Biotechnology Focus Podcast

On today's show, we go one-on- one with Borden ladner Gervais' Jeff Graham to discuss his article in our latest issue of Biotechnology Focus Magazine, Genetic Discrimination: Creating a New Canadian Law, plus we look at the week that was and the top stories on the Canadian biotech scene.

Hello this is Shawn Lawrence, senior writer for Biotechnology Focus, and your host for Biotechnology Focus Podcast.

We start today's show with our weekly rundown of the top stories in Canadian biotech this week, and our first story takes us to Vancouver, BC, with AbCellera Biologics Inc., a biotechnology company specializing in the rapid discovery of monoclonal antibodies from natural immune cells, successfully completing its antibody discovery partnership with Kodiak Sciences. Through this collaboration, AbCellera applied its antibody discovery platform to perform ultra-deep screening of immunized animals and identified hundreds of antibodies against an undisclosed target. Multiplexed single cell binding assays were used to enrich for antibodies with high affinity and desired properties, resulting in the generation of multiple lead antibody families with picomolar affinity and potent blocking activity. Dr. Victor Perlroth, chairman and CEO of Kodiak Sciences said that AbCellera has outperformed Kodiaks expectations while adding that AbCellera's technology platform leverages the many benefits of in vivo selection and maturation while avoiding the inefficiency of traditional hybridoma approaches. In all, AbCellera screened over one million antibody-producing single cells with its platform and delivered relevant antibody sequences covering a diverse functional and sequence space. Under the terms of the agreement, Kodiak has now executed its option to advance selected candidates for clinical development with AbCellera eligible to receive downstream payments. Other financial terms were not disclosed.

Retaining and growing talent is a challenge for many Canadian biotech and life science companies, and there is a real need for new programs that are able help train and develop Canadian talent and help them become qualified workers with industry experience. In response to this need, Toronto-based Clinical immuno-oncology company Trillium Therapeutics says it is launching an industry postdoctoral fellowship program that will help young research scientists make the transition from their academic training to rewarding careers in the biopharma industry.

In a press release Dr. Niclas Stiernholm, president and CEO of Trillium Therapeutics stated that as part of Trillium's commitment to help grow the Canadian biotechnology industry, Trillium wants to provide an environment in which bright scientific minds will be challenged and nurtured.   "We believe this will help drive the development of tomorrow's innovative cancer therapies," he said. "We take great pride in establishing an official industry-sponsored postdoctoral fellowship program here in Canada, enabling outstanding Ph.D. graduates to prepare for a career in the biopharmaceutical industry," he added. Under the program, Trillium will annually accept up to four postdoctoral fellows for an initial one-year term, with the possibility of an extension for an additional one or two years based on project needs. Through the program,pPostdoctoral fellows will be paired with mentors in a state-of- the-art laboratory environment, conducting independent research that is commercial and translational in nature.

The postdoctoral projects will be focused on cutting-edge research in immunology or oncology research, with the goal of elucidating disease biology or the mechanism of action of a novel drug candidate or target. Individuals will work independently but as part of cross-functional teams made up of scientists and research associates. Additional information on the program can be found at: http://trilliumtherapeutics.com/Contact/Careers/IndustryPostdoc

The University of Saskatchewan marked the official launch of its unique Plant Phenotyping and Imaging Research Centre (P2IRC) with an international symposium and demonstration of new drone technology to be used in novel crop development approaches. The creation of the P2IRC stems from a $37.2-million award over seven years from the Canada First Research Excellence Fund (CFREF) for the research program "Designing Crops for Global Food Security"—one of only five CFREF grants awarded across Canada last year in the inaugural competition. The new centre will not only enhance the U of S biosciences cluster—but also capitalize on one of the largest clusters of food-related researchers in the world." The P2IRC is led by Maurice Moloney, executive-director of the university's Global Institute for Food Security (GIFS), who has been building a multi-disciplinary team of researchers from across the U of S campus and from other Canadian universities and centres. "By 2022, we hope to create a unique global resource for plant breeders seeking to develop new crop varieties at unprecedented speed and scale," Moloney said. "The new science we are developing at the P2IRC will elevate Canada's position as a global powerhouse in agricultural research and lead to commercial spin-offs involving field and aerial sensors, satellite imaging, robotics, and big data analytics." He noted the P2IRC is unique in that it combines plant genomics with crop phenotyping (the identification of useful traits), high-performance computing, and digital imaging technology, as well as undertakes research to address societal and developing world impacts. Research projects in four theme areas have undergone rigorous international peer review involving an eight-member International Scientific Advisory Committee that includes experts from Australia, Germany, France, and the U.K., several of whom are participating in the symposium. The new centre also involves partnerships with four Canadian universities, three international institutes, and more than 15 private and public organizations, including the National Research Council and Agriculture and Agri-Food Canada. The P2IRC is currently recruiting graduate students, technicians, software developers, and researchers. Over the seven years, 60 graduate students and 35 post-doctoral

fellows will be trained at P2IRC, and four or five new faculty positions will be created.

It was a banner week at 13 postsecondary institutions across Canada , as Kirsty Duncan, Canada's announced that the federal government will invest $900 million towards their research efforts through the Canada First Research Excellence Fund. Among the biggest recipients are the Université de Montréal, McGill University, Polytechnique Montréal, and HEC Montréal who together received $213 million to be split between three projects related to research in artificial intelligence, brain health, and medical technologies. According to a release, Polytechnique Montréal will use its hospital's infrastructure to shape future medical technologies for complex diseases, while McGill's Healthy Brains for Healthy Lives project will establish a central hub to work on projects to improve brain health in Canada. Two of the other more larger grant recipients included Dalhousie University, which is getting $93 million for its Safe and Sustainable Development of the Ocean Frontier to develop Canada's oceans, and the University of Waterloo which is receiving $72 million for Transform Quantum Technologies, an initiative for advancing its work in developing quantum technologies. Additionly, the University of Saskatchewen is receiving $77.84 million for World Water Futures: Solutions to Water Threats in an Era of Global Change, while the University of Guelph is receiving approximately $76.6 million for Food from Thought: Agricultural Systems for a Healthy Planet. Rounding out the list is the University of Alberta, getting $75 million for the Future Energy Systems Research Institute, the University of Calgary getting $75 million for the Global Research Initiative in Sustainable Low Carbon Unconventional Resources, Western University getting $66 million for BrainsCAN: Brain health for life, $63.744 million to Queen's University for the Canadian Particle Astrophysics Research Centre, $49.2 million going to Laurentian Universtiy for Metal Earth and York University receiving $33.3 million for Vision: Science to Applications (VISTA).

An interesting side note, the Canada First Research Excellence Fund was actually established by the Harper Conservatives in the 2014 budget to help support research on big ideas and big themes. The funding is allocated on a competitive basis with researchers applying to an arms-length selection board.

Borden Ladner Gervais (Canada's largest law firm) released their annual Life Signs report earlier this year, which looks at legal trends in the Canadian Life Sciences sector.  The report includes contributed articles from multi-disciplinary BLG  professionals  and as well from Canada's national  and regional life sciences associations. Jeff Graham, BLG's National Life Sciences

Group Leader, is here with us today to discuss genetic discrimination and Canadian law, just one of areas explored in this report

Welcome to Biotechnology Focus Podcast. I'm your host Shawn Lawrence.

We kick things off this week with the release of our latest issue of Biotechnology Focus and as such we thought we should give you our listeners a rundown or sneak peak of all the stories you'd see. As most or our listeners know, there are comprehensive changes to the way the Federal Government supports science and innovation in Canada coming with consultations on Canada's Innovation Agenda drawing to a close and this issue reaches your desk with this Call to Action in mind, beginning with our cover story on the results from our latest Hot Button Issue Survey. The survey was open to everyone in both the private and public sectors and at all organizational levels, from the c-level right on down. In all, 163 of you got back to us with what you'd like to see from the Innovation Agenda. Under the microscope for many of you was one of the biggest sources of government cash for private sector research and innovation in Canada, the Scientific Research and Experimental Development program or SR&ED's as they are more commonly known. It was interesting to see how many of you rely on this program, and why many of you feel this program needs not only to be maintained but expanded upon. Be sure to check out this and what other priorities were included in our special report. And for further reading on the SR&ED program, Elizabeth Pringle, the National Life Sciences Industry Lead for Ernst & Young LLP's Business Tax Incentives Practices, discusses recent changes to the program itself while suggesting more alternatives to strengthening the overall tax credit regime for life science companies in Canada, including looking south of the border for some inspiration. Moreover, she highlights some of the recent CRA programs added to help increase tax claim certainty and reduce the administrative burden of applying for SR&EDs including the Pre-Claim Consultation program which launched on June 29 and the Pre-Claim Review (PCR) pilot project which launched August 2. In addition to underlining your priorities that you'd like to get before

