MIAMI, Florida — Longeveron Inc. (NASDAQ: LGVN), a clinical stage biotechnology company developing cellular therapy for life-threatening, rare pediatric and chronic aging-related conditions, today announced that the independent Data Monitoring Committee (DMC) has completed its final prespecified data review for the ongoing, fully enrolled, Phase 2b clinical trial (ELPIS II) evaluating laromestrocel (Lomecel-B®) as a potential adjunct therapy for hypoplastic left heart syndrome (HLHS). The DMC based its review on available data for all participating patients enrolled. The DMC performed a risk-benefit assessment, indicated no safety concerns, and approved the study to continue as designed to completion.
“We are grateful to the members of the DMC for their effort and diligence in this review,” said Nataliya Agafonova, M.D., Chief Medical Officer at Longeveron. “Based on the scientific rationale supporting laromestrocel and the clinical data generated to date, we remain encouraged by the potential of our investigational therapy in HLHS. With our Phase 2b clinical trial for HLHS having achieved full enrollment last June, we continue to anticipate top-line trial results in August 2026 and look forward to sharing the data.”
ELPIS II enrolled 40 pediatric patients at 12 leading pediatric cardiac centers across the country. The clinical trial is being conducted in collaboration with the National Heart, Lung, and Blood Institute (NHLBI) through grants from the National Institutes of Health (NIH). ELPIS II builds on findings from ELPIS I study, in which treated children demonstrated 100% transplant-free survival up to five years of age. Published historical outcomes in HLHS populations have reported substantial mortality despite surgical palliation.
The U.S. Food and Drug Administration (FDA) has granted laromestrocel Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation, for development for HLHS.
About Hypoplastic Left Heart Syndrome (HLHS)
HLHS is a rare congenital heart defect that effects approximately 1,000 infants per year in the U.S. Infants with HLHS are born with an underdeveloped left ventricle, which creates a life-threatening condition due to the heart’s inability to pump adequate amounts of blood throughout the body. The current treatment requires infants to undergo a complex three-stage heart reconstruction surgery process over the first five years of their life. Despite staged surgical palliation, long-term mortality and morbidity remain substantial, with progressive right ventricular dysfunction representing a major contributor to adverse outcomes. There is clearly an important unmet medical need to improve right ventricular function in these infants to positively impact both short- and long-term patient outcomes.
About laromestrocel (Lomecel-B®)
Laromestrocel is a living cell product made from specialized cells isolated from the bone marrow of young healthy adult donors. These specialized cells, known as mesenchymal stem cells (MSCs), are essential to our endogenous biological repair mechanism. MSCs have been shown to perform a number of complex functions in the body and support tissue repair and regenerative signaling pathways. They also have been shown to respond to sites of injury or disease and secrete bioactive factors that are immunomodulatory and regenerative. We believe that laromestrocel MSCs may have multiple potential mechanisms of action that may lead to anti-inflammatory, pro-vascular regenerative responses, and therefore may have broad application for a range of rare and aging related diseases.
About Longeveron Inc.
Longeveron is a clinical stage biotechnology company developing regenerative medicines to address unmet medical needs. The Company’s lead investigational product is laromestrocel (Lomecel-B™), an allogeneic mesenchymal stem cell (MSC) therapy product isolated from the bone marrow of young, healthy adult donors. Laromestrocel has multiple potential mechanisms of action encompassing pro-vascular, pro-regenerative, anti-inflammatory, and tissue repair and healing effects with broad potential applications across a spectrum of disease areas. Longeveron is pursuing four pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer’s disease (AD), Pediatric Dilated Cardiomyopathy (DCM) and Aging-related Frailty. Laromestrocel development programs have received five distinct and important FDA designations: for the HLHS program – Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation; and, for the AD program – Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation. For more information, visit www.longeveron.com or follow Longeveron on LinkedIn, X, and Instagram.
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