Longeveron FDA Meeting: ELPIS II Primary Endpoint Not Pivotal
Longeveron Inc. announced a Type C meeting with the U.S. FDA regarding its ELPIS II Phase 2b clinical trial for laromestrocel (LOMECEL-B®) in hypoplastic left heart syndrome (HLHS). The FDA stated that the trial's primary endpoint (RVEF) is not appropriate for demonstrating efficacy and, due to an ongoing interim analysis, a new primary endpoint could not be agreed upon while the trial is active. Consequently, the FDA no longer considers ELPIS II a pivotal trial, reversing its previous position from 2024. Despite this, the FDA expressed willingness to meet again after the trial's completion to discuss results and a potential path forward, suggesting objective measures like mortality and major adverse cardiac events (MACE) could be informative. Longeveron plans to submit a new Statistical Analysis Plan (SAP) with a composite primary endpoint and remains optimistic that trial results will support a Biologics License Application (BLA) filing following the anticipated top-line data readout in August 2026.
Key Highlights
- FDA Type C meeting held in late March, with summary provided in late April, regarding ELPIS II trial.
- FDA asserted that the primary endpoint of right ventricle ejection fraction (RVEF) in ELPIS II is not appropriate for efficacy.
- FDA no longer refers to the ELPIS II trial as pivotal, reversing its 2024 stance.
- Top-line results from the randomized, controlled Phase 2b ELPIS II clinical trial are anticipated in August 2026.
- FDA is willing to meet with Longeveron again after ELPIS II study completion to discuss results and path forward.
- Longeveron intends to submit a Sponsor Statistical Analysis Plan (SAP) for ELPIS II with a composite primary endpoint.
- Laromestrocel HLHS program holds Orphan Drug, Fast Track, and Rare Pediatric Disease designations.
Price Impact
More from LGVN