Longeveron Shares Halve After Heart Drug Fails Key Clinical Trial

Shares of Longeveron Inc. (NASDAQ:LGVN) plunged by 50% in premarket trading on Thursday after the biotechnology company announced its ELPIS II Phase 2b clinical trial did not meet its primary goal. The trial was evaluating the company's investigational stem cell therapy, laromestrocel, for a rare and complex congenital heart defect in infants.

Trial Fails to Meet Primary Endpoint

The ELPIS II study assessed the efficacy of laromestrocel in 40 infants with hypoplastic left heart syndrome (HLHS). The trial's primary efficacy endpoint was the change from baseline in right ventricular ejection fraction at the 12-month mark, a key measure of heart function.

Longeveron reported that the trial failed to show a statistically significant improvement in the treatment group compared to the control group. The key results included:

• The least-squares mean difference between the groups was -0.7 percentage points.
• The result was not statistically significant, with a p-value of 0.8336.

Company Explores Strategic Options

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In response to the trial results, Longeveron's management has initiated a process to review all strategic options with the stated goal of maximizing shareholder value. The company announced it plans to engage an investment bank to serve as a strategic advisor.

Alongside the strategic review, Longeveron will implement cash conservation measures to control costs. The company stated it still intends to discuss the full results with the U.S. Food and Drug Administration (FDA) to determine potential next steps for the HLHS development program.

Secondary Data and Future Plans

While the primary endpoint was missed, Longeveron noted some exploratory clinical outcomes. There were no deaths in the laromestrocel-treated group over 12 months, compared to one death in the control group. Major adverse cardiovascular events were approximately 31% fewer in the laromestrocel arm, though this analysis was also not statistically significant.

The company confirmed that laromestrocel's safety profile was consistent with prior trials and no new safety signals were identified. Longeveron said it plans to pursue funding to continue developing laromestrocel for other indications, including longevity and aging-related frailty, noting its recent selection as a finalist in the XPRIZE Healthspan competition.