A phase 3 AA amyloidosis study has failed to meet its primary endpoint.
The drug being tested, Kiacta (eprodisate), has had a long history of showing potential for AA amyloidosis then failing to deliver in the end. Back in 2006, the FDA told the drug’s developer at the time (Neurochem
) that more data would be needed to get the drug approved following a review.
Ten years later, NeuroChem is now called Bellus Health and the Canada-based company is working with Auven Therapeutics, a company headquartered in the US Virgin Islands, to get the drug ready for FDA review.
And in the latest announcement that the phase 3 study did not meet its primary endpoint, the prospect of the drug getting FDA approval seems less likely.
Eprosidate binds to the glycosaminoglycan binding sites on serum amyloid A (SAA), which inhibits the formation of the glycosaminoglycan-amyloid fibril aggregate. This prevents the formation of amyloid deposits in certain organs, especially the kidneys, in AA amyloidosis.
In a press release
, Roberto Bellini, President and Chief Executive Officer of Bellus Health said, "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."
The confirmatory Phase 3 study took 5 years to complete and randomized 261 patients with AA Amyloidosis to receive eprosidate (800mg) or placebo twice a day. Data from the study will be presented July 6 at the XV International Symposium on Amyloidosis
About AA Amyloidosis
AA amyloidosis is a rare condition in which amyloid A accumulates in major organs, particularly the kidneys, and can lead to organ dysfunction, failure, and eventually death. AA amyloidosis is a rare complication of more common auto-immune conditions or infections such as rheumatoid arthritis or tuberculosis.
There is currently no available treatment for AA amyloidosis.