Tegoprubart

Enrolled 12 participants in Phase 2 BESTOW trial evaluating tegoprubart for the prevention of kidney rejection Tegoprubart used as a component of the immunosuppressive treatment regimen following the first-ever transplant of a kidney from a genetically modified pig to a human Additional data from 11 participants in Phase 1b trial in kidney transplantation demonstrated that tegoprubart was generally safe and well tolerated, successfully prevented rejection and permitted above historical average post-transplant kidney function IRVINE, Calif., March 28, 2024 (GLOBE NEWSWIRE) -- Eledon Pharmaceuticals Inc. (“Eledon”) (Nasdaq: ELDN) today reported its fourth quarter and full year 2023 operating and financial results and reviewed recent business highlights. “Eledon continues to execute on time and as promised towards our goal of extending the functional life of transplanted organs,” said David-Alexandre C. Gros, M.D., Chief Executive Officer of Eledon. “Our latest Phase 1b data further support the potential of tegoprubart to significantly reduce the risk of rejection as well as harmful side effects associated with current standard of care in immunosuppression. In addition to our clinical development progress, tegoprubart was used for immunosuppression in historical kidney and heart pig-to-human xenotransplant procedures. As we advance our clinical program to assess the use of tegoprubart in kidney transplant procedures, we remain committed to supporting groundbreaking advancements that can lead to broader organ availability for patients around the world.” “Looking ahead, we anticipate completing enrollment of our Phase 2 BESTOW study by the end of the year as we also continue to enroll participants in the second cohort of our Phase 1b study and open-label extension study designed to provide additional insights into tegoprubart’s long-term effectiveness,” continued Dr. Gros. “We look forward to providing updated interim clinical data from both the Phase 1b and open-label studies in the second quarter of this year.” Fourth Quarter 2023 and Recent Corporate Developments Announced the use of tegoprubart as a component of the immunosuppressive treatment regimen following the first-ever transplant of a kidney from a genetically modified pig to a human. The procedure was completed on March 16, 2024, at Massachusetts General Hospital on a 62-year-old man with end-stage kidney disease. Announced 12th participant enrolled in the Phase 2 BESTOW trial assessing tegoprubart head-to-head with tacrolimus for the prevention of rejection in kidney transplantation. Reported updated safety and efficacy data from the ongoing Phase 1b open-label trial evaluating tegoprubart for the prevention of rejection in patients undergoing kidney transplant. Data from 11 participants demonstrated that tegoprubart was generally safe and well-tolerated in patients undergoing kidney transplantation, with aggregate mean estimated glomerular filtration rate (eGFR) above 70 mL/min/1.73m² at all reported time points after 90 days post-transplant. Results were presented at the American Society of Nephrology Kidney Week 2023 Annual Meeting held in Philadelphia, PA in November 2023. Amended Phase 1b trial protocol to add a second cohort, now allowing enrollment of up to 24 trial participants who are undergoing kidney transplantation. Enrolled first patient in a Phase 2 open-label extension (OLE) study, which will evaluate the long-term safety, pharmacokinetics, and efficacy of tegoprubart in participants who have completed one year of treatment in either the ongoing Phase 1b or Phase 2 BESTOW study. The participant completed the Phase 1b study with an eGFR of 91 at one year (day 374). Partnered with the University of Chicago Transplantation Institute to secure financing from the Juvenile Diabetes Research Foundation (JDRF) and The Cure Alliance to fund an investigator sponsored study in pancreatic islet cell transplantation in participants with type 1 diabetes. Tegoprubart treatment will be evaluated for the prevention of transplant rejection. Strengthened leadership team with appointment of Eliezer Katz, M.D., FACS as Chief Medical Officer and strengthened board of directors with appointment of Allan Kirk, M.D., Ph.D. and James Robinson. Anticipated 2024 Milestones Second quarter 2024: Report updated interim clinical data from the ongoing Phase 1b trial of tegoprubart in kidney transplantation. End of 2024: Complete enrollment in the Phase 2 BESTOW trial of tegoprubart in kidney transplantation. 