) detecting a combination of biomarkers.

Genomic DNA is considered a product of nature, and based on the Myriad decision is therefore not patentable. Possible strategies for bypassing this roadblock can be claims directed at cDNA, nonnaturally occurring DNA (such as mutagenized or chemically modified DNA), synthetic RNA or synthetic proteins (it is important to avoid using the term “isolated”), as well as their respective probes and primers. 

Imaging Smart Medical Systems Digital Prevention and Care Early Detection & Prediction Big Data Management & Analytics Regulatory Affairs Market Access Strategies Supplier Manufacturer Relations

 1000 Participants  120 Exhibitors  65 high level lectures in three parallel sessions  International Delegations  Job Wall

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2 - 3 July 2014 NCC Ost NürnbergMesse

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www.medtech-pharma.de

Supported by:

02.04.2014 16:40:25 Uhr

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Euro|Biotech|News

BIG DATA & IP ONCOLOGY

Leveraging Big Data for new medicines Manuela Müller-Gerndt, Healthcare & Pharma/Life Sciences, IBM Germany; Dr. Wolfgang Hildesheim, Watson Group, IBM Europe; Fatema Maher, IBM Germany

Almost US$200bn are spent annually on R&D in science-driven sectors such as healthcare, life sciences, consumer products or chemicals. An estimated 60% of pharmaceutical R&D investments, however, are spent on products that will never reach the market. This article looks at how best practices for R&D workspaces could be applied to ensure successful drug development. It all starts with a step-by-step approach towards Big Data, for example based on IBM’s Watson Foundations.

Cancer is the second most common cause of death in the developed world. In fact, one in three people will be diagnosed with carcinoma at some stage in their life. To tackle this global problem, pharma firms are investing billions of dollars every year in the development of cancer treatments. Even though R&D costs for drug development are exploding, there are currently about 1,900 cancer medicines in the pipeline.

At the same time, the volume, velocity, variety and veracity of data is rising exponentially in medicine. We know today that cancer is often related to genetic factors.

To sequence insightful DNA and achieve better treatment options, billions of samples need to be compared. That is generating huge sets of data, so that drug development for oncology has increasingly become a data-driven science bringing together physicians, pharmacologists, molecular biologists, computer scientists and mathematicians to solve complex problems that none of these disciplines could solve individually.

The Big Data challenge This complexity is also reflected in the related data itself, which are often referred to as the characteristics of “Big Data”:

38-39_EBSIN_4_14_Special_IBM_tg.indd 38

– Volume of data: Research data is spread across huge databases in a number of different areas, including patents, compounds, journals, biomarkers, structure activity relationships, medical records and genomics. For example, around one million human genomes were sequenced in 2013, and we expect around five million human genomes to be sequenced by the end of 2014 worldwide with around 15 million new cancer patients each year worldwide. Over 26 million unique molecules are available for new drug development from 400+ different sources in the ChemSpider chemical database.

– Velocity of data: Real-time analysis of device data, images and alerts will change the role of monitoring devices in healthcare outcomes and patient well-being.

Bedside monitoring devices today capture more than 1,000 vital signs per second.

– Variety of data: Around 80% of all data today is unstructured, and this percentage will increase dramatically over time. As health and personalised medicine make advances in the population, even more data inputs can be expected from medical records, notes and dictation, public health reports, scientific papers, social media and the Internet.

– Veracity: How trustworthy is the data?

The ambition to deal with these four ’V’s’ can be understood as the definition of the Big Data trend, which we see in oncology, but also in healthcare and nearly all other industries.

A business need in pharma R&D Oncology R&D researchers are challenged by huge amounts of data from different sources in heterogeneous formats that they have to digest and turn into new products more quickly. Given the sensitivity of health records and medical information, as well as the need to protect intellectual property, one of the most compelling issues is security.

More and more R&D employees, administrators, managers and senior executives are asking for role-specific workplaces with instant access and proactive delivery of changes to many kinds of information from many internal systems – both within their own organisation and external databases/ online libraries. They want access to patient information, physician opinions, clinical data, medical research studies, product and market information, and regulatory & compliance standards.

Getting up to speed To stay at the forefront, R&D departments in oncology need to tackle the Big Data challenge and prepare to take advantage of the upcoming era of Cognitive Computing. To get up to speed, typically, they go through three phases: – The first phase starts with Information Exploration & Discovery to enable complete visibility into new and historical internal data sets, as well as external research results and publications.

– The second phase concentrates on Content Analytics by applying text mining, pattern recognition and predictive analytics (e.g. for target discovery and validation) in order to find correlations between genes and diseases.

– Phase three introduces Cognitive Computing, adding natural interaction with oncologists and systems that learn through interactions, deliver evidence-based medical responses and drive better outcomes in the near future.

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BIG DATA & IP Since most oncology R&D departments are currently in the first phase, or even a pre-stage of it, this article introduces how organisations can get started with information exploration. Usually the first step is selecting a defined pilot, working with an innovative group and tying together a few internal and external information sources.

State-of-the-art data exploration Modern R&D workplaces ideally include intelligent information exploration that virtually integrates multiple sources into one single access point, provides personalised content, uses best-of-breed search and unique, automatic clustering and categorisation capabilities, and allows users to browse all kinds of historical research information within the organisation and beyond.

Dynamic clustering helps users to gain new insights that have not been discovered before. Having the ability to tag, bookmark and comment on documents helps foster collaboration and improve treatment outcomes. Proactive alerts and “push intelligence” let people know when new or updated information is available.

The rapid application of building capabilities helps to quickly present customised, role-specific views to the end user.

This kind of information exploration is available today, allowing organisations

to maximise the value of their intellectual property, reduce their product development cycle and significantly decrease R&D costs.

One real-life example A leading pharmaceutical company that has been developing innovative medicines for decades needed to focus on competitiveness. That involved transforming information and data sharing capabilities to support faster time to market and new product introductions. R&D needed instant access to patient and research information across the company’s intranet, to many different applications and file systems, as well as information from several external toxicity databases and subscription sources. The company’s executives created initiatives to increase data transparency and productivity throughout the enterprise, worldwide.

Data Exploration Initiative The Watson Data Explorer software was deployed within 10 weeks and gave 30,000 employees worldwide an authorised access to intranet portal-like applications. These portals were built rapidly to cover real-time internal and external information, including networked file systems, the enterprise content management system, files in Microsoft SharePoint, an employee directory

Unlock the value of information when users need it the most

Watson Explorer

gation Discovery & navigation Data access & integration Clustering rization • C lustering & categorization Providing unified, real-time access • Index structured & unstructured data—in place ence • Contextual intelligence and fusion of big data unlocks • Support existing security ications • Easy-to-deploy applications greater insight and ROI • Federate to external sources oday’s big data • All at the scale required for today’s big data • Leverage MDM, governance, and taxonomies challenges

Create LL unified view of ALL Create unified view of ALL Improve customer service & me information for real-time information for real-time reduce call times monitoring monitoring

n risk nce

&

Increase productivity & leverage past work increasing speed to market

Analyze customer data to unlock true customer value

Identify areas of information risk & ensure data compliance

Watson Explorer – a first step towards intelligent 360º view workplaces including structured and unstructured data.

