Longeveron’s pediatric heart stem-cell trial misses primary endpoint, prompting cost cuts

Longeveron reported that its phase 2b Elpis II trial of the stem-cell product laromestrocel in infants with hypoplastic left heart syndrome failed to meet its primary efficacy endpoint.

The study enrolled 40 infants who received the cell therapy during stage-2 palliative surgery, while a control group received no drug. At 12 months, the change in right ventricular ejection fraction was not statistically different between the two arms, leading to the missed endpoint.

Safety signals were modest; no deaths occurred in the treated group versus one in controls, and major adverse cardiovascular events were lower in the treatment arm (12 vs 19), though the analysis did not reach statistical significance.

Facing the setback and limited cash, Longeveron said it will review all options, cut costs, continue exploratory analyses, and discuss a possible path forward with the FDA.