European Biotechnology News 9/2012 - Free Excerpt - Gene therapy rounds the bend in Europe

Nº 9 | Volume 11 | 2012 | 10.00 € | ISSN 1618-8276 | A 60711 |

European Biotechnology News nds th

end in Europe eb

Gene therapy rou

Science & Industry

CENTRAL EUROPE

Molecular Partners signs US$1.1bn deal with Allergan NORTHERN EUROPE

Russian investors swallow Denmark's Affitech A/S WESTERN EUROPE

Sanofi files to switch indications for alemtuzumab Special

EuroBiotech Compass 2012 Guide to Life Sciences Events SOUTHERN EUROPE

FRE

T P R E E EXC

Antibióticos in bioplastics deal with US developer Metabolix Eastern EUROPE

Polish insulin maker Bioton cashes in final Bayer cheque science & technology

Research teams prove existence of cancer stem cells

Roche X-tremeGENE Transfection Reagents

Now proven in 275 cell types  Efficiently transfect a broad spectrum of cells including difficult cells  Generate physiologically relevant results with low cytotoxic reagents  Obtain top-level protein yields  Quickly establish your transfection protocols

Free sample and protocols at www.x-tremegene.roche.com

For life science research only.

Not for use in diagnostic procedures.

Roche Diagnostics GmbH Sandhofer Straße 116 68305 Mannheim, Germany

X-TREMEGENE is a trademark of Roche.

© 2012 Roche Diagnostics.

All rights reserved.

N –º 9 | Volume 11 | 2012

Euro|Biotech|News

Intro

FREE EXCERPT

Editorial

Challenges and answers: HTA in the economic crisis

Andrea Rappagliosi, Co-Chair EFPIA HTA Task Force, Sanofi Pasteur MSD, Lyon

D

Andrea Rappagliosi is Vice President Market Access, Vaccine Advocacy & Medical Affairs at Sanofi Pasteur MSD, the only company in Europe totally dedicated to vaccines. During his career in both the public and the private sectors, he has focused on access issues that impact patients and healthcare systems in Europe.

Rappagliosi is also VP of the European Vaccine Manufacturers Group and co-chair of the EFPIA HTA Task Force. The author would like to express a special thank-you to Edith Frenoy, Director at EFPIA, for her contributions to these reflections.

uring the last thirty years, healthcare expenditure has been growing much more rapidly than GDP in OECD countries, causing increasing concerns about the long-term sustainability of current trends.

As we have seen with the global impact of the financial crisis, economic realities are moving faster than political ones. The standards of healthcare that European citizens are provided with today risk becoming economically unsustainable, if the efficiency of healthcare systems is not factored in when assessing the value of prevention and treatment of diseases.

Against this background, we need to look in more detail at the determinants of healthcare expenditure in the countries of Europe, explicitly taking into account aspects like the role of income, the effects of an ageing population, life habits, technological progress, and institutional and budgetary variables.

When developing products and solutions for European patients today, the research-based pharmaceutical industry is responding to evi­ dence­-based requirements put in place by regulatory agencies – which request proof of efficacy, quality and safety – and 30+ Health Technology Assessment (HTA) agencies across Europe, which want proof of relative effectiveness. In the area of prevention, the vaccine industry has to provide dedicated evidence to public authorities, which in turn make recommendations prior to any public prevention programme. There is a potential for unnecessary duplication between these different requirements, as they are actually sourced from the same sets of data.

The need to secure timely, equitable and sustainable patient/citizen access to healthcare services has led to a growth in collaboration between HTA agencies recently, and this has been strongly supported by the European Commission. Industry believes there is room for this

initiative, whilst simultaneously keeping regulatory and HTA processes separated and keeping pricing and reimbursement decisions fully at the national level.

