UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 Longeveron Inc. (Exact name of registrant as specified in its charter) | Delaware | 47-2174146 | | --- | --- | | (State or Other Jurisdiction | (IRS Employer | | of Incorporation) | Identification No.) | | th 1951 NW 7 Avenue, Suite 520, Miami, Florida | 33136 | | (Address of principal executi ...

Core Viewpoint - Longeveron Inc. is set to report its first quarter 2025 financial results and provide a business update on May 8, 2025, after U.S. 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] Pipeline and FDA Designations - Longeveron is pursuing three pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer's disease (AD), and aging-related frailty [3] - The HLHS program has received three FDA designations: Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation [3] - The AD program has received two important FDA designations: Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation [3]

Core Viewpoint - Longeveron Inc. has made significant progress in developing cellular therapies for life-threatening and chronic aging-related conditions, with a focus on advancing its lead product, laromestrocel (Lomecel-B™), towards a potential Biological License Application (BLA) submission in 2026 [1][22]. Strategic Overview and 2024 Progress - The field of stem cell therapy has evolved significantly over the past decade, transitioning from a theoretical concept to one that delivers tangible clinical outcomes, highlighting its potential in regenerative medicine [3]. - Longeveron is at the forefront of this evolution, with laromestrocel derived from mesenchymal stem cells (MSCs) showing promise in treating aging-related conditions and rare cardiovascular diseases [4]. Clinical Development and Market Opportunities - Laromestrocel has shown positive results in multiple clinical trials, including Phase 1 & 2 trials for Alzheimer's disease, aging-related frailty, and Hypoplastic Left Heart Syndrome (HLHS), with estimated market opportunities of over $5 billion for Alzheimer's, $4 billion for aging-related frailty, and up to $1 billion for HLHS [5]. - The World Health Organization approved "laromestrocel" as the non-proprietary name for Lomecel-B™, marking a key milestone for future commercialization [5]. HLHS Program - HLHS is a rare pediatric disease with a high mortality rate, where Longeveron's program aims to improve heart function in affected infants, achieving 100% transplant-free survival in a Phase I trial compared to a historical 20% mortality rate [7][8]. - The company is nearing completion of enrollment for the pivotal Phase 2b trial (ELPIS II), with nearly 95% enrollment expected to finish in Q2 2025 [9]. - A Type C meeting with the FDA confirmed that ELPIS II could be pivotal for BLA submission, with potential eligibility for a Priority Review Voucher (PRV) upon approval [10]. Alzheimer's Disease Program - The Alzheimer's disease program has received recognition, with positive results from the CLEAR MIND Phase 2a trial indicating safety and potential efficacy in slowing disease progression [11][12]. - A Type B meeting with the FDA established a clear regulatory pathway for a potential BLA submission based on positive interim results from a proposed seamless adaptive Phase 2/3 trial [13]. Financial Position and Capital Strategy - Longeveron anticipates needing additional capital to support its strategic objectives, with existing cash expected to fund operations into Q4 2025 [19]. - The company aims to manage expenses effectively, having reduced total operating expenses by 13% in 2024 while advancing development programs [22]. 2025 Goals and Priorities - Key objectives for 2025 include completing enrollment for ELPIS II, achieving BLA preparedness for HLHS, and seeking strategic partnerships for the Alzheimer's disease program [23]. - The company is focused on efficient execution of its strategic plan, with multiple transformational milestones anticipated over the next 12 months [20].

