Biotechnology Focus February/March 2017

INSIGHTS FOR THE LIFE SCIENCE INDUSTRY

February/march 2017 VOLUME 20, NUMBER 1

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Anniversary

A Network for

Personalized Cancer Care Building a Pan-Canadian Molecular Registry

INSIDE:

Reimbursement & Market Access for Innovative Canadian Biotechnologies

Publication Mail Registration Number: 40052410

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FEATURES

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contents

BUILDING A PAN-CANADIAN MOLECULAR REGISTRY TO PERSONALIZE CANCER CARE The emergence of personalized medicine has brought with it a paradigm shift to both cancer care and drug development (By Valerie Higenell, Richard Fajzel and Gerald Batist)

FEBRUARY/MARCH 2017 – VOLUME 20 – NUMBER 1

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RECAPPING THE 2017 JP MORGAN HEALTHCARE CONFERENCE Lumira’s Benjamin Rovinski gives his take on what took place at JPM 2017 in San Francisco (By Benjamin Rovinski)

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PROTECTING PRECISION DIAGNOSTIC INVENTIONS Overcoming the obstacles that may limit the pace of commercialization for precision biomarker based diagnostic advances (By Carmela De Luca)

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THE SPARTAN CUBE: REVOLUTIONIZING DNA ANALYSIS Ottawa’s Spartan Bioscience is set to make point-of-care DNA testing a reality with the development of the world’s smallest DNA analyzer!

(By Shawn Lawrence)

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(Compiled by Shawn Lawrence)

DEPARTMENTS 6

RESEARCH NEWS

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BUSINESS CORNER

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NEW PRODUCTS

29 CALENDAR OF EVENTS www.biotechnologyfocus.ca

The unique organization that combines Canada’s cancer research community’s strengths to take immunotherapy discoveries into the clinic (By Dr. John Bell)

BIOTECHNOLOGY 20TH ANNIVERSARY SPECIAL: Looking back at our past issues, Years 1 to 3, to highlight various moments in time that speak not just of then, but of today as well.

BIOCANRX TAKES UP CANADA’S CANCER FIGHT

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THE LAY OF THE LAND A run down of what’s available to Canadian biotech companies in terms of Government grants & incentives (By Chris Chapman)

IN EVERY ISSUE

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ACROSS CANADA

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THE LAST WORD

Reimbursement and market access for innovative Canadian biotechnologies (By Kaitlyn Proulx and W. Neil Palmer)

The 2017 Canadian Biotech Imperative: Remaining competitive in the face of uncertainty (By Andrew Casey) February/March 2017 BIOTECHNOLOGY FOCUS 3

PUBLISHER’S note PUBLISHER/ EDITOR-IN-CHIEF SENIOR WRITER CONTRIBUTING WRITERS

Terri Pavelic Shawn Lawrence Andrew Casey

Benjamin Rovinski Carmela De Luca Chris Chapman Gerald Batist John Bell

Reflecting BACK ON two decades of Biotechnology Focus!

Kaitlyn Proulx

Twenty years ago, I made decision to launch Biotechnology Focus Magazine. I’ve seen this anniversary coming for some time, and pondered things, big and small, to reflect upon reaching this milestone starting with what initially inspired me to launch the publication.

Well, as a science graduate, I loved to learn how things worked, but I found working in a lab monotonous. I was looking for a career that would allow me to keep in touch with the pursuit of science discovery. I stumbled upon publishing and found it was the right fit because it incorporated my banking experience along with the entrepreneurial drive my parents instilled in me. I haven’t looked back since.

Over the last 20 years, the life science industry has seen many twists and turns, however, it has always been about discovery and innovation – And I find this exciting!

During these two decades of publishing, I’ve also been asked many times what’s the secret to Biotechnology Focus’s staying power? As Canada’s oldest life science trade magazine, I belive our staying power is due to staying true to our mission statement; helping life science executives find useful information, insights, resources and inspiration for running and growing their businesses. We do this in a number of ways. One of our most important resources is the contributions from leading Canadian biotech and life science experts that make up our readership community. Each month, they continue to come up with thought provoking articles. To these folks, thank you for your ideas and stories, and please keep them coming!

Likewise, our staying power can be attributed to our ability to adapt. I’ve learned the biggest challenge to media is the constant change to technology. It has fragmented how we keep abreast of what is happening within our industry and it is constantly changing how everyone accomplishes their job. And it’s no longer just about producing a magazine, it’s about understanding the preferred medium an individual wants to receive their information. Nowadays, people stay on top of what affects them through a number of mediums such as; magazines, web sites, newsletters, conferences, social media, blogs, etc. So media companies have to provide valuable information in multiple mediums.

This means more work, more deadlines and quicker turn around. And truthfully, we wouldn’t have it any other way.

Simply put, we at Biotechnology Focus know that commercialization in the life science field is not for the faint-hearted. Our goal is to help you get there. Moreover, I feel privileged to have watched these people grow their companies. Some succeed and some do not. Regardless, I am constantly impressed at the resourcefulness of these entrepreneurs, their sheer drive and their persistence. It is always inspirational to be able to write about these people who have the courage to give their all.

Looking ahead, I see the future as something that is fluid and constantly evolving.

But one thing is certain, Biotechnology Focus wants to continue reporting on the commercialization journey of Canadian life science organizations, in whatever new medium technology introduces.

And if you’ve read this far — by which I mean this pub note, not for two decades — I just want to say thanks, from all of us to all of you, for being part of our community, your ongoing support means so much to us!

W. Neil Palmer

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Richard Fajzel Valerie Higenell GRAPHIC DESIGNER CONTROLLER MARKETING MANAGER

Elena Pankova John R. Jones Mary Malofy

CIRCULATION DIRECTOR Mary Labao circulation@promotivemedia.ca Tel: 905-841-7389

EDITORIAL ADVISORY BOARD Christine Beyaert, Roche Canada; Pierre Bourassa, IRAP, Montréal; Murray McLaughlin, Sustainable Chemistry Alliance; Ulli Krull, UTM; John Kelly, KeliRo Company Inc.; Peter Pekos, Dalton Pharma Services; Robert Foldes, Viteava Pharmaceuticals Inc.; Gail Garland, OBIO; Barry Gee, CDRD; Bonnie Kuehl, Scientific Insights Consulting Group Inc.; Raphael Hofstein, MaRS Innovation; Roberto Bellini, Bellus Health; Peter van der Velden, Lumira Capital; Albert Friesen, Medicure Inc.; Ali Tehrani, Zymeworks Inc.; Dr. Jason Field, Life Sciences Ontario; Andrew Casey, BIOTECanada

Biotechnology Focus is published 6 times per year by Promotive Communications Inc. 1-226 Edward Street, Aurora, ON L4G 3S8, Phone 905-727-3875 Fax 905-727-4428 www.biotechnologyfocus.ca E-mail: biotechnology_focus@promotive.net Subscription rate in Canada $35/year; USA $60/year; other countries $100/year. All rights reserved. No part of this publication may be reproduced without written consent. Publications Mail Registration Number: 40052410 Return undeliverable Canadian addresses to: circulation department – 1-226 Edward Street, Aurora, ON L4G 3S8 National Library of Canada ISSN 1486-3138 All opinions expressed herein are those of the contributors and do not necessarily reflect the views of the publisher or any person or organization associated with the magazine.

If you would like to order hard copy or electronic reprints of articles, contact sales@promotivemedia.ca

2017

CANADA’S NATIONAL BIOTECHNOLOGY AWARD PROGRAM – GOLD LEAF AWARDS

PROGRAMME NATIONAL DE PRIX DE BIOTECHNOLOGIE DU CANADA – LES FEUILLES D’OR

BIOTECanada’s Gold Leaf Awards program is an opportunity to recognize both companies and individuals who have contributed to the biotech industry.

Le programme des Feuilles d’or de BIOTECanada vous permet de rendre hommage aux entreprises et aux personnes qui ont accompli des réalisations importantes au sein de notre industrie.

AWARD CATEGORIES

CATEGORIES DE PRIX

NEW FOR 2017 – ECOSYSTEM BUILDER

NOUVEAU POUR 2017 – BÂTISSEUR DE L’ÉCOSYSTÈME

An ecosystem builder will be an organization or company who has distinguished itself through its contributions to Canadian communities beyond their respective business and economic impact. The respective nominee demonstrates leadership, showing significant contributions to the Canadian biotech ecosystem through their engagement and responsibility.

Une organisation ou société s’étant distinguée par ses apports aux collectivités canadiennes, au-delà de son impact commercial et économique naturel. Le candidat doit avoir fait preuve de leadership dans des initiatives locales, régionales ou nationales et avoir contribué de façon considérable, par ses activités et les responsabilités qu’il assume, à l’écosystème canadien des biotechnologies.

NEW FOR 2017 – INDUSTRY CATALYST An individual or group of individuals have challenged the status quo, broken down barriers, and re-written the rules for their biotech business.

Their dedication and everyday actions are leading the biotech revolution.

Respected by people inside and outside their organizations, these individuals are viewed as true innovators.

EMERGING COMPANY

NOUVEAU POUR 2017 – CATALYSEUR ÉCONOMIQUE Ce visionnaire a remis en cause le statu quo, éliminé des barrières et remanié les règles de son secteur d’affaires en biotechnologie. Son dévouement et ses actions de tous les jours font avancer la révolution des biotechs. Cette personne, respectée par les membres de son organisation comme par les intervenants externes, est vue comme réellement innovatrice.

ENTREPRISE EN DÉMARRAGE

(1 Health, 1 Agriculture/ Industrial)

(1 en santé et 1 dans le secteur agricole et industriel)

INNOVATION IN BIOTECH RESEARCH

INNOVATION DE LA RECHERCHE EN BIOTECHNOLOGIE

COMPANY OF THE YEAR

ENTREPRISE BIOTECHNOLOGIQUE DE L’ANNÉE

“We’re fiercely proud to be a Canadian company and we’re humbled to be recognized by BIOTECanada and our peers.

We’re looking forward to continuing the important work that has taken us to this point to make a difference for patients, for our team, and for the Canadian biotechnology industry. “ –Dr. Ali Tehrani, President and CEO of Zymeworks.

Winner Gold Leaf Award Company of the Year 2016

“Nous sommes farouchement fiers d’être une entreprise canadienne et nous sommes humbles d’être reconnus par BIOTECanada et nos pairs. Nous sommes impatients de poursuivre l’important travail qui nous a permis de faire une différence pour les patients, pour notre équipe et pour l’industrie canadienne de la biotechnologie.”

–Dr. Ali Tehrani, président et chef de la direction de Zymeworks.

Lauréat Les Feuilles d’Or, Entreprise Biotechnologie de l’année 2016

Nominations now being accepted | Candidatures maintenant acceptées Deadline for nominations is April 30, 2017

| La date limite pour la présentation des candidatures est le 30 avril 2017

goldleafawards.com

R&D news CQDM and Brain Canada team up again to fund two new brain research projects

Edward Fon MONTREAL, QC – CQDM and Brain Canada are teaming up to fund two new multi-disciplinary institutional research projects under the second edition of their joint Focus on Brain strategic initiative.

In all, $3 million will go to two research teams developing cutting-edge technologies designed to accelerate the discovery of new drugs for brain and nervous system disorders. The two research teams include nine researchers from seven public and private organizations across Canada.

The first project is led by Edward Fon at the Montréal Neurological Institute and Hospital (MNI) joined by multi-provincial collaborators from McGill University, Université Laval and the University of British Columbia. The project involves the development of a novel drug discovery platform for Parkinson’s disease and amyotrophic lateral sclerosis (ALS).

The platform is based on the isolation of pluripotent stem cells derived from the blood of affected patients. Under appropriate cell culture conditions, these stem cells will differentiate into neurons, thus mimicking in vitro what is happening to these cells in patients. The team will also develop three assays to monitor the properties of these neurons, and thus better understand how they are affected in these diseases. The neuronal cell lines will be used to screen for drugs that could correct their defective properties.

The second project led by Jean-Martin Beaulieu at University of Toronto is a public-private collaboration with the Research Institute of the McGill University Health Centre and ImStar Therapeutics in Vancouver.

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Jean-Martin Beaulieu The project involves the development of an exclusiveRNA-binding proteins platform to screen, identify, and validate new drug candidates that affect a large, still relatively unexplored family of proteins. These RNAbinding proteins may be what are needed to address a large spectrum of major brain disorders, such as autism, depression, and Alzheimer’s disease. The technology will, among other things, help tackle the shortage of disease-relevant targets; a main hurdle to the development of new therapeutics to treat these conditions. Moreover, this screening platform will enable identification and development of new classes of drugs while reducing risks at early stages of development, thereby expediting the availability of new CNS-relevant drugs to patients.

In addition to receiving funding, the research teams will participate in CQDM’s unique mentoring program, and will have the opportunity to collaborate with influential senior scientists from the pharmaceutical industry.

“These two very exciting projects could have a huge impact on our understanding of neurodegenerative diseases such as Parkinson’s, Alzheimer’s and ALS,” said Diane Gosselin, president and CEO at CQDM. The Brain Canada funds are provided through a partnership with Health Canada, known as the Canada Brain Research Fund.

