Tacrolimus

Completed enrollment of Phase 2 BESTOW trial of tegoprubart in kidney transplantation four months ahead of schedule; on track to report topline results in fourth quarter of 2025 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 Announced oversubscribed $85 million underwritten offering, with proceeds expected to extend cash runway to the end of 2026 IRVINE, Calif., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Eledon Pharmaceuticals, Inc. (“Eledon”) (Nasdaq: ELDN) today reported its third quarter 2024 operating and financial results and reviewed recent business highlights. “We believe tegoprubart has best-in-class potential as a novel immunosuppressive treatment option to prevent transplant rejection, with promising clinical results across kidney, xenograft, and now islet transplantations," said David-Alexandre C. Gros, M.D., Chief Executive Officer of Eledon. “Recent encouraging data from the UChicago Medicine trial in type 1 diabetes, combined with our accelerated enrollment in the Phase 2 BESTOW trial and our strong capital position following our recent oversubscribed financing, has put us in a strong position to advance tegoprubart’s development. We look forward to sharing results from our BESTOW trial in the fourth quarter of 2025.” Recent Business Highlights Completed enrollment for the Phase 2 BESTOW clinical trial, which is designed to assess the safety and efficacy of tegoprubart for the prevention of organ rejection in patients undergoing kidney transplantation. The trial reached its target enrollment of 120 participants approximately four months earlier than originally planned.Announced positive initial data for the first three islet transplant recipients treated with tegoprubart as part of an immunosuppressive 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 Transplantation Institute. The first two subjects achieved insulin independence and remain insulin free, with glucose control in the normal range; the third subject was recently transplanted and remains on a trajectory to also achieve insulin independence. Treatment with tegoprubart was generally well tolerated in all subjects with no unexpected adverse events. The data demonstrated potentially the first human cases of insulin independence achieved using an anti-CD40L monoclonal antibody immunosuppressive therapy without the use of tacrolimus, the current standard of care for prevention of transplant rejection.Completed an oversubscribed, underwritten offering of common stock and pre-funded warrants for total gross proceeds of $85.0 million, or net proceeds of approximately $79.5 million after deducting underwriting discounts, commissions, and offering expenses. The offering, which priced at a premium, included participation from both new and existing leading healthcare investors. 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.2025: Report longer-term follow up from the investigator-led clinical trial with the UChicago Medicine Transplant Institute for pancreatic islet transplantation in subjects with type 1 diabetes. Third Quarter 2024 Financial Results Cash, cash equivalents and short-term investments totaled $78.2 million as of September 30, 2024, which does not include net proceeds of approximately $79.5 million received in the October 2024 underwritten offering. The company expects current cash, cash equivalents and short-term investments, together with the net proceeds from the October 2024 underwritten offering, to fund operations to the end of 2026. 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, an increase in employee compensation and benefits related to increased headcount, and greater chemistry, manufacturing and controls (CMC) expenses related to the production of clinical trial materials. General and administrative expenses for the third quarter of 2024 were $4.0 million, compared to $3.3 million for the comparable period in 2023, an increase of $0.7 million. The increase was primarily driven by a rise in professional services and an increase in general operating expenses. Net income for the third quarter of 2024 was $77.0 million, or $1.05 per basic share, compared with a net loss of $9.9 million, or $0.33 per basic share, for the comparable period in 2023, an increase of $86.9 million. The increase in net income was primarily driven by a non-cash gain of $96.4 million related to changes in the fair value of warrant liabilities and the fair value of financial instruments issued in excess of proceeds, recorded in the third quarter ending September 30, 2024. Excluding this gain, the company would have recorded a net loss of $19.5 million for the third quarter of 2024. 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 sites, 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 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 JasperGilmartin Group(858) 525 2047stephen@gilmartinir.com Media Contact: Jenna UrbanBerry & Company Public Relations(212) 253 8881jurban@berrypr.com Source: Eledon Pharmaceuticals ELEDON PHARMACEUTICALS, INC.CONDENSED CONSOLIDATED BALANCE SHEETS(In thousands, except share data)(Unaudited) September 30,2024 December 31,2023 (As Restated) ASSETS Current assets: Cash and cash equivalents$6,751 $4,612 Short-term investments 71,440 46,490 Prepaid expenses and other current assets 3,318 5,027 Total current assets 81,509 56,129 Operating lease asset, net 471 365 In-process research and development 32,386 32,386 Other assets 210 186 Total assets$114,576 $89,066 LIABILITIES AND STOCKHOLDERS’ EQUITY Current liabilities: Accounts payable$5,860 $967 Current operating lease liabilities 186 383 Accrued expenses and other liabilities 6,443 2,545 Total current liabilities 12,489 3,895 Deferred tax liabilities 1,752 1,752 Non-current operating lease liabilities 315 — Warrant liabilities 23,962 76,211 Total liabilities 38,518 81,858 Commitments and contingencies Stockholders’ equity: Preferred stock, $0.001 par value, 5,000,000 shares