Lomecel-B granted FDA RMAT status for mild Alzheimer’s
Designation follows results from Phase 2a clinical trial
The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to Longeveron’s experimental cell therapy Lomecel-B for mild Alzheimer’s disease.
The designation is designed to accelerate the development and review processes for regenerative medicine candidates, including cell therapies, intended to treat, modify, reverse, or cure serious or life-threatening conditions. It provides therapy developers certain benefits, including early interactions with the FDA and potential priority review once the therapy is submitted for approval.
The designation was based on results from the CLEAR MIND Phase 2a clinical trial (NCT05233774) showing Lomecel-B led to an overall slowing or prevention of disease progression compared with a placebo. Full trial results will be presented at the 2024 Alzheimer’s Association International Conference, held July 28-Aug. 1 in Philadelphia and online.
“The RMAT designation is an important milestone for Longeveron and the Lomecel-B program that recognizes the potential of our cellular therapy to have a positive impact on patients afflicted with this devastating disease,” Joshua Hare, MD, co-founder, chairman, and chief science officer at Longeveron, said in a company press release.
“[W]e look forward to meeting with the FDA to discuss the path forward and the development plans for Alzheimer’s Disease in the very near future,” said Nataliya Agafonova, MD, Longeveron’s chief medical officer.
Reducing brain inflammation, damage
Alzheimer’s is a progressive neurodegenerative disorder characterized by declining mental abilities, including memory and cognitive function, as well as mood and personality changes. Therapeutic options are currently very limited.
Lomecel-B is a medicinal signaling cell, or MSC, therapy based on stem cells derived from the bone marrow of healthy adult donors. Preclinical studies suggest that Lomecel-B may address multiple hallmarks of Alzheimer’s, reducing brain inflammation and damage associated with the disease progression. The therapy may also improve the function of blood vessels in the brain and promote regenerative responses.
In a Phase 1 trial (NCT02600130), the cell therapy was generally well tolerated and slowed cognitive decline in people with mild Alzheimer’s, when compared with a placebo. The therapy was also linked to increased levels of anti-inflammatory markers and was shown to improve patients’ quality of life.
The CLEAR MIND trial evaluated the treatment’s safety and efficacy in 49 adults ages 60-85 with mild Alzheimer’s disease, who were randomly assigned to receive four monthly Lomecel-B infusions at two different doses, or a placebo.
Top-line data demonstrated the treatment was generally safe and well-tolerated, with no treatment-related serious adverse events reported.
The treatment was also shown to significantly improve scores on the Composite Alzheimer’s Disease Score (CADS) assessment, a validated measure of cognitive function, dementia, and ability to perform daily life activities. Patients given the highest dose also experienced significant life quality improvements and less brain volume loss.
Lomecel-B was also associated with significantly less volume loss in the hippocampus — a brain region critical for memory that is strongly affected in Alzheimer’s — as well as lower levels of brain inflammation.
“The trial achieved the primary safety and secondary efficacy endpoints and showed statistically significant improvements in pre-specified clinical and biomarker endpoints in specific Lomecel-B groups compared to placebo,” Hare said.
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