Prothena Corporation’s Phase III AFFIRM-AL trial of birtamimab in individuals with amyloid light-chain (AL) amyloidosis misses its primary endpoint of time to all-cause death, leading to the discontinuation of the therapy’s development.
The company will also halt the open-label extension of the trial based on these outcomes.
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The global, double-blind, placebo-controlled trial enrolled 207 newly diagnosed, treatment-naïve subjects with Mayo Stage IV AL amyloidosis.
Its secondary endpoints, including the six-minute walk test distance and the Short Form-36 version 2 Physical Component Score, were also not met.
The subjects in the active arm of the trial were administered birtamimab every 28 days at a dose of 24 mg/kg, with a maximum dose not surpassing 2500 mg.
Both the active and control arms were given a bortezomib-containing chemotherapy regimen as standard of care, and the daratumumab’s use was permitted.
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By GlobalDataDespite the trial’s outcomes, the therapy was found to be generally safe and well-tolerated, in line with its established safety profile.
Prothena anticipates to outline plans to minimise its current operating costs in June. Details of the company’s review of its business options will be communicated once the analysis and plans are finalised.
Prothena CEO and president Gene Kinney said: “This is not the outcome that we expected, and we are surprised and disappointed by these results for patients, their families and caregivers, and for the entire AL amyloidosis community.
“With these results, we believe that the most appropriate action is to discontinue all development of birtamimab. Prothena would like to thank all of the patients, their families and caregivers, and the investigators, site staff, and Prothenians that made this clinical trial possible.”
The company focuses on the pipeline of investigational therapeutics aimed at treating neurodegenerative and rare peripheral amyloid conditions.
Its pipeline includes programmes for diseases such as transthyretin amyloidosis (ATTR) amyloidosis with cardiomyopathy, Parkinson’s disease, Alzheimer’s disease, and other neurodegenerative conditions.