authorized at September 30,2024 and December 31, 2023: Series X1non-voting convertible preferred stock, $0.001 par value,515,000 shares designated; 110,086 shares issued and outstandingat September 30, 2024 and December 31, 2023 — — Series X non-voting convertible preferred stock, $0.001 par value,10,000 shares designated; 4,422 shares issued and outstandingat September 30, 2024 and December 31, 2023 — — Common stock, $0.001 par value, 200,000,000 shares authorized at September 30, 2024 and December 31, 2023; 41,183,102 and 24,213,130 shares issued and outstanding at September 30, 2024 and December 31, 2023, respectively 41 24 Additional paid-in capital 386,884 326,586 Accumulated other comprehensive income 102 — Accumulated deficit (310,969) (319,402)Total stockholders’ equity 76,058 7,208 Total liabilities and stockholders’ equity$114,576 $89,066 ELEDON PHARMACEUTICALS, INC.CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS(In thousands, except share and per share data)(Unaudited) For the Three MonthsEnded September 30, For the Nine MonthsEnded September 30, 2024 2023 2024 2023 (As Restated) (As Restated) Operating expenses Research and development$16,520 $7,931 $34,036 $23,245 General and administrative 3,990 3,267 11,845 9,417 Total operating expenses 20,510 11,198 45,881 32,662 Loss from operations (20,510) (11,198) (45,881) (32,662)Other income, net 1,042 849 2,485 1,962 Change in fair value of warrant liabilities and fair value of financial instruments issued in excess of proceeds 96,439 443 51,829 (55,738)Net income (loss)$76,971 $(9,906) $8,433 $(86,438) Net income (loss) attributable to common shares - basic$54,429 $(9,906) $5,551 $(86,438)Basic net income (loss) per common share$1.05 $(0.33) $0.13 $(3.79)Weighted-average number of shares outstanding - basic 51,945,920 29,974,400 41,443,049 22,813,085 Net income (loss) attributable to common shares - diluted$(17,504) $(9,906) $(61,086) $(86,438)Basic net income (loss) per common share$(0.32) $(0.33) $(1.42) $(3.79)Weighted-average number of shares outstanding - diluted 55,478,342 29,974,400 43,106,746 22,813,085

PITTSBURGH, Nov. 12, 2024 (GLOBE NEWSWIRE) -- Lipella Pharmaceuticals Inc. (Nasdaq: LIPO), a clinical-stage biotechnology company focused on developing innovative therapies for serious diseases with unmet medical needs, today announced that U.S. Patent No. 12,138,345 for its proprietary liposomal drug delivery platform will be officially issued by the U.S. Patent and Trademark Office (USPTO) on November 12, 2024. This patent, titled "Delivery of Agents Using Metastable Liposomes," covers key technological innovations that enable the targeted delivery of therapeutic agents using liposome-based vehicles. The patent’s claims cover Lipella’s method of using metastable liposomes to deliver therapeutic agents, providing broad intellectual property protection for the company’s drug delivery platform. This protection extends market exclusivity for Lipella’s two lead clinical assets, LP-10 and LP-310, which are currently in Phase 2 clinical trials. LP-10, a liposomal formulation of tacrolimus for treating hemorrhagic cystitis, and LP-310, an oral rinse for managing oral lichen planus, represent promising new approaches to addressing diseases with limited treatment options. “We believe our proprietary liposomal drug delivery platform holds significant potential to enhance efficacy and tolerability across various therapeutic areas, including oncology, cancer survivorship and immunotherapy. This platform offers a safer, more effective way to administer therapeutics like tacrolimus, providing patients with targeted treatment options that minimize side effects,” said Dr. Michael Chancellor, Chief Medical Officer of Lipella Pharmaceuticals. “The official issuance of this strategically important patent marks a pivotal advancement for Lipella, strengthening our intellectual property portfolio and supporting our commitment to advancing treatments for critical conditions such as hemorrhagic cystitis and oral lichen planus.” With additional patents in the United States, Australia and Canada extending exclusivity until 2035, Lipella Pharmaceuticals is committed to providing value as it advances its clinical pipeline and explores new therapeutic applications. About Lipella’s Lead Clinical Assets: LP-10 and LP-310 LP-10 is a liposomal formulation of tacrolimus designed for intravesical administration to treat hemorrhagic cystitis (HC), a rare but severe condition characterized by bleeding from the bladder. LP-10 has shown promising safety and efficacy results in a multicenter Phase 2a trial, where it improved urinary symptoms in patients. The FDA has granted Orphan Drug Designation to LP-10 for the treatment of moderate to severe HC, further underscoring its potential to address this critical unmet need. A Phase 2b multicenter placebo-controlled trial is ready to begin.LP-310 is an innovative oral rinse formulation of LP-10, designed to treat oral lichen planus (OLP), a chronic autoimmune disease affecting the mucous membranes of the mouth. LP-310 offers a promising new approach to treating OLP, which affects millions of Americans and currently lacks an approved pharmacotherapy. A Phase 2a multicenter trial is underway with anticipated top-line data by year-end and trial completion by mid-2025. To find out more about our clinical trial visit: https://lipella.com/oral-lichen-planus-treatment/ About Lipella Pharmaceuticals Inc.Lipella Pharmaceuticals is a clinical-stage biotechnology company focused on developing new drugs by reformulating active agents in existing generic drugs and optimizing these reformulations for new applications. Lipella targets diseases with significant unmet needs, where no approved drug therapies currently exist. The company completed its initial public offering in December 2022. Learn more at lipella.com and follow us on X and LinkedIn. CONTACTJeff RamsonPCG Advisoryjramson@pcgadvisory.com646-863-6893