Eledon Pharmaceuticals, Inc. · NASDAQ:ELDN
Eledon Pharmaceuticals — Investor Day (July 2025)
114 slides from Eledon Pharmaceuticals's investor day presentation, Jul 9, 2025
Slide 1: Research & Development Day
Research & Development Day July 9, 2025 1
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation contains forward‐looking statements that involves substantial risks and uncertainties. Any statements about the company’s future expectations, plans and prospects, including statements about its strategy, future operations, development of its product cand
Slide 3: Welcome & Introduction to Eledon
Welcome & Introduction to Eledon David-Alexandre C. Gros, MD Chief Executive Officer 3
Slide 4: Management Attendees and Guest Speakers
Management Attendees and Guest Speakers David-Alexandre C. Gros, MD Andrew Adams, MD, PhD Chief Executive Officer Professor and Chief, Division of Transplantation Eledon Pharmaceuticals University of Minnesota Eli Katz, MD William (Bill) E. Fitzsimmons, PharmD, MS Chief Medical Officer Adjunct Profe
Slide 5: Agenda
Agenda 1 Welcome and Introduction to Eledon David-Alexandre C. Gros, MD 2 Overview of Tegoprubart Steven Perrin, PhD 3 Solid Organ Transplantation – Kidney and Liver Andrew Adams, MD, PhD 4 Endpoints in Kidney Transplantation Bill Fitzsimmons, PharmD, MS 5 Eledon Kidney Transplantation Program Eli K
Slide 6: Tegoprubart is a Pipeline in a Product Opportunity
Tegoprubart is a Pipeline in a Product Opportunity DEVELOPMENT STAGE Early Human Trials/ INDICATIONS PRE-CLINICAL PHASE 1 PHASE 2 PHASE 3 NOTES TRANSPLANTATION • Phase 1b data expected Aug. 2025 Kidney • Phase 2 BESTOW data expected Nov. 2025 • U. Chicago investigator sponsored trial Islet Cell • Re
Slide 7: Areas of Focus
Areas of Focus Tegoprubart is positioned to replace 1 Need for a new standard of care in 5 tacrolimus with improved long-term immunosuppression outcomes and better tolerability 2 Limitations of current CNIs on graft longevity, 6 Xenotransplantation may greatly expand patient health and quality of li
Slide 8: Overview of Tegoprubart: An Anti CD40L Antibody
Overview of Tegoprubart: An Anti CD40L Antibody Steven Perrin, PhD President & Chief Scientific Officer 8
Slide 9: A Signal From Activated T Cells is Required For Maximal B Cell Maturation
A Signal From Activated T Cells is Required For Maximal B Cell Maturation A B C • Cell membranes from activated but not resting T cells (D10) induce B cell proliferation irrespective of antigen priming • Protease digestion of activated T cells in culture reduces B cell proliferation • Inhibition of
Slide 10: Mechanism Overview of CD40L Inflammatory Signaling
Mechanism Overview of CD40L Inflammatory Signaling • Interaction of CD40 with CD40L on immune cells mediates activation of the co-stimulatory immune pathway, controlling "cross talk” between the adaptive and innate immune systems • Maximal activation of inflammatory system is a 3-step process requir
Slide 11: Anti CD40L Treatment is Efficacious in Multiple Preclinical Models
Anti CD40L Treatment is Efficacious in Multiple Preclinical Models Neuromuscular Dermatology ALS MG NMO Vitiligo Alopecia MS Polymyositis Areata Hepatic Immune PsO PsA Gastrointestinal PSC Atopic Ah Ulcerative Dermatitis RA PBC Colitis IBD AS ALS Celiac Disease Sjogren;s ITP CLE T1D Syndrome Lupus N
Slide 12: BG9588 and IDEC-131 Ameliorate Disease in Lupus Nephritis & ITP
BG9588 and IDEC-131 Ameliorate Disease in Systemic Lupus Nephritis & Idiopathic Thrombocytopenia Purpura Systemic Lupus Nephritis Idiopathic Thrombocytopenia Purpura • BG9588 decreased proteinuria in patients with SLE • In a Phase 2 study, BG9588 and IDEC-1131 improved platelet • There was a decreas
Slide 13: Next Generation Anti-CD40Ls Required Elimination of Fc Effector Function
Next Generation Anti-CD40Ls Required Elimination of Fc Effector Function Platelet Activation Requires CD40L Activation of Platelets is Dependent on the Fc Activation & FcgRII Receptors Activity of the Bound Antibody • Anti-CD40L antibodies bind CD40L on platelets resulting in platelet activation Pla
