Biotechnology Focus October/November by Promotive Communications

INSIGHTS FOR THE LIFE SCIENCE INDUSTRY

AG-WEST BIO 25 YEARS

OF GROWING BIOSCIENCES INSIDE: JONATHAN GOODMAN AND HIS LATEST BIOPHARMACEUTICAL ENDEAVOUR: KNIGHT THERAPEUTICS

Publication Mail Registration Number: 40052410

OCTOBER/NOVEMBER 2014 VOLUME 17, NUMBER 5

ABIC2014-BiotechFocusOct-FullPage-2.pdf 1 23/09/2014 3:03:39 PM

CMY

contents OCTOBER/NOVEMBER 2014 – VOLUME 17 – NUMBER 5

Ag-West Bio: 25 years of growing biosciences

Funding Clinical Trials: Challenges for Small Biotechs

FEATURES PRECISION MEDICINE: PATENT AND REGULATORY FRAMEWORKS IN CANADA A brief review of Canadian specific scenerios that influence the development of personalized medicine technologies (By Carmela DeLuca and Amy Dam)

Opinion

AG-WEST BIO: 25 YEARS OF GROWING BIOSCIENCE Championing Saskatewan’s ag biotech sector for two and a half decades (By Ag-West Bio)

FUNDING CLINICAL TRIALS Cynapsus Therapeutics CEO Anthony Giovinazzo discusses the challenges of funding clinical trials specific to smaller biotech companies (By Anthony Giovinazzo)

LOOKING TO REPEAT THE FEAT With the sale of Paladin Labs Inc. to the highest bidder in the rear view mirror, Jonathan Goodman believes he can strike gold again with his new company Knight Therapeutics (By Shawn Lawrence)

DEPARTMENTS 6 9 26 28

IN EVERY ISSUE

RESEARCH NEWS BUSINESS CORNER NEW PRODUCTS CALENDAR OF EVENTS

Q&A with Ag-West Bio director of commercialization Brad Bly (Compiled by Biotechnology Focus with files from Ag-West Bio)

24 RESEARCH NEWS

8 www.biotechnologyfocus.ca

A QUARTER CENTURY OF SUPPORTING COMMERCIALIZATION

Partnership creates research laboratory for translational cancer research

ACROSS CANADA Highlights from the business of regenerative medicine course (By Mark Curtis)

THE LAST WORD The Bioeconomy and sustainability: Role of cellulosic biomass (By Murray McLaughlin)

October/November 2014 BIOTECHNOLOGY FOCUS 3

PUBLISHER’S NOTE

PUBLISHER/ EDITOR-IN-CHIEF SENIOR WRITER CONTRIBUTING WRITERS

Lots to see in Saskatoon

NATIONAL ACCOUNT MANAGER GRAPHIC DESIGNER CONTROLLER

Collaboration and co-operation among academic, government and industry partners is at the core of Saskatchewan’s biotech industry. It starts in the City of Saskatoon, where on a bluff high above the South Saskatchewan River sits the 80-acre University of Saskatchewan Innovation Place. With such leading centres as the Bio Processing Centre, the Vaccine and Infectious Disease Organization (VIDO), the International Vaccine Centre (InterVac) and of course the Canadian Light Source synchrotron, you’ll also find a healthy mix of great scientific research institutes and cutting edge bioscience companies. If you’ve ever been, you would know that it’s an impressive sight to behold. And if you haven’t been, we highly recommend going. It all makes for a great backdrop to what marks the 14th edition of the world’s premier ag-bio event, the Agricultural Biotechnology International Conference (ABIC) being held October 5 to 8. It’s fitting that a city and a province with so much going for it, as the epicentre of Canada’s ag-biotech industry and as a booming town for biotech is hosting this event once more. Keeping with this theme, we’ve tried to tie in ABIC’s homecoming in this issue by spotlighting the organization responsible for launching this event in our cover story. It’s quite surprising to realize this year marks the 25th anniversary of Ag-West Bio Inc.’s launch. It certainly has come a long way in its efforts to promote and support the growth of Saskatchewan’s agricultural bio-industry. Other highlights of this issue include Carmela DeLuca’s and Amy Dam’s article on the challenges of determing patent and regulatory frameworks in Canada for personalized medicine. We’ve also included a best practices for funding clinical trials piece written by a veteran in the industry at running biotech’s Anothony Giovinazzo. And lastly, our feature entrepreneur this month is the man behind the biggest biotech deal in Canada for 2014, former Paladin Labs CEO and current Knight Therapeutics CEO Jonathan Goodman. We hope you enjoy this issue as much as we enjoyed putting it together.

MARKETING MANAGER

Terri Pavelic Shawn Lawrence Anthony Giovinazzo Amy Dam Carmela DeLuca Mark Curtis Murray McLaughlin Marcello Sukhdeo Elena Pankova John R. Jones Mary Malofy

CIRCULATION DIRECTOR James Watson circulation@promotive.net Tel: 705-812-0611

EDITORIAL ADVISORY BOARD Celine Bak, Analytica Advisors; Rob Henderson, BioTalent Canada; Najla Guthrie, KGK Synergize; Pierre Bourassa, IRAP, Montréal; Murray McLaughlin, Sustainable Chemistry Alliance; Carol Reynolds, AdFarm; Ulli Krull, UTM; John Kelly, KeliRo Company Inc.; Peter Pekos, Dalton Pharma Services; Brad Thompson, Oncolytics; Darrell Ethell, CanReg; John Hylton, John H. Hylton & Associates; Robert Foldes, Viteava Pharmaceuticals Inc.; Randal R.Goodfellow, P.Ag., Senior Vice President, Corporate Relations, Ensyn; Bob H. Sotiriadis, Robic LLP; Dale Patterson, Genome Canada; Darcy Pawlik, Syngenta Seeds Canada Inc; Gail Garland, OBIO; Barry Gee, CDRD; Bonnie Kuehl, Scientific Insights Consulting Group Inc.; Raphael Hofstein, MaRS Innovation Biotechnology Focus is published 10 times per year by Promotive Communications Inc. 24-4 Vata Court, Aurora, Ontario L4G 4B6 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 dept – 24-4 Vata Court, Aurora, Ontario L4G 4B6 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.

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4 BIOTECHNOLOGY FOCUS October/November 2014

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R & D NEWS World breakthrough: a new molecule allows for an increase in stem cell transplants

Clinical Trials & Patents OncoGenex Pharmaceuticals, Inc. (Vancouver, BC) has completed patient enrollment for its Phase 3 AFFINITY trial. The trial is randomized, open-label and is designed to evaluate the potential of custirsen to improve survival outcomes in approximately 630 men with metastatic castrate-resistant prostate cancer (CRPC). Patients have been randomized to receive second-line Jevtana® (cabazitaxel) and prednisone with or without custirsen. The timing of the survival primary endpoint data is event-driven and results are currently expected in late 2015 or early 2016. The U.S. Food and Drug Administration (FDA) has already granted Fast Track designation for the evaluation of custirsen in the AFFINITY trial.

Specialty pharmaceutical company Nuvo Research Inc. (Mississauga, ON) reports it has completed enrollment for its Phase 2 clinical trial of WF10 in patients with refractory allergic rhinitis. WF10 is a solution of OXO-K993 containing stabilized chlorite ions that focuses on supporting the immune system by targeting the macrophage, a type of white blood cell that coordinates much of the immune system to regulate normal immune function. The 16-week trial is a randomized, double-blind, placebo-controlled study that will assess the efficacy, safety and tolerability of a regimen of five infusions of either WF10 or its main constituents (sodium chlorite and sodium chlorate) relative to saline control in patients with multiple airborne allergies who suffer from moderate to severe allergic rhinitis. The trial will measure total nasal symptom score (TNSS) and other secondary endpoints. The company says it expects the trial to be completed in late 2014 with top-line results anticipated in the first quarter of 2015.

From left to right, Jalila Chagraoui, Research Officer, Iman Farès, Ph.D. Student and Dr. Guy Sauvageau, Principal Investigator at IRIC. Photo: IRIC. Investigators from the Institute for Research in Immunology and Cancer (IRIC) at the Université de Montréal have just published in Science magazine, their discovery of a new molecule, the first of its kind, which allows for the multiplication of stem cells in a unit of cord blood. Directed by Dr. Guy Sauvageau, principal investigator at IRIC and hematologist at the Maisonneuve-Rosemont Hospital, this breakthrough has the potential to multiply by 10 the number of cord blood units available for a transplant in humans. In addition, it will considerably reduce the complications associated with stem cell transplantation. A clinical study using this molecule, named UM171 in honor of the Université de Montréal, and a new type of bioreactor developed for stem culture in collaboration with the University of Toronto will be initiated in December, 2014 at the Maisonneuve-Rosemont Hospital. According to Dr. Guy Sauvageau, “This new molecule, combined with the new bioreactor technology, will allow thousands of patients around the world access to a safer stem cell transplant. Considering that many patients currently cannot benefit from a stem cell transplant for lack of 6 BIOTECHNOLOGY FOCUS October/November 2014

matching donors, this discovery looks to be highly promising for the treatment of various types of cancer.” The Centre of Excellence for Cellular Therapy at the Maisonneuve-Rosemont Hospital will serve as production unit for these stem cells, and grafts will then be distributed to patients in Montréal, Québec City and Vancouver for this first Canadian clinical study. Tangible results should be available one year later, that is, in December 2015. The significance of this new discovery is such that over time, conclusive clinical results could revolutionize the treatment of leukemia and other blood-related illnesses. “These extraordinary advances result from the efforts of a remarkable team that includes extremely gifted students and postdoctoral investigators working in the IRIC laboratories,” adds Dr. Guy Sauvageau. “Among them, the first authors of this publication: Iman Farès, doctoral student, and Jalila Chagraoui, research officer, along with the professionals in IRIC’s medical chemistry core facility under the direction of Anne Marinier, who optimized the therapeutic properties of this new molecule.” To see this story online visit http://biotechnologyfocus.ca/?p=12523

n Oncolytics Biotech Inc. (Calgary, AB) reports that patient enrollment has been completed in its randomized Phase 2 study of paclitaxel plus REOLYSIN® versus paclitaxel alone in patients with persistent or recurrent ovarian, fallopian tube or primary peritoneal cancer (GOG186H). The study chair is David E. Cohn, MD, gynecologic oncology division director at The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James). The study is to be done by the Gynecologic Oncology Group, now incorporated into NRG Oncology and is sponsored by the U.S. National Cancer Institute (NCI) through a clinical trials agreement between the Cancer Therapy Evaluation Program, Division of Cancer Treatment and Diagnosis, NCI and Oncolytics. Oncolytics is providing clinical supplies of REOLYSIN.

