Longeveron Inc. · NASDAQ:LGVN
Longeveron — Investor Presentation (August 2023)
30 slides from Longeveron's investor presentation, Aug 8, 2023
Slide 1: A Regenerative Medicine Company
Cell-Based Therapies A Regenerative Medicine Company May 2023
Slide 2: Forward Looking Statements
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 esti
Slide 3: Our Mission: Deliver Regenerative Medical Therapies for Unmet Medical Needs
Our Mission: Deliver Regenerative Medical Therapies for Unmet Medical Needs Lomecel-B™ -- A Pipeline in a Product Three Clinical-Stage Programs Allogeneic medicinal signaling cells (MSCs) isolated • Hypoplastic Left Heart Syndrome: from bone marrow of healthy young adults (Open-label Ph. 1 data led
Slide 4: Longeveron Overview
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 Frailty, and
Slide 5: Lomecel-B: Multiple Mechanisms of Action (MOA)
Lomecel-B: Multiple Mechanisms of Action (MOA) Pro-vascular, Pro-regenerative and Anti-inflammatory: Repairs Tissue and Promotes Healing LOMECEL-B Anti-inflammatory Cell-cell Interactions Exosome and Mitochondrial Transfer Cytokines (stem cells) MicroRNA Release (somatic cells) Key Advantages: • Sup
Slide 6: Robust Clinical Pipeline
Robust Clinical Pipeline Indication Geography Phase 1 Phase 2 Phase 3 Hypoplastic Left Phase 2a trial actively U.S. enrolling. Heart Syndrome 2b Single-Dose Trial complete; data Aging-Related U.S. & Japan announced Q3 2021. Frailty Ongoing Phase 2 trial in Japan. Ongoing Phase 2a trial; enrollment A
Slide 7: Lomecel-B for Hypoplastic Left Heart Syndrome
Lomecel-B for Hypoplastic Left Heart Syndrome
Slide 8: Large Unmet Need in Hypoplastic Left Heart Syndrome
Large Unmet Need in Hypoplastic Left Heart Syndrome • HLHS is a rare congenital heart defect in which the left side of the heart fails to normally develop • Children with HLHS require 3 staged open-heart surgeries in order to survive – these surgeries reconfigure the heart so that the right side of
Slide 9: Lomecel-B: Hypoplastic Left Heart Syndrome (HLHS)
Lomecel-B: Hypoplastic Left Heart Syndrome (HLHS) Clinical Approach • Lomecel-B administered directly into heart Glenn: Redirects ‒ Injected into cardiac tissue of right ventricle during Stage II surgery at Blood to PA (St II – 4 mo.) approximately 4 months of age (“Glenn or Hemi-Fontan Procedure”)
Slide 10: Advantages of Lomecel-B 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: Lomecel-B: Hypoplastic Left Heart Syndrome (HLHS)
Lomecel-B: Hypoplastic Left Heart Syndrome (HLHS) Positive ELPIS 1 (Phase 1b) Trial Results (N=10) • Open-label study provided sufficient initial safety data to warrant randomized, controlled trial of Lomecel-B in HLHS (ELPIS II) ‒ No SAEs attributed to treatment • No transplants required in the 10
Slide 12: Long-term Survival in HLHS from ELPIS I Trial of Lomecel-B
Long-term Survival in Hypoplastic Left Heart Syndrome (HLHS) from ELPIS I Trial of Lomecel-B TM Results from ELPIS I trial showed 100% survival (transplant-free) In Lomecel-B treated patients at up to 5 years. In SVR historically matched controls, 5-year mortality was ~20% The NIH/NHLBI Pediatric He
Slide 13: Lomecel-B: Hypoplastic Left Heart Syndrome (HLHS)
Lomecel-B: Hypoplastic Left Heart Syndrome (HLHS) ELPIS II: Ongoing Phase 2 Trial of Lomecel-B 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
Slide 14: Lomecel-B for Aging-Related Frailty
Lomecel-B for Aging-Related Frailty
Slide 15: Aging-Related Frailty: Diminishing Health, Independence and QoL
Aging-Related Frailty*: Diminishing Health, Independence and QoL Frailty Etiology of Aging-Related • Age-associated decline in reserve and function Frailty across multiple physiologic systems leading to inability to cope with stressors • Characterized by mobility disability, weakness, fatigue, weigh
Slide 16: Lomecel-B for Aging-Related Frailty
Lomecel-B for Aging-Related Frailty Phase 2b Study Aging-Related Frailty Study (N=143) • Designed to determine whether there was a dose response to a single infusion of Lomecel-B in Results (graphs on next slide) aging-related frailty • There was a statistically significant • There were 5 treatment
Slide 17: Lomecel-B Improved 6MWT Walking Distance
