Longeveron DMC Backs Completing Phase 2b HLHS Trial; New CEO Appointed

Longeveron (NASDAQ: LGVN) announced that an independent Data Monitoring Committee (DMC) completed its final planned review of the fully enrolled Phase 2b ELPIS II trial of laromestrocel (Lomecel-B) for hypoplastic left heart syndrome (HLHS) and recommended the study continue as designed to completion. The DMC found no new safety concerns, and top-line results are anticipated in August 2026.

ELPIS II enrolled 40 pediatric patients at 12 pediatric cardiac centers and is being conducted with NHLBI support. The trial is designed to compare laromestrocel plus standard surgery versus standard surgery alone, with endpoints including survival at 12 months, length of hospitalization, and change in right ventricular ejection fraction between baseline and 12 months. Full enrollment was achieved in June 2025.

Prior Phase 1 data from the ELPIS I study showed 100% transplant-free survival to age five in treated children, compared to historical controls of approximately 83% at five years (with about 5% requiring transplant) in the comparable population. In that study, laromestrocel was well tolerated with no major adverse cardiovascular events or infections related to therapy through one year, meeting the primary safety endpoint.

HLHS is a rare and life-threatening congenital heart defect in which the left ventricle is severely underdeveloped or absent. Even with the standard of care — typically a series of three reconstructive surgeries over the first few years of life — survival into adolescence is only around 50–60%. Longeveron's approach administers laromestrocel directly into the right ventricle during the second stage of surgery.

Laromestrocel for HLHS holds FDA Orphan Drug, Fast Track, and Rare Pediatric Disease designations. Following a Type C meeting with the FDA in September 2024, the agency confirmed that the ELPIS II trial could serve as a pivotal study — and if positive, could form the basis for a Biologics License Application (BLA) submission for full traditional approval.

Separately, the company announced the appointment of Stephen Willard as permanent CEO, effective immediately. The CEO is deferring 50% of his $500,000 salary to support the financial needs of the company. This is part of a larger range of cost containment measures that includes furloughing some employees, travel limitations, and a reduction in directors' fees. These measures are estimated to reduce administrative expenses by approximately 25% in the coming quarters, extending the capital runway as investors await the anticipated top-line results from the Phase 2b HLHS trial in the third quarter of 2026.