38-39_EBSIN_4_14_Special_IBM_tg.indd 39

and external internet pages and subscription sources. The solution preserved existing security parameters, so that employees could only access content they were authorised to view. Security is supported at group, user, document and the even more granular field levels.

Watson Explorer has enabled touch points throughout the drug discovery process, from initial research to clinical trials. R&D staff obtained an easy navigation tool and a way to filter data quickly. Scientists can now retrieve an overview, drill down into specific topics and discover content that might remain uncovered. They can also scan multiple synonyms for medical terms and phrases, while critical R&D employees receive alerts of changes in support-of-compliance efforts. Collaboration capabilities allow users to identify relevant content to colleagues by commenting and tagging results. By globally leveraging past research around the history of compounds for formulation and noted effects, R&D was able to drastically reduce duplicate efforts. In addition, sales representatives became more productive by gaining access to the newest policies, external news, marketing collateral and a doctor’s latest purchases.

Finally, the company knowledge base supported hiring and retraining transitioning employees. A knowledge-sharing and collaboration culture was created, as well as subject-matter experts, who maximise one of the company’s most valuable assets – their data.

Watson Data Explorer has already delivered measurable business value to many pharma companies. In the example described above, the efficiency of R&D was improved by 90%. Cutting search by 50% saved the company millions of dollars in the first year alone. In addition, sales rose by 4.1%, training costs were reduced by 10% and new staffing requirements decreased by 1.2%, saving US$13.4m per year.  References [1] Jaruzelski, B., Loehr, J., Holman, R.: THE GLOBAL INNOVATION 1000: Making Ideas Work. B. Company,Editor. 2012.

[2] IBM Institute for Business Value analysis.

[3] Cancer.org, DKFZ 2013

02.04.2014 16:42:09 Uhr

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BIG DATA & IP Interview

Big Data can’t make gold from bad data Life sciences companies are growing increasingly interested in Big Data as they seek to combine information about disease pathways with patient data and critical inclusion criteria for clinical trials or electronic medical records. EuroBiotechNews spoke with SAP’s Peter Langkafel, the software giant’s General Manager Public Sector/Healthcare MEE, about the impact of Big Data and its current limitations in life sciences applications.

Euro|BioTech|News

?

Dr Langkafel, is the idea of Big Data being over­hyped?

LANGKAFEL:

!

© mhristov - Fotolia.com

“Big Data” in healthcare is an El Dorado for some people, but it is still a very diffuse term. There is a huge potential in some areas – such as better integrating research data and clinical day-to-day data. But Big Data can also mean “going beyond borders”, which means the integration of ambulatory and in-house care and the analysis of what is happening there. This is at times a complex topic due to organisational and legal issues. But there’s no doubt there is huge potential.

That said, there is hype as well. For some people, Big Data is pretty much only associated with personalised medicine – with genomic analysis, which “promises” cures for all kinds of diseases in the

future. For me, the so-called “potential” here is hype.

Euro|BioTech|News

?

In which areas does SAP see the greatest market potential for Big Data solutions within the healthcare industry?

LANGKAFEL:

!

We’re involved in projects with providers like the Charité (Europe´s biggest university clinic), healthcare providers like the AOK (Germany´s biggest health insurer), research organisations like the cancer research centres in Heidelberg and Stanford, and Health Maintenance Organisations like Kaiser Permanente in the US. I would describe three phases for Big Data against this backdrop. First, it is about understanding and maybe better visualising data. Then you have to align and match data sources – which has not been possible to this extent before. The third phase is to really create new data or add some data outside of the traditional ecosystem. And I’d like to point out that just a few years ago we still had technical difficulties, but today technology hurdles like memory represent unleashed potential. Technology is no longer necessarily the obstacle.

Euro|BioTech|News

?

What is Big Data about, and how can data integration help improve patient recruitment for clinical trials? Or the identifica-

40-41_EBSIN3_14_Interview_SAP_tg.indd 40

Dr. med. Peter Langkafel is SAP AG’s General Manager Public Sector/Healthcare MEE (Middle and Eastern Europe) and the President of the Berlin/Brandenburg German Association of Medical Informatics (BVMI e.V.) He has over 20 years of experience in healthcare and information technology.

With a PhD in medicine, a degree in medical informatics, and an executive MBA, Langkafel was a clinician and researcher himself for many years. He has written a long list of publications, and has appeared as a speaker at over 100 healthcare conferences.

tion of drug responders in areas like oncology, the identification of new uses for already approved drugs, establishing electronic medical records, etc.?

LANGKAFEL:

!

There are many existing cases that can illustrate this process. We’ve tried to understand what has happened in the past with traditional reporting. Currently we are on our way to understanding the situation in real-time. And Big Data technology will help us to predict and look into the future…so it’s not just about what has happened, but what is happening and what will happen.

Euro|BioTech|News

?

What tools are needed and used to manage inherently imprecise data types like that involving stratification biomarkers?

LANGKAFEL:

!

The golden rule of any business intelligence project is: garbage in — garbage out.

If you have bad data, you can’t turn it into gold dust through some miraculous process. We do provide tools for data cleansing

03.04.2014 14:49:20 Uhr

N –º 4 | Volume 13 | 2014

or master data management, but these are mainly technical tools that can’t “understand” the content of imprecise data. There is a lot of effort being put into trying to get unstructured data (like a doctor’s discharge letter) into the system. Most of these projects promise a lot, but only reach accuracy levels of 60% to 80%. Something is missing, but it’s not clear what. So we can’t use this approach. My recommendation is always to try to feed structured data into the system!

Euro|BioTech|News

»Larger is good.

Smarter is better.«

?

What are current drivers, and where do you see limitations?

LANGKAFEL:

!

Technology is certainly a driver, as well as the growing volume of digital data in healthcare. We’ve started to view “data” as something very valuable – but we are in an early phase of really understanding its power. Data security is for me not a limitation, but an absolute prerequisite. We should be asking our data security officers as well about who will protect our data from NOT being used. Our main limitations are the silos in the organisation and a lack of statistical understanding. Or let me put it this way: Big Data gives a lot of answers, but we have to ask the right questions first.

Euro|BioTech|News

?

What has been already achieved by SAP, and what are your plans for the future?

LANGKAFEL:

!

