Longeveron Says the FDA No Longer Sees Trial for Heart Syndrome Treatment as Pivotal

By Kelly Cloonan

Longeveron said the Food and Drug Administration no longer refers to its Phase 2b clinical trial for a potential treatment for hypoplastic left heart syndrome (HLHS) as pivotal.

The biotechnology company said Friday the two parties did not land on an appropriate primary endpoint for the study in their Type C meeting in March. The FDA indicated the study's primary endpoint of right ventricle ejection fraction is not an appropriate endpoint to demonstrate efficacy, and indicated that the two parties would not be able to agree on a new primary endpoint while the trial remains ongoing given an interim analysis mandated and conducted by the National Institute of Health.

However, Longeveron said the FDA indicated it is willing to meet again when the study is complete to discuss the results and align on a potential path forward. During the Type C meeting, the FDA acknowledged that HLHS is a rare disease associated with significant morbidity and mortality with a high unmet medical need for safe and effective therapies, the company said.

Longeveron said it plans to submit a Sponsor Statistical Analysis Plan for the trial to the FDA with a composite primary endpoint and secondary endpoints for review and approval. The company said it remains optimistic that the trial results and other evidence will support filing a Biologics License Application following the readout of top-line results.

Longeveron expects top-line results from the Phase 2b clinical trial of the drug in August.

Write to Kelly Cloonan at kelly.cloonan@wsj.com

(END) Dow Jones Newswires

May 08, 2026 16:57 ET (20:57 GMT)