Slide 14: CD40L/CD40 is a Validated Clinical Target
CD40L/CD40 is a Validated Clinical Target Antibody Company Target FC Clinical Data VIB4920 Amgen CD40L None SS, Kidney Tx, LN, RA Tegoprubart Eledon CD40L IgG1 Kidney Tx, ICTx, ALS Frexalimab Sanofi CD40L IgG1 MS Dapirolizumab UCB / Biogen CD40L None SLE Antova Biogen CD40L IgG4 ITP, LN, Kidney Tx I
Slide 15: Tegoprubart: Potential Best-in-Class Anti-CD40/CD40L Antibody
Tegoprubart: Potential Best-in-Class Anti-CD40/CD40L Antibody Targeting CD40 Ligand vs. CD40 Receptor IgG1 vs. fusion protein CD40L and CD40 CD40L only or pegylated FAB ✓Blocking anti-CD40L also inhibits CD11 costimulatory receptors on antigen presenting cells, thus blocking the ✓Up to over 2x times
Slide 16: Tegoprubart (AT-1501): Next Generation Humanized IgG1 Antibody
Tegoprubart (AT-1501): Next Generation Humanized IgG1 Antibody Lacking Fc Effector Function 5c8 AT-1501 • AT-1501 is a humanized IgG1 anti-CD40L antibody with high affinity for CD40L – EC50: 0.084 ng/ml • Rationally designed to address prior safety issues in class • AT-1501 Fc region lacks effector
Slide 17: Lack of Fc Effector Function Eliminates Platelet Activation
Lack of Fc Effector Function in Tegoprubart (AT-1501) Eliminates Platelet Activation 5c8 Tegoprubart AT-1501 binds CD40L on platelets but compared to 5c8 no longer activates human platelets as determined by PAC1 expression (Orange) on the surface of platelets measured by FACS Source: Gill, ATC 2021.
Slide 18: Inhibition of CD40L Improved Survival in NHP Kidney Transplantation
Inhibition of CD40L Improved Survival vs. Other Approaches in Non- Human Primate (NHP) Kidney Transplantation Monotherapy Studies NHP Survival Post Kidney Transplant Anti-CD40L Tac Untreated Belatacept Anti-CD40 Sources: Perrin, 2022; Song, 2014; Song, 2016; Duan, 2017. Note: In aggregated data from
Slide 19: NHP Renal Xenotransplantation: Advantage of Blocking CD40L
NHP Renal Xenotransplantation Experience has Demonstrated Advantage of Blocking CD40L vs. Tacrolimus or CD40 NHP Survival Post Xeno Kidney Transplant 100 75 Percent Survival p = 0.0031 50 25 Tac Anti-CD40 Anti-CD40L 0 0 50 100 150 200 250 300 350 400 Post-Transplant Days Source: Lovasik et al., “Ant
Slide 20: Solid Organ Transplantation
Solid Organ Transplantation Andrew Adams, MD, PhD Professor and Chief, Division of Transplantation University of Minnesota 20
Slide 21: Kidney and Liver Transplantation: Unmet Needs and Limitations of Current Therapy
Kidney And Liver Transplantation: Unmet Needs and Limitations of Current Therapy Andrew Adams MD/PhD University of Minnesota
Slide 22: Transplantation as a Therapy for End-stage Organ Disease
Transplantation as a Therapy for End-stage Organ Disease • Kidney Failure- • >800,000 people in US, >550,000 on dialysis • up to 30% mortality rate in the first year after diagnosis • 35.5 million with some form of kidney disease • Liver Failure/Cirrhosis • 3rd most common cause of death for adults
Slide 23: ESRD: Increasing Prevalence and Costs
ESRD: Increasing Prevalence and Costs 23
Slide 24: True size of the organ shortage could be many times larger
suggests that the true size of the organ shortage could be many times larger than is reflected by transplant waiting lists 24
Slide 25: Health Resources & Services Administration
Health Resources & Services Administration organdonor.gov U.S. Government Information on Organ Donation and Transplantation Statistics at a Glance & HRSA ABOUT US Number of men, women, and 109, OOO+ srsrerwatintt2.o September 2020. 39,718 17 transplants were performed in people die each day waiting
Slide 26: Increasing Demand, Limited Supply
Increasing Annual Kidney Transplants 30,000 28,142 27,759 Demand, 28,000 26,362 25,528 26,000 24,294 23,664 24,000 22,038 21,916 Limited 21,341 22,000 20,681 20,498 19,913 19,547 Number of Patients 20,000 18,639 18,247 18,418 17,686 17,583 17,912 17,909 17,800 17,711 17,844 17,367 17,645 17,342 17,4
Slide 27: How do dialysis and transplant compare?