SAP is the frontrunner in memory technology: With SAP HANA, we have an extremely powerful tool in our portfolio, which is now even available in a cloud offering. The SAP Healthcare platform brings together new ways of using and integrating data.

We are delivering solutions in that area, and we are co-innovating with customers – like those I mentioned before – around the world.

Euro|BioTech|News

?

Data from academic institutions are often not compatible with the requirements of the biopharmaceutical industry when it comes to things like assay design, reproducibility or biomarker cut-off values. What kind of standardisation do you think is needed to support efficient translation into applications?

LANGKAFEL:

!

Any discussion about standardisation should ask the same open question: Why is there no standard? Who has interest in NOT having a standard? Maybe for economic reasons, to protect an area (or a market) or to protect fraud and abuse, or to protect AGAINST fraud and abuse? I am personally a big fan of the the British Medical Journal (BMI) Open Data Initiative. Everybody who has nothing to hide should allow others to have a look in – under a certain operation mode, of course (data security, IP, legal). Here we need new ways for academia and industry to collaborate.

F

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U.S. and EUropEan LifE SciEncE patEnt proSEcUtion

nEW

New office at the innovation and start-up center for Biotechnology in Martinsried: am Klopferspitz 19 | 82152 planegg/Martinsried | Germany

www.amirsehhi.com | ra@amirsehhi.com Tel.: +49 (0)89 4516 9990 | Mobile: +49 (0)176 64 63 33 81

07.04.2014 10:48:09 Uhr

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Per spec tives Eu funding

EuropaBio/EY

Early sepsis Dx

Improving incentives in the EU

A Brussels – Early diagnosis of sepsis is crucial to prevent the dramatic drop in blood pressure that occurs in the course of the socalled hyperdynamic response. An EU consortium led by Meez Islam from Britain’s Teesside University has now bagged a1.6m in FP7 money to try to establish a new method for detecting sepsis biomarkers that is 100 times more sensitive than existing technologies. Together with three institutes, Islam’s spin-out Anasyst and five other companies are aiming to establish Cavity Enhanced Absorption Spectrometry (EAS) as the gold standard for measuring sepsis biomarkers colorimetrically in bioassays. The work of the CE MicroArray consortium builds on a US patent – which involves the use of CEAS in microenvironments – as well as three additional patent filings that are part of a patent family for the CEAS technology licensed exclusively to Anasyst. A German-Spanish research team recently demonstrated that the bio­marker Adrenomedullin (AM) is strongly associated with severity of sepsis, vasopressor requirement and 28-day mortality (Critical Care, doi:10.1186/cc13731).

B

A Brussels – National governments in the EU have moved to encourage innovation through a web of tax incentives for R&D. However, the bi-annual report ”Biotechnology in Europe: The Tax, Finance and Regulatory Framework and Global Policy Comparison” says that plenty remains to be done to effectively transfer knowledge into products and economic growth. Jointly tabled by EuropaBio and EY in mid-March in Brussels, the study profiles biotech­-relevant tax, financial and regulatory incentives in 17

European Commission

Tighter rules A Brussels – The European Commission (EC) is closing a regulatory loophole in offlabel prescription. As a first step, the EC will launch a study to identify potential fields of legal action. The move comes on the heels of a request from Italian drug agency AIFA, which wants to clarify if and under what conditions the off-label use of Avastin instead of Lucentis might be allowed as a treatment for age-related macular degeneration (see p. 25). Unlike AIFA, the French regulatory agency ANSM wants to greenlight off-label use of Avastin. Meanwhile, a German court ruled that providing prefilled syringes with Avastin requires separate market authorisation.

B

42_EBSIN4_14_IPOroundup_ml.indd 42

Member States with thriving biotech sectors. It also identifies substantial variations in regulatory policy when it comes to startup financing, as well as overall attraction for entrepreneurs and managers, which affect an SME’s ability to transform innovation into manufactured products. ”National governments, together with the EU, need to ensure that the right policies are in place to make funding more accessible to SMEs,” concludes EY’s Philip Robinson. The report is available at www.europabio.org.

D

Start-up considerations across selected EU Member States Country

Corporate tax rate

Use of Losses

R&D tax credit

A Austria

25%

Yes, cap at 75% of income

10% up to a cap of a1m

A Belgium

33%

Yes, but no carry back

Investment deduction: 14.5% of acquisition value/21.5% of depreciation amount, or R&D credit

A Denmark

24.5%, progressive reduction to 22% in 2016 and onwards

Restrictions on carry forward, 100% offset ≤ DKK7.5m ≤ 60%

100% deduction for qualifying expenditure, R&D refund of negative tax up to DKK5m

A France

33.3%

Limit a1m, 50% of taxable profits exceeding the limit

30%, up to a100m + 5% qualif. expenses > a100m

A Germany

15.825% + municipal (10%-18%)

Limit a1m, 60% of taxable profits exceeding the limit

No

A H ungary

19% and 10% on first a1.7m

Up to 50% of tax base

Double deduction for R&D expenses

A Ireland

12.5% and 25% (passive)

Full relief

25% (effective 37.5%)

A Italy

31.4%

Up to 80% of tax base

50% tax credit in increm. R&D, max.: a2.5m/year

A Luxembourg

29.22%

Yes

Yes

A Netherlands

19% and10% on first a200,000

Carry forward nine years, carry back one year

R&D tax credit 35% (start-ups: 50%) of a250,000 total wage cost for R&D, 14% for remainder; R&D deduction of 60% cost

A Norway

27%

Yes, carry forward indefinite

18% for SMEs, max. NOK8m

A Poland

19%

50% capped at 50% of loss

Innovation fund ­– 50% of eligible cost

A Portugal

23%

Max. 70% of taxable profits

32.5% on qualified expenses, a1.5/1.8m cap

A Spain

30% and 25% on first a120,202

For 18 years

General rate 25%, up to 42%

A Sweden

22%

Yes, carry forward indefinite

Expenses deductible

A Switzerland

12-24%

Carry forward for 7 years

Capitalised expenditure

A UK

23%

Carry forward full relief

225% deduction for SMEs, 130% for large companies

03.04.2014 14:54:38 Uhr

N –º 4 | Volume 13 | 2014

43

Euro|Biotech|News

biopeople EFSA

Prothena

New food safety top watchdog

mAB executive

A T he Eu ropea n Food Safet y Au­ thority’s Manage­ ment Boa rd h a s n o m i n a t e d D r.

Bernhard Url as EF­ SA’s next Executive Director. Follow­ ing the departure of former Director Bernhard Url Catherine GeslainLanéelle, Url has been EFSA’s acting Ex­ ecutive Director since the autumn of 2013.

A qualified veterinarian by training, he brings high-level management experi­ ence from public and private food-safety organisations to the role, most recently as Managing Director for the Austrian Agen­ cy for Health and Food Safety (AGES). Un­ til March 2012, Url served as a member of EFSA’s Management Board.

