Longeveron Announces Positive Type B Meeting With FDA Regarding Pathway to BLA for Laromestrocel in Alzheimer’s Disease

Longeveron Inc. recently announced the positive outcome of a Type B meeting with the US FDA, supporting the advancement of laromestrocel (Lomecel-B), 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-B) 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-B) 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.

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.

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.

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.