2024: Dose the first islet cell transplant participant for the treatment of type 1 diabetes at the University of Chicago Transplantation Institute. Fourth Quarter 2023 Financial Results The Company reported a net loss of $9.6 million, or $0.32 per share, for the three months ended December 31, 2023, compared to a net loss of $58.4 million, or $4.09 per share, for the same period in 2022. The net loss for the three months ended December 31, 2022 includes a non-cash goodwill impairment charge totaling $48.6 million. Excluding the non-cash impairment charge, net loss would be $9.7 million, or $0.68 per share. Research and development expenses were $7.1 million for the three months ended December 31, 2023, compared to $7.3 million for the comparable period in 2022, a decrease of $0.2 million. General and administrative expenses were $3.3 million for the three months ended December 31, 2023, compared to $2.8 million for the comparable period in 2022, an increase of $0.5 million. Full Year 2023 Financial Results The Company reported a net loss of $40.3 million, or $1.64 per share, for the year ended December 31, 2023, compared to a net loss of $88.0 million, or $6.16 per share, in 2022. The net loss for the year ended December 31, 2022 includes a non-cash goodwill impairment charge totaling $48.6 million. Excluding the non-cash impairment charge, net loss would be $39.3 million, or $2.75 per share. Research and development expenses were $30.3 million for the year ended December 31, 2023, compared to $27.1 million for the year ended December 31, 2022, an increase of $3.2 million. General and administrative expenses were $12.7 million for the year ended December 31, 2023, compared to $12.7 million for the year ended December 31, 2022. The Company ended the year with approximately $51.1 million in cash and cash equivalents and short-term investments, compared to $56.4 million in cash and cash equivalents as of December 31, 2022. 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 CD40 Ligand, a well-validated biological target within the costimulatory CD40/CD40L cellular pathway. 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 . 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-Qs, annual 10-K, and other filings with the U.S. Securities and Exchange Commission, which can be found at . 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 Berry & Company Public Relations (212) 253 8881 jurban@berrypr.com Source: Eledon Pharmaceuticals ELEDON PHARMACEUTICALS, INC. CONSOLIDATED BALANCE SHEETS (In thousands, except share and per share data) December 31, 2023 2022 ASSETS Current assets: Cash and cash equivalents $ 4,612 $ 56,409 Short-term investments 46,490 — Prepaid expenses and other current assets 5,027 3,109 Total current assets 56,129 59,518 Operating lease asset, net 365 739 In-process research and development 32,386 32,386 Other assets 186 150 Total assets $ 89,066 $ 92,793 LIABILITIES AND STOCKHOLDERS’ EQUITY Current liabilities: Accounts payable $ 967 $ 2,200 Current operating lease liability 383 363 Accrued expenses and other liabilities 2,545 3,912 Total current liabilities 3,895 6,475 Deferred tax liability 1,752 1,752 Non-current operating lease liability — 383 Total liabilities 5,647 8,610 Commitments and contingencies Stockholders’ equity: Preferred stock, $0.001 par value, 5,000,000 shares authorized at December 31, 2023 and 2022: Series X¹ non-voting convertible preferred stock, $0.001 par value, 515,000 shares designated; 110,086 and 117,970 shares issued and outstanding at December 31, 2023 and 2022, respectively — — Series X non-voting convertible preferred stock, $0.001 par value, 10,000 shares designated; 4,422 and 6,204 shares issued and outstanding at December 31, 2023 and 2022, respectively — — Common stock, $0.001 par value, 200,000,000 shares authorized at December 31, 2023 and 2022; 24,213,130 and 13,776,788 shares issued and outstanding at December 31, 2023 and 2022, respectively 24 14 Additional paid-in capital 326,586 287,034 Accumulated deficit (243,191 ) (202,865 ) Total stockholders’ equity 83,419 84,183 Total liabilities and stockholders’ equity $ 89,066 $ 92,793 ELEDON PHARMACEUTICALS, INC. CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (In thousands, except share and per share data) Year Ended December 31, 2023 2022 Operating expenses Research and development $ 30,312 $ 27,080 General and administrative 12,688 12,700 Goodwill impairment — 48,648 Total operating expenses 43,000 88,428 Loss from operations (43,000 ) (88,428 ) Other income, net 2,674 462 Loss before income taxes (40,326 ) (87,966 ) Income taxes — — Net loss and comprehensive loss $ (40,326 ) $ (87,966 ) Net loss per share, basic and diluted $ (1.64 ) $ (6.16 ) Weighted-average common shares outstanding, basic and diluted 24,619,197 14,285,254