How do dialysis and transplant compare? Dialysis can only do 10-15% of what anormal kidney does People on dialysis have to follow a rigid diet plan People on dialysis typically have low energy levels A transplanted kidney can do 50- 85% of what a normal kidney does dialysis and can go back to work H
Slide 28: Early Focus on Reducing Acute Rejection
Early Focus on Reducing Acute Rejection (<1yr) decreasing early rejection 28
Slide 29: Long-term survival of kidney grafts according to donor type
Long-term survival of kidney grafts according to donor type aft_survival (%) Kidney gr > o = Live donor 40 4 ~~ Deceased donor (non-ECD) -— Deceased donor (ECD) 0 T so —t T r 0 3 12 36 48 60 72 84 108 120 Time post-transplantation (months) ECD: Expanded criteria donor http://srtr.transplant.hrsa.gov
Slide 30: Transplant Outcomes
Transplant Outcomes HU Meier-Kriesche AJT March 2011, 11(3):450-462 30
Slide 31: Developing New Immunosuppression for the Next Generation of Transplant Recipients
Developing New Immunosuppression for the Next Generation of Transplant Recipients: The Path Forward M. D. Stegall”*, R. E. Morris?, R. R. Alloway? and R. B. Mannon* "Departments of Surgery and Immunology, von Liebig Transplant Center, Mayo Clinic, Rochester, MN Department of Cardiothoracic Surgery,
Slide 32: Why Kidney Transplants Fail
Why Kidney Transplants Fail Causes of Graft Loss > 6 months Causes of Death with Function Glomerulonephritis Other 8% Stroke 6% 6% Cardiovascular 36% Infection/ Sepsis 18% Chronic Rejection 36% Malignancy Other 9% 10% Death with function GI disorder 2% 50% Accident/ Unknown Suicide2% Ojo AO. Kidney
Slide 33: Non-Specific Immunosuppression
Non-Specific Immunosuppression Neurotoxicity Near Ubiquitous Target Distribution • CNI Hypertension • Cellcept Hyperlipidemia • Steroids • Rapamycin Immunosuppression Diabetes Nephrotoxicity 33
Slide 34: Chronic Renal Failure after Transplantation of a Nonrenal Organ
The NEW ENGLAND JOURNAL of MEDICINE ESTABLISHED IN 1812 SEPTEMBER 4, 2003 VOL.349 NO.10 Chronic Renal Failure after Transplantation of a Nonrenal Organ M.D., M.S., Robert A. Wolfe, Ph.D r, M.P.H., Laura Christensen, M.S Akinlolu O. Ojo, M.D., Ph.D., Philip J. Held, Ph.D., Friedrich K. Por Alan B. Le
Slide 35: CNI Side Effects Play a Leading Role in Kidney Graft Pathology
Calcineurin Inhibitor (CNI) Side Effects Play a Leading Role in Kidney Graft Pathology Over Time Source: Nankivell 2003; Vincenti ATC 2018 . . 35 35
Slide 36: Targeted immunosuppression
Targeted immunosuppression Specifically, Target T cell signaling Immunosuppression (e.g. CD40L) 36
Slide 37: The Promise of Targeted Immunosuppression
The Promise of Targeted Immunosuppression • Less medication related side effects • better cardiovascular profile • lower rates of diabetes, high blood pressure, high cholesterol • lower neurologic side effects • Improved kidney function • Less chronic rejection/injury • lower rates of antibody forma
Slide 38: CD40L Pathway: Opportunity for Improved Outcomes
CD40L Pathway: Opportunity for Improved Outcomes • Tegoprubart targets the most important pathway in transplant rejection • Prioritize long-term survival while minimizing side effects • Increase kidney function • limit cardiovascular risk- diabetes, high blood pressure/cholesterol • More effective a
Slide 39: Clinical Trial Endpoints in Kidney Transplantation
Clinical Trial Endpoints in Kidney Transplantation William E. Fitzsimmons, Pharm.D., M.S University of Illinois at Chicago Transplant Therapeutic Consortium 39
Slide 40: Clinical Trial Endpoints in Kidney Transplantation
Clinical Trial Endpoints in Kidney Transplantation William E. Fitzsimmons, Pharm.D., M.S. University of Illinois at Chicago Transplant Therapeutics Consortium 40
Slide 41: Background on Primary Endpoint for Immunosuppressant Approvals
Background on Primary Endpoint for Immunosuppressant Approvals ▪ Since the 1990’s the primary endpoint has been a composite of efficacy failure based on: • Biopsy Proven Acute Rejection • Graft Loss • Death • Loss to follow-up ▪ Acute rejection rates are less than 10% in the first year resulting in
Slide 42: iBOX as a novel surrogate endpoint for long-term graft survival
