Core Viewpoint - Longeveron Inc. is advancing its clinical programs, particularly laromestrocel, with significant progress in treating life-threatening pediatric and chronic conditions, aiming for pivotal trial results in 2026 and potential FDA approval thereafter [2][10]. Development Programs Update - Longeveron's lead investigational product, laromestrocel (Lomecel-B), is being evaluated for multiple indications, including Hypoplastic Left Heart Syndrome (HLHS), Alzheimer's disease, and Pediatric Dilated Cardiomyopathy (DCM) [3][4][5]. - The pivotal Phase 2b clinical trial (ELPIS II) for HLHS is fully enrolled with 40 pediatric patients, and top-line results are expected in Q3 2026 [9][10]. - Laromestrocel has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the FDA for HLHS, and RMAT and Fast Track designations for Alzheimer's disease [10][15]. Financial Results Summary - For the nine months ended September 30, 2025, revenues decreased by 53% to $0.8 million compared to $1.8 million in 2024, primarily due to reduced demand for clinical trials and contract manufacturing services [14]. - General and administrative expenses increased by 22% to approximately $9.1 million, while research and development expenses rose by 52% to approximately $9.3 million [14]. - The net loss for the nine months ended September 30, 2025, increased to approximately $17.3 million, up from $11.9 million in the same period in 2024 [14][22]. Corporate Updates - The company appointed Than Powell as Interim CEO and announced key leadership changes, including Dr. Joshua Hare as Executive Chairman [14]. - Longeveron completed a public offering in August 2025, raising approximately $5.0 million, with potential additional proceeds of up to $12.5 million [14]. - The company is seeking strategic collaborations and partnerships to advance laromestrocel in treating Alzheimer's disease [10][14].

Financial Performance - As of September 30, 2025, the company had an accumulated deficit of $126.9 million and expects to continue generating operating losses for the foreseeable future[127]. - Revenues for the three months ended September 30, 2025, were $0.1 million, a decrease of $0.7 million, or 82%, compared to $0.8 million in 2024, primarily due to reduced demand for contract manufacturing services[155]. - For the nine months ended September 30, 2025, revenues were $0.8 million, a decrease of $1.0 million, or 53%, compared to $1.8 million in 2024, driven by decreased participant demand for the Bahamas Registry Trial[162]. - Net loss for the three months ended September 30, 2025, increased to approximately $7.2 million, up $2.8 million, or 63%, from a net loss of $4.4 million in 2024[161]. - General and administrative expenses increased to approximately $3.6 million for the three months ended September 30, 2025, up $0.5 million, or 15%, from $3.1 million in 2024[158]. - Research and development expenses rose to approximately $3.9 million for the three months ended September 30, 2025, an increase of $1.7 million, or 75%, compared to $2.2 million in 2024[159]. - General and administrative expenses for the nine months ended September 30, 2025, increased to approximately $9.1 million, up $1.7 million, or 22%, from $7.4 million in 2024[165]. - Research and development expenses for the nine months ended September 30, 2025, increased to approximately $9.3 million, an increase of $3.2 million, or 52%, compared to $6.1 million in 2024[166]. - Net cash used in operating activities for the nine months ended September 30, 2025, was $13.3 million, compared to $10.5 million in 2024[169]. - Cash and cash equivalents as of September 30, 2025, were $9.2 million, with working capital of approximately $5.4 million[173]. - The company anticipates that existing cash and cash equivalents will fund operating expenses into the first quarter of 2026 based on current forecasts[174]. Funding and Financing - The company raised approximately $5.0 million from a public offering of 5,882,354 shares of Class A common stock at a price of $0.85 per share on August 11, 2025[125]. - The company has access to an At The Market Offering Agreement with an aggregate market value of $10.7 million for the sale of Class A common stock[125]. - The company plans to seek additional financing through capital raises and non-dilutive funding options to support its operational needs[127]. - The company has been awarded approximately $11.5 million in governmental and non-profit association grants since 2016, which have funded clinical trials and research[183]. - The company has entered into an At The Market Offering Agreement with a market value of $10.7 million for the sale of Class A common stock[180]. - The company has no credit facility or committed sources of capital, which may limit its operational flexibility[188]. - The company may need to raise additional funds through equity offerings or other means to meet operational needs and capital requirements[187]. Clinical Trials and Research - The company is currently conducting a Phase 2b clinical trial (ELPIS II) for laromestrocel, targeting Hypoplastic Left Heart Syndrome (HLHS) with a total of 40 subjects enrolled[132]. - The ELPIS II trial is ongoing, with full enrollment achieved, and top-line results expected in Q3 2026, potentially leading to a BLA filing in 2027 if successful[145]. - The CLEAR MIND trial for mild Alzheimer's disease (AD) showed laromestrocel's safety profile was well tolerated, with significant results in secondary efficacy endpoints, including a 48% reduction in whole brain volume loss and a 62% reduction in hippocampal volume loss compared to placebo[137]. - The FDA granted RMAT and Fast Track designations to laromestrocel for mild AD in July 2024, marking it as a unique investigational product candidate[138]. - The FDA approved the IND application for laromestrocel as a potential treatment for pediatric Dilated Cardiomyopathy (DCM) in July 2025, allowing for a Phase 2 pivotal trial in 2026[144]. - The company has discontinued its clinical trial in Japan for aging-related frailty to focus on HLHS and AD, reflecting a strategic shift in clinical priorities[145]. - The company has initiated enhancements to its Chemistry, Manufacturing and Controls (CMC) infrastructure to support future Biologics License Application (BLA) submissions[121]. - The company has filed patent applications for laromestrocel in multiple countries, including the U.S., Canada, and Japan, to protect its intellectual property[135][140]. Operational Strategy - The company has not yet achieved profitable operations and relies on grants and contract manufacturing for revenue generation[127]. - Research and development expenses are expected to remain significant as the company increases headcount and clinical trial activities[151]. - The company plans to expand manufacturing capabilities to achieve cost-effective production for clinical trials and CDMO obligations[145]. - General and administrative expenses primarily consist of salaries, equity-based compensation, and professional fees, indicating a focus on operational efficiency[152]. - The ongoing U.S. federal government shutdown may delay regulatory communications and affect the company's clinical development programs[122]. - The company intends to pursue regulatory approval for product candidates, including a potential BLA filing with the FDA in 2027 for HLHS[178].

