Longeveron(LGVN) - 2025 Q2 - Quarterly Results

Company Overview and Business Update Introduction and CEO Statement Longeveron Inc. reported Q2 2025 financial results and business updates, with CEO Wa'el Hashad highlighting positive stem cell therapy progress, productive FDA communications for HLHS and Alzheimer's, and IND approval for pediatric DCM, preparing for HLHS BLA submission by late 2026 - Longeveron Inc. is a clinical-stage regenerative medicine biotechnology company focused on developing cell therapies for life-threatening rare pediatric and chronic aging-related diseases[2] - CEO Wa'el Hashad stated that its stem cell therapy, Laromestrocel, showed positive preliminary results across five clinical trials for three indications[3] - The company held positive, clear-path meetings with the FDA for HLHS and Alzheimer's development programs and received IND approval for pediatric dilated cardiomyopathy, allowing direct entry into pivotal clinical trials[3] - The pivotal Phase 2b clinical trial for HLHS has completed patient enrollment, with top-line results expected in 2026 and a BLA submission planned for late 2026[3] Development Programs Update Longeveron's Laromestrocel (Lomecel-B) is being evaluated across multiple indications, with HLHS Phase 2b enrollment complete and BLA planned for late 2026, Alzheimer's showing potential with FDA agreement on Phase 2/3 design, and pediatric DCM receiving IND approval for a pivotal Phase 2 trial expected to start in H1 2026 - Longeveron's investigational therapeutic candidate is Laromestrocel (Lomecel-B), a proprietary, scalable, allogeneic cell therapy[4] Hypoplastic Left Heart Syndrome (HLHS) The HLHS program has completed enrollment for its pivotal Phase 2b clinical trial, with top-line results expected in Q3 2026 and a BLA submission planned for late 2026 if results are positive - The pivotal Phase 2b clinical trial for HLHS (ELPIS II) completed enrollment of all 40 pediatric patients in June 2025[5][6] - Top-line results from ELPIS II are anticipated in the third quarter of 2026[5][6] - If ELPIS II results are positive, a Biologics License Application (BLA) for Laromestrocel in HLHS is expected to be submitted in late 2026[5][6] - The FDA confirmed ELPIS II as a pivotal trial, potentially supporting a BLA submission for HLHS if sufficient evidence of efficacy is demonstrated[6] Alzheimer's Disease (AD) The Alzheimer's disease program showed therapeutic potential in Phase 2a, with FDA agreement on a pivotal Phase 2/3 trial design, and the company is seeking strategic partnerships - Results from the Phase 2a clinical trial (CLEAR MIND) were published in Nature Medicine in March 2025, supporting Laromestrocel's therapeutic potential in treating mild Alzheimer's disease[10] - A positive Type B meeting was held with the FDA in March 2025 regarding the Alzheimer's BLA submission pathway, with agreement on the design, population, and endpoints for a proposed single pivotal Phase 2/3 clinical trial[10] - The FDA granted Laromestrocel Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations for the treatment of mild Alzheimer's disease[10] - The company is seeking strategic collaborations and/or partnerships to advance Laromestrocel in treating AD[10] Pediatric Dilated Cardiomyopathy (DCM) The pediatric DCM program received FDA IND approval for direct entry into a pivotal Phase 2 clinical trial, anticipated to start in H1 2026 pending financing - In July 2025, the FDA approved Longeveron's IND application for its stem cell therapy, Laromestrocel, as a potential treatment for pediatric dilated cardiomyopathy (DCM)[10] - The approved IND application allows direct entry into a single pivotal Phase 2 registrational clinical trial[10] - The company currently anticipates initiating the pivotal Phase 2 clinical trial in the first half of 2026, subject to securing necessary financing[10] Corporate Milestones and Strategic Initiatives Longeveron achieved significant regulatory milestones, including multiple FDA designations for HLHS and RMAT/Fast Track for AD, while actively seeking AD strategic collaborations, securing an XPRIZE Healthspan award, appointing a Chief Business Officer, licensing new stem cell technology, and raising approximately $5 million through a public offering with potential additional funds from warrant exercises - ELPIS II builds upon positive clinical results from ELPIS I, where children treated with Laromestrocel achieved 100% transplant-free survival before five years of age[10] - The FDA granted Laromestrocel Orphan Drug, Fast Track, and Rare Pediatric Disease designations for the treatment of HLHS[10] - The company was selected as a semifinalist team for the XPRIZE Healthspan competition and received a $250,000 Milestone 1 award[10] - Than Powell was appointed Chief Business Officer, responsible for overall corporate business strategy, Alzheimer's program partnerships, and international strategy for the HLHS program[10] - The company licensed a new, composition-of-matter patented stem cell technology from the University of Miami with broad