Developing drugs for rare diseases

BELLUS Health is focused on developing drugs for rare diseases, starting with conditions that affect the kidneys.

The lead program is KIACTA™, a novel drug candidate currently in a Phase III Confirmatory Study for the treatment of AA amyloidosis, a rare disease resulting in renal dysfunction that often leads to dialysis and death. KIACTA™ is partnered with global private equity firm Auven Therapeutics. The Phase III Confirmatory Study is expected to conclude by the end of the first quarter of 2016, with top-line data expected to be available in the middle of 2016.

KIACTA™ is also being developed as a treatment in a second indication called sarcoidosis, a rare, potentially fatal inflammatory condition that affects the lungs​. An investigational new drug application (IND) for a Phase II clinical study is expected to be filed with the U.S. Food and Drug Administration by the end of 2015.

In addition, BELLUS Health is developing Shigamab™, an antibody treatment for Hemolytic Uremic Syndrome caused by Shiga toxin-producing E. coli ("sHUS"). sHUS often leads to dialysis, chronic kidney disease and in some cases death, particularly in children. Shigamab™ pre-clinical studies for the treatment of sHUS are currently underway.

BELLUS Health’s pipeline also includes a research-stage project for the treatment of AL amyloidosis, a rare disease in which amyloid protein builds up and causes dysfunction in various parts of the body.