For more R&D news visit http://biotechnologyfocus.ca/ category/topics/science/

R & D NEWS

Dr. Ravi Retnakaran

New combination therapy is game-changer in treatment of type 2 diabetes

To see this story online visit http://biotechnologyfocus.ca/ ?p=12507

EACH DAY, THERE ARE

4,900

CHILDREN

Adverse effects of the current drug treatments for patients with type 2 diabetes, such as weight gain and low blood sugar levels, could be virtually eliminated by adopting a treatment strategy that is proving effective and safe in clinical trials worldwide, according to a study published in The Lancet. The study, led by investigator Dr. Ravi Retnakaran of Mount Sinai Hospital in Toronto, ONT, shows that, when combined with long-acting insulin, a new class of medication that mimics a naturally occurring gut hormone, can obtain excellent blood sugar control without increased risk of low sugars or weight gain. “The ability to achieve this ideal trifecta – glucose control, plus weight loss and no increased risk of low sugars or weight gain – offers the opportunity to change the paradigm of clinical management for type 2 diabetes,” notes Dr. Retnakaran. The hormone (glucagon-like peptide-1 agonist, or GLP-1 agonist) is produced in the gut as part of normal digestion. It is triggered by the presence of food in the small intestine, and acts almost instantly and with exquisite sensitivity to fine-tune the balance of insulin to blood sugar. Combining a GLP-1 agonist with long-acting insulin gives better blood glucose control and induces weight loss, with no increased risk of low sugars, as compared to other anti-diabetic therapies. In type 2 diabetes, the body’s ability to produce insulin and effectively metabolize sugar decreases over time. To control their blood sugar levels, patients typically are treated with different medications, and many ultimately need insulin itself. With current drugs and even with insulin treatment, however, many patients are not able to reach target levels for blood sugar control, experiencing increased risks of low blood sugar and weight gain. The research team’s meta-analysis and systematic review examined 15 recent randomized controlled trials, which involved a total of 4,348 participants. Based on the study’s results, the research team at Mount Sinai Hospital is currently starting a new clinical trial (called PREVAIL) of this combination therapy. “The closer we are to mimicking normal blood glucose control, the better for patients with diabetes,” notes Dr. Jim Woodgett, director of the Lunenfeld-Tanenbaum Research Institute at Mount Sinai Hospital. “This study should provide the impetus to change standards of care to benefit everyone with this disease.” The new treatment protocol is not the only advance in type 2 diabetes, Dr. Retnakaran adds. “We are also seeing big gains in using insulin alone for short periods in the early stages of type 2 diabetes, rather than reserving it as a ‘rescue’ medication for later in the disease. Using insulin early and for only a few weeks at a time is another strategy for obtaining excellent blood sugar control without weight gain or increased risk of low blood sugars.” His team is testing this strategy in a clinical trial at Mount Sinai Hospital (Toronto) called RESET IT.

WHO RELY ON THE SUPPORT OF CHILDREN’S MIRACLE NETWORK

MEMBER HOSPITALS IN CANADA CHILDREN’S MIRACLE NETWORK FUNDS

CRITICAL PRIORITY NEEDS FOR

CHILDREN’S

HOSPITALS

ACROSS CANADA

What is Children’s Miracle Network? Children’s Miracle Network® raises funds for 170 children’s hospitals in North America, 14 of which are in Canada. These hospitals, in turn, use the money where it’s needed the most. When a donation is given, it stays in the community, ensuring that every dollar is helping local kids. These donations have gone to support critical research and training, purchase life-saving equipment, and ensure excellence in care - all in support of our mission to save and improve the lives of children.

Learn more at: ChildrensMiracleNetwork.ca October/November 2014 BIOTECHNOLOGY FOCUS 7

R & D NEWS

The Vancouver Prostate Centre (VPC)

Partnership creates research laboratory for translational cancer research

The Vancouver Prostate Centre (VPC) is teaming up with the world’s largest genomics organization, China-based BGI to establish a joint research laboratory. The BGI-VPC Joint Research Laboratory, established at the VPC facility in Vancouver and currently available for use, will apply genome research to develop new biomarkers and cancer therapies. The facility will specialize in sequencing and translational research facilitating basic and translational programs in oncology, including personalized oncology. The primary focus initially will be on bladder, kidney and prostate cancer, although the collaboration could be expanded to include other cancers of spe-

cial interest say the two organizations. Over the next five years, scientists will utilize clinical expertise, a unique repository of metastatic and post-treated cancer specimens, and cutting edge genomics with the goal of improving patient outcomes and quality of life through development of targeted therapies and ultimately evidence based precision oncology. In addition, with the participation of Dr. Yuzhuo Wang, senior scientist at Vancouver Prostate Centre and BC Cancer Agency, researchers will use unique and clinically relevant patient derived xenograft models to dissect the inner workings of tumours. It is anticipated that future collaborative research will range from health and agricul-

ture to environmental research. “The primary focus of this agreement is to achieve a deeper understanding of advanced and drug-resistant cancers with the implicit goal of improved quality of life for patients,” stated Dr. Colin Collins, professor at the University of British Columbia and senior scientist and director of Laboratory for Advanced Genome Analysis at VPC. Added Dr. Martin Gleave, director of the VPC, “this partnership is creating tangible synergy that will expand and accelerate our drug discovery pipeline for cancer patients.” “We welcome this opportunity to collaborate with The Vancouver Prostate Centre, and to make a meaningful contribution to advancing potential new therapies for cancer,” stated Yingrui Li, CEO of BGI Tech Solutions Company. “With our contribution of next generation sequencing equipment and advanced data processing and analysis, we expect the Joint Research Laboratory to make significant discoveries resulting in new, validated therapeutic targets as well as new molecules for commercial development.” The collaboration is the first between BGI and any Canadian research institution. To see this story online visit http://biotechnologyfocus.ca/?p=12494

The Ontario Institute for Cancer Research (OICR) is providing $7 million in funding over four years to its Immuno and Bio-therapies (ORBiT) program, led by Dr. John Bell at the Ottawa Hospital Research Institute. The funds will be used to further promising research into oncolytic viruses, immunotherapies and oncolytic vaccine therapies, including a clinical trial evaluating the program’s Maraba Oncolytic Vaccine platform. The program includes collaborators at the Children’s Hospital for Eastern Ontario (CHEO), McMaster University, NCIC Clinical Trials Group and the University Health Network (UHN). “Oncolytic viruses and vaccines have long shown a great deal of promise, with the potential to offer patients targeted treatment for many forms of cancer,” commented Dr. Tom Hudson, president and scientific director of OICR. “The recent advances in the field of immunotherapy for the treatment of cancer patients further underline the importance of developing innovative therapies targeting the immune system. The work of Dr. Bell and his colleagues in the 8 BIOTECHNOLOGY FOCUS October/November 2014

ORBiT Program is turning this promise into reality, bringing these therapies to the clinic and building the infrastructure in Ontario to ensure their long term success. OICR is proud to fund this innovative and exceptional work.” Oncolytic viruses and vaccines take advantage of the fact that cancer cells grow quickly and as a result have a weaker structure than normal cells. This makes them more susceptible to viral attacks than normal cells, meaning virus-based treatments can be designed and targeted to harm just cancer cells while leaving healthy cells unaffected. “This support for ORBiT is critical to moving our discoveries forward, so patients can benefit sooner in the clinic,” said Dr. Bell. “With the funding announced today, we will soon be able to begin a clinical trial for a therapy that combines a new cancer-fighting virus, developed here in Ottawa, with a vaccine therapy. It is clearly helping to position us as leaders in the field.” The ORBiT Program was designed to address some of the key challenges in the

Dr. John Bell

New national consortium to tackle dementia

development of bio-therapies in Ontario and rapidly translate leading laboratory discoveries into the clinic. This is the second round of funding for the ORBiT Program, which initially received $10 million from the Government of Ontario in 2009. The new funding brings the total to $17 million over nine years. To see this story online visit http://biotechnologyfocus.ca/?p=12572

BUSINESS CORNER SQI Diagnostics to begin trading on OTCQX Marketplace

Common shares for SQI Diagnostics Inc., a life sciences company that develops and commercializes technologies and products for advanced microarray diagnostics, have been approved for trading in the U.S. on the OTCQX marketplace. The company will be listed under the symbol SQIDF. Company CEO Andrew Morris commented on the news that, “trading on the OTCQX will help increase awareness of SQI in the U.S investment community and is an important step for SQI’s continued growth.” The OTCQX marketplace is for established, investor-focused U.S. and global companies. To qualify for OTCQX,

companies must meet high financial standards, demonstrate compliance with U.S. securities laws, be current in their disclosure, and be sponsored by a professional third-party advisor. U.S. investors can find information and Real-Time Level 2 quotes for the company on www.otcmarkets.com. Dorsey & Whitney LLP will act as the company’s principal American liaison on the OTCQX marketplace. Morris added that the company will continue to trade on the TSX Venture Exchange under its existing symbol SQD. To see this story online visit http://biotechnologyfocus. ca/?p=12574

Qu Biologics closes private funding round of $5 million plus Qu Biologics Inc. reports it has closed an oversubscribed private financing of $5.1 million, exceeding its target goal of $4.5 million. The financing brings the total money raised by company in 2014 to $8.2 million. The company is currently developing Site Specific Immunomodulators (SSIs) that aim to restore the body’s immune system. Dr. Hal Gunn, CEO of Qu Biologics commented the company was very pleased with the high level of interest from new and returning investors. He explains much of this interest is due to the promise of the technology. “SSIs represent a new approach to treating cancer and immune-related disease, such as Crohn’s disease,” he said. “Rather than blocking or disrupting a process in the body, SSIs aim to restore the body’s normal immune response.” Qu Biologics is currently enrolling participants into a Phase 1/2 placebo-controlled, randomized clinical trial designed to evaluate the safety and efficacy of QBECO SSI in Crohn’s disease. The Crohn’s disease trial is progressing as planned and primary endpoint results are expected Q2 2015. Qu Biologics is also preparing to initiate a Phase 2a clinical trial of QBKPN SSI in lung cancer later this year and a Phase 2a clinical trial of QBECO SSI in ulcerative colitis in the first half of 2015. To see this story online visit http://biotechnologyfocus.ca/?p=12560

Knight Therapeutics Inc. acquires Orphan Canada Inc. Canadian specialty pharmaceutical company Knight Therapeutics Inc. has entered into an asset purchase agreement with Orphan Canada Inc. related to the Canadian rights for ATryn® and PHOTOFRIN® (porfimer sodium), two pharmaceutical products approved for sale in multiple jurisdictions. As part of the agreement, Jason Flowerday and Joost van der Mark, the founders of Orphan, will join the Knight leadership team; Flowerday as vice president commercial operations and Mark as vice president corporate development. “Joost and I are excited to bring our combined experience of 40 years in the pharmaceutical industry to Knight.” said Jason Flowerday, chief commercial officer of Orphan Canada Inc, formerly a privately held, Toronto-based company. “We are proud of this agreement which

marks the beginning of a relationship with Knight where we will work together to bring innovative products to the Canadian market,” he added. Also in connection with the agreement, Bourne Partners, a healthcare-focused merchant banking firm and co-founders of Orphan Canada with Flowerday and Mark, has been engaged by Knight to assist with its corporate development efforts. When commenting on the overall agreement, Jonathan Ross Goodman, CEO of Knight, stated, “With this transaction, Knight has established its first product offering in Canada, significantly increased the size and experience of its leadership team, and expanded its business development reach.” To see this story online visit http://biotechnologyfocus.ca/?p=12403 October/November 2014 BIOTECHNOLOGY FOCUS 9