Core Viewpoint - Longeveron Inc. is set to report its third quarter 2025 financial results and provide a business update on November 4, 2025, after market close, followed by a conference call and webcast [1]. Company Overview - Longeveron Inc. is a clinical stage biotechnology company focused on developing regenerative medicines to meet unmet medical needs [3]. - The company's lead investigational product is laromestrocel (Lomecel-B™), an allogeneic mesenchymal stem cell therapy derived from the bone marrow of young, healthy adult donors [3]. - Laromestrocel has multiple potential mechanisms of action, including pro-vascular, pro-regenerative, anti-inflammatory, and tissue repair effects, with applications across various disease areas [3]. - Longeveron is pursuing four pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer's disease, Pediatric Dilated Cardiomyopathy (DCM), and Aging-related Frailty [3]. Regulatory Designations - Laromestrocel development programs have received five significant FDA designations: - For the HLHS program: Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation - For the Alzheimer's disease program: Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation [3].

Core Insights - Longeveron Inc. is advancing its clinical-stage cellular therapy, laromestrocel, aimed at treating hypoplastic left heart syndrome (HLHS), a severe congenital heart defect [1][2][4] - The pivotal Phase 2b clinical trial, ELPIS II, has completed enrollment, with top-line results expected in Q3 2026 [2][7] - Laromestrocel has shown promising results in previous trials, achieving 100% transplant-free survival up to five years of age compared to a historical mortality rate of approximately 20% [4] Company Overview - Longeveron Inc. focuses on developing regenerative medicines for unmet medical needs, with laromestrocel as its lead investigational product [5] - The company is pursuing multiple indications, including HLHS, Alzheimer's disease, Pediatric Dilated Cardiomyopathy (DCM), and Aging-related Frailty [5] - Laromestrocel has received several FDA designations, including Orphan Drug, Fast Track, and Rare Pediatric Disease designations for HLHS, and RMAT designation for Alzheimer's disease [5] Clinical Trial Details - ELPIS II is a pivotal trial conducted in collaboration with the National Heart, Lung, and Blood Institute (NHLBI) and supported by NIH grants [4] - The trial aims to evaluate laromestrocel's efficacy as an adjunct treatment for HLHS, a rare pediatric disease [7] - The FDA has confirmed that successful results from ELPIS II could lead to a Biologics License Application (BLA) submission for full approval [4]

