Longeveron Inc. (NASDAQ: LGVN) announced positive long-term data from the ELPIS I Phase 1 clinical trial, demonstrating a 100% five-year transplant-free survival rate in hypoplastic left heart syndrome (HLHS) patients treated with Lomecel-B™ after undergoing the Glenn procedure. The data were presented at the Congenital Heart Surgeons’ Society (CHSS) 51st Annual Meeting in Chicago. This outcome suggests a significant improvement compared to historical data from the Single Ventricle Reconstruction (SVR) Trial.
Long-Term Outcomes of Lomecel-B in HLHS
The ELPIS I trial, an open-label Phase 1 study, evaluated the safety of Lomecel-B in HLHS patients. The follow-up study assessed long-term survival benefits over five years post-Glenn surgery. Key findings revealed that the Kaplan-Meier survival rate was 100% in patients treated with Lomecel-B, with no heart transplants required. In contrast, the SVR trial reported 83% transplant-free survival (95% CI= [77.4, 89.3]) and a 5.2% heart transplantation rate (95% CI= [2.0, 8.3]) within the same timeframe.
Expert Commentary
"HLHS is a devastating disease for patients and their families, so I am impressed and very encouraged to see the continued long-term survival of patients treated with Lomecel-BTM in the ELPIS I study," said Sunjay Kaushal, M.D., Ph.D., Professor of Surgery, Cardiovascular and Thoracic Surgery at University of Nevada, Las Vegas. He further emphasized the unmet medical need to improve right ventricular function in these infants to positively impact both short- and long-term patient outcomes, suggesting stem cell therapy, particularly Lomecel-B, has the potential to play a critical role in treating these patients.
Ongoing ELPIS II Phase 2b Trial
Building on the promising results of ELPIS I, the ELPIS II Phase 2b clinical trial is currently underway. This randomized trial is further evaluating Lomecel-B, compared to standard of care, as a potential adjunct therapy for HLHS. The trial is enrolling 38 pediatric patients across twelve institutions and is being conducted in collaboration with the National Heart, Lung, and Blood Institute (NHLBI) through grants from the National Institutes of Health (NIH).
Regulatory Designations
The U.S. Food and Drug Administration (FDA) has granted Lomecel-B Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation, which could expedite its development for HLHS.
About Lomecel-B
Lomecel-B is a cell-based therapy derived from medicinal signaling cells (MSCs) isolated from the bone marrow of healthy adult donors. These MSCs are believed to promote tissue repair, modulate the immune system, and stimulate regenerative responses. Longeveron believes Lomecel-B may have broad applications for various rare and aging-related diseases.
About Hypoplastic Left Heart Syndrome (HLHS)
HLHS is a severe congenital heart defect affecting approximately 1,000 infants annually in the U.S. It is characterized by an underdeveloped left ventricle, impairing the heart's ability to pump blood effectively. Current treatment involves a complex three-stage surgical process, but even with this, only 50% to 60% of infants survive to adolescence due to right ventricular failure.
