Tacrolimus

Completed enrollment in Phase 2 BESTOW trial assessing tegoprubart in kidney transplantation four months ahead of schedule; on track to report topline results in fourth quarter of 2025 Presented updated data on 13 participants from ongoing Phase 1b trial that continue to support safety and tolerability of tegoprubart for prevention of organ rejection in kidney transplantation Announced positive initial data from first three subjects with type 1 diabetes treated with tegoprubart as part of immunosuppression regimen following islet transplantation in investigator-initiated trial at UChicago Medicine Completed two financings totaling $135 million in combined gross proceeds, with funds expected to support operations through end of 2026 IRVINE, Calif., Jan. 13, 2025 (GLOBE NEWSWIRE) -- Eledon Pharmaceuticals, Inc. (“Eledon”) (Nasdaq: ELDN) today announced a summary of 2024 accomplishments and provided guidance for anticipated 2025 business milestones. “2024 was a transformative year for Eledon as we achieved multiple key clinical milestones for tegoprubart across kidney, islet cell, and xenograft transplantation,” said David-Alexandre C. Gros, M.D., Chief Executive Officer of Eledon. “Moreover, the data we reported reinforced the potential of tegoprubart to be a best-in-class immunosuppression therapy to prevent transplant rejection and disrupt the current standard of care. We now look forward to another pivotal year ahead.” 2024 Key Highlights Completed enrollment of 120 patients in the Phase 2 BESTOW clinical trial approximately four months earlier than originally planned. The Phase 2 trial is designed to assess the safety and efficacy of tegoprubart for the prevention of organ rejection in patients undergoing kidney transplantation. Presented updated data at the American Transplant Congress (ATC) from the ongoing Phase 1b open-label trial evaluating tegoprubart for the prevention of organ rejection in kidney transplant patients. Updated data from 13 participants demonstrated that tegoprubart was generally safe and well tolerated, with an overall mean estimated glomerular filtration rate (eGFR) of 70.5 mL/min/1.73m2 at all reported time points after day 30 post-transplant. Two participants completed more than 12 months on therapy post-transplant, and both demonstrated mean eGFRs above 90 mL/min/1.73m2 at one-year post-transplant. Announced positive initial data for the first three islet transplant recipients treated with tegoprubart as part of an immunosuppression regimen for the prevention of islet transplant rejection in subjects with type 1 diabetes in an investigator-initiated trial at the University of Chicago Medicine’s Transplant Institute. The data demonstrated potentially the first human cases of insulin independence achieved using an anti-CD40L monoclonal antibody immunosuppression therapy without the use of tacrolimus, the current standard of care for prevention of transplant rejection. Announced the use of tegoprubart as part of the immunosuppression treatment regimen used following the first-ever kidney xenotransplant procedure of a genetically modified pig kidney to a human. 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. Anticipated 2025 Milestones 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. 2025: Report longer-term follow up results from the investigator-led clinical trial at UChicago Medicine Transplant Institute for pancreatic islet transplantation in subjects with type 1 diabetes involving use of tegoprubart as part of immunosuppression regimen. About Eledon Pharmaceuticals and tegoprubart Eledon Pharmaceuticals, Inc. is a clinical stage biotechnology company that is developing immune-modulating therapies for the management and treatment of life-threatening conditions. The Company’s lead investigational product is tegoprubart, an anti-CD40L antibody with high affinity for the CD40 Ligand, a well-validated biological target that has broad therapeutic potential. The central role of CD40L signaling in both adaptive and innate immune cell activation and function positions it as an attractive target for non-lymphocyte depleting, immunomodulatory therapeutic intervention. The Company is building upon a deep historical knowledge of anti-CD40 Ligand biology to conduct preclinical and clinical studies in kidney allograft transplantation, xenotransplantation, and amyotrophic lateral sclerosis (ALS). Eledon is headquartered in Irvine, California. For more information, please visit the Company’s website at www.eledon.com. Follow Eledon Pharmaceuticals on social media: LinkedIn; Twitter Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. Any statements about the company’s future expectations, plans and prospects, including statements about planned clinical trials, the development of product candidates, expected timing for initiation of future clinical trials, expected timing for receipt of data from clinical trials, the company’s capital resources and ability to finance planned clinical trials, as well as other statements containing the words “believes,” “anticipates,” “plans,” “expects,” “estimates,” “intends,” “predicts,” “projects,” “targets,” “looks forward,” “could,” “may,” and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are inherently uncertain and are subject to numerous risks and uncertainties, including: risks relating to the safety and efficacy of our drug candidates; risks relating to clinical development timelines, including interactions with regulators and clinical sides, as well as patient enrollment; risks relating to costs of clinical trials and the sufficiency of the company’s capital resources to fund planned clinical trials; and risks associated with the impact of the ongoing coronavirus pandemic. Actual results may differ materially from those indicated by such forward-looking statements as a result of various factors. These risks and uncertainties, as well as other risks and uncertainties that could cause the company’s actual results to differ significantly from the forward-looking statements contained herein, are discussed in our quarterly 10-Q, annual 10-K, and other filings with the U.S. Securities and Exchange Commission, which can be found at www.sec.gov. Any forward-looking statements contained in this press release speak only as of the date hereof and not of any future date, and the company expressly disclaims any intent to update any forward-looking statements, whether as a result of new information, future events or otherwise. Investor Contact: Stephen Jasper Gilmartin Group (858) 525 2047 stephen@gilmartinir.com Media Contact: Jenna Urban CG Life (212) 253 8881 jurban@cglife.com Source: Eledon Pharmaceuticals Source: Eledon Pharmaceuticals, Inc.

