Wa'el Hashad Wa'el Hashad, CEO, Longeveron MIAMI, April 22, 2025 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN), a clinical stage regenerative medicine biotechnology company developing cellular therapies for life-threatening and chronic aging-related conditions, today announced that the Company’s CEO, Wa’el Hashad, issued the following letter to Longeveron shareholders. Dear Fellow Shareholders, Twenty twenty-four was a year of execution for Longeveron. We made significant progress advancing Longeve ...

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]

Nataliya Agafonova MIAMI, March 20, 2025 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN), a clinical stage regenerative medicine biotechnology company developing cellular therapies for life-threatening and chronic aging-related conditions, today announced the positive outcome of a Type B meeting with the U.S. Food and Drug Administration (FDA), supporting the advancement of laromestrocel (Lomecel-B), a proprietary, scalable, allogeneic, investigational cellular therapy, as a potential treatment for Alzhe ...

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]

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]

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] - 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]

Revenue Growth - Total revenue for 2024 increased 237% year-over-year to $2.4 million, primarily due to successful contract manufacturing services and increased participant demand for the Bahamas Registry Trial[6]. - Total revenues for the year ended December 31, 2024, increased to $2,392,000, representing a significant growth of 237% compared to $709,000 in 2023[18]. - Clinical trial revenue from the Bahamas Registry Trial for 2024 was $1.4 million, representing a 110% increase compared to $0.7 million in 2023[9]. - Clinical trial revenue reached $1,402,000 in 2024, up from $668,000 in 2023, indicating a growth of 109%[18]. Profitability and Expenses - Gross profit for 2024 was approximately $1.9 million, an increase of 752% compared to a gross profit of $0.2 million in 2023[9]. - Gross profit for 2024 was $1,884,000, compared to $221,000 in 2023, reflecting a substantial increase[18]. - General and administrative expenses decreased by 16% to approximately $10.3 million in 2024, down from $12.2 million in 2023[9]. - Research and development expenses decreased by 10% to approximately $8.1 million in 2024, down from approximately $9.1 million in 2023[9]. - Total operating expenses decreased to $18,406,000 in 2024 from $21,250,000 in 2023, a reduction of approximately 13%[18]. Net Loss and Shareholder Impact - Net loss decreased to approximately $16.0 million for 2024, a reduction of 25% from a net loss of $21.4 million in 2023[9]. - Net loss attributable to common stockholders for 2024 was $24,623,000, compared to $22,211,000 in 2023, indicating an increase in losses[18]. - Basic and diluted net loss per share improved to $2.62 in 2024 from $10.22 in 2023, showing a reduction in loss per share[18]. Cash and Assets - Cash and cash equivalents as of December 31, 2024, were $19.2 million, expected to fund operations into Q4 2025[9]. - Cash and cash equivalents increased significantly to $19,232,000 in 2024 from $4,949,000 in 2023, a growth of 288%[16]. - Total assets rose to $25,558,000 in 2024, up from $12,078,000 in 2023, marking an increase of 112%[16]. - Total liabilities decreased to $3,671,000 in 2024 from $5,337,000 in 2023, a reduction of approximately 31%[16]. - Total stockholders' equity increased to $21,887,000 in 2024 from $6,741,000 in 2023, reflecting a growth of 225%[16]. Clinical Trials and Future Plans - The pivotal Phase 2b clinical trial (ELPIS II) for Lomecel-B in Hypoplastic Left Heart Syndrome has achieved over 90% enrollment and is expected to complete enrollment in Q2 2025[6]. - The FDA meeting to discuss development paths for Lomecel-B in mild Alzheimer's disease is anticipated in late Q1 2025[6]. - The company anticipates a potential BLA submission for Lomecel-B in 2026, contingent on the success of the ELPIS II trial[9].