To see this story online visit http://biotechnologyfocus.ca/cqdm-andbrain-canada-join-forces-again-fund-twonew-brain-research-projects/

Zymeworks opens new state-of-the-art lab facility VANCOUVER, BC – Zymeworks Inc. has opened a new state-of-the-art, 10,000 square foot laboratory facility in Vancouver, BC. According to company president and CEO, Dr. Ali Tehrani, the new lab will provide the company with increased control over its discovery research activities including antibody generation, medicinal chemistry, bioconjugation for generating antibody drug conjugates and developing multi-functional proteins.

“Zymeworks’ new lab is an investment in our future, it will enable us to perform our own internal research and development in a fully integrated manner,” Tehrani said. “As the Zymeworks family continues to grow, so do our requirements, and the capabilities our new lab provides demonstrate our commitment to efficiently advance and expand our therapeutic pipeline.”

He adds that having Zymeworks’ discovery components together in one centralized location will enhance the company’s ability to expedite certain processes on an as-needed basis.

To see this story online visit http://biotechnologyfocus.ca/zymeworks-opens-new-state-of-the-art-labfacility/

Dr. Ali Tehrani

R&D news

Immune therapy scientists discover distinct cells that block cancer-fighting immune cells

Dr. Pamela Ohashi, Director, Tumour Immunotherapy Program at the Princess Margaret Cancer Centre, University Health Network.(Photo credit: UHN) TORONTO, ON – Princess Margaret Cancer Centre scientists have discovered a distinct cell population in tumours that inhibits the body’s immune response to fight cancer.

According to principal investigator Pamela Ohashi, director, Tumour Immunotherapy Program at the cancer centre, University Health Network, the findings are critical to understanding more about why patients will or will not respond to immune therapies.

“We’ve uncovered a potential new approach to modulate the immune response to cancer,” Dr. Ohashi explains. “By looking at tumour biology from this different perspective we’ll have a better understanding of the barriers that prevent a strong immune response. This can help advance drug development to target these barriers.”

The research team with international collaborators analyzed more than 100 patient samples from ovarian and other cancer types to discover the distinct population of cells found in some tumours.

They found that this population of cells suppresses the growth of cancer-fighting immune cells, thereby limiting the ability of the immune system to fight off cancer.

For patients, down the road Dr. Ohashi envisions a new era of combined therapies

to simultaneously target and kill these suppressive cells while augmenting the immune response against cancer. “This would really strengthen the way clinicians can treat cancer using immune therapy, which holds so much promise for patients.”

The team’s next avenue of research will be focused on identifying a “biomarker” that can reveal this distinct suppressive cell elsewhere in the body – for example, in blood or other samples – as a potential predictive clinical tool to determine when these cells are present in patients, which currently cannot be done.

“That knowledge will guide clinical decisions to personalize cancer treatment to unleash an individual’s immune response,” says Dr. Ohashi. “We need to identify ways to track these cells and find another source and ways to grow these cells for further study.”

Dr. Ohashi’s research, published online in Nature Medicine, was funded by the Canadian Institutes of Health Research and The Princess Margaret Cancer Foundation.

To see this story online visit http://biotechnologyfocus.ca/ immune-therapy-scientists-discoverdistinct-cells-that-block-cancerfighting-immune-cells/

A messenger RNA vaccine to protect against Zika virus

VANCOUVER, BC – New findings published in Nature highlight the potential of a new messenger RNA vaccine to protect against Zika virus.

Acuitas Therapeutics Inc., a Vancouverbased private biotechnology company is behind the published findings. The company is developing a lipid nanoparticle (LNP) delivery technology for the messenger RNA (mRNA), and the company has demonstrated that a single low dose immunization with a messenger RNA delivered in an Acuitas LNP carrier protects against infection by the Zika virus.

According to the published paper, Acuitas Therapeutics scientists and academic researchers including Dr. Drew Weissman, a professor of Infectious Diseases in the Perelman School of Medicine at the University of Pennsylvania, were able to show that single low-dose immunization with mRNA-LNP encoding the pre-membrane and envelope (prM-E) glycoproteins of a Zika virus strain responsible for the 2013 outbreak elicited potent, durable and protective neutralizing antibody responses in animals.

“I am delighted that our long-standing collaboration with Dr. Weissman continues to demonstrate the exciting clinical potential of mRNA therapeutics,” said Thomas Madden, president and CEO of Acuitas Therapeutics. “The current data show that a single low dose administration of mRNA-LNP encoding a Zika protein completely protected animals from a subsequent challenge with the virus.”

The company says it is continuing its collaboration with Dr. Weissman to further advance this new therapeutic modality.

To see this story online visit http://biotechnologyfocus.ca/new-findings-show-the-potential-of-a-messengerrna-vaccine-to-protect-against-zika-virus/ February/March 2017 BIOTECHNOLOGY FOCUS 7

R&D news Clinical Trials & Patents Avivagen undertakes human health proof-ofconcept study with its OxC-beta™ technology

DelMar Pharmaceuticals (Vancouver, BC) has commenced enrollment in its Phase 2 study of VAL-083 at the

University of Texas MD Anderson Cancer Center in Houston, TX. The Phase 2 study of VAL083 (dianhydrogalactitol) in patients with MGMT-unmethylated, Avastin (bevacizumab)-naïve recurrent glioblastoma will enroll 48 patients in a singlearm design to determine if treatment with VAL-083 improves overall survival, compared to historical control. VAL083 is a “first-in-class,” small-molecule chemotherapeutic that according to the company has demonstrated clinical activity against a range of cancers including GBM in historical clinical trials sponsored by the U.S. National Cancer Institutes.

DelMar has also announced plans to advance VAL-083 into a pivotal randomized multi-center Phase 3 clinical trial for the treatment of bevacizumab-failed GBM and into a separate international Phase 2 trial for newly diagnosed GBM patients with an unmethylated MGMT promoter.

OTTAWA, ON – Avivagen Inc., a life sciences company commercializing products intended to replace the antibiotics added to livestock feeds, has launched a new project to explore the potential application of its OxC-beta™ technology in humans.

With the help of the National Research Council of Canada (NRC), the company plans to evaluate the efficacy of OxC-beta in an established research model of a yet to be announced human infectious disease.

As part of the project, work will be conducted by NRC experts at its facilities based upon a jointly developed protocol and is expected to be completed in 2017.

The OxC-beta™ technology is derived from Avivagen discoveries about carotenoids, compounds that give certain fruits and vegetables their bright colors and is a non-antibiotic means of maintaining optimal health and growth.

“We are pleased to be working with NRC on this project to establish a proof-of-concept for use of OxC-beta for a first potential human application – likely as a preventative intervention or as part of a therapeutic regimen,” Dr. James (Jamie) Nickerson, Avivagen’s director of Product Validation, commented. He adds that OxC-beta has demonstrated safety and effectiveness across more than a dozen livestock trials and those results suggest it may also prove 8 BIOTECHNOLOGY FOCUS February/March 2017

to be important to human health and wellbeing.

“The results of this project should help to provide such evidence,” he said.

“Antibiotic drug resistance in bacteria is a major concern that could affect the health of Canadians as previously curable infections may become untreatable and spread through the community,” said Dr. Lakshmi Krishnan, program leader in Human Health Therapeutics at the National Research Council of Canada. “We have a longstanding expertise in infectious disease research and are pleased to collaborate with Avivagen on this innovative project which will evaluate the potential of OxC-beta to provide additional effective human health treatments.”

Avivagen says it is not disclosing the specific disease target at this time to help ensure the patentability of this potential new application for the OxC-beta™ technology.

However, the company does say that the disease target is one of the top 18 urgent, serious or concerning drug-resistance threats listed by the U.S. Centers for Disease Control.

To see this story online visit http://biotechnologyfocus.ca/avivagenundertakes-human-health-proof-of-concept-study-with-its-oxc-beta-technology/

Aurinia Pharmaceuticals (Victoria, BC) has chosen Worldwide Clinical Trials as its clinical research organization (CRO) service provider for its upcoming AURORA Phase 3 study of volcosporin to treat active lupus nephritis (LN). With support from the CRO Aurinia says it will now proceed with conducting a randomized, placebo-controlled, double-blind global 52-week trial in approximately 320 patients. Lupus Nephritis (LN) in an inflammation of the kidney caused by Systemic Lupus Erythematosus (SLE). Voclosporin is a calcineurin inhibitor (CNI)/immunosuppressant that has a dual mechanism of action. By inhibiting calcineurin, voclosporin blocks IL-2 expression and T-cell mediated immune responses. It is made by a modification of a single amino acid of the cyclosporine molecule which has shown a more predictable pharmacokinetic and pharmacodynamic relationship, an increase in potency, an altered metabolic profile, and potential for flat dosing. Aurinia reports it is on track to commence the AURORA trial in the second quarter of 2017, with results from this study to be used to support the company’s New Drug Application (NDA) submission to the FDA.

Quark Venture and GF Securities team up again- invest in Biscayne Neurotherapeutics with the leadership, and their unique approach to finding a solution for this devastating disease for which there are currently no therapies available.”

Among the other investments made by Quark Venture and GF Securities are a January US$25 million investment in Microbion

BUSINESS corner

Corp., and a $4 million investment into SQZ Biotech made in late 2016.

To see this story online visit http://biotechnologyfocus.ca/quarkventure-and-gf-securities-team-up-againinvest-in-biscayne-neurotherapeutics/

Aequus lands IRAP funding in support of its Aripiprazole patch development VANCOUVER, BC & MIAMI, FL – Quark Venture Inc. and GF Securities are investing US$1.6 million in Biscayne Neurotherapeutics , through the firm’s $656-million Global Health Sciences Venture Fund.

A clinical-stage company, Biscayne Neurotherapeutics (Biscayne) was established by Biscayne Pharmaceuticals to develop its biopharmaceutical assets for the treatment of neurological disorders. Biscayne’s lead compound BIS-001, is in clinical development as a novel anti-epileptic agent.

The Series B investment will help Biscayne initiate and complete a Phase 1b proof-ofconcept trial of its new extended release formulation of BIS-001 in 2017. BIS-001 is a novel synthetic form of huperzine A, a traditional Chinese medicine that has been used as a treatment for cognitive disorders for centuries. Biscayne is initially developing BIS-001 to treat refractory forms of focal epilepsy, including refractory partial complex seizures and children with Dravet syndrome.

Huperzine A, offers a unique mechanism of action for the treatment of epilepsy. It has shown promising efficacy in highly predictive preclinical models of refractory complex partial epilepsy, providing 100 per cent elimination of seizures in the majority of the animals. In a Phase 1 trial in patients with drugresistant epilepsy, BIS-001 appeared safe, and serum levels were obtained that support its development as an effective anticonvulsant. Biscayne has developed an extended release formulation to ensure convenient and compliant patient dosing.

“The goal of the Global Health Sciences Fund is to invest in the best science the world has to offer, so we are pleased to partner with Biscayne. whose innovative science was developed by researchers at Harvard and Yale,” said Karimah Es Sabar, director GHSF and CEO, Quark Venture. “We are impressed

VANCOUVER, BC – Aequus Pharmaceuticals reports it will receive up to $100,000 in federal funding through the National Research Council’s Industrial Research Assistance Program.

The company plans to use the funds towards its ongoing proof-of-concept clinical study for its lead product candidate, AQS1301, a once-weekly transdermal aripiprazole patch. Aripiprazole is an atypical antipsychotic and the active ingredient in Abilify®, a leading medication in the U.S. used for the treatment of a number of psychiatric disorders including bipolar I disorder, schizophrenia, major depressive disorder and irritability associated with autistic disorder.

Aripiprazole is currently available in once-daily oral tablets and a once-monthly injectable form, however, medication adherence continues to be a significant challenge for patients.

Offering the medication in a patch form potentially could make it more convenient to use, and improve patient adher-

ence says Anne Stevens, COO and director of Aequus Pharmaceuticals .

“The current study will help define our clinical and commercial prototype patch and moves us one step closer to finalizing partnering discussions for the next steps in the development and commercialization of this product,” she said.

The product is currently in clinical development, butAequus expects to confirm its regulatory development plan in a pre-Investigational New Drug (pre-IND) meeting with the US Food and Drug Administration (FDA) in the second half of 2017.

Aequus anticipates results of the current repeat dose, 28-day study in the first quarter of 2017. The results will be used to inform the final design of the patch to be advanced into the regulatory phase of its clinical trials.

To see this story online visit http://biotechnologyfocus.ca/aequuslands-irap-funding-in-support-of-itsaripiprazole-patch-development/

February/March 2017 BIOTECHNOLOGY FOCUS 9

BUSINESS corner

Dealmakers

MaRS pays $300 million loan from Ontario three years ahead of schedule

TORONTO, ON – A massive infusion of private funds from three private companies has allowed Toronto-based technology innovation incubator MaRS Discovery District to repay the $300 million it borrowed from the province of Ontario.

Manulife, Sun Life Financial and iA Financial Group led the $290-million transaction by investing in 19-year bonds issued by Phase II Investment Trust.

A press release from the innovation centre said, “The proceeds of this transaction will be used to repay most of the Ontario government’s interest-bearing loans to MaRS almost three years ahead of schedule.”

