Longeveron® Announces Positive Type B Meeting with U.S. FDA Regarding Pathway to BLA for Laromestrocel (Lomecel-BTM) in Alzheimer’s Disease
Longeveron has achieved a positive Type B meeting with the FDA regarding the development pathway for laromestrocel (Lomecel-B) as a potential Alzheimer's disease treatment. The FDA agreed to consider a Biological License Application (BLA) based on a single, pivotal Phase 2/3 clinical trial's interim results.
The company has already completed two positive clinical trials in mild Alzheimer's patients: a Phase 1 study and the CLEAR-MIND Phase 2a trial. The latter demonstrated favorable safety, no amyloid-related imaging abnormalities, and improvements in cognition, function, quality of life, and reduced brain atrophy. These results led to FDA Regenerative Medicine Advanced Therapy (RMAT) designation.
The pivotal Phase 2/3 trial is planned for second half of 2026, subject to securing non-dilutive funding or partnership support. Laromestrocel has also received Fast Track designation and is reportedly the first cellular therapeutic candidate to receive RMAT designation for Alzheimer's disease.
Longeveron ha raggiunto un incontro positivo di Tipo B con la FDA riguardo al percorso di sviluppo per laromestrocel (Lomecel-B) come potenziale trattamento per la malattia di Alzheimer. La FDA ha concordato di considerare una domanda di licenza biologica (BLA) basata sui risultati intermedi di un singolo, fondamentale studio clinico di Fase 2/3.
L'azienda ha già completato due studi clinici positivi su pazienti con Alzheimer lieve: uno studio di Fase 1 e il trial CLEAR-MIND di Fase 2a. Quest'ultimo ha dimostrato una sicurezza favorevole, assenza di anomalie di imaging correlate all'amiloide e miglioramenti nella cognizione, nella funzionalità, nella qualità della vita e una riduzione dell'atrofia cerebrale. Questi risultati hanno portato alla designazione di Terapia Avanzata per la Medicina Rigenerativa (RMAT) da parte della FDA.
Il trial fondamentale di Fase 2/3 è previsto per la seconda metà del 2026, soggetto a ottenere finanziamenti non diluitivi o supporto da partnership. Laromestrocel ha anche ricevuto la designazione Fast Track ed è riportato come il primo candidato terapeutico cellulare a ricevere la designazione RMAT per la malattia di Alzheimer.
Longeveron ha logrado una reunión positiva de Tipo B con la FDA en relación con la trayectoria de desarrollo de laromestrocel (Lomecel-B) como un posible tratamiento para la enfermedad de Alzheimer. La FDA acordó considerar una Solicitud de Licencia Biológica (BLA) basada en los resultados interinos de un único ensayo clínico pivotal de Fase 2/3.
La empresa ya ha completado dos ensayos clínicos positivos en pacientes con Alzheimer leve: un estudio de Fase 1 y el ensayo CLEAR-MIND de Fase 2a. Este último demostró una seguridad favorable, ausencia de anomalías de imagen relacionadas con la amiloide y mejoras en la cognición, la función, la calidad de vida y una reducción de la atrofia cerebral. Estos resultados llevaron a la designación de Terapia Avanzada de Medicina Regenerativa (RMAT) por parte de la FDA.
El ensayo pivotal de Fase 2/3 está previsto para la segunda mitad de 2026, sujeto a asegurar financiamiento no dilutivo o apoyo de asociaciones. Laromestrocel también ha recibido la designación Fast Track y se informa que es el primer candidato terapéutico celular en recibir la designación RMAT para la enfermedad de Alzheimer.
롱에버론은 알츠하이머병 치료제로서 라로메스트로셀 (로메셀-B)의 개발 경로에 대해 FDA와 긍정적인 B형 회의를 진행했습니다. FDA는 단일 주요 2/3상 임상 시험의 중간 결과를 바탕으로 생물학적 라이센스 신청서(BLA)를 고려하기로 합의했습니다.
회사는 이미 경증 알츠하이머 환자를 대상으로 두 개의 긍정적인 임상 시험을 완료했습니다: 1상 연구와 CLEAR-MIND 2a상 시험입니다. 후자는 안전성이 우수하고 아밀로이드 관련 영상 이상이 없으며 인지, 기능, 삶의 질 개선과 뇌 위축 감소를 보여주었습니다. 이러한 결과는 FDA의 재생 의학 고급 치료(RMAT) 지정으로 이어졌습니다.
