Longeveron Inc. · NASDAQ:LGVN
Longeveron — Investor Presentation (June 2023)
30 slides from Longeveron's investor presentation, Jun 23, 2023
Slide 1: Cell-Based Therapies: A Regenerative Medicine Company
Cell - Based Therapies A Regenerative Medicine Company May 2023
Slide 2: Forward Looking Statements
2 Forward Looking Statements Certain statements in this press release that are not historical facts are forward - looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 , which reflect management's current expectations, assumptions, and
Slide 3: Our Mission: Deliver Regenerative Medical Therapies for Unmet Medical Needs
3 Our Mission: Deliver Regenerative Medical Therapies for Unmet Medical Needs Lomecel - B Ρ -- A Pipeline in a Product Allogeneic medicinal signaling cells (MSCs) isolated from bone marrow of healthy young adults Three Clinical - Stage Programs • Hypoplastic Left Heart Syndrome: (Open - label Ph. 1
Slide 4: Longeveron Overview
4 Longeveron Overview Leader in development of MSCs for therapeutic indications Well - characterized allogeneic MSC (Lomecel - B) product in Phase 2 trials Robust clinical pipeline with clinical efficacy signals in multiple indications with large unmet need: Alzheimer’s Disease, Aging - Related Frai
Slide 5: Multiple Mechanisms of Action (MOA)
5 Lomecel - B: Multiple Mechanisms of Action (MOA) Pro - vascular, Pro - regenerative and Anti - inflammatory: Repairs Tissue and Promotes Healing Jimenez - Puerta GJ, et al. Journal of Clinical Medicine . 2020 Feb;9(2):445. Mazhari R and Hare JM. Nature Clinical Practice Cardiovascular Medicine . 2
Slide 6: Robust Clinical Pipeline
6 Robust Clinical Pipeline Phase 3 Phase 2 Phase 1 Geography Indication Phase 2a trial actively enrolling. U.S. Hypoplastic Left Heart Syndrome 2b Single - Dose Trial complete; data announced Q3 2021. Ongoing Phase 2 trial in Japan. U.S. & Japan Aging - Related Frailty Ongoing Phase 2a trial; enroll
Slide 7: for Hypoplastic Left Heart Syndrome
Lomecel - B for Hypoplastic Left Heart Syndrome
Slide 8: Large Unmet Need in Hypoplastic Left Heart Syndrome
8 Large Unmet Need in Hypoplastic Left Heart Syndrome HLHS Normal Heart Source: Abu - Harb . Arch Dis Child Fetal Neonatal Ed. 1994; Best. Front Ped. 2021; Liu. J Am Heart Assoc. 2018; Mahle . Pediatrics. 2001; Newburger . Circulation. 2018; Siffel . Pediatrics. 2015; Stasik . J Thorac Cardiovasc Su
Slide 9: Clinical Approach: Hypoplastic Left Heart Syndrome (HLHS)
9 Clinical Approach Lomecel - B: Hypoplastic Left Heart Syndrome (HLHS) ClearView Healthcare Partners Analysis. • Lomecel - B administered directly into heart ‒ Injected into cardiac tissue of right ventricle during Stage II surgery at approximately 4 months of age (“Glenn or Hemi - Fontan Procedure
Slide 10: Advantages in HLHS
Advantages of Lomecel - B in HLHS • Findings in studies of adults using MSCs: ‒ Boosts ejection fraction (EF) in patients with dilated cardiomyopathy (DCM) • EF increased by 8.0 percentage points (95% CI: 2.8 to 13.2 percentage points; p = 0.004) ‒ Reduces ventricular chamber size in adults with DCM
Slide 11: Positive ELPIS (Phase 1b) Trial Results (N=10)
11 Positive ELPIS 1 (Phase 1b) Trial Results (N=10) Lomecel - B: Hypoplastic Left Heart Syndrome (HLHS) Published historical data (blue and red curves) adapted from Figure 2 in Son JS, et al. Circulation: Cardiovascular Imaging. 201 8;11(7):e006983. ELPIS 1 transplant - free survival data depicted i
Slide 12: Long-term Survival in HLHS from ELPIS I Trial
Slide 12 Long - term Survival in Hypoplastic Left Heart Syndrome (HLHS) from ELPIS I Trial of Lomecel - B TM The NIH/NHLBI Pediatric Heart Network Single Ventricle Reconstruction Trial dataset was used in preparation of this work. Dat a w ere downloaded from https://www.pediatricheartnetwork.com/pud
Slide 13: ELPIS II: Ongoing Phase 2 Trial in HLHS
• Total population = 38 patients • Randomized, controlled study comparing best medical care to the same treatment with addition of Lomecel - B (5 - minute additional procedure during surgery) • Primary endpoint: Change in Right Ventricular Ejection Fraction (RVEF) at 12 months Eligibility Criteria A
Slide 14: for Aging-Related Frailty
Lomecel - B for Aging - Related Frailty
Slide 15: Aging-Related Frailty: Diminishing Health, Independence and QoL
15 Frailty • Age - associated decline in reserve and function across multiple physiologic systems leading to inability to cope with stressors • Characterized by mobility disability, weakness, fatigue, weight loss, slowness, low activity, etc. Higher risk for poor clinical outcomes • Infections, fall
Slide 16: Phase 2b Study in Aging-Related Frailty (N=143)
16 Phase 2b Study Aging - Related Frailty Study (N=143) Lomecel - B for Aging - Related Frailty Results (graphs on next slide) • There was a statistically significant increase in 6MWT in multiple Lomecel - B treatment groups 9 months after a single infusion of Lomecel - B compared to placebo • There
Slide 17: Improved 6MWT Walking Distance
17 Lomecel - B Improved 6MWT Walking Distance * Change from baseline; p<0.05 ** Change from baseline; p<0.01 # Versus placebo; p<0.05 ## Versus placebo; p<0.01 Statistically - significant dose response at 180 days.