“Canada now has an urban innovation hub at a scale that is fully competitive with any global counterparts,” commented Ilse Treurnicht, CEO of MaRS. She adds that the West Tower is also now fully leased, and could soon generate the operating income required to be entirely self-sustaining, “putting MaRS on stable footing for decades to come.”

But this wasn’t the case just a few short years ago.

Back in 2014, the Liberal government of Ontario extended a $309 million interestbearing loan to MaRS, with the assumption that the tower would achieve 80 per cent occupancy. About $65 million of that money was used to buy out an American 10 BIOTECHNOLOGY FOCUS February/March 2017

developer. For months, the building was close to two-thirds empty, that is until 2015, when prospects for the West Tower picked up gradually, and then Facebook Canada announced it was setting up shop in the tower.

Today, the centre offers more than 1.5 million square feet of state-of-the-art lab and office space and is home to more than 140 research labs and companies spanning the entire innovation ecosystem.

Noteworthy life science tenants include multinational medical firms such as Johnson & Johnson’s JLABS @ Toronto, which itself houses over 40 biotech and health startups, biopharmaceutical company Merck, as well as a growing number of emerging life science firms such as Synaptive Medical, Deep Genomics, Interface Biologics, Highland Therapeutics and Triphase Accelerator. Additionally, biotech incubator Blueline Bioscience and leading research groups from the University of Toronto (which owns a 20 per cent stake in the West Tower), University Health Network (including Princess Margaret Cancer Centre), Ryerson University and the Ontario Institute for Cancer Research, all now call the West tower home.

To see this story online visit http://biotechnologyfocus.ca/marspays-300-million-loan-from-ontariothree-years-ahead-of-schedule/

Astellas Pharma (Markham, ON) has entered into a research collaboration with

GenomeDx Biosciences (San Diego, CA) to use latter’s Decipher® Classifier and Decipher GRID® as a potential aid for genomic tumour profiling and to better identify which prostate cancer patients will respond to treatment with XTANDI® (enzalutamide). As part of the agreement, Astellas will provide GenomeDx with tumour samples from its Phase 2 ENACT trial (NCT02799475), which is comparing the time to prostate cancer progression between patients treated with enzalutamide versus patients undergoing active surveillance. GenomeDx will profile all samples and provide Astellas with an analysis of tumour aggressiveness based on its Decipher Classifier score, and a Decipher GRID profile which will assess the biological behavior of a patient’s tumor based on a set of signatures that may be associated with enzalutamide response.

Quebéc-based venture capital fund AmorChem has launched its second and third spin-off companies, Corbin Therapeutics and SemaThera.

Corbin Therapeutics will focus on developing therapeutics to treat various inflammation-based diseases via inhibition of the ubiquitin carboxyl-terminal hydrolase 15 (USP15) enzyme. As part of the company launch, all rights to the USP15 technology initially held by AmorChem have been transferred to Corbin Therapeutics, along with a first seed investment of $1 million.

Corbin Therapeutics says it will use the funds to screen various compound libraries in order to identify lead candidates which efficiently inhibit the USP15 enzyme, and eventually develop them as therapies for multiple sclerosis (MS). SemaThera is a Montréal-based biotech company focused on developing novel SEMA 3A TRAP inhibitors for the treatment of various retinopathies, including diabetic macular edema (DMA). The company is currently testing different SEMA 3A biological TRAPs for intravitreal administration. Development of these TRAPS could result in either standalone therapy or as adjunct to current therapeutic strategies to more effectively counter diabetic retinopathies. AmorChem has transferred all rights to the SEMA 3A technology to SemaThera, along with a first seed investment of $1 million. SemaThera also signed an exclusive, worldwide, license with Univalor on a series of SEMA 3A TRAP inhibitors to be developed and used in various retinopathies and other non-ocular inflammatory diseases.

BUSINESS corner Evotec and MaRS Innovation launch Fibrocor Therapeutics

TORONTO, ON & HAMBURG, GERMANY – Toronto’s MaRS Innovation is teaming up with Evotec AG, a German-based drug discovery and development partnership company, to launch Fibrocor Therapeutics LP. According to both parties, Fibrocor will focus on developing first-in-class therapeutics that target fibrotic diseases. Taking a new approach to understanding and treating such diseases, Fibrocor plans to bring together clinical expertise and access to disease tissue with high-throughput molecular analysis infrastructure and expertise in clinically-predictive animal models of fibrosis.

The company is being backed with a $ 2.8 million seed financing round, which includes cash from MaRS Innovation. Evotec will pro-

vide all drug discovery activities and will also receive an equity stake. The company is cofounded with leading academic and clinician scientists from world-class academic institutions in Toronto, including: Dr. Richard Gilbert, Canada Research chair in Diabetic Complications and Head of Endocrinology at St. Michael’s Hospital and scientist with the hospital’s Keenan Research Centre for Biomedical Science; Dr. Darren Yuen, Nephrologist with St. Michael’s and scientist with its Keenan Research Centre for Biomedical Science; and Dr. Jeff Wrana, senior investigator at Lunenfeld-Tanenbaum Research Institute, Sinai Health System.

The academic/clinical team is to be complemented by management and business development from MaRS Innovation and drug discovery and development services from Evotec.

“Through Fibrocor we have created a transformational partnership with Evotec to translate Toronto’s world-class fibrosis expertise into therapies for patients worldwide,” MaRS Innovation president and CEO Dr. Rafi Hofstein, said.

Established in 2008, MaRS Innovation is

supported by the Government of Ontario through the Ontario Centres of Excellence.

The nonprofit organization has a portfolio of more than 60 companies. Late last year, one of MaRS’s leading portfolio companies, Triphase Accelerator, sold its first investigational anticancer compound, marizomib, to Celgene.

Hofstein hopes to see Fibrocor enjoy similar success.

“We are building on our recent success of Triphase in the area of oncology and applying it to a new therapeutic cluster in the area of fibrosis,” he said.

Fibrocor has already identified a lead program and will partner with Evotec to develop novel molecules with the anticipation of nominating a lead candidate in 2018. In addition, Fibrocor will collaborate with its academic co-founders to expand access to tissue samples to include several relevant tissues, such as lung, liver, kidney, colon and skin.

To see this story online visit http://biotechnologyfocus.ca/evotecand-mars-innovation-launch-fibrocortherapeutics/

FACIT-seeded Fusion Pharmaceuticals closes US$25M Series A led by Johnson & Johnson Innovation Fushion Pharmaceuticals is a spinout from TORONTO, ON – Fusion Pharmaceuticals Inc. (Fusion) reports it has closed a US$25M Series A financing, with Johnson & Johnson Innovation – JJDC, Inc. (JJDC) as the lead investor.

The other investors taking part in the Series A round include venture groups HealthCap, TPG Biotechnology Partners, and Genesys Capital, as well as founding investor Fight Against Cancer Innovation Trust (FACIT). The financing is strengthened by HealthCap’s specialized expertise, having pioneered a new wave of successful radiotherapeutic companies.

Fusion Pharmaceuticals says it will use the proceeds to advance its lead program, FPX01, into human clinical trials. FPX-01 is an antibody-targeted radiotherapy that seeks out a specific biomarker present on nearly all types of treatment-resistant cancers.

According to the company, its technology, targeted alpha-particle emitting radiotherapeutics, combine the precision of molecular targeting agents such as antibodies with the potency of alpha-particle emitting radioisotopes to specifically attack and eradicate cancer cells. Alpha-particle emitting medical

isotopes attack cancer cells using multiple mechanisms, including causing the type of DNA damage that the cancer cell cannot easily repair, reducing the likelihood of developing drug resistance. At the same time, alpha particle-emitting agents offer improved safe-handling features and can be administered in conventional cancer treatment facilities unlike previous generations of radiotherapeutics.

In addition, Fusion is building its pipeline through access to a centyrin protein targeting platform in two licensing agreements with Janssen Biotech in transactions facilitated by J&J Innovation.

the Centre for Probe Development and Commercialization (CPDC), which is a Centre of Excellence for Commercialization and Research Centre (CECR) located at McMaster University. The CPDC has facilities and major partnerships in Hamilton, Boston, Toronto and Ottawa.

The company also received OICR funding support from its inception. As OICR’s commercialization partner, FACIT provided corporate leadership (through its Executive-inResidence program) as well as seed funding to launch Fusion and to attract the strategic partners, investors and the larger capital infusion necessary to grow the company, and continue its research around radiotherapeutics.

The company is led by Dr. John Valliant, who is the founder of both Fusion and CPDC. He was selected as one of the Canada’s top 40 under 40 in 2010.

To see this story online visit http://biotechnologyfocus.ca/facitseeded-fusion-pharmaceuticals-closesus25m-series-a-led-by-johnson-johnsoninnovation/ February/March 2017 BIOTECHNOLOGY FOCUS 11

Oncology

| By Valerie Higenell, Richard Fajzel, Gerald Batist

Building a Pan-Canadian Molecular Registry: A Network for Personalized Cancer Care Personalized Medicine in Oncology The past 20 years have brought major advances in understanding the molecular pathways underlying cancer, and new recognition that each cancer type represents a collection of molecularly diverse diseases.1 Each patient’s cancer bears a unique pattern of molecular alterations, which can be analyzed to help develop a treatment strategy. Increasingly, clinicians can tailor therapy to the molecular profile of a patient, a personalized approach which increases the likelihood of response while sparing patients the expense and toxicity of therapies unlikely to be effective.2 The emergence of personalized medicine has brought a paradigm shift to both cancer care and drug development: identifying biomarkers which predict drug response has become a research priority for industry,3 with personalized therapies representing almost a quarter of all new molecular entities approved by FDA in each of the past three years.4

Sample Profiling for a Therapeutic Match Appropriate matching of therapy and patient requires analysis of patient samples for the biomarker specific for each drug. The type of biomarker which predicts patient response depends on how the drug achieves its effects: eligible patients may carry a single nucleotide mutation, overexpress a receptor, bear a chromosomal rearrangement, etc.5 Biomarkers of interest evolve with the emergence of new profiling technologies,6,7 the development of drugs with novel mechanisms of action, and deeper understanding of the molecular pathways underlying cancer.

The recent influx of immuno-oncology drugs in particular will markedly change how biomarkers of response are analyzed, given the dynamic nature of the immune system and the need to assess cellular as well as DNA and protein markers.8 For example, early reports indicate that predicting response to therapies targeting PD-1/PD-L1 checkpoint proteins may require analysis of transient PD-L1 expression in the tumour or tumour microenvironment,9 density and clonality of tumour-infiltrating lymphocytes,10 overall mutational load,11 expression of T-effector/IFN-γ associated genes,12 or mutation of genes such 12 BIOTECHNOLOGY FOCUS February/March 2017

as the tumour suppressor PTEN.13 With the discovery that certain treatments can drive the expression of immune biomarkers such as PD-L1, a new area of research interest has developed to find combinations of therapies which first drive the expression of immune biomarkers in tumour cells, then kill the cells expressing the marker.11

Operational Challenges in Personalized Cancer Research Although the advantages of personalized oncology care are clear, developing new personalized therapies entails operational and scientific challenges which are difficult to accommodate in the current clinical research model. Many biomarkers for personalized therapy are expressed in only a small fraction of patients (for example, most of the molecular signatures targeted by drugs in use or under development for non-small cell lung adenocarcinoma are present in less than 5 per cent of patients).14 Enrolling these rare patients into clinical trials presents an enormous challenge, especially in a large and sparsely populated country like Canada. Of the patients who bear the biomarker predictive of response to a given drug, some patients will never respond and others will become resistant to therapy. Assessing mechanisms of resistance to therapy or disease evolution requires longitudinal data collection, which is beyond the scope of most clinical research.15 Access to patient samples for molecular profiling represents an

additional hurdle, as patients who are already ill do not always consent to multiple tumour biopsies, and sample collection and processing is generally not standardized across research sites.16 Moreover, the rapid pace of drug development requires that sample collection and analysis constantly adapt to the moving target of industry interest; such agility is not offered by traditional biobanking.

Working Together for Cancer Research Given these challenges, a consensus in the international medical community is growing that a co-operative network of cancer centres is fundamental to streamline and coordinate research efforts in personalized medicine.17-19 Central to this concept is a unified infrastructure for patient recruitment, allowing for coordinated screening and enrollment of patients with distinct molecular profiles.

The ability to identify patients with biomarkers of interest across the country is essential to compete with larger patient populations elsewhere, and attract clinical trials to Canada.

A common database would allow identification of patients regardless of geography, a key consideration in a healthcare system that is managed provincially.20 A national network is equally important to facilitate patient access to investigational drugs, and to enable collection of patient biospecimens. Harmonizing sample collection, quality control procedures, and analysis platforms would improve costeffectiveness and comparability of data across research centres. Furthermore, a national

Personalized Medicine: Therapy which Targets an Individual’s Molecular Profile

ONCOLOGY network promotes communication between the many stakeholders in drug development and healthcare, including scientists, clinicians, patients, trial sponsors, healthcare administrators, regulators, ethics boards, and government at the provincial and federal level. A network of cross-functional members could create a sustainable solution to meet the needs of the Canadian oncology arena as a whole.21

Innovative Networks for Personalized Medicine in Canada To this end, Exactis Innovation (Exactis) launched a pan Canadian cancer patient registry – Personalize My Treatment (PMT) – which aims to facilitate the recruitment and matching of patients to clinical trials of innovative personalized therapies. Patients are recruited to PMT by their cancer care institution, where they consent to have their clinical and molecular data collected, to have their samples profiled, and to be re-contacted if they become eligible for a trial based on the molecular characteristics of their cancer.

