Longeveron Inc. · NASDAQ:LGVN
Longeveron — H.C. Wainwright BioConnect 2022 Virtual Conference
30 slides from Longeveron's conference presentation, Jan 10, 2022
Slide 1: H.C. Wainwright BioConnect 2022 Virtual Conference
Nasdaq: LGVN www.longeveron.com H.C. Wainwright BioConnect 2022 Virtual Conference Cell Therapy for Aging - Related Disease & Life - Threatening Conditions
Slide 2: Forward Looking Statements
Forward Looking Statements 2 Certain statements in this presentation that are not historical facts are forward - looking statements that reflect management's current expectations, assumptions, and estimates of future performance and economic conditions, and involve risks and uncertainties that could
Slide 3: Longeveron At-a-Glance
Longe v e r o n A t - a - G l an c e (N AS D A Q: L G V N) 3 Biotechnology Regenerative Medicine / Cell Therapy 2014 February 12, 2021 $19.0 million (September 30, 2021) $20.5 million private placement (gross proceeds; December 2021) Industry S ec tor Found e d IPO Last Reported Cash Recent Financin
Slide 4: Cell Therapy for Aging-Related Disease & Life-Threatening Conditions
Cell Therapy for Aging - Related Disease & Life - Threatening Conditions 4 • Lomecel - B Œ : medicinal signaling cells (MSCs) from young, healthy donors (bone marrow) • Manufactured in Longeveron’s cGMP facility • Ongoing Phase 2 trials in Hypoplastic Left Heart Syndrome (HLHS) and Alzheimer’s disea
Slide 5: Experienced and Successful Leadership
Experienced and Successful Leadership Geoff Green, MBA Chief Executive Officer 25+ years in life sciences, with leadership roles in public and private companies, and experience in corporate strategy, operations, business development, and clinical affairs. Previously President and Acting CEO of DOR B
Slide 6: Robust Clinical Research Pipeline
Robust Lomecel - B Clinical Research Pipeline *Sub study of two US Aging Frailty trials; ARDS=Acute Respiratory Distress Syndrome; Central Nervous System ^National Institute on Aging Grant for Phase 2b Aging Frailty and Metabolic Substudy; MSCRF grants for Phase 1/2 Aging Frailty “HERA”, Phase 1 HLH
Slide 7: U.S Market Opportunity: Large Markets with Unmet Medical Needs
U.S Market Opportunity: Large Markets with Unmet Medical Needs 7 Aging Frailty A l z heime r ’ s Disease The Metabolic Syndrome Acute Respiratory Distress Syndrome (ARDS) Hypoplastic Left Heart Syndrome (HLHS) 8.1 million 1 5.8 million 2 87.2 million 3 150,000 4 1,000 5 1 Company estimate based on U
Slide 8: Medicinal Signaling Cells (MSC) Therapeutics
Lomecel - B Medicinal Signaling Cells (MSC) Therapeutics 8
Slide 9: Multimodal Mechanisms of Action (MOA)
Lomecel - B: Multimodal Mechanisms of Action (MOA) Pro - vascular, Pro - regenerative and Anti - inflammatory: Repairs Tissue & Promotes Healing 9 1 A n ti - i n fl a mm a t o ry cytokines 2 3 Exosome and m i c r o RN A r e l ease 4 Mitochondrial transfer (somatic cells) Cell - cell interactions (st
Slide 10: Production and Administration
Lomecel - B Production and Administration 10 H ea lt h y A du lt Donors 18 - 45 Y ea r s ; R i go r ous ly Screened MSCs Isolated from Bone M a rr o w A sp ir a te MSCs Culture - Expanded into Billions of Cells 40 Minute IV Infusion
Slide 11: Proprietary, Scalable, Off-the-Shelf Cellular Therapy
Lomecel - B: Proprietary, Scalable, “Off - the - Shelf” Cellular Therapy 11 • Robust quality assurance processes ensure batch - to - batch consistency • Cryogenically stored long - term, easy to prepare and administer • Delivered systemically or locally • In response to inflammatory mediators, MSCs
Slide 12: Clinical Research Hypoplastic Left Heart Syndrome
Lomecel - B Clinical Research Hypoplastic Left Heart Syndrome 12
Slide 13: Hypoplastic Left Heart Syndrome (HLHS)
Lomecel - B for Hypoplastic Left Heart Syndrome (HLHS) Normal Heart Left Ventricular Defect • Congenital heart defect (~1,000 babies/year in US) 1 • Characterized by underdeveloped or absent left ventricle • Three staged operations currently standard of care • ~30% mortality rate at 3 years 2 • Lome
Slide 14: HLHS: Phase 1 Trial (ELPIS I) Complete Results
Phase 1 Trial (ELPIS I) HLHS: Phase 1 Complete Results * Safety • No major adverse cardiac events • No infections considered related to Lomecel - B Secondary/exploratory endpoints • No statistically significant deterioration in cardiac structure or function at 6 or 12 months compared to baseline • G
Slide 15: HLHS: ELPIS II Trial: Actively enrolling
