Biotechnology Focus October/November 2015

INSIGHTS FOR THE LIFE SCIENCE INDUSTRY

October/november 2015 VOLUME 18, NUMBER 5

A crucial moment in time for

stem cell R&D Special Report:

Surveying the Biotechnological HR Landscape

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contents October/November 2015 – VOLUME 18 – NUMBER 5

8

11

15 The Evolving Clinical Trial Landscape in Canada

FEATURES Treating Acute Coronary Syndrome: This time it’s personal

Thanks to the promise of precision medicine, Dalcetrapib, a failed heart drug is getting a second shot at a Phase 3 trial (By Shawn Lawrence)

A crucial moment in time for stem cell R&D

Opinion

11 A crucial moment in time for Stem Cell R&D Traditionally a powerhouse in stem cell research, Canada is at risk of falling back to the pack (By Joe Sornberger)

13

Banking on CORD blood

Recent technological innovations in umbilical cord blood expansion suggests that a future of better therapies no not that far away (By Lisa Willemse)

18 Canada and Subsequent Entry Biologics Merck Canada’s Chirfi Guindo discusses opportunities around Subsequent Entry Biologics, and how Canada can capitalize (By Shawn Lawrence)

IN EVERY ISSUE

Special Report:

Surveying the Biotechnological HR Landscape

First-of-its-kind employee survey of Canada’s biotechnology industry brings future of industry into focus (By Jeff Mackey)

www.biotechnologyfocus.ca

15

aCROSS CANADA

22

The Last Word

The evolving clinical trial landscape-an update (By Diane Gajewczyk) Corporate governance for a vibrant life science ecosystem (By Nathalie Richard)

October/November 2015 BIOTECHNOLOGY FOCUS 3

PUBLISHER’S NOTE

Celebrating with Canada’s stem cell research community; and Results are in for our HR survey!

Recently, I had the pleasure of attending the Till & McCulloch meetings, Canada’s premier stem cell research event. Named for the University of Toronto researchers Drs. James Till and Ernest McCulloch who discovered transplantable stem cells in 1961, it certainly lived up to its billing with more than 450 leading stem cell scientists, clinicians, bioengineers and ethicists, as well as representatives from industry, government, health and NGO sectors from around the world in attendance.

Among the event highlights was a special Award Lecture presented by University of British Columbia researcher Dr. Timothy Kieffer presided over by Dr. Till himself. Dr. Kieffer landed the prestigious slot by developing a protocol that converts human stem cells into glucose-responsive insulin-secreting cells capable of reversing diabetes in mice.

While not claiming to cure diabetes, Dr. Kieffer’s paper which was published in Nature Biotechnology last year, is considered an important step to curing a disease that affects over nine million Canadians.

The agenda also included a special session with the Canadian Stem Cell Foundation, presented by James Price, Foundation president & CEO, and Dr. Alan Bernstein, chair of the Board of Directors and president & CEO of the Canadian Institute for Advanced Research.

Coupled with the great science on display via a plethora of posters, there were some interesting debates on the state of stem cell research both here and abroad.

“Canada is a leader in regenerative medicine research and the Till & McCulloch Meetings is our chance to shine in front of industry, government and invited guest speakers from around the world,” said Dr. Michael May, president and CEO of the Centre for Commercialization of Regenerative Medicine, one of the hosts of the event. He further commented that the conference was a homecoming for the community and an opportunity for collaborations to flourish. He also mentioned some of the great Canadian companies working in the space.

So with so much going right, where does the debate on the state of Stem Cell Research in Canada come into play?

As Joe Sornberger, the author of the University of Toronto Press book Dreams & Due Diligence: Till and McCulloch’s Stem Cell Discovery and Legacy explains in his column in this issue, many of Canada’s elite stem cell/regenerative medicine researchers are on the verge of delivering new treatments for a number of diseases, but they are producing this kind of world-leading work remarkably on a shoestring budget, with diminishing funding resources. He cites the impending closing down of the Stem Cell Network as well as the absence of a comprehensive national strategy to help move stem cells out of the lab and into the clinic as key challenges to Canada’s stem cell research community.

Moreover, he says we’ve reached a crucial moment in time for stem cell R&D. It’s an interesting piece, and definitely a much needed call to action.

Lisa Willemse keeps the stem cell discussion going in her editorial piece. Her article focuses on recent technological innovations by Canadian researchers in umbilical cord blood expansion and how by improving the use of these life-giving cells they are delivering on a the promise of a future of better therapies.

In addition to these two pieces, this issue of Biotechnology Focus includes many other stories on hot industry topics including: precision medicine, biosimilars and clinical trials.

Rounding things out, we’re excited to announce that the results are in on our first-ever independent survey identifying what employees in the life science and biotech space value in their workplace. Be sure to check out the special pull out section to see the results. Overall, we hope you enjoy this issue as much as we enjoyed putting it together!

4 BIOTECHNOLOGY FOCUS October/November 2015

PUBLISHER/ EDITOR-IN-CHIEF

Terri Pavelic

SENIOR WRITER

Shawn Lawrence

CONTRIBUTING WRITERS

Diane Gajewczyk

Jeff Mackey

Joe Sornberger

Lisa Willemse

Nathalie Richard

Director, Content & Business Development

José Labao

GRAPHIC DESIGNER CONTROLLER MARKETING MANAGER

Elena Pankova John R. Jones Mary Malofy

CIRCULATION DIRECTOR Mary Labao circulation@promotive.net Tel: 289-879-4272

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Biotechnology Focus is published 6 times per year by Promotive Communications Inc. 23-4 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 – 23-4 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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R & D NEWS Destructive disease shows potential as a cancer treatment

Mads Daugaard, an assistant professor of urologic science at UBC Scientists at the University of British Columbia, Vancouver Coastal Health and the BC Cancer Agency have discovered a protein from malaria that could one day help stop cancer in its tracks.

This new approach, which halted the growth of various tumours in mice, was based on a discovery by collaborators at the University of Copenhagen. While exploring why pregnant women are particularly susceptible to malaria, they found that the mosquito-borne parasite produces a protein that binds to a particular type of sugar molecule in the placenta.

That discovery led to another: that same sugar molecule is also found in most cancers. This commonality is understandable, because both cancers and placentas grow rapidly, often pushing aside other tissues in the process.

The Copenhagen and Vancouver researchers realized that the sugar molecule thus could be a target for anti-cancer drugs,

and that the malarial protein, called VAR2CSA, could provide the tool for carrying such drugs to tumours.

“Scientists have spent decades trying to find biochemical similarities between placenta tissue and cancer, but we just didn’t have the technology to find it,” said project leader Mads Daugaard, an assistant professor of urologic science at UBC and a senior research scientist at the Vancouver Prostate Centre, part of the Vancouver Coastal Health Research Institute. “When my colleagues discovered how malaria uses VAR2CSA to embed itself in the placenta, we immediately saw its potential to deliver cancer drugs in a precise, controlled way to tumours.”

To test that theory, Daugaard and colleagues enlisted the expertise of John Babcook and his team at The Centre for Drug Research and Development (CDRD). They attached a novel toxin to VAR2CSA and treated hundreds of normal and cancer cell lines. The drug compound specifically tar-

geted and killed more than 95 per cent of the cancer cell lines.

The drug was then tested on mice that were implanted with three types of human tumours. With non-Hodgkin’s lymphoma, the treated mice’s tumours were about a quarter the size of the tumours in the control group. With prostate cancer, the tumours completely disappeared in two of the six treated mice a month after receiving the first dose. With metastatic breast cancer, five out of six treated mice were cured from metastatic disease. The mice showed no adverse side-effects, and their organs were unharmed by the therapy. The results were published in Cancer Cell.

“This is an extraordinary finding that paves the way for targeting sugar molecules in pediatric and adulthood human cancer, and our groups are vigorously pursuing this possibility together,” said Poul Sorensen, a UBC professor of Pathology and Laboratory Medicine and distinguished scientist with the BC Cancer Agency and co-senior investigator on the study.

“There is some irony that a disease as destructive as malaria might be exploited to treat another dreaded disease,” said Ali Salanti, a professor of immunology and microbiology at the Centre for Medical Parasitology, at University of Copenhagen.

Two companies, Vancouver-based Kairos Therapeutics and Copenhagen-based VAR2 Pharmaceuticals, are developing the compound for clinical trials in humans, which will take another three to four years.

To see this story online visit http://biotechnologyfocus.ca/?p=15987

CQDM and AiF Projekt GmbH join forces to enhance biopharmaceutical R&D internationally CQDM and AiF Projekt GmbH, the project management agency of BMWi, the German Ministry for Economic Affairs and Energy, are teaming up on a new international funding initiative entitled the Canada/Germany joint program, within CQDM’s Canada/Europe joint funding initiative.

This collaboration agreement aims to fund and facilitate collaborative research between the two countries, financing the development of novel and potentially transformative next-generation technologies or tools with the potential to improve, enhance or accelerate the state of the art and/or development process.

For Canada, the program is focused on biomedical research aimed at developing 6 BIOTECHNOLOGY FOCUS October/November 2015

tools and technologies to improve, accelerate or enhance the drug discovery process.

The German funding system is generally technology open. The program is not intended to fund research on new drugs for specific therapeutic segments. According to the two organizations, this gives the opportunity to R&D projects jointly conducted

by Canadian and German private and public research entities to develop new expertise and to expand into new markets.

Each project will benefit from a funding between $500,000 and $1 million over a two or three year period. The contribution

by Germany, intended to the German research entities involved in the project, will be provided by the Central Innovation Program for SMEs (ZIM Cooperation projects)

and is technology open. Funds provided

by Canada, for the Canadian research organizations collaborating on the project, will come from CQDM and is aimed at creating value for the life sciences and biopharmaceutical industry.

To see the call for proposals and to know more about the Program’s requirements, we invite you to visit http://www.cqdm. org/en/programs-and-competitions/ canada-germany-program.php.

Applicants have until January 15th, 2016 to apply.