Patient data is stored in a centralized digital biobank: through one portal, patients across the country can be identified, selected for biomarker profiling, recontacted, and matched to clinical trials. Patient recruitment focuses on common cancers of clinical research interest which represent a high unmet need. The foundation of the PMT initiative is its network of cancer centres: a Canada-wide collaboration of outstanding scientists and clinicians working together to further personalized cancer care. As part of this infrastructure, a federated network of labs provides flexible analysis of different types of samples using various platforms to determine patients’ eligibility for clinical trials.

The PMT initiative is a cross-Canada collaboration to promote patient access to cutting-edge therapies, and to strengthen the clinical research environment in oncology.

PMT and complementary programs such as the Canadian Partnership for Tomorrow,22 and the Canadian Cancer Clinical Trials Network23 are innovative solutions to some of the ongoing challenges of personalized cancer research in Canada. Pioneering consortia such as these will be important elements for Canada to maintain competitiveness on a global scale: to promote drug discovery, attract clinical trials, and support the integration of personalized oncology medicines into sustainable healthcare.20

About Exactis Innovation: Exactis Innovation is a National Centre of Excellence, non-profit organization that launched PMT, a pan‑Canadian patient molecular registry to facilitate the recruit-

ment of patients to trials of innovative personalized therapies. Exactis also assists in the design, start-up and execution of clinical studies within the PMT network. www.exactis.ca

References 1. Hoadley KA, Yau C, Wolf DM, et al.

Multiplatform analysis of 12 cancer types reveals molecular classification within and across tissues of origin. Cell 2014;158:929-44.

2. Schilsky RL. Personalized medicine in oncology: the future is now. Nat Rev Drug Discov 2010;9:363-6.

3. Milne CP, Cohen JP, Chakravarthy R. Market watch: Where is personalized medicine in industry heading? Nat Rev Drug Discov 2015;14:812-3.

4. Personalized Medicine at FDA: 2016 Progress Report. (Accessed Jan 30 2017, at http://www.personalizedmedicinecoalition.org/Userfiles/PMCCorporate/file/PM-at-FDA.pdf.)

5. Ludwig JA, Weinstein JN. Biomarkers in cancer staging, prognosis and treatment selection. Nat Rev Cancer 2005;5:845-56.

6. Friedman AA, Letai A, Fisher DE, Flaherty KT. Precision medicine for cancer with next-generation functional diagnostics. Nat Rev Cancer 2015;15:747-56.

7. Patel LR, Nykter M, Chen K, Zhang W. Cancer genome sequencing: understanding malignancy as a disease of the genome, its conformation, and its evolution. Cancer Lett 2013;340:152-60.

8. Dracopoli NC, Boguski MS. The Evolution of Oncology Companion Diagnostics from Signal Transduction to Immuno-Oncology. Trends Pharmacol Sci 2017;38:41-54.

9. Sathyanarayanan V, Neelapu SS. Cancer immunotherapy: Strategies for personalization and combinatorial approaches. Mol Oncol 2015;9:2043-53.

10. Tumeh PC, Harview CL, Yearley JH, et al. PD-1 blockade induces responses by inhibiting adaptive immune resis-

tance. Nature 2014;515:568-71.

11. Yuan J, Hegde PS, Clynes R, et al.

Novel technologies and emerging biomarkers for personalized cancer immunotherapy. J Immunother Cancer 2016;4:3.

12. Fehrenbacher L, Spira A, Ballinger M, et al. Atezolizumab versus docetaxel for patients with previously treated non-small-cell lung cancer (POPLAR): a multicentre, open-label, phase 2 randomised controlled trial. Lancet 2016;387:1837-46.

13. Peng W, Chen JQ, Liu C, et al. Loss of PTEN Promotes Resistance to T CellMediated Immunotherapy. Cancer Discov 2016;6:202-16.

14. Minuti G, D’Incecco A, Cappuzzo F. Targeted therapy for NSCLC with driver mutations. Expert Opin Biol Ther 2013;13:1401-12.

15. Murugaesu N, Chew SK, Swanton C. Adapting clinical paradigms to the challenges of cancer clonal evolution.

Am J Pathol 2013;182:1962-71.

16. Mardis ER. The translation of cancer genomics: time for a revolution in clinical cancer care. Genome Med 2014;6:22.

17. Abrams J, Conley B, Mooney M, et al.

National Cancer Institute’s Precision Medicine Initiatives for the new National Clinical Trials Network. Am Soc Clin Oncol Educ Book 2014:71-6.

18. SPECTA: Screening Patients for Efficient Clinical Trial Access. (Accessed Jan 31 2017, at http://www.eortc.org/ wp-content/uploads/2015/08/SPECTA-flyer-2015.pdf.)

19. Mendelsohn J. Personalizing oncology: perspectives and prospects. J Clin Oncol 2013;31:1904-11.

20. Syme R, Carleton B, Leyens L, Richer E. Integrating Personalized Medicine in the Canadian Environment: Efforts Facilitating Oncology Clinical Research. Public Health Genomics 2015;18:372-80.

21. Canadian Cancer Research Alliance.

Pan-Canadian Cancer Research Strategy: A plan for collaborative action by Canada’s cancer research funders. 2010.

22. Canadian Partnership for Tomorrow Project. (Accessed Jan 30 2017, at http:// www.partnershipfortomorrow.ca/.)

23. Canadian Cancer Clinical Trials Network. (Accessed Jan 31 2017, at http://3ctn.ca/.)

To see this story online visit www.biotechnologyfocus.ca/building-a-pancanadian-molecular-registry-a-network-forpersonalized-cancer-care/ February/March 2017 BIOTECHNOLOGY FOCUS 13

Spotlight on JPM 2017

| By Benjamin Rovinski

Lumira’s Insights from the 2017 JP Morgan Healthcare Conference As usual during the second week of January the entire Lumira team made their way to San Francisco, CA for the JP Morgan Healthcare Conference.

14 BIOTECHNOLOGY FOCUS February/March 2017

W

hile a few of our team members always attend the actual JPM Conference, the week is about so much more and at least for us, the preparation, diligence and screening process with respect to who we will meet and who we will hear presentations from typically begins three months before we actually get to San Francisco.

In fact, before we get to the conference we would have reviewed and screened over 500 presenting companies across the various conferences that occur in parallel that week, we would have looked at another 75+ presentations of companies that we are looking to pro-actively meet with during the week, and we would have coordinated over two dozen meetings with the business development and leadership teams of leading global and regional biotech, pharma and medtech companies. The preparatory work is a time consuming but essential part of the process.

Despite the lead up work, there is nothing quite like the spontaneity that occurs when

you have 20,000 people all engaged in the innovative healthcare sector in one place for a week. That spontaneity is often highlighted by a chance conversation with a CEO, investor, or corporate manager as you run between meetings or do the dance at one of the countless number of cocktail parties and breakfast meetings. Unfortunately, this was the first year in many where the torrential and often unrelenting rains took away a fair amount of that spontaneity, as people hunkered down rather than risk another soaking.

So, what did we learn and take away from the conference?

In terms of investment themes there was little doubt that immuno-oncology was continuing to enjoy strong support with the focus centred on companies developing drug candidates that can potentially be combined with checkpoint inhibitors to elicit a synergistic response. Despite this continuing enthusiasm, there were also signs of caution particularly with respect to the fact that the space is already crowded, clinical trials are

Spotlight on JPM 2017 becoming extremely expensive, and it is becoming much harder to recruit volunteers for these trials. One number we heard bandied about is that collectively Merck, Bristol Myers, and Genentech have started more than 500 trials in partnership with private companies to evaluate their checkpoint inhibitors in combo studies.

So, if immune-oncology is exciting but crowded, where else are investors looking?

Liver diseases was one of the other areas frequently mentioned as a very hot area of investment activity. NASH, fatty liver, and Hep B seem to be the most favoured. The third most commonly cited theme appeared to be around autoimmune disorders, and in our discussions with one Foundation closely affiliated with one of our companies, we were told that they had identified more than 300 drug candidates being clinically tested for autoimmune diseases such as arthritis, IBD, lupus, type 1 diabetes, MS, and psoriasis. A lot of interest is also focused on novel approaches to elicit immune tolerance, Treg modulation, and more selective regulation of cytokines.

In terms of unmet need probably the most often referenced area was anti-infectives. This enthusiasm was somewhat tempered by the fact that at this time there was no meaningful investments from the big pharmas, and that

there remained a few regulatory hurdles, particularly for novel antibiotics. Of course, we have seen this before and so we suspect investment from all participants will pick up in 2017/2018.

Some other areas worth a mention include novel therapeutics to treat some viral infections such as HPV and Hep B, and preventative vaccines to prevent infections from few selected viruses such as RSV. On a more macro level, the important questions are always with respect to M&A and IPOs. To that end, most of the CEOs and investors we spoke with believed that 2017 would see a significant increase in M&A activity. Perhaps the two most commonly cited biotech companies were Gilead and Celgene.

Gilead because it has suffered lately in terms of pipeline productivity and clinical success while still fortunately having tonnes of cash, and Celgene which was broadly viewed as needing to catch-up to competitors targeting the solid tumor market.

Many folks also expressed optimistic views on improved deal making conditions based on the believe that there is a good chance that the Trump administration would provide the pharma sector with strong incentives to repatriate their large amounts of overseas cash and that the most logical use for that cash would

be to drive shareholder value via pipeline accretive acquisitions.

In terms of the IPO market, while there seemed to be a lack of clear consensus, sentiment seemed to favour a modestly strong market with one of the bankers we are working with pointing out that there are already several more biotech companies in the IPO queue compared to last year at this time.

There is also a lot of institutional appetite for pre- and early-clinical stage companies, particularly in the immuno-oncology sector.

This, coupled with the fact that there are also a large number of companies with prior S-1 filings, seems to bode well for the public biopharma market, but as always, this is probably one of the more risky dimensions of the sector to predict with any comfort or certainty.

All in all, the take aways were positive and we are hugely enthusiastic about 2017.

Benjamin Rovinski is a Managing Director with Lumira whose focus is on novel drug and biological-based therapeutic drugs

To see this story online visit www.biotechnologyfocus.ca/lumirasinsights-from-the-2017-jp-morgan-healthcare-conference/

February/March 2017 BIOTECHNOLOGY FOCUS 15

20th anniversary

| Compiled by Shawn Lawrence

Biotechnology 20th Anniversary Special: Years 1 to 3!

Biotechnology Focus at the years 1998 to 2000

In celebration of Biotechnology Focus’ twentieth anniversary, we’ve decided to take a trip down memory lane and look back at our past issues to highlight various moments in time that speak not just of then, but of today as well. This month March 1998

Biotechnology Focus publishes its first issue: Vol. 1, No. 1

With an emphasis on the pharmaceutical industry, our premier issue hit news stands with features on Patented Medicines and the Linkage Regulations; a status report on ICH Guidelines relating to the preclinical safety evaluation of new biopharmaceuticals, and a piece explaining Capillary electrophoresis as a tool to bring macromolecular based drugs to the market faster.

We wrapped things up with a piece on the importance of proteomics and its emerging importance in analyzing protein expression.

we’ll begin by looking through the eyes of Biotechnology Focus magazine at the years 1998 to 2000, highlighting some of the major milestone moments in Canadian and global biotech history.

May 1998

January/February 1999 Vol 2, No 1

May/June 1999

Sept

Vol. 1, No. 2

For our second issue we touched upon the agricultural aspects of biotech and we debuted our Research News section (now R&D News).

With our feature stories, we looked at nutraceuticals and how gene technologies allow food producers to develop innovative products with more desirable traits such as pest resistance, enhanced nutritional value and medicinal purposes. Sticking with the ag-biotech theme, Pippa Wysong’s contributed piece The Venture Capital Journey focused on Saskatoon Saskatchewan’s Innovation Place, which 20 years later continues to house Canada’s largest concentration of leading ag-biotech companies. Closing things up we had a technical piece by Fred Rabel on HPLC Method Development.

Cloning was all the rage when this issue hit news stands, starting with the first cloning of a human embryo in South Korea.

Of course, that was overshadowed by the first book about Dolly the Sheep hitting book stands in paperback form. Our Research News section also included a story about scientists unveiling a revised map of the human genome containing what they thought was half of what turned out to be an overly estimated 60,000 genes. Meanwhile, feature stories from the issue included Animal Research in Drug discovery: Trends and Issues; Are Canadian students receiving the right education to land jobs in today’s biotech economy; and Canadian Biosensors: The Next Generation.

The big news story from this issue was Toronto’s Hospital for Sick Children unveiling a then state-of-the-art supercomputer to be used in probing gene sequence homologies and performing 3D protein modeling. Our feature story lineup included an outsourcing piece which discussed what CMOs need from sponsors to keep production running the way it should.

