tegoprubart (AT-1501) / Eledon - LARVOL DELTA

Efficacy and Safety of Tegoprubart for the Prevention of Rejection in Kidney Transplantation: Results from the Phase 2 BESTOW Trial (KIDNEY WEEK 2025) - P2 | "All received rabbit anti-thymocyte globulin as induction, corticosteroids, and mycophenolate. Patients were randomized 1:1 to intravenous tegoprubart 20 mg/kg on days 1, 3, 7, 14, 21, 28, and every 21 days thereafter, or twice daily oral tacrolimus at a target whole blood trough concentration of 6–12 ng/mL to month 6, and 6–8 ng/mL thereafter...Initial efficacy and safety results at 12 months will be presented. Conclusion The results of the BESTOW study may provide a basis for continuing the development of tegoprubart as a potential alternative to tacrolimus for preserving allograft function and preventing kidney transplant rejection."

Clinical • Late-breaking abstract • P2 data • Cardiovascular • Chronic Kidney Disease • Diabetes • Diabetic Nephropathy • Hypertension • Immunology • Metabolic Disorders • Movement Disorders • Nephrology • Transplant Rejection • Transplantation • CD40LG

AT-1501-K207: Safety and Efficacy of Tegoprubart in Patients Undergoing Kidney Transplantation (clinicaltrials.gov) - P2 | N=127 | Completed | Sponsor: Eledon Pharmaceuticals | Active, not recruiting ➔ Completed | Trial completion date: Mar 2025 ➔ Sep 2025 | Trial primary completion date: Mar 2025 ➔ Sep 2025

Trial completion • Trial completion date • Trial primary completion date • Immunology • Transplant Rejection • Transplantation

Eledon Pharmaceuticals to Present Results from Phase 2 BESTOW Trial of Tegoprubart for the Prevention of Rejection in Kidney Transplantation at the American Society of Nephrology Kidney Week 2025 Annual Meeting

P2 data • Transplant Rejection • Transplantation

Tolerance Through Mixed Chimerism (Sip-Tego) (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Tatsuo Kawai, MD, PhD | Not yet recruiting ➔ Recruiting

Enrollment open • Renal Disease • Transplantation

First and Second Genetically Modified Pig Kidney Xenotransplants in Living Human Recipients: Immunological and Physiological Insights (WTC 2025) - "Induction immunosuppression included rituximab, rabbit ATG, steroids, and complement inhibition (ravulizumab in Patient 1, pegcetacoplan in Patient 2). Maintenance therapy consisted of tegoprubart (anti-CD154mAb), tacrolimus, mycophenolate, and prednisone.* Clinical Course: Patient 1: Achieved immediate urine production, with serum creatinine decreasing from 8 to 2 mg/dL within one week... These first-in-human xenotransplants showed immediate graft function, stable metabolism, and no early antibody-mediated rejection, demonstrating feasibility of kidney xenotransplantation. Differences in Ca-Phos metabolism were likely related to absence of PTH on the first patient. Xenotransplantation offers a promising solution for patients facing prolonged wait times."

Anemia • Antibody-mediated Rejection • Cardiovascular • Congestive Heart Failure • Coronary Artery Disease • Diabetes • Endocrine Disorders • Heart Failure • Hematological Disorders • Infectious Disease • Metabolic Disorders • Nephrology • Pain • Peripheral Arterial Disease • Transplant Rejection • Transplantation • CD40LG

Updated Data from Eledon Pharmaceuticals’ Ongoing Phase 1b Trial of Tegoprubart in Patients Undergoing Kidney Transplantation to be Presented at the World Transplant Congress 2025 (GlobeNewswire) - "Eledon Pharmaceuticals...that updated clinical data from its ongoing open-label Phase 1b study evaluating tegoprubart for the prevention of rejection in subjects announced undergoing kidney transplantation will be presented at the World Transplant Congress (WTC) taking place in San Francisco from August 2-6, 2025. The oral presentation will feature updated results from approximately 30 kidney transplant recipients and will be presented by Dr. John Gill from the University of British Columbia....In addition to the oral presentation, new preclinical data, utilizing tegoprubart for the prevention of rejection in non-human primates undergoing liver transplantation, will be presented by Dr. Andrew Adams in a poster session."