German researchers are invited to submit their letters in intent with AiF Projekt GmbH and Canadian researchers with CQDM. To see this story online visit http://biotechnologyfocus.ca/?p=15920

BUSINESS CORNER Antibe Therapeutics acquires Citagenix Antibe Therapeutics Inc. has entered into a share purchase agreement to acquire an 85 per cent interest in Citagenix Inc., a Montréal-based sales and distribution company with a focus on regenerative medicine.

Citagenix markets, manufactures, sells and distributes a broad range of dental and orthopaedic surgical products, as well as a line of German-made surgical instruments. The company has a fully established commercial infrastructure with annual revenues of $9.7 million for the 12-month period ending August 31, 2015.

Antibe believes there is opportunity for organic growth of the existing product

portfolio, plus the addition of new products and expansion into the U.S., Europe, Middle East and Asian markets in the near term.

Daniel Legault, CEO of Antibe, commented, “We believe this transaction is transformative for Antibe. We have obtained a commercial infrastructure and we are excited for the op-

Precision NanoSystems raises $13.4 million in Series A financing Precision NanoSystems, Inc. (PNI), a nanomedicine company, says it has completed a $13.4 million private Series A financing.

The investment was led by 5AM Ventures and Telegraph Hill Partners, with additional participants that include the Rising Tide Fund and other individual investors. PNI says it will use the funding to expand product development, increase manufacturing capacity and accelerate commercialization of its novel instruments and reagents platform.

PNI is a manufacturer of instruments, kits and reagents for the nanomedicine market including tools for drug development and cell-specific delivery to study, diagnose and treat disease. PNI’s flagship product, the NanoAssemblr™ Benchtop instrument, allows scientists to rapidly develop novel nanomedicine drug candidates for pre-clinical testing. PNI has sold over 60 NanoAssemblr Benchtop instruments worldwide to leading pharmaceutical and biotechnology companies as well as several elite academic institutions.

PNI’s scale-up platform for producing nanomedicines is in beta test at several locations and under development for the Good Manufacturing Practice (GMP) manufacture of nanomedicines. PNI’s instru-

portunity to work with our new colleagues at Citagenix. The Citagenix product portfolio and large existing customer base provide a platform to grow the business and we expect sustained growth of these revenues over time by selectively adding class-leading and innovative products to our product line.”

Antibe has agreed to further develop the sales capacity of Citagenix by investing an additional $2.25 million in Citagenix in the next year. Antibe adds that it will acquire the remaining 15 per cent interest in Citagenix upon fulfillment of a regulatory condition. Citagenix will operate as a subsidiary of Antibe.

To see this story online visit http://biotechnologyfocus.ca/?p=15911

Aeterna Zentaris closes Québec office, moves operations to Charleston, SC

ments and consumables aid clients in developing a wide range of lipid and polymer nanoparticles for the delivery of genetic medicines (such as DNA, siRNA, mRNA, miRNA, CRISPR, etc.), small-molecule drugs and proteins.

Concurrent with the financing, Andy Schwab, founder and managing partner of 5AM Ventures, Deval Lashkari, senior partner of Telegraph Hill Partners, Andrew Booth, CFO STEMCELL Technologies, and Joe Victor, CEO of Startide Sciences, will join existing co-founding directors James Taylor and Dan Nixon on the company’s board. Victor will take on the role of executive chairman.

Aeterna Zentaris says it intends to shut down its Québec City head office by the end of December, 2015.

The company, which specializes in developing oncology and endocrinology treatments with a focus on women’s health, did not confirm how many employees would remain with the company, but did say that the bulk of the Québec City office’s functions and operations will be transferred to its Charleston, South Carolina facility. Additionally, the company’s chief financial officer Dennis Turpin is leaving the company.

“This decision was difficult but necessary,” said company chairman, president and CEO, David A. Dodd. “After a comprehensive review, the company’s board reached the decision to take these actions, including the closure of our Quebec City office by the end of December, making all associates based there redundant.”

The company also reported chief accounting officer Keith Santorelli will fill in as a replacement for Turpin in the interim and that a comprehensive search for a CFO and the other finance and accounting personnel required is underway.

Aeterna Zentaris was founded in 1991 as Aeterna Laboratories. In May, 2014, the company selected Charleston, SC as the new location for its North American business and global commercial operations.

To see this story online visit http://biotechnologyfocus.ca/?p=15882

To see this story online visit http://biotechnologyfocus.ca/?p=15948 October/November 2015 BIOTECHNOLOGY FOCUS 7

By: Shawn Lawrence

Precision Medicine

Treating Acute Coronary Syndrome: This time

it’s personal

Robert McNeil, managing director of Sanderling and CEO of DalCor

Thanks to the promise of precision medicine, a heart drug ditched three years ago by European multinational Roche Holding AG (Roche) is getting a second shot at a Phase 3 trial.

A

s a potential treatment for patients with acute coronary syndrome (ACS), the drug, Dalcetrapib, had in its first go around made it all the way through to Phase 3 before Roche halted its development due to a lack of meaningful efficacy results. Now, a Sanderling Ventures spin-out company in Montréal, DalCor Pharmaceuticals, has licensed the drug from Roche and launched a new Phase 3 trial to prove its viability as a personalized cardiovascular therapy. This will be a first in cardiovascular disease.

As part of this new trial, DalCor says it will work with the Montréal Heart Institute and Roche Diagnostics to screen more than 33,000 patients across 30 countries in the hopes of identifying 5,000 patients who stand to benefit from its ability to boost “good” cholesterol because they have the right genetic profile. As Robert McNeil, managing director of Sanderling and CEO of DalCor explains, the big difference is that this time personal8 BIOTECHNOLOGY FOCUS October/November 2015

ization is the name of the game.

“It all comes down the underlying principle behind precision medicine, a patients’ different genetic profile impacts how useful or harmful a drug is to the system,” he says, elaborating that not all drugs are one size fits all. In the case of Dalcetrapib, two Montréal Heart Institute (MHI) researchers Drs. JeanClaude Tardif and Marie-Pierre Dubé, have shown that patients with a certain genotype on a specific gene called ADCY9 (adenylate cyclase 9 on chromosome 16) exhibited reduced cardiovascular risk when treated with dalcetrapib as compared to placebo.

It was the pharmacogenomics data presented in this study that convinced McNeil that Dr. Tardif’s findings were groundbreaking. He remembers vividly his excitement when he heard about these findings from Dr. Tardif first-hand.

“A mutual friend called me up one day and said, “I’d like you to come and listen to a story

of a colleague of mine that you might find interesting, about a possible drug.” So about a week later I was in his office with Dr. Tardif and basically, he showed me the results of Dalcetrapib in the original Roche Phase 3 trial, which I was aware of and he said, “But if you take what we discovered and look at a particular genotype, there is quite a different outcome.””

“Basically, there is a distinct difference in how Dalcetrapib performs in a group of people with a certain genetic composition,” says McNeil, citing that in Tardif and Dubé’s study, this patient group had a 39 per cent decrease in combined clinical outcomes of heart attacks, strokes, unstable angina, coronary revascularizations and cardiovascular deaths when taking the drug.

“First a 39 per cent decrease in hard outcomes on top of statins is a very big improvement, and second, it was the first time in cardiovascular disease that we had evidence that a drug could impact those who have a certain

Precision Medicine “We met with experts, clinicians and geneticists and they were all curious, wondering how true those data were. And we all agreed we had to go for it; we had to do a Phase 3 trial in this target population. We saw this as an opportunity because having this type of data is very rare.”

— Louise Proulx, DalCor’s chief development officer genetic composition,” continues McNeil.

McNeil had always thought that eventually someone would try to take a precision medicine approach to treat cardiovascular disease, especially with advancements in the field deepening our understanding of the molecular and genetic basis of more and more diseases. Now, Dr. Tardif’s study had shown definite proof that this was the case.

“I’ll say a lot of people did not pick up on it with the vast majority of people probably saying, “Oh, this is a drug which failed a clinical trial” and one of the most dangerous things in the world is to try to parse through a failed clinical trial, finding the patients where it did work, and then do a trial on those patients. Those fail very frequently.

However, Dr. Tardif’s data was unique: first, there were two Phase 3 trials, second there were prospective studies undertaken and it’s very rare to have such excellent data from a

pharmacogenomics analysis. So I said, “Fine, I’ll do it. What do you need?” And he says, “$50 million.” And I said, “Okay, you got it.”

That was the beginning.”

McNeil wasn’t the only DalCor representative blown away by Dr. Tardif’s research.

Louise Proulx, DalCor’s chief development officer was very impressed by the pharmacogenomics data as well.

“We met with experts, clinicians and geneticists and they were all curious, wondering how true those data were. And we all agreed we had to go for it; we had to do a Phase 3 trial in this target population.”

She adds there were other factors that made the decision to license the drug and sponsor the trial an easy one.

“For starters, the drug was already de-

“The purpose of the companion diagnostic or CDx test as we call it, is to select or find the patients within the larger group that have the specific genotype linked to a positive response to dalcetrapib.”

— Michele D’Elia, manager, Medical Affairs at Roche Diagnostics

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Precision Medicine “For us, with this particular test, it would be breaking through with the first companion diagnostic test in the cardiovascular disease space, so we’re very proud to be associated with this project.”

— Francois Drolet, director of Business Development Roche Diagnostics

risked in a sense, having showed an excellent safety profile in studies with more than 17,000 patients. One other thing that reassured us in terms of the drug itself, is the formulation of the tablets and all that has already been agreed upon with the FDA. It’s very reassuring to know there should be no issues with that,” says Proulx.

Best of all, DalCor isn’t in this alone. Like many precision medicines, there is a need for a companion diagnostic to help find the target population the drug is meant to treat.

In this case, DalCor has turned to Roche Diagnostics to develop a diagnostic test that will be a key part of a pharmacogenomicsguided dalcetrapib treatment and be used in the planned Phase 3 clinical trial.