BUSINESS CORNER

Pﬁzer acquires license option for Medgenesis’s potential Parkinson’s treatment technology Canadian biotech company MedGenesis Therapeutix says it has granted Pfizer an exclusive worldwide option to license its glial cell line-derived neurotrophic factor (GDNF) protein and convection enhanced delivery (CED) technology to be used in research for potential treatments for Parkinson’s disease. MedGenesis is currently running a proof of concept study in Parkinson’s disease with the GDNF and CED technology. Convectionenhanced delivery (CED) is a minimally invasive technique that provides for targeted, local treatment of serious conditions such as Parkinson’s, epilepsy, brain cancer and other severely debilitating diseases of the central nervous system. MedGenesis’ GDNF program has been supported by a grant from the Michael J Fox Foundation for Parkinson’s Research, and benefitted from a direct grant from Parkinson’s UK to an investigator-sponsored clinical trial being conducted by the North Bristol Trust. “We believe that delivering drugs, in-

cluding proteins, precisely to the regions of the brain where they are required will allow the potential for better treatments for a whole range of central nervous system (CNS) disease, and opens up the opportunity for the potential development of truly disease-modifying treatments of neurological diseases,” commented Dr. Erich Mohr, CEO and chairman of MedGenesis. “We are delighted to be collaborating with Pfizer to complete our GDNF program, and we hope that this collaboration will result in potential treatments for people living with Parkinson’s that could radically alter the course of their disease.” “Pfizer is committed to improving the lives of people living with neurological diseases, including Parkinson’s disease,” said Michael Ehlers, senior vice president, Neuroscience Research Unit, Pfizer. “With limited treatment options currently available, Parkinson’s disease is a devastating illness for patients and families. We believe that this collaboration with MedGenesis has

Dr. Erich Mohr

Michael Ehlers

the potential to provide the first diseasemodifying treatment that slows the progressive decline in patients, consistent with our commitment to making a difference in areas of high unmet medical need.” Under the terms of the agreement MedGenesis will receive an upfront option fee and upon exercise of the option by Pfizer will be eligible for further milestone and royalty payments. To see this story online visit http://biotechnologyfocus.ca/?p=12556

Dealmakers n Concordia Healthcare Corp. (Oakville, ON) through its subsidiary, Concordia Pharmaceuticals Inc. has entered into an agreement with Eisai Inc. (Eisai) to acquire Zonegran® for commercialization and sale in the U.S. and Peurto Rico. The company has agreed to acquire Zonegran for US$90 million in cash, plus approximately US$1.5 million for purchased inventory. Management plans to pay for the acquisition through debt financing. Zonegran® was first approved by the U.S. Food and Drug Administration in March 2000 indicated for adjunctive therapy in the treatment of partial seizures in adults with epilepsy. It is available in 25mg and 100mg capsules. It is a registered trademark of Sumitomo Dainippon Pharma Co., Ltd. and licensed exclusively to Eisai Inc. Torreya Partners is acting as financial advisor to Concordia. n Canadian biotherapeutics company Zymeworks Inc. (Vancouver, BC) has hired American contract manufacturing organization

10 BIOTECHNOLOGY FOCUS October/November 2014

CMC Biologics, Inc. (Bothell, WA) for the process and formulation development, and cGMP clinical manufacturing of its recombinant human IgG1 bi-specific (heterodimeric) antibody. Terms of the agreement were not disclosed. Clinical stage regenerative medicine company RepliCel Life Sciences Inc. (Vancouver, BC), is the newest member of the Centre for Commercialization of Regenerative Medicine’s (CCRM) industry consortium. RepliCel says it is also pursuing CCRM as a secondary manufacturing site for the company’s fibroblast platform. CCRM is currently providing RepliCel with contract regulatory services including documentation and filings related to RepliCel’s RCT-A-01 product for chronic Achilles tendinosis. This program is the subject of a planned clinical trial in Canada to start in 2014 that will involve clinical testing to address chronic tendinosis, a degenerative disease of the tendon caused by a cycle of injury, improp-

er healing and re-injury until there are very few pain free and functional periods. Concordia Healthcare Corp. (Oakville, ON) through its subsidiary, Pinnacle Biologics, Inc. has signed a collaboration agreement for PHOTOFRIN® (porfimer sodium) with Toronto-based Orphan Canada (now part of Knight Therapeutics Inc.). Under the agreement, Pinnacle and Orphan Canada will partner to support the continued supply of Photodynamic Therapy (PDT) using PHOTOFRIN in Canada. PHOTOFRIN is approved in Canada for the treatment of certain forms of gastrointestinal, lung and bladder cancers. PHOTOFRIN was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) in 2004 for cholangiocarcinoma (CCA), a rare bile duct cancer.

For more Business Corner news visit http://biotechnologyfocus.ca/category/ topics/business/

By: Carmela DeLuca and Amy Dam

INTELLECTUAL PROPERTY

PRECISION MEDICINE:

Patent and Regulatory Frameworks in Canada

“P

ersonalized medicine”, or perhaps more accurately “precision medicine” (“PM”) has been described as the next revolution in healthcare and the “coolest part of medicine.”1,2 Generally speaking, PM refers to the tailoring of medical interventions to individual (“personalized”) and/ or disease (“precision”) characteristics.3 PM promises to radically improve patient care by making medicine more predictive, preventive and precise. Accordingly, there is a strong desire for protecting and commercializing PM innovations. The following provides a brief review of some Canadian specific scenarios under the current legal regimes that may influence development of PM technologies.

Patent Framework Claims define the scope of patent protection and PM innovations can be protected by a variety of claim types. Examples of claim types include prognostic method type claims (e.g. that extract baseline patient/disease characteristic information that can affect outcome regardless of treatment) and companion diagnostic method type claims (e.g. that identify whether a patient is more or less likely to benefit from a specific treatment or intervention). A generalized example could be a method of predicting a likelihood of developing disease Y (e.g. prognostic) or responding to a particular treatment (e.g. companion) for disease Y in a patient comprising: i) detecting a mutation in ABC; and ii) identifying the patient with a greater likelihood of developing disease Y or responding to a particular treatment for disease Y if the mutation in ABC is detected. Assuming

such claims meet the requirements for patentability, including novelty, non-obviousness and utility, such claims can generally be obtained. Method of treatment claims would seem to be a useful category of claim type to protect PM innovations as PM tests can be used to identity a patient subpopulation that, for example, is more likely to respond to a treatment. However, Canadian Courts have clearly established that methods of medical treatment claims do not constitute patentable subject matter.4 The decision is based on the principle that inventions should not prevent physicians from exercising their skill and judgement while delivering a medical treatment.5 It is generally possible to convert traditional method of treatment claims into use claims (e.g. use of compound X for treating disease Y, a.k.a. “Canadian method of treatment claims”), though the drafter must remain mindful to remove all elements that may be construed as limiting the skill and judgement of a physician. A patent examiner will consider a use claim not patentable if an essential element only serves to instruct a medical professional “how” to treat a patient (e.g. dosing schedule, administration site or narrowing treatment to a patient sub-population), rather than “what” to use to treat the patient (e.g. compound, composition, dosage form).6 The question that arises is ‘does inclusion of “how” elements equate to a patentability bar for personalized medicine Canadian style treatment claims?’ Recently the Canadian Intellectual Property Office (CIPO) released a practice notice entitled Examination Practice Respecting Medical Uses7 and Examples of purposive construction analysis of medical use claims for statutory subject-matter evaluation.8 One of the examples describes use of a known compound X to treat a disease Y in a specific patient population having the gene mutation ABC, and finds that the claim is not patentable because the invention is directed to “how” to treat and restricts the choices of the physician on how to use X. Moreover, the example states that the invention does not qualify as a “selection” patent because in a selection all of the range claimed must be novel and in this case in the population already treated, some patients already had the ABC gene mutation, and therefore treating the sub-population with X for Y is not novel.9 The example notes that if the solution was to provide a different formulation of X to patients with the ABC mutation, for example a lower dose unit, then such a claim could be statutory. No case law is cited to support the lack of patentability and although several court decisions have considered the patentability of October/November 2014 BIOTECHNOLOGY FOCUS 11

Intellectual Property dosage ranges whose selection relies on the skill of a physician , no cases have specifically considered the scenario provided in the ABC mutation example.10 As mentioned above, meaningful protection is still available in the form of prognostic/ diagnostic claims. However, medical use claims may have advantages not shared by prognostic/diagnostic type claims. For example, a patent with a claim for the use of a medicinal ingredient, where the use has been approved through the issuance of a Notice of Compliance in respect of the submission would be eligible for listing on the Patent Register under the Patented Medicines (Notice of Compliance) Regulations and periods of exclusivity therein provided.11 Such benefits are not available for patents directed to prognostic/diagnostic claims. The Canadian position also stands in contrast to our neighbours to the south. Notably, the breadth of prognostic/diagnostic claim types allowed by CIPO is far greater than what is currently allowable by the United States Patent and Trademark Office (USPTO) in light of recent case law and a USPTO guidance document that have significantly diminished the breadth of protection available to prognostic/diagnostic type claims.12 Interestingly, although patenting PM technologies and particularly prognostic/ diagnostic method type claims have faced mounting hurdles in the United States, method of treatment claims are a category of claim that is generally available for personalized medicine type claims.

Regulatory Framework Under Canada’s Constitution , legislative powers are divided between federal and provincial governments and as such, ‘diagnostic’ tests may fall under federal or provincial regulations depending on whether they are sold as a kit or delivered solely in a laboratory.13 In vitro diagnostic tests (“IVDs”) are regulated under the Medicines Devices Regulations14 pursuant to the Food and Drug Act , while laboratorydelivered tests (“LDT”) are subject to provincial regulations. In vitro diagnostic devices are defined in the Medicines Devices Regulations as a medical device intended to be used in vitro for the examination of specimens taken from the body, and genetic testing is defined as the analysis of DNA, RNA or chromosomes for purposes such as the prediction of disease or vertical transmission risks, or monitoring, diagnosing or prognosis. Based on a risk-based classification system of medical devices, IVDs intended to be used for genetic testing are considered Class III devices16 (which represent moderate public health risk or high individual risk). A manufacturer desiring to commercialize a Class III device in Canada is required to obtain a Medical Device Licence issued by Health Canada. As such, it must submit a premarket review comprising among other things a summary of investigational testing conducted on the device using human subjects representative of the intended users and under conditions similar to the conditions of use, as well as provide a copy of the quality management system certificate certifying that the quality management system under which the device is designed and manufactured satisfies National Standard of Canada CAN/CSA-ISO 13485:03, Medical devices — Quality management systems — Requirements for regulatory purposes.17 LDTs, on the other hand, are designed, manufactured and used in a laboratory setting and are not meant to be sold as standalone kits. 12 BIOTECHNOLOGY FOCUS October/November 2014

Therefore, they do not fall within the scope of the Medical Devices Regulations or the Food and Drug Act. They are regulated by provincial regulations relating to laboratory safety and quality. Accordingly, all laboratories offering diagnostic testing are required to obtain an operation licence issued by the provincial Minister of Health, as well as self-regulated accreditation and quality controls. Because each province has its own regulations relating to medical laboratories, these rules have been shown to vary considerably from one provincial jurisdiction to another.18 In some provinces, there is a lack of binding rules whereas other provinces have developed elaborate legal frameworks.19 Given that diagnostic testing is offered predominantly as a laboratory service, and given the variations between provincial regulations on LDTs in Canada, harmonized oversight of diagnostic tests has been proposed, particularly for high risk LDTs. At present, LDTs that are considered as posing a high individual risk (i.e. equivalent to Class III devices) are not required to comply with Health Canada regulations which may be more stringent. Interestingly, this concern was expressed by the United States Food and Drug Agency (“FDA”) which regulates IVDs (much like Health Canada), and has suggested that the FDA oversight be based on the risk level to patients, and not on whether the diagnostic test is made by a conventional manufacturer or in a single laboratory.20 Although the FDA has historically exercised enforcement discretion over LDTs, it has recently taken steps to ensure that diagnostic tests provide accurate, consistent and reliable results by publishing a Draft Guidance entitled Framework for Regulatory Oversight of Laboratory Developed Tests (LDTs). LDT providers who were not required to provide clinical study data to support their tests may now be urged to conform to FDA guidelines.