주요 2/3상 시험은 2026년 하반기에 계획되어 있으며, 비희석 자금 조달 또는 파트너십 지원 확보에 따라 달라질 수 있습니다. 라로메스트로셀은 또한 패스트 트랙 지정을 받았으며 알츠하이머병에 대한 RMAT 지정을 받은 첫 번째 세포 치료 후보로 알려져 있습니다.
Longeveron a obtenu une réunion positive de type B avec la FDA concernant le chemin de développement de laromestrocel (Lomecel-B) en tant que traitement potentiel de la maladie d'Alzheimer. La FDA a accepté d'examiner une demande de licence biologique (BLA) basée sur les résultats intermédiaires d'un essai clinique pivot de phase 2/3.
La société a déjà terminé deux essais cliniques positifs chez des patients atteints d'Alzheimer léger : une étude de phase 1 et l'
L'essai pivot de phase 2/3 est prévu pour la deuxième moitié de 2026, sous réserve de l'obtention de financements non dilutifs ou de soutien de partenariat. Laromestrocel a également reçu la désignation Fast Track et serait le premier candidat thérapeutique cellulaire à recevoir la désignation RMAT pour la maladie d'Alzheimer.
Longeveron hat ein positives Typ-B-Treffen mit der FDA bezüglich des Entwicklungswegs für Laromestrocel (Lomecel-B) als potenzielle Behandlung der Alzheimer-Krankheit erreicht. Die FDA stimmte zu, einen Antrag auf biologische Lizenz (BLA) auf der Grundlage der Zwischenresultate einer einzigen, entscheidenden Phase-2/3-Studie zu prüfen.
Das Unternehmen hat bereits zwei positive klinische Studien bei Patienten mit leichter Alzheimer-Krankheit abgeschlossen: eine Phase-1-Studie und die CLEAR-MIND Phase-2a-Studie. Letztere zeigte eine günstige Sicherheit, keine amiloidbezogenen Bildgebungsanomalien und Verbesserungen in Kognition, Funktion, Lebensqualität sowie eine Verringerung der Hirnatrophie. Diese Ergebnisse führten zur Regenerative Medicine Advanced Therapy (RMAT) Auszeichnung durch die FDA.
Die entscheidende Phase-2/3-Studie ist für die zweite Hälfte des Jahres 2026 geplant, abhängig von der Sicherstellung von nicht verwässernden Finanzierungen oder Unterstützung durch Partnerschaften. Laromestrocel hat auch die Fast-Track-Auszeichnung erhalten und wird als der erste zelluläre therapeutische Kandidat berichtet, der die RMAT-Auszeichnung für die Alzheimer-Krankheit erhalten hat.
- FDA agreement to consider BLA based on single pivotal trial interim results
- Successful completion of two positive clinical trials showing favorable safety and efficacy
- Received both RMAT and Fast Track designations from FDA
- First cellular therapeutic to receive RMAT designation for Alzheimer's disease
- Trial initiation dependent on securing additional funding or partnership
- Significant timeline to potential commercialization with trial starting in 2H 2026
Insights
Longeveron's announcement represents a significant regulatory advancement for laromestrocel (Lomecel-B) in Alzheimer's disease treatment. The FDA's agreement to consider a BLA based on a single pivotal trial—rather than multiple Phase 3 studies—potentially shortens the development timeline by years and substantially reduces costs.
The RMAT designation is particularly notable as laromestrocel is reportedly the first cellular therapy to receive this status for Alzheimer's disease. This designation is relatively rare, with the FDA granting it to only a small percentage of applicants, signaling regulatory recognition of the therapy's potential. Combined with Fast Track designation, these regulatory advantages provide enhanced FDA interactions and eligibility for accelerated approval pathways.
The Phase 2a results published in Nature Medicine demonstrated favorable safety outcomes without the amyloid-related imaging abnormalities (ARIA) that have plagued other Alzheimer's therapies. This safety profile could represent a significant competitive advantage if efficacy is maintained in larger trials.