“The purpose of the companion diagnostic or CDx test as we call it, is to select or find the patients within the larger group that have the specific genotype linked to a positive response to Dalcetrapib,” says Michele D’Elia, manager, Medical Affairs at Roche Diagnostics. He adds that the test will work like most genetic based PCR assays.

“In every healthy cell we have all the same genomes; you take blood, then you isolate the white blood cells and you’re able then to look at specific genes that are located on a specific chromosome. In this case, it’s a specific gene called ADCY9, which shows at a certain sequence that there is a signature that would identify patients that are likely to respond more to Dalcetrapib,” he explains.

He adds that the diagnostic test will not change anything related to the objective of the drug treatment except to ensure the patients who receive it will be the ones that actually benefit from the compound.

There is a process in place to generate a commercial version of this test approved by the FDA. “Eventually, if the outcomes of the Phase 3 trial reflect what the retrospective analysis of the Dr. Tardif’s study did, then the companion diagnostic will be sold commercially alongside the drug,” he says.

Roche’s familiarity with the drug should

aid its efforts in this regard. Moreover, this collaboration is important for Roche Diagnostics in breaking new ground in the precision medicine space. According to Francois Drolet, director of Business Development Roche Diagnostics, the company has made a personalized healthcare strategy an integral part of its business plan.

“It’s funny how it works, something takes you somewhere you hadn’t anticipated,” he remarks. “After the company pulled the plug on the Dalcetrapib program three years ago, I’m not sure we expected to be involved again with the success of this drug.

But this concept of precision medicine and having companion diagnostics is moving to dominate medical practice. For example, if you look at the oncology space today, one third of the drugs that are prescribed have a biomarker associated with them. With Her2 and Herceptin® (trastuzumab) as probably the best example. Herceptin is not overly compelling as a treatment for breast cancer in a general population, but if you administer the drug to those with the correct genetic marker it shows significant medical benefit.

That is what we’re trying to achieve, and I think we’re getting pretty good success lately if you look at our pipeline. We are bringing the concept of companion diagnostics into other disciplines, such as neuroscience and respiratory disease. For us, with this particular test, it would be breaking through with the first companion diagnostic test in the cardiovascular disease space, so we’re very proud to be associated with this project,” says Drolet.

The economics are confidential, but there will also be a financial reward for Roche if it’s successful.

Roche isn’t the only partner in this initiative. The Montréal Heart Institute will be retained by DalCor as the lead Academic Research Organization provider for the Phase 3 trial, to be conducted in partnership with MedPace, an international Contract Research Organization. This also insures that Dr. Tardif and his team remain heavily involved in the

10 BIOTECHNOLOGY FOCUS October/November 2015

project and can further investigate the drug’s biological mode of action.

As for the cost of taking Dalcetrapib back into Phase 3 trials, DalCor estimates it will take $250 million to complete the project.

“The way we established that number, is there’s a screening portion to the trial where we have to distinguish who our trial population will be, then establishing the trial in 30 countries and so on. That’s a major part of that $250 million,” says Proulx.

Aligning the development of a drug and diagnostic design program also requires a lot of careful planning she adds.

“Once we develop the companion diagnostic, then we need drug supply for the trial, so manufacturing and distribution around the world over a period of five years.

We have a very detailed budget,” she says.

The payoff if successful could be in the billions. Specifically, before Roche’s setback with the molecule, some industry analysts had estimated that dalcetrapib could have achieved up to $10 billion in annual sales.

“That number changes of course with this new personalized medicine or precision medicine approach, but it’s still a significant figure, in the billions,” Proulx states.

Going forward, McNeil says DalCor is the fifth company that Sanderling has launched in Quebec since 2013, and it won’t be the last. As part of its program dedicated to help bolster the biomedical industry in Quebec, Sanderling has plans to form as many as eight companies in Quebec, representing a potential investment of more than $500 million.

“We do like investing in new technologies and innovative big products, that’s where the excitement is,” says McNeil. “With DalCor we hope to build a very large, substantial organization which can fund other projects which we will have over time and reduce our need for venture capital.”

For now though, the focus will be on DalCor, and proving that Dalcetrapib actually works. He is excited by the promise this trial holds for the future in personalized treatment for cardiovascular patients based on genomics.

“There’s a lot of evidence that says it does, but on the other hand we still have to prove it and if we do, there will be a substantial benefit to a meaningful part of our world population,” says McNeil.

To see this story online visit www.biotechnologyfocus.ca/ treating-acute-coronarysyndrome-this-time-its-personal/

By Joe Sornberger

Stem Cells Medicine By Design - CFREF Funding Announcement

A crucial moment in time for

stem cell R&D

In an interview with the Edmonton Sun in April, Dr. James Shapiro sketched out a not-far-off future in which diabetics won’t need daily insulin injections.

“We have successfully and reliably reversed diabetes in our preclinical models,” the University of Alberta researcher/clinician told his hometown paper. “This approach is new and especially exciting as it opens up a world of opportunities.”

Dr. Shapiro, who was part of the team that developed the Edmonton Protocol to treat type 1 diabetes with transplanted pancreatic islets, received Health Canada’s approval earlier this year to conduct a Phase 1/2 clinical trial of a stem cell-derived islet replacement treatment for diabetes. The therapy, which he helped develop with San Diego-based ViaCyte, Inc., involves inserting a device about half the size of a credit card and loaded with pancreatic progenitor cells under the patient’s skin, where new blood vessels grow around it and the body’s immune system doesn’t try to destroy it, enabling regulation of blood glucose levels. The Edmonton trial, supported by Alberta Innovates – Health So-

lutions and JDRF, follows one ViaCyte began last year in San Diego, CA. If the device works, the impact could be transformative. According to Statistics Canada, more than 2 million Canadians have diabetes. The Canadian Diabetes Association puts the number at over 10 million when people with type 2 diabetes and those Canadians in pre-diabetes are included.

News this summer of the commencement of the Canadian trial couldn’t be timelier.

For the past 17 years -- since the discovery of embryonic stem cells -- the field of regenerative medicine has been long on promise, short on product. But this is changing. In many ways, Dr. Shapiro is at the same crucial moment in time as many of Canada’s elite stem cell/regenerative medicine researchers: on the verge of delivering new treatments for a number of diseases.

To note a few: • Researchers at Toronto’s University Health

Network have begun a pilot study to treat patients with knee osteoarthritis using mesenchymal stem cells.

• Clinical trials are in the works using a molecule called UM171 developed by Dr. Guy Sauvageau of the Université de Montréal to expand umbilical cord blood stem cells while maintaining their properties. (Currently, donated umbilical cords have too few cells to treat adult cancer patients.)

• Dr. Duncan Stewart of the Ottawa Hospital Research Institute leads a two-year, 100-patient study in Ottawa, Montréal and Toronto to test using genetically enhanced stem cells to rebuild heart tissue damaged by heart attacks.

• Dr. Mark Freedman at the Ottawa Hospital Research Institute co-leads a 40-patient trial with Dr. James J. Marriott of the University of Manitoba, called MESCAMS (for MEsenchymal Stem cell therapy for CAnadian MS patients).

October/November 2015 BIOTECHNOLOGY FOCUS 11

Stem Cells

• Dr. Michael Fehlings of Toronto’s University Health Network leads a first-of-its kind North American study to test using neural stem cells to treat spinal cord injury.

What’s remarkable is that Canadian labs are producing this kind of world-leading work on a shoestring, with funding resources that are diminishing. The sunsetting Stem Cell Network, which co-ordinated the efforts of stem cell researchers from St. John’s to Victoria, was able to leverage the almost $83 million in taxpayer funding it received over its 15-year run many times over. But compare that to the $3 billion that California -- with a similar population to Canada -- assigned to advance the translation of stem cell research into new therapies and you get a pretty good idea of what Canadian scientists are up against.

The federal government’s recent announcement of $114 million over seven years for the University of Toronto’s Medicine by Design project, which includes stem cell/regenerative medicine R&D, is very good news for the GTA research hub, as is the Ontario’s $25-million, five-year commitment to the Ontario Institute of Regenerative Medicine.

But compare those investments to Japan’s all-in approach: $1 billion to turn induced pluripotent stem (iPS) cells discovered by Nobel Prize-winner Shinya Yamanaka into treatments. Japan also revamped its legislation to make it a magnet for clinical trials.

“I want us, Japan, to dominate the world in the area of therapies using iPS cells,” Dr. Masayo Takahashi, who leads a macular degeneration study now underway at the government-funded Riken research centre, told Bloomberg News. “It feels like the government is opening one door after another to help.”

Meanwhile Canada, the country where stem cells were discovered by Drs. James Till and Ernest McCulloch in the early 1960s,

able diseases, the Strategy & Action Plan will transform the stem cell/regenerative medicine sector into a thriving industry that generates thousands of high-quality jobs.”

During the election campaign, the CSCF hosted a website at www.stemcellstrategy. ca for Canadians to tell their federal riding candidates and party leaders that they support the Canadian Strategy & Action Plan and urging the Government to implement it.

The notion is gaining traction with those who see a need for Canada to ease its reliance James Price, on a resource-based economy. In September, president and CEO the Globe & Mail challenged economists and of the Canadian Stem Cell Foundation public policy gurus to produce 15 smart ideas “that wring as much as possible out of the old economy and help a new economy flourish.” has no comprehensive strategy in place for Stem cell research was Number 10, with the moving stem cells out of the lab and into article suggesting Canada could become a the clinic. magnet for such research.

“Generally, the pace (of funding) is slower As for what it will cost, the Strategy calls and I think we are losing ground compared for a $1.5 billion private/public investment to other jurisdictions,” Dr. Stewart told the over 10 years. While that may seem like a Ottawa Citizen in July. The article pointed big gulp, the federal government’s share is out that just as the promise of potential new pegged at an annual average investment of stem cell therapies is blossoming, research $50 million. Two-thirds of the funding will funding is more uncertain than ever. “Cancome from non-federal sources, with one ada has been a leader in this area,” said Dr. firm alone, STEMCELL Technologies of VanStewart. “It would be a shame if we were to couver, pledging $350-million in investment slide back.” over the timeframe.

