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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 completed its targeted enrollment in May 2014 and is expected to conclude in 2016.

KIACTA™ is also being developed as a treatment in a second indication called sarcoidosis, a rare inflammatory condition that affects the lungs.

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™ proof of concept studies for 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 including the kidneys, heart, liver and peripheral nerves.