Core Viewpoint - Longeveron Inc. reported significant advancements in its clinical programs and financial results for 2024, highlighting the potential of its cellular therapy candidate, Lomecel-B™, in treating Hypoplastic Left Heart Syndrome (HLHS) and mild Alzheimer's disease, with multiple key milestones anticipated in the coming year [2][8]. Financial Results - Total revenue for 2024 increased by 237% year-over-year to $2.4 million, driven by contract manufacturing services and increased demand for the Bahamas Registry Trial [8][19]. - Clinical trial revenue rose to $1.4 million in 2024 from $0.7 million in 2023, marking a 110% increase due to higher participant demand [8][19]. - Gross profit for 2024 was approximately $1.9 million, a 752% increase compared to $0.2 million in 2023 [8][19]. - General and administrative expenses decreased by 16% to approximately $10.3 million in 2024, while research and development expenses decreased by 10% to approximately $8.1 million [8][19]. - The net loss for 2024 decreased to approximately $16.0 million from $21.4 million in 2023, representing a 25% reduction [8][16][19]. Development Programs - Lomecel-B™, the company's investigational cellular therapy, is being evaluated for HLHS and mild Alzheimer's disease, with over 90% enrollment achieved in the pivotal Phase 2b clinical trial (ELPIS II) for HLHS [2][8][9]. - The FDA has granted Lomecel-B™ several designations, including Orphan Drug, Fast Track, and Rare Pediatric Disease for HLHS, and Regenerative Medicine Advanced Therapy (RMAT) and Fast Track for Alzheimer's disease [8][11]. - The WHO approved "laromestrocel" as the non-proprietary name for Lomecel-B™ in February 2025 [3][8]. Future Outlook - The company anticipates completing enrollment in the ELPIS II trial by the second quarter of 2025 and plans to submit a Biological License Application (BLA) for HLHS in 2026, contingent on trial success [2][8]. - A meeting with the FDA is expected in late Q1 2025 to discuss the development pathway for Lomecel-B™ in mild Alzheimer's disease [8][9].