Of particular note is the collaboration between HTA agencies, which is supported by the European Commission and will be formalised in a permanent network as of next year. The current network (EUnetHTA) is working on developing methodological guidance for relative effectiveness assessment. Industry is supporting this approach, and is contributing to this discussion in order to feed in experience and know-how. Moving forward, industry would certainly favour increased collaboration between the regulatory world and the European collaboration on HTA, as this would ensure that evidence-based requirements receive the best scientific attention and are aligned across the range of decisionmakers, allowing for an ongoing dialogue between the parties. What is crucial is that consultation with stakeholders, including those in industry, is warranted.

Today, the key challenge for governments is designing pluralistic systems of healthcare delivery and financing. In this area, a wellbalanced mix of public and private financing would put market forces to work promoting investment and innovation, without imposing unsustainable burdens on public budgets or denying prevention and care to the disadvantaged population.

B

3

4

N –º 9 | Volume 11 | 2012

Euro|Biotech|News

CIRCUL ATION

FREE EXCERPT

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

CMYK

Net work

Europe: european-biotechnology.net

Blue: 100/15/0/35 Orange: 0/75/90/0

Germany: biodeutschland.org

Switzerland: swissbiotech.org

Council of European BioRegions

Europe: cebr.net

Europe: ebe-biopharma.org

Europe: europabio.org

Norway: biotekforum.no

Ireland: ibec.ie/ibia

Finland: finbio.net

Denmark: danskbiotek.dk

Sweden: swedenbio.com

The Netherlands: niaba.nl

Belgium: bio.be

Spain: asebio.com

France: france-biotech.org

Italy: assobiotec.it

Hungary: hungarianbiotech.org

EuroBiotechNews covers the biotechnology sector of the current 27 EU member states, Norway and Switzerland. If you would like to subscribe, please refer to Portugal: www.apbio.pt

www.eurobiotechnews.eu

N –º 9 | Volume 11 | 2012

contents

FREE EXCERPT

Insight

Cover Story 10

© Fotolia.de

Heard in Brussels

5

Euro|Biotech|News

First EuroBiotechMonitor survey finds business climate good for biotech in EU 1 2 Commissioners to reform Eu clinical trials directive; New EMA registry 13 IP Flash; Biotech SMEs meet in Brussels 14 Regulatory Affairs Breaking news from the EMA

16

Update on clinical trials

17

Perspectives

Is gene therapy finally coming into its own?

Interview: Olivier Litzka, Edmond de Rothschild Invest. Partner 18

After a balancing act that went down to the wire with regulators – and left its predecessor company bankrupt – uniQure biotherapies now looks set to become the first pharma company to gain approval for a gene therapy in Europe. The concept of ‘curing’ rare inherited diseases by altering a patient’s genome in key cells has been around for decades. But disastrous, highly-publicised failures to harness the technology in the past frightened off developers and patients alike. Now that looks set to change.

Economic Focus on newsflow and dividends

19

Stock markets

20

Northern Europe Denmark, Sweden, Finland, Norway, Iceland 22 Central Europe Germany, Austria, Switzerland

24

Western Europe UK, France, Belgium

26

Southern Europe Italy, Spain, Portugal Eastern Europe Poland, Hungary, and Slovenia

Services

Special

Regional News

28

30

6

Imprint

EuroBiofairs Compass Navigating in the events season 33

5

Biopeople News from Photocure, ADC Therapeutics, FENS, Wilex, Ariana Pharma, and Horizon Discovery

46

Company index

47

BioSpain 2012, Pamplona

34

BioPartnering Future Europe, Brussels

36

Planet xMAP 2012, Monaco

38

euroPLX, Sorrento, Italy

40

New products

48

Journées Internationales de Biologie, Paris

42

Events What’s on in Sept.-October 2012

49

BIO-Europe 2012, Hamburg

44

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, Dr. Philipp Graf, Dr. Bernd Kaltwaßer, Dr. Martin Laqua; Advertising: Oliver Schnell, +49-30-2649-2145, Advertising USA: Avani Media, Inc. , Leslie Hallanan, Tel.: +1-415-331-2150 , Fax: +1-415-289-0402, E-Mail: leslie@avanimedia.com; Distribution: Angelika Werner, +49-30-2649-2140; 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: © fotolia.de

® BIOCOM is a registered trademark of BIOCOM AG, Berlin, Germany.

