ASH 2023 | Safety of birtamimab in AL amyloidosis: pooled analysis from multiple studies

I will be presenting a poster at this American Society of Hematology meeting on pooled safety analysis of birtamimab in patients treated on clinical trials for systemic Al amyloidosis. Birtamimab is an anti-fibril agent that is developed against the C-terminus of kappa and lambda light chains, which targets the amyloid deposits as well as it targets the soluble oligomers. It targets the amyloid deposits by activation of macrophages and creating phagocytosis.

This molecule, birtamimab, has been studied in PRONTO trial and VITAL trial, Phase I and II clinical trials with open-label extension and also crossover with open-label extension of PRONTO trial. And the pooled safety analysis was performed in 302 patients for who had participated in all these trials. The median exposure of birtamimab of these 302 patients was 12.2 months, and the most commonly reported side effects or adverse events were fatigue, nausea, constipation, and dyspnea. Moreover, it was also shown that all cause adverse events as well as birtamimab-related adverse events were no different from birtamimab versus placebo in PRONTO and VITAL trials, because these were the placebo-controlled trials. Birtamimab is currently being investigated for safety and efficacy in patients with newly diagnosed stage four Al amyloidosis in AFFIRM-AL trial, which is currently enrolling and accruing.