Core Insights - Longeveron Inc. is participating in the 4th Annual ROTH Healthcare Opportunities Conference on October 9, 2025, in New York, where management will hold one-on-one meetings with investors [1] Company Overview - Longeveron Inc. is a clinical stage biotechnology company focused on developing regenerative medicines to meet unmet medical needs [2] - The company's lead investigational product is laromestrocel (Lomecel-B™), an allogeneic mesenchymal stem cell therapy derived from the bone marrow of young, healthy adult donors [2] - Laromestrocel has multiple potential mechanisms of action, including pro-vascular, pro-regenerative, anti-inflammatory, and tissue repair effects, with applications across various disease areas [2] - Longeveron is pursuing four pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer's disease, Pediatric Dilated Cardiomyopathy (DCM), and Aging-related Frailty [2] - The development programs for laromestrocel have received five important FDA designations: - For the HLHS program: Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation - For the Alzheimer's disease program: Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation [2]

Core Insights - Longeveron Inc. has elected Dr. George Paletta, Jr. to its Board of Directors, bringing extensive experience in medicine and entrepreneurship to the company [2][3] - The company is focused on developing cellular therapies for life-threatening, rare pediatric and chronic aging-related conditions, with a promising pipeline of stem cell therapies [2][6] Company Overview - Longeveron is a clinical stage biotechnology company that develops regenerative medicines to address unmet medical needs, with its lead product being laromestrocel (Lomecel-B™), an allogeneic mesenchymal stem cell therapy [6] - The company is pursuing four pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer's disease, Pediatric Dilated Cardiomyopathy (DCM), and Aging-related Frailty [6] - Laromestrocel has received five important FDA designations, including Orphan Drug designation and Fast Track designation for the HLHS program [6] Leadership and Expertise - Dr. Paletta is a recognized orthopedic surgeon and has significant experience in developing ambulatory surgical centers, having participated in the sale of two ASCs valued at nearly $1 billion [2][3] - He holds multiple patents in orthopedic and cardiovascular fields and has been involved in various start-up ventures, providing advisory roles [3][4] - Dr. Paletta's educational background includes a Doctor of Medicine from Johns Hopkins University and an MBA from Washington University in St. Louis [4]

Core Insights - Longeveron Inc. is participating in the Alliance for Regenerative Medicine's Cell & Gene Meeting on the Mesa from October 6-8, 2025, to explore partnerships for its Alzheimer's disease stem cell therapy program [1][2] Company Overview - Longeveron is a clinical stage biotechnology company focused on developing cellular therapies for life-threatening, rare pediatric and chronic aging-related conditions [1][5] - The lead investigational product is laromestrocel (Lomecel-B™), an allogeneic mesenchymal stem cell therapy derived from the bone marrow of young, healthy adult donors [5] - The company is pursuing four pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer's disease, Pediatric Dilated Cardiomyopathy (DCM), and Aging-related Frailty [5] Alzheimer's Disease Program - Laromestrocel has shown promising results in a completed Phase 2a clinical trial (CLEAR MIND) for mild Alzheimer's disease, demonstrating an overall slowing of disease progression compared to placebo [3][6] - The trial met primary safety and secondary efficacy endpoints, with statistically significant improvements in clinical and biomarker outcomes for specific laromestrocel groups [3][7] - Full results from the CLEAR MIND study were presented at the 2024 Alzheimer's Association International Conference [4] Regulatory Designations - Laromestrocel has received multiple FDA designations: Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation for the HLHS program; and Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation for the Alzheimer's disease program [5][6] Clinical Development Pathway - A positive Type B meeting with the FDA in March 2025 established a clear regulatory pathway for a Biologics License Application (BLA) submission for laromestrocel in Alzheimer's disease [7] - The FDA has granted laromestrocel both RMAT and Fast Track designations, facilitating its development [6][7]

Leadership Changes - Longeveron Inc. appointed Than Powell as interim Chief Executive Officer, succeeding Wa'el Hashad who left to pursue other opportunities [1][6] - Dr. Joshua Hare, the founder and Chief Science Officer, has been appointed as Executive Chairman of the Board [1][6] - The Board plans to conduct a national search for a permanent CEO [1][6] Pipeline and Clinical Trials - Longeveron has a robust pipeline centered on laromestrocel, a stem cell therapy, with positive initial outcomes in five clinical trials across three indications [2] - The pivotal Phase 2b clinical trial for laromestrocel as a treatment for hypoplastic left heart syndrome (HLHS) has achieved full enrollment, with top-line results expected in 2026 [2] - The company has expanded its pipeline to include pediatric dilated cardiomyopathy, advancing three unique programs to pivotal clinical trial stage [2][7] Regulatory Designations - Laromestrocel has received five important FDA designations, including Orphan Drug, Fast Track, and Rare Pediatric Disease designations for the HLHS program, and Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations for the Alzheimer's disease program [7]

