Safety and tolerability of anselamimab in participants with light chain amyloidosis: Results from a phase 2 study.
Jason Valent, Jeffrey Zonder, E Scott Swenson et al.
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In 30 seconds
This phase 2, multicentre, open-label, sequential-cohort dose-selection study assessed anselamimab with standard anti-plasma-cell therapy in adults with AL amyloidosis and measurable hematologic disease. Among 25 participants, all experienced at least one treatment-emergent adverse event, mostly mild to moderate; 16 (64.0%) had serious events. Investigators adjudicated all serious and fatal events as unrelated to study drugs, and no dose-limiting toxicities occurred. Best cardiac response was reported in 47.4% of evaluable participants at Week 50, while best renal response was 85.7% at Weeks 62 and 98.
Key findings
- All 25 participants experienced at least one treatment-emergent adverse event, most of which were mild to moderate.
- Serious treatment-emergent adverse events occurred in 16 participants (64.0%); all serious and fatal events were adjudicated as unrelated to study drugs.
- Four participants (16.0%) discontinued study drug because of adverse events, and 3 (12.0%) experienced fatal adverse events.
- No dose-limiting toxicities occurred; best cardiac and renal responses were 47.4% at Week 50 and 85.7% at Weeks 62 and 98, respectively, among evaluable participants.
Why it matters
AL amyloidosis involves organ-damaging deposits that current therapies described in the study do not directly remove. This early clinical evaluation provides safety and tolerability information for an investigational antibody intended to target those deposits when given alongside standard anti-plasma-cell treatment. The reported organ responses are exploratory findings from a small study and should be interpreted in that context.
What to keep in mind
The study was small, open-label, and non-randomised, with sequential cohorts and no comparator group; organ responses were reported only among evaluable participants.