Longeveron Presents Long-term Survival Data for Lomecel-B in HLHS at CHSS 51st Annual Meeting

Longeveron Inc., a clinical-stage biotechnology company focused on regenerative medicine, has showcased significant long-term data from its ELPIS I Phase 1 clinical trial at the Congenital Heart Surgeons’ Society (CHSS) 51st Annual Meeting. The study's findings were presented on October 27-28, 2024, in Chicago, Illinois.

The company has reported exceptional long-term survival rates for patients with Hypoplastic Left Heart Syndrome (HLHS) treated with their product Lomecel-B™. HLHS is a severe congenital heart defect where the left ventricle is underdeveloped, impeding the heart’s ability to adequately pump blood. Despite current surgical interventions, survival rates to adolescence remain between 50% to 60%. The quest for improved treatments is crucial for enhancing both short and long-term outcomes for these infants.

The ELPIS I trial, an open-label and Phase 1 study, aimed at evaluating the safety of Lomecel-B™ in HLHS patients. These patients underwent the Glenn Procedure, a type of open-heart surgery, at around 4-5 months old. The results, previously published in the European Heart Journal Open in 2023, revealed that the primary safety endpoint was met. All patients survived, did not require transplants, and maintained expected growth rates one year post-treatment.

Further analysis involved following the ten ELPIS I participants (seven males and three females) for five years post-Glenn surgery and Lomecel-B™ treatment. The goal was to assess long-term survival and transplant-free survival. Comparisons were made with historical data from the Single Ventricle Reconstruction (SVR) Trial, which involved patients who had the same shunt type at Stage I Norwood operation followed by Glenn procedures. Key outcomes demonstrated a 100% survival rate without the need for heart transplants for five years among those treated with Lomecel-B™. In contrast, the SVR trial showed 83% survival with a 5.2% heart transplantation rate.

No major adverse cardiovascular events (MACE) or safety issues related to Lomecel-B™ were observed during the study. The data support Lomecel-B™ as a valuable adjunct to HLHS reconstruction surgery, potentially enhancing transplant-free survival.

Building on these promising results, the ELPIS II Phase 2b clinical trial is currently underway, comparing Lomecel-B™ with the standard care for HLHS. This trial involves 38 pediatric patients across twelve premier treatment institutions in the U.S. Conducted in collaboration with the National Heart, Lung, and Blood Institute (NHLBI) and supported by grants from the National Institutes of Health (NIH), the trial seeks to validate the initial positive outcomes of ELPIS I.

Lomecel-B™ has garnered multiple designations from the U.S. Food and Drug Administration (FDA), including Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation for HLHS. These recognitions facilitate greater regulatory support during the product's development.

Dr. Joshua Hare, Co-founder, Chief Science Officer, and Chairman at Longeveron, emphasized the significance of these findings. He underscored the potential of Lomecel-B™ to positively impact patients suffering from HLHS, a devastating condition. Encouraged by the ELPIS I data, Longeveron is actively progressing with the ELPIS II Phase 2b trial, which could form the basis for a Biologics License Application (BLA) for potential approval.

HLHS affects approximately 1,000 infants annually in the U.S. Current treatment involves a complex three-stage heart reconstruction surgery over five years, yet many infants fail to reach adolescence. Improving right ventricular function remains a critical unmet medical need, and Lomecel-B™ could play a crucial role in addressing this challenge.

Lomecel-B™ is derived from specialized cells in the bone marrow of young, healthy adult donors. Known as medicinal signaling cells (MSCs), these cells are vital to the body's repair mechanisms, performing functions such as tissue formation and responding to injury or disease. They secrete bioactive factors with immunomodulatory and regenerative properties, suggesting wide-ranging applications for rare and age-related diseases.

Longeveron continues to explore the therapeutic potential of Lomecel-B™ across various indications, including HLHS, Alzheimer's disease, and aging-related frailty, with multiple FDA designations supporting its development.