As most or our listeners know, there are comprehensive changes to the way the Federal Government supports science and innovation in Canada coming with consultations on Canada's Innovation Agenda drawing to a close and this issue reaches your desk with this Call to Action in mind, beginning with our cover story on the results from our latest Hot Button Issue Survey. The survey was open to everyone in both the private and public sectors and at all organizational levels, from the c-level right on down. In all, 163 of you got back to us with what you'd like to see from the Innovation Agenda. Under the microscope for many of you was one of the biggest sources of government cash for private sector research and innovation in Canada, the Scientific Research and Experimental Development program or SR&ED's as they are more commonly known. It was interesting to see how many of you rely on this program, and why many of you feel this program needs not only to be maintained but expanded upon. Be sure to check out this and what other priorities were included in our special report. And for further reading on the SR&ED program, Elizabeth Pringle, the National Life Sciences Industry Lead for Ernst & Young LLP's Business Tax Incentives Practices, discusses recent changes to the program itself while suggesting more alternatives to strengthening the overall tax credit regime for life science companies in Canada, including looking south of the border for some inspiration. Moreover, she highlights some of the recent CRA programs added to help increase tax claim certainty and reduce the administrative burden of applying for SR&EDs including the Pre-Claim Consultation program which launched on June 29 and the Pre-Claim Review (PCR) pilot project which launched August 2. In addition to underlining your priorities that you'd like to get before government, this issue also spotlights what makes Canadian biotech great, and highlights some of the areas we can become stronger. It starts with Wayne Schnarr's roundup piece, "Looking for success stories in Canadian Healthcare." As he explains, measuring success in Canada's health technology sector is no easy task, and varies depending on one's perspective. His article looks at the Canadian biotech landscape from the viewpoint of an investor and reminds us that they judge successful companies differently than government, economic development offices and the companies themselves. With the investors wish list in mind, he highlights some Canadian companies to keep an eye on, including one who is featured later in this podcast Cynapsus.

Unfortunately, investors may have missed the boat on that company, but we'll get to that later.

Next in our editorial lineup TO Health's Ella Korets-Smith, Montreal In Vivo's Frank Beraud and Teralys Capital's Cedric Bisson cowritten piece "Canada Poised to be best in the World in Life Sciences Sector" recognizes Canada as a world class biotech hub whose greatest strength is working together to promote our successes in this arena. However, they add that there is still room to grow, and we must begin by looking to other jurisdictions for best practices especially in areas such as venture capital investment.

Next, Zymeworks lead man Ali Tehrani, no stranger to success, and perhaps the type of entrepreneur we need to hear more ideas from on how to improve the industry, asks the question why isn't Canadian biotech with all its great science, visionary entrepreneurs and angel investors, ranked among the best in the world. In addition to posing the question, he endevors to answer it, offering three near and long-term solutions. We have all this and more packed into this issue, so be sure to sign up for our digital edition service to see this issue and past issues as well.

Story 2

Our first news story this week is a science story, and features a breast cancer research breakthrough. Specifically, a team of researchers in Canada have discovered that mutations found outside of genes that accumulate in estrogen receptor positive breast tumours throughout their development act as the dominant culprits that drive the disease.

The multi-institutional research team collaborated with the Princess Margaret Genomics Centre and Bioinformatics group to analyze changes in the DNA sequence that accumulate in patients' tumours with respect to the epigenetic identity of estrogen receptor-positive breast cancer cells..

The research, published online in Nature Genetics, focuses on the most common type of breast cancer, estrogen receptor positive, and was led by principal investigator Dr. Mathieu Lupien, senior scientist, Princess Margaret Cancer Centre, University Health Network and associate professor in the Department of Medical Biophysics, University of Toronto. His team investigated acquired mutations found outside of genes through the power of epigenetics, and identified that functional regulatory components can be altered to impact the expression of genes to promote breast cancer development.

Dr. Lupien discusses the study in the following audio provided by UHN Toronto

Dr.Lupien's research builds on a previous study that identified why 44 known genetic variations increased breast cancer risk (Nature Genetics, Sept. 23, 2012). He adds that the convergence of more knowledge about inherited risk variants and the role of acquired mutations should readily enable translating the science into more precise clinical tests to diagnose and monitor patients.

His work was funded by The National Cancer Institute at the National Institute of Health, The Princess Margaret Cancer Foundation, Canadian Cancer Society, Susan G. Komen Foundation, the Ontario Institute for Cancer Research, the Canadian Institute of Health Research and a

Movember Rising Star award from Prostate Cancer Canada.

Story 3

Enerkem Inc., a Canadian headquartered waste-to- biofuels and chemicals producer, has obtained certification from the International Sustainability and Carbon Certification (ISCC) system and will now be able to begin biomethanol production at its Biofuels full-scale facility in Edmonton,AB. According to the company the third-party certification confirms that Enerkem meets high ecological and social sustainability requirements. Enerkem makes biofuels and renewable chemicals from non-recyclable municipal solid waste. The company owns a full-scale commercial facility in Alberta as well as a demonstration plant and a pilot facility in Quebec.

The company is also developing additional biorefineries in North America and globally, based on its modular manufacturing approach. With the certification, the Edmonton biorefinery is now the first ISCC certified plant in the world to convert municipal solid waste into biomethanol.

Story 4

In business news, Laval's ProMetic Life Sciences has entered into a binding agreement to acquire Belleville, Ontario's Telesta Therapeutics in an all share transaction. By way of a plan of arrangement under the Canada Business Corporations Act, ProMetic will acquire all of the share capital of Telesta at a share price of $0.14 payable in ProMetic common shares. The number of common shares to be issued by ProMetic will be based on the volume-weighted average closing price (VWAP) of ProMetic's common shares for the five (5) trading days prior to the closing date of the acquisition. According to ProMetic, the deal gives the company the opportunity for further integration of its manufacturing capabilities and longer term capacity expansion in a 150,000 sq. ft. facility in Belleville, ON. At the same time it gives  ProMetic  a significant foothold in Ontario, consolidating its presence as a major player in the Canadian market. Completion of the deal is subject to the approval of Telesta's shareholders and a number of customary closing conditions including court and regulatory approvals (such as the approval of the Toronto Stock Exchange). The acquisition is expected to close in early November, 2016.

Story 5

Rapid infectious disease test developer bioLytical Laboratories has filed two patent applications with the U.S. Patent and Trademark Office relating to its multiple colour rapid detection assay systems for the detection of antibodies to pathogens including Zika and other flaviviruses. The two new patent applications are based on the company's INSTI platform and describe methods and kits allowing either simultaneous or sequential detection of different antibody classes to multiple pathogens using a single sample of blood or blood product. The first patent application describes a method for detecting active or past infection using Zika as an example. The second patent application describes a method for differentiating early from later stages of infection.

According to the company, these technology breakthroughs could revolutionize point-of- care rapid assays for detection of acute infections including Zika. The Flaviviridae family has become a major concern in many areas of South America and more recently in the continental U.S.  As of early February 2016, the Brazil Ministry of Health has estimated that 500,000 to 1,500,000 cases of Zika virus disease have occurred since the beginning of the outbreak. The company says that with the high degree of cross-reactivity within the flaviviridae family, a diagnostic assay that can rapidly detect and differentiate between them, and determine new vs old infection, is of upmost importance.

Story 6

Our final, and perhaps biggest story this week is a Canadian biotech business news story with Marlbourough, MA based-Sunovion Pharmaceuticals announcing that it is buying Toronto's Cynapsus Therapeutics Inc. for approximately CDN$820 million. The transaction is the biggest the industry has seen in awhile for sure and has already received unanimous approval by the board of directors of both companies and The acquisition will be funded with cash on hand and is expected to close in the fourth quarter of 2016 (third quarter of Sunovion's fiscal year). Pursuant to the terms of the agreement, and upon closing of the proposed transaction, shareholders of Cynapsus will receive US$40.50 per common share in cash, and holders of warrants and stock options will receive a cash payment equal to the difference between US$40.50 and the exercise price of such warrant or stock option. The offer of US$40.50 per common share in cash represents a premium of 123 per cent based on the volume weighted average closing price of Cynapsus' common shares on the NASDAQ Global Market for the last twenty trading days. As part of the acquisition, Sunovion is getting Cynapsus' lead product candidate, APL-130277, a sublingual thin film formulation of apomorphine designed to be an easy-to- use on-demand treatment option for managing OFF episodes associated with Parkinson's disease (PD), a chronic, progressive neurodegenerative disease that affects more than four million worldwide. One of the biggest benefits of the sublingual thin film delivery of apomorphine is that avoids the normal gastrointestinal route of delivery used by most PD medications. Prior to the deal, Cynapsus had just announced that the Phase 3 clinical program for APL-130277 was nearing completion and that the company had plans to submit a new drug application (NDA) to the FDA in the first half of 2017.Nobuhiko Tamura, chairman and CEO, Sunovion said that his company sees APL-130277 as a novel late-stage candidate with the potential to make a real difference for patients and their families. He adds that Sunovian has high regard for the Cynapsus team led by Anthony Giovinazzo, president and CEO of Cynapsus and the work they have done with the APL-130277 program. For Cynapsus, the deal culminates years of dedicated work, and Anthony J. Giovinazzo, president and CEO, Cynapsus believes Sunovian is the ideal company to take APL-130277 forward. values Cynapsus at approximately CDN$820 million. The transaction will be completed by way of a plan of arrangement under the Canada Business

Corporations Act. The arrangement will require approval of at least two-thirds of the votes cast by Cynapsus shareholders and warrantholders voting together as a single class at a special meeting of such securityholders of Cynapsus. This meeting will be held in October.

