Longeveron completes enrollment of Phase 2b trial of laromestrocel in HLHS

Longeveron (LGVN) has achieved full enrollment of the Phase 2b clinical trial evaluating its investigational cellular therapy laromestrocel as a potential adjunct treatment for Hypoplastic Left Heart Syndrome, or HLHS, a rare pediatric and orphan-designated disease. Top-line trial results are anticipated in Q3 2026, after the final follow-up at 12-months. Laromestrocel is a proprietary, scalable, allogeneic cellular therapy being evaluated in multiple indications. The trial’s primary endpoint is a composite of survival at 12 months, length of hospitalization and change in right ventricular ejection fraction measured between baseline and 12 months. ELPIS II is being conducted in collaboration with the National Heart, Lung, and Blood Institute through grants from the National Institutes of Health. ELPIS II builds on the positive clinical results of ELPIS I, in which children in the trial experienced 100% transplant-free survival up to five years after receiving laromestrocel during the Glenn surgery, compared to an approximate 20% mortality rate observed from historical control data. Five-year post-Glenn procedure Kaplan-Meier survival was 100% in patients treated with laromestrocel in ELPIS I with none requiring heart transplant. This compared to 83% survival in the Single Ventricle Reconstruction trial through 5 years post-Glenn surgery, and a 5.2% heart transplantation rate. These findings support the use of laromestrocel as a potential adjunct to HLHS reconstruction surgery to improve transplant-free survival.The FDA has granted laromestrocel Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation for the treatment of HLHS. Currently, the Rare Pediatric Disease PRV program is facing uncertainty regarding its future, as it was not renewed by the U.S. Congress in December 2024.