Longeveron (LGVN) 37th Annual ROTH Conference summary

Company overview and pipeline strategy

• Focuses on allogeneic mesenchymal stem cell therapies with anti-inflammatory and pro-vascular mechanisms, targeting multiple diseases including aging-related frailty, Alzheimer's, and hypoplastic left heart syndrome (HLHS).

• Prioritized HLHS for fastest commercialization, aiming for a BLA filing next year.

• Technology licensed from the University of Miami; public since 2021.

HLHS program and clinical progress

• HLHS is a rare, life-threatening congenital heart disease with about 1,000 US cases annually; current standard is multi-stage surgery with ~50% survival to adolescence.

• ELPIS I phase 1 trial showed 100% five-year survival with no transplants among 10 treated infants, leading to Fast Track, Orphan, and Rare Pediatric Disease designations.

• ELPIS II is a 38-patient, head-to-head phase IIb trial at top US academic centers, with >90% enrollment and completion expected in Q2.

• Endpoints include survival, hospitalization, and a composite/win ratio, following FDA guidance.

• Top-line data expected summer 2026, with BLA rolling submission to follow immediately.

Manufacturing and commercialization plans

• Manufacturing facility in Miami can produce 1,500 doses per lot, enough for US annual demand; 2–3 lots/year suffice globally.

• Evaluating in-house vs. CDMO for commercial-scale fill/finish and packaging.

• Commercialization will leverage relationships with top 10 academic centers, which handle 80% of HLHS cases; expects a lean commercial team of ~15 people.

• Conservative pricing modeled at $500,000–$1 million per treatment, with risk-adjusted NPV over $500 million.