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.