With that we've come to the end of this week's program. We hope you enjoyed it. A big thanks to our production manager Laskey Hart and the rest of the Biotechnology Focus team. You can find past episodes online at www.biotechnologyfocus.ca and we're always looking for your feedback, story

ideas and suggestions so we'd love to hear from you. Simply reach out to us on twitter:

@BiotechFocus or by email [email protected].

Zymeworks, ProMetic Life Sciences, Innovative Targeting Solutions all back in the news again; Saskatchewan researchers make a breakthrough on a devastating pig virus, and healthcare behemoth GE partners with STEMCELL Technologies.We have this and more on this week's Biotechnology Focus Podcast!

Welcome to Biotechnology Focus Podcast. I'm your host Shawn Lawrence. Story 1 We kick things off this week in the prairies, where in less than a year, University of Saskatchewan (U of S) scientists have developed and tested a prototype vaccine that could protect the North American swine industry from a virus that has killed more than eight million pigs and cost more than $400 million in lost income since 2013. The Porcine Epidemic Diarrhea Virus (PEDV) hit the U.S. in 2013 and spread to Canada in 2014. It is a coronavirus, a virus group which includes important emerging human diseases such as SARS and MERS. It was first discovered in Europe, and has become increasingly problematic in Asian countries. Occurring only in pigs, PEDV can kill up to 100 per cent of infected piglets. Using its new containment Level 3 facility, the Universtiy of Saskatchewan Vaccine and Infectious Disease Organization-International Vaccine Centre (VIDO-InterVac) quickly launched a vaccine development project. The project has resulted in a prototype vaccine that protects 100 per cent of the piglets who have received it, according to  Dr. Volker Gerdts, VIDO-InterVac's research director. The successful vaccine results have since triggered the interest of several animal health companies including Huvepharma, which has partnered with VIDO-InterVac to develop the technology for commercial production in North America. With the support of the swine industry, the vaccine is now undergoing field testing in Saskatchewan, as well as in Manitoba where it is being used to help protect piglets from a recent PEDV outbreak. VIDO-InterVac director Andrew Potter said such a project wouldn't be possible without this facility, adding that "This is a perfect example of why InterVac was constructed – it is one of the only facilities available internationally with the capacity to conduct vaccine development and testing on this scale for emerging infectious diseases and It helps Canada remain prepared to quickly respond to outbreaks like this," he said. The PEDV vaccine development project has been supported by a variety of funders including the Government of Saskatchewan (ADF), Sask Pork, and the Canadian Swine Health Network. Story 2 The US Food and Drug Administration (FDA) has given Toronto's Trillium Therapeutics Inc. the go-ahead to initiate a Phase 1 clinical trial for its lead drug candidate, TTI-621 (SIRPaFc), in solid tumours and mycosis fungoides. Trillium is developing TTI-621 as a novel checkpoint inhibitor of the innate immune system, and the drug is currently being evaluated in an ongoing Phase 1 dose escalation study in patients with relapsed or refractory hematologic malignancies. Patient enrollment in the Phase 1 trial is anticipated to commence by year end. The trial will be multicenter and open-label, with TTI-621 being delivered to patients with relapsed and refractory, percutaneously-accessible cancers by intratumoral injection that increase in dose and dosing frequency to characterize safety, pharmacokinetics, pharmacodynamics and preliminary evidence of antitumour activity. In addition, detailed evaluation of serial, on-treatment tumor biopsies of both injected and non-injected cancer lesions will help characterize tumor microenvironment changes anticipated with CD47 blockade.

Story 3 As part of its strategy to leverage and attract investor interest to Québec's life sciences sector, the Fonds de solidarité FTQ has made a $15 million investment in Genesys Capital's latest venture fund Genesys Ventures III. The fund itself will used to back companies at the seed-stage, considered by many in the industry the valley of death, to help these companies advance technologies and products for unmet medical needs. Genesys Capital is one of the largest Canadian-based venture capital firms exclusively focused on the life sciences industry. Of note, Knight Therapeutics Inc., a specialized biopharmaceutical company based in Montreal, QC is also committing $1 million to Canadian-based life sciences venture capital fund Genesys Ventures III LP. For Knight the investment is the eighth life sciences equity fund investment Knight has made to date, having committed over $125 million. In terms of the Genesys Ventures III fund, Genesys Capital says it has closed $90 million of its $125 million target for the fund, while Managing director Damian Lamb adds that the firm expects to reach its goal for Genesys Ventures III early next year. Should the fund reach this target, it would be the largest Genesys has raised in its 16-year history. Story 4 Zymeworks is back in the news this week as the United States Food and Drug Administration (FDA) has accepted the company's Investigational New Drug (IND) application for its lead product ZW25 as a treatment for certain HER2-expressing cancers. ZW25 is a novel bi-specific antibody, developed using the company's Azymetric™ platform, to target two different epitopes (bi-paratopic targeting) of the human epidermal growth factor receptor 2 (HER2) proteinThe protein is known to be over-expressed on the surface of many tumour types, including certain breast, gastric, lung, and ovarian cancers. ZW25 will be evaluated in the clinic for safety as well as efficacy in patients with tumours with low to moderate levels of HER2 expression. The company anticipates it will begin a Phase 1 clinical trial for ZW25 in late August of this year. Additionally, last week  the FDA granted Orphan Drug Designation to ZW25 and a second product, ZW33 for the treatment of ovarian cancer. ZW33 is a drug-conjugated version of ZW25 that is currently in development in preparation for an IND filing in early 2017. Orphan designation qualifies Zymeworks for a number of development incentives, including tax credits for clinical testing and marketing exclusivity for a period of seven years if ZW25 and/or ZW33 is approved for this indication.

Story 5 The Ontario Institute for Cancer Research (OICR), Thermo Fisher Scientific and Queen's University are collaborating on a research study to help bring more targeted diagnosis and treatment to breast cancer patients in the future. The study, led by Dr. John Bartlett, director of OICR's Transformative Pathology Program, and Dr. Harriet Feilotter, Department of Pathology and Molecular Medicine, Queen's University, aims to identify mutations and copy number changes found in breast cancer samples and establish whether these abnormalities correlate with on-market drugs, available clinical trials or published studies. OICR-affiliated researchers and collaborators at Queen's University and Sunnybrook Health Sciences Centre will process the same breast cancer samples to establish whether the results are reproducible at different sites. This study will also characterize more than 400 additional retrospective breast cancer samples supporting ongoing clinical research efforts of Dr. Bartlett's team at OICR, which strives to improve the clinical management of the disease. Collaborators Drs. Martin Yaffe and Arun Seth at Sunnybrook Health Sciences Centre will provide laboratory space and additional technical support for the study. Dr. Yaffe is also co-leader of OICR's Smarter Imaging and Imaging Translation Programs. The study will use Thermo Fisher's Oncomine Comprehensive Assay, a targeted, next-generation sequencing (NGS) research tool that analyzes 143 genes relevant to cancer and which is the NGS assay used for the NCI-MATCH study in the United States. The data generated can be further studied with the Oncomine Knowledgebase Reporter, which is a curated set of published evidence that matches driver genetic variants with relevant information, such as on-market drugs, available clinical trials, or published studies. The findings of the OICR study will be used to assess the technology and could inform subsequent clinical trials. Drs. Feilotter and Bartlett have also engaged six laboratories in Ontario, including at Hamilton Health Sciences, London Health Sciences Centre, Ottawa General Hospital, Sunnybrook Health Sciences Centre, Sudbury Health Science North and University Health Network to look at the robustness and reproducibility of the assay across different cancer samples. This collaboration could extend the findings of this study beyond breast cancer to other common cancers. Story 6 Vancouver's Innovative Targeting Solutions has teamed up with yet another major life science company, this time striking a research collaboration deal with Janssen Biotech, Inc. (Janssen), through Johnson & Johnson Innovation.  The deal will allow Janssen to utilize Innovative Targeting Solutions' proprietary HuTARG™ research platform to discover antibody candidates useful for modulating immune responses in autoimmunity or cancer. The HuTARG™ protein engineering platform is able to engineer both T-cell receptors and fully human antibodies that bind major histocompatibility complex (MHC)/peptide complexes displaying fragments of intracellular proteins of interest.  Essentially, it allows researchers to generate and engineer fully human antibodies. The technology underlying the platform is based upon the natural process of V(D)J recombination, employed by the human immune system to produce a diverse repertoire of antibodies. Details of the collaboration including the specific targets, number of targets, and specific therapeutic indications have not yet been disclosed nor have financial details. In June, ITS announced a research collaboration with Merck, to utilize the HuTARG™ research platform to help identify and develop biologic therapeutic candidates directed towards targets that have historically been a challenge for biologic therapies, and just two weeks ago, ITS also partnered with another Vancouver-based company known for its own string of deals with pharma, Zymeworks. Other disclosed ITS collaborators include Novartis and Amgen. Story 7 A few months ago, Biotechnology Focus spoke with Phil Vanek (of GE Healthcare) and Michael May (of CCRM) about BridGE@CCRM, the centre they co-created at the MaRS West Towere to help accelerate the creation of cellular therapeutic tools. Now GE is taking another step in its mission to bring the right infrastructure to the global cell therapy industry, with another unique partnership here in Canada, signing an exclusive licensing agreement with Vancouver-based STEMCELL Technologies Inc., to develop and commercialize cGMP grade versions of STEMCELL's T-Cell reagents for the isolation, activation, and culture of T-cells in clinical applications. Both parties say these reagents are critical tools in the development and manufacturing of cell and gene therapies intended for administration to patients.