iBOX as a novel surrogate endpoint for long-term graft survival ▪ iBox is a composite biomarker panel incorporating: • Kidney Function ✓eGFR ✓Proteinuria ▪ Immunologic Response of the Recipient to the donor organ • Donor Specific Antibody (DSA) to HLA antigens in blood ▪ +(full)/-(abbreviated) Histo
Slide 43: iBox Composite Biomarker Panel (CBP)
iBox Composite Biomarker Panel (CBP) ▪ Developed by Loupy et al at the Paris Transplant Group (BMJ 2019;366:l4923) in 4,000 recipients ▪ Validated in 4 Independent Cohorts1 (2 single center and 2 RCTs) • Mayo Clinic Rochester • Helsinki • BMS BENEFIT Phase 3 study • BMS BENEFIT-Ext Phase 3 study 1 C
Slide 44: C Statistic for Discrimination
C Statistic for Discrimination C Statistic 0.5 = Random Chance >0.7 = Good model >0.8 = Strong model 1.0 = Perfect model 44
Slide 45: 1-year iBox as a predictor of 5-year graft loss
1-year iBox as a predictor of 5-year graft loss A. Klein, et al. Transpl. Int., 24 September 2023 doi: 10.3389/ti.2023.11951 45
Slide 46: 12-month eGFR is the best single predictor of long-term graft loss
12-month eGFR is the best single predictor of long-term graft loss Clinical Events and Renal Function in the First Year Predict Long-Term Kidney Transplant Survival Schold, Jesse D.1,2; Nordyke, Robert J.3; Wu, Zheng4; Corvino, Frank4,5; Wang, Weiying4; Mohan, Sumit5 Key Points • eGFR at 1 year post
Slide 47: iBox Components – eGFR as the major driver
iBox Components – eGFR as the major driver; Combined iBOX further improves upon eGFR A. Klein, et al. Transpl. Int., 24 September 2023 doi: 10.3389/ti.2023.11951 47
Slide 48: iBox superior to BPAR as a surrogate for long-term graft loss
iBox superior to biopsy proven acute rejection (BPAR) as a surrogate for long-term graft loss Klein et al. Comparing the prognostic performance of iBOX and biopsy-proven acute rejection for long-term kidney graft survival. American Journal of Transplantation, Volume 24, Issue 10, 1784 - 1793 48
Slide 49: Minimal Clinically Important Difference (MCID) for the iBOX
Minimal Clinically Important Difference (MCID) for the iBOX iBOX Difference Distribution Based ◦ 0.5 × SD (0.76-0.90) 0.38-0.45 0.40 difference in iBOX translates into a 4-5% Anchor Based improvement in 5-year ◦ eGFR difference of 5-10 mL/min/1.73 m2 0.23-0.46 graft loss ◦ cg score on biopsy 0 vs. 1
Slide 50: iBox Composite Biomarker Panel Regulatory Status
iBox Composite Biomarker Panel Regulatory Status ▪ Qualified by the European Medicines Agency (EMA) • Secondary endpoint prognostic for death-censored graft loss • Intended to be used in Phase 2/3 clinical trials to support the evaluation of novel IST applications • Both full and abbreviated iBOX qu
Slide 51: Impact of Reasonably Likely Surrogate Endpoints and Accelerated Approval
Impact of Reasonably Likely Surrogate Endpoints and Accelerated Approval on Novel Drug Development • IgA nephropathy 2017: No approved therapies; FDA accepted proteinuria as a RLSE at End of Phase 2 meetings with sponsors 2021-2025: Four accelerated approvals: Vanrafia (2025) Fabhalta (2024) Filspar
Slide 52: iBox Composite Biomarker Panel Conclusion
iBox Composite Biomarker Panel Conclusion • Only endpoint in FDA Biomarker Qualification Program addressing transplant patient, regulatory and clinician needs. • Best prognostic endpoint for long-term graft survival. • Allows for superiority of a new therapy and a new indication. • Facilitates use o
Slide 53: Clinical Development in the Prevention of De Novo Kidney Transplant Rejection
Clinical Development in the Prevention of De Novo Kidney Transplant Rejection Eliezer Katz, MD, FACS Chief Medical Officer 53
Slide 54: Phase 1b, Phase 2 and Long-Term Extension Kidney Transplantation Studies
Phase 1b, Phase 2 and Long-Term Extension Kidney Transplantation Studies are Running in Parallel 52-week, open label, single arm study Primary endpoints: Phase 1b • Safety & tolerability ATG induction therapy plus Secondary endpoints: Up to 36 participants • Graft function (eGFR) undergoing kidney C
Slide 55: Phase 1b Kidney Transplant Study Design