Company Overview - Longeveron Inc. is a clinical stage biotechnology company focused on developing regenerative medicines to address unmet medical needs [2] - The company's lead investigational product is laromestrocel (Lomecel-B™), an allogeneic mesenchymal stem cell therapy derived from the bone marrow of young, healthy adult donors [2] - Laromestrocel has multiple potential mechanisms of action, including pro-vascular, pro-regenerative, anti-inflammatory, and tissue repair effects, with applications across various disease areas [2] Pipeline and FDA Designations - Longeveron is pursuing four pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer's disease, Pediatric Dilated Cardiomyopathy (DCM), and Aging-related Frailty [2] - The HLHS program has received three important FDA designations: Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation [2] - The Alzheimer's disease program has received two FDA designations: Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation [2] Upcoming Events - Longeveron will participate in the H.C. Wainwright 27th Annual Global Investment Conference from September 8-10, 2025, in New York City [1] - The company's presentation is scheduled for September 8, 2025, from 4:00 to 4:30 p.m. ET, with a webcast available on the company's website [1]

Financial Data and Key Metrics Changes - Revenues for the six months ended June 30, 2025, were $700,000, a decrease of $300,000 or 31% compared to $1,000,000 for the same period in 2024, primarily due to decreased participant demand for the Bahamas registry trial and reduced demand for contract manufacturing services [25][26] - Clinical trial revenue for the same period was $600,000, a decrease of $200,000 or 31% compared to $800,000 in 2024 [26] - General and administrative expenses increased to approximately $5,500,000, up from $4,300,000, representing a 28% increase [27] - Research and development expenses rose to approximately $5,500,000, a 39% increase from $3,900,000 in 2024 [27] - Net loss increased to approximately $10,000,000 for the six months ended June 30, 2025, compared to a net loss of $7,500,000 for the same period in 2024 [27][28] Business Line Data and Key Metrics Changes - The company is focused on three primary operational goals for 2025: advancing the pivotal Phase 2b study for SLHS, SLHS BLA preparedness, and pursuing strategic collaboration for the Alzheimer's disease program [6][30] - The SLHS program is considered a key strategic priority with a high probability of success and a shorter path to potential regulatory approval [6][30] Market Data and Key Metrics Changes - The U.S. market opportunities for the company's three initial indications are approximately $5 billion for Alzheimer's disease, over $4 billion for aging-related frailty, and up to $1 billion for hypoplastic left heart syndrome (HLHS) [5][6] - The prevalence of pediatric dilated cardiomyopathy is estimated to be around 2,000 to 3,000 patients in the U.S., while HLHS affects about 1,000 newborns [37] Company Strategy and Development Direction - The company is expanding its pipeline to include pediatric dilated cardiomyopathy and has licensed additional stem cell technology from the University of Miami [21][30] - The strategy focuses on excellent science, lower required investments, speed to market, and addressing important unmet medical needs [21] - The company plans to initiate a pivotal Phase 2 clinical trial for pediatric dilated cardiomyopathy in 2026 [18][29] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential BLA submission for SLHS in late 2026, contingent on positive trial results [28][30] - The company is focused on prudent capital allocation strategies to advance development programs, which are considered highly cost-efficient [28][29] - Management highlighted the importance of FDA interactions and the collaborative approach taken by the agency [8][56] Other Important Information - The company completed a public offering in August, raising approximately $5 million in gross proceeds [28] - The FDA has approved the IND application for evaluating laramestrocel as a treatment for pediatric dilated cardiomyopathy, allowing the company to move directly to a pivotal Phase 2 trial [17][18] Q&A Session Summary Question: Are the target patient populations and addressable markets for HLHS and pediatric dilated cardiomyopathy similar? - The markets are similar but not identical; HLHS has a one-time use administration while pediatric dilated cardiomyopathy involves continuous use [35][36] Question: Will a favorable approval decision in HLHS impact the regulatory outlook for pediatric dilated cardiomyopathy? - A positive outcome from the HLHS trial would support the regulatory review process for pediatric dilated cardiomyopathy, but the FDA will still require study results specific to that indication [41][42] Question: What is the estimated budget for the pediatric dilated cardiomyopathy trial? - Preliminary budget estimates for the entire trial range from $15 million to $20 million, with annual costs around $3 million [43][44] Question: Will the company retain the pediatric designation and associated benefits if the PRB sunsets? - The company is cautiously optimistic about the renewal of the PRB and has mechanisms in place for rolling submissions [51][53] Question: What gave the FDA confidence to allow the company to move directly to pivotal Phase 2 for pediatric dilated cardiomyopathy? - The FDA was confident due to the design of clinically meaningful endpoints and the company's robust safety data set from previous studies [67][72]