This comes despite the Federal GovernIt plays to Canada’s strengths. Our country ment’s Advisory Panel on Healthcare Innois an enticing location for clinical research, vation citing stem cells as an important part with highly qualified clinicians supported of the solution to transforming health care. by experienced research staff, cutting-edge The panel, led by the University of Toronto’s facilities, a varied patient population and atformer president Dr. David Naylor, found that tractive R&D tax credits. And it would help “the inter-related areas of stem cell science, ease the strain on health care. In Canada, tissue engineering and regenerative medicine treating incurable diseases consume 67 per have opened up new therapeutic vistas.” cent of all direct health care costs, according Many of the Advisory Panel’s suggestions to the Canadian Institute for Health Informaresonate with sentiments contained in the tion -- a burden that is building. Stem cells Canadian Stem Cell Strategy & Action Plan, offer cures, not just continuous and evera private-sector-led plan to deliver up to 10 more-costly symptomatic relief. new curative therapies within 10 years by “Ultimately, it just makes sense,” says Mr. aligning key players in the field – includPrice. “We founded this field in Canada. We ing researchers, clinicians, health charities, excel at it. All we need to do is pull it all toindustry leaders and philanthropists. The gether and follow through on the enormous Action Plan was developed over 18 months potential.” of cross-Canada consultations and comes endorsed by an international panel of exJoe Sornberger is the author of the University perts headed by Harvard Medical School’s of Toronto Press book Dreams & Due Diligence: Dr. George Q. Daley and supported by a Till and McCulloch’s Stem Cell Discovery and detailed report by KPMG. Legacy. He works with the Canadian Stem Cell “We need a national strategy to maintain Foundation as Director of Communications our position as a global leader in the field that Programs. we pioneered and have pushed forward,” says James Price, president and CEO of the Canadian Stem Cell Foundation (CSCF), To see this story online visit which is championing the Action Plan. “The www.biotechnologyfocus.ca/aupside is enormous. Beyond helping provide crucial-moment-in-time-for-stemrelief to thousands of Canadians and their cell-rd/ families who are dealing with chronic, incur-

12 BIOTECHNOLOGY FOCUS October/November 2015

Special Report:

By Jeff Mackey

Surveying

the Biotechnological HR Landscape First-of-its-kind employee survey of Canada’s biotechnology industry brings future of industry into focus

H

igh speed photography is a challenge even for the most talented of photographers. This is because when things move fast they can be difficult to capture—even for a snapshot.

Human Resource (HR) professionals in rapidly expanding industries, like the biotechnology and life science industries, face a similar problem. How can you capture and understand that which is constantly moving and changing?

For photographers the answer has to do with shutter speed and aperture. For HR departments, on the other hand, the answer has everything to do with people. What do the people in these industries think about their work? Where do they think trends are heading? How can the industry be improved?

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Employee Opinion of the Biotech Workplace

In hindsight these are easy questions to answer. However, if you’re building a business plan for a biotechnology start-up, or even if you’re changing the direction of a major pharmaceutical company; you need to know where the industry is going; not where it was five years ago.

That is why employee surveys are relied on for valuable insights. By talking to the people in the trenches you can gain valuable perspective about where the industry is currently and, more importantly, where it’s going.

“What you have right now is employers looking for talent and recent grads looking to break into the industry but they don’t know where to go. It’s a serious problem,” said Janet LeClair, Senior HR Consultant with Stratford Managers Corporation.

That’s why the Stratford Managers Corporation partnered with Biotechnology Focus to conduct the Employee Opinion of the Biotech Workplace survey. The survey, which was conducted online, was filled out by 161 respondents from eight provinces and territories. This survey will be used as an early benchmark for follow up surveys in the future on the same topic.

“We were trying to reach any employee who was working in Canada in biotechnology or life science sector organizations,” said Dean Fulford, Vice President of HR Consulting with Stratford Managers. “In order to spread the word, Biotechnology Focus engaged with their network of associations across Canada who agreed to distribute the survey to their membership. We started a bit of a social media campaign to share the process about the survey, both Stratford and Biotechnology Focus.”

Given a choice, respondents were allowed to fill out the survey anonymously, to allow for more honest and frank answers.

“We saw this as an opportunity for employees in the sector to BIOTECHNOLOGY FOCUS October/November 2015

Employee Opinion of the Biotech Workplace

provide some feedback on their experiences working in biotechnology and life sciences,” said Fulford. “We had hoped as well that this would spur some positive activity in those organizations to improve their human resource processes.”

“Ultimately the survey process would provide us a base line for the workplace experience aspect, and ultimately contribute toward developing recognition of best employers’ practices for the sector,” he continued.

The survey found that generally people were pretty happy within the biotechnology field. In aggregate, employees rated the key indices, of Job Clarity, Organization Climate, Visionary

Employee Opinion of the Biotech Workplace Index Performance

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Employee Opinion of the Biotech Workplace Response by Index

Leadership and Recognition and Reward as providing a “Strong Performance” in the industry.

“We asked 25 questions on the survey about different aspects of the work environment in biotech and life science workplaces.

The goal was to build awareness of the employee’s perception of the work environment and their ability to impact the things that affect their productivity,” said Fulford.

Job Clarity, an index which scored 77 out of 100, is about whether employees understand their job requirements and how they contribute to organizational success. Organizational Climate, an index which scored a 72, has to do with the design aspects of the organization and job that empower employees to be most effective. The Recognition and Reward index, which scored the lowest with a 67 out of 100, looks at how an organization recognizes employee contributions with available organizational resources. Finally Visionary Leadership, an index which scored a 71, has to do with focusing effort and developing commitment toward achieving the organization’s mandate.

“Generally employees were saying that there are some strong

Employee Opinion of the Biotech Workplace Response by Index

practices in their organization, but there are also some specific areas where the organizations can improve,” said Fulford.

For example, the Job Clarity index, the highest scoring index for the industry, found that 84 per cent of employees ‘Strongly Agree’ and nine per cent of employees ‘Agree’ with the statement that “I can generally decide for myself the best way to get my work done.”

In many other industries the responses are far more distributed.

On the other hand, employees possibly found that the often security-centric nature of the industry a barrier to sharing information, with only 42 per cent of respondents ‘Strongly Agreeing’ that “Information and knowledge are shared openly within this organization.” Unfortunately, this finding might be par for the course when it comes to the biotechnology industry.

“One area that the survey respondents highlighted was having their organization invest in their continuous development and learning,” said Fulford. “This would be where organizations make a commitment to allowing employees to develop abilities within their roles as well as providing support for developing skills that they may need in the future.”

“The number one thing that the organizations are doing well at is employees are saying they are empowered to do their jobs in the best way they know how. Job empowerment is another top driver of employee engagement and demonstrates a high level of trust exists in the organization. Those are both really important elements of a high-performing organization.”

— Dean Fulford, Vice President of HR Consulting with Stratford Managers Corporation October/November 2015 BIOTECHNOLOGY FOCUS

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Employee Opinion of the Biotech Workplace Response by Index

Employee Opinion of the Biotech Workplace Response by Index

“More than one quarter of survey respondents list learning and development as one of their top factors of engagement, yet almost one in four don’t agree that their organization is investing in this area. This is a powerful set of responses when looked at together,” he continued.

“When I say the word engagement; that is when [employees] give their commitment to the organization’s success and not just success in their own jobs,” said Fulford. “The importance of great leadership is often the number one employment engagement driver identified by employees. Allowing employees to understand how their work contributes towards senior management’s strategy can be an area for improvement.”

But biotech organizations are doing many things right, as well.

“The number one thing that the organizations are doing well at is employees are saying they are empowered to do their jobs in the best way they know how,” said Fulford. “Job empowerment is another top driver of employee engagement and demonstrates a

high level of trust exists in the organization. Those are both really important elements of a high-performing organization.”

Employee engagement was a special focus of the survey, and Fulford is considered the employee engagement specialist within Stratford Managers.

“If people are highly engaged they tend to be most productive, they get more done. They tend to be more committed (and less likely to leave the organization) and they are more active in the organization, meaning they initiate activity to improve workflow, customer satisfaction and other factors,” said Fulford. “If you have weak engagement you tend to see higher workplace attrition and less employee productivity.”

In all, 19 different organizational activities were reported as being important to employees in the survey as engagement drivers.

The top five most important engagement drivers were: flexible work schedules, strong leadership from senior management, professional development opportunities, rewards for performance,

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and challenging employees with greater responsibility or increasing the variety of their tasks.

Employee Opinion of the Biotech Workplace

Senior-level respondents and demographics

The demographics of the respondents for the Stratford Managers Corporation proved interesting.

“A large number of people who responded have been with the organization for more than ten years,” said Dean Fulford, Vice President of HR Consulting with Stratford Managers. “That shows they have been in business for a long period and they want to see their organizations improve so they can take it to the next level.”

Fulford pointed out that, generally speaking, employees with up to five-years of experience increase engagement with their organization, many of those with more than five-years in one organization plateau their engagement, and at 10-years people are either highly committed to the organization and increase engagement—or they check out and engagement drops.

“Because there is such a high level of senior-level people that responded shows that there are a lot of people in this industry that still want to see their industry grow.”

Where the industry goes next

Janet LeClair, is a Senior HR Consultant with Stratford Managers Corporation. She believes that a survey like this can have longterm effect on the biotechnology and life science industries.

“It’s exciting, this is innovation, it’s Canada’s future,” said LeClair.

“Why we think employee engagement is important in the bio and life science sector is because the sector is still developing,” said LeClair. “So we want to get information from the employees about the work, we already know from the employer how hard it is to get set up and commercialize, but we haven’t heard much from employees on their challenges.”

By entrenching a regular employee survey into the industry many, from individual HR professionals all the way to executive boards, will be able to make decisions empowered with vital information about their employees and their industry.