Conclusion As innovators continue to invest heavily in PM, it is expected that PM technologies promising to revolutionize health care will continue to emerge. Patent and regulatory challenges remain. Methods of treatment are not per se patent eligible subject matter in Canada although protection for some therapeutic methods and meaningful protection for prognostic/diagnostic claims is available. It remains to be seen if the courts will endorse CIPO’s example relating to subpopulations. The Canadian regulatory frameworks relevant to PM technologies are complex owing in part to the division of powers between the federal and provincial governments. Whether regulatory harmonization is on the horizon, and whether harmonization would address the regulatory challenges, remain to be seen.

References: 1. Katsnelson A. Momentum grows to make ‘personalized’ medicine more ‘precise’. Nature Medicine 19, 249 (2013). 2. Peter Byers, MD, director of the new Center for Precision Diagnostics at the University of Washington, calls it “the coolest part of medicine.” UW Medicine 2014 Annual Address: Precision Medicine; Ibid. 3. Personalized Medicine Coalition, available at http://www.personalizedmedicinecoalition.org/About_Us/About_PMC. 4. Canadian Intellectual Property Office, Examination Practice Respecting Medical Uses (PN 2013-04) released June 10, 2013, at

INTELLECTUAL PROPERTY

5. 6. 7. 8.

9. 10.

11. 12.

13. 14. 15. 16.

page 1, available at http://www.cipo.ic.gc.ca/eic/site/cipointernet-internetopic.nsf/vwapj/wr03655-eng.pdf/$file/wr03655eng.pdf. Janssen Inc. v. Mylan Pharmaceuticals ULC, 2010 FC 1123, par. 51. Ibid. 4, at page 2. Ibid. 4. Canadian Intellectual Property Office, Examples of purposive construction analysis of medical use claims for statutory subjectmatter evaluation, released November 14, 2013, available at http://www.cipo.ic.gc.ca/eic/site/cipointernet-internetopic. nsf/eng/wr03714.html. Ibid, see section 7. Known compound, for a specific group of patients, Example 7.1 sub-patient population. Novartis Pharmaceuticals Canada Inc v. Cobalt Pharmaceuticals Company, 2013 FC 985 at paras. 92-99; Bayer Inc. v. Cobalt Pharmaceuticals Company, 2013 FC 1061 at paras. 160-162. Patented Medicines (Notice of Compliance) Regulations, S.O.R./93-133. USPTO, Guidance For Determining Subject Matter Eligibility Of Claims Reciting Or Involving Laws of Nature, Natural Phenomena, & Natural Products. The Guidance document at the time of writing was under public consultation and it is expected that changes will be released shortly. Constitution Act Medical Devices Regulations, S.O.R./98-282. Food and Drug Act, R.S.C. 1985, c. F-27. Health Canada, Therapeutic Products Programme, Guidance For The Risk Based Classification System of In Vitro Diagnostic Devices, see section 5.1.2, available at http://www.hc-sc.gc.ca/

dhp-mps/md-im/applic-demande/guide-ld/ivd-rsk_idiv-rsqeng.php. 17. Ibid. 14, section 32. 18. Petit E, Tassé AM, Godard B. An empirical analysis of the legal framework governing genetic services labs in Canadian provinces. Health Law Review 2008;16:65-72, at page 66. 19. Ibid, at p. 69. 20. FDA News Release, FDA takes steps to help ensure the reliability of certain diagnostic tests (July 31, 2014), available at http:// www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ ucm407321.htm Carmela DeLuca is a partner with Bereskin & Parr LLP and member of the Life Sciences practice group. Her practice focuses on patent matters, including advising on the management of patent portfolios in Canada and abroad, the preparation and prosecution of patent applications in the life sciences and the analysis of patent issues such as validity, infringement and freedom to operate. She also has experience advising on regulatory compliance. Amy Dam is an associate at Bereskin & Parr LLP and member of the Life Sciences practice group. Her practice focuses on patents relating to the life sciences, including biotechnology, chemical and pharmaceutical inventions. To see this story online visit http://biotechnologyfocus.ca/precision-medicinepatent-and-regulatory-frameworks-in-canada

CELEBRATING

YEARS

Of Delivering High Impact Results

Ontario Bioscience Innovation Organization Taking thought leadership into action

OBIO RESPONDS TO INDUSTRY NEEDS: Capital Access, Innovation Adoption and Interconnectivity initiatives.

OBIO REPRESENTS A POWERFUL AND MOTIVATED RESOURCE: Our member companies, partners and sponsors.

TOGETHER WE ARE BUILDING AN ONTARIO that will lead the way in providing health technology, products and services to the international marketplace. Join today and experience the benefits of membership.

www.obio.ca October/November 2014 BIOTECHNOLOGY FOCUS 13

Photo: Liam Richards

ag BIOTECH

ag-wesT bio:

25 years of growing bioscience

lot can happen in 25 years. That is especially true in the agriculture sector, where the introduction of agricultural biotechnology has led to dramatic changes in the last two and a half decades. Ag-West Bio has been a champion of Saskatchewan’s ag biotech sector every step of the way.

The history In the late 1980s the Government of Saskatchewan began an analysis of the province’s ag biotech industry and soon realized that the biggest gap was in marketing; it was determined that an organization was needed to facilitate, coordinate and promote ag biotech on a global scale. “Saskatchewan already had the research infrastructure. It also had programs for commercialization of technology that were so successful they were replicated by others,” says Royal Hinther, at the time a policy analyst involved in the strategy. The University of Saskatchewan (U of S), the National Research Council Canada (NRC) and Agriculture and Agri-Food Canada (AAFC) labs provided the foundation of research infrastructure; German life science company Hoechst had transferred its canola research to Saskatoon, and the city had a few biotech companies, including Philom Bios and Micro Bio Rhizogen. That, combined with some hard work and political will was enough to get the ﬂedgling cluster off the ground.

The Government approved a strategy that recommended the creation of a marketing and networking organization, and committed funding of $9 million over five years. “The convincing argument,” remembers Hinther, “was ‘ag biotech is one of the few sectors that has a multi-billion dollar market at its back door; expenditures by farmers for agricultural inputs are $5 billion.’ This was a way to add value to an existing industry.” The new bioscience industry association, Ag-West Biotech, was the first of its kind – it was something other jurisdictions did not have. It was unique in that it was connected to the community and had support from all stakeholders, from the public research institutions, to all levels of government and industry. The name was shortened in 2004 when the company merged with the Saskatchewan Bioproducts Association and the Saskatchewan Nutraceutical Network. Murray McLaughlin, the organization’s first president and CEO, says timing was important. “The cluster in Saskatoon realized that agricultural biotechnology would be important to Saskatchewan from a research and business perspective.” The priority for Ag-West in the early days was business attraction and education. Plant Genetic Systems Inc. out of Belgium, with a novel hybrid system, was a key target. The company had no revenue, but in spite of that, the belief was, ‘if you get them, every-

14 BIOTECHNOLOGY FOCUS October/November 2014

one else will follow.’ The plan worked. The second order of business was educating both the research community and international business interests about what Ag-West Biotech was, and what Saskatoon had to offer: Research capacity and business support. “We had to explain to researchers the importance of connecting their research to commercial opportunity,” says Hinther. Outside recognition was quick to appear. McLaughlin remembers, “About three years into our mandate, a researcher was at a World Summit. He reported back that when he said he was from Saskatoon, four of the five international scientists at the table told him that they considered Saskatoon a leading centre for ag biotech.” From then on, he was comfortable referring to Saskatoon as a ‘world leader.’ To increase research capacity in the cluster, a steering committee that included the Mayor of Saskatoon, Saskatoon Regional Economic Development Authority (SREDA), the Government of Saskatchewan, Ag-West Biotech and the U of S, pushed for the relocation of numerous AAFC research positions from Ottawa to Saskatoon. That, and the previous rebranding of NRC’s lab as the Plant Biotechnology Institute, and ultimately, the Canadian Light Source (CLS) synchrotron, rounded out the community. Once again timing proved right for the organization, as the right people seemed to be in the right

Ag Biotech

places at the right time. There was support from Innovation Place, Limagrain moved to Saskatoon and John Hyshka was hired at SREDA. Innovation Place built research greenhouses to support the sector and the federal and provincial agricultural ministers were supportive of developing the ag biotech industry in Saskatchewan. John Cross, founder of Philom Bios, echoes the sentiment. “We had political impetus, initiatives from the University, recognition from academia that biological sciences were a pathway to the future. NRC and AAFC were on board at the same time. Ag-West further contributed that feeling of enhanced capabilities.” With investments in more than 50 companies, hundreds of events organized to bring researchers and entrepreneurs together from around the globe, and hosting delegations from dozens of countries, the value of AgWest Bio can be seen in the hard numbers. Abdul Jalil, executive director, Agriculture Research Branch at the Saskatchewan Ministry of Agriculture, reports, “Ag-West’s strategic investments of an estimated $12.9 million since 1989 has resulted in Gross Domestic Product impact of $1.2 billion and created an estimated 3,375 person years of employment in Saskatchewan.” A paper by Dr. Stuart Smyth (U of S) published in 2012 substantiates the economic impact of Ag-West Bio.