We also ran a feature on monoclonal antibodies and their ability to not just overcome initial limitations but also fulfill a wide range of research, therapeutic and clinical needs. Finally, we ran a technical piece on the potential of fluorescence polarization and the possibility of this method eclipsing manipulated radioisotopebased (RAD) methods for high throughput screening.

Patrica come Canad and m scale d to mic Richar benefi munic comp advice Danie go ab tors, a Sotiria an inte

March/April 1999

July/August 1999

Nov

Editorial themes from this issue include the emerging importance of bioinformatics in biotech, as well as emerging new technologies in genomics and proteomics.

Meanwhile, Jamie Cuticchia’s Right Stuff article addressed the types of skills needed to be a bioinformatician. Lastly, our cover story Gene Genies focused on new gene pathway research technologies like microarrays, two-dimensional gel electrophoresis (2DE) and protein characterization.

Hot topics from this issue include isothermal alternatives muscling in on Polymerase Chain Reaction (PCR) territory, single nucleotide polymorphisms playing a key role in multi-factorial disease treatment, and looking deeper into the potential of gene therapies. On this front, we looked at some of the challenges of targeting and delivering therapeutic genes, and predicting how Canadian courts and legislators would respond to the then landmark Harvard Mouse decision, particularly its impact on Canada’s Patent Act. Also, scientists at Hadassah University Hospital in Jerusalem discovered the HPA gene, a major cause in the spread of cancer to tissues and organs. Wrapping things up, we recap the 1999 Bio International Convention and spotlight Nova Scotia’s biotechnology sector.

Closin our M we ta the In scien plant conse head of the write Edwa serva Prize

1998

Vol 2, No 3

Vol 2

1999

Biotechnology Focus at the years 1998 to 2000

1998 September 1998 Vol. 1, No. 3

In global news Nature reported the first reproducible cloning of mammals from adult cells, which appeared in our Research News Section. For feature stories, we had a spotlight piece on New DNA Technologies, in which author Carol Thomas mentions it had only been a year since the creation of the first human artificial chromosome. Interestingly, her piece also cites the Human Genome Project and its aim to map all 100,000 human genes by the year 2005. Of course, scientists eventually beat that deadline by roughly five years, and the actual total figure was an estimated 19,000 to 20,000 human protein-coding genes. covered current trends and applications for PCR Technologies and We also branched out with more ran a retrospective piece on ABIC special sections including two Tech 1998 and Bio 1998. An interesting focused articles; the first a Canadian sidebar, Bio 1998 was held in NY Perspective on Patenting Higher Life City and attracted a then record of Forms and the other on the FLAG 4,800 attendees. For comparison’s recombinant protein expression sake the event now attracts 16,000 system developed by Sigma (now attendees annually.

Sigma-Aldrich). Additionally we also

16 BIOTECHNOLOGY FOCUS February/March 2017

Vol 2, No 2

Vol 2, No 4

Vol 2

20th anniversary We hope you enjoyed this retrospective look at Biotechnology Focus Years 1 to 3. Be sure to check back in the next issue to see our timeline from 2001 to 2003!

September/October 1999

January/February 2000

May/June 2000

Patrica Nicholson’s piece, Big Things come in Small Packages, highlights Canadian advances in microarray and microfluidics technology that scale down lab-bench procedures to microchip size. Next, PR expert Richard Rothstein discusses the benefits to a well-planned communications strategy for biotech companies both big and small. The advice pieces keep coming with Daniel Pharand’s article on how to go about choosing a board of directors, and editorial board alumni Bob Sotiriadis article on how to approach an intellectual property audit.

We kicked off the new century with the unveiling of our inaugural Editorial Advisory Board.

Composed of some the most senior individuals in the business, the initial roster included: Dr. Cynthia Robbins-Roth, Dr. John Clement, Dr. Peter Morand, Randal Goodfellow and Bob Sotiriadis. In R&D News, over 200 scientists from six countries successfully pieced together the order of 33 million DNA bases for nearly 700 genes on chromosome 22, the first human chromosome to be sequenced under the Human Genome Project. Meanwhile, our editorial lineup featured such compelling topics as how Canadian biotech firms are vulnerable to espionage; the launch of new bioscience co-op programs across Canada to address the industry’s skill shortage gap, and research into apoptosis to decipher the pathogenesis of disease.

Our first real kick at the can in compiling a special issue devoted to understanding outsourcing, this issue featured our picks of contract research and manufacturing outfits specializing in hard-to-meet biotech outsourcing needs. Other hot emerging topics include the emergence of an expensive protein characterization miracle worker called quadruple time-of-flight (QTOF) hybrid mass spectrometry that was on every proteomics lab’s wish list, and the trend of big pharma turning to the Internet to rejig its massive sales and marketing campaigns.

Editorial themes from this issue include a look beyond the genome to both demystify and explore the value of the proteome, coverage of what’s hot and what wasn’t in protein science, and understanding the value of biotech careers. Finally, we wrapped up this issue with the next segment in our Canadian biotech blueprint series showcasing Québec.

Vol 2, No 5

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Vol 3, No 1

1999

September/October 2000

Vol 3, No 3

Vol 3, No 5

2000

2000 2000

November/December 1999

March/April 2000

Vol 2, No 6

Vol 3, No 2

July/August 2000

November/December 2000

Closing the 20th century with our Millennium special issue, we tackled such topics as how the Internet is changing the life science industry, xenotransplantation R&D and the genetic consequences of settlement. Our headliner for this issue was one of the most prominent science writers of the 20th century, Dr. Edward O. Wilson, a leading conservationist and two-time Pulitzer Prize winner.

As our publisher, a much younger Terri Pavelic notes in her pub note, June 26th, 2000 would go down in history with the completion of the first rough draft of the entire human genome. Remarkably, the work was completed several years earlier than anticipated. Even today, this historic achievement figures prominently in the way we currently conduct biotechnology research. Coinciding with this milestone achievement and the beginning of the post-genomic era, The top stories from this issue we ran two features on bioinformatics, include Reign of the brain: how the first highlighting it as a key tool for to attract and secure the best hu21st century biotechnology, and the man capital for your organization, second piece spotlighting the National dispelling the “Frankenfood” myth Research Council of Canada’s emergin Agbiotech and understanding ing bioinformatics center in Nova why investing in this space is Scotia. Deviating a bit from the central important. Other topics included, theme, we also had editorial contriburesearching and developing tions on innovations in space-age consumer friendly drug-delivery biomaterials that would revolutionize systems, and capillary array medical and dental care, a policy piece electrophoresis (CAE) instruments on regulatory developments that relax setting new benchmarks for acthe burden on biotech companies raiscuracy and throughput in DNA ing capital, and a regional spotlight on sequencing.

Alberta biotech.

Vol 3, No 4

Vol 3, No 6

Key topics covered in this issue were genomics, 3D imaging and cancer research in Canada. On the latter, we highlighted some of the leading Canadian oncology biotech’s in business at the time. Next, our editorial advisory board and the Ottawa Life Sciences Council compiled their ones to watch list of top Canadian biotech firms on the national scene — a notable inclusion still in operation today was ProMetic Life Sciences. In our Across Canada section, Canada’s bright light dream is realized with the news that the Canadian Light Source (CLS) would be completed by January 2004. Also, sadly on October 4, 2000, Canada’s biotech community lost a legend with the passing of Nobel Laureate Dr. Michael Smith. In her pub note Terri Pavelic discussed his passing as well as the importance of his research and its impact on the field of protein engineering. His impact is still felt even today, as the Michael Smith Foundation for Health Research (MSFHR) which was created in 2001 by the BC government continues to drive BC’s capacity to develop new treatments and cures.

February/March 2017 BIOTECHNOLOGY FOCUS 17

Special Report | By Carmela De Luca, J.D. Ph.D., Partner at Bereskin & Parr LLP

Protecting Precision Diagnostic Inventions

R

esearch directed to understanding the impact of genetic and environmental variables on disease emergence and progression has led to the identification of patient populations that share molecular features or signatures, laying the foundation for precision medicine. These molecular features and signatures are being exploited and have led to the identification of new targeted treatments, the tailoring of existing treatment options by identifying which patient populations are more likely to respond to particular drug classes or treatment modalities and prognostic indicators that identify patient populations that are more or less likely to progress warranting more or less aggressive intervention. A number of targeted treatments have been approved and even more are being assessed in clinical trials. As a result, the promise of precision medicine of more effective and less toxic or invasive treatments, better individualized prognosis and the potential for disease prevention for a number of cancers and a variety of other diseases seems closer than ever.

Precision diagnostics, which can be understood as the “tools” necessary to identify the populations that can be treated with targeted therapy or who are more likely to respond or share a prognosis, is a necessary prerequisite for precision medicine and relies on identifying biological markers or signatures including genomic, other “omic” and phenotypic biological markers or signatures (collectively which can be referred to as “biomarkers”), that identify the populations of patients that can benefit from precision medicine.

Considerable discovery research and in particular development research dollars are necessary to identify and validate biomarkers requiring adequate market incentives to encourage market participation.

18 BIOTECHNOLOGY FOCUS February/March 2017

There are a number of obstacles that may limit the pace of commercialization of precision biomarker based diagnostic advances.

Reimbursement and regulatory challenges have been identified as bottleneck issues for the introduction of new diagnostic innovations. In Canada, different provincial regimes and practices for approving and reimbursing diagnostic tests adds a further layer of complexity.

In addition, recent changes in the practice of examining diagnostic innovations before the Canadian and United States patent offices and recent legal changes to US patent law, have added some uncertainty as to the patent protection available requiring deeper consideration of the strategies that may be available for protecting precision diagnostics.

It is generally accepted, that intellectual property rights (IPR) are a key influence on private innovative activity in the biotech field and it is widely accepted that one of the pillars for building value in a biotech company is a sound patent strategy and robust patent position for key assets of a company.

Diagnostic tests that include a new reagent such as a new antibody or diagnostic tests that involve a new method or combination of methods are still generally considered patentable subject matter. However, diagnostic tests that use standard technologies to detect or measure a level of a newly correlated biomarker face hurdles in both the Canadian and US patent offices.

A key high growth area for precision diagnostics is companion diagnostics.

For companion diagnostics, the correlation discovery can generate the most valuable intellectual property (IP) (after claims to the therapeutic composition or use thereof) as it can impact exclusivity for both the drug

and the associated diagnostic. Assays and assay reagents can be less valuable as they can usually be designed around with the general exception of platform technologies.

Business models providing diagnostic services can also involve specific patent challenges. For example, it can be more challenging to patent routine methods applied to a newly discovered biomarker correlation or the patents can be more challenging to enforce (i.e., enforcing patents against customers) and this can have an impact on valuation and the barriers for competitors entering the market.

Depending on the type of invention, the jurisdictions of interest and, the business model and the associated value propositions, patentability and infringement considerations in the markets of interest need to be considered. Future freedom to operate risks can be managed to some extent by publishing or filing a patent application. Filing a patent application has the further benefit of allowing for reassessment of the commercial value of the invention. Alternate options such as trade secret protection should also be considered.

Trade secret protection does not prevent others from independently making the invention but particularly where the business model is focused on diagnostic services and where regulatory approval and reporting may be less stringent such as for laboratory developed tests, trade secret protection can prevent others from using or improving upon the invention. If the trade secret is kept secret and not independently made, the owner can benefit from the trade secret indefinitely. Of course, if the trade secret is not guarded, the owner is typically left without any protection.

A combination of the two may be warranted for providing the most robust protection for precision diagnostic inventions.

Innovator

| by Shawn Lawrence

Ottawa’s Spartan Bioscience set to make point-of-care DNA testing a reality

W

e live in interesting times, bearing witness to a convergence of accelerated healthcare technologies that are empowering patients to manage their own personal health. Examples include such innovations like home blood glucose meters, pregnancy tests, and various other pointof-care diagnostics. And now, thanks to the efforts of an Ottawa-based firm, Spartan Bioscience, the field of DNA analysis is about to take an exponential leap forward.

It’s all thanks to the invention of a tiny new device called the Spartan Cube. As Paul Lem, CEO and founder of the company explains, the Spartan Cube is on the verge of pushing the power of DNA analysis farther, faster and closer to the end user than ever before.

Billed as the world’s smallest DNA analyzer, Lem and his team have constructed a device that not only shrinks the DNA testing process, it takes it out of the hands of lab technicians working with large machines and puts it into those of the consumer. At the same time, it has cut the time it takes to produce DNA analysis results from weeks and months, to as few as 30 minutes. It’s small, roughly the size of a coffee cup, and it packs a powerful punch to accurately diagnose a range of infections.

A former medical doctor with a back-

ground in infectious diseases, Lem says his goal was to make DNA testing accessible with the Cube. On this point, he compares the evolution of DNA analysis to that of computer technologies. Specifically, how such technological advances have changed our everyday lives, giving users instant access to information. In much the same way that computer technologies have evolved from cumbersome clunky machines into more personal, faster and portable devices, Lem believes the Cube will do the same for healthcare diagnostics.

He adds that the device has quite a few of the D’s of exponential technology covered.

For example, it’s digitized, disruptive, demonetized, and one day, hopefully he says, it will be fully democratized.