Core Insights - Longeveron Inc. announced a positive outcome from a Type B meeting with the FDA regarding laromestrocel (Lomecel-B) as a potential treatment for Alzheimer's disease [2][3] - The FDA and the company aligned on the study design for a pivotal Phase 2/3 clinical trial, which may lead to a Biological License Application (BLA) based on interim results [3][9] - Laromestrocel has received both RMAT and Fast Track designations from the FDA, facilitating closer interactions during its development [7][9] Company Overview - Longeveron is a clinical stage biotechnology company focused on developing regenerative medicines to address unmet medical needs, with laromestrocel as its lead investigational product [14] - The company is pursuing multiple indications, including hypoplastic left heart syndrome, Alzheimer's disease, and aging-related frailty [14] Clinical Trial Details - The planned pivotal Phase 2/3 clinical trial is expected to start in the second half of 2026, contingent on securing additional funding or partnerships [6][9] - Previous clinical trials, including a Phase 1 study and a Phase 2a trial (CLEAR-MIND), demonstrated a favorable safety profile and potential clinical efficacy in patients with mild Alzheimer's disease [4][5] Mechanism of Action - Laromestrocel is derived from mesenchymal stem cells (MSCs) and is believed to have multiple mechanisms of action that may address inflammatory responses associated with Alzheimer's disease [10][13] - The therapy aims to tackle the underlying pathology of Alzheimer's without the limitations of existing treatments [6] Alzheimer's Disease Context - Alzheimer's disease is a leading cause of dementia, significantly impacting patients and society, with costs in the U.S. reaching hundreds of billions annually [11] - Neuroinflammation is a key feature of Alzheimer's, contributing to the disease's progression and associated cognitive decline [12]

Core Insights - Longeveron Inc. announced a positive outcome from a Type B meeting with the FDA regarding laromestrocel (Lomecel-B™) as a potential treatment for Alzheimer's disease [1][2] - The FDA and the company aligned on the study design for a pivotal Phase 2/3 clinical trial, which includes patient population, placebo control, dosing, trial duration, and endpoints [2][8] - The FDA granted laromestrocel both RMAT and Fast Track designations, facilitating closer interaction during development [6][8] Company Overview - Longeveron is a clinical stage biotechnology company focused on developing regenerative medicines for unmet medical needs, with laromestrocel as its lead investigational product [12] - The company is pursuing multiple indications, including Alzheimer's disease, hypoplastic left heart syndrome, and aging-related frailty [12] Clinical Trial Details - The planned pivotal Phase 2/3 clinical trial is expected to begin in the second half of 2026, contingent on securing non-dilutive funding or partnerships [5][8] - Previous clinical trials, including a Phase 1 study and a Phase 2a trial (CLEAR-MIND), demonstrated a favorable safety profile and potential clinical efficacy in patients with mild Alzheimer's disease [3][4] Mechanism of Action - Laromestrocel is derived from mesenchymal stem cells (MSCs) and is believed to have multiple mechanisms of action that may address inflammatory responses associated with Alzheimer's disease [7][11] - The therapy aims to tackle the underlying pathology of Alzheimer's without the limitations of existing treatments [5] Alzheimer's Disease Context - Alzheimer's disease is a leading cause of dementia, significantly impacting patients, families, and society, with substantial economic costs [9] - Neuroinflammation is a key feature of Alzheimer's, contributing to the disease's progression and associated cognitive decline [10]

Core Insights - Longeveron Inc. announced the publication of results from the CLEAR MIND Phase 2a clinical trial for laromestrocel (Lomecel-B™) as a potential treatment for mild Alzheimer's disease, reinforcing its safety and efficacy [2][3] - The trial involved 48 patients aged 60-85, with results indicating improvements in cognitive function, quality of life, and brain volume [5][11] - Laromestrocel (Lomecel-B™) is the first cellular therapeutic candidate to receive U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) designation for Alzheimer's disease [5][7] Company Overview - Longeveron is a clinical stage biotechnology company focused on developing regenerative medicines to address unmet medical needs, with laromestrocel (Lomecel-B™) as its lead investigational product [9] - The company is pursuing multiple indications, including hypoplastic left heart syndrome, Alzheimer's disease, and aging-related frailty [9] Clinical Trial Details - The CLEAR MIND trial was randomized and placebo-controlled, achieving primary safety and secondary efficacy endpoints [3][6] - Key findings showed laromestrocel (Lomecel-B™) improved cognitive function and minimized brain volume loss associated with Alzheimer's disease [11] - Safety evaluations indicated that laromestrocel (Lomecel-B™) was well tolerated, with no significant adverse effects reported [6][11] Mechanism of Action - Laromestrocel (Lomecel-B™) is derived from mesenchymal stem cells (MSCs) and is believed to have multiple mechanisms of action, including anti-inflammatory and pro-vascular effects [8][9] - The therapy aims to address the underlying pathology of Alzheimer's disease without the limitations of previous treatments [3][5] Regulatory Status - The FDA has granted laromestrocel (Lomecel-B™) both RMAT and Fast Track designations, facilitating greater access to the FDA during its development [7][9] - The company plans to meet with the FDA to discuss future development paths for laromestrocel (Lomecel-B™) in Alzheimer's disease [7]

