Celtic Therapeutics and Bellus Health initiate confirmatory Phase III clinical study for KIACTA

21-12-2010: BELLUS Health Inc. and Celtic Therapeutics announced that a global confirmatory phase III clinical study was initiated on December 14, 2010 for KIACTA™ (eprodisate). KIACTA™ is a drug candidate being developed for the treatment of AA amyloidosis, a life-threatening orphan disease that occurs in patients with long-lasting inflammatory conditions, most commonly due to rheumatoid arthritis. The study is designed to confirm the safety and efficacy of KIACTA™ in preventing renal function decline in patients with AA amyloidosis.

"KIACTA™ has the potential to offer a much awaited treatment to patients with AA amyloidosis, a devastating disease affecting an estimated 50,000 patients in the United States, Europe and Japan, for which there is currently no specific treatment available," said Dr. Peter B. Corr, Co-Founder and General Partner of Celtic Therapeutics. "In its initial phase III clinical study, KIACTA™ reduced the risk of renal decline or mortality by 42% (Cox proportional hazards regression model, p=0.025) as compared to placebo over a two-year period, and showed an excellent safety profile. KIACTA™ also continues to be safe and well tolerated upon chronic administration, with some patients exposed to the compound for more than eight years in the compassionate use program. These past findings make us confident regarding the outcome of the confirmatory phase III study," Dr. Corr added.

The international, randomized, double-blind, placebo-controlled, event-driven study will involve approximately 230 patients diagnosed with AA amyloidosis enrolled from approximately 90 sites in 30 countries worldwide. The primary efficacy composite endpoint of the study is based on patients reaching an event linked to the deterioration of their renal function, defined as a persistent decrease in creatinine clearance (CrCl) of 40% or more, a persistent increase in serum creatinine (SCr) of 80% or more, or progression to end-stage renal disease (ESRD). The primary efficacy analysis will be the time from baseline to the first renal deterioration event of the primary composite endpoint.

The study is tentatively planned to end when approximately 104 renal events have occurred. The number of events and enrollment target for the study will be reviewed and may be refined following an interim analysis which will be conducted following the occurrence of at least 20 events and 15 months after the enrollment of the first patient. It is currently estimated that the study will be completed in 2014.