Longeveron (LGVN) Investor presentation summary

Clinical platform and pipeline

• Laromestrocel (Lomecel-B) is an allogeneic mesenchymal stem cell therapy made from bone marrow cells of healthy young adult donors; programs span HLHS, Alzheimer’s disease and aging-related frailty.

• The HLHS program is in an ongoing Phase 2b trial; Alzheimer’s disease development has completed Phase 2a, and the U.S. aging-related frailty Phase 2b trial is complete.

• The presentation cites positive initial results across five clinical trials and large U.S. market opportunities across the three indications.

HLHS program

• ELPIS I enrolled 10 patients; all remained alive and transplant-free through five years after Stage II surgery, versus 80% five-year transplant-free survival in a propensity-matched historical control group.

• No laromestrocel-related safety issues or major adverse cardiovascular events were reported in ELPIS I; the trial met its primary safety endpoint through one year.

• ELPIS II is a randomized, blinded Phase 2b study targeting 38 patients, comparing standard care with laromestrocel injected during Stage II surgery; it is actively enrolling.

• Following an August 2024 FDA meeting, ELPIS II was deemed pivotal; if positive, it may support a BLA submission for potential traditional approval, with FDA alignment on endpoints and CMC requirements.

• FDA designations for HLHS include Rare Pediatric Disease, Orphan Drug and Fast Track.

Alzheimer’s disease program

• CLEAR MIND Phase 2a met its primary safety and secondary efficacy endpoints, with statistically significant improvements in prespecified clinical and biomarker endpoints in specific treatment groups versus placebo.

• Results indicated slowing or prevention of worsening versus placebo; measures included statistically significant MoCA results and ADCS-ADL improvement, with trending improvements in CDR-SB and MMSE.

• Brain MRI results showed a 49% reduction in brain volume loss and improved cerebral blood flow; no hypersensitivity, infusion-related reactions or ARIA cases were reported.

• FDA granted laromestrocel RMAT and Fast Track designations for Alzheimer’s disease; Phase 2a results were selected for publication in the March 2025 issue of Nature Medicine.

• Further development plans included an anticipated FDA meeting in Q1 2025 and seeking partnerships and non-dilutive funding.