HLHS: ELPIS II Trial: Actively enrolling • First infant treated July 7 2021 during Stage II Glenn surgery • Will be enrolling at up to seven major pediatric cardiac centers • Primary endpoint: Change in Right Ventricular Ejection Fraction (RVEF) at 12 months • US FDA granted both Rare Pediatric Dise
Slide 16: Clinical Research Alzheimer's Disease
Lomecel - B Clinical Research Alzheimer’s Disease 16
Slide 17: Alzheimer's Disease: Targeting Neuroinflammation
Alzheimer’s Disease: Targeting Neuroinflammation 17 • Chronic neuroinflammation a hallmark of dysregulated microglia, cause of nerve cell death • Systemic inflammation likely contributes to disease progression • MSCs in animal models of Alzheimer’s: – Cross the blood brain barrier (BBB) – ↓ pro - in
Slide 18: Safety: Primary Endpoint Met
Safety: Primary Endpoint Met Single infusion of two different doses well tolerated: No Amyloid Related Imaging Abnormalities (ARIA) identified Secondary/Exploratory Endpoints Lomecel - B group’s MMSE score declined more slowly compared to placebo (Low dose vs placebo: stat sig at 3 & 9 months) Lomec
Slide 19: Phase 2a Alzheimer's Disease Study Design
19 © Longeveron. CONFIDENTIAL Phase 2a Alzheimer’s Disease Study Design 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 Eligib
Slide 20: Robust Clinical Program Aging Frailty
Lomecel - B Robust Clinical Program Aging Frailty 20
Slide 21: Aging Frailty: Diminishing Health, Independence and QoL
Aging Frailty*: Diminishing Health, Independence and QoL 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 . Hi
Slide 22: Can We Prevent or Reverse Frailty Progression?
Can We Prevent or Reverse Frailty Progression? Inflammation • Lifelong wear & tear • Subclinical disease • Chronic sterile inflammation • Deterioration of mobility, strength and exercise tolerance; • Weight loss; muscle loss; • Susceptibility to Infections; • Loss of i ndependen c e • Increased risk
Slide 23: Phase 2b Study Aging Frailty Study Design
Phase 2b Study Aging Frailty Study Design Group 1 Placebo Group 2 25M Lomecel - B Group 3 50M Lomecel - B Single Infusion (randomized, blinded) Clinical Screen Major Eligibility Criteria • 70 - 85 year old male or female • Clinical Frailty Scale score 5 – 6 (mild to moderate) • Can walk 200 to 400 m
Slide 24: Improved Walking Distance Over Months
Lomecel - B Improved Walking Distance Over 9 Months 24 * 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 25: HERA Trial: Immune Response to Influenza Vaccine in Aging Frailty
“HERA” Trial: Immune Response to Influenza Vaccine in Aging Frailty* 25 *As noted previously, Frailty/Aging Frailty” is currently not a recognized indication by US FDA or Japan PMDA HAI=Hemagglutination Inhibition O b j ec ti v es • Safety • Can Lomecel - B improve adaptive immunity against influenz
Slide 26: Aging Frailty Clinical Trial in Japan
Aging Frailty* Clinical Trial in Japan 26 Objectives • Safety • Can Lomecel - B improve exercise tolerance and mobility? Efficacy Endpoints • Change in 6MWT; grip strength, walking speed, patient - reported outcomes biomarkers, fear and risk of falling, depression, sexual function, clinical outcomes
Slide 27: Intellectual Property & Barriers to Competition
Intellectual Property & Barriers to Competition Proprietary Manufacturing Process • Exclusively licensed from University of Miami • Improved and optimized by Longeveron • In - house manufacturing to maintain proprietary trade secrets Company - Owned Patent Applications • Allogeneic MSCs for use as a
Slide 28: What are we focused on in 2022?
What are we focused on in 2022? 28 Alzheimer’s Disease • Rapidly recruit & enroll our Phase 2 a Trial • Publish Phase 1 trial results Aging Frailty (JP) • Initiate Japan Phase 2 Trial • Partnership for commercial rights in Japan Aging Frailty (US) • Publish Phase 2b trial results • Review new data:
Slide 29: Summary
S umma ry Leader in regenerative medicine for aging - related and life - threatening diseases and conditions Well - characterized allogeneic MSC product in development Robust clinical pipeline with advanced trials in Alzheimer’s disease, aging frailty, HLHS and other indications State - of - the - a
Slide 30: Thank You
Thank You 30 Website : www.longeveron.com Chief Financial Officer : James Clavijo jclavijo@Longeveron.com Investor Relations : Brendan Payne (212) 362 - 1200 Brendan.payne@sternir.com
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