The future When Ag-West Bio was founded, not a single genome had been sequenced. Today genomics research is far more commonplace and affordable, and the potential applications are infinite. Genomics research itself is no longer based on technology development; it is primarily a tool available for com-

the director of Business Development at the CLS. “Plant imaging is the next big opportunity.” It appears the only limit to progress in the ag biotech sector is our imaginations—and possibly funding. Momentum lagged in Saskatchewan in the first decade of the 21st century, but today, public and private sectors are paying closer attention to what the industry needs to move forward, and working to close those gaps. “The provincial government has set a goal to become a global leader in ag biosciences by 2020, and has increased its financial support to ag research in Saskatchewan more than 100 per cent in the past seven years. Ag-West Bio will be key to the province reaching that goal,” says Abdul Jalil. John Cross is adamant that while the province’s 2020 plan is encouraging in its support of bioscience, a return to the types of business incentives that existed in the late 1980s would be helpful. “If you invested a dollar, you got a 30-cent tax credit. Philom Bios used it. We need the same thing again.” With the rate of change in the ag biotech sector, there will be no resting on our laurels. McLaughlin notes, “Genetic engineering and canola are synonymous, and herbicide tolerant canola has had an obvious impact on the provincial economy. We still need to push for it and keep supporting it to stay on top.” Ag-West Bio’s education component has changed, but is still paramount. While the cluster is on board, there has been resistance to ag biotech research and development. “The need for education is very important,” says Keller. “We want the farm-to-table consumer to understand that all food products are the result of innovation over many years.” Ag West Bio’s role continues to revolve around business development, networking and education in a rapidly evolving industry. The organization is shepherding a cluster through what McLaughlin describes as “continuous change in the future, as far as I can see.”

mercial improvement of crops and livestock. “There is a rapid evolution of biotech tools,” says Ag-West Bio’s president and CEO Wilf Keller, “from DNA sequencing, to metabolic profiling, to synchrotron-based imaging, to bioinformatics. One can ultimately envision the creation of the virtual cell or plant.” Ten years ago, there was talk of convergence among sectors, and that is now happening at a faster rate. “Research in the health of livestock and humans is growing in terms of innovation,” says Keller. “Genomic profiling tools for health will move us to a more preventive strategy, and genomics will play a role in DNA-based diagnostics for diseases such as cancer. Similar tools are being used in developing improved varieties of crops and strains of livestock.” Bioremediation, which uses living systems to detoxify environments, is a growing field, notes Keller, “The interface of living systems of microbes and plants with mining and petroleum are growth areas from which Saskatchewan can benefit.” “We’ll continue to see a lot of emphasis on building environmental quality, responding to societal demands for sustainable production of high-quality, safe food with reduced contaminants. There will be greater use of a plant’s natural processes to reduce chemical input. This is already happening, and it will continue at an increasingly rapid pace.” Murray McLaughlin notes the trend towards non-food oriented biofeedstocks. “In the next five years we’ll see a move away from using food products for biochemicals, and move to non-food products of crops, such as straw and hulls. We’ll be using waste This article was originally published in the components of crops and forests to develop Ag-West Bio annual report available on their website @ www.agwest.sk.ca bio-based industry.” There is emerging potential for using the CLS and the Sylvia Fedoruk Centre for Nuclear Innovation at the U of S in novel To see this story online visit methods of plant imaging. “We’re unique www.biotechnologyfocus.ca/ in having a synchrotron within walking ag-west-bio-25-years-of-growingdistance from all of this agricultural research bioscience infrastructure,” says Hinther, who is now

October/November 2014 BIOTECHNOLOGY FOCUS 15

By: Anthony Giovinazzo

CLINICaL TRIaLS

FUNDING CLINICAL TRIALS: Challenges for Small Biotechs

Because clinical trials are required to obtain regulatory approval for a drug, they are one of a biotech company’s highest priorities.

However, the challenges of funding these trials are significant, both for Big Pharma and even more so for smaller biotechs that have relatively fewer resources at their disposal. What challenges are specific to these smaller companies? In Canada, small biotechs can compete for funds from the Canadian Institutes of Health Research (CIHR), Canada’s federal funding agency for health research. Composed of 13 separate “virtual” institutes, CIHR provides leadership and support to more than 13,200 health researchers and trainees across Canada. Each institute is dedicated to a specific area of focus, linking and supporting researchers pursuing common goals. Each institute embraces a range of research from fundamental biomedical and clinical research, to research on health systems, health services, the health of populations, societal and cultural dimensions of health and environmental inﬂuences on health. Specific funding opportunities are listed on their website. Alternatively, small biotechs in Canada can compete for funding from from the National Research Council Canada’s Industrial Research Assistance Program (NRC-IRAP), which is Canada’s premier innovation assistance program for small and medium-sized enterprises. NRC-IRAP oversees a set of programs and collaboration opportunities in areas ranging from biologics to natural health products. In order to be considered for possible IRAP funding, the basic eligibility criteria are: to be a small and medium-sized enterprise in Canada, incorporated and profit-oriented; have 500 or fewer

16 BIOTECHNOLOGY FOCUS October/November 2014

full-time equivalent employees; and have the objective to grow and generate profits through development and commercialization of innovative, technology-driven new or improved products, services or processes in Canada. Before entering into a financial commitment with a client, both the firm and the project are evaluated on an individual basis during a consultation with one of NRC-IRAP’s advisors. Specifically, the due diligence process assesses the business and management capabilities of the firm and the company’s potential to achieve the expected results and outcomes associated with the proposed project; the financial capabilities of the firm and its plan to commercialize the developed technologies; and the technical aspects of the project and its potential impact on the firm. The NRC’s Human Health Therapeutics (HHT) portfolio works hand-in-hand with industry to help small biotech firms achieve success and save costs. As an R&D partner, NRC HHT de-risks critical steps in the development of biologics, vaccines and delivery of large molecules to the brain, to help improve the health of patients. The program adds value to collaborators’ products and accelerates their progress to market by overcoming challenges in preclinical and early clinical development, expanding product characterization, optimizing bioprocesses and their scale-up, and developing biomarkers. For example, the NRC’s vaccines program works with Canadian and international partners to develop novel and improved vaccines and enabling technologies. Through these partnerships, co developed vaccine candidates progress through an expanded value chain that includes biomanufacturing and early clinical trials. As a result, the NRC is able to accelerate development, reduce risk and add value to each vaccine candidate. The NRC has established best practices to guide the management and commercialization of intellectual property for co developed products and enabling technologies. It observes the rigorous standards set forth by Health Canada’s Biologics and Genetic Therapies Directorate (BGTD), ensuring that all of its co developed products meet their regulatory approval requirements to be sold in Canada and other jurisdictions. Canadian cGMP vaccine manufacturers who benefit from our bioprocessing expertise will be well positioned to supply both domestic and global markets. The NRC’s collaborative research projects span a very broad spectrum of activities and business structure models;

Clinical Trials The challenges of funding clinical trials are real and serious. A small biotech that is unable to allocate the necessary funding for clinical trials will be unable to bring its product to market, potentially depriving healthcare providers and their patients with innovation that could alleviate suffering. access to NRC expertise and equipment provides its collaborators—including small biotechs—with the opportunity to accelerate their commercial development timelines. The challenges are different in the U.S., where the federal government funds a large portion of clinical research primarily through the National Institutes of Health (NIH). Clinical trial costs can vary widely depending on the number of patients being sought, the number and location of research sites, the complexity of the trial protocol and the reimbursement provided to investigators. Funding can therefore come either from the NIH or from private grants. The congressionally mandated Small Business Innovation Research (SBIR) and Technology Transfer (STTR) programs make up about three per-

cent of the NIH extramural budget—about $700 million a year. However, businesses must be U.S.-owned to apply for funding through these programs. If a small biotech is developing a product targeting a specific patient population, it might seek to apply for funding from an organization founded to support that group. For example, since its founding in 2000, The Michael J. Fox Foundation for Parkinson’s Research has granted more than $450 million in research. Interested companies are invited to apply to a number of open funding programs in various areas of Parkinson’s research. Because competition for these grants is fierce, a small biotech must submit an application that is sufficiently compelling to the organization judges. A small biotech

can maximize its chances of success by offering a well-defined description of the drug it is developing, how it will address an unmet need, and how it compares favorably to other drugs currently on the market or in development either at other small biotechs or at larger companies. The challenges of funding clinical trials are real and serious. A small biotech that is unable to allocate the necessary funding for clinical trials will be unable to bring its product to market, potentially depriving healthcare providers and their patients with innovation that could alleviate suffering. Anthony Giovinazzo is President and CEO of Cynapsus Therapeutics, Inc., which is developing the only non-injectable (sublingual) delivery of the only approved drug (apomorphine) to be used as a rescue therapy for “off” motor symptoms of Parkinson’s disease.

To see this story online visit http//biotechnologyfocus.ca/ funding-clinical-trials-challengesfor-small-biotechs

October/November 2014 BIOTECHNOLOGY FOCUS 17

By Shawn Lawrence

innovator

Photo: Christinne Muschi

Looking to repeat the feat

With the sale of Paladin Labs Inc. to the highest bidder in his rear view mirror, Jonathan Goodman believes he can strike gold again with his new company Knight Therapeutics For Montréal entrepreneur Jonathan Goodman, 2014 has been a very big year. After all, there aren’t many CEO’s in the biotech space who can say they took a company they’ve founded from a market cap of just $6 million through to a sale of over $3.2 billion. The sale of Paladin Labs, one of Canada’s most successful specialty pharmaceutical companies to Pennsylvania-based Endo Health Solutions took many by surprise. But perhaps even more surprising than Goodman’s decision to walk away from the company he founded in 1995, was his decision to

jump back into the game with the launch of Knight Therapeutics on the same day of the landmark deal. It’s a question he gets asked a lot, even today, “you sold your business for $3 billion, why didn’t you take a week off or even a day?” “I love selling drugs, I kind of feel like it’s what I was born to do,” he says. And why wouldn’t he feel this way? Based on the success of Paladin, it’s obvious he’s very good at it. The most compelling evidence of this is the interest that Knight is generating from the industry and in the investor community. Perhaps it is because many see Knight as a sort of Paladin 2.0, and the reason for that is that Knight’s core business strategy is very much the same. Even things such as the company name (Paladin in the dictionary is defined as a knight in shining armor), the logo, colours and fonts share similarities. Most importantly, like Paladin, Knight Therapeutics is a specialty pharmaceutical company focused on acquiring and in-licensing innovative pharmaceutical products for Canadian and select world markets. As Goodman explains, “It’s about carving out our piece of the pie in markets that the big

18 BIOTECHNOLOGY FOCUS October/November 2014

players don’t care as much about, markets that are inefficient. For example, Canada roughly represents only 1.86 per cent of the world pharmaceutical market, hardly worth it to a big pharma company, but for a company such as Knight, it can be very lucrative. As I like to say, big pharma’s trash is often our treasure.” According to Goodman, in addition to focusing licensing efforts towards certain undervalued markets, the company plans to source its products in the same fashion as Paladin, buying low risk, late clinical stage products through licensing deals and partnerships. “Preclinical risk is something I’m just not comfortable taking. It’s too difficult to discern what will work and what won’t. I’ve always preferred to chase products in the later stages of the clinical development path. These are products that need just a little bit of a tweak to get approved and we will spend the money to do that.” The best part is Knight won’t have the burden of facing the same uphill battles that other start-ups do. The company’s biggest asset is its cash, of which it has plenty of. “Within the first three weeks of operating we raised $255 million without even trying

innovator “I had always run Paladin as a sort of benevolent dictator, and those that know me know I’m a bit of a control freak. In my absence Paladin became more of a democracy and it couldn’t go back to the way it was. So, I felt that if I couldn’t run the company the way I wanted to, I didn’t want it anymore and made plans to sell it.” and the message I’m getting is the investor community is thrilled with us and they like our plan of execution. I know how to sell drugs in Canada, and I think that fact is not lost on them. More importantly, there’s a certain level of trust going through this a second time. It’s very important that when you say you’re going to do something you do it. You set your milestones and you meet them on time. That’s how I ran Paladin, and that’s how I’m going to run Knight.” With the balance sheet sitting pretty, Goodman believes the company needs to now deploy the capital it has raised. He already has big plans on how this money will be spent and the shopping spree has already started. While a specialty area of therapeutic focus has yet to be decided, he expects it won’t take long to find one. “At Paladin, I used to joke that our specialties were urology, endocrinology and opportunology. With Knight, since its early days, it’s too soon to tell. But the strategy is the same; we’re in the market to capitalize on opportunity. That’s really the core of our business going forward. And the source of these new products will include the aforementioned pharmaceutical companies willing to trade off Canadian rights for their products, as well as emerging specialty pharmaceutical and biotech companies who have developed something interesting and mature.” Such is the case with the company’s recent acquisition of Orphan Canada Inc. Through the purchase it acquired new staff as well as two new products. “The two assets we brought in are very near term, in fact, they’re approved in other markets. We just need to get one of them approved in Canada, and the other we’re looking to expand on by getting a new indication approved.” There’s also the caveat that through its ownership of Orphan Canada Inc. the company has partnered with Concordia Healthcare. “Our goals are completely aligned, they’re focused on the U.S. market and we’re focused on Canada so it’s easy to work together.”