The following proprietary reagents from STEMCELL Technologies will be qualified as cGMP-grade materials and licensed by GE Healthcare:  ImmunoCult™ Human CD3/CD28 T-Cell Activator, ImmunoCult™ Human CD3/CD28/CD2 T-Cell Activator, EasySep™ Release Human CD3 Positive Selection Kit and the ImmunoCult™–XF T-Cell Expansion Medium.

According to Allen Eaves, president and CEO of STEMCELL Technologies Inc. this partnership with GE will give STI customers the confidence of a path to the clinic with a suite of critical cGMP grade T-cell reagents as well as help the industry evolve and make these promising therapies, such as CAR-T cells and TCR-engineered T cells, a clinical reality.

The agreement closely follows GE's acquisition of Biosafe Group SA, as well as many other investments in the space to make available a turnkey, scalable, flexible manufacturing solution that will enable access to these critical therapies. With that we've come to the end of this week's program. We hope you enjoyed it. A big thanks to our production manager Laskey Hart and the rest of the Biotechnology Focus team. You can find past episodes online at www.biotechnologyfocus.ca and we're always looking for your feedback, story ideas and suggestions so we'd love to hear from you. Simply reach out to us on twitter: @BiotechFocus  or by email [email protected]. For all of us here at Biotechnology Focus, thank you for listening.

ProMetic Life Science hits two significant clinical milestones, Antibe Therapeutics reports promising Phase 2 results, In BC researchers, take a closer look at the DNA of the world's greatest wines, and using genomics to treat and track tuberculosis We have this and more on this week's Biotechnology Focus Podcast!

Welcome to Biotechnology Focus Podcast. I'm your host Shawn Lawrence.

Story 1 We start this week's show in beautiful British Columbia, highlighting two local projects with an international flavor.

The first story sees UBC researchers Dan Durall and Mansak (Ben) Tantikachornkiat getting ever closer to identifying the biological personalities of the world's greatest wines.

In a recent study, published in the International Journal of Food Microbiology , the duo developed a technique that combines a process to identify the full spectrum of DNA in yeast and bacteria samples with a technique that distinguishes between live and dead micro-organisms. As Durall, an associate professor of biology at UBC's Okanagan campus, explains, since only live micro-organisms are relevant in the various stages of fermentation as they relate to the senses, this study provides some of the important tools that will be necessary to determine why different types of wine taste and smell as they do. Their findings could also lead to the identification and elimination of micro-organisms that are responsible for spoilage. In undertaking the study, the pair used a number of different kinds of yeast and bacteria specimens, including those typically found in wine fermentations. Key in the development of the new scientific technique was the use of a light-sensitive dye, propidium monoazide, which binds to dead DNA and prevents it from being detected. This allows scientists to identify and focus on the more relevant aspects of a test sample. According to Tantikachornkiat, this technique has allowed them to quickly and accurately monitor in one experiment what previously could have taken multiple experiments and months of trial and error. The next stages of their research will focus this technique on different types of wine making methods to see how they change micro-organisms that affect the final wine product.

Story 2 Our second BC story focuses on a new collaborative project involving the BC Centre for Disease Control (BCCDC), Oxford University and Public Health England (PHE). Together they are working to build data-sharing capacity between eachother to accelerate the use of genomics as a tool for the diagnosis, treatment and tracking of tuberculosis (TB). Led by Dr. Jennifer Gardy at BCCDC and Dr. Derrick Crook, University of Oxford and PHE, the research project is exploring how to communicate the complex data from a genomics-based test in a simple and effective laboratory report allowing clinicians, many of whom have not worked with genomic data before, to quickly and easily find the information and get the interpretation they need to ensure a direct benefit for patients. Funded in part by Genome BC, the project also supports PHE and BCCDC's efforts to validate the use of a genomic platform in a clinical setting through developing user-friendly reports to assist doctors in faster and more effective diagnosis and treatment. The use of genomics in the clinic means patients will have access to the most effective treatment several weeks earlier. Through a previous collaboration, the researchers have already demonstrated that using genomics to diagnose and characterize TB infections can reduce the time to diagnose and fully characterize an infection from an average of 31 days to just five days. Genomics also provides important information on the drug resistance profile of the tuberculosis strain, which helps doctors to identify the best treatment and avoid using antibiotics that will not be effective. Valued at $168,000, the initiative called SMAC: Sharing Mycobacterial Analytic Capacity will use techniques from the field of information visualization to design the better laboratory reporting form. Through a series of online surveys and iterative designs, the researchers hope to develop a simple, two-page report that describes a patient's diagnosis, the antibiotics that are predicted to work to treat the infection, and whether or not the patient is part of an outbreak. As part of SMAC, the UK and Canadian teams are also sharing resources and expertise in TB genomics and bioinformatics in order to accelerate the clinical validation and implementation of genomics-based TB diagnostics, first in the UK, and ultimately in BC. The partnership is a product of a MOU signed by Genome British Columbia and Genomics England last year to improve diagnostic capability and outcomes for patients with cancer, rare diseases and infectious diseases.

Story 3 In Atlanta, Georgia, Toronto based med tech company Synaptive Medical has launched a revolutionary brain surgery technology at Emory University Hospital. The technology, called BrightMatter™ is an innovative neurosurgery solution that offers advanced imaging, surgical planning and navigation through robotic visualization. Synaptive's technology shares a common imaging hub, which analyzes and assesses the quality of imaging scans in real-time prior to surgical planning and creates the foundation for a clinically-integrated imaging informatics research platform. Using an imaging method called diffusion tensor imaging, or DTI, BrightMatter enhances MRI images of the entire brain's pathways, allowing physicians to consider approaches for navigating around critical structures in neurological surgery. Synaptive's integrated imaging and navigation systems allow physicians to see details that can't be seen with the naked eye or a standard MRI, and may allow access to brain locations previously deemed inoperable. The automatic positioning system with an attached camera follows the physician's tools, showing an image of the patient's anatomy with unprecedented detail. This robotic arm includes a hands-free optical visualization system that allows for better surgical ergonomics, facilitates collaboration with operating room staff, and consumes less surgical time without the need to manipulate cumbersome optics.

Dr. Gustavo Pradilla, an Emory assistant professor of Neurosurgery, and chief of neurosurgery for Grady, co-director of the Grady Skull Base Center, and director of the Cerebrovascular Research Laboratory said that acquiring Synaptive's platform will bring innovative neurosurgical treatments that are the next technological frontier in intraoperative navigation, robotic-assisted visualization, corridor-based neurosurgery and clinical informatics. He adds that the technology will expand the hospitals ability to treat previously inoperable lesions in delicate areas of the brain, leading to safer and more efficient procedures, smaller incisions, shorter hospital stays.