Phase 1b Kidney Transplant Study Design Day of Transplant D0 D1 D7 D14 D21 D28 D49 D70 D91 D112 D133 D154 D175 D196 D217 D238 D259 D280 D301 D322 D343 D364 Tegoprubart Cohort 1: 20 mg/kg Cohort 2: 10 mg/kg ATG Cohort 1: Up to 6 mg/kg Cohort 2: 4.5 mg/kg Steroids 5 mg MPA/MMF 1000/720 mg BID 55
Slide 56: Phase 1b Study: Thirteen Subjects Demographics and Disposition
Phase 1b Study: Thirteen Subjects Demographics and Disposition Age/ Donor HLA Ethnicity Donor Underlying Disease Gender Age Mismatch 60/F White Living Polycystic Kidney Disease 40 5 77/F White Deceased Diabetes 42 5 62/M White Living Cystic Disease 55 4 68/M White Living Diabetes 57 4 • Median recip
Slide 57: Phase 1b Study: Treatment Emergent Adverse Events
Phase 1b Study: Thirteen Subjects Treatment Emergent Adverse Events* System Organ Preferred Term N (%) Diarrhea 5 (38%) Constipation 5 (38%) • No cases of hyperglycemia, new Gastrointestinal onset diabetes, or tremor Nausea 4 (31%) Vomiting 3 (23%) Polyomavirus (BK) viremia 5 (38%) • 1 participant d
Slide 58: eGFRs Over Time Post Transplant
eGFRs Over Time Post Transplant: Mean ~53 mL/min/1.73m2 After 12 Months Using CNIs 50 40 40 eGFR eGFR 30 20 20 Donor type Treatment type Deceased CNI 10 Living CNI-free 0 0 16 32 48 0 2 4 6 Days Years Note: n = 4,868 patients. eGFR estimated using MDRD 4-variable GFR Equation. Source: Kosinski et al
Slide 59: Phase 1b Kidney Transplantation Aggregate Mean eGFR
Phase 1b Kidney Transplantation Aggregate Mean eGFR • Overall mean eGFR of all reported time points after day 30 post- transplant of 70.5 mL/min/1.73m² • Mean eGFR measured above 60 mL/min/1.73m² at all reported time points after day 30 post- transplant Note: Estimated glomerular filtration rate (eG
Slide 60: Updated Phase 1b Data to Be Presented at the World Transplant Congress
Updated Phase 1b Data to Be Presented at the World Transplant Congress Eledon will participate in an oral presentation at the World Transplant Congress featuring updated data from ~30 subjects in the ongoing open-label Phase 1b trial, including from both the 20 mg/kg and 10 mg/kg cohorts • Title: Te
Slide 61: Phase 2 BESTOW Kidney Transplant Study Design
Phase 2 BESTOW Kidney Transplant Study Design Randomization 1:1* Arm A Tegoprubart (20 mg/kg) + Corticosteroid†+ MMF Extension Study (K209) Tacrolimus + Corticosteroid †+ MMF Extension Study Arm B Screening (K209) (Day -28 to 0) rATG D0 D1 D3 D7 D14 D21 D28 M6 M12 M13 (Day of Transplant) Primary Ana
Slide 62: Upcoming Kidney Transplant Clinical Milestones
Upcoming Kidney Transplant Clinical Milestones September 2025 November 2025 August 2025 Phase 1b BESTOW BESTOW Report Updated Data, WTC LPLV (Month 12) Topline Results, Kidney Week Two kidney transplant catalysts (Phase 1b data and Phase 2 data) are expected in the coming months, each marking a key
Slide 63: Islet Cell Transplantation
Islet Cell Transplantation Piotr Witkowski, MD, PhD Associate Professor of Surgery and Director of Pancreatic and Islet Transplant Program University of Chicago 63
Slide 64: Islet Cell Transplantation
Islet Cell Transplantation Piotr Witkowski, MD, PhD Professor of Surgery and Director of Pancreas and Islet Transplant Program University of Chicago 64
Slide 65: Natural History of Type 1 Diabetes
Natural History of Type 1 Diabetes • Type 1 diabetes is an autoimmune disease that destroys insulin-producing cells of the pancreas, thus causing hyperglycemia and related health complications • ~2 million Americans live with Type 1 diabetes (T1D) • Insulin injection remains a standard of care thera
Slide 66: ICT Procedure
ICT Procedure • Minimally invasive alternative for whole pancreas tx • Today, it still requires immunosuppression medications • Isolated from deceased donor pancreas or • Manufactured from stem cells Source: Merani and Shapiro. Clin Sci 2006 Source: ? 66
Slide 67: Immunosuppressive Regimen
Immunosuppressive Regimen N=3 with up to 2 Islet Islet Transplant Transplants per Individual (T) Tegoprubart 20mg/kg IV Thymoglobulin 1.5 mg/kg x 3days Solumedrol 1mg/kg IV x 3 days T T T T T T T T T ~ -1Month -1 0 1 3 7 14 21 28 q3weeks MPS 720mg BID Day 0 50mg IV, Anti-TNF- Etanercept Day 3,7,10 2
Slide 68: Patient 01 Follow up
