Longeveron Inc. · NASDAQ:LGVN
Longeveron — Investor Presentation (October 2021)
36 slides from Longeveron's investor presentation, Oct 1, 2021
Slide 1: Cell Therapy for Aging-Related Disease & Life-Threatening Conditions
Nasdaq: LGVN October 2021 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
Longeveron At - a - Glance (NASDAQ: LGVN) 3 Biotechnology Regenerative Medicine / Cell Therapy 2014 February 12, 2021 $21.4 million (June 30, 2021) Industry Sect o r Found ed IP O Cash Lo cat ion Miami, Florida (Corporate headquarters, manufacturing, research and development)
Slide 4: Developing Cell Therapy For Aging-Related Disease & Life-Threatening Conditions
Longeveron: Developing Cell Therapy For Aging - Related Disease & Life - Threatening Conditions • Lead investigational therapeutic: Lomecel - B Œ (Allogeneic (donor - derived) medicinal signaling cells (MSCs)) – MSCs isolated from the bone marrow of young healthy adult donors • Lomecel - B produced
Slide 5: Medicinal Signaling Cells (MSC) Cell Therapy
Lomecel - B Medicinal Signaling Cells (MSC) Cell Therapy 5
Slide 6: Multimodal Mechanisms of Action
Lomecel - B: Multimodal Mechanisms of Action (MOA) Pro - vascular, Pro - regenerative and Anti - inflammatory: Repairs Tissue & Promotes Healing 6 1 A nt i - i nf l ammator y cytokines 2 3 Exosome and m ic ro RNA re l ease 4 Mitochondrial transfer (somatic cells) Cell - cell interactions (stem cells
Slide 7: Production and Administration
Lomecel - B Production and Administration 7 Healthy Adult Donors 18 - 45 Years; Rigorously Screened MSCs Isolated from Bone Marro w Aspi rat e MSCs Culture - Expanded into Billions of Cells 40 Minute IV Infusion
Slide 8: Proprietary, Scalable, Off-the-Shelf Cellular Therapy
Lomecel - B: Proprietary, Scalable, “Off - the - Shelf” Cellular Therapy 8 • 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 h
Slide 9: Robust Clinical Research Pipeline
Robust Lomecel - B Clinical Research Pipeline Focus Indication Location Funding/Partner ^ Phase 1 Phase 2 Phase 3 Central Nervous System Alzheimer’s Disease U.S. Aging Frailty Aging Frailty Phase 2b U.S. Flu Vaccine in Aging Frailty (Phase 1/2 “HERA” Trial) U.S. Aging Frailty Phase 2 Japan Metabolic
Slide 10: Clinical Research: Alzheimer's Disease
Lomecel - B Clinical Research Alzheimer’s Disease 10
Slide 11: Alzheimer's Disease: Targeting Neuroinflammation
Alzheimer’s Disease: Targeting Neuroinflammation 11 • 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 12: Phase 1 Study Designed for Safety & Exploratory Efficacy
Phase 1 Study Designed for Safety & Exploratory Efficacy 12 Group 1 Placebo Group 2 20M Lomecel - B Group 3 100M Lomecel - B Infusion (randomized, blinded) Clinical Screen Major Eligibility Criteria • 50 - 80 year old male or female • Clinically diagnosed with mild AD according to NIA - AA criteria
Slide 13: Low Dose: Slower Decline in MMSE in Phase 1 Trial
Low Dose Lomecel - B: Slower Decline in MMSE in Phase 1 Trial 13 MMSE: Mini Mental State Exam; 20M: 20 million. † Pl ac eb o ch ang e f rom ba s e li ne , p<0 . 05 * 20M Lomecel - B compared to placebo, p<0.05 * 20M Lomecel - B change from baseline, p<0.05 † Change from baseline, p<0.05 †† Change fr
Slide 14: 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 Low dose Lomecel - B group’s MMSE score declined more slowly than placebo Low dose Lomecel - B group showed positive change Q
Slide 15: Robust Clinical Program: Aging Frailty
Lomecel - B Robust Clinical Program Aging Frailty 15
Slide 16: 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 17: Aging Frailty Phase 2b Clinical Trial
Aging Frailty* Phase 2b Clinical Trial General objectives of the trial: • Can Lomecel - B improve physical function? • Is there a dose - response relationship with Lomecel - B? • Are there patient reported outcomes (PROs) that correlate with change in physical function? • Are there biomarkers that s
Slide 18: 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 19: Improved Walking Distance Over Months