“Because it’s small enough to fit into your hand and extremely portable, you can bring it anywhere.” he says.

He expects that the device when approved, will be in every doctor’s office, pharmacies, and eventually in millions of homes, all at a relatively cheap price.

“With the Cube we’re striving to drive the price of DNA analysis down significantly and make it affordable to everyone,” he adds.

This is the evolution of DNA testing that he envisioned when he first launched Spartan Bioscience in 2005. Back then he was a doctor specializing in medical microbiology.

As Paul Lem, the man behind the invention, and CEO and founder of the company explains, the Spartan Cube is on the verge of pushing the power of DNA analysis farther, faster and closer to the end user than ever before.

February/March 2017 BIOTECHNOLOGY FOCUS 19

Innovator “For the healthcare industry, we’re looking at people who work in hospitals, doctor’s offices and pharmacies.

For the food and water testing industry, we’re hoping to get it into the hands of building managers and restaurant owners.

The ultimate goal is to get this technology to wherever people want it.”

Training to run a diagnostic lab housing mainframe DNA analyzers, he also had a penchant for inventing. He was presented with a problem, one that he believed he could solve.

“We’d see many patients in these labs, and I remember what was so frustrating was that we weren’t able to give patients their results right away,” he recalls. He adds that often patients would have to wait weeks, even months for their results. He felt the anxiety of not knowing and waiting on these results must have been excruciating for these patients. He believed that someone needed to figure out a way to take mainframe DNA analyzers out of the lab and bring them to the bedside. More importantly, he felt that he could be that someone.

“That was the seed that planted the Spartan Cube idea,” he says.

The decision to change careers and become an entrepreneur and inventor was an easy one he recalls. He adds that transitioning was equally about making more of a difference in the world.

“As a doctor, I realized that I was limited to the number of hours I had in my day and the number of patients that I could see. At most maybe, I could see 10 or 20 patients a day. I thought if I could make products like the Spartan Cube, I could scale well beyond this where potentially, I could reach millions of people with these devices or products.”

And yet, there’s a big difference between using such a device and actually inventing 20 BIOTECHNOLOGY FOCUS February/March 2017

one. Likewise, constructing a complex tool like the Spartan Cube required a unique skill set. For most of his peers, it would have been difficult to do so, but not for Lem. An avid tinkerer, Lem says he had always been fascinated by such healthcare tools and wanting to get to the root of how and why they work.

“I would say throughout my undergrad, and even in high school, where I once won a silver medal at a Canada-wide science fair, I have just loved to play with the tools sometimes even building my own.”

According to Lem, everything he has done since leaving medicine has been part of a grand strategy.

“The actual goal is to change the world,” he says.

For Lem, the first significant step on this path was the creation of Spartan’s first generation device, a four-well endpoint PCR instrument called the Spartan DX. This device after years of tinkering eventually evolved into Spartan Bioscience’s first commercial generation Spartan RX platform. The RX, which included a front-end DNA collection plus DNA extraction components all together in one device, was approved by the FDA in 2013 for one test, identifying patients with a mutation of the CYP2C19 gene.

This was important from a pharmacogenetics point of view, Lem explains, as it was used to identify cardiac stent patients that had the mutation and were taking the blood

thinner Plavix®. The reason that identifying this mutation is important, is it interferes with the efficacy of the drug. If you have the gene, taking the drug can also lead to serious complications. Likewise, by pre-identifying such patients, cardiologists can instead prescribe other drugs in Plavix’s place.

That first-generation device was roughly the size of a toaster, impressive of course. But Lem knew he and his team could find a way to make it even smaller. With the invention of the Cube, they have done just that, as well as expanding on its applications.

Explaining how the Cube works, Lem says it takes all the steps of DNA analysis and puts it into one small sample-to-result box. “It is a PCR-based system that fully integrates DNA extraction, and analysis, with the results shown on an easy-to-use interfaced wireless tablet.”

Lem adds that integrating all these steps makes the Cube a game changer.

The process begins with DNA collection, using a swab that comes with the box. After the user collects the right amount of DNA sample, the swab is then inserted into the box where the DNA extraction is automatic and quick. He adds the cells are broken open through polymerase chain reaction (PCR) and the DNA is released. The machine then uses fluorescence to determine what is present in the DNA. “There’s a software algorithm that interprets the PCR result and the spits out the actual result that says, for example, whether some-

Innovator

thing like Strep bacteria is there or it isn’t,” he explains.

Further, the Cube isn’t just for medical applications. Rather, according Lem, because it can do any type of DNA or RNA application, wherever either is present, the device can detect it. This means the device can also be used as a detection tool for certain environmental contaminants on top of its human health applications.

“The way that we think about who can use the device, is that there are three major industry verticals that we want to go after: infectious disease, pharmacogenetic, and finally food and water safety testing.”

Under these verticals, he says the instrument will have tests that can identify Strep A (which is the cause of strep throat) in DNA; it can determine whether a person carries a certain gene tied to the development of Alzheimer’s; and environmentally, it can be used to monitor ventilation systems for Legionella, a bacteria that when inhaled by humans can cause a potentially fatal type of pneumonia called Legionnaires’disease.

Moreover, on the pharmacogenetics front, the company is focusing on the development of two tests – ApoE and the aforementioned CYP2C19. ApoE (apolipoprotein E) genetic mutations increase the risk of developing Alzheimer’s disease, while CYP2C19 mutations affect how people metabolize drugs. And additionally, for food and water safety,, Lem

says the Cube could also be used for detection of other bacteria such as E. Coli, Listeria and Salmonella.

The company also has plans to further expand the testing in the other two verticals as well.

“Our hardware team is always hard at work figuring out how we can eventually approach all diagnosis like the old tricorders on Star Trek,” he says. “You simple insert the sample, press go and away you go.”

Regardless of the vertical, Lem also expects users to come from many different types of industries.

“For the healthcare industry, we’re looking at people who work in hospitals, doctor’s offices and pharmacies. For the food and water testing industry, we’re hoping to get it into the hands of building managers and restaurant owners. The ultimate goal is to get this technology to wherever people want it,” he says.

And as alluded to, into people’s homes.

Already the company’s first generation device, the Spartan RX, is selling around the world, giving the company a revenue stream to further develop the Spartan Cube. The RX has been approved by Health Canada, both European and Asian Regulatory Agencies, and most importantly, it has FDA 510(k) approval. Now the company is focused on getting FDA 510 (k) approval for the Cube.

Lem adds that fortunately, the company has

already learned some valuable lessons from the RX approval pathway that should make the process simpler for the Cube. The company also has a very big player in its corner.

Specifically, Spartan has benefitted from a partnership with consumer product giant Canon Inc., a world leader in the camera and copier market. Through its U.S. arm, Canon BioMedical is helping Spartan in various ways to both develop and market the Cube worldwide. The partnership has also given Spartan access to advanced manufacturing facilities, specifically that scale of manufacturing that makes it possible for Spartan to execute on its vision.

“They have manufacturing expertise at an enormous scale, as well as a massive global network for sales and service,” says Lem. “They also have decades of consumer electronic design expertise, so they know how to design the user interfaces and how to make things easy to use for the average person.”

They also have a huge fleet of technicians at their disposal to service their device.

“I think they have field technicians that are servicing almost every office building in the world because like Canon photocopiers or printers are in every office building, pharmacy and library. So, they already have all these feet on the street which could prove useful if we get the Cube to where we want it to go.”

According to Lem, prior to this partnership, Canon had been developing its own DNA diagnostics division for years, having a very difficult time making a point-of-care device.

They decided to look externally for a more user-ready device.

“The sent their business development people all over the world asking key opinion leaders and top scientists for any up and coming companies, and they kept getting referred back to us, and that’s how they found us,” he says.

Lem says it has meant a lot to have such a major player to lean on, and he expects the two sides now are very close to achieving their mutual goals. Likewise, Lem sees a very bright future for the device and for the future Spartan devices to come “It’s amazing to think as little as two decades ago, machines like the Cube took up so much space, even fillingoffices. And here we are today – we’ve built the world’s smallest DNA analyzer and we’re on the verge of making it accessible to millions.”

To see this story online visit www.biotechnologyfocus.ca/spartanbioscience-set-to-make-point-of-care-dnatesting-a-reality/ February/March 2017 BIOTECHNOLOGY FOCUS 21

Oncology

| By Dr. John Bell

Combining strengths to fight cancer

BioCanRx supports the translation of key immunotherapy discoveries to the clinic

I

t’s been nearly 50 years since U.S. President Richard Nixon declared war on cancer. Since then, some battles have been won, others lost. But the traditional weaponry – chemotherapy and radiation – while refined and improved, is still carrying the brunt of battle duty.

In recent years, these two warhorses have been joined by a powerful new weapon. The most promising new approach to cancer to emerge, immunotherapy enlists the patients’ own immune systems to fight their cancer.

It’s an exciting field, with new therapeutic approaches being rapidly developed.

BioCanRx was created to build on these discoveries and approaches, to help them reach the clinic, where they can save lives as quickly as possible. In Toronto, for instance, Dr. Pamela Ohashi of the Princess Margaret Cancer Centre has developed a novel method of taking tumour-infiltrating lymphocytes (TILs), a 22 BIOTECHNOLOGY FOCUS February/March 2017

form of T-cells, from patients, growing them in the lab with dendritic cells, another kind of immune cell, and returning them to the patient, where they can attack ovarian cancer tumours. Now, the world’s first clinical trial to use this type of adoptive cell therapy, as it is known, for ovarian cancer is giving women who are no longer responsive to conventional treatment another option.

But there still remain challenges to the widespread implementation of immunotherapy. It is now clear that even within a single patient, cancer is a genetically complex and heterogeneous disease. Its ability to evolve during therapy coupled with its propensity to shroud itself with an immunosuppressive cloak makes metastatic or widespread cancers essentially incurable with the current therapies available to the oncologist. However, our immune systems have the “potential” to evolve in lock-step with the cancer and rec-

ognize novel tumor specific antigens as they appear during progression from a normal cell to a malignant cancer. Strategies need to be developed to exploit the ability of our immune systems to recognize foreign cancer antigens while at the same time finding ways to overcome the barriers that tumours set up to block immune cell activity.

Scientists within the BioCanRx Network believe that it is not sufficient to attack each of these barriers on its own. Our approach is to develop novel combination strategies creating a multi-faceted attack on cancer to enable immune recognition and destruction of tumours wherever they hide in the body.

That’s why, today, we are focusing our funding on exactly that – research into combination therapies that together are stronger than the sum of their parts. For example: • Dr. Julian Lum and his colleagues at the BC Cancer Agency are combining radioactive

Oncology

Dr. Julian Lum

Dr. Marcus Butler

Dr. Yonghong Wan

Dr. Jonathan Bramson

Dr. Raja Ghosh

leading a multidisciplinary team in developing a template for evaluating future therapeutics and designing clinical trials that are feasible, safe, effective and economical. The team will address issues that most first-in-human/early phase trials encounter and offer Dr. Manoj Lalu Dr. Dean Fergusson Dr. Rob Holt Dr. Pamela Ohashi a structured method to evaluate available data and design an evidence-informed trial protocol, cess. The development of new therapies as drugs with checkpoint inhibitors so that the beginning with CAR-T cell therapy for single agents in a “siloed” approach is slow, immune response generated by the radiablood cancers. expensive, ineffective and unlikely to make tion can better infiltrate the cancer tumour. the rapid progress cancer patients need. We • Dr. Rob Holt is working with a team at the BC Their work could provide a new treatment Cancer Agency and Ottawa Hospital Research at BioCanRx are investing our funds to make option for metastatic prostate cancer that Institute to build the manufacturing capacity to these combinations happen sooner. has become resistant to hormone therapy. enable CAR-T therapy in Canada. This will bring The other thing we’ve learned is that we • Dr. Marcus Butler of the Princess Margaret this necessary, effective and highly publicized need a better way to select those patients Cancer Centre is conducting the world’s first therapy to cancer patients for whom conventional who will benefit most from these promisclinical trial combining an oncolytic vaccine treatment has not worked and who are in dire ing, but expensive, therapies. The pace of with checkpoint inhibitor antibodies for canneed of a new approach. research advances today is so quick that we cer treatment. The vaccine he is using was are missing opportunities to capture the data developed in Canada and its testing remains that results, in particular, from clinical trials. The result of our investments is the rapid exclusive to Canada, as, likely, will its comBioCanRx is capturing that data and using it testing and introduction into the clinic of mercialization. The trial will involve patients to learn how to best identify those patients combination therapies targeted to the patients with advanced solid tumours who already who will benefit from specific immuno- who will benefit most from them, so that canshow an anti-cancer immune response and therapy approaches. This helps to speed up cer becomes part of their past and not their have not responded to conventional therathe product development process and ensure present. In doing so, we are contributing to a pies. The combination of oncolytic vaccines that decision-making is strongly rooted in the huge improvement in quality of life of people with checkpoint inhibitors is one of the most with cancer, lowering healthcare costs by best possible evidence. exciting prospects in oncology, according to With our funding, we are also helping to delivering the most effective therapy first and Dr. Stephen Russell of the Mayo Clinic and a develop the foundations and infrastructure to reducing the social and economic toll of this member of the BioCanRx Research Managetoo-often fatal disease. support future immunotherapy discoveries: ment Committee.