Patient 01 Follow up: 13 months 1st ITx HbA1c • 42 years old female 8.4 2nd ITx • Weight 88kg → 93kg 7.8 • BMI 30 → 32 7.2 7.1 6.9 • Transplant 1 (Day 0) • Islet IEQ = 363,000 6.3 6.1 6 5.9 • Islet IEQ/kg = 4,092 5.6 5.5 5.6 5.5 5.5 5.4 5.4 5.3 • Transplant 2 (Week 17) • Islet IEQ = 505,000 • Islet
Slide 69: Patient 02 Follow up
Patient 02 1st ITx HbA1c Follow up: 11 months • 30 years old female 8.5 8.2 • Weight 50kg • BMI 21 7.6 7.3 • 2 weeks after ATG 6.6 patient experienced pre-transplant serum 5.8 sickness and fully 5.4 5.3 5.1 5 5.2 5.1 5.1 recovered 4.9 5 4.9 5 4.7 4.8 • Transplant (Day 0) • Islet IEQ = 326,000 • Isle
Slide 70: Patient 02 Stable islet graft function at 9 months
Patient 02 Stable islet graft function at 9 months MMTT glucose MMTT c-peptide 180 4 160 3.5 140 3 120 2.5 100 2 80 1.5 60 40 1 20 0.5 0 0 0 15 30 60 90 120 0 15 30 60 90 120 Day 30 9M Day 30 6M 9M A1c 6.6 5.1. 5.0 70
Slide 71: Patient 03 Follow up
Patient 03 1st ITx HbA1c Follow up: 8 months 2 ITx • 37 years old male • Weight 92kg 9.9 9.7 • BMI 29.9 9.1 • HbA1c 9.9% • 90u of insulin per day 8.4 8.4 8 7.7 7.7 7.8 • Transplant 1 (Day 0) 7.1 7.1 7.2 7 • IEQ = 376,000, 6.8 6.9 6.7 • IEQ/kg = 4,086 • Transplant 2 (Week 28) • IEQ = 396,000 • IEQ/kg
Slide 72: Patient 03 Follow up
Patient 03 Follow up: 8 months • 37 years old male • Weight 92kg • BMI 29.9 • HbA1c 9.9% • 90u of insulin per day • Transplant 1 (Day 0) • IEQ = 376,000, • IEQ/kg = 4,086 • Transplant 2 (Week 28) • IEQ = 396,000 • IEQ/kg = 4,211 • Second transplant delayed for personal reasons 72
Slide 73: Patient 01, Patient 02, Patient 03
Patient 01 Patient 02 Patient 03 Weight 89kg, BMI 30 Weight 50kg, BMI 21 Weight 92kg, BMI 30 IEQ 363,000 IEQ. 4,091 IEQ/kg IEQ 326,000 6,775 IEQ/kg IEQ 376,000 4,086 IEQ/kg Day 75 Day 75 Day 75 Insulin requirements down 64 u/day Insulin requirements down 60 u/day Insulin requirements down 74 u/day f
Slide 74: Patient 01, Patient 02, Patient 03
Patient 01 Patient 02 Patient 03 Weight 89kg, BMI 30 Weight 50kg, BMI 21 Weight 92kg, BMI 30 IEQ 363,000 IEQ. 4,091 IEQ/kg IEQ 326,000 6,775 IEQ/kg IEQ 376,000 4,086 IEQ/kg On day 75 after a single donor islet transplant, all 3 patients demonstrated robust islet function and insulin production as re
Slide 75: Summary
Summary All 3 subjects: • Achieved stable islet graft function, improved blood glucose control and insulin independence – No unexpected Adverse Events: • No severe hypoglycemic episodes • No opportunistic infections • No thromboembolic events • No signs of kidney toxicity or of neurotoxicity • No GI
Slide 76: Islet Engraftment Results with anti CD40L mAb
Islet Engraftment on day 75 (AUC c-pep)/IEQ Results with anti CD40L mAb replacing tacrolimus 80 Islet Engraftment 2-3 fold higher 70 RESULTS WITH TRADITIONAL TACROLIMUS BASED pmol/ml*min/IEQ*10^5 60 IMMUNOSUPRESSION 50 40 30 20 10 0 BMI 29 BMI 29 01 02 03 Anti-inflammatory → Etanercept (anti TNF) Re
Slide 77: Conclusions & Next Steps
Conclusions & Next Steps • Preliminary results of our pilot study to date support the safety and effectiveness of tegoprubart in islet graft protection after transplantation in patients with T1D • Next steps: – Current patients will continue Tegoprubart for another year – Breakthrough T1D has funded
Slide 78: Islet Cell Transplantation: Program & Next Steps
Islet Cell Transplantation: Program & Next Steps Steve Perrin, PhD President & Chief Scientific Officer 78
Slide 79: Islet Cell Transplantation Program Next Steps
Islet Cell Transplantation Program Next Steps • Complete enrollment of 9 subjects in UChicago ICT IST • Support ICT for Patients with Impaired Kidney Function IST • 1st subject enrolled in Sernova ICT collaboration • Engage FDA on ICT path to approval in 1H2026 79
Slide 80: Xenotransplantation
Xenotransplantation John D. Cleveland, MD Associate Professor of Clinical Surgery Program Director, Congenital Cardiac Surgery Fellowship Children’s Hospital Los Angeles Department of Surgery, Keck School of Medicine 80
Slide 81: Altering Care Paradigms with Co-Stimulation Blockade
ALTERING CARE PARADIGMS WITH CO-STIMULATION BLOCKADE John D. Cleveland, MD Associate Professor of Clinical Surgery Program Director, Congenital Cardiac Surgery Fellowship Children’s Hospital Los Angeles Department of Surgery, Keck School of Medicine
