Bellus Health Inc., a drug development company focused on rare diseases, today announced top-line results from the Phase 3 study of Kiacta (eprodisate) for the treatment of AA amyloidosis.
In the study, Kiacta did not meet the primary efficacy endpoint in slowing renal function decline. Kiacta was shown to be safe and well tolerated over treatment periods of greater than 4 years. Further analysis of the data is ongoing.
“While we are 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,” said Roberto Bellini, President and Chief Executive Officer of Belllus Health.
AA amyloidosis is a rare disease 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.
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 Phase 3 study was an event driven study that lasted 5 years meeting its completion target of 120 patient events linked to the deterioration of kidney function in January 2016.
A more detailed data set of the Kiacta Phase 3 study will be presented July 6 at the XV International Symposium on Amyloidosis.