“What I get excited about is that this survey will be the benchmark going forward and there is a lot that can be done going from this point,” said LeClair. “From a career development perspective it’s a great start, we want to get to the point where we have a clear outlook of the options in this sector, because right now some of these jobs are so new that the kids out there don’t even know about them.”

Companies in Canada will now be able to create the preconditions to attract and keep great talent in the biotechnology industry because, unlike in some other markets, the industry will be aware of how it can improve.

“There are really small companies who don’t even have HR,” said LeClair. “Then there are the huge pharma companies and research institutes, so overall it’s a unique sector.” “[This survey] kicks starts these small companies into thinking about HR.” she continued. “Too many people believe that as the

“Why we think employee engagement is important in the bio and life science sector is because the sector is still developing. So we want to get information from the employees about the work, we already know from the employer how hard it is to get set up and commercialize, but we haven’t heard much from employees on their challenges.”

— Janet LeClair, Senior HR Consultant with Stratford Managers Corporation

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Employee Opinion of the Biotech Workplace

Employee Opinion of the Biotech Workplace

industry goes; the talent will just be there, but that often isn’t the case.”

(63 per cent) or ‘Agreed’ (23 per cent) with the statement.

However, it would appear that biotech workers do not feel they receive the feedback they need to do their jobs better. Only 42% of respondents ‘Strongly Agree’ with the statement “I receive constructive feedback from my manager that helps me improve my contribution at work.” While 18 per cent of respondents ‘Disagreed’ or ‘Strongly Disagreed’ with the statement.

By the numbers: Organizational Climate

Nurturing a strong Organizational Climate, which are the design aspects of the organization that empower employees to be most effective, can be a tricky thing. It can be difficult to accommodate different personalities, perspectives and professional objectives that have all been bottled into a single organization—sometimes a single cubicle. The key to a good Organizational Climate is the development of good relationships through frequent and honest feedback, understanding and strong communcation skills.

Despite the biotech stereotype of being impersonal or overlyacademic, it would appear that the industry actually has a fairly strong Organizational Climate.

Only nine per cent of respondents to the Employee Opinion Survey disagreed or strongly disagreed with the statement “This organization cares about the health, safety and well-being of its employees while at work.” While it would of course be better to see this number lower than nine per cent, the industry can take heart at the fact with 86 per cent of respondents ‘Strongly Agreed’

By the numbers: Recognition and Reward

The results of the survey found that while the biotechnology industry fared well in satisfying the productivity factors important to its employees, one area of improvement might be in Recognition and Rewards. Though this index still received a ‘Strong Performance’ rating by survey respondents, it was the worst performing of all indices in the study.

Only 44 per cent of respondents ‘Strongly Agree’ that their organization invests in their continuous learning and development while 25 per cent either ‘Disagree’ or ‘Strongly Disagree.’ This is not ideal for an industry that relies on the creation of new ideas and retaining talented workers.

Continuous learning is a driver of employee engagement, and

“More than one quarter of survey respondents list learning and development as one of their top factors of engagement, yet almost one in four don’t agree that their organization is investing in this area. This is a powerful set of responses when looked at together.”

— Dean Fulford, Vice President of HR Consulting with Stratford Managers Corporation

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high engagement drives commitment. Talent can be retained and attracted with a focus on professional development as recognition for contribution, as opposed to a line item in your budget.

A closer look: Job Clarity

According to the survey, the Job Clarity index in the biotechnology industry “increases with organization tenure, indicating an improved awareness of job impact to organizational success and improved capability to use judgment to complete work at the expected level of contribution.”

Job Clarity “is strongest in organizations with less than 100 employees alluding to increased access to relevant information for their work and more awareness of the direct impact of individual contribution to organization success.”

A closer look: Visionary Leadership

According to the survey, the Visionary Leadership index in the

Employee Opinion of the Biotech Workplace

biotechnology industry is “weaker in organizations with more than 100 employees, likely indicating an increased expectation of senior leadership to drive organizational success. This is aligned with employees in the same size organizations having relatively less awareness of their own contribution to organization success.”

A closer look: Recognition and Reward

According to the survey, the Recognition and Rewards index in the biotechnology industry is “consistent across all age and tenure categories, however senior level employees with greater than 15 years of experience report strongest results.” Performance of this index is significantly weaker for employees in manufacturing roles, possibly indicating less satisfaction with personal development opportunities and less recognition for contribution.”

A closer look: Organizational Climate

According to the survey, the Organizational Climate index in the biotechnology industry is “weaker for employees in the 6 to 10 year tenure range, indicating a point where employees are increasing their expectation of their organizations (more involvement, more feedback, better tools). This is after years 1 to 5 where employees are still learning, and followed by tenure of 11 or more years where employees have ‘settled in’ and their organization expectations have often plateaued.”

They say a picture is worth a thousand words. The health and future of your organization, on the other hand, is priceless. It would be wise to take heed of this survey, engage your employees and start preparing for the future of the industry—put your success into focus before it slips out of frame.

To see this story online visit www.biotechnologyfocus.ca/surveying-thebiotechnological-hr-landscape/ October/November 2015 BIOTECHNOLOGY FOCUS

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By Lisa Willemse

Stem Cells

Banking on cord blood Recent technological innovations in umbilical cord blood expansion and other research aimed at improved use of these life-giving cells suggest that a future of better therapies for many patients is not that far away.

I

n late June, two coincidental announcements, one by Canadian Blood Services and another by an emergent business with strong ties to research, marked the future of blood stem cell treatments in Canada. The two announcements were not coordinated or linked in any way, but together, they promise significant impact: Canadian Blood Services’ new national cord blood bank enables collection of umbilical cord blood at four Canadian hospitals that are now being used for treatment or research purposes, while the cord blood expansion technologies supported by ExCellThera will help make more of these units usable within the clinical setting, thereby allowing more patients to receive the life-giving stem cells each unit of cord blood contains. These pivotal advances highlight an extraordinary breadth of research and technology development that is taking place in labs across Canada, and particularly in Ontario.

Within a single unit of umbilical cord blood, there exists a mix of cells, from mature or nearly mature blood cells, such as red and white blood cells that perform necessary functions of oxygen transport and conferring immunity against disease, to primitive hematopoietic stem cells or HSCs – cells that have the unique ability to form all of the different kinds of cells within the blood system, but can also make exact copies of themselves in order to maintain a reservoir of replacement cells. For patients with particular blood diseases, such as leukemia, this reservoir can be quickly depleted, either by the illness itself, or by aggressive treatment protocols. Under such circumstances, a hematopoietic stem cell transplant, rich in new stem cells, can help trigger the body to heal. Transplants, however, require a source of new stem cells.

Currently, there are only three viable sources of these cells: the bone marrow, peripheral blood stem cells (from the blood-

stream) and umbilical cords. Of these, the most common source is peripheral blood stem cells, which provides one of the richest source of blood stem cells and therefore is the preferred treatment option. Less rich in HSCs, cord blood units are nevertheless an important source needed to meet clinical demand.

“Cord blood transplants are one of the most expensive forms of allogeneic therapy out there, because there are typically fewer cells available to engraft,” says Dr. Nicolas Pineault, a development scientist at Canadian Blood Services and a member of the Ontario Institute for Regenerative Medicine.

“This is because of the large costs associated with the length of time a patient must stay in the hospital, and the cost of cord blood units, which can be as high as $50,000 each.”

“The success of a cord blood transplantation is proportional to the number of stem cells that a patient receives,” Dr. Steven

October/November 2015 BIOTECHNOLOGY FOCUS 13

11:28 AM

Stem Cells

One year of cord blood collection

Canadian Blood Services began collecting cord blood units in Ottawa in September, 2013. Units that have insufficient quantities of stem cells for therapeutic use can be useful in research. In the first year: • 60% of mothers donating their baby’s cord blood consented to donate for research.

• 8 applications from Canadian researchers (Ontario, Alberta, BC) were approved.

• 168 cord blood units were distributed to researchers nationally.

Klein, CEO of ExCellThera explains further.

“If you’re an adult, you’re only looking at the ability to access the five or six per cent of the cords that have the required level of cells available in an unexpanded form.”

As a result, many adult patients currently require more than one unit of cord blood, which drives up costs. A further complication is that the units must be matched as closely to the patient as possible in order to reduce the chance of immune rejection.

This is the challenge being taken up by Canadian researchers in several ways – by better understanding the properties of the cells before and after engraftment, and by finding methods to increase the numbers and improve the quality of the cells.

ExCellThera’s approach takes advantage of two of these Canadian advances. The first facet of the technology is a small molecule, developed in the lab of Dr. Guy Sauvageau at the Université de Montréal capable of expanding the number of the most primitive blood stem cells in culture by at least ten times that of standard cultures. This, on its own, is impressive, but that number jumps significantly when combined with the second part of the protocol, a fed-batch culture

bioreactor developed by Drs. Peter Zandstra and Elizabeth Csaszar at Toronto’s Centre for Commercialization of Regenerative Medicine (CCRM).

Csaszar, a development scientist at CCRM and former PhD student in the Zandstra lab, first began working on the fed batch system four years ago. “People have been refining the ability to expand blood stem cells for many years, but the challenge has always been that when you put these cells in culture, the stem cells tend towards differentiation and become mature blood cells, and over time you lose the real stem cell component,” she said.

Zandstra and Csaszar theorized that this occurs because the mature cells produce a particular signal that prevents the production of new stem cells; as the number of mature blood cells grows in the culture, so does the concentration of the inhibitory signal. Their goal was to find a way to prevent the overaccumulation of the inhibitory signal so that more stem cells would be produced.

Using a mathematical modelling approach that mimicked the blood system, the team tested several different scenarios for predicting optimal cell growth and arrived at the fed-batch culture system. “It was surprisingly

14 BIOTECHNOLOGY FOCUS October/November 2015

simplistic,” Csaszar explains, “It operates on the principle of dilution. The system has a continuous inflow of culture media [food for the cells] but doesn’t have an outflow, so this means over time the volume will increase, but the concentration of the inhibitory signal remains low enough to allow the production of stem cells.”

