Phase 2a results demonstrated laromestrocel (Lomecel-B™) improved cognitive function, quality of life, and brain volume in the treatment of mild Alzheimer’s disease
This encouraging clinical trial data adds to the body of evidence supporting the safety of single and multiple doses of laromestrocel (Lomecel-B™) treatment for mild Alzheimer’s disease and provides indications of efficacy in combating decline of brain volume and potentially cognitive function
Laromestrocel (Lomecel-B™) is, to Company’s knowledge, the first cellular therapeutic candidate to receive U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) designation for Alzheimer’s disease
MIAMI, March 11, 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 publication in Nature Medicine of results from the CLEAR MIND Phase 2a clinical trial evaluating laromestrocel (Lomecel-B™) as a potential cellular therapy for mild Alzheimer’s disease (AD). The article is also available online here. The International Nonproprietary Names (INN) Expert Committee of the World Health Organization recently approved “laromestrocel” for the non-proprietary name of Lomecel-B™.
“This Nature Medicine publication reinforces laromestrocel’s safety and efficacy as a potential treatment for mild Alzheimer’s disease and paves the way for more advances in utilizing cell therapy for Alzheimer’s disease,” said Joshua Hare, M.D., Founder and Chief Science Officer of Longeveron. “As a mesenchymal stem cell therapy that has multiple potential mechanisms of action to address inflammatory responses in the brain, laromestrocel offers a new potential strategy to address the underlying pathology of Alzheimer’s disease without the limitations of previous therapies. Cell based therapy with MSCs is a particularly attractive treatment candidate as it encompasses pro-vascular, immunomodulatory, and tissue repair mechanisms of actions, with findings validated in a murine Alzheimer’s disease model.”
The randomized, placebo-controlled Phase 2a CLEAR MIND trial evaluated a total of 48 patients (36 were treated with laromestrocel (Lomecel-B™) and 12 received placebo) who were 60-85 years old and had a diagnosis of mild AD in accordance with National Institutes of Health – Alzheimer’s Association (NIA-AA) criteria, a Mini-Mental State Examination (MMSE) score of 18-24, and a brain MRI and positron emission tomography (PET) scan consistent with AD. The clinical trial achieved its primary safety and secondary efficacy endpoints, and the study results being accepted for publication in Nature Medicine support the therapeutic potential of laromestrocel (Lomecel-B™).
Key findings include:
The trial safety evaluations supported that laromestrocel (Lomecel-B™) is safe and well tolerated in the study population, in both single and multiple dosing regimens, and that patients showed no incidence of hypersensitivity, infusion-related reactions, and no cases of amyloid-related imaging abnormalities (ARIA).
Patients treated with laromestrocel (Lomecel-B™) showed an overall slowing of disease worsening compared to placebo.
Positive efficacy results were demonstrated via a change from baseline at week 39 of the trial at prespecified levels using the Composite Alzheimer’s Disease Score (CADS) – a secondary outcome measure that combines information across cognitive, functional capacity, and brain MRI domains.
Administration of laromestrocel (Lomecel-B™) was associated with slowing cognitive and functional decline as demonstrated by statistically significant results in the Montreal Cognitive Assessment and statistical trending improvements compared to placebo in CDR-SB and MMSE.
There was a statistically significant improvement relative to placebo observed in the Alzheimer’s Disease Cooperative Study Activities of Daily Living (ADCS-ADL).
Patients treated with laromestrocel (Lomecel-B™) showed minimized loss in brain volume in areas associated with AD (total brain volume, Hippocampus, Temporal and Frontal lobes, and Thalamus), statistically significant relative to placebo. Along with positive changes in brain volumes, there was 20-30% reduction in left and right ventricular enlargement, respectively.
Diffusion tensor imaging (a form of MRI) supports the concept that laromestrocel (Lomecel-B™) has the potential to reduce neuroinflammation compared to placebo.
Laromestrocel (Lomecel-B™) treated patients demonstrated a numerical improvement relative to placebo in quality of life observed by caregivers and measured by the Alzheimer’s Disease Related Quality of Life (ADRQOL) and Quality of life AD (QOL-AD) scales.
“Alzheimer’s disease is a disorder of neurocognition with a major unmet medical need. We believe these results provide important validation of both the safety and therapeutic potential of laromestrocel in the treatment of mild Alzheimer’s disease and suggest, with further clinical evaluation, that this cellular therapy has the potential to have a positive impact on patients afflicted with this devastating disease,” said Nataliya Agafonova, M.D., Chief Medical Officer at Longeveron. “We look forward to our planned meeting with the FDA in March to review this data and discuss the future development path for laromestrocel (Lomecel-B™) in Alzheimer’s disease.”
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 the FDA during laromestrocel’s (Lomecel-B™) development 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 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.
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