Slide 82: Organ Shortages
Organ Shortages • Over 100,000 Americans are on transplant waiting lists – 83% Kidney; 11% Liver; 3% Heart; 2% Lung • ONLY ~39,000 transplants performed annually • Over 20 patients die each day waiting for an organ • Demand > Supply 82
Slide 83: Microcosm of the Organ Deficit
Microcosm of the Organ Deficit 10-year Volumes in U.S. Pediatric Heart Transplant Total 2025 2024 2023 2022 2021 2020 2019 2018 2017 2016 2015 2014 < 1 1,323 20 96 118 114 111 124 133 119 111 130 127 120 Year 1-5 1,164 18 108 93 97 106 100 114 113 107 101 110 97 Years 6-10 753 16 80 78 66 67 67 80 6
Slide 84: A Growing Waitlist
A Growing Waitlist • Waitlist has increased • Transplant rates 36.7% since 2011 decreasing in candidates – <1 yr (21.4%) – 1-5 yrs (23.6%) – <1yr (41.8%) – 1-5yrs (37%) > Median Wait Time 110days – 6-11 yrs (20.5%) – 6-11 yrs (~) – 12-17 yrs (29%) 84
Slide 85: The Current Paradigm
Seas rSCuneaet, The Current Paradigm
Slide 86: The Clinical Vision
The Clinical Vision Cardiac Xenotransplantation as a Bridge
Slide 87: Enabling Technology
Enabling Technology • Triple Knockout Pigs + Co-Stimulation Blockade (Tegoprubart) Gal (-) Neu5Gc (-) Sda (-) 87
Slide 88: Animal Model for Proof of Concept
Animal Model for Proof of Concept Immunosuppression Regimen -Tegoprubart -Calcineurin Inhibitor -Mycophenylate -Rapamycin mofetil -Corticosteroid -Corticosteroid 88
Slide 89: Results
RESULTS OCXT Total Graft Ischemic CPB Time Survival Time Outcome Time (min) (min) #1 136 122 <24 hours PCXD #2 77 119 90 days AMR #3 71 81 241 days AMR #4 68 85 < 24 hours SIXR #5 63 80 < 24 hours Technical Error #6 60 85 9 days AMR, CMR #7 59 90 2 days SIXR #8 43 85 693 days Alive and Well #9 31 79
Slide 90: Cardiac Xenotransplant Performance
Cardiac Xenotransplant Performance OCXT 10 Hemodynamics 200 45 180 40 LVEDP, PA Diastolic, PA Systolic 160 35 Heart Rate, LVSystolic 140 30 120 25 100 20 80 15 60 40 10 20 5 0 0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 Post operative Week LVSystolic HeartRate LVEDP PASystolic PADiastolic 90
Slide 91: Xenosensitization on Tegoprubart
Xenosensitization on Tegoprubart Longitudinal DSA for Xeno-heart NHP Transplants 6 IgG (B000822) IgM (B000822) 5 IgG (B004922) Pre- to Post-Transplant Levels IgM (B004922) DSA Fold Change from IgG (B002821) 4 IgM (B002821) IgG (B004321) 3 IgM (B004321) IgG (B010022) 2 IgM (B010022) IgG (B006922) 1 I
Slide 92: Allosensitization on Tegoprubart
Allosensitization on Tegoprubart 92
Slide 93: Impact of Removing Tegoprubart
Impact of Removing Tegoprubart C4d Removal of Tegoprubart 93
Slide 94: T-regulatory Cells on Tegoprubart
T-regulatory Cells on Tegoprubart 94
Slide 95: What We've Learned
What We’ve Learned • The impact of co-stimulation blockade with Tegoprubart appears to be significant: 1. Prevents Xenosensitization 2. Prevents Allosensitization 3. Allows T-regulatory Cell Recovery 4. Allows Prolonged Xenograft Function (~700 days) 5. Development and growth are not hindered 6. Lim
Slide 96: Spin-Off Applications
Childrens Bley 3 USC University of Hospital WW Southern California Spin-Off Applications
Slide 97: Valve Transplants
Valve Transplants • ~50,000 Valve Replacements in the U.S. annually (adults) – ~1,500 pediatric valve replacements • ~300,000 worldwide • Ideal Valve – Organic – No blood thinners – Resistant to infection – Grow with recipient • Xenograft valves poised to meet this need • Potential Single Medication
Slide 98: Allotransplant Immunosuppression
Allotransplant Immunosuppression • >4,000 Cardiac Allotransplants (adult + pediatric) performed yearly in the U.S. • Impact of prolonged calcineurin based immunosuppression – Nephrotoxicity (eventual need for renal transplant) – Increased risk of infection – Increased risk of cancers • Potential tra
Slide 99: Xenotransplantation: Program & Next Steps
Xenotransplantation: Program & Next Steps Eli Katz, MD Chief Medical Officer 99
Slide 100: Clinical Xenotransplantation Update
