Longeveron (LGVN) Investor presentation summary

Platform and pipeline

• Lomecel-B is an allogeneic cell therapy derived from bone marrow of healthy young adults, culture-expanded and frozen in patient doses.

• Development spans HLHS, Alzheimer's disease and aging-related frailty; Phase 2b is ongoing in HLHS, while studies through Phase 2a in Alzheimer's and Phase 2b in frailty are complete.

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

HLHS program

• HLHS affects ~1,000 U.S. babies annually and requires three staged surgeries; estimated survival to adolescence is 50%–60%, with ~80% five-year transplant-free survival.

• In ELPIS I, all 10 treated patients were alive and transplant-free through five years; five-year transplant-free survival was 100% versus 80% in propensity-matched historical controls, with no reported treatment-related safety issues or MACE.

• Phase 2b ELPIS II is ongoing, with 38 participants randomized to standard of care or Lomecel-B during Stage II surgery; primary endpoints include change in right-ventricle ejection fraction, survival and hospitalization length.

• FDA deemed ELPIS II pivotal and, if positive, acceptable for a potential BLA submission; the parties aligned on endpoints, CMC and potency-assay plans.

Alzheimer's disease program

• The program targets CNS inflammation; FDA granted Lomecel-B RMAT and Fast Track designations.

• CLEAR MIND Phase 2a tested placebo and single- or multi-dose regimens in four groups, with safety as the primary endpoint and imaging and neurocognitive measures among secondary endpoints.

• Results showed a positive benefit-risk profile, met primary safety and secondary efficacy endpoints, and indicated slowing or prevention of disease worsening versus placebo; no hypersensitivity, infusion-related reactions or ARIA were reported.

• MRI results showed 49% less brain volume loss and improved cerebral blood flow; cognitive and functional measures also showed statistically significant or trending improvements versus placebo.

• A meeting with FDA was anticipated for Q1 2025 to review future clinical and regulatory strategy; partnerships and non-dilutive funding were being pursued for further development.