P1 data • Preclinical • Transplant Rejection • Transplantation

Eledon Presents Updated Data from Ongoing Phase 1b Trial Evaluating Tegoprubart for Prevention of Rejection in Kidney Transplantation (GlobeNewswire) - P1b/2 | N=42 | NCT05027906 | Sponsor: Eledon Pharmaceuticals | "Results from the oral presentation...were presented today at the World Transplant Congress (WTC)....As of the July 2025 cutoff date, 32 patients undergoing kidney transplantation have been enrolled in the Phase 1b study. Updated data showed that kidney function, as assessed by estimated glomerular filtration rate (eGFR), stabilized after the first month post-transplant and remained in the range of approximately 68 mL/min/1.73 m2 through 12 months for patients (n=12) who remained on tegoprubart​. Kidney function in the intention-to-treat population (n=15) was approximately 63 mL/min/1.73 m2 at 12 months....Topline results from the Phase 2 BESTOW trial are anticipated in November 2025."

P1 data • P2 data • Transplant Rejection • Transplantation

Tegoprubart, an Anti-CD40L Antibody, for the Prevention of Rejection in Kidney Transplantation: An Ongoing Phase 1B Study (WTC 2025) - "All participants received rabbit anti-thymocyte globulin and a loading dose of tegoprubart followed by a maintenance regimen consisting of tegoprubart 20 mg/kg IV q3 weeks, mycophenolate mofetil and corticosteroids. It is postulated that tegoprubart could be an alternative therapy for prevention of rejection in kidney transplant recipients with the potential for improved kidney function compared to current standard of care. Thus far tegoprubart has been well tolerated and participants have shown good kidney function. Diagnosis and management of rejection on tegoprubart are areas of further studies and are a critical part of the development plan."

IO biomarker • P1 data • Cardiovascular • Diabetes • Hypertension • Metabolic Disorders • Transplantation • CD40LG

CD40L Co-Stimulation Blockade as a Non-Toxic Calcineurin Inhibitor-Free Immunosuppression for Beta Cell Replacement Therapy: Preliminary Results from a Pilot Clinical Study (WTC 2025) - "Preliminary data from the first three patients indicate that tegoprubart with mycophenolate is well-tolerated and effective in preventing islet graft loss, with no unexpected adverse events. Further studies are warranted to validate these findings."

Clinical • Diabetes • Hypoglycemia • Infectious Disease • Metabolic Disorders • Type 1 Diabetes Mellitus • CD40LG

The Promise and Perils That Novelty Bestows: A Case of Pulmonary Mucor in a Patient Treated with Tegoprubart (WTC 2025) - "Source control was achieved via right lower lobectomy with intercostal muscle flap and cryoablation, mycophenolate was temporarily withheld, and he was treated with isavuconazonium sulfate. Tegoprubart, as part of the BESTOW trial, offers a potential alternative to CNI, but further studies are needed to understand its safety profile and efficacy fully. Moreover, investigators and clinicians alike must remain vigilant for invasive fungal infections in patients treated with the study drug."

Clinical • Cardiovascular • Chronic Kidney Disease • Congestive Heart Failure • Coronary Artery Disease • Diabetes • Dyslipidemia • Focal Segmental Glomerulosclerosis • Glomerulonephritis • Heart Failure • Hypertension • Infectious Disease • Metabolic Disorders • Nephrology • Pneumonia • Respiratory Diseases • Solid Organ Transplantation • Type 2 Diabetes Mellitus

Anti-CD154 Promotes Long-Term Liver Transplant Survival in Nonhuman Primates (WTC 2025) - "Tegoprubart, a next generation anti-CD154 antibody, is currently in a phase II clinical trial for kidney transplantation. The purpose of this study was to evaluate anti-CD154 therapy in a preclinical model of liver transplantation.* 8 NHP underwent orthotopic liver transplantation and received maintenance immunosuppression with mycophenolate mofetil and steroids... Anti-CD154 promoted long-term liver allograft survival without adverse events or opportunistic infections. Anti-CD154 treatment represents a promising strategy to suppress the allo-immune response in liver transplantation and may facilitate future studies of tolerance."