Story 4 In clinical trial news, Toronto's Antibe Therapeutics Inc. has posted positive results from its Phase 2 clinical trial of ATB-346 in osteoarthritis (OA). ATB-346, is an NSAID (non-steroidal anti-inflammatory drug), and a hydrogen sulfide-releasing derivative of naproxen, the most-prescribed NSAID in North America. As part of the trial, 12 patients with OA of the knee were treated once daily for 10 days with the drug at a dose of 250 mg. The dose contains one-sixth of the typical daily dose of naproxen for treating OA. According to the company, the lower dose was found to be very effective at reducing pain, and equal to or better than naproxen or celecoxib in comparable studies. The drug was also found to be safe and well-tolerated. As part of the trial, patients recorded their level of pain one day prior to starting treatment and again on days four and 10 of treatment. The "WOMAC pain scale", the gold standard in arthritis clinical trials, was used as the measure of beneficial effect. The enhanced effectiveness of ATB-346 as compared to the market-leading drugs for osteoarthritis was a pleasant surprise, particularly considering the low dose of ATB-346 that was used said both the company's chief science officer John Wallace and the company's CEO Dan Legault. Legault added that the company plans to expeditiously perform additional clinical trials to confirm the results seen in this phase 2 study, and explore the effectiveness of even lower doses of ATB-346. The Phase 2 clinical trial was carried out in Toronto, Canada by Topstone Research Ltd.

Story 5 A research team at the Krembil Research Institute has discovered a pair of tissue biomarkers that directly contribute to the harmful joint degeneration associated with spine osteoarthritis. In a study study, published in the Journal of Clinical Investigation Insight, the researchers were able to show that elevated levels of both of these biomarkers cause inflammation, cartilage destruction and collagen depletion. Osteoarthritis affects about three million Canadians and is characterized by a breakdown of the protective cartilage found in the body's spine, hand, knee and hip joints. There is no known cure. The study involved tissue biopsies from 55 patients undergoing decompression or discectomy at the Krembil Neuroscience Centre at Toronto Western Hospital. As part of the study, the research team – led by Dr. Mohit Kapoor at the Krembil Research Institute and comprising Dr. Akihiro Nakamura, a post-doctoral fellow, and Dr. Y. Raja Rampersaud, a clinical expert and spine surgeon – explored the role, function and signaling mechanisms of two tissue biomarkers: microRNA-181a-5p and microRNA-4454. The study screened 2,100 microRNAs and found that measuring the levels of these two specific biomarkers can help clinicians determine the stage to which the disease has progressed, and provide a tool for determining the degree of cartilage destruction.

Dr. Kapoor discusses his team's discovery of the pair of tissue biomarkers in the following audio provided by (Video: UHN From 0:26-1:09,1:30-2:02).

The discovery represents the end of the first stage of research. The team is now investigating whether these biomarkers can be detected in the blood – which would help clinicians more simply determine the stage of spine osteoarthritis – and whether further studying the biomarkers will allow researchers to halt and reverse spine degeneration.

Story 6 It's been a week filled with successful milestones for Laval, QC's ProMetic Life Sciences. A developer of products used in the purification of biologics, drug development, proteomics and the removal of pathogens, the company announced on August 9th it had completed enrollment of the adult patient cohort for its pivotal intravenous immunoglobulin (IVIG) Phase3 clinical trial for the treatment of primary immunodeficiency diseases (PIDD). The company also announced on August 11 that it had completed patients enrolment of the congenital plasminogen deficient patients in its pivotal phase 2/3 clinical trial required for the accelerated regulatory approval pathway with the U.S. Food and Drug Administration In terms of the Phase 3 trial, completion of enrollment for the adult patient population is five months ahead of schedule and puts the company on the fast track to becoming the first Canadian-based company to locally produce IVIG. It's also a further indication of the near-term commercial prospect of what will be the company's second plasma protein.

According to company CEO and president Pierre Laurin, Canadian patients are amongst the largest consumers of IVIG on a per capita basis worldwide and the demand continues to grow at a rapid pace. He believes that the manufacturing advantages provided by the company's proprietary PPPS™ technology can help alleviate Canada's current dependence on foreign plasma derived therapeutics. IVIG is a preparation of antibodies purified from plasma donations from normal individuals. It is indicated for the maintenance treatment of patients with primary immunodeficiencies including common variable immunodeficiency, X-linked agammaglobulinemia, severe combined immunodeficiency and as a treatment of immune thrombocytopenic purpura (ITP). It is also used for the treatment of many other autoimmune diseases, including Guillain-Barré syndrome, Kawasaki disease. The Phase 3 trial is an open label, single arm, two-cohort multicenter study investigating the safety, tolerability, efficacy and pharmacokinetics of ProMetic's plasma derived IVIG in a total of 75 patients suffering from PIDD, and the adult cohort includes the 50 enrolled adults (cohort 1) and will also include 25 children (cohort 2). ProMetic anticipates the completion of enrollment for cohort 2 to go quickly with completion of the IVIG Phase 3 clinical trial expected in the second half of 2017.

As for the second trial, the FDA has agreed to an accelerated regulatory approval pathway, given the rarity of the condition and the related unmet medical need. To secure an accelerated pathway approval, a drug must treat a serious condition, provide a meaningful advantage over available therapies and demonstrate an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit. Dr John Moran, Chief Medical Officer of ProMetic commented that the ongoing clinical trial has enabled ProMetic to meet the primary end-point of achieving the targeted increase in plasma concentration of plasminogen and to define the optimal treatment regimen.

Plasminogen is a naturally occurring protein that is synthesized by the liver and circulates in the blood. Activated plasminogen, plasmin, is a fundamental component of the fibrinolytic system and is the main enzyme involved in the lysis of blood clots and clearance of extravasated fibrin. Plasminogen is therefore vital in wound healing, cell migration, tissue remodeling, angiogenesis and embryogenesis. ProMetic's Plasminogen has received an Orphan Drug Designation by the FDA and the European Commission for the US and the European markets respectively. ProMetic also received a Fast Track Designation by the FDA, a process designed to facilitate the development and expedite review of drugs and biologics intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.

With that we've come to the end of this week's program. We hope you enjoyed it. Thanks to Laskey Hart our production manager. You can find us online at www.biotechnologyfocus.ca and we're always looking for your feedback, story ideas and suggestions so we'd love to hear from you. Simply reach out to us on twitter: @BiotechFocus .

For all of us here at Biotechnology Focus, thank you for listening.

Genome Canada names its latest GAPP Recipients, DelMar Pharma lists on NASDAQ, and OPKO health acquires Toronto's Transition Therapeutics. We have this and more on this week's Biotechnology Focus Podcast!

Welcome to Biotechnology Focus Podcast. I'm your host Shawn Lawrence.

Story 1 We start this week's show in the biotech business world with a longtime player on the Toronto Life Science scene being acquired. Miami based OPKO Health says it is buying clinical stage Toronto-based biotechnology company Transition Therapeutics, in an all-stock transaction valued at about $60 million. Through the acquisition of Transition, OPKO will take over the full clinical portfolio of the company including a treatment for type 2 diabetes and obesity, a treatment for low testosterone and a third neuropsychiatric drug candidate that targets patients with Alzheimer's disease and Down syndrome. The first product, Transition's lead metabolic drug candidate TT401, is a once- or twice-weekly oxyntomodulin for type 2 diabetes and obesity that the company calls the most advanced candidate among glucagon-like peptide 1 (GLP1)/glucagon receptor dual agonists. In a recently completed Phase 2 study of 420 patients with type 2 diabetes, subjects receiving the highest dose of TT401 peptide once weekly demonstrated significantly superior weight loss compared with currently approved extended release exenatide and placebo after 12 and 24 weeks of treatment. TT401 also provided a reduction in HbA1c, a marker of sugar metabolism, similar to exenatide at weeks 12 and 24. Transition had regained full development and commercialization rights to TT401 in April of this year after Eli Lilly and Co pulled out of an existing development partnership that had the two companies working together on what they called at the time a next generation diabetes drug. In doing, Lilly opted to not advance the compound into Phase 3. The assets is a fit for Opko as it already has a couple of development stage oxyntomodulin in its pipeline – including MOD-6031, a long lasting version, and TT401 fits nicely as a complimentary shorter term treatment. The second product included in the deal is Transition's TT701, a once-daily oral selective androgen receptor modulator for patients with androgen deficiency. In terms of its clinical showing, In a 12-week study of 350 male subjects, TT701 showed significantly decreased fat mass and increased lean body mass and muscle strength without significantly changing prostate specific antigen levels. The third product is ELND005, a neuropsychiatric drug candidate that is an orally administered small molecule. It has completed Phase 2 clinical studies in Alzheimer's disease and Down syndrome patients, however it failed in this trial. This is probably why many news outlets reported that only two products were being acquired in this deal. It will be interesting to see what Opko Health has in mind for its future clinical development. Both companies expect the transaction to close during the second half of 2016, subject to approval of Transition Therapeutics stockholders and other customary conditions.