Financial Condition - As of December 31, 2024, the company had cash and cash equivalents of $19.2 million, which is expected to fund operations into Q4 2025 based on current forecasts[191]. - The company has accumulated a deficit of approximately $109.6 million as of December 31, 2024, up from $85.0 million in 2023, indicating ongoing financial losses[194]. - The company anticipates significant increases in operating expenses and capital expenditures in 2025 due to ramped-up activities related to Biologics License Application (BLA) enabling activities[195]. - The company expects to incur additional losses in the future, with operating expenses likely to increase as it conducts more preclinical studies and clinical trials[190]. - The company has ongoing challenges with liquidity and access to capital, which could impact its ability to fund operations and clinical programs[190]. - The company’s ability to continue as a going concern is dependent on its available cash and successful management of its operating requirements[198]. - The company has been funded in part by government and non-profit association grants, which are not guaranteed sources of future funding[199]. - The company anticipates that its current cash resources will be insufficient to fund operations beyond Q4 2025, necessitating additional funding[356]. Regulatory Challenges - The company faces significant risks related to the transition from research to commercial activities, which may require substantial additional capital[167]. - There are no FDA-approved allogeneic, cell-based therapies for Aging-related Frailty or other conditions the company is studying, complicating potential regulatory approval[172]. - The FDA has indicated that the concept of "Frailty" will require additional clinical data and discussion before marketing authorization can be obtained[173]. - The lengthy and uncertain process of obtaining regulatory approvals for product candidates may hinder the company's ability to generate sufficient revenues[245]. - Delays in clinical trials or regulatory approvals could shorten exclusive commercialization periods, allowing competitors to enter the market earlier[243]. - The complexity of obtaining regulatory approval for mesenchymal stem cells may result in longer timelines compared to conventional drugs, impacting commercialization efforts[246]. - Regulatory approvals for product candidates may be subject to limitations and require costly post-marketing testing, including Phase 4 clinical trials[258]. - The FDA has historically been stringent with Alzheimer's disease drug approvals, with only two drugs, Aduhelm® and Leqembi™, receiving accelerated approval, both requiring confirmatory trials[251]. - The company has not had success in developing therapeutics for Alzheimer's disease, despite significant investment from the biopharmaceutical industry[252]. - The acceptance of clinical trial data by regulatory authorities such as the U.S. FDA and Japanese PMDA is uncertain, which could impact the commercialization of product candidates[310]. - Changes in regulations and future legislation could prevent or delay regulatory approval of product candidates, affecting profitability[325]. Competition and Market Risks - The company is facing increased competition in the cell therapy field from both established companies and academic institutions, which may have greater resources[186][187]. - If the company's product candidates are approved, potential competitors may seek to introduce biosimilar products using abbreviated approval pathways[223]. - The biotechnology industry is highly competitive, and the company's success depends on its ability to respond to rapid technological changes and attract skilled personnel[222]. - The company may face challenges in protecting its proprietary rights, as competitors could independently develop similar technologies[225]. - The company may need to obtain licenses from third parties to advance research or commercialization, and failure to do so could significantly harm its business[218]. Intellectual Property Risks - The company has pending patent applications related to MSC technology, but these may not issue or may issue with narrower claims, potentially allowing competitors to develop similar products[206]. - The company faces risks regarding the assignment of patents from third parties, which could affect its control over patented technology and its ability to commercialize products[208]. - The extensive time required for product development may lead to patent expirations before commercialization, reducing competitive advantages[209]. - The Leahy-Smith America Invents Act transitioned the U.S. to a "first inventor to file" system, increasing the risk of third parties obtaining patents for inventions made independently[228]. - The U.S. patent law changes may increase uncertainties and costs related to patent application prosecution and enforcement, potentially adversely affecting the company's competitive position[230]. - Patent terms in the U.S. generally last 20 years from the earliest filing date, which may not provide sufficient protection against competition if patents expire before product commercialization[233]. - Patent term extensions of up to five years may be available, but failure to obtain these extensions could lead to reduced revenue as competitors may launch products post-expiration[234]. - The company may face challenges in protecting intellectual property rights globally due to varying enforcement levels and legal complexities in foreign jurisdictions[236]. - Compulsory licensing laws in many countries could force the company to grant licenses to third parties, impairing its competitive position[238]. - Non-compliance with procedural requirements could lead to the loss of patent rights, significantly impacting the company's business and financial condition[240]. Operational Risks - The company relies on a limited supply of bone marrow donors, which may impact its ability to produce sufficient quantities of product candidates for clinical trials[178]. - Future government regulations or health concerns may further reduce the number of available bone marrow donors, limiting production capabilities[179]. - The company’s processing and storage facility is located in a hurricane-prone area, posing risks to its operations and product candidates[183]. - Ethical concerns surrounding stem cell therapy may negatively affect public perception and regulatory approval of the company’s products[184]. - The company relies on research institutions for clinical trials, which may lead to increased costs and delays if agreements are not reached or maintained[254]. - The reliance on third parties for clinical trials and manufacturing poses risks, including potential delays and increased costs if these parties fail to meet obligations[273][280]. - Future dependence on third-party manufacturers may expose the company to supply shortages and price fluctuations, potentially harming its operations[285]. - Risks associated with third-party suppliers include supply interruptions, compliance failures, and delays in product shipments, which could adversely affect the company's ability to meet clinical trial demands[286][287]. Legislative and Healthcare Environment - Legislative changes, such as the Budget Control Act, have resulted in Medicare payment reductions of 2% per fiscal year since April 2013, impacting revenue[262]. - Increasing governmental efforts to cap healthcare costs may limit coverage and reimbursement for newly approved products[263]. - The Inflation Reduction Act of 2022 allows CMS to negotiate prices for certain high-cost drugs under Medicare Part B and Part D, starting with ten drugs in 2026 and increasing to 20 drugs by 2029[272]. - Medicare beneficiaries' annual out-of-pocket drug expenses are capped at $2,000 under the Inflation Reduction Act[272]. - Legislative changes may lead to reductions in Medicare and other healthcare funding, impacting the prices for product candidates and their prescription frequency[268]. - The trend towards managed healthcare and cost containment initiatives is expected to exert pricing pressures on future approved products[268]. - The FDA has been directed to clarify the approval framework for generic drugs, which may affect competition in the pharmaceutical market[266]. - State legislatures are implementing regulations to control pharmaceutical pricing, which may include price constraints and access restrictions[267]. - The evolving legislative landscape in healthcare reform may materially impact revenue prospects and operational stability[269]. - Increased scrutiny over drug pricing practices has resulted in Congressional inquiries and proposed legislation aimed at enhancing transparency and reforming reimbursement methodologies[330]. - The company anticipates that future healthcare reforms may impose more rigorous coverage criteria and downward pressure on product pricing, impacting revenue generation and profitability[331]. Stock and Market Risks - Class A common stock closing bid price as of February 18, 2025, was $1.58[364]. - Nasdaq's minimum bid price requirement is $1.00 per share[364]. - Company executed a reverse stock split on March 26, 2024[364]. - If closing bid price drops below $1.00 before March 26, 2025, immediate delisting proceedings will commence[364]. - Loss of Nasdaq listing would significantly reduce liquidity and adversely affect stock value[364]. - The company is at risk of delisting if share price does not meet requirements[364]. - Current market conditions may impact the ability to maintain share price above the minimum requirement[364].