According to Goodman, more deal flow is on the horizon in terms of seeking out and adding new products to the portfolio with the company looking at approximately 80 such opportunities. In addition to this, Knight has two other assets at its disposal— a voucher that allows the bearer to get faster review time by the U.S. Food and Drug Administration; and rights to Impavido, an oral treatment for leishmaniasis, a tropical disease transmitted by the sandfly. Both were inherited from Paladin when Knight was launched. Of the two, Goodman considers Impavido rather insignificant in the grand scheme but keeping this product and getting it approved was a means to an end he says. “It’s the reason really that we have this sort of uncashed lottery ticket U.S. “priority voucher”. When I was selling the company, what was important to me was getting the best value I could for the company. During negotiations, I conveyed to Endo that we expected to get this product approved by the FDA in a couple of weeks and it was going to come with this priority voucher. To include it in the sale I wanted more cash. In the end, Endo wasn’t willing to pay for it; they said they didn’t value it. So I said perfect, if you don’t want it, I’m going to keep it and give it to my shareholders.” Goodman recalls that the whole conversation lasted just 30 seconds, but it led to the first brick in the foundation of Knight being laid. And in the end, Knight got its approval for Impavido and with it the voucher. As such, Endo’s missed opportunity has turned into Knight’s gain. “What’s unique about it really is that it’s like a fast pass in terms of your FDA process. The program was designed by professors at Duke University to encourage drug companies to develop drugs for diseases where there is no market incentive, i.e. tropical infectious diseases. The idea is to create economic incentive for companies to create drugs for these neglected disease areas and if you do, you get this voucher, you give it to FDA and they will review any product in six months instead of a year. The key there, it can be used on any product. Just imagine what a big pharma company would pay for

six more months of patent protection.” And yet with all this going for it, Goodman admits there was a time Knight Therapeutics almost didn’t happen, a time where he very nearly left the business of biotech altogether. Due to complications stemming from a cycling accident that happened in 2011, Goodman felt he longer could head up a company. The accident which was hugely disruptive to his life had him in a coma for five weeks, and led to several hospital complications including contracting C. difficile. This forced his long-time partner at Paladin, Mark Beadet to step into his role. Goodman endured a long struggle back to health, and it was a scary time, one with lots of self-reflection, he says. When he was well enough to return, he found things had changed too dramatically and he longer enjoyed running the business. “I had always run Paladin as a sort of benevolent dictator, and those that know me know I’m a bit of a control freak. In my absence Paladin became more of a democracy and it couldn’t go back to the way it was. So, I felt that if I couldn’t run the company the way I wanted to, I didn’t want it anymore and made plans to sell it.” But as the offers started to roll in, Goodman started to have a change of heart. “I thought to myself, I love what I’m doing, why would I give this up and during the process, my cognition improved to the point that I felt I could still run a pharmaceutical company. That’s really when I decided that selling Paladin didn’t mean I’d have to leave the business, that creating Knight was a very real possibility.” And now, for Goodman, it’s all happening very quickly. “People say Paladin was an ‘over-nightsuccess’ but in reality that is not entirely true. It took a lot of time, a lot of luck and patience,” he says. Refreshed, rejuvenated and excited about Knight’s prospects he adds; “I’m a lot more experienced and a lot wiser the second time around. I guess the best evidence is if I didn’t think I could do it again, I wouldn’t have written a cheque for $65 million.” If the Paladin story can be likened to a blockbuster movie, there’s a very good chance that Knight Therapeutics could be Goodman’s blockbuster sequel.

To see this story online visit www.biotechnologyfocus.ca/ looking-to-repeat-the-feat

October/November 2014 BIOTECHNOLOGY FOCUS 19

Q&a

a QuaRTeR CeNTuRY of supporting

CommeRCialiZaTioN Commercialization support has been a very important part of the work at Ag-West Bio from the beginning. Ag-West Bio provides seed capital and helps leverage additional funding for qualiﬁed, early-stage or expanding companies. Brad Bly, Ag-West Bio director of Commercialization, touches on many aspects of commercialization in the following Q&A: Opportunities, like crowd funding; the importance of mentorship; and challenges, such as ﬁnding adequate resources. Q: What are some major milestones for Ag-West Bio?

Over the past 25 years we have had many milestones. Ag-West Bio created the world’s premier ag-bio conference, the Agricultural Biotechnology International Conference (ABIC), held internationally. Ag-West has been recognized by Tourism Saskatoon for its dedication in bringing spectacular events to the city; and Ag-West’s Technology Commercialization Fund has made unique investments, totalling $12.9 million to more than 50 companies.

Q: What are the biggest challenges facing Saskatchewan-based biotechnology companies currently?

Human capital and financial capital. Commercializing bioscience requires heightened business development skills, and compensating individuals with these skills requires sufficient financial capital. Ag-West Bio works to fill these gaps by helping bioscience companies attain mentorship and advisory input, while our Technology Commercialization Fund can provide repayable investment capital for business development purposes.

Q: What are the biggest opportunities for Saskatchewan biotechnology companies?

There are many opportunities, as Saskatchewan is poised to take advantage of the increasing global focus on food security and environmentally sustainable products. We are respected globally for the magnitude and sophistication of our food production. That, and our research infrastructure, gives us 20 BIOTECHNOLOGY FOCUS October/November 2014

a real advantage in developing new and sustainable technologies and products. Two of our companies exemplifying such leader ship are Agrisoma Biosciences Inc. and Open Mind Developments.

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Q: What steps are needed to address quicker commercialization of projects?

Commercializing bioscience is not usually quick; on the up-side however, the benefit from commercialization can be substantial. Mentoring and advice are extremely important steps. Bioscience start-ups that obtain experienced input early on have a better chance of getting to market faster. Similarly, the networks that develop from good advisory input help companies find the strategic commercialization partners needed to accelerate their goals.

Q: How does Ag-West Bio identify companies with strong potential?

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Lots and lots of networking. Over the past five years Ag-West has hosted, on average, more than 15 networking events per year. These events can be as big as ABIC, with more than 500 delegates from around the world, or as small as local bioscience showcases that highlight exciting new research initiatives and companies for an audience of 40. These events, matched with our diligent involvement in the provincial research environment and our participation in relevant initiatives globally, keep us well-informed of opportunities.

Q: What community partners does Ag-West Bio work with and what is their role in the development of commercialization projects? CONTINUED ON PAGE 22

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Q&a

CONTINUED FROM PAGE 20 We have wonderful, informal and mutually beneficial relationships with numerous stakeholders. Just a few of the many partners on speed dial are: U of S Industry Liaison Office, National Research Council – Industrial Research Assistance Program (NRC-IRAP) and Saskatchewan Economic Development Association. Mentoring and education partners such as the Raj Manek Mentorship Program and CETAC-WEST are also very important in increasing the commercialization capacity of the companies we assist.

Q: What are the most prominent grant programs available for Saskatchewan biotechnology companies?

Key grant programs available to companies early on are: NRC-IRAP, which can provide non-repayable funding to promising companies at early stages; the Saskatchewan Agri-Value Initiative (SAVI) enables the development and expansion of companies in their efforts to add value to agricultural products; National Sciences and Engineering Research Council of Canada (NSERC), MITACS, and BioTalent Canada have unique programs aimed at helping companies fill human capital gaps and turn research into commercialization; and of course, the Government of Canada’s Scientific Research and Experimental Development (SR&ED) Tax Incentive Program is available to all technology companies.

Q: Describe the nature of the private equity

financing environment in Saskatchewan. What can be done to enhance this environment for companies to access capital? The private equity financing environment in Saskatchewan is quite limited, but growing. Investment funds managed by Westcap Mgt. Ltd. and PFM Capital Inc. are interested in technology companies in the commercialization and growth stage, while we have seen PIC Investment Group Inc. display interest in promising companies at the pre-commercialization stage. At very early stages, capital is limited. However, the Saskatchewan Capital Network is gaining good momentum in building

22 BIOTECHNOLOGY FOCUS October/November 2014

an angel investment environment and has facilitated investments through its work.

Q: Do you see the crowd funding concept being an avenue of capital for the Saskatchewan biotechnology industry?

Yes: Crowd funding campaigns generally work for raising capital to fund initiatives that have social or environmental benefits. Bioscience research often involves products with significant public good, such as replacing petroleum or chemical-based products with natural bioproducts. I believe there is potential for bioscience companies to obtain crowd funding dollars if they can demonstrate substantial environmental benefit…along with an exciting new concept. Ag technology companies are now raising capital through crowd funding vehicles such as AgFunder.

Q: Why did Ag-West Bio partner with the Raj Manek Mentorship Program and what are the benefits to program participants?

Ag-West Bio has been working with entrepreneurs for 25 years and we know from experience that mentoring and advisory input are critical to success. Simply put: Those that attain these components can succeed; those that don’t, do not succeed. The Raj Manek Business Mentorship Program is high calibre, unique and, we believe, the best of its kind. Program participants can obtain access to suitable experienced mentors. Alternately, the mentors we recommend to the program can exercise the desire to share their knowledge and wisdom. It is all wellmanaged by the small Raj Manek Mentorship Program team who have it down to a well-tuned science.

To see this story online visit www.biotechnologyfocus.ca/qa-aquarter-century-of-supporting-commercialization

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HiGHliGHTs FROM THE BUSINESS OF REGENERATIVE MEDICINE COURSE By Mark Curtis, Business Development Analyst Centre for Commercialization of Regenerative Medicine (CCRM) Ask a student of any age what makes a course great and it always comes down to the teacher. Interesting subject matter helps, but a great teacher can make any subject worth staying awake for. The “students” who attended The Business of Regenerative Medicine course, in Toronto, in July 2014, were treated to more than 40 great “teachers” all sharing their vast knowledge of critical issues relevant to commercialization in the world of regenerative medicine and cell therapy (RM). As experts in their fields – whether they were discussing investing, drug screening, reagents/tools/manufacturing, tissue engineering/regenerative molecules or cell therapy – they all brought frankness, enthusiasm and insight to their talks. Here are some highlights from the 2.5 day course. Reimbursement and accelerated approval were popular themes. It is clear that demonstrating value to payers will be absolutely essential for the successful uptake of RM technologies. Activity in the RM space is picking up as clinical data continues to substantiate the use of live cells for myriad different disease areas and indications. Importantly, the global clinical pipeline shows a greater percentage of ongoing mid-stage RM studies versus early-stage, suggesting a bolus of commercial outputs could be on its way. Pharma is becoming more comfortable working in the RM space, having identified the major bottlenecks and gaps in the industry, which is reflected by increased partnering activity. Accelerated approval regulatory pathways are also contributing to Pharma involvement, as clearer and shorter paths to market are forged. Early-stage biotechnology companies will have to be creative, at least in the short-term, and take advantage of Pharma’s willingness to partner to secure funds for development. Last quarter saw an alltime historical low for first-time venture capital (VC) financings in the biotech space. The financing outlook for mid-to-late-stage companies is more promising, as these companies can still take advantage of the IPO (initial public offering) window that burst open in 2013, but this window is beginning to constrict.

