5 Year AA Amyloidosis Study Fails to Meet Primary Endpoint

James Radke, PhD

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.

Next Steps

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.
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