cardiac indications[10] - The company raised approximately $5 million through a public offering, with potential for an additional $12.5 million in gross proceeds from the full exercise of short-term warrants[10] Second Quarter 2025 Financial Results Summary Financial Performance (Six Months Ended June 30, 2025) For the six months ended June 30, 2025, Longeveron's total revenue decreased by 31% to $0.7 million, gross profit fell by 37% to $0.4 million, while G&A and R&D expenses significantly increased by 28% and 39% respectively, leading to a 34% expansion in net loss to $10.0 million, partially offset by a $0.5 million increase in other income Summary Financial Performance (Six Months Ended June 30, 2025) | Metric | Six Months Ended June 30, 2025 (million dollars) | Six Months Ended June 30, 2024 (million dollars) | Change (million dollars) | Change (%) | | :----------------------- | :--------------------------------- | :--------------------------------- | :---------------- | :------- | | Revenue | 0.7 | 1.0 | (0.3) | -31% | | Cost of Revenue | 0.3 | 0.3 | 0.0 | 0% | | Gross Profit | 0.4 | 0.7 | (0.3) | -37% | | General and Administrative Expenses | 5.5 | 4.3 | 1.2 | 28% | | Research and Development Expenses | 5.5 | 3.9 | 1.6 | 39% | | Other Income, Net | 0.5 | 0.1 | 0.4 | 400% | | Net Loss | (10.0) | (7.5) | (2.5) | 34% | - The decrease in revenue was primarily due to reduced demand from Bahamian registered trial participants and lower demand for contract manufacturing services from third-party customers[11] - The increase in general and administrative expenses was mainly associated with higher personnel and related costs, including equity-based compensation, in 2025[11] - The increase in research and development expenses was primarily due to higher personnel and related costs, including equity-based compensation, to support CMC and manufacturing readiness activities, and increased patent cost amortization in 2025, partially offset by reduced clinical trial expenses due to the discontinuation of aging-related frailty clinical trial activities[11] Balance Sheet Highlights As of June 30, 2025, Longeveron's total assets decreased to $16,749 thousand from $25,558 thousand at December 31, 2024, with cash and cash equivalents falling to $10,334 thousand, while total liabilities slightly increased to $4,093 thousand, and total stockholders' equity declined to $12,656 thousand primarily due to increased accumulated deficit Balance Sheet Highlights | Metric (thousand dollars) | June 30, 2025 | December 31, 2024 | Change (thousand dollars) | | :-------------------- | :------------- | :------------- | :------------- | | Assets: | | | | | Cash and Cash Equivalents | 10,334 | 19,232 | (8,898) | | Total Current Assets | 11,269 | 19,624 | (8,355) | | Total Assets | 16,749 | 25,558 | (8,809) | | Liabilities: | | | | | Total Current Liabilities | 3,284 | 2,582 | 702 | | Total Long-Term Liabilities | 809 | 1,089 | (280) | | Total Liabilities | 4,093 | 3,671 | 422 | | Stockholders' Equity: | | | | | Accumulated Deficit | (119,646) | (109,607) | (10,039) | | Total Stockholders' Equity | 12,656 | 21,887 | (9,231) | - As of June 30, 2025, cash and cash equivalents significantly decreased to $10.334 million from $19.232 million at December 31, 2024[19] - As of June 30, 2025, total assets decreased to $16.749 million from $25.558 million at December 31, 2024[19] - As of June 30, 2025, the accumulated deficit increased to $119.646 million from $109.607 million at December 31, 2024[19] Statements of Operations Highlights For the three months ended June 30, 2025, total revenue was $316 thousand with a net loss of $5,028 thousand, while for the six months, total revenue decreased to $697 thousand and net loss expanded to $10,039 thousand, though basic and diluted net loss per share decreased to ($0.67) due to a significant increase in weighted average common shares outstanding Statements of Operations Highlights | Metric (thousand dollars) | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------- | :-------------------- | :-------------------- | :-------------------- | :-------------------- | | Total Revenue | 316 | 468 | 697 | 1,016 | | Gross Profit | 146 | 344 | 421 | 673 | | Total Operating Expenses | 5,543 | 3,844 | 10,999 | 8,263 | | Operating Loss | (5,397) | (3,500) | (10,578) | (7,590) | | Other Income, Net | 369 | 87 | 539 | 119 | | Net Loss | (5,028) | (3,413) | (10,039) | (7,471) | | Basic and Diluted Net Loss Per Share | (0.33) | (1.83) | (0.67) | (3.54) | | Weighted Average Common Shares Outstanding | 15,013,072 | 6,509,881 | 14,982,075 | 4,511,734 | - For the six months ended June 30, 2025, clinical trial revenue decreased from $0.802 million to $0.557 million, contract manufacturing lease revenue decreased from $0.191 million to $0.012 million, and contract manufacturing revenue increased from $0.023 million to $0.128 million[21] - For the six months ended June 30, 2025, operating loss expanded from $7.59 million to $10.578 million[21] - Basic and diluted net loss per share decreased from ($3.54) to ($0.67) for