Longeveron (LGVN) Investor presentation summary

Pipeline and platform

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

• Development status: HLHS Phase 2b ongoing; Alzheimer’s disease completed through Phase 2a; aging-related frailty completed through Phase 2b.

• Initial positive results reported across 5 clinical trials in 3 indications, with an established safety profile and FDA designations across HLHS and Alzheimer’s disease.

Hypoplastic left heart syndrome

• ELPIS I treated 10 patients; all remained alive and transplant-free up to 5 years after Stage II surgery, versus 83.1% estimated 5-year transplant-free survival in matched SVR controls.

• No laromestrocel-related safety issues or major adverse cardiovascular events were reported in ELPIS I.

• ELPIS II is a randomized, blinded Phase 2b study enrolling 38 patients, comparing standard of care with laromestrocel during Stage II surgery; enrollment was approaching completion.

• Primary endpoints include change in right ventricular ejection fraction, survival at 12 months and length of hospitalization; FDA deemed ELPIS II pivotal and said positive results could support a BLA submission.

• FDA designations: Orphan Drug, Fast Track and Rare Pediatric Disease; U.S. market estimate ~ $1 billion.

Alzheimer’s disease

• CLEAR MIND Phase 2a met its primary safety and secondary efficacy endpoints; selected laromestrocel groups showed statistically significant improvements on prespecified clinical and biomarker endpoints versus placebo.

• Results included statistically significant improvements on MoCA and ADCS-ADL, trends on CDR-SB and MMSE, and 49% less brain volume loss with improved cerebral blood flow on MRI.

• FDA granted RMAT and Fast Track designations; a positive Type B meeting supported a planned single pivotal Phase 2/3 trial, anticipated to start in 2H 2026 subject to non-dilutive funding and/or partnering support.

• Positive Phase 2a results were selected for publication in Nature Medicine in March 2025; U.S. market estimate ~ $5+ billion.