Longeveron Inc. (NASDAQ: LGVN) announced positive long-term data from a follow-on study to its ELPIS I Phase 1 clinical trial, showcasing the potential of Lomecel-B in treating Hypoplastic Left Heart Syndrome (HLHS). The data, presented at the Congenital Heart Surgeons’ Society (CHSS) 51st Annual Meeting, highlighted a 100% five-year transplant-free survival rate in HLHS patients treated with Lomecel-B after the Stage 2 Glenn surgery. This compares favorably to the 83% survival rate observed in the Single Ventricle Reconstruction (SVR) trial, which also reported a 5.2% heart transplantation rate.
Long-Term Outcomes of Lomecel-B in HLHS
The ELPIS I trial initially focused on evaluating the safety of Lomecel-B in HLHS patients undergoing the Glenn procedure. The trial met its primary endpoint, demonstrating safety through one-year post-treatment, with all patients alive, transplant-free, and maintaining expected growth rates. The subsequent five-year follow-up aimed to assess the long-term survival benefits of Lomecel-B.
Key findings from the follow-up study include:
- A 100% Kaplan-Meier survival rate at five years post-Glenn procedure in patients treated with Lomecel-B, with no heart transplants required.
- Comparison to the SVR trial, which showed 83% transplant-free survival and a 5.2% heart transplantation rate over the same period.
- Absence of Major Adverse Cardiovascular Events (MACE) during the study.
- No reported safety issues related to Lomecel-B.
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-B 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, stating, "Even with current comprehensive treatment, only 50% to 60% of infants survive to adolescence due to right ventricular failure and for those that are surviving, parents are strongly seeking to avoid transplant... 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, Longeveron is currently conducting the ELPIS II Phase 2b randomized clinical trial. This trial further evaluates Lomecel-B, compared to standard of care, as a potential adjunct therapy for HLHS. The ELPIS II trial is enrolling 38 pediatric patients across twelve institutions and is supported by grants from the National Heart, Lung, and Blood Institute (NHLBI) through the National Institutes of Health (NIH).
Regulatory Designations and Future Prospects
The U.S. Food and Drug Administration (FDA) has granted Lomecel-B Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation, which facilitate greater access to the FDA during its development for HLHS. Joshua Hare, M.D., Co-founder, Chief Science Officer, and Chairman at Longeveron, stated, "Based on the strength of the ELPIS I data, we are currently conducting the ELPIS II Phase 2b clinical trial evaluating our cellular therapy Lomecel-B as an adjunct therapy for treating HLHS, which, if positive, may serve as the foundation for a BLA submission for potential approval of Lomecel-B."
About HLHS and Lomecel-B
HLHS is a rare congenital heart defect affecting approximately 1,000 infants per year in the U.S. It involves an underdeveloped left ventricle, leading to inadequate blood circulation. Current treatment involves a complex three-stage surgical process, but even with this, survival to adolescence is only 50% to 60%. 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.