10

N –º 9 | Volume 11 | 2012

Euro|Biotech|News

Cover Story LPLD patients who had already had acute pancreatitis one or more times.

Euro|BioTech|News

Aldag:

!

The patient population is smaller. I would estimate that 350-700 of LPLD patients in Europe have already had acute pancreatitis. But that's not a limitation to our business, because we won’t treat hundreds of patients in the first year after approval. We will first have to identify patients that carry the gene defect and have had pancreas inflammations. To do that, we have to go to special hospitals in Europe where patients with acute pancreatitis are treated and provide the doctors with our companion diagnostics chip. Our aim is to identify patients in about 20 hospitals throughout the EU.

Euro|BioTech|News

?

What price do you think is realistic?

Aldag:

!

You have to look at prices for other orphan drugs on the market. Our approach has a big advantage: it's a one-time treatment that is effective for years. But if Glybera offers at least the same benefit as comparable orphan treatments, prices should be similar. Prices for orphan drugs like Provenge, Cere­zyme or Naglazyme range from a100,000-a450,000 annually. Since our therapy has already proven efficacy over several years after just one treatment, I think it's reasonable to start at the high end of this range. I think you can do the math.

Before we have any agreement in place with healthcare payers, I am, however, reluctant to spell out pricing details in the media. I am sure you appreciate that.

Euro|BioTech|News

?

What’s your outlook?

Aldag:

Heard in Brussels

?

How will this reduction in the patient population affect your business?

!

We have learnt an incredible amount throughout the approval process. Now we have a unique technology platform to make hundreds of medicines.

B

FREE EXCERPT

M

Fixing Europe (again)

Brussels – Well ladies and gentlemen of the bio­technology community, welcome back after the silly season. I hope you had a nice one, whether it was in the sunny bit, the rainy bit or the bits that were on fire.

As usual, summer in Europe was quiet.

I see we still have a Eurozone, which is nice, and shows that the revolutionary, bio­tech­-driven solution I came up with earlier in the year is still working.

But to business – what startling insights and wisdom can I provide from Brussels this month? Very little based on activity over the summer, so I shall instead turn once again to my crystal ball, and present a list of predictions based on my incurable optimism.

European public funding: after a year of wrestling with the Framework pro­ gramme and Innovative Medicines In­ itiative, my gut tells me that the agencies will at last start plan­ning their funding together, finally al­lowing partners to plan an annual cal­endar of collaborative project applications without having to worry about deadlines all landing in the space of a single month.

National funding: countries in Europe will suddenly remember, in the midst of reducing funding for innovation, that it is innovation that drives economic growth. If all your scientists are fired, particularly those in SMEs, you throttle economic progress, making early-stage research pointless and large company growth in your country impossible.

Policymakers: these will return from their holidays miraculously alive to the fact that some things are not worth arguing about any more – I refer of course to GM technology in food crops (just say yes or no), the EU patent law (just get it finished for crying out loud) and consumer genetic testing legisla­tion (don’t let quacks and charlatans sell tests).

Claire Skentelbery, Secretary General of the European Biotechnology Network

Big companies: my inner eye is particular focussed here, foreseeing that the pipeline crisis and continued shrinkage of R&D capacity will revolutionise the way that large companies work with others. There will be less of a ‘business on our terms’ approach to partnership and more quick movement, shared skills and earlier-stage partnerships.

Small companies: those rascals, they're taking up a lot of space in my crystal ball. Biotech SMEs will certainly start working more closely together, rather than trying to nurse a single technology through to blockbuster status.

And they will start to understand the strength brought about by partnership with other SMEs – that they are stronger together than alone.

That's it for my post-summer predictions – and given my astounding success at curing the Eurozone crisis earlier this year, I'm sure they will all have come to pass by Christmas.