However, investors should carefully weigh several important factors:
- The trial initiation timeframe (2H 2026) places potential approval and commercialization years into the future
- The company's
$25.5M market capitalization raises questions about its ability to independently fund development - The explicit contingency on securing non-dilutive funding or partnerships indicates current financial constraints
- While early results are promising, they don't guarantee Phase 3 success in this notoriously challenging therapeutic area
The Alzheimer's market opportunity is substantial, with over 6 million patients in the US alone and growing rapidly with the aging population. If successful, laromestrocel's cellular therapy approach targeting inflammatory responses offers a mechanistically distinct alternative to the recently approved antibody therapies focused on amyloid clearance.
- Planned single, pivotal Phase 2/3 clinical trial, if positive, acceptable for Biological License Application (BLA) submission for Alzheimer’s disease
- Alignment with FDA on proposed trial study design, population and endpoints
- Initiation of planned pivotal Phase 2/3 clinical trial anticipated in 2H 2026, contingent upon obtaining non-dilutive funding and/or partnering support
- Laromestrocel (Lomecel-B™) has received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for Alzheimer’s disease which provides frequent interactions with the FDA, intensive guidance on drug development, and eligibility for accelerated approval and priority review
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-BTM), a proprietary, scalable, allogeneic, investigational cellular therapy, as a potential treatment for Alzheimer’s disease (AD).
The Company and the FDA reached foundational alignment on the overall study design for a proposed single, pivotal, seamless adaptive Phase 2/3 clinical trial, including proposed AD patient population, proposed placebo control, laromestrocel (Lomecel-BTM) dose selection and frequency, trial duration and trial endpoints. To accelerate the pathway to potential approval, the FDA agreed to consider a BLA based on positive interim trial results from the planned single study.
Longeveron has previously completed two positive clinical trials studying patients with mild AD: a Phase 1 study and a multi-center, randomized, double-blind, placebo-controlled Phase 2a clinical trial (CLEAR-MIND), results of which were presented at the 2024 Alzheimer's Association International Conference (AAIC) and published in Nature Medicine in March 2025.
The Phase 2a CLEAR-MIND study results showed a favorable safety profile, absence of amyloid-related imaging abnormalities (ARIA) with laromestrocel (Lomecel-BTM) administration, and several domains of potential clinical efficacy, including cognition, function, quality of life, and reduction in brain atrophy. The results of the CLEAR-MIND trial formed the basis for the FDA RMAT designation.
“We are pleased to have alignment with the FDA on the development pathway for our laromestrocel development program in Alzheimer’s disease, a debilitating neurodegenerative disorder that leads to progressive memory loss and has a significant burden on patients, families, caregivers and society in general,” said Nataliya Agafonova, Chief Medical Officer of Longeveron. “Laromestrocel’s multiple potential mechanisms of action to address inflammatory responses offer the potential to address the underlying pathology of Alzheimer’s disease without the limitations of previous therapies. We look forward to further advancing this potentially transformative stem cell therapy treatment option for patients suffering from mild Alzheimer’s disease.”
Contingent upon obtaining additional non-dilutive funding and/or partnering support, the Company anticipates initiating the pivotal clinical trial in the second half of 2026.
The FDA has granted laromestrocel (Lomecel-B™) both Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation for the treatment of mild Alzheimer’s disease, which allow greater access to FDA interaction during laromestrocel’s (Lomecel-B™) development for Alzheimer’s disease. Laromestrocel (Lomecel-B™) is, to Company’s knowledge, the first cellular therapeutic candidate to receive FDA RMAT designation for Alzheimer’s disease.
About laromestrocel (Lomecel-B™)
Laromestrocel is a living cell product made from specialized cells isolated from the bone marrow of young healthy adult donors. These specialized cells, known as mesenchymal stem cells (MSCs), are essential to our endogenous biological repair mechanism. MSCs have been shown to perform a number of complex functions in the body, including the formation of new tissue. They also have been shown to respond to sites of injury or disease and secrete bioactive factors that are immunomodulatory and regenerative. We believe that laromestrocel may have multiple potential mechanisms of action that may lead to anti-inflammatory, pro-vascular regenerative responses, and therefore may have broad application for a range of rare and aging related diseases.
About Alzheimer’s Disease (AD)
Alzheimer’s disease is neurodegenerative disorder that leads to progressive memory loss and death and currently has very limited therapeutic options. AD is the leading form of dementia. This disease affects millions of Americans, leads to early mortality, and creates a tremendous burden on families and society that costs the U.S. hundreds of billions of dollars annually in direct costs and lost productivity. One in three older adults dies with Alzheimer’s or another dementia.