Core Insights - Longeveron Inc. announced the publication of results from the CLEAR MIND Phase 2a clinical trial for laromestrocel (Lomecel-B™) as a potential treatment for mild Alzheimer's disease, reinforcing its safety and efficacy [1][2] Clinical Trial Details - The Phase 2a trial involved 48 patients aged 60-85 with mild Alzheimer's disease, where 36 received laromestrocel and 12 received a placebo [2][3] - The trial achieved its primary safety and secondary efficacy endpoints, with results published in Nature Medicine [2][3] Key Findings - Laromestrocel (Lomecel-B™) improved cognitive function, quality of life, and brain volume in patients with mild Alzheimer's disease [3] - The treatment demonstrated safety and tolerability, with no significant adverse effects reported [4] - Statistically significant improvements were observed in cognitive assessments and brain volume preservation compared to placebo [4] Mechanism of Action - Laromestrocel is a mesenchymal stem cell therapy that may address inflammatory responses in the brain, offering a new strategy for treating Alzheimer's disease [2][6] - The therapy has multiple potential mechanisms, including pro-vascular and immunomodulatory effects [6][7] Regulatory Designations - Laromestrocel (Lomecel-B™) has received both Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA for the treatment of mild Alzheimer's disease [5][7] Future Development - The company plans to meet with the FDA to discuss the data and future development paths for laromestrocel in Alzheimer's disease [5]

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 Lomecel-B™, an allogeneic medicinal signaling cell therapy derived from the bone marrow of young, healthy adult donors [2] - Lomecel-B™ 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 three pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer's disease, 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 significant FDA designations: Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation [2] Upcoming Events - Longeveron will participate in the 37th Annual Roth Conference on March 17-18, 2025, with a fireside chat scheduled for March 18, 2025, from 9:00 to 9:25 a.m. PT [1] - The conference presentation will be accessible via webcast, with a replay available on the company's website for 90 days following the event [1]

Financial Data and Key Metrics Changes - Revenues for 2024 were $2.4 million, up $1.7 million or 237% compared to 2023, primarily due to increased participant demand for clinical trials and new contract manufacturing revenue [23] - Total operating expenses for the year declined 13% year-over-year, with G&A expenses decreasing to approximately $10.3 million from $12.2 million in 2023, a decrease of approximately $1.9 million or 16% [24] - Net loss decreased 25% to approximately $16 million for 2024 from a net loss of $21.4 million for 2023 [26] Business Line Data and Key Metrics Changes - Contract manufacturing revenue for 2024 was $1 million, consisting of $0.5 million from manufacturing lease services and another $0.5 million from manufacturing services contracts [23] - R&D expenses for 2024 decreased approximately $1 million or 10% to approximately $8.1 million, primarily due to reductions in expenses related to completed clinical trials [25] Market Data and Key Metrics Changes - The U.S. market opportunity for the company's three initial indications is approximately $5 billion for Alzheimer's disease, $4 billion for aging-related frailty, and up to $1 billion for HLHS [7] Company Strategy and Development Direction - The HLHS program is a key strategic priority, with high probability of success and the shortest path to potential regulatory approval and commercialization [8] - The company plans to initiate a rolling submission of a BLA with the FDA in 2026 if the current ELPIS II trial is successful [10] - The company aims to focus on partnerships for the Alzheimer's disease program to avoid dilution of efforts while pursuing global opportunities [85] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential for a transformative year in 2025, with clarity on the Alzheimer's development pathway and completion of enrollment for the HLHS Phase II trial [11] - The company is ramping up activities for the BLA submission, which will increase operating expenses and capital expenditure requirements in 2025 [27] Other Important Information - The international non-proprietary name for the company's cellular therapy, Lomecel-B, was approved by the WHO, marking an important step in its development and potential commercialization [12] - The company is currently conducting a Phase IIb clinical trial, ELPIS II, which has achieved over 90% enrollment and is expected to complete enrollment by the second quarter of 2025 [16] Q&A Session Summary Question: What is causing the enrollment delay in ELPIS II? - Management noted that HLHS is a rare disease, making it difficult to predict enrollment timelines accurately, with surgery dates being a key factor [36][38] Question: Will the FDA consider a composite endpoint for ELPIS II? - Management confirmed discussions with the FDA regarding the acceptance of composite endpoints, including hospitalization and survival rates [51][53] Question: What are the prospects for receiving a pediatric disease priority review voucher for Lomecel-B? - Management indicated that they believe they will have an extension for the PRV, pending agreement with the agency at the pre-BLA meeting [60] Question: How many patients remain to be enrolled in ELPIS II? - Management confirmed that 3 patients remain to be enrolled, with the possibility of over-enrollment if additional eligible patients are identified [65][66] Question: What is the commercialization strategy for Lomecel-B in HLHS? - Management stated that a minimal commercial infrastructure would be needed, as there are only about 50 treating physicians in the U.S. [78][80] Question: What are the plans for international regulatory discussions regarding Alzheimer's disease? - Management plans to engage with international regulatory authorities after hiring a full-time regulatory person to lead these discussions [83][84]