B

Novimmune

Moneymaker A Geneva-based anti­body develop­ er Novimmune SA has appointed Andrew Oakley as its new CFO. The fi­ nance expert has been the Chief Fi­ nancial Officer and Vice President of Andrew Oakley Actelion Pharma­ ceuticals U.S., Inc. since January of 2003.

Prior to that, he held executive positions in major multinational building material companies, and spent several years as an equity analyst with banks in Australia, the UK and the US. Oakley has been a mem­ ber of the Australian Institute of Chartered Accountants since 1987.

B

43_EBSIN4_14_Biopeople_tg.indd 43

A Irish antibody de­ veloper Prothena Corporation plc has named Tara Nickerson as its Chief Business Officer.

Previously she held the position of Head of Corporate and Business Develop­ Tara Nickerson ment at the com­ pany, where she was instrumental during Prothena’s demerger from Elan and Pro­ thena’s worldwide collaboration with Ro­ che to develop and commercialise antibod­ ies that target alpha-synuclein. Prior to Elan, Nickerson was a senior scientist at Celera Genomics (Axys Pharmaceuticals).

B

Merck

Biologic lead A Germany’s ­Merck KGaA (Darmstadt) has hired the former head of Boehring­ er Ingelheim’s biopharmaceuticals devel­ opment team to lead its biosimilar unit, which was set up in 2012. Prior to the stint at Boehringer, Simon Sturge was CEO of Dutch biotech OctoPlus NV, and he also acted as CEO of Vernalis plc (UK). In the past, Sturge has held several roles of in­ creasing seniority – from Business Devel­ opment, Marketing and Operations to CEO at Lonza Biologics, as well as at Celltech Bio­logics and at AstraZeneca. The native of Britain was award­ ed the UK Medi­ science CEO of the Year award in 2005.

St u rge succeeds Thierry Hulot, who created Merck’s bio­ similars unit and took over respon­ sibility for Global Manufacturing & Simon Sturge

Supply for the Merck Serono division at the end of 2013. Along with Sturge, Merck has also strengthened its team internally with ­Friederike Rotsch, who has now tak­ en over as chief legal advisor.

B

Roche

R&D translator A Roche’s pRED unit has a new head of on­ cology and translational research. William Pao, currently director at the HematologyOncology division and Personalised Can­ cer Medicine unit at Vanderbilt University Medical Center, will lead Roche’s discov­ ery, translational medicine and ear­ ly development of innovative oncolo­ gy medicines. Pao, who has focused on academic clini­ cal research in the past, is one of the world’s top oncolo­ William Pao gy experts.

B

Canbex

The doctor is in A Canbex Therapeu­ tics Ltd has hired Alberto Lledó as its new Chief Medi­ cal Officer (CMO).

Lledó will lead Can­ bex’s preparations in proof-of-concept trials for VSN16R, a small molecule Alberto Lledó for the treatment of spasticity in multiple sclerosis. Lledó is a Senior Consultant Neurologist at the Clíni­ ca Creu Blanca (Barcelona), and spent six­ teen years at Eli Lilly. He will take over as CMO from Dr. Miroslav Ravic, who is guid­ ing the company through the completion of its Phase I study.

B

04.04.2014 12:57:24 Uhr

44

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

Per spec tives base containing additional information that can be accessed through the unique device identifier.

© luchshen, fotolia.com

Paths to unique device identifiers

MeDtech

Development of UDI systems

Jesús Rueda Rodríguez, EDMA, Director, Regulatory Affairs, Brussels

Knowing the tools of your trade is an essential part of any profession, but identifying the various kits and instruments that are used in a clinical lab has to date been done in an ad-hoc manner. Each manufacturer chooses how they want to refer to their products, and many laboratories have developed their own shorthands to refer to routinely-­used pieces of equipment and reagents.

With the move to the information age, a consistent way of identifying devices electronically is needed to ensure that information regarding the devices can be recorded in a consistent form. This is essential for information management systems and potentially for electronic patient records, though in the case of in­vitro diagnostiscs (IVDs) it is often not the device but rather the result that needs to be encoded.

To address this issue, the last few years have seen the development of the concept of unique device identification systems for

44_EBSIN4_14_EDMA_tg.indd 44

all medical devices, including IVDs. Much of the early work has been carried out at the international level by the Global Harmonization Task Force, and more recently by its successor, the International Medical Devices Regulators Forum. We are now seeing the birth of the first major unique identification system in the US, with other planned systems beginning to be developed in the EU and Japan.

Unique device identification systems are made up of two components: a unique device identifier on the product in the form of a barcode (linear or 2D), and a data-

The unique device identifier will always contain two elements. A device identifier (UDI-DI), which is a unique code that refers to device type, and a production identifier (UDI-PI), which identifies that specific device amongst all others of its type – normally in the form of a serial, batch, lot number or – in some cases – in the form of the date of manufacture.

The UDI-DI is in turn composed of two elements: the first is a code identifying the manufacturer, provided by an international entity that operates systems for the assignment of UDIs and guarantees that the code will be unique to the manufacturer.

The second is a code assigned by the manufacturer that is specific for that device.

In addition to the unique device identifier, other information may be encoded in the barcode for immediate access. How­ ever, most of the information will be available through a UDI database that contains further information on the device, including details on how to contact the manufacturer, relevant safety information, and a description of the device itself. Critically, it would also allow the rapid dissemination of information on whether a given device has been subjected to a recall or other specific action that users need to be aware of. One of the main challenges of UDI is ensuring that the various databases under development are interoperable, so that information can be shared easily and issues can be identified rapidly. Interoperability between the projects in the US and the EU would therefore be a valuable fundamental step.

Regulators have gotten involved in UDI mainly because they want incidents to be reported more consistently and to implement product recalls more effectively. However, once devices start to be codified throughout Europe – something that is still some years off, 2016-2017 seem likely dates – other applications within laboratories for information and stock management will undoubtedly be developed to leverage this new information.

B

02.04.2014 16:53:54 Uhr

Celebrating excellence in Europe Vol. 4

The new European Biotechnology Science & Industry Guide 2014, showcasing brilliant science and excellent business from companies, universities, research institutes, and expert support providers.

This 4th edition of the Guide gives new depth to the organisations profiled and demonstrates how Europe translates its life sciences into everyday life. Plus, brand-new reports about the European biotechnology sector.

Available at your local bookseller or online ISBN 978-3-928383-49-3 EUR 19.80

BIOCOM AG | Luetzowstr. 33-36 | 10785 Berlin | Germany | www.biocom.de

45_EBSIN4_14_Euroguide-2014.indd 1

03.04.2014 14:58:23 Uhr

46

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

Per spec tives ers trying to understand the complex pathways information takes in translation from complex, highly-regulated genomes to phenotypic changes.