See you next edition – by which time that holiday tan will have faded and the silly season will be a distant memory.

B

12

N –º 9 | Volume 11 | 2012

Euro|Biotech|News

INSIGHT EUROPE

FREE EXCERPT

EUROPE

More cash than expected?

EuroBiotechMonitor: surprising optimism

External financing was one problem most companies experienced. While public or larger companies have enough critical mass to attract significant amounts of external financing and licensing deals, the majority of smaller biotechs do not. According to the survey, 21% of the respondents said that “bad access to financing hindered business development.” Around the same number said that the EU should increase its investment incentives to support biotech SMEs. Although many companies said that establishing EU-funded partnerships was “easy” (48%) or “manageable”, around a quarter thought that improvements in R&D funding on the part of the EU and less bureaucracy (25%) could help the sector.

Surprisingly, 37% of the firms specified that they had enough cash to last for 1224 months, while an additional 20% had enough for even longer. Around the same number said that access to external sources of financing during the last 12 months was “good”, while a further 33% called it “satisfactory”. One in four said it was “bad” or “not relevant”.

Most European biotechnology companies expect higher revenues in 2012 than in 2011.

Even though access to external financing, especially venture capital, remains a wearisome prospect on the continent, the vast majority of biotechs say they aren’t planning to cut any jobs this year. In an exclusive, EuroBiotechNews has received those top-line results from the 1st EuroBiotechMonitor survey. The poll across 2,100 European biotech companies was commissioned by the Brussels-based European Biotechnology Network. The results of the very first business climate report dedicated to the European biotech sector show that the field remains optimistic despite a challenging business environment.

(A) Our business outlook for next year is …



good



in 2010. In light of the demands for more money from national and European biotech interest groups, an unexpected result was the positive business outlook for next year. Just 7% of company management figures believed that their business will decline in 2013, while 93% expected performance to remain constant or improve (Fig. 1A). Half of the responding fi rms expected to make more money in 2012, while 35% anticipate that business will remain stable (Fig. 1B). However, the mostly small companies also said that support from the European Commission needed to be extended.

(B) Our revenues in 2012 are expected to be …

satisfactory



weak

© European Biotechnology Foundation

“The EuroBiotechMonitor survey gives us a tool to improve our unterstanding of the trends and challenges biotech companies are facing in Europe,” says study director Dr. Sebastian Delbrück. According to the survey1, which included all dedicated EU biotech companies that met OECD criteria, more than two out of three (68%) respondents judged their enterprise’s current business situation as either “satisfactory” or “good”. Half of the mostly small and medium-sized companies said that their total revenues in 2011 outperformed those of the previous year, while 34% stated that they had reached the same levels as

Good general business climate Regarding the entire European biotech sector, the respondents felt positive. Over 90% appraised the overall business climate as good (58%) or satisfactory (34%). However, when it comes to future expectations, most firms remain cautious. Only 24% believed that the business climate will continue to improve, while a majority of 63% thought it would remain as it is at the moment. In another positive twist, many of the companies (40%) expect to hire more staff in the next 12 months. Over half specified that they are not planning any changes in staff numbers, and less than one in ten were reducing the employees on the payroll. According to the survey, 86% of the responding firms employ less than 50 staff. 10% had 50-250 employees, and just 4% provided more than 250 jobs.  1

Fig. 1: Business outlook (A) and sales expectations (B) of EU biotech companies for 2012.

All data taken from 1st EuroBiotechMonitor, conducted from 11 May–13 July 2012.