Reimbursement and Accelerated Approval There was particular excitement around accelerated approval regulatory pathways that are being developed to facilitate the commercialization of live cell technologies. As Gil Van Bokkelen, chairman and CEO of Ohio-based Athersys put it, we’re experiencing a “magical era of accelerated approval.” But he also wonders about the fate of cell therapy technologies upon approval, and what is being done to link accelerated approval with reimbursement. This sentiment was echoed by cell therapy consultant Lee Buckler, who pointed to Korea as an example of a country that has been progressive with RM approval, having approved 16 therapies to date – the most of any country in the world – but has failed to support 24 BIOTECHNOLOGY FOCUS October/November 2014

technologies through reimbursement. So far, none are paid for or exported out of the country. Lee stressed the need to show effectiveness in the clinic, worried that if the necessary precautions are not taken we could wake up and find ourselves working in a “withering industry with a plethora of approvals.” It is evident that if these therapies are to gain market access, a dialogue between regulators and payers must be opened imminently. On the topic of reimbursement, we heard from Chris McCabe, a leading health economist based out of the University of Alberta. He laid out the path to de-risking reimbursement through valueengineered translation: completing therapeutic headroom analysis, value-based market access risk assessment, and efficient research and development design. The impact of reimbursement on RM companies could not be illustrated more clearly than in the case of U.S. company Organogenesis, which currently manufactures and markets the two dominant skinsubstitute products for chronic wounds – Apligraf and Dermagraft. Geoff MacKay, CEO, gave a heroic account of corporate survival following a decision by the Centers for Medicare and Medicaid Services (CMS), late in 2013, to bundle payment of cellular products for wound healing with acellular products. To get by, the company slashed $200 million from its cost-structure, cut competition by purchasing the primary competing product Dermagraft from Shire, and put an end to its R&D program across multiple indications to shift its focus to being a manufacturer of products solely for wound healing. Attendees also discussed the notion of conditional approval of cell therapies – essentially leap-frogging Phase 3 studies and launching a Phase 4 study post-marketing. Arnold Caplan, professor at Case Western Reserve University and founder of the course seven years

Across Canada vided some keen insight into issues around the logistics of moving live cells. She believes that the clear-cut distinction in a RM business model should be dictated by patient-specific versus bulk cell therapies rather than by specific cell types. Different therapies will have different requirements in terms of intake and shipping. Dr. Carmen says frozen-in and frozen-out is generally easy to manage; however, receiving cells fresh, and particularly having to ship cells fresh, comes with significant logistical challenges. Patient-specific therapies have the greatest logistical and regulatory burden, and hence cost, as they must be tracked within a chain of custody. They are shipped fresh, which means they have a short shelf life. Advances in logistics and the supply chain will be critical in driving down costs of patient-specific therapies.

Tissue Engineering and Drug Screening

ago, argues this might be an effective means of getting cell therapies to the bedside more rapidly by approving them with only preliminary safety and efficacy data. A precedent can be found with Japan, where the government has recently implemented a conditional approval system. Cell therapy developers are only required to have a single, albeit larger, Phase 1 study to get marketing approval. Remarkably, all cell therapies currently approved in Japan are entitled to reimbursement.

A company to watch in the 3D printing space is Aspect Biosystems, which is out to create disruptive tissue engineering technologies, “on demand.” Sam Wadsworth, co-founder and director of Biology for this Vancouver-based start-up, explained that whole organ replacement is their goal, but we must first focus on understanding the individual building blocks of the organ before we can pursue synthesizing whole organs. The company’s first product focus will be airway tissue followed by liver. A more immediate use of these tissue constructs is in drug screening, which is evolving to use multicellular, 3D constructs as a more predictive means to screen for toxicity during preclinical development. Between 1990 and 2010, there were 160 Phase 3 or postmarket withdrawals due to toxicity issues. Estimates for the all-in cost of developing a drug have ballooned to $4 billion (when accounting for development costs of alternate leads that didn’t make it into the clinic). Increasing the current success rate from 10-20 per cent would save a pharmaceutical company ~$1.5 billion in development costs. So, this is big business. While screening for cardiac drugs presents a large market opportunity, targeting disease areas where animal models have failed to translate to humans is a smart business model. Dr. Wadsworth pointed out that airway fibrosis is a great example of this, with 100+ treatments that have proven successful in mice, but zero that have gone on to succeed in the clinic.

Manufacturing, Tools, and Logistics

Bottom-Line

Cost of goods in manufacturing cell therapies is typically high, primarily due to expensive growth factors required to drive differentiation of stem cells to specific cell types. Nick Timmins, director of Product and Process Development at CCRM, gave an overview of some of the cost reduction efforts being pursued at the Centre, using natural killer (NK) cells as an example. NK therapies for cancer are expected to have an all-in cost per patient of $100,000 to $150,000. Through bioprocess optimization, he and his team were able to reduce the cost of consumables from $20,000 to ~$8,000, by minimizing media, serum replacement, and growth factor requirements. Bioprocess optimization must occur early in cell therapy development. Once a therapy’s protocol is locked in from a regulatory perspective, it is very difficult to go back and carry out this type of cost of goods reduction. Dr. Timmins explained some of the tools that will facilitate the efficient production of cell therapies, including analytical devices for timely measurements of process quality. He urged us to think beyond scale-up and scale-out, and focus on yield. Jessica Carmen, Business Development manager at Lonza, pro-

Reimbursement is everything. While accelerated approval will get RM developers to approval, ultimately they will need to demonstrate value to the patient and cost-effectiveness to payers. Without this, easing of regulatory burdens will do little to facilitate the adoption of RM technologies and unlock their health benefits. Product and process development, and scale-up, will be essential for the industrialization of the cell therapy industry and cost of goods reduction, which will give a boost to cost-effectiveness. Logistics and a consistent, reliable, global supply chain for cellular products will help cement cell therapies as the next pillar in health care. The Business of Regenerative Medicine course was hosted in Canada, for the first time, by the Centre for Commercialization of Regenerative Medicine. To see a copy of the program, please visit http://events.ccrm. ca/program/preliminary-schedule/ To see this story online visit www.biotechnologyfocus.ca/highlights-from-thebusiness-of-regenerative-medicine-course October/November 2014 BIOTECHNOLOGY FOCUS 25

NEW PRODUCTS Microplates BrandTech Scientific, Inc. announces the availability of inertGrade™ microplates for the cultivation of non-adherent cell lines, spheroids, and stem cells in a 96-well plate format. These polystyrene plates are manufactured in one of the largest, most modern cleanroom facilities for laboratory disposable items in the world. Rather than the more common treatments to enhance the natural hydrophobic characteristics of polystyrene, these new microplates are treated with a proprietary

high recovery. The column is compatible with organic solvent and aqueous mobile phase. It is designed for rugged stability and very low carry-over.

Web: www.thermoscientific.com/ mabpacHIC Liquid Chromatography Shimadzu Scientific Instruments has introduced two new integrated liquid chromatography systems, Prominence-i and Nexera-i.The iSeries systems feature innovative, intuitive and intelligent design so users can begin building the lab of the future. Through the integration of these systems with LabSolutions software, Shimadzu fosters a new relationship between users and instrumentation. The data acquired by the Prominence-i and Nexera-i via interactive communication mode (ICM) is sent to a lab’s data center by the LabSolutions network and managed uniformly by a server. ICM allows users to perform operations such as purging mobile phases and con-

hydrogel. This creates a hydrophobic interface between the surface of the plastic and cellular material to inhibit cell and protein attachment. This unique low-binding microplate surface successfully suppresses the adhesion of a wide variety of adherent cell lines, can enhance the formation and maintenance of uniform spheroid cultures, and can inhibit early differentiation of stem cells or neurospheres. The microplates are available with round, flat, or curved bottoms in clear, white, black, and colored with transparent bottoms.

Web: www.BrandTech.com Columns Designed for separation of monoclonal antibodies (MAbs) and related biologics by hydrophobic interaction, the Thermo Scientific™ MAbPac™ HIC-10 LC column is a high resolution silica column. The MabPac HIC-10 column’s unique chemistry provides high resolution, excellent biocompatibility, and selectivity that is different from other HIC columns on the market and is able to separate many native mAbs, fragments, aggregates, oxidation variants, PEGylated mAbs and ADC. The column’s proprietary chemistry is designed to provide the new selectivity in a high-resolution column with excellent biocompatibility and

Western Blotting System The TransBlot® Turbo™ Transfer System from Bio Rad Laboratories is a high-performance western blotting system designed to provide rapid transfers with high efficiency. It combines traditional blotting techniques with modern filter paper and buffers, allowing rapid transfer of proteins from gels to membranes with minimal preparation time. The system integrates a high amperage power supply that directs current between a built-in platinized titanium anode and a stainless steel cathode. This enables blot transfer of protein in as little as three minutes without sacrificing performance. The entire system is in a readyto-use format that allows researchers to obtain their results faster and easier, with reproducibility that is difficult to achieve using traditional blotting methods. Paired with Trans-Blot Turbo Transfer Packs or Trans-Blot RTA Kits, it provides a complete solution for incredibly fast, high efficiency transfers-including high molecular weight proteins. Web: www.bio-rad.com

firming analytical results from anywhere in the facility with a smart device. It also permits easy access to a system installed in a closely supervised area such as under a hood where highly active ingredients are being analyzed.In addition to the temperature control function in flow cells, the systems harness new technology for detector optical systems called TC-Optics, which provides excellent baseline stability. This ensures high precision of validation and quantitation of trace components. The i-Series also allows the direct injection of highly concentrated samples without dilution. Even when injecting ultra-low sample volumes, the systems provide repeatability of one percent or less. The i-Series can be used for a wide range of industries such as pharmaceutical, biopharmaceutical, chemical, environmental, food, and academic teaching.

Web: www.ssi.shimadzu.com/products/product.cfm?product=i-Series_1.

26 BIOTECHNOLOGY FOCUS October/November 2014

Fluid extraction The Supercritical Fluid Technologies (SFT) bench top supercritical fluid extractor (SFE) model SFT-110XW is the latest addition to the SFE product line. The completely redesigned restrictor valve with integrated micrometer allows for very precise flow control, which is essential for demanding applications. A completely removable oven lid and large side panel allow the user easy access to the high pressure vessel(s). An indicator light on the SFT-10 pump module alerts the user to proper operation of the Peltier pre-cooler, ensuring CO2 is maintained in the liquid state. A robust outlet from the restrictor ensures that users will not accidently damage the outlet tube when inserting it into the collection container. Extraction vessels up to 500 ml, single or dual configurations, afford maximum versatility.The SFT-110XW SFE is well suited for both research and process development applications. Web: www.supercriticalfluids.com.

