Longeveron(US:LGVN) Globenewswire·2025-06-24 10:30Core Insights - Longeveron Inc. has completed full enrollment of the Phase 2b clinical trial (ELPIS II) for laromestrocel, targeting Hypoplastic Left Heart Syndrome (HLHS) [1][2] - The trial aims to evaluate laromestrocel as a potential adjunct treatment for HLHS, with top-line results expected in Q3 2026 [1][5] - Laromestrocel has received multiple FDA designations, including Orphan Drug, Fast Track, and Rare Pediatric Disease designations, indicating its potential significance in addressing unmet medical needs [4][5] Company Overview - Longeveron is a clinical stage biotechnology company focused on developing regenerative medicines, with laromestrocel as its lead investigational product [9] - The company is pursuing treatments for HLHS, Alzheimer's disease, and aging-related frailty, with laromestrocel being an allogeneic mesenchymal stem cell therapy [9] Clinical Trial Details - ELPIS II enrolled 40 pediatric patients across twelve leading treatment institutions, with primary endpoints including survival at 12 months and changes in right ventricular ejection fraction [2][3] - The trial builds on the success of ELPIS I, which reported 100% transplant-free survival at five years for patients treated with laromestrocel [3][6] Market Potential - The U.S. market potential for laromestrocel in treating HLHS is estimated to be up to $1 billion, highlighting the significant unmet medical need in this area [5] - If successful, the company could be eligible for a Priority Review Voucher (PRV), which can expedite the review process for future drug applications [7] Regulatory Status - The FDA has confirmed that ELPIS II is a pivotal trial, and positive results could lead to a Biological License Application (BLA) submission for full approval [4][5] - The Rare Pediatric Disease PRV program is currently facing uncertainty, but recent legislative efforts may extend its availability [7]