The true power of the fed batch system comes when combined with the expansion molecule, when it is then capable of producing as much a 150-fold expansion in just 12 days.

“It’s currently the best system out there,” said Klein. “We’ve looked at the published literature and the public disclosures and can say with confidence that there is nothing better. With it, we’re hoping to achieve 80 per cent usability of the cord blood units, rather than the current five or six per cent.”

It’s a confidence that bears up in the preclinical data, but is yet lacking verification from human trials. If all goes according to plan, the human data will not be long in coming: the company is in the process of starting a Phase 1/2 clinical trial in fall of this year. The first patients will be recruited in Québec. Anticipating proof of concept through this first trial, ExCellThera has an eye to financing a Phase 3 registration trial, for which multiple international centres have already expressed an interest.

It’s quite possible, however, that part of the trial could stay in Canada. In Ontario, a coordinated effort is underway to develop a clinical trials network, led by the Ontario Institute for Regenerative Medicine (OIRM).

Translational clinicians from within the OIRM are already leading seven of the eight cell therapy trials that are currently underway in Ontario. OIRM is working on several fronts to streamline the steps that take a cell therapy into Phase 1 trials, including regulatory, ethical, multi-centre frameworks and coordinated funding models.

Nicolas Pineault, for one, is optimistic about the momentum in the field. “I think we’re looking at about five to 10 years, and we’ll see improvements from this research and these technologies having a direct impact on the health of patients, with more being treated and spending less time in recovery.”

To see this story online visit http://biotechnologyfocus.ca/ banking-on-cord-blood/

By Diane Gajewczyk

Across Canada

The Evolving Clinical Trial Landscape in Canada An Update

T

here has been a flurry of activity in recent years to address some of the challenges in Canada’s ability to compete globally for clinical trial participation. It is encouraging to see the broad range of stakeholders from provincial and federal governments, to academic research institutions to industry associations join together at conferences, workshops and discussion forums to address Canada’s languishing rate of clinical trial participation. These efforts are vital if we are to improve the business climate for Canadian clinical trials.

Multiple Players-Who Does What?

From the perspective of an industry sponsor looking to initiate a multi-site trial in Canada, however, the plurality of efforts can be confusing as a singular united national coordinating voice has not been established yet. Nonetheless, solid advances are being made, albeit in a patchwork manner. A quick guide to some of the multiple resources follows.

First, at the national level, the CCTCC (Canadian Clinical Trials Coordinating Centre) was established in April 2014 and is responsible for implementing the recommendations from the 2011 Clinical Trials Summit.

Susan Marlin, President and CEO of CTO

The outcome of the summit brought forth nine recommendations and three organizations (Rx&D, CIHR and HealthCareCAN) have joined together to fund the CCTCC to implement these recommendations. The most recent notable accomplishment of Rx&D and the CCTCC was the roll-out of the CCTAM (Canadian clinical trials asset map) in June 2015. The CCTAM is a real time database containing information on investigators, research institutes etc. across the country- a useful tool for an industry sponsor looking to initiate a national multi-site clinical trial. Also at the national level is the N2 network (Network of Networks). Membership in this network facilitates knowledge sharing in operations (SOP’s training etc.), and also networking among specialty knowledge networks. However, industry sponsor membership in this group is currently quite limited, and perhaps there is an opportunity for further industry engagement.

At the regional/provincial level there are also promising developments. Clinical Trials Ontario (CTO) has been one of the most active organizations. CTO is a non-profit organization funded by the Government of Ontario with a mandate to provide a streamlined approach to conducting multi-centre clinical trials in

“I would like to reach a state where a protocol is submitted and receives province wide ethics approval within a month to two of submission and trial sites are approved merely days after.”

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Across Canada Ontario. There are three strategic initiatives that CTO has outlined in its strategic plan to achieve this goal: 1. To streamline the Research ethics board (REB) review process 2. To engage/educate patients and other stakeholders in order to enhance clinical trial recruitment rates 3. To increase the number of global clinical trials in Ontario

CTO’s Streamlined REB Process in Ontario In March 2015, CTO launched the CTO streamlined research ethics review system.

The REB review model in Ontario is aimed at one REB review per protocol in the province of Ontario. It is not a centralized single review, instead, it relies on a network of member organizations that formally join CTO so that an REB review at one institution is recognized at all member institutions, acting as a “Board of Record.” This model provides for efficiency, flexibility and capacity balancing in the REB system so that investigators and study sponsors do not need to rely on a local REB approval for every research site activated. CTO works within the largest clinical trial infrastructure in Canada, covering 75 REB’s reviewing clinical trials, 250 hospitals and multiple academic research institutions.

Currently, within the first six months from launch, CTO has qualified nine REB’s with six more REB’s in review and has signed up 40 research sites into the system. Their extensive pre-consultation is likely reaping the benefits in the solid level of uptake and engagement in the CTO process.

The engagement on the REB participant side is matched with active participation by study investigators and sponsors. As of September 2015, there are 16 studies in the system under review and approximately 20 studies in the pipeline. In June 2015, CTO announced that a GSK multi-centre trial had received province-wide ethics approval in just two months from the time of submission—a definite and very welcome first for Ontario’s clinical research environment and proof of concept that the CTO model works.

As the number and diversity of protocols submitted through the system increases over time, it will be interesting to monitor the performance metrics to ensure that the core timeline savings can be retained. As Susan Marlin, President and CEO of CTO, states, she “would like to reach a state where a protocol is submitted and receives province wide ethics approval within a month to two of submission and trial sites are approved merely days after.”

With this single improvement in cutting red tape and needless duplication of effort, Ontario would be able to begin to compete competitively on the global stage for protocol start up.

CTO’s REB Model Benefits Study Sponsors as well as Emerging Research Centres One of the clear benefits of the CTO model is simplifying duplicative processes and paperwork which results in savings of time and money. However, there are additional benefits to the Ontario clinical research landscape. For example, the participation of some community hospitals such as Southlake Regional Health Centre in Newmarket, Ontario is a case in point.

Southlake is a community hospital but it also runs regional centres for both cancer and cardiac tertiary care. As a result, there are very talented specialized staff and a pool of well-characterized patients available at this centre. However, as a community hospital, there are limited funds available to build the clinical research infrastructure required to participate in major global clinical trials.

As Gary Ryan, Chief Innovation Officer at Southlake stated “Southlake sees three key benefits in participating in the CTO process: 1. Participate in a functional system that improves the operating standards of all participating REB’s; 2. Potentially have increased numbers of studies come to the Southlake centre; 3. Leverage the CTO’s electronic resources so that Southlake does not have to invest in an independent electronic system.”

Southlake has completed its prequalification and has a scheduled on site review this fall.

It is expecting CTO certification by the end of this year. Once certified, Southlake will be one of three community based hospitals, in addition to Markham-Stouffville and Peterborough, participating in the CTO system.

Future Developments of the Streamlined REB Model – Taking it to the National Stage Although it is still early days in the implementation of CTO’s model, it appears that they are off to a good start. However, from

16 BIOTECHNOLOGY FOCUS October/November 2015

an industry perspective, there is a desire to conduct Canada-wide trials so that physicians and patients have access to the newest cutting edge treatments and an appropriate diverse Canadian patient population can be included in the trials, The ideal state would be to have a national network of REB’s where a single REB review could be recognized at research centres across Canada. For a number of reasons, this ideal state does not yet exist. There are, nonetheless, hints of interprovincial cooperation—CTO and BCCRIN—Alberta and UBC—for example.

It appears that this grassroots approach to a national collaboration may be what is required to build the bridges and further evolve the system so that it can be both robust enough to serve as a nationally recognized system, but flexible enough to accommodate the regional differences found in our provincially influenced health care infrastructure.

As Susan Marlin of CTO stated “ CTO ‘s mandate is to improve Ontario’s environment for clinical trials, but we also recognize that we need to reach out nationally to improve the Canadian environment as well in order to truly succeed in Ontario. There is a tremendous will and excitement to work towards a national solution.” However, that solution may not necessarily be a national body per se, but rather a quasi-national system built of a coalition of willing partners.

Recently, Health Canada has established an advisory committee on REB’s to review potential standards that may result in a regulatory requirement for REB accreditation.

This initiative could also help to move to a more national vs interprovincial/provincial approach. At this point in time, the provinces have not been invited to the committeehopefully that consultation will occur before final recommendations are made.

CTO is also looking at initiating an REB council in Ontario to look at differences in reviews and practices at different institutions in Ontario in the hope of identifying the differences, the underlying need (if any) for the difference and a potential solution or revised new best practice. Perhaps some of the findings and recommendations from the Ontario experience could then be taken to the national level to address interprovincial differences.

Across Canada Gary Ryan, Chief Innovation Officer at Southlake

CTO’s Patient Engagement Strategy One aspect of study protocol initiation is the institutional ethics review of the protocolbut study sites must be selected. Site selection involves a number of factors; the reputation of the investigators at the site, quality of research personnel, access to specialized equipment and, perhaps most importantly, access to the right types of patients for the study. Given the amount of money and time invested in study site start-up, it is important that the site meet a minimum patient recruitment level in order to remain economically competitive with other trial sites on a per patient costing basis. The CCTAM and CTO asset maps could assist in identifying sites with appropriate expertise, but how can the site recruitment rates be improved? Another strategic imperative for CTO is to increase patient engagement in clinical trials and address this very question.

To date, CTO is still in early stages of this initiative. They have started conversations with patient advocacy groups and health charities to begin to understand the perspectives and attitudes of patients towards participation in clinical trials. They have also partnered with BCCRIN on a public survey to examine attitudes towards and perceptions of clinical trials. CTO intends to seek more public and patient engagement and will soon appoint an individual representing the patient perspective to their board and reach out to industry stakeholders to discuss patient centricity in trial design and operations. CTO will also work on patient education initiatives to increase awareness of clinical trials and address some of the questions that may be serving as barriers to participation.

