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News
A promising stem cell therapy for a severe infant heart defect didn't hit its primary goal in a mid-stage trial. Longeveron's laromestrocel failed to improve right-heart pumping function at 12 months compared to standard care alone in infants with hypoplastic left heart syndrome. The company's stock dropped nearly 60% after hours, and it's now exploring strategic options.
Longeveron's experimental stem cell therapy, laromestrocel, has stumbled in a mid-stage clinical trial for hypoplastic left heart syndrome (HLHS) — a rare and severe birth defect where the left side of the heart is underdeveloped. The drug, tested alongside standard surgery in 40 infants, failed to show meaningful improvement in right-heart pumping function at the 12-month mark compared to surgery alone. The company's shares nosedived nearly 60% in after-hours trading following the announcement.
Despite missing the primary endpoint, the therapy showed a favorable safety profile with no new concerns identified. Longeveron says it plans to review the full data set and meet with the FDA to discuss potential next steps before making any final decisions about the program's future.
By the Numbers:
Why it matters: HLHS carries a high mortality risk, and effective adjunct therapies remain an unmet need. While the trial missed its primary goal, the survival signal in longer-term follow-up data may still offer a path forward — pending FDA discussions and further analysis.