• Drs. Jonathan Bramson and Raja Ghosh • Dr. Yonghong Wan of McMaster University from McMaster University are develop- Dr. John Bell is Sr. Scientist, Centre for is also using an oncolytic vaccine, this time ing a “bioreactor in a box” to enable the Innovative Cancer Research, Ottawa with adoptive cell therapy, to target cancers production of T-cells for immunotherapy Hospital Research Institute; Professor, Dealready showing an immune response. This in a closed system that more closely mim- partments of Medicine and Biochemistry, exciting combination of therapies has a ics the body’s production of T-cells, while Microbiology & Immunology, University of clear mechanism for working together to reducing the costs, lab space and human Ottawa and Scientific Director, BioCanRx kill cancer cells. resources required for current methods - Biotherapeutics for Cancer Treatment. of producing T-cells for immunotherapy.

This is a key example of the critical role that Their work will ensure that the high cost BioCanRx is playing in the immunotherapy of producing the needed immune-system field. Pharmaceutical companies, very justifiTo see this story online visit cells is no longer a barrier for providing ably, are focused on developing their own www.biotechnologyfocus.ca/combiningimmunotherapy in the clinic. agents. Combining these agents with those strengths-to-fight-cancer-biocanrx-supports• Drs. Manoj Lalu and Dean Fergusson of of other companies, if it happens at all, the-translation-of-key-immunotherapy-disthe Ottawa Hospital Research Institute are generally happens quite a bit later in the procoveries-to-the-clinic/ February/March 2017 BIOTECHNOLOGY FOCUS 23

across canada

| By Kaitlyn Proulx and W. Neil Palmer

Reimbursement & Market Access for Innovative

Canadian Biotechnologies

R

eimbursement and market access are critical milestones for commercial success of new biotechnologies, and planning for reimbursement should begin long before regulatory approval. The road to reimbursement is marked by many challenges, not least of which is demonstrating value in terms of both clinical and cost effectiveness. No longer is it sufficient to demonstrate only efficacy, safety and quality (the requirements for regulatory approval). Payers (public and private) now insist on clear evidence of clinical and economic value confirmed through rigorous health technology assessments (HTAs). And budget impact is an additional factor that can limit access even for drugs (eg. Sovaldi (sofosbuvir) for hepatitis C) that have clearly demonstrated both clinical and cost effectiveness.

Health Technology Assessments (HTAs) Formal HTA reviews are a requirement in most developed countries and Canada has been a leader developing HTA standards and procedures. The Canadian Agency for Drugs and Technology in Health (CADTH)1 and the Institut national d’excellence en santé et en services sociaux (INESSS)2 in Québec conduct assessments of all new prescription drugs and biologics and recommend whether the new technologies should be reimbursed by the federal, provincial and territorial drug benefit plans. Not only are these recommendations critical for public reimbursement, they are increasingly important for private reimbursement as well.

Although the formal CADTH / INESSS HTA review processes commence just before or after market authorization (Notice of Compliance or “NOC” from Health Canada), planning for the HTA review should begin well before and often concurrently with Phase 2 clinical trials, if not earlier. This timing is important because the Phase 3 trials need to be developed with HTA, reimbursement and market access in mind. Trial design (double-blind random24 BIOTECHNOLOGY FOCUS February/March 2017

ized controlled trial is the gold standard), selection of comparators, primary and secondary outcomes, patient sub-populations, and quality of life measures are important elements that should be considered in the Phase 3 clinical trial program.

For most new drugs and biotechnologies, manufacturers face considerable challenges when it comes to developing and conducting clinical trial programs. For rare (orphan) diseases the barrier is small patient populations that make it difficult to recruit sufficient patients to power statistically significant differences in clinically meaningful outcomes.

Moreover, the outcomes may only be surrogate markers and not the clinically relevant outcomes preferred by payers.

Case Study: PCSK9s For example, approval of the recently launched PCSK9s3 (ie, Praluent (alirocumab), Repatha (evolocumab)), a new class of lipidlowering medications for the treatment of

familial hypercholesterolemia, relied primarily on reduction of low-density lipoprotein cholesterol (LDL-C) as the primary outcome.

LDL-C is a laboratory measure that often serves as a surrogate marker for cardiovascular events. However, HTA agencies would prefer to see changes in morbidity (e.g., cardiovascular events) and mortality as the primary outcomes in the clinical trials. However, this would have been difficult for the PCSK9s given the small patient population and the extended time horizon necessary to record sufficient morbidity and mortality events to be clinically and statistically meaningful.

Ultimately, HTA agencies accept surrogate markers as proxies for outcomes as long as there is sufficient evidence demonstrating a clear correlation between the surrogate marker and the outcome. Ideally there will be follow-on or extension trials that will help confirm that the new technology truly offers benefits in morbidity and mortality.

across canada It is important to note that manufacturer – payer relationships are not about becoming “partners” or “friends”, but rather they are about manufacturer representatives establishing credibility with the payers.

Early Scientific Advice Leading pharma and biotechnology companies that recognize the challenges in developing clinical trials that are relevant to both regulators and HTA agencies will often seek early scientific advice from HTA agencies (sometimes in conjunction with regulatory agencies). This advice, while non-binding and offered on a fee-for-service basis, can provide valuable insights and suggestions for developing a clinical trial program that address both the requirements of the regulator and the evidence expectations of the HTA agency and payer.

The National Institute for Health and Care Excellence (NICE)4 in the UK was the first HTA agency to develop a scientific advice service, and this type of service is now offered in several countries including by CADTH in Canada which modelled its program on NICE.

Payer Engagement Early scientific advice is but one element of the pre-market activities that are critical to market access success. Payer engagement is important throughout the product life cycle and as such it is about the continuing relationship between the manufacturer and the national and regional decision makers. The objective of payer engagement is to establish professional relationships between manufacturers and payers that maintain clear lines of communication, foster understanding of each others organization and over time develop trust and credibility. Larger pharma companies maintain regional staff that are tasked with maintaining relationships locally whereas smaller firms will have a national person in a senior role with responsibilities for establishing and maintaining relationships with payers and HTA agencies at all levels.

It is important to note that manufacturer – payer relationships are not about becoming “partners” or “friends”, but rather they are about manufacturer representatives establishing credibility with the payers. These relationships will pay dividends when it comes to negotiating risk sharing agreements typically referred to as product listing agreements or “PLAs” negotiated with public payers through the pan Canadian Pharmaceutical Alliance (pCPA)5 process. And this is not because longstanding relationships result in better deals per se, but rather because trust and credibility foster frank and honest negotiations. It

is no surprise therefore that the most difficult and protracted negotiations are those led by foreign-based global pricing teams or their equally foreign consultants. Foreign-based global pricing teams are a fixture with large pharma companies, however, the best firms empower their Canadian affiliates (and local consultants) to lead negotiations.

Price Regulation, pCPA and PMPRB The PLAs established through the pCPA process establish (usually) confidential net prices however this is not the only mechanism for limiting prices in Canada. The Patented Medicine Prices Review Board (PMPRB)6 has a mandate to ensure prices of patented medicines in Canada are not excessive. The PMPRB applies to both domestic therapeutic class price comparison and to international price comparisons to limit introductory prices of new patented medicines. Post introduction, prices cannot increase faster than inflation as measured by the consumer price index (CPI) and may never exceed the highest international price among the PMPRB seven reference countries.

The PMPRB and provincial payers (on their own or through pCPA) work independently of each other such that an agreeable price to one may appear excessive to the other.

And the confidential rebates paid by manufacturers to provincial payers are typically not included the calculation of the average transaction price that is regulated by the PMPRB. The PMPRB is currently engaged in a comprehensive review of its mandate with an objective of becoming more relevant to payers. New, tougher PMPRB price guidelines are expected in 2018. Meanwhile, the pCPA process continues to evolve. Taken together, it is expected that there will be greater collaboration between PMPRB and pCPA and perhaps even coordination or harmonization at certain levels.

Market Access is Complex and Evolving While the specific pricing, reimbursement and market access mechanisms described above are unique to Canada, complexity is not.

Each country has its own complex systems of national and regional HTA, pricing and reimbursement mechanisms reflecting the underlying health care systems they serve.

Although there are many similarities between Canada and other countries in terms of reliance on evidence-based methodologies for assessment, implementation and decision making processes are unique to each country.

Market access is at best a challenging endeavour in Canada (and globally!) that is ever evolving with new regulations, policies and methodologies employed by payers and regulators to assess value. Investors and analysts have become more sophisticated when it comes to market access – no longer are they looking only at regulatory approval but more importantly they need to be convinced that timely reimbursement is both feasible and probable.

Start Early!

Above all else it is critical to start early. Too often, small emerging biotechs find out the hard way (and too late) that convincing payers (public and private) to pay for innovative new technologies is a significant challenge.

Big Pharma recognized some time ago that regulatory approval and market authorization are only the first step of a long journey and that it is essential that there be careful planning and sufficient resourcing to support strategic planning, model development, dossier preparation and payer engagement.

About the Authors Kaitlyn Proulx (Kaitlyn.Proulx@pdci.ca) is PDCI’s Managing Director with responsibility for managing the firm and leading the team of twenty senior consultants and support staff.

W. Neil Palmer (Neil.Palmer@pdci.ca) founded PDCI in 1996 and serves as principal consultant on many of PDCI’s pricing and reimbursement engagements.

About PDCI Market Access Inc. PDCI is Canada’s leading pricing and reimbursement consultancy featuring senior consultants with clinical, health economic, HTA, pricing and strategic reimbursement expertise. (www.pdci.ca)

References 1 2.

3.

4.

5.

https://cadth.ca/ http://www.inesss.qc.ca/ Proprotein convertase subtilisin/kexin type 9 https://www.nice.org.uk/ http://www.pmprovincesterritoires.ca/en/ initiatives/358-pan-canadian-pharmaceutical-alliance 6. http://www.pmprb-cepmb.gc.ca/home

To see this story online visit www.biotechnologyfocus.ca/reimbursement-market-access-for-innovative-canadian-biotechnologies/ February/March 2017 BIOTECHNOLOGY FOCUS 25

Reimbursement

| By Chris Chapman

The lay of the land

for Canadian Government Grants & Incentives

T

o remain competitive globally it is imperative for companies to continue to innovate and grow. For the Canadian life sciences industry, the Globe and Mail reported in April of 20151 that the biotech sector is “ready and eager” for growth but without adequate financing, it will go nowhere. Canada does offer a multitude of government grants and incentives to promote innovation and growth for Canadian businesses.

The federal government as well as all provincial governments offer support in the form of non-repayable grants, favourable loans, and tax credits, with Ontario and Québec having the largest number of programs available. While the other provinces have fewer program offerings, new programs are announced on a monthly basis in an effort to support their regional priorities and compete with the other provinces.

Regardless of the geographic region in the country, government incentive programs are available to companies operating in various industries, including life sciences. The programs available can be broadly grouped into four categories relating to the initiatives they support: 1. Business and Market Expansion 2. Innovation Development 3. People Development 4. Environmental Sustainability Programs in the Business and Market Expansion category generally support projects that include facility expansions, new product line expansions and expansion to new markets.

For Ontario businesses, projects that involve expanding innovation capacity, improving productivity, performance and competitiveness and are proposed be in excess of $10 million in expenditures could be eligible for up to a 20 per cent non-repayable grant contribution. For similar projects that are expected to be less than $10 million, they could be eligible for a 15 per cent non-repayable grant to a maximum of $1.5million if project costs exceed $500,000.

Program offerings in the Innovation Development category generally support all industries, including life science-based projects that require significant R&D investment for new product and technology development; acquisition of new equipment and technology to improve productivity; as well as industry/ university collaboration initiatives.

26 BIOTECHNOLOGY FOCUS February/March 2017

Canada’s longstanding flagship Scientific Research & Experimental Development (SR&ED) program provides up to a 35 per cent federal credit for SR&ED eligible labour, materials and subcontractor expenditures. In addition to SR&ED, federal programs such as the National Research Council’s (NRC) Industry Research Assistance Program (IRAP) provide R&D project-based non-repayable funding up to $500,000 per project.

The Ontario Centres of Excellence (OCE) AdvancingHealth2 program is designed to bolster innovation in Ontario’s public healthcare sector by matching healthcare needs with innovative products and services through partnerships between public healthcare organizations, companies and academic institutions.

The aim of the program is to advance healthcare innovation directed to improved health outcomes, enhanced patient experience and efficient use of resources through investments in collaborative demonstration projects that show clear potential for scaling-up to the system-level and establish a strong case for adoption. Up to $250,000 is available from OCE per demonstration project and applicants can also access two academic vouchers per demonstration project (valued at $50,000) each to support academic researchers and students to work on the demonstration project and/or to assist with adoption.

In many provinces, both federal and provincial assistance is available to help Canadian companies hire and retain staff, improve their effectiveness and train their employees in areas such as advanced technologies or operational processes. Specific programs support initiatives towards hiring of recent university/college graduates; training programs for upskilling staff; training on new technology or equipment installations; and supporting the temporary hiring of co-ops students and interns. Companies looking to hire recent university graduates from science, technology, engineering, and math disciplines can access

funding to subsidize 50 per cent of the first year salary for a new hire.