Clinical Xenotransplantation Update • First patient who received a porcine kidney died at 4 months from cardiac event with functional xenograft • Second kidney patient continues to do well at ~6 months – He is now the longest living ever individual with a xeno organ – Threw out the 1st pitch at a Re
Slide 101: Commercial Opportunity
Commercial Opportunity David-Alexandre C. Gros, MD Chief Executive Officer 101
Slide 102: Transplant Immunosuppression is a Large Market
Transplant Immunosuppression is a Large Market with Multiple Drugs Having Achieved Blockbuster Revenues • Global organ transplant immunosuppressant Novartis’ Astellas’ tacrolimus, first market size estimated cyclosporine approved in the U.S. in $5.3+ billion franchise achieved 1994, reached blockbus
Slide 103: Over 48,000 Organs Were Transplanted in the United States in 2024
Over 48,000 Organs Were Transplanted in the United States in 2024 Lungs Corneas 3K performed in 2024 Can help improve 20 sight for YEARS 68% 3-year survival rate Liver Heart 11.5K performed in 2024 4.6K performed in 2024 75% 5+ year survival 5-year survival 70% rate of OR MORE Pancreas Kidneys perfo
Slide 104: The Demand for Kidney Transplants is Expected to Continue Growing
The Demand for Kidney Transplants is Expected to Continue Growing… 900,000 • Over the past decade, the U.S. ESRD 815,896 800,000 and dialysis population has grown by approximately 2.5% annually, reflecting 700,000 a 28% total increase from 2012 to 2022 600,000 550,859 • This growth is driven by risi
Slide 105: Supply of Organs and Number of Transplants Performed
…Along with the Supply of Organs and the Number of Resulting Transplants Performed Annual Kidney Transplants • While the supply of kidneys for 30,000 transplant has increased, this 28,142 27,759 28,000 26,362 growth has come almost entirely 26,000 25,528 from deceased donors. 24,294 23,664 24,000 22
Slide 106: Most Kidney Transplant Recipients Have One or More Risk Factors
Most Kidney Transplant Recipients Have One or More Factors That Increase Risk and the Percentage is Expected to Increase Distribution of transplant recipients and donors across various risk factor groups (2023) • Several measures are employed to Number of Segment patients Segment Number of patients
Slide 107: Nearly All Transplant Recipients Eligible for Immunosuppressant Coverage
Nearly All Transplant Recipients in the U.S. are Eligible for Coverage of Immunosuppressants Drugs Primary Payer Coverage Among Adult Current Medicare Immunosuppression Kidney Transplant Recipients (2022) Drug Coverage Other/unknown Medicaid 3% • The Comprehensive Immunosuppressive Drug 7% Coverage
Slide 108: Kidney Transplantation is Highly Concentrated Market
Kidney Transplantation is Highly Concentrated Market Distribution of Kidney Transplants Among • U.S. kidney transplant market is highly U.S. Transplant Centers concentrated, with half of kidney (100% = 27,759 transplants) transplants performed at the top 20% of centers (46 centers) 20% • 80% of tran
Slide 109: Potential Future Expansion Areas for Tegoprubart in Transplant
Potential Future Expansion Areas for Tegoprubart in Transplant Beyond Kidney, Islet Cell and Xeno • Liver Transplantation (11,500 US patients per year) Other Transplantation Types • Heart Transplantation (4,600 US patients per year) • Lung Transplantation (3,000 US patients per year) • Switch from O
Slide 110: Key Takeaways
Key Takeaways David-Alexandre C. Gros, MD Chief Executive Officer 110
Slide 111: Eledon's Vision: One Transplant for Life
Eledon’s Vision is Transforming Transplant Immunosuppression to Deliver “One Transplant for Life” Developing tegoprubart to CNIs cause kidney toxicity, Treatment goal is preventing BESTOW is the first Phase 2 become the new standard of hypertension and diabetes, graft loss which in kidney head-to-he
Slide 112: Expected Catalysts Over Next 12 Months
Expected Catalysts Over Next 12 Months 2H2025 1H2026 • Phase 1b Kidney Transplant update at World • Engage FDA on Phase 3 Kidney Transplant Transplant Congress in August 2025 • Engage FDA on ICT path to approval • NHP Liver Transplant data at World Transplant Congress in August 2025 • 1st subject en
Slide 113: Q&A
Q&A Yip aE |
Slide 114: Pharmaceuticals
Pharmaceuticals