Story 2

Our next business story involves DelMar Pharmaceuticals, a BC-based company developing and commercializing cancer therapies in new orphan drug indications. The company commenced trading on July 12 on the NASDAQ Capital Market under the trade symbol DMPI.In honor of the listing, the company also got to ring the Opening Bell at the NASDAQ MarketSite in Times Square, New York City, on July 14, 2016. Jeffrey Bacha, chairman and CEO of DelMar Pharmaceuticals commented that listing on NASDAQ will better position the company in broadening its shareholder base, increase its appeal to institutional investors, and provide improved liquidity. The company's lead drug in development, VAL-083, is currently undergoing clinical trials in the U.S. as a potential treatment for refractory glioblastoma multiforme. It has been extensively studied by the U.S. National Cancer Institute, and is currently approved for the treatment of chronic myelogenous leukemia and lung cancer in China. Published pre-clinical and clinical data suggest that VAL-083 may be active against a range of tumour types via a novel mechanism of action that could provide improved treatment options for patients. And now for a quick break, and when we return, and when we come back we'll talk about Health Canada's plan to protect Canadians against the increasing risk of antimicrobial resistance.

If you could have a face-to-face conversation with government to discuss the life science industry, what would you say? Biotechnology Focus would like to give you the chance to engage with government and have your voices heard via our special "Hot Button Issues survey". We kicked off the survey June 24, and response thus far has been terrific, so much so that we've decided to extend its run time through to Friday July 22. We want to hear from you on such hot topics as reimbursement of new technologies, funding challenges like access to capital, intellectual property, procurement, the state of the industry and more. This survey is open to everyone in the industry, including the business sector, academics, research institutions, service providers and more. It's also open to individuals at all levels, from the C-level to support staff. The timing for this survey couldn't be more right as the Federal government has just launched a call to help shape Canada's "Innovation Agenda", and, Canada's Minister of Innovation, Science and Economic Development Navdeep Bains has asked us all to step up and voice our opinions, to help further shape the future of Canada's knowledge based economy. This survey is your chance to get involved! Your answers will be featured in a special high profile issue that will be sent to Deputy Ministers, Assistant Deputy Ministers, Cabinet, Directors and Generals, working in Health Canada, Industry Canada, Agriculture & Agri-Food, Foreign Affairs & Internationals trade, the Privy Council, as well as other public sector executives in federal and most provincial departments. So, don't miss this opportunity to participate and have your voices heard. Simply visit www.surveymonkey.com/r/biotechfocus. Remember, Friday July 22, the survey closes.

And welcome back to the Biotechnology Focus Podcast, and to get us started on the second half our show we have some exciting funding news related to a partnership between a major charitable organization and an Ontario clinical stage company.

Story 3 The funding partnership involves Sernova Corp., a company based in London Ontario that is developing disruptive regenerative medicine technologies for the long-term treatment of chronic diseases including diabetes and hemophilia. They have entered into a research funding agreement with JDRF, a leading global organization that provides funds to type 1 diabetes research and is an advocate of such research. JDRF is providing Sernova up to $2.45 million and these funds will be used to advance human clinical trials of Sernova's CPS technologies for treatment of hypoglycemia unawareness patients with severe type 1 diabetes at a major transplantation center in the U.S. Type 1 Diabetes is a life-threatening disease in which the body's immune system mistakenly attacks and kills the pancreatic cells that produce insulin—a hormone that is essential for life because of its role to help the body use glucose. The goal of the study is to provide patients with hypoglycemia unawareness a novel cell therapy treatment utilizing Sernova's proprietary Cell Pouch System™, a highly vascularized, cell macro-encapsulated implantable and scalable device to reduce or eliminate the need for injections of exogenous insulin. JDRF has previously provided funding to advance the development of Sernova's technologies through a preclinical collaboration with Massachusetts General Hospital and according to Derek Rapp, JDRF president and CEO, JDRF is excited about this collaboration as well. Initiation of the clinical trial is scheduled for the second half of 2016.

Story 4 In an effort to protect Canadians against the increasing risk of antimicrobial resistance, Health Canada is taking the first steps towards implementing a new Federal Action Plan on Anti-Microbial Resistance. Antimicrobial resistance occurs when an antimicrobial drug – an antibiotic, antifungal or antiviral drug – is no longer effective at controlling an infection it was once used to treat. This can cause an increased risk of infection, longer infection times, and a greater risk of death. The decreasing effectiveness of antimicrobial drugs is having a significant impact on our ability to protect Canadians from infectious diseases. It also has profound impacts on our healthcare system, global trade, agriculture, environment and health sectors. As part of Health Canada's action plan, the department is seeking feedback on a regulatory proposal that it hopes will strengthen rules governing the importation, sale and use of antimicrobial drugs in livestock. This covers Surveillance – Detecting and monitoring trends and threats to inform strategies to reduce the risks and impacts of antimicrobial resistance, Stewardship – Conserving the effectiveness of existing treatments through infection prevention and control guidelines, education and awareness, regulations, and oversight, and finally Innovation – Finding new solutions to counteract loss in antimicrobial effectiveness through research and development. According Canada's Ministry of Health the proposed changes to the Food and Drug Regulations would restrict the importation of certain veterinary drugs, require drug manufacturers to follow stricter rules, require provision of sales information to regulators to allow for improved monitoring of antimicrobial use, and introduce an easier way for manufacturers to sell low-risk veterinary health products. Consultations on the proposed regulatory changes are already underway, running for a 75 day period, ending on September 8, 2016. The proposal was published in the Canada Gazette, Part I, on July 2, 2016.

Story 5 Our final story of the week features Genome Canada announcing the recipients of Round 5 funding under its Genomic Applications Partnership Program. GAPP partners academic researchers with users in the private and public sectors to promote genomics-derived solutions to address challenges or opportunities facing users. The projects are expected to have considerable economic and social impacts in the near term, spurring innovation, commercialization and growth in Canada. Further, through a Genome Canada and Mitacs partnership, GAPP supports training the next generation of graduate students and post-doctoral fellows. Several of the projects announced in this funding round involve research internships, which will prepare Canada's next entrepreneurs through hands-on experience. As part of the round, a total of $5.3 million in federal funding through Genome Canada will go to five projects, with an additional $11.6 million invested through partners including provinces, private and public sector organizations. The five projects in this round are: Dr. Casey Hubert, University of Calgary, who is working with the Nova Scotia Department of Energy to derisk offshore oil and gas exploration in the province using innovative genomic approaches. Dr. Christoph Borchers, University of Victoria and Dr. Gerald Batist, McGill University, who are working with the pharmaceutical company AstraZeneca to validate new technology that will help healthcare professionals match the right cancer treatment to the right patient. Dr. Charles Goulet, Université Laval, who is working with Vineland Research and Innovation Centre to use variation in aroma-related genes to develop more flavourful tomatoes. Dr. Elizabeth Edwards, University of Toronto, who is working with SiREM to use specific microbes to accelerate the rate of biodegradation and enable less expensive cleanup of contaminated industrial sites. And Dr. Xiao-Yan Wen, St. Michael's Hospital in Toronto, who is working with Edge Therapeutics Inc. to develop drugs for preventing intracerebral hemorrhage – a form of brain hemorrhage responsible for 10 per cent of all strokes. You can find out all the details of these GAPP Round 5 projects via a backgrounder on the Genome Canada website.

We're always looking for your feedback, story ideas and suggestions so we'd love to hear from you. Simply reach out to us on twitter: @BiotechFocus

Biotechnology Focus senior writer Shawn Lawrence goes one-on-one with Mike Woollatt, chief executive officer of the Canadian Venture Capital & Private Equity Association (CVCA), to discuss the upswing of venture capital investment in Canada, and to find out just  how much is going to Canadian biotech and life science companies.

Bellus Health is left reeling as its lead drug Kiacta fails its Phase 3 clinical study, Vancouver based QLT inks a merger deal with Aegerion Pharmaceuticals and looks to a fresh new start, and Biotechnology Focus relaunches its Hot Button Issue Survey. We have this and more on this weeks show!

Show Notes:

We kick things off this week with some bad news on both the business and the clinical trial front with BELLUS Health the rare disease drug development firm reporting top line results from its Phase 3 study of KIACTA™ (eprodisate) as a treatment of AA amyloidosis. The disease is characterized by secondary to severe chronic inflammation or infection leading to the formation and deposition of amyloid fibrils in organs, often resulting in end-stage renal disease and death. Currently there are no therapies available that target the disease directly. According to company CEO Roberto Bellini, KIACTA™ failed to meet its primary efficacy endpoint in slowing renal function decline. The news, announced on June 20, was somewhat surprising to both the company and its investors. Shares for the company on the TSE under the trade symbol BLU plummeted, going from $2.42 on June 17, to around $0.39 on the day of the announcement, holding at .40 cents today. Many investors had rallied to the company over the past year due to the drug being in later stages of clinical trial development.