Synthetic Biology

Building yeast from scratch

Real-world synthetic organism

For the first time, researchers have built a functional eukaryotic chromosome from the ground up. The engineered baker’s yeast Saccharomyces cerevisiae was able stably pass down its man-made genes to its offspring. Reported at the end of March in the journal Science, the major breakthrough in synthetic biology is now fuelling hopes that bio­engineers will soon be able to equip yeast with a full set of synthetic and changeable genes for the production of pharma and chemical compounds.

“We can shuffle genes into these chromosomes like a deck of cards,” explains Jef Boeke from the New York University Langone Medical Center’s Institute for System Genetics. His team conducted the study together with researchers from the French Institut Pasteur. But what newspapers are calling “the first step to artificial life” is in fact just another milestone in synthetic bio­logy. After all, the researchers didn’t synthesise all 16 chromosomes in the yeast genome. However, the work has shown the future potential of genomic engineering, as the researchers inserted tiny markers into 2.5% of the single-cell organism’s nucleotides. They then changed

or deleted non-essential sites (loxPsym) to see if the yeast survived, gaining valuable insights into the yeast’s software along the way. According to Boeke, “we have changed the letters of this chromosome as we would in a book.”

Over the last five years, scientists like genome pioneer Craig Venter have mainly built bacterial chromosomes in an attempt to identify the basic set of genes required for life, and have outfitted synbio constructs with the genes required to produce novel medicines, or raw materials for food and biofuels. The new study, dubbed the “Mt. Everest of synbio”, is especially interesting to academic research-

The researchers put their artificial chromosomes in living yeast cells and tested their ability to grow on different nutrient substrates under different conditions. In each case, the version equipped with a synthetic chromosome functioned indistinguishably from native yeast.

What’s more, the synthetic chromosome was stable over more than 125 cell divisions. After deletion or alterations for distinct loxPsym sites, Boeke’s team found that some cells grew more slowly, while others with different recombinations of genes grew very quickly. By recombining the DNA in different ways, the researchers hope to be able to engineer organisms that make more ethanol than natural yeast, for example, or grow better in difficult environments.

Some researchers see huge application potential in the results. “This is significant as an example of synthetic genomics aimed well beyond making mere copies of chromosomes – the new trend being the making of significant functional changes – ideally changes useful for biotech productivity and safety,” says Harvard biologist George Church. Boeke also is enthusiastic about progress in the field. “We are entering a new era in biotechnology,” he says.

“This is just the beginning of a whole series of products in the works that spring from the ancient relationship between humans and yeast.”

© IAndrea Dant/fotolia.com

Huge market potential

46_EBSIN4_14_SynBio_tg.indd 46

According to Transparency Market Research, the global synthetic biology market is still in its infancy. Worth US$1.5bn in 2011, it grew to US$2.1bn in 2012, and is expected to reach US$16bn by 2018 – an annual growth rate of over 40%. According to market researcher estimates, Europe has the biggest market share, and is expected to maintain that lead position in terms of revenue until 2018.

B

02.04.2014 16:55:13 Uhr

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

47

Produc ts & Services Gilson

Promocell

Eppendorf

High throughput: Optimising cell As easy as it gets transfection

Single-use vessel for microbiology

A Limburg-Offheim – PlateMaster is an easy-to-use, accurate solution for highthroughput pipetting of 96 and 384-well plates. Its compact and ergonomic design means it can be used anywhere and by anyone for fast, effortless and reproducible pipetting of 96 channels. The manual 96-channel pipetting PlateMaster can easily be operated by everyone after an introduction of just five minutes. Since it is a manually controlled instrument, there is no complicated and time-consuming programming. The operation is as easy and intuitive as with a manual pipette.

Filling a 96-well plate requires less than 10 seconds. Loading 384-well plates is possible in four pipetting steps. Using PlateMaster, high-throughput experiments can be performed while avoiding high costs and saving staff resources. When using high-quality Pipetman tips, accuracy and precision levels equalling those of an 8- or 12- channel pipette are achieved in a single motion. Additionally, it allows you to avoid the risk of double-pipetting entire rows or columns. Through a variety of accessories, such as adapters for loading 384-well plates, reagent reservoirs or sample heater blocks, the PlateMaster can be fully adapted to various needs.

D

A Heidelberg – Magnet-assisted transfection (MATra) is an easy-to-handle, very fast (15 minutes!) and highly efficient technology to transfect cells in culture. With MATra, all

A Hamburg – The BioBLU 0.3f and 1f Single-use Vessels allow microbial process development at a new level. These rigidwall stirred-tank vessels have been specifically designed for high cell density fermentation with bacteria, fungi and yeast.

BioBLUf vessels can be used with the Eppendorf DASbox and DASGIP parallel bioreactor systems. Compared to cell culture applications, fermentation processes require much higher kLa values for proper mass transfer, as well as suitable heating and cooling options. Proven stirred-tank design, powerful overhead drives featuring Rushton-type impellers and innovative baffles for cooling make the BioBLUf vessels achieve these demands.

Contact

Contact

Contact

Phone: +49-06431-212-150

Phone: +49-6221-649-340

bioprocess-info@eppendorf.de

www.gilson.com

www.promokine.info/promotion

www.eppendorf.com/BioBLUf

47_EBSIN4_14_PIs_ml.indd 47

types of nucleic acids can be delivered. The technique does not disturb the membrane architecture, nor does it cause chromosomal damage. MATra can be used with adherent or suspension cells – with or without serum.

Compared to other transfection techniques like electroporation or lipofection, MATra results in extremely low cytotoxicity, and is very cost-effective. MATra can also be used in high-throughput transfection assays.

Promocell’s PromoFectin transfection reagent provides highly efficient and reproducible delivery of nucleic acids into a variety of cell types, including many hard-to-transfect cell lines and primary cells. It consists of a non-liposomal polymer free of any components of animal origin. PromoFectin compacts and protects the nucleic acid of interest, enables efficient transport into the cells via endocytosis and allows for a rapid and almost complete release of the intact nucleic acid into the cytosol – all of which favour the delivery to the nucleus. Special cell typespecific PromoFectin variants are also available (endothelial cells like HUVECs or HDMECs, hepatocytes, macrophages, dendritic or neuronal cells).