MENLO PARK, Calif.--( BUSINESS WIRE )--Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today presented positive long-term data on its lead investigational allogeneic T-cell immunotherapy, Orca-T, at the 66th American Society of Hematology (ASH) Annual Meeting. Three-Year Survival Data with Orca-T vs PTCy The results of a three-year follow-up analysis with Orca-T plus single-agent tacrolimus (TAC) showed encouraging overall survival (OS) in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL) and high-risk myelodysplastic syndrome (MDS). These results also showed an improvement when compared to a historical cohort of patients receiving an allogeneic stem cell transplant (alloHSCT) with post-transplant cyclophosphamide (PTCy)-based graft versus host disease (GvHD) prophylaxis. “With the current standard of care, physicians must carefully balance the risk of relapse with the risk of toxicities when treating patients with serious hematological malignancies, as both impact overall survival,” said presenting author Caspian Oliai, MD, medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center. “The preliminary results with Orca-T have been promising, and now observational outcomes show sustained improvement in comparison to a similar cohort of patients who received a conventional alloHSCT with PTCy at one, two and three years. These data add to the growing body of evidence suggesting Orca-T could potentially offer an important new treatment option for patients.” This analysis from the multicenter Phase 1b clinical trial evaluated a subset of patients (n=77) who received Orca-T with TAC. These outcomes were compared to patients from a CIBMTR literature-based cohort (n=293) who received alloHSCT with a PTCy-based GvHD prophylaxis regimen. All patients across both groups received myeloablative conditioning (MAC) and used a related or unrelated fully matched donor. These subsets of patients were selected based on disease, disease status and conditioning regimens that most closely resemble the patients enrolled in the ongoing pivotal Phase 3 clinical trial evaluating Orca-T. The analysis included patients who had a median follow-up time of 33 months (range 5-54 months) and 24 months (range 0-53 months) in the Orca-T Phase 1b and PTCy cohorts, respectively. The OS at 12, 24 and 36 months (p=0.0021) are summarized in the table below: Follow-up Phase 1b Orca-T plus TAC OS AlloHSCT with PTCy OS 1 year 96% (range 88%-99%; 95% CI) 82% (range 78%-87%; 95% CI) 2 year 88% (range 78%-94%; 95% CI) 73% (range 68%-79%; 95% CI) 3 year 86% (range 73-92%; 95% CI) 67% (range 61%-74%; 95% CI) Further analyses of these patient subsets found Orca-T may reduce non-relapse mortality (NRM) and increase relapse-free survival (RFS) compared to PTCy. At one year, NRM was 1.4% in the Orca-T group and 7.4% in the PTCy cohort. The rate of RFS at one year was 83% and 71% for patients receiving Orca-T and PTCy, respectively. Results also found that age had no significant impact on OS at one year. For patients receiving Orca-T, OS was 95% (88%-100%) in patients under the age of 50 and 97% (92%-100%) in patients over the age of 50. For patients in the PTCy cohort, OS was 86% (81%-92%) in patients under the age of 50 and 77% (70%-85%) in patients over the age of 50. Across all patients in the Phase 1b trial, Orca-T was manufactured reliably and delivered with vein-to-vein times of 72 hours or less across the U.S. “We’re pleased to present positive long-term follow-up data for Orca-T as these findings provide valuable insight into how the cell therapy performs over extended periods of time,” said Scott McClellan, MD, PhD, chief medical officer at Orca Bio. “As we eagerly await the results of our pivotal Phase 3 clinical trial evaluating Orca-T in a similar patient population, we are one step closer to gathering the information needed for BLA submission and toward potentially making a significant difference for patients.” Combining Orca Bio Approach with CAR-T Technology A second oral presentation highlighted initial feasibility, safety and efficacy data from an investigator-sponsored Phase 1 clinical trial evaluating OrCAR-T, a platform that combines the Orca Bio technology with CAR-T technology. This presentation highlighted data following treatment with Orca-T and allogeneic CD19/CD22 CAR-T cells in patients with high-risk B-cell acute lymphoblastic leukemia (B-ALL). B-ALL remains incurable for many patients despite treatment with an alloHSCT or autologous CAR-T cells, which can also lead to serious complications including GvHD, infections and CAR-related toxicities. Preliminary results for 10 patients treated with OrCAR-T who reached the primary endpoint of 42 days post-treatment showed neutrophil and platelet engraftment at 14 days. At a median follow up of 381 days (range 61-762 days, including 6 patients with >12 months of follow-up) across three dose escalation levels (1x10 6 CAR+ cells/kg; 2x10 6 CAR+ cells/kg; 3x10 6 CAR+ cells/kg), there were no cases of acute or chronic GvHD, no grade 3 or greater cytokine release syndrome and no neurotoxicity observed. These early findings suggest a single treatment that combines a high-precision cell therapy like Orca-T with specific CAR-T cells has the potential to benefit patients with a range of hematologic diseases. About Orca-T Orca-T is an investigational allogeneic T-cell immunotherapy being evaluated in clinical trials for the treatment of multiple hematologic malignancies. Orca-T is comprised of highly purified regulatory T-cells, CD34+ stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors. Orca-T is currently being evaluated in Precision-T, a pivotal Phase 3 clinical trial, which has completed enrollment at leading transplant centers across the U.S. Orca-T has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration. About Orca Bio Orca Bio is a late-stage biotechnology company developing high-precision cell therapies for the treatment of cancer and autoimmune diseases. Our investigational products are designed to replace a patient’s diseased blood and immune system with a healthy one, with the goal of improving outcomes with fewer risks than the standard of care. Our manufacturing platform uses single-cell precision to create proprietary, uniquely-defined products that have the potential to transform allogeneic cell therapy. At Orca Bio, our mission is to make curative cell therapies both more effective and safer, and in doing so, push past the field’s current boundaries and redefine its future. For more information, visit www.orcabio.com .