Longeveron (LGVN) Emerging Growth Conference77 summary

Strategic overview and pipeline

• Lead asset Lomecel-B has shown positive results in five clinical trials across HLHS, Alzheimer's, and aging-related frailty.

• HLHS program has received Rare Pediatric Disease, Orphan Drug, and Fast Track designations from the FDA; Alzheimer's program has RMAT and Fast Track designations.

• Lomecel-B is an allogeneic, donor-derived mesenchymal stem cell therapy produced under controlled conditions.

• Cash and cash equivalents of $22.8 million as of Q3 2024, with no debt and funding runway through Q1 2025.

• Management team strengthened with new CMC leadership and proven track record in advancing therapies.

Clinical development and trial progress

• HLHS phase 2b trial is over 80% enrolled, with completion expected by early 2025; BLA submission targeted for early 2026.

• Phase 1 HLHS trial showed 100% five-year survival and no transplants, outperforming historical controls.

• Alzheimer's phase 2a trial met safety and efficacy endpoints, showing slowed cognitive decline and significant biomarker improvements.

• Aging-related frailty phase 2b trial showed significant improvement in the Six-Minute Walk Test after a single infusion.

• Expanded access program in The Bahamas provides additional safety data for regulatory submissions.

Market opportunity and commercialization

• HLHS represents a $1 billion U.S. market, with rare disease pricing and a concentrated prescriber base.

• Alzheimer's disease market opportunity exceeds $10 billion in the U.S., with global prevalence nearing 35 million patients.

• Aging-related frailty affects 8.1 million in the U.S., representing a $4–8 billion market.

• Commercialization for HLHS is streamlined due to a small number of treating physicians and one-time treatment model.

• Partnership and non-dilutive funding are being pursued for large-scale Alzheimer's trials.