Financial Data and Key Metrics Changes - Revenues for 2024 were $2.4 million, up $1.7 million or 237% compared to 2023, primarily due to increased participant demand for clinical trials and new contract manufacturing revenue [23] - Net loss decreased 25% to approximately $16 million for 2024 from a net loss of $21.4 million for 2023 [26] - Total operating expenses for the year declined 13% year-over-year, with G&A expenses decreasing to approximately $10.3 million from $12.2 million in 2023 [24] Business Line Data and Key Metrics Changes - Contract manufacturing revenue for 2024 was $1 million, consisting of $0.5 million from manufacturing lease services and another $0.5 million from manufacturing services contracts [23] - R&D expenses for 2024 decreased approximately $1 million or 10% to approximately $8.1 million, primarily due to reductions in expenses related to completed clinical trials [25] Market Data and Key Metrics Changes - The U.S. market opportunity for the company's three initial indications is approximately $5 billion for Alzheimer's disease, $4 billion for aging-related frailty, and up to $1 billion for HLHS [7] Company Strategy and Development Direction - The HLHS program is a key strategic priority, with high probability of success and the shortest path to potential regulatory approval and commercialization [8] - The company plans to initiate a rolling submission of a BLA with the FDA in 2026 if the current ELPIS II trial is successful [10][27] - The international non-proprietary name for Lomecel-B was approved by the WHO, marking an important step in its development and potential commercialization [12] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about 2025 being a transformative year, with clarity on the Alzheimer's development pathway and completion of enrollment for the HLHS Phase II trial [11] - The company is focused on seeking additional financing and non-dilutive funding options to support increased operating expenses and capital investments in 2025 [28] Other Important Information - The company has received multiple FDA designations for its programs, including orphan-drug designation and fast track designation for HLHS, and RMAT designation for Alzheimer's disease [14][15] Q&A Session Summary Question: What is causing the enrollment delay in ELPIS II? - Management noted that HLHS is a rare disease, making it difficult to predict enrollment timelines, with surgery dates being a key factor [36][38] Question: Will the FDA consider a composite endpoint for ELPIS II? - Management confirmed that they are working with the FDA to propose a composite endpoint that includes additional factors beyond RVEF [50][52] Question: What are the prospects for receiving a pediatric disease priority review voucher for Lomecel-B? - Management indicated that they believe they will have an extension for the PRV, pending agreement with the agency at the pre-BLA meeting [60] Question: How many patients remain to be enrolled in ELPIS II? - Management confirmed that 35 out of 38 patients have been enrolled, with the possibility of over-enrollment if additional eligible patients are identified [65][66] Question: What is the potential commercial scenario for Lomecel-B in HLHS? - Management stated that commercialization would require minimal infrastructure, targeting approximately 50 treating physicians in the U.S. [78][80] Question: What are the plans for international regulatory discussions regarding Alzheimer's disease? - Management plans to engage with international regulatory authorities after hiring a full-time regulatory person to lead these discussions [83][85]