NEW PRODUCTS Liquid Handling Pumps VICI Valco’s Cheminert Model M6 and M50 liquid handling pumps are a new generation of pumps for precision handling of liquids and/or gases. Producing a bidirectional pulseless flow with a range of over six orders of magnitude (5 nL/min to 5 mL/

weighing cell, which measures the weight of each individual fill with an accuracy rate of +/-0.002g. Additionally, it handles filling requirements for clinical trials and medium batch production with peristaltic aseptic filling technology from 0.1 to 100ml fill sizes. For operator convenience, the FPC50W new card:Layout 1 1/31/2013 9:09 AM Page 1 system can also accommodate a wide range of vial sizes and neck finishes without the need of extra format parts. The weighing cell is connected to the filling system, allowing for fully automatic recalibration of the pump throughout the batch. Additionally, the unit can employ a changeover of the single-use fluid path, which includes

pharmaceutical grade silicone tubing and plastic filling needles, to adapt to multiple batch processes or different product fill volumes. The system is equipped with an automated precision turntable infeed, an in-line filling station, and a rubber plug closure applicator that is capable of filling and capping vials at rates up to 20 vpm. The FPC50W comes standard with a onetouch 12 inch screen/HMI for easy setting of all process parameters. If desired, the machine can be configured for password protection in three levels ensuring batch security ease of validation.

Web: www.watson-marlow.com

SRC101

min for the M6 Pump; 1 µL/min to 25 mL/ min for the M50 Pump), they represent an option to syringe pumps by eliminating the need for refill cycles and syringe changes. The M6 and M50 are positive displacement pumps, which means they are self-priming and tolerant of any gas which may find its way into the fluid lines. There is no separate fill cycle, and the capacity is unlimited. RS-232 and RS-485 communication protocols are incorporated into the microprocessor-driven controller. (USB interface requires an adapter.) The included software package controls flow rates, flow direction and metered volumes. While the standard package includes software and instructions to run the M6 as a stand-alone unit, the software can also integrate a multi-position stream selection valve.

Web: www.vici.com Automated Filling System WatsonMarlow offers its Flexicon FPC50W, an operator-friendly, fully-automated filling,

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C87 Which products are used in your lab? A B C D E F G

Analysis Instruments Basic Lab Equipment Chemicals/Biochemicals Chromatography – Gas Chromatography – Liquid Filtration, Water Purification LIMS

H I J K L M Y

Liquid Handling & Sample Prep Microscopes, Optics, Cameras Safety & Hygiene Spectroscopy Testing Systems/Equipment Vacuum Equipment None of the Above

C88 Product Development Stage (check all that apply)

COMPANYs PRIMARY BUSINESS ACTIVITY

plugging and capping system that offers accuracy, flexibility for aseptic filling in clinical trials and scale-up applications. The FPC50W is equipped with a Wipotec

Yes

BIOTECHNOLOGY FOCUS

C Specify

A Research/Development B Pilot/Scaleup

C Production/Manufacturing D Tech Transfer E Not applicable

C89 Are you building a new lab?

Yes

C90 We have the following enewsletters: 1 2 3 4

Biotechnology Focus eBulletin Laboratory focus eBulletin BioPharma BioMedical

5 Health Care 6 Agri-Food 7 Clean Tech 8 Industry Inte

For a quick response please fax: 905-727-4428 or e-mail: circulation@promotive.net October/November 2014 BIOTECHNOLOGY FOCUS 27

CALENDAR OCTOBER 2014

Tel: (902) 421-5705 Email: info@bionova.ca Web: www.bioportatlantic.ca

October 5-8 ABIC 2014 Venue: Saskatoon, SK Tel: 306-668-2650 Email: abic2014@agwest.sk.ca Web: www.abic.ca/abic2014/

October 20-23 30th Annual International Conference on Soils, Sediments, Water and Energy Venue: Amherst, MA Tel: 413.549.5170 Email: brenna@aehsfoundation.org Web: http://www.aehsfoundation.org/ Events.aspx

October 7-8 Bio Investor Forum Venue: San Francisco, CA Email: bd_registration@bio.org Web: www.bio.org/events/conferences/ 13th-annual-bio-investor-forum

October 12-16 MS&T – Materials, Science & Technology 2014 Venue: Pittsburgh, PA Tel: 240-646-7054 Email: customerservice@ceramics.org Web: http://www.matscitech.org/

October 15-17

October 20-21

Neuroscience 2014 Venue: Washington, DC Tel: (202) 962-4000 Email: program@sfn.org Web: http://www.sfn.org/Annual-Meeting/ Neuroscience-2014

November 24-26

AusBiotech 2014 National Conference Venue: South Yarra, Australia Tel: (03) 9828 1400 Email: admin@ausbiotech.org Web: https://www.ausbiotech.org/

Genomics: The Power & The Promise Venue: Ottawa, ON Twitter: #powerofgenomics, @GenomeCanada Web: www.genomecanada.ca

NOVEMBER 2014

November 25

November 2-6

OSCI Stem Cell Symposium Venue: Toronto, ON Web: http://ontariostemcell.ca/node/405

November 12-15

Bioport Atlantic 2014 Venue: Halifax, NS

November 15-19

October 29-31

AAPS Annual Meeting Venue: San Diego, CA Tel: 703-243-2800 Email: Meetings@aaps.org Web: www.aaps.org

Canadian Science Policy Conference Venue: Halifax, NS Web: http://cspc2014.ca/

Venue: Dusseldorf, Germany Tel: 416-598-1524 Email: messeduesseldorf@germanchamber.ca Web: www.medica-tradefair.com

MEDICA 2014

November 30-December 5 2014 MRS Fall Meeting & Exhibit Venue: Boston, MA Tel: 724-779-3003 Email: info@mrs.org Web: www.mrs.org

Ca so life

Don

Company & Advertiser Index COMPANY

Page Website

Ag-West Bio..........................................................................................................2.............................................................................................www.agwest.sk.ca AlbertatBay.........................................................................................................23..................................................................................... www.albertatbay.com Bereskin & Parr ..................................................................................................17...................................................................................www.bereskinparr.com Best Western Victoria Park Suites...................................................................23....................................................................................www.victoriapark.com Bio Rad Laboratories.................................................................................... 26........................................................................................ www.bio-rad.com BrandTech Scientific Inc............................................................................... 26...................................................................................www.BrandTech.com Canadian Government Executive...................................................................21.....................................................www.canadiangovernmentexecutive.ca Centre for Commercialization of Regenerative Medicine............................ 10............................................................................................... www.ccrm.ca Children’s Miracle Network...............................................................................7.................................................................www.childrensmiraclenetwork.ca Concordia Healthcare Corp.......................................................................... 10.................................................................................www.concordiarx.com Eppendorf...........................................................................................................32.......................................................................................... www.eppendorf.ca Knight Therapeutics................................................................................... 9,10................................................................................www.gud-knight.com OncoGenex Pharmaceuticals Inc................................................................... 6...................................................................................www.oncogenex.com Oncolytics Biotech Inc.................................................................................. 6........................................................................ www.oncolyticsbiotech.com Nuvo Research Inc........................................................................................ 6...............................................................................www.nuvoresearch.com Ontario Bioscience Innovation Organization...............................................13......................................................................................................www.obio.ca POI Business Interiors.......................................................................................31........................................................................................................ www.poi.ca Qu Biologics.................................................................................................. 9.................................................................................. www.qubiologics.com Thermo Scienitific........................................................................................ 26..........................................................................www.thermoscientific.com VICI Vlaco..................................................................................................... 27.............................................................................................. www.vici.com VWR.......................................................................................................................5......................................................................................................... ca.vwr.com Zymeworks Inc. ........................................................................................... 10................................................................................. www.zymeworks.com 28 BIOTECHNOLOGY FOCUS October/November 2014

Read it here first. Canada’s foremost news source on the Canadian life science industry. Don’t get left behind.

www.biotechnologyfocus.ca

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By Murray McLaughlin

The BioEconomy and Sustainability

Role of Cellulosic Biomass

I Murray McLaughlin

is Executive Director of the Bioindustrial Innovation Centre and President and CEO of the Sustainable Chemistry Alliance.

n my area of bioindustrial development we see our vast Canadian resources in agriculture and forestry that provide tremendous amounts of biomass which is not used today - Forestry bark; sawdust; straw (wheat, barley, etc.); corn stover; and purpose grown crops. Some people call it waste, some underutilized opportunity - regardless it is biomass that creates opportunities to create more sustainability for the future. There are those that question the efforts to work with biomass and convert it to chemicals, energy, biofuels, and biomaterials because they are unsure of the long term sustainability and meeting the promises made or the costs of getting there. Certainly in Canada, it has become very clear that our forestry industry will not be sustainable without moving to a broader focus on bioindustrial opportunities - chemicals, fuels, energy, new uses for wood, and improved processes from our present biorefineries for wood. In agriculture the use of our biomass is a new component of our farm production that is being recognized as a way to improve the bottom line at the farm gate. As Atul Bali, Competitive Green Technologies, describes it - “We need to think of agriculture (and forestry), not just at the farm and traditional uses of crops, but think BEYOND.” Where can we see products from agriculture impact our daily lives - automobiles, planes; clothing, tires, furniture, plastics, paints; cosmetics, running shoes, and the list goes on. Think about making products sustainably and you can understand the importance of agriculture and forestry, (the producers of cellulose); in the 21st Century (The Sustainable Century). I do worry about our ability in Canada to really move to building sustainable industries from cellulose biomass because we are not willing to step up and support the movement in the ways needed, - be it funding for Research and Development; investments for start-ups; ensuring our regulatory system is modernized to handle new technologies in a expeditious and timely manner; having appropriate taxation; etc. to ensure Canada is business friendly. However, let me put worry in context, because it reflects the Canadian cautious way of always getting to the right decision, so I am fully confident that we will make the decisions needed to be the global leader

30 BIOTECHNOLOGY FOCUS October/November 2014

in cellulosic biomass as a key part of our bioeconomy. So here I am talking about the importance of sustainability for our cellulosic industry and the importance it has in ensuring a future for agriculture and forestry that will be viable long term. However, I am assuming everyone thinks about sustainability the same way, and as we all know, we should never “assume.” We hear the words “sustainable” and “sustainability” on a regular bases, but what does it mean? Is it about people and culture, our environment, jobs or money? Is it about urban or rural? Is it something for you and me or others to worry about? In actual fact, sustainability is about all of the above and more. It is about taking what we need to live now, without jeopardizing the potential for people in the future to meet their needs. A sustainable activity should be able to continue forever. Bringing this back to the discussion on cellulosic biomass and its importance to the forestry and agriculture industry, it is just as important to a sustainable bioeconomy. Hence at Bioindustrial Innovation Canada (BIC) we are strongly focused on the development of green and sustainable technologies for the future and cellulosic biomass is a key part of that focus. Looking at the knowledge we have gained about biomass and biomass opportunities, many will think that we are on the verge of break through ideas, however that is the easy part and is already done - our challenge now is to implement the ideas by staying focused on the tasks. Greg Van Alstyne, futurist, stated “The secret to achieving breakthrough is to grasp and combine the “normal” and the “new”. Innovation is inevitably “the new normal.” This quote fits nicely with cellulosic biomass and its use to create the new normal in agriculture and forestry - sustainability for the 21st Century. As my friend Don McCabe, farmer and VP of Ontario Federation of Agriculture (OFA) would tell you, when he hears the term “Biomass (corn stover, straw, etc.) is just a waste material”, he will quickly correct you - “In agriculture there is no such thing as waste, just underutilized resources waiting for us to find the right opportunity.” So, cellulosic biomass is the opportunity for us to add value to Canadian agriculture and forestry. We need to recognize this and move forward with the right programs to ensure Canada is a Global Leader in the bioeconomy. This is important to creating the sustainability needed to ensure agriculture and forestry continue to be thriving industries.

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