Diabetes • Hepatology • Infectious Disease • Metabolic Disorders • Nephrology • Transplantation • CD40 • CD40LG

AT-1501-K102: Safety, Pharmacokinetics, and Efficacy of AT 1501 in Patients Undergoing Kidney Transplant (clinicaltrials.gov) - P1/2 | N=48 | Recruiting | Sponsor: Eledon Pharmaceuticals | N=24 ➔ 48 | Trial completion date: Sep 2025 ➔ Jan 2027 | Trial primary completion date: Sep 2025 ➔ Jan 2027

Enrollment change • Trial completion date • Trial primary completion date • Transplantation

Eledon Pharmaceuticals to Host R&D Day on July 9, 2025 in New York City (GlobeNewswire) - "Eledon Pharmaceuticals...announced that it will host an R&D Day in New York City on Wednesday, July 9, 2025 beginning at 8:30 am ET....The R&D Day event will highlight Eledon’s lead investigational candidate, tegoprubart, with a focus on its clinical development in organ and cell transplantation, including the ongoing Phase 2 BESTOW trial. Additional sessions will address unmet needs in solid organ transplantation, the evolution of clinical endpoints such as iBox and eGFR, and the Company’s strategic opportunities in islet cell transplantation and xenotransplantation."

P2 data • Immunology • Solid Organ Transplantation • Transplant Rejection

Tegoprubart as Cd40l Co-Stimulation Blockade in a Non-Toxic Calcineurin Inhibitor-Free Immunosuppression for Beta Cell Replacement Therapy: Preliminary Results from a Pilot Clinical Study (IPITA 2025) - "Preliminary data from the first three patients indicate that tegoprubart with mycophenolate is well-tolerated and effective in preventing islet graft loss, with no unexpected adverse events. Further studies are warranted to validate these findings and explore the long-term benefits of novel regimen."

Clinical • Diabetes • Hypoglycemia • Infectious Disease • Metabolic Disorders • Type 1 Diabetes Mellitus • CD40LG

Tolerance Through Mixed Chimerism (Sip-Tego) (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: Tatsuo Kawai, MD, PhD

New P1 trial • Renal Disease • Transplantation

Eledon Pharmaceuticals Reports First Quarter 2025 Operating and Financial Results (GlobeNewswire) - "Anticipated Upcoming Milestones: August 2025: Report updated interim clinical data from the ongoing Phase 1b open-label trial evaluating tegoprubart for the prevention of organ rejection in kidney transplant patients; 4Q 2025: Report topline results from the Phase 2 BESTOW trial of tegoprubart in kidney transplantation; Late 2025: Report updated interim clinical data from the investigator-led clinical trial with UChicago Medicine in subjects with type 1 diabetes treated with tegoprubart as part of an immunosuppression regimen for the prevention of pancreatic islet transplant rejection."

Clinical data • Transplant Rejection • Transplantation • Type 1 Diabetes Mellitus

Eledon Pharmaceuticals Reports Fourth Quarter and Full Year 2024 Operating and Financial Results (GlobeNewswire) - "Research and development (R&D) expenses for the year ended December 31, 2024 were $52.0 million, including $4.3 million of non-cash stock-based compensation expense, compared to $30.3 million, including $1.5 million of non-cash stock-based compensation expense, for the comparable period in 2023. The increase was primarily driven by a rise in clinical development expenses related to the Phase 1b, Phase 2 BESTOW and Phase 2 open-label extension trials for kidney transplantation..."

Commercial • Transplant Rejection

Eledon Pharmaceuticals Announces Use of Tegoprubart as Key Component of Immunosuppression Regimen in its Second Transplant of a Genetically Modified Pig Kidney into a Human (GlobeNewswire) - "Eledon Pharmaceuticals...today announced that tegoprubart, the company’s investigational anti-CD40L antibody, was used as a key component of the immunosuppression therapy regimen in a patient who recently received a transplanted kidney from a genetically modified pig...MGH received Food and Drug Administration (FDA) approval to proceed with this transplant and plans to perform two additional xenotransplants this year, further advancing the field of xenotransplantation....'Our anti-CD40L antibody tegoprubart represents a novel approach to immunosuppression therapy with the potential to improve safety and efficacy and enable patients to live longer with their transplanted organs.'"

