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.

• Lead product laromestrocel (Lomecel-B) is an allogeneic mesenchymal stem cell therapy with multiple mechanisms of action, including anti-inflammatory, pro-vascular, and regenerative effects.

• Clinical pipeline includes Hypoplastic Left Heart Syndrome (HLHS), Pediatric Dilated Cardiomyopathy (PDCM), Alzheimer's Disease (AD), and Aging-related Frailty (AF), with positive initial results in five clinical trials across three indications.

• HLHS and AD programs have received multiple FDA designations, including Orphan Drug, Fast Track, Rare Pediatric Disease, and RMAT.

• Large U.S. market opportunities estimated at $1B for HLHS, $1B+ for PDCM, $5B+ for AD, and $4B+ for AF.

Clinical trial highlights and results

• ELPIS I trial in HLHS showed 100% transplant-free survival at five years post-surgery, with no major adverse cardiovascular events.

• ELPIS II pivotal Phase 2b trial for HLHS completed enrollment in June 2025, with top-line data expected in Q3 2026.

• CLEAR MIND Phase 2a trial in Alzheimer's demonstrated a positive benefit/risk profile, slowing cognitive and functional decline, and significant improvements in clinical and biomarker endpoints.

• Phase 2b study in Aging-related Frailty showed statistically significant improvements in physical endurance (6MWT) and a dose-response relationship, with no serious adverse events attributed to treatment.

• IND approved for PDCM in July 2025, allowing direct progression to a pivotal Phase 2b/3 trial.

Regulatory and development milestones

• Positive FDA Type C meeting for HLHS and ongoing pivotal trial; positive FDA Type B meeting for AD, with a planned pivotal Phase 2/3 trial.

• Five FDA designations across HLHS and AD programs, supporting accelerated development and regulatory pathways.

• Planned initiation of pivotal Phase 2/3 trial in AD in 2H 2026, contingent on funding or partnerships.

• ELPIS II data read-out and pre-BLA meeting with FDA are key priorities for the next 12 months.

• Expansion of partnership efforts and initiation of PDCM pivotal trial dependent on financing.