D

The advantages in short: (1) Reliable scalability through industrial design; (2) Sealed magnetic overhead drives with encapsulated bearings and Rushton-type impellers for excellent mixing; (3) High-performance mass and heat transfer suitable for high cell density fermentation; (4) Minimal set-up times and easy handling; (5) Liquid-free Peltier exhaust condenser; and (6) All wetted materials free of animal components (USP Class VI). The BioBLU vessels hence combine the benefits of single-use bioreactors with the reliable performance of conventional glass or stainless steel bioreactor technology. D

02.04.2014 16:55:41 Uhr

48

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

Company And Advertiser Inde x A A1M Pharma AB (S)… …………………………… 23 AB Science SA (F)………………………………… 19 Ability Pharmaceuticals SL (E)…………………… 29 AC Immune SA (CH)……………………………… 12 Acteaventures GmbH (GER)……………………… 25 Activaero GmbH (GER)…………………………… 27 Active Biotech (S)………………………………… 19 Adaptimmune Ltd. (UK)… ……………………… 27 Adocia S.A.S. (F)… …………………………… 16, 19 Algeta AS (N)……………………………………… 23 Almirall S. A. (E)…………………………………… 50 Amgen Inc. (USA)………………………………… 11 Amirsehhi Intellectual Property Law (GER)…… 36, 41 Amneal Pharmaceuticals (USA)… ……………… 30 Anasyst (B)………………………………………… 42 Ark Therapeutics (UK)… ………………………… 12 AstraZeneca AB (S/UK)………………………… 43 Atopix (UK)………………………………………… 16

B Bayer AG (GER)…………………………………… 23 Bayer Healthcare AG (GER)……………………… 25 Betagenon AB (S)………………………………… 16 BioAlliance (F)… ………………………………… 19 Biocartis SA (CH)… ……………………………… 24 BIOCOM AG (GER)… …………………… 11, 19, 45 Biocrates Life Sciences AG (A)…………………… 24 BioInvent International AB (S)…………………… 19 Biotech Saniona AB (DK)………………………… 22 BioTOP Berlin-Brandenburg (GER)… …………… CP3 bluebird bio France … …………………………… 18 BOB - Best of BIOTECH (A)………………………… 9 Boehringer Ingelheim (GER)… ……………… 16, 18

C Caixa Capital Biomed (E)…………………………

Canbex Therapeutics Ltd. (UK)… ………………

Capital Markets (India) Private Ltd.………………

Cardio3 Biosciences (B)… ………………………

Cargill Dow LLC (USA)… …………………………

Celera Genomics (USA)… ………………………

Cell2B (PT)…………………………………………

Celltech Group plc (UK)… ………………………

Chiesi Farmaceutici S.p.A. (I)… …………………

Circassia Ltd. (UK)…………………………………

CMC Supply (DK)… ………………………………

Cobra Biologics (UK)………………………………

Codexis, Inc. (HUN)… ……………………………

Collaborative Drug Discovery Inc. (USA)…………

Crossject (F)… ……………………………………

Curevac GmbH (GER)… …………………………

28 43 26 12 29 43 28 43 17 12 23 15 30 13 12 25

D Danske Bank (DK)…………………………………

DBV Technologies (F)… …………………………

Debiopharm S.A. (CH)……………………………

Dell Inc. (USA)… …………………………………

Discuva Ltd. (UK)… ………………………………

21 19 24 33 25

E Elan Corp. (IRL)…………………………………… 43 Eli Lilly (USA)…………………………………… 16, 43

48_EBSIN4_14_Index_mak.indd 48

Elsa GmbH (GER)………………………………… 24 Endocyte Inc. (USA)… …………………………… 18 Eppendorf AG/Bioprocess Center Eur. (GER)…… CP2 Eppendorf AG (GER)……………………………… 47 Ernst & Young GmbH (GER)……………………… 12 Erytech Pharma (F)… ………………………… 3, 19 Esteve S.A. (E)… ………………………………… 28 Eurofins Scientific (F)… ………………………… 21 European Biotechnology Network (B)…………… 36 EY Farmindustria (I)… …………………………… 29

F/G Ferrer Group Int. SA (E)… ……………………… 28 Ferring International Center SA (CH)… ………… 27 Fort Knox F√∂rvaring AB (S)……………………… 17 Forum MedTech Pharma e.V. (GER)… ………… 37 Galapagos NV (B)………………………………… 17 Galmed Pharmaceuticals (IL)… ………………… 12 Genfit SA (F)… ………………………………… 19, 20 Genticel S.A. (F)… ……………………………… 12 Gilson International B.V. Deutschland…………… 47 GlaxoSmithKline (UK)… ………………… 16, 20, 26 Glycotope GmbH (GER)… ……………………… 24 GSK Pharmaceuticals Ltd. (IND)… ……………… 26

H/I Horizon Discovery Ltd. (UK)……………………… 12 Hospira (UK)… …………………………………… 11 Hospira One 2 One Global Pharmac. (USA)… … CP4 HSBC Trinkaus & Burkhardt KGaA (GER)………… 26 Human Genome Sciences Inc. (USA)…………… 33 IBM gmbH. (GER)……………………………… 33, 38 IMS Institute for Healthcare Informatics (I)……… 33 Index Pharmaceuticals AB (S)… ………………… 50 InHouse Patent Counsel LLC (USA)……………… 33 Innate Pharma SAS (F)…………………………… 19 Intrexon Corp. (USA)……………………………… 30

J Janssen-Cilag (B)… …………………………… 18, 22 Johnson & Johnson (USA)… …………………22, 30 Jossa Arznei GmbH (GER)… …………………… 24

K/L Karo Bio AB (S)…………………………………… 19 Kempen & Co (NL)… …………………………… 33 KPMG (GER)… ……………………………………… 3 Lee‘s Pharmaceutical Holdings Ltd. (HK)… …… 26 Life Technologies Corp. (USA)…………………… 25 Lombard Medical Techn. plc (UK)… …………… 12 Lubrizol Corp. (USA)……………………………… 29

M Mater-Biotech S.P.A. (I)…………………………… 29 Medivir AB (S)… ………………………………… 19 Merck KGaA (GER)… ………………………… 17, 43 Merck Serono (GER)……………………………… 43 Molecular Partners AG (CH)……………………… 12 Mologen AG (GER)… …………………………… 16 Morgan Stanley (DK)……………………………… 21 Myriad Genetics (USA)…………………………… 37

N Neovacs SA (F)…………………………………… 17 NeuroSearch A/S (DK)…………………………… 22 NeuroVive Pharmaceutical AB (S)… ………… 19, 23 Nicox (F)…………………………………………… 19 NNIT A/S (DK)… ………………………………… 21 Novamont S.p.A. (I)… …………………………… 29 Novartis AG (CH)… ……………………………… 25 Novartis Europharm Ltd. (UK)…………………… 18 Novimmune SA (CH) … ……………………… 12, 43 Novo Nordisk A/S (DK)………………………… 21, 23 Novozymes A/S (DK)… ………………………… 22 Noxxon Pharma AG (GER)… …………………… 50

