Longeveron (LGVN) Investor presentation summary

Strategic focus and clinical pipeline

• Developing stem cell therapies for life-threatening conditions in children and the elderly, targeting rare pediatric and chronic aging-related diseases.

• Clinical pipeline includes Hypoplastic Left Heart Syndrome (HLHS), Pediatric Dilated Cardiomyopathy (PDCM), Alzheimer's disease (AD), and Aging-related Frailty (AF).

• Positive initial results in five clinical trials across three indications, with a well-established safety profile.

• Multiple FDA designations: HLHS (Orphan Drug, Fast Track, Rare Pediatric Disease), AD (RMAT, Fast Track).

• Large U.S. market opportunities: HLHS ($1B), PDCM ($1B+), AD ($5B+), AF ($4B+).

Laromestrocel (Lomecel-B) product and mechanism

• Allogeneic mesenchymal stem cell therapy derived from healthy young adult bone marrow, culture-expanded and cryopreserved for off-the-shelf use.

• Demonstrates pro-vascular, pro-regenerative, and anti-inflammatory mechanisms, supporting tissue repair and healing.

• Shown to be immuno-evasive, with enhanced safety and convenient administration.

• Mechanisms support use in diseases with endothelial dysfunction, inflammation, fibrosis, and need for endogenous repair.

Clinical highlights by indication

• HLHS: Ongoing pivotal Phase 2b trial (ELPIS II) fully enrolled; ELPIS I showed 100% transplant-free survival at 5 years post-surgery, compared to 80% in historical controls.

• PDCM: IND approved July 2025; FDA supports direct progression to pivotal Phase 2b/3 trial.

• AD: Completed Phase 2a (CLEAR MIND) with positive safety and efficacy; RMAT and Fast Track designations; pivotal Phase 2/3 trial planned for 2H 2026.

• AF: Completed Phase 2b showing significant improvement in physical endurance (6MWT) and dose-response; no serious adverse events attributed to treatment.