N –º 9 | Volume 11 | 2012 FREE EXCERPT

Euro|Biotech|News

47

Company And Advertiser Inde x

A

EBEWE Arzneimittel GmbH… …………………… 18

P

Abbott Laboratories (USA)… ………………… 16,44

Edmond de Rothschild Investment Partners (F)… 18

Pfizer Inc. (USA)…………………… 6, 16, 18, 21, 22

Actavis (CH)… …………………………………… 30

Elan Corp. (IRL)…………………………………… 21

PharmaMar (Grupo Zeltia) (E)…………………… 11

Actinium Pharmaceuticals Inc. (USA)… ………… 28

Endocyte Inc. (USA)… …………………………… 44

Pharming Group N.V. (NL)… …………………… 18

ADC Therapeutics Sarl (CH)……………………… 46

Endosense SA (CH)… …………………………… 18

Philogen S.p.A. (I)………………………………… 28

Affitech (DK)… …………………………………… 22

Evotec AG (GER)… ………………………………… 8

Photocure ASA (N)… …………………………… 18

Amsterdam Molecular Therapeutics (NL)… ……… 8

F/G

ProBioDrug AG (GER)… ………………………… 18

Amylin Pharmaceuticals Inc. (USA)……………… 44

FIT Biotech Oyi Plc (FI)…………………………… 22

Prometheus Labs (USA)… ……………………… 37

Antibioticos S.A. (E)… …………………………… 28

Galapagos NV (B)………………………………44, 50

PromoCell GmbH (GER)… ……………………… 48

AOP Orphan Pharmaceuticals AG (A)…………… 16

GE Healthcare (GER)……………………………… 48

Apeptico GmbH (A)… …………………………… 25

Genmab A/S (DK)……………………………… 18, 20

Q

Archer Daniels Midland Co. (ADM) (USA)… …… 28

Genomed Management SA (PL)………………… 30

Quartz Bio (CH)…………………………………… 21

Ariana Pharma (F/USA)…………………………… 46

Genzyme (USA)………………………………… 6, 26

Quintiles Limited (UK)… ………………………… 16

ASEBIO (E)……………………………………… 34, 35

GlaxoSmithKline (UK)… ……………………… 17, 22

AstraZeneca (UK/S)… …………………………… 44

GPC Biotech AG (GER)…………………………… 25

R

Grünecker Patent- und Rechtsanwälte (GER)…… 14

RauCon Business Development (GER)… 33, 40, 41

Allergan Inc. (USA)… …………………………… 24

B

Prexton Therapeutics SA (CH)…………………… 21

Reed Expositions France (F)…………………… 42, 43

BASF AG (GER)… ………………………………22, 46

H

Bayer Healthcare AG (GER)……………………… 30

Horizon Discovery Ltd. (UK)……………………… 18

Bayern Innovativ GmbH (GER)… …… 32BB Biotech

Hospira (USA)… ………………………………… 16

(CH)… …………………………………………… 19

Hospira One 2 One Global Pharmaceutical (USA)…7

BIOCOM AG… …………………………………… 25

Hovione FarmaCiencia Sa (PT)… ……………… 28

BIO.NRW (GER)…………………………………… CP3

Roche Diagnostics GmbH (GER)………… CP 2, CP 4

S SALANS LLP (GER)… …………………………… 15 Sanofi SA (F)…………………………… 3, 6, 22, 26

Bioquell Ltd. (UK)………………………………… 23

J

BIOQUELL UK Limited… ………………………… 48

Janssen AI (IRL)…………………………………… 21

BioSystems International (F)… ………………… 46

Janssen Biologics LLC (IRL)… …………………… 17

Bioton S.A. (PL)…………………………………… 30

Janssen Biotech Inc. (USA)… …………………… 20

BioTOP Berlin-Brandenburg (GER)… …………… 17

Janssen-Cilag International NV (B)… …………… 16

BioWin - The Health Cluster of Wallonia (B)… 27, 49

JenaValve Technology GmbH (GER)… ………… 18

Boehringer Ingelheim GmbH (GER)… ………… 22

Johnson & Johnson (USA)… …………………… 20

Bristol-Myers Squibb (USA)……………………… 44

ReNeuron Group plc (UK)… …………………… 17

L

SAP AG (GER)… ………………………………… 20 SciGen Ltd. (SG)… ……………………………… 30 Seagarden ASA (N)… …………………………… 22 Serono SA (CH)…………………………………… 21 SuperSonic Imagin (F)… ………………………… 18