Slide 18: Japan Clinical Trial Strategy Offers Fast Track for Regenerative Medicine
The Act on the Safety of Regenerative Medicine (ASRM) • Comprises all medical technologies for which the safety and efficacy have not yet been established • Only approved for medical care and clinical research use and not intended for marketing • Treatment at one’s own expense with no insurance cove
Slide 19: Clinical Trial in Japan (N=45) for Aging-Related Frailty
19 • Primary Objective ‒ Safety • Japanese National Center for Geriatrics and Gerontology - sponsored trial ‒ NCGG (Nagoya) and Juntendo University (Tokyo) selected as clinical sites • Amended protocol accepted by PMDA on August 8, 2022 • Ongoing Phase 2 trial Clinical Trial in Japan (N=45) Lomecel
Slide 20: for Alzheimer's Disease
Lomecel - B for Alzheimer’s Disease
Slide 21: Alzheimer's Disease: Targeting CNS Inflammation
21 Lomecel - B for Alzheimer’s Disease: Targeting CNS Inflammation Figure from Congdon EE, Sigurdsson EM. Tau - targeting therapies for Alzheimer disease. Nat Rev Neurol. 2018 Jul;14(7):399 - 415. *Shi Y, Holtzman DM (December 2018). Nature Reviews. Immunology . 18 (12): 759 – 772 ᐩ Heppner FL; Rans
Slide 22: Phase 1b Trial in Alzheimer's Disease (N=33)
22 Lomecel - B for AD • Designed to study safety (primary objective) of single infusion of Lomecel - B in mild AD patients ‒ Exploratory endpoints included cognitive measures such as Mini - Mental State Exam (MMSE) • There were 3 treatment groups: placebo and 2 different doses of Lomecel - B: 20 and
Slide 23: Low Dose: Slower Decline in Cognition (MMSE) in Phase 1b Trial
23 Low Dose Lomecel - B: Slower Decline in cognition (MMSE) in Phase 1b Trial than placebo MMSE: Mini Mental State Exam; 20M: 20 million. † Placebo change from baseline, p<0.05 * 20M Lomecel - B compared to placebo, p<0.05 Number of participants 7 8 8 8 Placebo 13 15 15 15 20M Lomecel - B 8 10 10 10
Slide 24: Phase 2a Alzheimer's Disease Study Design
48 patients with mild AD 24 Designed to explore the potential efficacy of multiple doses of Lomecel - B in mild AD Phase 2a Alzheimer’s Disease Study Design Ongoing placebo single dose of Lomecel - B 25 million cells 4 doses of Lomecel - B 25 million cells 4 doses of Lomecel - B 100 million cells Tr
Slide 25: Experienced and Successful Leadership
25 Experienced and Successful Leadership Our new Chief Medical Officer will be announced in late July. Chief Medical Officer Co - founded Longeveron in 2014 and is the founding director of the Interdisciplinary Stem Cell Institute at the University of Miami’s Miller School of Medicine. Joshua M. Har
Slide 26: Near-term Milestones
26 Near - term Milestones First patient to enroll in Japan Phase 2 Aging - Related Frailty Trial 2023 Full data from the Phase 1 ELPIS I trial published in the European Heart Journal Open Topline data from Phase 2a Alzheimer’s Disease trial anticipated by end of 2023 Long - term survival data disclo
Slide 27: Longeveron Overview
27 Longeveron Overview Leader in development of MSCs for therapeutic indications Well - characterized allogeneic MSC (Lomecel - B) product in Phase 2 trials Robust clinical pipeline with clinical efficacy signals in multiple indications with large unmet need: Alzheimer’s Disease, Aging - Related Fra
Slide 28: Thank You
Converge Miami 1951 NW 7th AVENUE Suite 520 Miami, FL 33136 844 - 470 - 2550 longeveron.com Website www.longeveron.com Chief Financial Officer James Clavijo jclavijo@Longeveron.com Investor Relations: Stern IR, Inc. (212) 362 - 1200 longeveron@sternir.com Thank You Social Media @LGVNSocial @Longever
Slide 29: U.S. Market Opportunity: Large Markets with Unmet Medical Needs
29 U.S Market Opportunity: Large Markets with Unmet Medical Needs Alzheimer’s Disease Aging - Related Frailty Hypoplastic Left Heart Syndrome (HLHS) 5.8 million 2 8.1 million 1 1,000 3 Patient Population ~$5 - 10B 5 ~$4 – 8B 4 Up to $1B 6 Market Potential 1 Company estimate based on US Census Bureau
Slide 30: Phase 2a Alzheimer's Disease Study Design (N=48)
30 Phase 2a Alzheimer’s Disease Study Design (N=48) Ongoing Group 1 Infusion 1 - 4: Placebo Group 2 Infusion 1: 25M Lomecel - B Infusion 2 - 4: Placebo Group 3 Infusion 1 - 4: 25M Lomecel - B Infusions (randomized, blinded): Day 0, Week 4, Week 8 & Week 12 Clinical Screening Major Eligibility Criter