Moreover, the science seemed sound as in earlier studies, KIACTA™ was shown to significantly delay the disease.  In both earlier studies and even in the failed trial, it was found to be safe and well tolerated over treatment periods of greater than four years. The confirmatory Phase 3 study of KIACTA™ was a global study across more than 70 sites in more than 25 countries that randomized 261 patients to receive either 800mg dose of KIACTA™ twice daily or placebo.

The  study was an event driven study that lasted five years meeting its completion target of 120 patient events linked to the deterioration of kidney function in January 2016. The company added that further analysis of the data is ongoing, and a more detailed data set of the KIACTA™

Phase 3 study will be presented July 6 at the XV International Symposium . Roberto Bellini, president and CEO of the company adds that while Bellus is disappointed that the Phase 3 study did not meet the primary efficacy endpoint, the full data set will be assessed to determine the best path forward. The company says in the aftermath of the failed trial, it will continue working on a number of other projects in its portfolio,  including its study of KIACTA™ as a treatment for sarcoidosis (set to enter Phase 2 trials), clinical development of Shigamab™ as a treatment for STEC-related Hemolytic Uremic Syndrome (sHUS), and the company's research-stage project for AL amyloidosis.

We go from negative news to the more positive, as QLT Inc., once upon a time Canada's hottest biotech company, and a company long-entrenched as a in the BC- biotech community inking a strategic merger agreement with Cambridge-based Aegerion Pharmaceuticals, Inc. The proposed deal has been approved by the board of directors of both companies, and is expected to close late in the third quarter or during the fourth quarter of 2016. Upon closing, QLT says it plans to change its name to Novelion Therapeutics Inc. adding that its common shares will trade on the NASDAQ Global Select Market and the Toronto Stock Exchange. QLT's current market cap is approximately $110 million (CDN) and Aegerion has a market cap of approximately $70 million (CDN) giving the new combined company a market value of approximately $180 million. This isn't QLT's first attempt to merge with another company, having been thwarted in the past in prior merger arrangements with Endo International, Auxilium Pharmaceuticals and InSite Vision. The planned merger with Endo International was terminated in October 2014, and likewise, Auxilium had also been carrying out plans to merge with QLT but chose instead to be bought by the aforementioned Endo International in a separate deal valued at $2.6 billion.

Likewise, last year, QLT and InSite announced a merger, but that deal fell through after InSite changed its mind and accepted a higher merger offer from Sun Pharmaceutical Industries. This time QLT won't be left at the alter so to speak, and likewise both companies under the Novelion Therapeutics Inc. banner will get a much needed rebrand and fresh start. The new company will also have quite a diversified portfolio consisting of Aegerion's two commercially branded products, Juxtapid® (lomitapide) capsules and Myalept® (metreleptin), and QLT's QLT091001 (Zuretinol Acetate or Zuretinol), a Phase 3-ready Ultra-Orphan Fast Track and Orphan Drug designated asset being developed for the treatment of Inherited Retinal Disease caused by underlying mutations in RPE65 or LRAT genes (IRD), which indication comprises Leber Congenital Amaurosis (LCA) and Retinitis Pigmentosa (RP). Aegerion's CEO, Mary Szela, will serve as CEO of Novelion following the close of the transaction. Additionally, an investor syndicate comprised of both new investors and existing shareholders of both companies has  committed to invest via a private placement approximately $22 million in QLT. The investment will be funded prior to the transaction close and is expected to provide Novelion with additional capital to support future operations and the potential opportunity for targeted business development initiatives.

In futher business news, TVM Life Science Ventures VII a venture capital fund domiciled in Montréal, Québec, has closed the fund's eleventh investment, establishing Mediti Pharma Inc., a company based in Montréal, Québec. The new company is developing a Phase 2 ready compound, MP-101, originally discovered by Eli Lilly and Company for the treatment of Alzheimer's disease (AD) psychosis. The behavioral symptoms associated with AD psychosis, include hallucinations, delusions and aggression. The company is managed by CEO Dr. Ivan Shaw, formerly with Merck Frosst, Aptalis and Actavis and neurobiologist and experienced drug developer and CFO Mark Cipriano, a Boston-based highly experienced and networked executive. As for the fund itself, and its initial association with the drug, it comes about as part of a unique arrangement between TVM Capital Life Science and numerous limited partners, which includes Eli Lilly and Company.  The goal of the fund is to finance and access innovation while managing risk and sharing reward.

Staying in Montreal, on the clinical trial front Montreal InVivo , the city's economic development organization with a focus on life sciences and health technologies (LSHT) sector. is partnering with Canadian Clinical Trails Coordinating Centre(CCTCC) to enhance Canada's clinical research capabilities. The partnership will result in the addition of Quebec clinical trial investigators "data from Montreal Goes Clinic to the Canadian ClinicalTrials Asset Map (CCTAM) and real-time updates down the line. The Montreal Goes Clinic itself is an initiative by Greater Montreal's life sciences"; cluster that aims to make the region a world-class centre of expertise in early stage clinical trials.  The goal of this initiative is to improve Greater Montreal's business environment by helping pharmaceutical, biotech, contract research organizations (CRO's) and medical device companies perform high quality clinical trials and run clinical proofs-of- concept, thus  increasing patient access to new treatments and improving medical practices by allowing clinicians to work with technologies of tomorrow and the best equipment.

Frank Béraud, CEO of Montréal InVivo adds that the portal will create more visibility for Quebec's researchers and investigators throughout Canada, and will also showcase the province's capabilities in clinical research. Dr. Shurjeel Choudhri, senior vice president and head, Medical and Scientific Affairs Bayer Inc. and the current Chair of the CCTCC Executive Committee adds that the CCTAM is a great example of the collaboration of multiple stakeholders from across Canada, including representatives from the Federal and Provincial governments, industry, the Canadian Institutes of Health Research and HealthCareCAN. He calls the addition of clinical investigator data from Montreal Goes Clinic an important step towards making the CCTAM a truly comprehensive inventory of Canada's clinical research assets.

If you could have a face-to- face conversation with government to discuss the life science industry, both the good and the bad what would you say? A number of years ago Biotechnology Focus used to run a special Hot Button Issue survey which gave our readers the opportunity to engage with government to get their concerns before the right people and have their voices heard. Well, with the Justin Trudeau government coming up to its ninth month in office, we thought the timing was right to do it all again.

The survey kicked off yesterday and will run to July 15, 2016. Similar to our past "Hot Button" issue surveys, we want your feedback on such things as regulatory policy including reimbursement of new technologies, funding challenges, intellectual property protection, the state of the industry and much more. This survey is open to everyone in the industry, from the business sector, to research institutions, and at all levels, essentially, if your field is biotech, bioscience or life science in general we want to hear from you. This already is vibrant industry, with strong representation at major research and knowledge translation institutions, a large base of Canadian biotech SMEs and MMEs, and we're home to many of the world's largest and leading pharmaceutical companies. Moreover, the timing for this survey couldn't be better as the Federal government as mentioned in last week's podcast has just launched the "Innovation Agenda", an action plan to both redesign and redefine how we as a nation support innovation and growth. They have embarked on this Innovation Agenda in partnership and coordination with the private sector; provinces, territories and municipalities; universities and colleges; and the not for-profit sector and they have also already taken the first steps in this plan already with Budget 2016, providing support for health research, genomics, regenerative medicine, brain research and drug development and committed $800 million to support innovation networks and clusters. So there's obviously lots that government is doing right to make Canada a friendly environment for our industry, but can we do more? Canada's Minister of Innovation, Science and Economic Development Navdeep Bains has asked us all to step up and voice our opinions, to help further shape the future of Canada's knowledge based economy. And our Hot Button Issue survey is your chance to do that!

Your answers will be featured in a special high profile issue that will be poly-bagged with our sister publication Canadian Government Executive magazine and sent to Deputy Ministers, Assistant Deputy Ministers, Cabinet, Directors and Generals, working in Health Canada, Industry Canada, Agriculture & Agri-Food, Foreign Affairs & Internationals trade, the Privy Council as well as other public sector executives in federal and provincial departments. So here is your chance to help influence the Innovation Agenda.

And remember, by taking only a couple of minutes to complete our survey, you're helping us grow Canada's knowledge-based economy, one answer at a time. You can access the survey by clicking the link below this podcast.

That wraps up this weeks show, you can find us online at www.biotechnologyfocus.ca. Likewise, we're always looking for feedback, story ideas and suggestions so we'd love to hear from you. Simply reach out to us on twitter: @BiotechFocus , email at [email protected]

For all of us here at Biotechnology Focus, thank you for listening.