O Omegam Laboratoria BV (NL)……………………

OncoDNA SA (B)… ………………………………

Oncos Therapeutics Ltd. (FI)… …………………

Oragenics (USA)… ………………………………

Orexo AB (S)… ……………………………………

21 27 18 30 19

P Patentopolis Holding BV (NL)… …………………

Pharmium Securities (F)… ………………………

Piramal Imaging (GER)……………………………

Polyphor Ltd. (CH)…………………………………

Prionics AG (CH)… ………………………………

PromoCell GmbH (GER)… ………………………

Prosensa BV (NL)… ………………………………

Prothena Corp. plc (IRL)… ………………………

29 19 17 12 25 47 12 43

Q/R Qiagen NV (NL)………………………………… 34, 35 Regenx Biosciences LLC (USA)… ……………… 28 Renovo (UK)… …………………………………… 12 Roche AG (CH)… …………………… 16, 20, 25, 43

S Salans FMC SNR Denton Europe LLP… ………… 32 Sanofi SA (F)……………………………… 16, 26, 27 SAP AG (GER)… ………………………………… 40 Sartorius AG (GER)… …………………………… 18 Sillajen Inc. (KR)…………………………………… 26 South Korean SK Chemicals Co. (KR)…………… 27 Synthon (NL)……………………………………… 17

T/U Takeda Pharmaceuticals (JP)… ………………… 18 TAYLOR WESSING Germany………………………… 7 Thermo Fisher Scientific (USA)… ……………… 25 Transgene SA (F)… …………………………… 19, 26 TxCell S.A. (F)…………………………………… 27, 12 UCB Pharma SA (B)… …………………………… 26 uniQure BV (NL)… ……………………………… 17 UPM (FI)…………………………………………… 23

V/Y Valneva (F)……………………………………… 17, 20 Vectura Group plc (UK)…………………………… 27 Venter Pharma S.L. (E)…………………………… 28 Vernalis plc (UK)… ……………………………… 43 Voisin Consulting (F)……………………………… 18 Vontobel AG (CH)………………………………… 12 Ysios Capital Partners (E)………………………… 28

04.04.2014 13:00:11 Uhr

N –º 4 | Volume 13 | 2014

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Euro|Biotech|News

E VENTS 23.-25.4.14: Systems Pharmacology in Drug Discovery & Development, Noordwijkerhout (NL) Info: Per Öhrngren, EUFEPS (E-Mail: manager@pharmscievents.com, Web: www.systemspharmacology.eu)

21.-22.5.14: Biosimilars 2014, Berlin (GER) Info: Informa Life Sciences (E-Mail: registrations@informa-ls.com, Web: www.informa-ls.com/event/ biosimilars2014)

27.-30.4.14: Human Genome Meeting 2014, Geneva (CH) Info: HUGO (E-Mail: hgm2014@mci-group.com, Web: www.hgm2014-geneva.org)

22.-23.5.14: REGATEC 2014 – 1st International Conference on Renewable Energy Gas Technology, Malmö (S) Info: Prof. Dr. Frank Scholwin, Institute for Biogas, Waste Management and Energy (E-Mail: info@biogasundenergie.de, Web: http://regatec.org/)

6.5.14: BioVaria 2014, Munich (GER) Info: Esther Lange, Ascenion GmbH (E-Mail: info@ascenion.de, Web: www.biovaria.org) 6.-7.5.14: European Algae Biomass, Sevilla (E) Info: ACI – Active Communications International (Web: www.wplgroup.com) 6.-8.5.14: Global Biobanking 2014, London (UK) Info: IQPC (Web: www.globalbiobanking.com) 7.-8.5.14: EuroMedtech 2014, Linz (A) Info: Simon Englhart, EBD Group (E-Mail: senglhart@ebdgroup.com, Web: www.ebdgroup.com/emt) 8.-10.5.14: ECRD 2014 – The European Conference on Rare Diseases & Orphan Products, Berlin (GER) Info: EURORDIS/DIA (Web: www.eurordis.org/ecrd2014-berlin) 8.-9.5.14: 3 Minicircle & DNA Vector Conference, Bielefeld (GER) Info: Dr. Martin Schleef, PlasmidFactory GmbH & Co. KG (E-Mail: Martin.Schleef@PlasmidFactory. com, Web: http://cms.plasmidfactory.com)

22.-23.5.14: Bio meets Economy – Science meets Industry, Halle/Saale (GER) Info: ScienceCampus Halle/Plant-based Bioeconomy (Web: www.sciencecampus-halle.de) 23.5.14: 2nd International Symposium of the Virtual Institute Viral Strategies of Immune Evasion – VISTRIE, Braunschweig (GER) Info: HZI Braunschweig (Web: www.helmholtz-hzi.de/) 28.-29.5.14: BioForum 2014, Lodz (PL) Info: Justyna Grzelak, Bio-Tech Consulting Ltd. (E-Mail: bioforum@bioforum.pl, Web: www.cebioforum.com) 28.-30.5.14: Medical Devices – European Regulatory and Market Access Trends, Opatija (HR) Info: Farmavita Regulanet d.o.o.

(Web: www.medical-device.farmavitar.com/en)

rd

10.-13.5.14: 24th European Congress of Clinical Microbiology and Infectious Diseases (ECCMID), Barcelona (E) Info: ESCMID, the European Society of Clinical Microbiology and Infectious Diseases (E-Mail: eccmid@kenes.com, Web: www.eccmid.org) 12.-14.5.14: BioTrinity 2014 - European Biopartnering and Investment Conference, London (UK) Info: David Boal, OBN Ltd (Tel.: +44-0845-5215222, E-Mail: david.boal@obn.org.uk, Web: www.biotrinity.com) 13.-15.5.14: European Lab Automation ELA 2014, Barcelona (E) Info: Select Biosciences (Web: http://selectbiosciences.com) 15.-18.5.14: European Biotechnology Congress 2014, Lecce (I) Info: (Web: www.eurobiotech2014.eu/index.php) 20.-22.5.14: 9 th World Stem Cells & Regenerative Medicine Congress, London (UK) Info: Terrapinn Ltd. (Web: www.terrapinn.com/conference/stem-cells) 21.-22.5.14: BioEquity Europe, Amsterdam (NL) Info: EBD (Web: www.ebdgroup.com)

49_EBSIN4_14_Events_mak.indd 49

31.5.-3.6.14: European Human Genetics Conference 2014, Milan (I) Info: ESHG (E-Mail: conference@eshg.org, Web: www.eshg.org/eshg2014) 3.-5.6.14: Medtec Europe 2014, Stuttgart (GER) Info: (E-Mail: medteceurope@ubm.com, Web: http://medteceurope.com) 11.-14.6.14: BioTech 2014 and 6 Czech-Swiss Symposium, Prague (CZ) Info: Olga Schreiberová, Inst. of Chemical Technology Prague/Inst. of Biotechnology, School of Life Sciences and Facility Management (LSFM), Zurich (E-Mail: info@biotech2014.cz, Web: www.biotech2014.cz) th

