Longeveron receives FDA approval for pediatric heart therapy IND

Published 08/07/2025, 14:24
Longeveron receives FDA approval for pediatric heart therapy IND

MIAMI - Longeveron Inc. (NASDAQ:LGVN), a clinical-stage biotechnology company with a current market capitalization of $19.4 million, has received U.S. Food and Drug Administration (FDA) approval for its Investigational New Drug (IND) application for laromestrocel, a stem cell therapy targeting pediatric dilated cardiomyopathy (DCM), the company announced Tuesday. According to InvestingPro data, the company maintains a strong liquidity position with more cash than debt on its balance sheet, though analysts note rapid cash utilization.

The approved IND allows Longeveron to bypass early-stage trials and proceed directly to a Phase 2 pivotal registration clinical trial, which is expected to begin in the first half of 2026. This development comes as the company maintains a healthy current ratio of 5.61, indicating strong short-term financial stability despite not being profitable over the last twelve months.

Pediatric DCM is a rare condition where heart chambers become enlarged and weakened, leading to difficulty pumping blood. According to information provided in the company’s statement, the disease affects at least 100,000 children worldwide, with approximately 40% of affected children requiring a heart transplant or dying within two years of diagnosis.

"Current treatment for DCM focuses on managing symptoms, improving heart function, and preventing complications rather than addressing the underlying cause," said Barry Byrne, Director of the Powell Gene Therapy Center at the University of Florida, in the press release.

Laromestrocel is described as an allogeneic cellular therapy derived from mesenchymal stem cells with potential anti-inflammatory and regenerative properties. The company suggests the therapy may help repair damaged heart tissue.

DCM is reportedly the most common form of cardiomyopathy in children, representing 50-60% of all pediatric cardiomyopathy cases, with higher prevalence in boys and infants under age one.

Longeveron’s development program for laromestrocel has previously received multiple FDA designations for other conditions, including Orphan Drug, Fast Track, and Rare Pediatric Disease designations for hypoplastic left heart syndrome, and Regenerative Medicine Advanced Therapy and Fast Track designations for Alzheimer’s disease.

The company is also investigating the therapy for aging-related frailty, according to the announcement.

In other recent news, Longeveron Inc. reported a significant decline in its first-quarter 2025 revenues, with a 30% decrease compared to the same period in 2024, resulting in a net loss of $5 million. Despite these financial challenges, the company remains focused on its clinical trials and potential market opportunities. The U.S. Food and Drug Administration approved Longeveron’s Investigational New Drug application for laromestrocel, allowing the company to proceed directly to a Phase 2 pivotal registration clinical trial for pediatric dilated cardiomyopathy. Longeveron also completed enrollment for its Phase 2b trial evaluating laromestrocel for Hypoplastic Left Heart Syndrome, with top-line results expected in the third quarter of 2026. The company appointed Than Powell as Chief Business Officer to lead its business strategy and international efforts. Powell brings over 25 years of experience from companies like GSK and Eli Lilly. The FDA has granted laromestrocel several designations, including Orphan Drug and Fast Track, for its various programs. These developments highlight Longeveron’s ongoing efforts to advance its stem cell therapy research amid financial setbacks.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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