Our first story takes us to Southwestern Ontario, where the government of Ontario and Bioindustrial Innovation Canada have partnered to launch a new commercialization hub in Sarnia-Lambton As part of the partnership, the Government of Ontario says it will invest $3 million over four years through the Business Growth Initiative to help build the Centre of

Excellence for the Commercialization of Sustainable Chemistry Innovations at the Western Sarnia-Lambton Research Park, Canada's largest clean-tech incubator. The new centre will focus on bridging critical gaps and addressing commercialization challenges facing the industrial biotechnology and manufacturing sectors, including developing expertise, forging commercial partnerships, and accessing risk-capital to help bring discoveries and technologies from the lab to the marketplace. The good news goes further as its expected that 400 high-value jobs will be created at the new site. The region is known for its Biomanufacturing prowess, turning sustainable feedstock — renewable resources such as agricultural and forestry by-products and wastes — into energy, value-added chemicals and materials for use in a variety of everyday products such as plastics, personal care products, automotive parts and food additives. The use of plant-derived chemicals and bio-manufacturing processes can significantly reduce greenhouse gas emissions when compared to conventional petroleum based production methods. One example of a successful company in this space is BioAmber, a sustainable chemical company in Sarnia that produces plant-derived succinic acid and is able to achieve a 100 per cent reduction in GHGs over conventional methods. Similarly, EcoSynthetix, a Burlington-based renewable chemicals company produces paper coatings while cutting GHG emissions by 63 per cent compared to the norm. The funding for the new centre was previously announced as part of this year's provincial budget.

In regulatory news coming out of Quebec, the Government there, through its Ministry of Health has denied Theratechnologies bid to have EGRIFTA®, an injectable prescription medicine to reduce the excess abdominal fat in HIV-infected patients with lipodystrophy, added to a list of reimbursed medications. Company CEO and president Luc Tanguay called the decision by the province, which also happens to be the province where EGRIFTA® was discovered, regretful.

"The government is sending a message that supporting research is not a priority," he said via a release adding that it is quite paradoxical that, after supporting the development of EGRIFTA® through tax credits, the Quebec government will not accept to reimburse it." The decision, made by the Quebec Minister of Health, Gaétan Barrette, was based on a recommendation by the Institut National d'Excellence en Santé et Services Sociaux(INESSS). Theratechnologies submitted to INESSS a comprehensive dossier which included data that led to public and private reimbursement by a large number of governmental agencies and insurers in Canada and in the United States. INESSS concluded that the decrease of visceral adipose tissue in HIV patients does not constitute a therapeutic advantage. Nevertheless, INESSS did recognize the efficacy EGRIFTA® and the quality of its clinical studies. In response, Theratechnologies did say that despite the decision, they will continue to work with the Ministry and INESSS, and that they will submit to INESSS, within the prescribed timelines, a request for a revision of their decision.

In Cancer Research News, the Movember Foundation, the Ontario Institute for Cancer Research (OICR) and Prostate Cancer Canada are providing $3 million in funding for a new Phase 3 clinical trial to evaluate if magnetic resonance imaging (MRI) can replace the current standard of care to diagnose prostate cancer. The trial, called PRECISE, will be led by Dr. Laurence Klotz of the Sunnybrook Research Institute in Toronto, a world leader in the field of prostate cancer research and in the global adoption of active surveillance, a standard practice to monitor patients with low risk prostate cancer. The primary objective of the multi-centre trial is to determine whether MRI imaging can spare some men from undergoing a biopsy and avoid the possible associated side effects. Dr. Klotz discusses the PRECISE TRIAL in the following audio...

Data management and analysis for the trial will be conducted by the Ontario Clinical Oncology Group (OCOG) in the Escarpment Cancer Research Institute, a Hamilton Health Sciences and McMaster University institute.

Earlier this month, five contenders of the third AmorChem KNOCK OUT™ competition climbed into the ring at a special Lumira-AmorChem hosted event to duke it out against a panel of Heavyweight Champions, or judges for a chance to win a coveted $500,000 financing from AmorChem. All the contenders displayed remarkable courage and agility in the ring, yet only one could stand victorious. In the end, Dr. Anne-Marie Mes-Masson, CRCHUM scientific director at the Institut du cancer de Montréal, for her project focused on the development of small molecule inhibitors of the Ran GTPase, was named the champion. The panel of "champion" judges included: Richard Lesniewski, a pharmaceutical industry veteran formerly from Abbott (AbbVie), GlaxoSmithKline and Madison Vaccines; John Gillard, science entrepreneur formerly from Merck Canada, BioChem Pharma and Aegera Therapeutics; John Bell, scientist-entrepreneur from the Ottawa Hospital Research Institute and the University of Ottawa; and Lloyd Segal, best known as a serial biotech entrepreneur and investor at Persistence Capital Partners and Trimera Capital. Christopher Hall, a renowned radio and newspaper columnist and comedian, was the master of ceremony. Both Elizabeth Douvile and Ines Holzbaur,, general partners at AmorChem which we've highlighted in past podacast shows called the competition a success, achieving its goal of increasing the Quebec and Canadian research communities' awareness of AmorChem and in reaching out to researchers who may not have been aware of the possibility of commercializing aspects of their work.

Among the other competitors were: Dr. John Coles, University of Toronto & Dr. Jason Maynes, The Hospital for Sick Children and University of Toronto; Dr. Julie Forman Kay, The Hospital for Sick Children and University of Toronto & Dr. Nahum Sonenberg, McGill University; Dr. Réjean Lapointe, Université de Montréal and CRCHUM & Dr. Sophie Lerouge, École de technologie supérieure and CRCHUM; and Dr. Moutih Rafei, Université de Montréal.

The Lumira-AmorChem conference also highlighted the respective roles of the two venture capital groups in the financing of the life sciences sector, and included presentations from Lumira Capital portfolio companies and from AmorChem's first spin-off company, Mperia.

Our final stories this week come out of Ottawa where the Government of Canada had two announcements related to supporting Canada's science community. The first saw Kirsty Duncan, Minister of Science, launch an independent review of federal funding for fundamental science, to assess the program machinery that is currently in place to support science and scientists in Canada. The scope of the review includes the three granting councils along with certain federally funded organizations such as the Canada Foundation for Innovation. The review will be led by an independent panel of distinguished research leaders and innovators including Dr. David Naylor, former president of the University of Toronto and chair of the panel. Other panelists include: Dr. Robert Birgeneau, former chancellor, University of California, Berkeley, Dr. Martha Crago, Vice-President, Research, Dalhousie University, Mike Lazaridis, co-founder, Quantum Valley Investments, Dr. Claudia Malacrida, Associate Vice-President, Research, University of Lethbridge, Dr. Art McDonald, former director of the Sudbury Neutrino Laboratory, Nobel

Laureate, Dr. Martha Piper, interim president, University of British Columbia, Dr. Rémi Quirion, Chief Scientist, Quebec, Dr. Anne Wilson, Canadian Institute for Advanced Research Successful Societies Fellow and professor of psychology, Wilfrid Laurier University

The panel will spend the next six months seeking input from the research community and Canadians on how to optimize support for fundamental science in Canada. The panel will also survey international best practices for funding science and examine whether emerging researchers face barriers that prevent them from achieving career goals. In addition to collecting input from the research community, the panel will also invite Canadians to participate in the review through an online consultation.

In further Canadian Science Policy news Navdeep Bains, Minister of Innovation, Science and Economic Development, has put forward a new Innovation Agenda . Jointly with Minister of Science Kirsty Duncan, and, Minister of Small Business and Tourism Bardish Chagger, the initiative or Agenda will aim at building an inclusive and innovative Canada focused on six areas for action: including promoting an entrepreneurial and creative society, supporting global science excellence, building world-leading clusters and partnerships, growing companies and accelerating clean growth, competing in a digital world and improving ease of doing business.

These six action areas, which form the Innovation Agenda, will be the focus of a summer-longperiod of public engagement that will result in an action plan. Central to this plan will be a call to action to all sectors of society. Minister Bains invited all Canadians to share their ideas on the Agenda, and should watch for the launch of an interactive website that will allow them to offer their suggestions on positioning Canada as a global leader in innovation. In addition, round-table discussions will take place across the country.

That wraps up this weeks show, be sure to You can find us online at www.biotechnologyfocus.ca and follow us on twitter @BiotechFocus check out our latest issue on our website!

On this week's show, we recap all the Canadian news coming out of the San Francisco and BIO2016, including: four announcements from the Quebec delegation, Valeant makes manufacturing moves, Merck backs another BC antibody developer and much more!

http://biotechnologyfocus.ca/canada-bio-podcast-ep-20/

On this week's show, Hot of the Press, we rundown all the storiesfrom our June/July issue of Biotechnology Focus, BIOTECanada names of two of its Gold Leaf Award Winners, CQDM extends its reach to Germany, and ProNAi Therapeutics licenses the rights to a cancer fighting molecule from Japan.