15.-19.6.14: Euromit 2014 – International Meeting on Mitochondrial Pathology, Tampere (FI) Info: Kaisa Immonen, Institute of Biomedical Technology, University of Tampere (E-Mail: kaisa.immonen@uta.fi, Web: www.euromit2014.org) 17.-18.6.14: Biomedica 2014 – The European Life Science Summit, Maastricht (NL) Info: Viktoria Lhomme, LifetecZONe/BioLiége/ LifeTech Limburg/LifeTec Aachen-Jülich e.V. (E-Mail: biomedicasummit@tema.de, Web: www.biomedicasummit.com) 22.-26.6.14: 21st European Biomass Conference and Exhibition, Copenhagen (DK) Info: Maddalena Grassi (E-Mail: biomass.conference@etaflorence.it, Web: www.conference-biomass.com)

Conference

DNA vectors A Bielefeld – Addressing scientists that wish to keep themselves updated with the recent advances in minicircle and DNA vector technologies, this event in the German city of Bielefeld (May 8–9) will feature invited speakers plus oral presentations by young scientists from selected abstracts. Focal topics include applications in DNA vaccination and gene therapy. D Contact Martin.Schleef@PlasmidFactory.com www.plasmidfactory.com

Congress

Talking shop A Lecce – Organised by the European Bio­technology Thematic Network Association (EBTNA) and Salento University, the main aim of the European Biotechnology Congress 2014 on 15–18 May is to share and discuss the latest advances, techniques and issues in the field. D Contact irmak.acar@devent.com.tr www.eurobiotech2014.eu

Conference

Microbial biotech A Prague – BioTech 2014 will concentrate on microbial biotechnology – a key factor in the bio-based economy that impacts foodstuffs, animal feed, cosmetics and pharma­ ceuticals. The event is organised by the Institute of Chemical Technology (Prague) and the ZHAW Institute of Biotechnology (Wädenswil). The 6th Czech-Swiss Symposium will take place concurrently in the Czech capital on 11–14 June.

D Contact info@biotech2014.cz www.biotech2014.cz

03.04.2014 15:01:28 Uhr

50

N –º 4 | Volume 13 | 2014

Euro|Biotech|News

ENCORE Minicircle & DNA Vector Conference, Bielefeld, Germany

Biovaria 2014, Munich

6-7

MAY

6

31-3

21-22

13-15



BioForum 2014, Łódz, Poland

8-9 European Lab Automation, Barcelona

LAST MINUTE

 Noxxon AG reports Phase IIa data from its emapticap pegol (NOXE36) trial in diabetic nephropathy. The spiegelmer neutralises the targeted pro-inflammatory chemokine – and reduces urinary albumin even after cessation of treatment.

BioEquity Europe, Amsterdam

Biosimilars 2014, Berlin

Taming viruses  DNA and RNA viruses are widely used in academic and therapeutic research, but one major problem is their safety profile – especially when it comes to oncolytic and live vaccine viruses.

Now German scientists from Heidelberg and Konstanz have diverted riboswitches – short RNA sequences whose enzymatic function can be modulated by adding ligands – to conditionally knock down viral gene expression (PNAS, doi: 10.1073/pnas.1318563111). The system doesn't rely on promoters to alter gene expression in cells, and is simple, efficient, and more flexible than e.g. the Tet on/off system, the team claims. 

… I am passionate about biopharma innovation and giving a voice to SMEs. Titta Rosvall-Puplett, Executive Director, European Biopharmaceutical Enterprises (EBE), Brussels

NUMBER CRUNCHER

Identity crisis  How much do doctors know about biosimilars? According to a survey conducted by the Alliance for Safe Biologic Medicines (ASBM) – not enough!  “If two medicines have the same non-proprietary scientific name, does this suggest to you or imply that the medicines are structurally identical?”

No Opinion 15%

Yes No

© ASBM

32%

Question from a web-based survey of 470 expert prescribers in France, Italy, Germany, Spain and the UK.

© EBE

 “No unduly distorting competition” – the Commission approves a43m in French state aid for a green chemistry public-private-partnership.

European Human Genetics Conference, Milan

SCIENCE

I'm in biotechnology because …

   Swedish Index Pharma and Spanish Almirall sign a license agreement for the European marketing rights to Index’ anti-inflammatory Phase III drug candidate Kappaproct.

The deal value could exceed a100m.

50_EBSIN4_14_Encore_ml.indd 50



28-29

European Algae Biomass, Sevilla, Spain

53%

JUNE



NEXT ISSUE

Cell-based success  Physiologically relevant cellular assays are increasingly used to test drug candidates or decipher complex signaling pathways. One major development in recent years has been the implementation of these assays in high-throughput systems. EuroBiotechNews 5/2014 will highlight how developers in biopharma in particular have discovered the advantages of cellbased assays for testing the cytotoxicity and potency of their prospects. The issue will hit kiosks and news stands on 15 May. Advertisements can be placed until 30 April. 

WINNERS & LOSERS

Different fates  Swedish biotechs Medivir AB and Swedish O r phan Biovit r um A B (Sobi) presented new clinical data in late March. One firm was happy, one was not.  Medivir's simeprevir against HCV scored good results in Phase III ATTAIN and Phase IIa LEAGUE-1 trials.

Phase III fail for Sobi: The enzyme treatment Kiobrina (rhBSSL) does not help growth in premature babies.

03.04.2014 15:03:09 Uhr

Life Sciences.

Living Research.

Berlin. Brandenburg.

A vibrant network.

Berlin-Brandenburg is one of Europe´s leading locations for the Life Sciences. It is also an R&D capital that magnetically attracts the world´s top scientists. Here you´ll find attractive subsidy conditions, close linkages between business and science, the highest concentration of R&D in Europe, a dynamic startup scene and a multifaceted entrepreneurial environment made up of more than 500 pharmaceutical, biotech and medical engineering companies. Come discover this one-of-a-kind landscape for scientists and entrepreneurs. www.businesslocationcenter.de/healthcareindustries Meet us at conhIT 2014 − May 6-8 | Hall 1.2 | Booth B115 conhIT Business Meetings 2014 − Find your business partner: www.b2match.eu/conhit2014

51_EBSIN4_14_BerlinPartner.indd 1 210x275_Motiv_LifeScience_eng.indd 1

02.04.2014 17:01:29 Uhr 31.03.2014 11:38:51

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KNOWLEDGE

Pharmapack 2014, Paris SWISS BIOTEC DAY 2014, Zurich ILSI-Biomed 2014, Tel Aviv

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04.04.2014 13:01:33 Uhr