Inflammation in the brain – a process called neuroinflammation – is a key feature of AD. This pro-inflammatory state appears essential for the clinical manifestation of dementia resulting from AD. In addition to affecting the brain tissue itself, neuroinflammation also impairs the blood vessels in the brain and the exchange barrier between the blood and the brain, called the blood-brain barrier (BBB). Ultimately, these pathological processes lead to the structural changes in the brain and resulting dementia.
Longeveron is testing laromestrocel (Lomecel-B) as a potential treatment for mild AD based on the hypothesis that its multiple possible mechanisms of action (MOAs) can simultaneously address multiple features of AD.
About Longeveron Inc.
Longeveron is a clinical stage biotechnology company developing regenerative medicines to address unmet medical needs. 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. Laromestrocel (Lomecel-B™) has multiple potential mechanisms of action encompassing pro-vascular, pro-regenerative, anti-inflammatory, and tissue repair and healing effects with broad potential applications across a spectrum of disease areas. Longeveron is currently pursuing three pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer’s disease, and Aging-related Frailty. Laromestrocel (Lomecel-B™) development programs have received five distinct and important FDA designations: for the HLHS program - Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation; and, for the AD program - Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation. For more information, visit www.longeveron.com or follow Longeveron on LinkedIn, X, and Instagram.
Forward-Looking Statements
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, which reflect management’s current expectations, assumptions, and estimates of future operations, performance and economic conditions, and involve known and unknown risks, uncertainties, and other important factors that could cause actual results, performance, or achievements to differ materially from those anticipated, expressed, or implied by the statements made herein. Forward-looking statements are generally identifiable by the use of forward-looking terminology such as “anticipate,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expects,” “intend,” “looks to,” “may,” “on condition,” “plan,” “potential,” “predict,” “preliminary,” “project,” “see,” “should,” “target,” “will,” “would,” or the negative thereof or comparable terminology, or by discussion of strategy or goals or other future events, circumstances, or effects and include, but are not limited to, statements about the various below-listed factors. Factors that could cause actual results to differ materially from those expressed or implied in any forward-looking statements in this release include, but are not limited to, our cash position and need to raise additional capital, the difficulties we may face in obtaining access to capital, and the dilutive impact it may have on our investors; our financial performance, and ability to continue as a going concern; the period over which we estimate our existing cash and cash equivalents will be sufficient to fund our future operating expenses and capital expenditure requirements; the ability of our clinical trials to demonstrate safety and efficacy of our product candidates, and other positive results; the timing and focus of our ongoing and future preclinical studies and clinical trials, and the reporting of data from those studies and trials; the size of the market opportunity for certain of our product candidates, including our estimates of the number of patients who suffer from the diseases we are targeting; our ability to scale production and commercialize the product candidate for certain indications; the success of competing therapies that are or may become available; the beneficial characteristics, safety, efficacy and therapeutic effects of our product candidates; our ability to obtain and maintain regulatory approval of our product candidates in the U.S. and other jurisdictions; our plans relating to the further development of our product candidates, including additional disease states or indications we may pursue; our plans and ability to obtain or protect intellectual property rights, including extensions of existing patent terms where available and our ability to avoid infringing the intellectual property rights of others; the need to hire additional personnel and our ability to attract and retain such personnel; and our estimates regarding expenses, future revenue, capital requirements and needs for additional financing.
Further information relating to factors that may impact the Company’s results and forward-looking statements are disclosed in the Company’s filings with the Securities and Exchange Commission, including Longeveron’s Annual Report on Form 10-K for the year ended December 31, 2024, filed with the Securities and Exchange Commission on February 28, 2025, its Quarterly Reports on Form 10-Q, and its Current Reports on Form 8-K. The Company operates in highly competitive and rapidly changing environment; therefore, new factors may arise, and it is not possible for the Company’s management to predict all such factors that may arise nor assess the impact of such factors or the extent to which any individual factor or combination thereof, may cause results to differ materially from those contained in any forward-looking statements. The forward-looking statements contained in this press release are made as of the date of this press release based on information available as of the date of this press release, are inherently uncertain, and the Company disclaims any intention or obligation, other than imposed by law, to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise.
Investor and Media Contact:
Derek Cole
Investor Relations Advisory Solutions
derek.cole@iradvisory.com
A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/e9e96d9c-d6c3-4aec-ba85-36631b520211
FAQ
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