Longeveron Achieves Full Enrollment in Phase 2b HLHS Trial, Top-line Results Anticipated in Q3 2026.

Longeveron Inc. has completed enrollment in its Phase 2b trial (ELPIS II) evaluating laromestrocel as a treatment for Hypoplastic Left Heart Syndrome (HLHS). Top-line results are expected in Q3 2026, with a BLA submission for full approval anticipated in 2026 if results are positive. The FDA has awarded laromestrocel HLHS program Rare Pediatric Disease, Orphan drug, and Fast track designations. The program addresses unmet medical needs with a U.S. market potential of up to $1 billion.

Longeveron Inc. (NASDAQ: LGVN) has announced the completion of enrollment for its pivotal Phase 2b clinical trial (ELPIS II) evaluating laromestrocel as a treatment for Hypoplastic Left Heart Syndrome (HLHS). The trial, which enrolled 40 pediatric patients across 12 premier institutions, marks a significant milestone in the development of a potential new therapy for this rare and devastating congenital heart defect [1].

The ELPIS II trial builds on the positive results of the ELPIS I trial, which demonstrated 100% transplant-free survival up to five years post-treatment, compared to historical control data showing 20% mortality. The composite primary endpoint of the ELPIS II trial captures both survival and functional cardiac improvements through right ventricular ejection fraction measurements, aligning with regulatory expectations and clinical relevance for HLHS patients [1].

The FDA has granted the laromestrocel HLHS program Rare Pediatric Disease, Orphan Drug, and Fast Track designations, indicating strong regulatory support. These designations create multiple acceleration pathways and potential exclusivity benefits if the therapy is approved. The U.S. market potential for a successful treatment is estimated at up to $1 billion, reflecting the dire need for new treatment options for this rare condition [1].

Top-line trial results are expected in Q3 2026, with a potential Biological License Application (BLA) submission for full approval anticipated in 2026 if the results are positive. The completion of enrollment strengthens Longeveron's commercialization timeline, with potential revenue and valuable Priority Review Voucher (PRV) opportunities on the horizon [1].

The enrollment completion also highlights the unmet need for new treatment options in HLHS, where current surgical approaches have a high mortality rate, particularly due to right ventricular failure. The slight over-enrollment of the trial (40 vs. 38 planned patients) reflects both investigator enthusiasm and the dire need for new treatment options [1].

Longeveron's announcement represents a critical value inflection point in their HLHS program development timeline. The company has significantly de-risked one major development hurdle and established a clear regulatory pathway with the FDA's confirmation that positive ELPIS II results could support a BLA submission [1].

References:
[1] https://www.stocktitan.net/news/LGVN/longeveron-announces-completion-of-enrollment-of-pivotal-phase-2b-yjhdhj2oqbad.html
[2] https://www.globenewswire.com/news-release/2025/06/24/3104065/0/en/Longeveron-Announces-Completion-of-Enrollment-of-Pivotal-Phase-2b-Clinical-Trial-Evaluating-Laromestrocel-as-a-Treatment-for-Hypoplastic-Left-Heart-Syndrome-HLHS.html