STAT+: Amylyx endocrine drug succeeds in pivotal study
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- Amylyx Pharmaceuticals said Tuesday its experimental drug achieved a 55% reduction in serious low blood sugar events versus placebo in a Phase 3 trial enrolling patients with post-bariatric hypoglycemia (PBH)
- The Phase 3 trial was statistically powered to detect just a 35% improvement over placebo, meaning the 55% result beat the prespecified bar by 20 percentage points
- Post-bariatric hypoglycemia causes sudden drops in blood sugar after bariatric surgery, with a serious event defined as a medical emergency that could involve loss of consciousness or seizure
- Amylyx co-CEO Justin Klee said any reduction in hypoglycemic events would be "very meaningful for people with PBH and their families," calling the large observed effect "just so exciting"
- The drug could become the first approved treatment for PBH if it clears regulatory review, filling a space where no FDA-approved therapy currently exists
Why it matters: Patients with post-bariatric hypoglycemia currently have no FDA-approved treatment for sudden, dangerous drops in blood sugar after bariatric surgery. A 55% reduction in events that can cause loss of consciousness or seizures — beating the trial's 35% statistical target — gives Amylyx a credible path to first-mover approval in an unmet-need indication.
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