Industry Pain Points-where are we on the journey to global competitiveness?

A quick pulse check of the Canadian clinical trial environment, and specifically the work of CTO seems to indicate that we are

Southlake has completed its prequalification and has a scheduled on site review this fall. It is expecting CTO certification by the end of this year. Once certified, Southlake will be one of three community based hospitals, in addition to Markham-Stouffville and Peterborough, participating in the CTO system.

strategically on the right path, but, more importantly, the strategy is being implemented. The typical industry pain points of REB paperwork and process duplication are being addressed through initiatives of CTO like REB streamlining which promises to reduce both time to study initiation and costs. Understanding the clinical trial resources available across the country is being addressed through asset map initiatives like the CCTAM. However, we still have gaps in terms of national standards and national/ interprovincial collaboration—so although it may be possible to get ethics approval within a couple of months in Ontario, it is unlikely at this time that this single approval could be adopted across a multi-site panCanadian trial. In this regard, and with other start-up issues like clinical trial agreements, there is still work to do to increase Canadian competitiveness overall.

Patient recruitment is another important aspect of the equation. New therapies in development tend to be highly targeted at the molecular level, and the era of “personalized medicine” has truly arrived. What does this mean for clinical trials in Canada? The ability for study sponsors to access patient pools that are well characterized from a disease and biomarker perspective will be key-often these tests will be performed as part of the standard of care, if not, then having patients located in tertiary care centers that could handle more advanced diagnostic testing will be key.

Another industry trend is to focus on the development of treatments for rare diseases. However, the challenge in finding these patients in Canada is inherent to their condition. Given the small Canadian patient population relative to other countries, by sheer numbers and statistical probability, more rare disease patients may be enrolled in global trials from countries other than Canada. This is sadly a lost opportunity, as often these rare disease patients have no treatment alternatives, or, if treatments are available, due to their high cost, patients cannot afford them. Participation in clinical trials for rare disease conditions gives these

patients an opportunity to access potential treatments that they otherwise could not afford or access. The development of the PTC (Permission to contact) database in Canada may help to bridge this gap—but only if it is truly adopted across the country.

Another complication when it comes to pan-Canadian multi-site studies is that the standard of care may vary significantly from province to province. As a result, there may be a provincial bias in recruitment that is dependent on what treatments patients may have access to in their province. One of the key drivers of this difference is the inter-provincial differences in pharmacare formularies.

A discussion has been underway for a national pharmacare strategy that perhaps may level the playing field between provinces in the future, and contribute to the “nationalization” of Canada’s clinical trial landscape.

Why is it important to continue to push for a Canadian agenda?

Canada clearly has the intellectual horsepower, talent and research resources to continue its legacy of making some important contributions to science and medicine. It is encouraging to see so much activity and the engagement of multiple stakeholders to improve Canada’s competitiveness. What is not clear at the moment is the sustainability of funding for these various provincial and stakeholder led initiatives and if a truly national approach will emerge over time. In the interim, hopefully government support and funding will continue so that we can build an infrastructure that will serve not only patients by providing them the opportunity to access cutting edge medicines and treatments, but also serve our Canadian researchers who can benefit from global networks and knowledge. As they say, if you build it…..they will come!

To see this story online visit www.biotechnologyfocus.ca/ the-evolving-clinical-trial-landscape-in-canada-an-update/

October/November 2015 BIOTECHNOLOGY FOCUS 17

By: Shawn Lawrence

biosimilars

Opportunities around Subsequent Entry Biologics,

Can Canada capitalize?

T

he term Subsequent-Entry Biologic (SEB) is used by Health Canada to describe “a biologic product that is similar to and would enter the market subsequent to an approved innovator biologic product,” otherwise known as biosimilars. While some jurisdictions, such as Europe, have embraced and enjoyed the entry of SEBs into their markets, Canada has taken a more conservative approach both in terms of its regulations and policies around SEBs which have limited their impact.

It should be pointed out that Health Canada does have a regulatory framework in place for SEBs that was released in March 2010. However, according to Chirfi Guindo, president and managing director at Merck Canada Inc., even with these guidelines in place, there is still more to be sorted out.

Moreover, he wants to see industry stakeholders, such as Merck, engage with regulators and policy makers to ensure Canada capitalizes on this emerging opportunity.

“One thing we would be looking forward to at Merck is to work with regulators, the provinces, health technology assessment bodies, patient and health care practitioner associations, and indeed within industry to help frame that policy environment so that there is a viable and sustainable SEB market here in Canada,” he says.

He’s not looking to reform the current environment, but rather he wants to find ways to make the system less arduous.

“Europe is in that sense significantly ahead of Canada,” he explains, noting the number

of SEB approvals in that market. So far, less than a handful of SEBs have been approved in Canada. In comparison, there are approximately twenty plus approved biosimilars in the European Union. He counters though that the European model is not necessarily what Canada should adopt. “There are certainly important differences between Europe and Canada in the way their healthcare system is organized; nonetheless, I think there is opportunity in Canada to really learn from them to help accelerate the policy and regulatory environment here.”

“Specifically, with how Canada’s healthcare system is currently organized, you’re not just dealing with one governing body

“One thing we would be looking forward to at Merck is to work with regulators, the provinces, health technology assessment bodies, patient and health care practitioner associations, and indeed within industry to help frame that policy environment so that there is a viable and sustainable SEB market here in Canada.”

— Chirfi Guindo for approvals and drug reimbursements, but rather you’re dealing with as many as 14, with 10 provinces and 3 territories and then the process for Canada as a whole,” he says.

He’d like to see a more harmonized and speedy approach for approvals and drug reimbursement, but recognizes this might be a tall order.

18 BIOTECHNOLOGY FOCUS October/November 2015

A second issue for Canada’s lag in SEB adoption is simply the lack of knowledge about biosimilars and the savings opportunity they present, and this is a big reason he wants to see more dialogue between various stakeholders.

“There is a real need for all of us to educate the consumers, patients, health care prac-

biosimilars titioners, policy makers and other decision makers, and help them fully understand what opportunities biosimilars can provide, and better explain for example, what we mean by extrapolation of indications and interchangeability.”

While he agrees on the necessity of a different approach because of the nature of biosimilars themselves, he still feels that there are some further discussions required with regulatory agencies on the issue of extrapolation of indications.

“When it comes to SEBs, you need to do substantial pre-clinical development requiring structural and functional studies, as well as conduct clinical trials,” he explains. “This is not the case with regular generics, where only the bioequivalence studies are required.

Then, the regulators typically give the generic companies all the indications of the original drug. For SEBs, it is a longer process where you have to do at least one clinical trial in at least one major indication to prove efficacy and safety, after which regulatory agencies

switch to the generic automatically. ” For the biologics and biosimilars, it’s another story. Because of the complexity and nature of these drugs, regulators tend to take the position that before you switch from a manufacturer’s biologic to another manufacturer’s biologic; the physician has to have their say.

There are other reasons beyond policy that put Canada behind the curve for the number of SEB product approvals, in comparison to European markets, of course. As the patent on the reference products for certain SEB products expired earlier in Europe than in North America, Europe’s SEB market developed first and moreover, the European Union’s (EU) regulatory agency was the first agency to establish a process to approve SEBs. Despite the stark differences in numbers, it doesn’t mean Canada can’t catch up, Guindo says, while adding that the payoff if they do catch up, in the end, could be huge in terms of savings for the healthcare system. He also

“You have a market here where biologics represent a significant part of the health care drug cost structure, so as they come off patent, and a biosimilar in the same space gets approved, you’re offering significant cost saving opportunities to the public and private health plans in this country.” — Chirfi Guindo are able to grant extrapolation.”

According to Guindo, replicating a biologic is not as simple as making a generic drug.

Biosimilars, like their biologic counterparts, are complex to make in comparison to small molecule chemical compounds. “You have to involve an extended team of biologists working together with chemists and manufacturing experts and undergo rigorous testing to come up with the product.”

Likewise, in regards to interchangeability, Health Canada doesn’t automatically support substituting SEBs for brand-name biologics because of their complexities, a position Guindo supports.

This is different from the way Health Canada treats generic drugs, he explains: “When you have a small molecule generic, regulators typically take a position that if they’re registered then it doesn’t matter who the manufacturer is and pharmacies can switch from one drug to another without physicians being involved. So you go to your pharmacy you’re given a script with product A, and product A happens to have a generic, pharmacies can

believes there are many indicators that SEBs could be very successful in Canada beginning with how many Canadians are already big consumers of biologics themselves.

“Canada is one of those markets globally where originator biologics have had a significant success and a significant level of adoption-we estimate that between 15 to 20 per cent of the market in Canada are biologics, with sales of close to $6 billion, and this is growing in double digits,” he says.

But there is the steep cost associated with biologics. Herein lies the opportunity.

“You have a market here where biologics represent a significant part of the health care drug cost structure, so as they come off patent, and a biosimilar in the same space gets approved, you’re offering significant cost saving opportunities to the public and private health plans in this country.”

It also comes down to creating a competitive marketplace.

“Essentially, in a post-patent context, once you will have two or three players in the same category which will create com-

Samsung Bioepis plant petition, and that’s what we all want since ultimately that is going to generate savings and be beneficial for the healthcare system overall. While I don’t think the biosimilars will displace the biologics completely, you’re giving patients and healthcare practitioners choices as to what they spend their money on and you’re effectively driving down price.”

He points to Europe where SEBs have already taken significant costs out of their healthcare system by creating a favorable environment for SEBs over brands once patent protection has expired. Merck has a vested interest in seeing that this happens here, as this would free up savings that would potentially help payers to fund innovations. This is why Merck entered into a partnership with Samsung Bioepis in the biosimilars area.