the six-month period, despite an increased net loss, due to a significant increase in weighted average common shares outstanding from 4,511,734 to 14,982,075[21] Cash Position and Financial Outlook As of June 30, 2025, the company held $10.3 million in cash and cash equivalents, with recent financing expected to support operations until Q1 2026, anticipating increased operating and capital expenditures for HLHS BLA preparation, and planning to seek additional financing, including non-dilutive funds and partnerships for the Alzheimer's program - As of June 30, 2025, cash and cash equivalents totaled $10.3 million[12] - The company anticipates that existing cash and cash equivalents will support operating expenses and capital expenditures through the first quarter of 2026[12] - Operating expenses and capital expenditures are projected to increase for the remainder of 2025 and throughout 2026, primarily for CMC (Chemistry, Manufacturing, and Controls) and manufacturing readiness to support the HLHS BLA submission[12] - The company plans to pursue additional financing opportunities, equity financing, and non-dilutive funding, with a focus on seeking collaboration opportunities and/or non-dilutive funding for the Alzheimer's disease program[12] Additional Information Conference Call Details Longeveron Inc. hosted a conference call and webcast on August 13, 2025, at 4:30 PM ET to discuss financial results and business updates, with details provided for dial-in, conference ID, webcast link, and an archived replay Conference Call Details | Item | Details | | :-------------------- | :-------------------- | | Conference Call Number | 1.877.407.0789 | | Conference ID | 13754295 | | Webcast | Accessible via link | - An archived replay of the webcast will be available in the "Events & Presentations" section of the company's website[13] About Longeveron Inc. Longeveron is a clinical-stage biotechnology company developing regenerative medicine products, with its lead candidate Laromestrocel (Lomecel-B™), an allogeneic mesenchymal stem cell therapy, demonstrating multiple potential mechanisms of action across four pipeline indications (HLHS, Alzheimer's, pediatric DCM, and aging-related frailty) and holding five key FDA designations - Longeveron is a clinical-stage biotechnology company developing regenerative medicine products to address unmet medical needs[14] - The company's lead investigational product is Laromestrocel (Lomecel-B™), an allogeneic mesenchymal stem cell (MSC) therapy product isolated from the bone marrow of young, healthy adult donors[14] - Laromestrocel possesses multiple potential mechanisms of action, including pro-angiogenic, pro-regenerative, anti-inflammatory, and tissue repair and healing effects[14] - The company is currently advancing four pipeline indications: Hypoplastic Left Heart Syndrome (HLHS), Alzheimer's disease, pediatric Dilated Cardiomyopathy (DCM), and Aging-related Frailty[14] - The Laromestrocel development program has received five important FDA designations, including Orphan Drug, Fast Track, and Rare Pediatric Disease designations for HLHS, and Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations for AD[14] Forward-Looking Statements This press release contains forward-looking statements under the Private Securities Litigation Reform Act of 1995, reflecting management's current expectations and estimates regarding future operations, performance, and economic conditions, which involve known and unknown risks, uncertainties, and other factors that could cause actual results to differ materially, and the company disclaims any obligation to update them - Certain statements in this press release that are not historical facts are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995[15] - Forward-looking statements involve known and unknown risks, uncertainties, and other important factors that could cause actual results, performance, or achievements to differ materially from expectations[15] - Factors that could cause actual results to differ materially from forward-looking statements include, but are not limited to: cash position and the need to raise additional capital, difficulties in financing, dilutive effects on investors, financial performance, ability to continue as a going concern, the period over which existing cash and cash equivalents will be sufficient to fund future operating expenses and capital expenditure requirements, the ability of clinical trials to demonstrate the safety and efficacy of product candidates, the timing and focus of clinical trials, the size of the market opportunity, the ability to scale manufacturing and commercialization, the success of competing therapies, the characteristics, safety, and efficacy of product candidates, the ability to obtain and maintain regulatory approvals, further development plans for product candidates, intellectual property protection, and the ability to recruit and retain personnel[15] - The company undertakes no obligation to update or revise any forward-looking statements, except as required by law[16]