Pictured: Illustration of gloved hands using tools to manipulate a DNA double helix/iStock, MicrovOne In a major medical milestone, Massachusetts General Hospital announced Thursday the world’s first successful transplant of a genetically-edited pig kidney into a 62-year-old man with end-stage kidney disease. Eledon Pharmaceuticals’ tegoprubart, an investigational anti-CD40L antibody, was used following Saturday’s surgical procedure to suppress the patient’s immune system and prevent his body from rejecting the transplanted organ. The Irvine, Calif.-based clinical-stage biotech is developing tegoprubart to protect transplanted organs and help reduce the risk of organ rejection. Eledon’s stock rose over 12% on Thursday after the announcement. Mass General and a biotech company called eGenesis, which provided the pig kidney, have been researching this form of transplantation—called xenotransplantation—for five years and published their findings in Nature in October 2023. In Thursday’s announcement, eGenesis said that without genetic modification, a pig kidney “would be immediately rejected by a human recipient” and credited an immunosuppression regimen that included Eledon’s “novel, investigational monoclonal antibody, tegoprubart, targeting the co-stimulatory CD40L pathway” for the success of the historic transplant. According to Eledon, tegoprubart has been used investigationally to prevent rejection in both kidney and heart pig-to-human xenotransplantations, as well as in human-to-human kidney transplantation. “Eledon has now participated in both heart and kidney xenotransplant procedures, further demonstrating tegoprubart’s broad potential in transplant,” David-Alexandre Gros said in a statement,” adding that the company is working towards its “goal of developing tegoprubart as a new and better immunosuppressive option for transplant patients.” Gros told BioSpace the biotech has been working with eGenesis to provide them with tegoprubart across their xenotransplantation efforts, including transplantation in non-human primates and humans. Saturday’s xenotransplantation was authorized by the FDA under the Expanded Access pathway. Currently, tegoprubart is being evaluated in two clinical trials for kidney allotransplantation, in which an organ from one individual is transplanted to another of the same species with a different genotype, eGenesis said in its announcement. Founded in 2004, Eledon has made tegoprubart and its multiple mechanisms of action the cornerstone of the company’s therapeutic approach to help effectively manage immune response. The antibody targets and blocks the CD40L which, according to Eledon Chief Science Officer Steven Perrin, was a receptor discovered back in the 1980s that is essential to controlling the activation of T cells and B cells to slow down disease progression. When CD40L is expressed on activated T cells, it can “talk” to its receptor, a protein on the surface of an antigen-expressing cell, which generates the immune response, Perrin told BioSpace. “The activation of this pathway is critical to eliminating viral infections and other things. But, unfortunately, it can become activated in autoimmunity when this pathway recognizes self, and it will start to destroy self and diseases like lupus or rheumatoid arthritis where the immune system thinks your joints are foreign and starts to destroy them,” Perrin said. The current standard of care when it comes to preventing a transplanted organ from being rejected is a multitude of drug cocktails and therapies, but the mainstay is a drug called tacrolimus. While tacrolimus is good at avoiding failure, the problem is that it is very toxic from the start of administration, mainly affecting the cardiovascular system, according to Perrin. Gros said that the company is looking to remove the toxicity of tacrolimus and to have a potent enough drug for humans to survive xenotransplantation, as the current treatments are not powerful enough. Perrin added that in Mass General’s announcement on Thursday the hospital noted that the standard of care lasts from one minute to five minutes in xenotransplantation before turning black and being rejected by the body. Eledon is focused on developing tegoprubart to become the standard of care across all transplantation types, including human and non-human organ sources. Gros added that the company will provide more data on a Phase Ib study in kidney transplantation in June 2024 at the American Transplant Congress. In addition, he said Eledon’s Phase II study—which puts tegoprubart head-to-head against tacrolimus—has finished enrollment and early data is expected in 2026. The company is also looking to use its anti-CD40L approach to target amyotrophic lateral sclerosis. Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.