T Takeda Pharmaceuticals (JP)… ………………… 16

C

Lehman Brothers Inc. (USA)……………………… 44

Technology Vision Group (BPFE 2012)(USA)…36, 37

Celgene Europe Ltd. (USA)… …………………… 16

LISA Vienna Region, Clustermanagement (A)…

29

ThromboGenics NV (NL)… ……………………… 17

Celltrion (KR)……………………………………… 16

Luminex (I)……………………………………… 38, 39

Thomson Reuters (USA)… …………………… 13, 50

Centocor BV (NL)… ……………………………… 17

Trans Nova Investments Ltd. (CY)… …………… 22

Chitinor (N)… …………………………………… 22

M

co.don AG (GER)… ……………………………… 22

Merck & Co. (USA)………………………………… 44

U

Crucell N.V. (NL)… ……………………………… 46

Merck KGaA (GER)… ………………………… 21, 50

uniQure BV (NL)… ………………………… 6, 8, 16

Cryo Innovation Ltd. (HUN)……………………… 30

Metabolix Inc. (USA)……………………………… 28

Cryo Management Ltd. (HUN)…………………… 30

Microsynth AG (CH)……………………………… 19

V

CSR plc. (UK)……………………………………… 18

Molecular Partners AG (CH)…………………… 8, 24

ViiV Healthcare (UK)……………………………… 22

MPM Capital GmbH (GER)… …………………… 25

Vitrolife AB (S)… ………………………………… 30

DBV Technologies (F)… ………………………… 17

N

W

deCode genetics (IS)… ………………………… 22

Neotope Bioscience (IRL)………………………… 21

Watson Pharmaceuticals (USA)… ……………… 30

Deloitte & Touche GmbH (GER)… ……………… 18

Newron Pharmaceuticals S.p.A. (I)……………… 28

Wilex AG (GER)………………………………… 18, 20

Diamyd Medical AB (S)…………………………… 22

Novartis AG… ……………………………………… 6

D

Dievini Hopp Biotech Holding GmbH (GER)… … 20

Novartis Europharm Ltd. (UK)…………………… 16

X

Donau-Universität Krems (A)… ………………… 25

Novexel S.A. (F)…………………………………… 18

Xcellerex Inc. (USA)… ……………………………… 9

Noxxon Pharma AG (GER)… …………………… 18

Xvivo Perfusion AB (S)… ………………………… 30

EBD Group (GER)………………………………44, 45

O

Z

European Biotechnology Network… …………… 13

Ono Pharma UK Ltd.……………………………… 50

Zoran Corp. (USA)………………………………… 18

E

n g & PR rketi fo Ma h in N R r c e t o W i B

,

ce

N Ex a h

rs

I n te r n a t i o na and al n and Conf l Fa s i it o tion er en i ib

s Te c

hno

log y Tran

s fe

r

a An

ps

Su

pp

,

s

io is of B tech Bu lys s cience in N ine S d RW s an

or t-u t of ar B i o te c h St Co ach ing ing & Financ

P ro m o t i o n of Yo s ung Ac a d e mic

bio.nrw.de Busyness for Biotech.

North Rhine-Westphalia’s biotechnology cluster BIO.NRW is a central catalyst for the sustainable development of the state’s biotechnology sector.

It activates cooperation between business, research, investors, and policymakers.

phone: +49-211-385469-9200 • E-Mail: bio.nrw@bio.nrw.de

Size: Reduced. Fun: Amplified.

Get to know a completely new type of real-time PCR instrument at: www.lightcycler-nano.com

For life science research only.

Not for use in diagnostic procedures.

LIGHTCYCLER is a trademark of Roche.

Roche Diagnostics GmbH Sandhofer StraĂ&#x;e 116 68305 Mannheim, Germany Š 2012 Roche Diagnostics.

All rights reserved.