Clinical • FDA event • Transplant Rejection

Costimulation blockade: the next generation. (PubMed, Curr Opin Organ Transplant) - "The focus in transplantation is shifting toward safer, long-term therapies with greater accessibility. Investigational agents with subcutaneous delivery methods could overcome logistical challenges, improve adherence, and redefine posttransplant care. These advancements in costimulation blockade may enhance long-term graft survival and transform the management of KT recipients."

Journal • Cardiovascular • Transplantation • CD40LG

AT-1501-K207: Safety and Efficacy of Tegoprubart in Patients Undergoing Kidney Transplantation (clinicaltrials.gov) - P2 | N=120 | Active, not recruiting | Sponsor: Eledon Pharmaceuticals | Recruiting ➔ Active, not recruiting

Enrollment closed • Immunology • Transplant Rejection • Transplantation

Eledon Pharmaceuticals Highlights Recent Business Milestones and Provides 2025 Outlook (GlobeNewswire) - "Completed enrollment of 120 patients in the Phase 2 BESTOW clinical trial approximately four months earlier than originally planned....Completed two financings for combined total gross proceeds of $135.0 million, before deducting any offering related expenses, which is anticipated to support company operations to the end of 2026....Summer 2025: Report updated interim clinical data from the ongoing Phase 1b and long-term efficacy extension studies of tegoprubart in kidney transplantation. 4Q 2025: Report topline results from the Phase 2 BESTOW trial of tegoprubart in kidney transplantation.'"

Enrollment closed • Financing • P1 data • P2 data • Transplant Rejection

Transplantation of a genetically modified porcine heart into a live human. (PubMed, Nat Med) - "He was maintained on a costimulation (anti-CD40L, Tegoprubart) blockade-based immunomodulatory regimen...No evidence of porcine cytomegalovirus replication in the xenograft was observed. Strategies to overcome the obstacle of antibody-mediated rejection are needed to advance the field of xenotransplantation."

Journal • Preclinical • Antibody-mediated Rejection • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Cytomegalovirus Infection • Fibrosis • Heart Failure • Immunology • Transplantation • CD40LG

New Developments and Therapeutic Drug Monitoring Options in Costimulatory Blockade in Solid Organ Transplantation: A Systematic Critical Review. (PubMed, Ther Drug Monit) - "The routine use of costimulation blockade in SOT is hindered by problems in efficacy compared with the standard of care. Costimulatory inhibitors could be combined in a calcineurin inhibitor-free regimen. Future PK/pharmacodynamic studies in costimulatory agents and personalized medicine could warrant TDM of these agents."

Journal • Review • Hepatology • Solid Organ Transplantation • Transplantation • CD40LG • CD80 • TNFSF4

Eledon Pharmaceuticals Announces Recent Business Highlights and Third Quarter 2024 Financial Results (GlobeNewswire) - "Anticipated Upcoming Milestones: Mid-2025: Report updated interim clinical data from the ongoing Phase 1b and long-term safety and efficacy extension studies of tegoprubart in kidney transplantation....4Q 2025: Report topline results from the Phase 2 BESTOW trial of tegoprubart in kidney transplantation....Research and development (R&D) expenses for the third quarter of 2024 were $16.5 million, compared to $7.9 million for the comparable period in 2023, an increase of $8.6 million. The increase was primarily driven by a rise in clinical development expenses related to the Phase 1b and Phase 2 BESTOW studies..."

Commercial • P1 data • P2 data • Immunology • Transplant Rejection

Safety and tolerability of tegoprubart in patients with amyotrophic lateral sclerosis: A Phase 2A clinical trial. (PubMed, PLoS Med) - P2a | "Tegoprubart appeared to be safe and well tolerated in adults with ALS demonstrating dose-dependent reduction in pro-inflammatory chemokines and cytokines associated with ALS. These results warrant further clinical studies with sufficient power and duration to assess clinical outcomes as a potential treatment for adults with ALS."

Biomarker • IO biomarker • Journal • P2a data • Amyotrophic Lateral Sclerosis • CNS Disorders • Fatigue • Immunology • Inflammation • Pain • Transplant Rejection • Transplantation • CD40LG • NEFL