“Canada is one of those countries that are covered by the agreement we have with Samsung Bioepis,” he says. “Samsung Bioepis will do the process development, characterization, manufacturing and regulatory filings and launch of the biosimilar products, with Merck being the commercial partner when the biosimilars hit the market. We’re going to be in a position where within this partnership, we’re going to be able to launch biosimilars in immunology, in oncology, as well as insulin. In total, we’re looking at several biosimilars that we hope to launch into the market in the next two to three years.”

There are many other pharma companies in the same position. As such, there is an ideal opportunity for Canada to embrace biosimilars.

To see this story online visit www.biotechnologyfocus.ca/ opportunities-aroundsubsequent-entry-biologicscan-canada-capitalize

October/November 2015 BIOTECHNOLOGY FOCUS 19

NEW PRODUCTS

Sequencing Illumina Inc. announces the launch of the TruSight® Tumor 15, a next-generation sequencing (NGS) panel designed to identify sequence variants in 15 genes commonly associated with marketed therapeutics. The panel is optimized

for low input DNA from tissue preserved in formalin and paraffin as well as for damaged and degraded formalin-fixed, paraffin embedded (FFPE) tumour samples, enabling low frequency somatic variant detection from limited nucleic acid inputs.

When paired with the Illumina MiSeq® system, the panel delivers high-quality sequencing with key content and features needed for tumour analysis in translational research. The panel is intended for research use only and not for use in diagnostic procedures.

Web: www.illumina.com/OncologyPanel Spectrophotometers The Thermo Scientific NanoDrop One and NanoDrop OneC UV-Vis microvolume spectrophotometers

are designed to help life science researchers gain a more complete understanding of sample quality and avoid costly delays due to troubleshooting and repeating experiments. These new spectrophotometers introduce the Thermo Scientific Acclaro Sample Intelligence technology, which enables researchers to identify sample contaminants and obtain corrected concentration results; receive instant feedback about sample quality with on-

demand technical support and guided troubleshooting; and confidently measure samples with embedded sensor and digital image analysis. Moreover, the instrument with its high-resolution touchscreen interface is simple to use, while a powerful auto-range path length technology facilitates accurate measurements for concentrated samples with no need for dilutions. The patented NanoDrop sample-retention system measures 1-2 µL of sample in seconds without the need for cuvettes. Workflows can be streamlined with Auto-Measure functionality. Modern connectivity allows results to be shared and archived via Wi-Fi, Ethernet or USB.

The rapifleX MALDI-TOF/TOF system is well suited for detailed protein characterization in life science research and biopharmaceutical laboratories. The system is for research use only.

Web: www.bruker.com Well plates The ExoComplete™ 96Well Plate and ExoComplete™ Tube kits from Hitachi Chemical Diagnostics Inc. are an integrated, fast and easy system for exosome collection and mRNA purification. Both products are intended for research use only and molecular biology applications. Neither are intended for the

Web: www.thermoscientific.com/ nanodrop Mass Spectrometry Bruker has released its new rapifleX MALDI-TOF/TOF mass spectrometer. With redesigned TOF/ TOF ion optics, the rapifleX system now offers significantly higher speed, mass resolution and mass accuracy. Additionally, the device integrates a 10 kHz smartbeam™ 3D scanning laser design for breakthrough mass-spec imaging capabilities with dramatically improved spatial resolution, image contrast and quality, and a new ion source for superior robustness and throughput in pathology research. Tissueand cell-type specific proteomics is a major advance for relevant biomarker discovery and identification, and requires MALDI tissue imaging studies which include the identification of detected biomarker candidates for validation and the subsequent development of targeted assays.

The rapifleX MALDI-TOF/TOF extends the MALDI Tissuetyper® imaging capabilities with rapid, spatially resolved MS/MS data. It supports the patented ImageID™ workflow for biomarker discovery in MALDI imaging cohort studies by obtaining matching identifications of tryptic peptides in bottom-up imaging studies.

20 BIOTECHNOLOGY FOCUS October/November 2015

diagnosis, prevention, or treatment of a disease, nor has either been validated for such use either alone or in combination with other products.The ExoComplete™ system isolates exosomal mRNA from biological samples such as plasma in a 96-well filter plate format, or large-volume samples such as urine in a single collection tube format. Exosomes and microvesicles (EMVs) in body fluids are captured with Hitachi Chemical’s Exosome Isolation Plate or Exosome Collection Tube with a proprietary filter material. The highly porous material allows fast filtration of biological samples without clogging, and reproducible isolation of EMVs without the use of conventional ultracentrifugation.

After lysing the captured EMVs, mRNA can be isolated through hybridization with single strand oligo (dT) immobilized in the wells of the 96-well mRNA Capture Plate.

A wide variety of downstream applications become available following isolation of mRNA, including mRNA quantification, single and double-stranded cDNA synthesis, RT-PCR, real time quantitative RT-PCR (qPCR), and next-generation RNA sequencing.

Web: www.hcdiagnostics.com

CALENDAR NOVEMBER 2015

MARCH 2016

November 22-25

March 6-11

BCN-AI Bio Conference Venue: Edmonton, AB new card:Layout 1 1/31/2013 Tel: (780) 492-1760 Email: vcheng@ualberta.ca Web: https://events.gobigevent.com/ events-web-public/event/start/774;jsessionid=d f2i5ilfgn7kHC7Jl+XHD4Xe?0

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March 30-April 2

Canadian Science Policy Conference Venue: Ottawa, ON Email: info@sciencepolicy.ca Web: cspc2015.ca

AMMI Canada - CACMID Annual Conference 2016

Venue: Vancouver, BC Tel: 613-260-3233 Fax: 613-260-3235 Email: info@ammi.ca Web: www.ammi.ca

APRIL 2016 April 17-20 World Congress on Industrial Biotechnology Venue: San Diego, CA Web: www.bio.org/events/conferences/ world-congress-industrial-biotechnology

DECEMBER 2015

SRC101

December 8 LSO Annual Policy Forum Venue: Toronto, ON Tel: (416) 426-7293 fax: (416) 426-7280 e-mail: admin@lifesciencesontario.ca Web: www.lifesciencesontario.ca/events/ eventsCalendar/FallSymposium2015.php

JANUARY 2016 January 11-14 J.P Morgan Healthcare Conference + Biotech Showcase 2016 Venue: San Francisco, CA Web: www.jpmorgan.com

January 23-27 SLAS2016 Venue: San Diego, CA Tel: (630) 256-7527 Fax: (630) 741-7527 Email: slas@slas.org Web: www.slas2015.org

FEBRUARY 2016 February 8-9 Bio CEO & Investor Conference New York, NY Web: www.bio.org/events/conferences/ bio-ceo-investor-conference

February 24 LSO Annual Awards Gala Venue: Toronto, ON Tel: (416) 426-7293 fax: (416) 426-7280 e-mail: admin@lifesciencesontario.ca Web: www.lifesciencesontario.ca/events/ eventsCalendar/LSOGala2016.php

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THE LAST WORD

By Nathalie Richard

Corporate Governance for

a Vibrant Life Science Ecosystem

T

Nathalie Richard Account Manager, Life Sciences & Health Services, Royal Bank of Canada

he formation of a strong life science ecosystem requires a critical mass of companies, advisors, networks and capital. For developing and high growth companies, capital remains one of the most challenging resources to obtain, as it requires strong and efficient capital markets, which can only exist if they are governed by equally strong corporate governance principals and regulatory frameworks.

Although boards of directors and advisory boards are well known for their focus on internal controls and fiduciary oversight, they offer other strategic advantages as well. Both can provide essential mentoring, coaching and, access to networks and resources.

How can high growth companies form strategic partnerships with their boards of directors and advisory boards, and leverage their knowledge and resources?

It starts by defining the role of corporate governance, understanding how it creates value and its relationship to capital markets.

Corporate governance is defined as “the balance between economic and social goals, and between individual and communal goals. The governance framework is there to encourage the efficient use of resources and equally to require accountability for the stewardship of these resources. The aim is to align as nearly as possible the interests of individuals, corporations and society.”1 In other words, organizations should not focus solely on maximizing shareholder value, but rather on improving economic growth, fostering an innovation ecosystem and creating value for society.

Life science companies have a long commercialization path, high levels of technical complexity and market risk, and require significant capital resources. This higher level of risk requires higher risk capital, which initially takes the form of equity. As a result, they are often financed by equity investors and must demonstrate that they can address important needs in the market in order for these investors to earn a return on their investment. Boards of directors and advisory boards strengthen the operational knowledge of companies, assist with formulating and executing on strategy and, provide the necessary relationships to assist with future rounds of financing and market launch. Companies can develop strong relationships with their boards by fostering an environment of collaboration, which is estab-

22 BIOTECHNOLOGY FOCUS October/November 2015

lished by identifying common goals in the commercialization process and determining what resources will be required to achieve them. The ability to engage in a review of different market data points, to establish and debate different alternatives, and to validate opportunities for each business milestone are among the key advantages that are provided by boards. The open dialogue between management teams and the boards reinforces the quality and depth of a company’s strategic plans, which in turn, reinforces investor confidence.

Shareholders form an equally important part of the corporate governance equation. Investors provide the capital that is required for the execution of business milestones. In return, the management team has a duty to maximize the return on these investments.

Boards of directors are responsible for monitoring the management team’s performance and ensuring that capital strategies are optimized. Strong boards of directors that establish clear and transparent communications with their investors will earn their confidence and attract additional capital.

The development of a vibrant life science ecosystem requires a credible and trustworthy capital markets system. The foundations of such a system rest on strong corporate governance principles and regulatory frameworks, which ensure that capital is allocated efficiently and reassures investors that their capital will be utilized to grow the business optimally. At the same time, strong corporate governance practices allow companies to balance the many interests of all of its stakeholders.

Companies with robust corporate governance practices have historically been more successful at raising new rounds of capital than those that were less committed to developing a strong corporate governance structure.

Corporate governance is a corner stone in fostering innovation, economic growth and the emergence of a mature life science industry.

Reference: 1. Cadbury, A. (1992). Report of the Committee on the Financial Aspects of Corporate Governance. Gee, London.

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