Lomecel - B Improved Walking Distance Over 9 Months * Change from baseline; p<0.05 ** Change from baseline; p<0.01 # Versus placebo; p<0.05 ## Versus placebo; p<0.01 19
Slide 20: Increase in Walking Distance (200 Million Cell Dose Group)
Lomecel - B led to ~ 50 meter increase in walking distance (200 million cell dose group) - 30m Δ = 19% increased mortality 1 (CHF patients) Day 270 Δ6MWT (200M vs Placebo) = 63.4 meters (CI 95% 17.1 – 109.62), p=0.0077 32m MCID 4 (Congestive Heart Failure (CHF) patients) 17.8m MCID 2 and 20m MCID 3
Slide 21: Statistically Significant Dose Response Using 6MWT
Statistically - significant dose response using 6MWT at 180 days. 21
Slide 22: Change in PROMIS-Physical Function Correlated with Change in 6MWT
Change in PROMIS - Physical Function SF - 20 correlated with change in 6MWT when all Lomecel - B subjects combined 22 Change in PROMIS - PF Change in 6MWT PROMIS - PF: Patient - Reported Outcome Measurement Information System - Physical Function
Slide 23: 200M Cohort Showed Decreased Serum Levels of sTie2
Lomecel - B 200M cohort showed decreased serum levels of soluble Tie2 (sTie2) and dose - response @ Day 270: Suggests possible improvement in endothelial function. ##p<0.05 Lomecel - B vs. placebo * p<0.05 vs. baseline Statistically significant dose - response for change in sTie2 @ Day 270. Tie2 and
Slide 24: Anticipated Next Steps
Anticipated Next Steps 24 • Continued deeper review of clinical data, subgroup analysis, biomarker analysis; • Work with Longeveron’s Frailty Steering Committee & potentially regulators to determine next steps in clinical development plan; • Publish results in peer - reviewed journal
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 Objectives • Safety • Can Lomecel - B improve adaptive immunity against influenza viru
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: Robust Clinical Program: Congenital Heart Defect
Lomecel - B Robust Clinical Program Congenital Heart Defect 27
Slide 28: 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 29: HLHS: Phase 1 Trial (ELPIS I) Complete
Phase 1 Trial (ELPIS I) HLHS: Phase 1 Complete D e s ign • Open label, single arm, n=10 • Lomecel - B injected during second staged surgery (Glenn Procedure) • • Funded in part by Maryland Stem Cell Research Fund (MSCRF) Subjects followed for 12 months Secondary/exploratory endpoints Results * Safet
Slide 30: HLHS: On to Phase 2
HLHS: On to Phase 2 • N=38; randomized, controlled, blinded • 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 Phase 1 Trial (ELPIS I
Slide 31: Near Term Events for Program
Q4 2021 Q1 2022 Biomarker Data from Aging Frailty Phase 2b Data from Aging Frailty Influenza Vaccine Phase 1/2 Trial Initiation of Phase 2 Alzheimer’s Trial Initiation of Phase 2 Japan Aging Frailty Trial Near Term Events for Lomecel - B Program *relative timelines; based on Company’s current foreca
Slide 32: Corporate Overview
32 Corporate Overview LONGEVERON
Slide 33: Intellectual Property & Barriers to Competition
Intellectual Property & Barriers to Competition 33 Company - Owned Patent Applications • Allogeneic MSCs for use as a vaccine adjuvant • Allogeneic MSCs for improving humoral immunity • Allogeneic MSCs to treat sexual dysfunction • MSC protein expression from stimuli (potency assay) • MSCs to treat
Slide 34: Experienced and Successful Leadership
Experienced and Successful Leadership Geoff Green, MBA CEO 25+ years in life sciences, with a variety leadership roles throughout his time in the industry, including operations, business development, clinical affairs. Previously President and Acting CEO of DOR BioPharma, (now Soligenix (NAS: SNGX)),
Slide 35: Summary
S um m ary Well - characterized allogeneic MSC product in development Robust clinical pipeline with advanced trials in Alzheimer’s disease, aging related frailty, HLHS and other indications State - of - the - art GMP facility for stringent manufacturing Lomecel - B well - tolerated to date; supporti
Slide 36: Thank You
Thank You 36 www.longeveron.com Nasdaq: LGVN