BELLUS Health Inc – “Our Big Shift Into Rare Diseases”

May 5, 2015

BELLUS Health Inc. (TSX: BLU) today published a new blog post on The Chairman’s Blog, written by the Company’s President and Chief Executive Officer, Roberto Bellini. is an exclusive online media publication that enables key executive officers a unique platform to share insights about their company and industry trends.

In his blog, Mr. Bellini discusses how Bellus has evolved from a multi-focus biotech company to a developer of drugs for rare diseases, starting with those that affect the kidneys. He highlights how this area has the potential to help rare disease patients and generate the greatest value for shareholders. Read the full blog post from Mr. Bellini on


The following is an excerpt from the post:

“Developing drugs for rare diseases starting with conditions that affect the kidneys.”  You can find this statement at the start of all our corporate presentations. It’s the first thing you see on our website and it’s repeated throughout our security filings. It is our mission.

Developing drugs for rare diseases hasn’t always been our focus though. When I first started in 2009 as Vice-President, Business Development, Bellus Health was a company focused around a technology, in this case a series of compounds that treated amyloid diseases.

Then, we had a pipeline that included a commercial stage nutraceutical, a Phase 3-ready program for a rare disease indication (AA amyloidosis) and a preclinical program for an Alzheimer’s disease drug.

How did we get to our current mission? Read on, this blog is about the principal reasons we decided to focus on the development of drugs for rare diseases.

Focused Business Model

In 2009, Bellus was doing a bit of everything. We had the full range of pharma activities from basic research including biology, chemistry and animal facilities to drug development and commercial activities.

We realized that as a small company we couldn’t be highly effective in all these different areas, so we decided to focus on what we knew how to do best – drug development.  This is the area that we have the most expertise. It’s also our opportunity to potentially help bring drugs to patients and concurrently generate the greatest value for our shareholders.

The corollary to this decision was halting our commercial activities and research functions. While this didn’t happen overnight, we shuttered our laboratories and sold off our commercial stage nutraceutical over the course of a few years.

Once completed, we had effectively reduced our footprint from more than 50 employees to nine. At the same time, we shifted to using contract organizations to conduct much of the work that had been previously done in-house.

Overall, this made us a leaner and more nimble organization.

Compete on a Global Basis

Rationalizing our business model wasn’t enough. It was also important to understand what kinds of drugs we wanted to develop. We were 100 percent committed to Kiacta in AA amyloidosis. We felt we had the expertise, the experience, and, with our partnership, the financing to move the project forward effectively.

This wasn’t the case for BLU-8499, our lead candidate in Alzheimer’s disease. It required too much funding and an ulterior level of expertise that we no longer had in-house.

Basically, we felt that we could neither effectively compete in the Alzheimer’s field nor have the right risk/return profile for our shareholders in this project. BLU-8499 was ultimately deemed non-core and licensed to Alzheon, a start-up based close to Boston, Massachusetts.

The Alzheon management team is a group that had strong expertise in Alzheimer’s disease and  they had recently raised a $10M Series A financing. This is good news for Alzheimer’s patients with another potential drug in development, and good news for Bellus shareholders who can reap the benefit of a share of future Alzheon revenues as well as a royalty linked to an eventual Alzheimer’s disease drug sales.

About BELLUS Health Inc.

BELLUS Health is a drug development company focused on rare diseases. It has a portfolio of rare disease projects including KIACTA(TM) in Phase III for AA amyloidosis, KIACTA(TM) for sarcoidosis, clinical stage Shigamab(TM) for sHUS and a research-stage project for AL amyloidosis. The lead program KIACTA(TM) is currently in a Phase III Confirmatory Study for the treatment of AA amyloidosis, an orphan indication resulting in renal dysfunction that often leads to dialysis and death. KIACTA(TM) is partnered with global private equity firm Auven Therapeutics.

About is an exclusive, online media publication where publicly and privately held firms alike share insights about their companies and industries. enables upper tier management to discuss issues that are of importance to their stakeholders, shareholders, and interested parties in an informal environment.


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