Lomecel-B Improved 6MWT Walking Distance Statistically-significant dose response at 180 days. * Change from baseline; p<0.05 # Versus placebo; p<0.05 ** Change from baseline; p<0.01 ## Versus placebo; p<0.01 17
Slide 18: Lomecel-B for Aging-Related Frailty
Lomecel-B for Aging-Related Frailty Japan Clinical Trial Strategy Offers Fast Track for Regenerative Medicine Regenerative Medicine (Gene and Cell Therapies) The Act on the Safety of Regenerative Medicine (ASRM) The Act on Pharmaceuticals and Medical Devices (PMDA) • Comprises all medical technologi
Slide 19: Lomecel-B for Aging-Related Frailty
Lomecel-B for Aging-Related Frailty Clinical Trial in Japan (N=45) • Primary Objective ‒ Safety • Japanese National Center for Geriatrics and Gerontology-sponsored trial ‒ NCGG (Nagoya) and Juntendo University (Tokyo) selected as clinical sites Tokyo • Amended protocol accepted by PMDA on August 8,
Slide 20: Lomecel-B for Alzheimer's Disease
Lomecel-B for Alzheimer’s Disease
Slide 21: Lomecel-B for Alzheimer's Disease: Targeting CNS Inflammation
Lomecel-B for Alzheimer’s Disease: Targeting CNS Inflammation • Previous therapies have targeted either amyloid plaques (b-secretase inhibitors and anti-amyloid antibodies) or neurofibrillary tangles (antibodies) with little evidence of improvement of disease state • Increasingly inflammation is rec
Slide 22: Lomecel-B for AD
Lomecel-B for AD Phase 1b Randomized and placebo-controlled trial (N=33) Results (graphs on next slide) • Designed to study safety (primary objective) of • There were no SAEs attributed to treatment single infusion of Lomecel-B in mild AD patients with Lomecel-B and most AEs were related to the proc
Slide 23: Low Dose Lomecel-B: Slower Decline in Cognition (MMSE)
Low Dose Lomecel-B: Slower Decline in cognition (MMSE) in Phase 1b Trial than placebo Worsening Worsening Number of participants Number of participants Placebo 8 8 8 7 Placebo 8 8 8 7 20M Lomecel-B 15 15 15 13 20M Lomecel-B 15 15 15 13 100M Lomecel-B 10 10 10 8 100M Lomecel-B 10 10 10 7 * 20M Lomece
Slide 24: Phase 2a Alzheimer's Disease Study Design Ongoing
Phase 2a Alzheimer’s Disease Study Design Ongoing Treatment: 41 weeks Primary Objective: Safety placebo Exploratory Endpoints: Topline data single dose of Lomecel-B include cognitive measures 48 patients 25 million cells anticipated such as MMSE, Clinical with by end of Dementia Rating Scale with mi
Slide 25: Experienced and Successful Leadership
Experienced and Successful Leadership Wa’el Hashad Joshua M. Hare, MD K. Chris Min, MD, James Clavijo Chief Executive Co-Founder & PhD Chief Financial Officer Chief Science Officer Acting Chief Medical Officer Officer 35+ years of leadership in global Co-founded Longeveron in 2014 25+ years in life
Slide 26: Near-term Milestones
Near-term Milestones 2023 Full data from the Phase 1 ELPIS I trial published in the European Heart Journal Open First patient to enroll in Japan Phase 2 Aging-Related Frailty Trial Long-term survival data disclosed from ELPIS I trial Topline data from Phase 2a Alzheimer’s Disease trial anticipated b
Slide 27: Longeveron Overview
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 Frailty, and
Slide 28: Thank You
Thank You Website www.longeveron.com Chief Financial Officer James Clavijo jclavijo@Longeveron.com Investor Relations: Stern IR, Inc. (212) 362-1200 longeveron@sternir.com Social Media Converge Miami 1951 NW 7th AVENUE @LGVNSocial Suite 520 Miami, FL 33136 @Longeveron 844-470-2550 @Longeveron Inc lo
Slide 29: U.S Market Opportunity: Large Markets with Unmet Medical Needs
U.S Market Opportunity: Large Markets with Unmet Medical Needs Hypoplastic Left Heart Syndrome Aging-Related Frailty Alzheimer’s Disease (HLHS) Patient Population 1,0003 8.1 million1 5.8 million2 Market Potential Up to $1B6 ~$4 – 8B4 ~$5-10B5 1 Company estimate based on US Census Bureau Population >
Slide 30: Phase 2a Alzheimer's Disease Study Design (N=48) Ongoing
Phase 2a Alzheimer’s Disease Study Design (N=48) Ongoing Clinical Screening Infusions (randomized, blinded): Follow-Up Visits Day 0, Week 4, Week 8 & Week 12 Major Eligibility Criteria Day 0, Week 4, 8 & 12: • 60-85 year old male or female Safety N=12 • Clinically diagnosed with mild Group 1 AD acco