USFDAMay 8, 2026, 04:08 PM
Longeveron: FDA deems ELPIS II RVEF endpoint insufficient for efficacy
AI Summary
Longeveron Inc. announced a Type C meeting with the FDA regarding its ELPIS II clinical trial for hypoplastic left heart syndrome (HLHS). The FDA acknowledged the high unmet medical need but stated that the primary endpoint of right ventricle ejection fraction (RVEF) is not appropriate for demonstrating efficacy, and a new endpoint cannot be agreed upon while the trial is ongoing. Consequently, the FDA no longer considers ELPIS II a pivotal trial. However, the FDA expressed willingness to meet with Longeveron after the trial's completion to discuss results and a potential path forward, and Longeveron plans to submit a new statistical analysis plan. Top-line results for ELPIS II are expected in August 2026.
Key Highlights
- FDA Type C meeting held in late March, summary received late April, regarding ELPIS II trial for HLHS.
- FDA stated right ventricle ejection fraction (RVEF) is not an appropriate primary endpoint to demonstrate efficacy.
- FDA indicated a new primary endpoint could not be agreed upon while the trial is ongoing due to NIH interim analysis.
- FDA no longer refers to the ELPIS II trial as pivotal, as previously discussed in 2024.
- FDA is willing to meet with Longeveron again after ELPIS II completion to discuss results and a path forward.
- Longeveron intends to submit a Sponsor Statistical Analysis Plan (SAP) for ELPIS II with a composite primary endpoint.
- Top-line results from the randomized, controlled Phase 2b ELPIS II clinical trial are anticipated in August 2026.
- ELPIS II has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the FDA.