Support for climate change initiatives and technology development projects has also increased in recent years. Grants are available for projects aimed at greenhouse gas reductions, energy conservation, and water conservation and usage reduction. These programs can improve the ROI for a company’s sustainability strategies, including renewable energy investments, energy efficiency improvements, recycling initiatives, advanced manufacturing and green building construction.

Over the past five years, there has been a focused effort by the federal and provincial governments to deliver grants and incentives to Canadian businesses through direct funding mechanisms (i.e., non-repayable cash contributions and favourable loan programs) over indirect funding mechanisms such as tax credits.

While these programs have defined eligibility criteria, the majority are discretionary and limited to budget availability. The discretionary nature of the majority of direct funding programs introduces a high level of uncertainty to companies who would otherwise be eligible.

In addition, businesses are required to submit their application and receive funding approval before commencing the project. This requires companies to plan ahead and develop a funding strategy to be successful.

Regardless of whether programs are statutory or discretionary, with proper planning and advice, there is significant opportunity for Canadian life science businesses to receive government funding for their upcoming projects.

About the Author Chris Chipman is the Canadian Incentives Leader at EY. He can be reached by email at chris.chipman@ca.ey.com.

References 1. Reguly, Eric, “Why is Canada’s life sciences sector flatlining?,” The Globe and Mail, 23 Apr 2015. http://www.theglobeandmail. com/report-on-business/rob-magazine/ why-is-canadas-life-sciences-sectorflatlining/article24030375/ 2. http://www.oce-ontario.org/programs/commercialization-programs/advancinghealth

To see this story online visit www.biotechnologyfocus.ca/the-layof-the-land-for-canadian-governmentgrants-and-incentives/

new PRODUCTS

Spectrometry IRsweep announces the release of the IRspectrometer, a tabletop frequency dual-comb spectrometer

offering microsecond time resolution, large spectral bandwidth and high spectral resolution. The IRspectrometer can be used in a variety of applications, including biological sample and chemical composition analysis. The device excels where fast time resolution or high throughput and superior brightness is required — including applications that involve a complex background matrix or where multiple similar molecules must be simultaneously quantified. Unique features of the IRspectrometer include: fast time resolution (as low as 1 µs for a full spectrum), the ability to monitor highthroughput applications without relying on techniques like step-scan in FT-IRs and brightness advantages that significantly ease sample preparation for liquid and solid analytes. The device also delivers microsecond resolution at high bandwidths, with center wavelengths between 6 µm (1700 cm-1) and 9.5 µm (1050 cm-1), covering fundamental amid absorption bands, are available.

ZEUS LT also incorporates Hamilton’s intelligent air displacement pipetting technology Qualitative Pipette Monitoring™ (QPM) for monitoring clogged tips or foam aspiration, Anti-Droplet Control (ADC™) for pipetting of volatile liquids, capacitive and pressure based level detection, tip detection and liquid class definitions. Easy mounting hardware enables attachment to an existing XYZ system, or development of an application-specific z-axis to accommodate space constraints or other design considerations.

Simple RS-232 or CAN commands combine with pressure and capacitance outputs for simple coordination of the pipette channel and the z-axis for liquid level detection, pipette tip following, and other coordinated movements.

Web: www.irvinesci.com Pipettes The Eppendorf epMotion 96 is an easy to use bench top system for high precision pipetting in 96- and 384-

Web: www.hamiltoncompany.com Cell Culture Irvine Scientific, a provider of cell culture media, introduces PRIME-XV® T Cell CDM, a chemically-defined, animal component-free medium for T cell culture.

Web: http://irsweep.com Pipettes Hamilton Company has expanded its pipetting family with the introduction of the ZEUS LT OEM pipetting module. Like the original ZEUS® module, the ZEUS LT is an ideal complement to analytical instruments and clinical diagnostic systems. Unlike the original, the ZEUS LT does not come with an integrated z-axis, but rather offers the option for integrators to custom design the z-axis.

More than a simple pipette module, the

growth, phenotype and the potential of T cells to polarize into therapeutic subtypes.

PRIME-XV T Cell CDM removes this variability to provide more consistency between lots. Chemically-defined media also reduces the risk of introducing foreign agents or impurities from undefined components, thereby facilitating scale-up to commercial production and the regulatory submission process.

The new medium has been developed to maximize consistent growth of T cells while maintaining their functionality and therapeutic potential. An increasingly important trend in cell culture media for gene therapies and immunotherapies is the move away from animal-derived and undefined components to serum and animal component-free, chemically-defined culture conditions. When working with T cells the advantage of this is two-fold: animal-derived components are variable between lots; and the naturally occurring cytokines and growth factors in them can result in undesirable effects. For example, cytokines and growth factors have been shown to impact

well or deep-well plates within life science research, pharmaceutical and industry laboratories. Overcoming the limits of manual multichannel pipetting, the device allows easy, rapid handling with high precision and accuracy compared to multi-channel manual pipettes. Thanks to its ability to dispense into all 96 wells at the same time, users can simultaneously start or stop a biological assay for all wells in a plate. Additionally, the epMotion 96 has a large volume range between 0.5 and 300µL using only one head or system so there is no need to switch pipette heads or employ a second device to achieve all volumes. Workflows benefiting from the epMotion 96 include but are not limited to: solid phase extraction, DNA extractions from blood and tissue, sequencing and PCR clean-up, total RNA isolation, NGS library preparation, distributing reagents and serial dilutions, media change, other cell culture applications and more.

Web: www.eppendorf.com February/March 2017 BIOTECHNOLOGY FOCUS 27

new PRODUCTS

Liquid Handling The BrandTech® Scientific Liquid Handling Station (LHS) pipetting robot reduces repetitive manual pipet-

ting and increases laboratory efficiency and reproducibility through automation. The LHS is an extremely compact benchtop instrument that has seven working positions and five available liquid ends in single channel volumes up to 1000µL and multichannel volumes up to 300µL. With a wide variety of accessories, this flexible system moves liquids between reservoirs, tubes and plates; and is ideal for plate reformatting, ELISA and PCR setup, cherry picking, etc.

the chassis and attacking the electronics or stirring mechanism. These units feature 10-program memory with 10 steps per program, temperature ramping, RS232 I/O port, membrane keyboard, and full function liquid crystal display where all parameters are continuously visible. Heater tops are 8″ square solid ceramic with 600 watts of power. Temperatures can be set to 450°C. The units are readable and settable to 1°C. Temperature control is by PID software and is controlled to ±1°C or ±1ºF. Stirrer speeds can be set from 100 to 1500 rpm. Temperature ramping can be set from 1ºC/hour to 450ºC/hour in 1ºC increments. The built-in timer is settable to 99 hours and is readable to 1 second. All units are available in 100, 115, and 230 VAC, 50/60 Hz models.

Web: www.torreypinesscientific. com Mixers MilliporeSigma’s Mobius® Power MIX single-use mixing systems are engineered with advanced technology to

plying CCD (EMCCD) camera platform for fluorescence microscopy. The iXon Life is available in 1024 x 1024 and 512 x 512 sensor formats, back-illuminated to deliver the highest and broadest QE of any microscopy camera and deep cooled down to -80°C for minimal darkcurrent.

Web: www.andor.com/ixon Mass Spectrometry Mass Spec Analytical is now shipping its Thermal Extraction Ion Source Sciex™ 3200 Triple Quadrupole Mass Spectrometer and other V-Series models to customers. The TEIS-3200 is ideal for applications where the method requires

Web: www.brandtech.com Hot Plates Torrey Pines Scientific introduces its EchoTherm™ Programmable Corrosion Resistant Digital Stirring Hot Plates for use with aggressive chemicals in environments where other stirring hot plates would be quickly destroyed by vapors or spills. The hot plates are designed to be purged using an inert gas through a fitting on the rear of the chassis. Purging provides a positive pressure inside the unit to prevent corrosive gasses from entering effectively handle difficult-to-mix buffers, culture media powders and other pharmaceutical ingredients. The system achieves a strong vortex using an impeller design and motor based on magnetically-coupled NovAseptic® technology-a proven mixing technology in stainless steel tanks. Sterile zero deadleg sampling can be performed directly from the mixing container. A probe port allows for insertion of either a reusable probe for non-aseptic processes or a presterilized, single-use sensor for in-process pH measurement of aseptic processes. The 100, 200 and 500 litre models are available in both jacketed stainless steel and highdensity polyethylene HDPE.

Web: emdmillipore.com/powermix 28 BIOTECHNOLOGY FOCUS February/March 2017

Imaging Solutions Andor Technology (Andor), an Oxford Instruments company, has released its iXon Life Electron Multi-

a large quantity of sample measurements in a relatively short period of time or where automation is required. When coupled with Mass Spec Analytical’s analysis software, results can be provided in as little as 4 seconds. The TEIS-3200 uses a patented thermal extraction sample introduction device. Compounds amenable to thermal desorption, such as many pesticides, drugs and explosives, can be rapidly analysed in this way without the need for any sample pre-treatment, solvents or glassware.

Web: www.msaltd.co.uk

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BC Tech Summit LABVOLUTION & BIOTECHNICA Venue: Vancouver, BC Venue: Hanover, Germany Hashtag: #BCTECH Summit new card:Layout 1 1/31/2013 Tel: 9:09416 AM598-7114 Page 1 Email: info@bctechsummit.ca Fax: 416 598-1840 Web: http://bctechsummit.ca/ Email: info@hf-canada.com March 20-22 Web: http://www.labvolution.de/home Web: http://www.biotechnica.de BIO Europe Spring Venue : Barcelona, Spain Web : https://ebdgroup.knect365.com/ bioeurope-spring/

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Bio International Conference Venue: San Diego, CA Email: convention@bio.org Web: http://convention.bio.org/2017/

June 26-27 WIN 2017 Symposium : Expediting Global Innovation in Precision Cancer Medicine Venue: Paris, France Email: vladimir.lazar@winconsortium.org Web: www.winsymposium.org

May 26-28 Labcon 2017 Venue : Banff, AB Web: https://labcon.csmls.org/

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APRIL 2017 April 3-7 Toronto Health Innovation Week Venue : Toronto, ON Email : info@tohealth.ca Web : http://healthinnovationweek.ca/

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LAST word

| By Andrew Casey, President & CEO of BIOTECanada

The 2017 Canadian Biotech Imperative: Remaining Competitive in the Face of Uncertainty 2016 was certainly an active year for the Canadian biotech ecosystem as several early stage Canadian companies including Northern Biologics, Zymeworks, Highland Therapeutics, Cynapsus and Innovative Targeting Solutions all hit milestones and enjoyed significant growth.

Moreover, the federal Venture Capital Action Plan investment dollars began to flow into the industry through investments by Teralys, Lumira and CTI Lifesciences. And as 2016 came to a close, Versant Ventures and Bayer AG partnered to launch stem cell focused BlueRock Therapeutics. The momentum was carried over into 2017 as Versant closed its $400M USD healthcare VI fund with Teralys as an LP, and Zymeworks announced more growth with the addition of a new 10,000 square foot lab facility in Vancouver, BC. Commercially, these are all very positive developments which generated considerable buzz at the recent BIOTECanada Investor Summit in Whistler.

From a public policy perspective, the recently released federal Economic Advisory Council on Economic Growth’s report to the Minister of Finance put forward some important conclusions and recommendations in relation to Canada’s biotech sector. Established in March 2016, the Council was mandated to identify policy actions to support the conditions for long-term economic growth.

Importantly, the Report highlighted biotech-heavy sectors, life sciences and agfood, as two of the four “high potential sectors in terms of their economic prospects and their strengths relative to global opportunities.” The Report also provided recommendations on how to move these sectors from just ‘potential’ to ‘commercial reality’, highlighting the importance of developing measures to attract investment capital and talent. It is anticipated that the upcoming Federal Budget (now expected in early March) and the soon to be released Federal Innovation Agenda will begin the process of putting in place a strategy 30 BIOTECHNOLOGY FOCUS February/March 2017

and corresponding measures to support the development and commercialization of life sciences and ag innovation.

Yet, while the Canadian industry continues to grow and the government’s policy agenda looks to support that growth, macro economic and political events outside Canada stand to re-define the global industry’s operating environment. In particular, the dramatically changed and unpredictable U.S. political landscape is generating more questions than answers at this point. Questions like what will be the impact of a ‘tweaked’ NAFTA, immigration policy, and ‘buy America’ policy approach be for Canada? How will the Trump administration balance the objectives of supporting biotech innovation while simultaneously trying to reduce drug prices? What will the replacement plan for Obamacare look like and how will it impact the pharmaceutical industry? Depending on the answers, there may be some silver linings for Canada- immigration policies might make it easier to attract and retain top talent and other economic measures and approaches could result in capital migration in our direction.

At a minimum, the shifting sands underscore the importance of Canada ensuring it is globally competitive. The federal Budget and corresponding Innovation Agenda will be important steps towards driving biotech innovation forward. With the right hosting conditions and strategies in place, Canada’s vibrant biotech sector can build on the momentum and success of 2016 to establish itself as a destination of choice for both investors and talent.

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