Longeveron's investigational cell therapy Lomecel-B is showing promising advancement in treating Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart defect. The company's pivotal Phase 2b ELPIS II trial has achieved over 90% enrollment across twelve premier pediatric treatment centers in the United States.
Significant Clinical Progress in HLHS Treatment
The ELPIS II trial, which is evaluating Lomecel-B as an adjunct therapy for HLHS, is expected to complete enrollment of 38 pediatric patients in the second quarter of 2025. The FDA has indicated that if the trial demonstrates sufficient efficacy, it could support a Biologics License Application (BLA) for full traditional approval, anticipated in 2026.
Early results from the Phase 1 ELPIS I study have been particularly encouraging. All ten children treated with Lomecel-B achieved 100% transplant-free survival up to five years post-Glenn surgery, marking a significant improvement over the historical control data showing an 80% transplant-free survival rate at five years.
Regulatory Support and Designations
The FDA has granted Lomecel-B multiple important designations for HLHS treatment:
- Orphan Drug designation
- Fast Track designation
- Rare Pediatric Disease designation
Expanding Therapeutic Applications
Beyond HLHS, Longeveron is advancing Lomecel-B's development in mild Alzheimer's disease, with an FDA meeting anticipated in late Q1 2025 to discuss potential development pathways. The therapy has received both Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation for this indication.
Financial Performance and Outlook
Longeveron reported strong financial results for 2024:
- Total revenue increased 237% to $2.4 million
- Net loss decreased by 25% to $16.0 million
- Cash position of $19.2 million as of December 31, 2024
"Throughout 2024, we continued to advance the development of our investigational cellular therapy candidate, Lomecel-B, as a potential treatment for both HLHS and mild Alzheimer's disease," said Wa'el Hashad, Chief Executive Officer of Longeveron. "Our team's expertise in clinical development and manufacturing, combined with several positive initial results across five clinical trials in three indications, continues to position Longeveron as a leader in stem cell therapy research."
Manufacturing and Development Progress
The World Health Organization's International Nonproprietary Names (INN) Expert Committee has approved "laromestrocel" as the non-proprietary name for Lomecel-B, marking another step forward in the therapy's development. The company is also expanding its manufacturing capabilities, having successfully initiated contract manufacturing services that contributed to the year's revenue growth.
