Longeveron Inc., a clinical stage regenerative medicine biotechnology company developing cellular therapies for certain life-threatening and chronic aging-related conditions, announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to Lomecel-B™ for the treatment of mild Alzheimer’s Disease. Lomecel-B™ is a proprietary, scalable, allogeneic cellular investigational therapy being evaluated across multiple indications, including Alzheimer’s Disease (Phase 2a completed), Aging-related Frailty (Phase 2b completed) and hypoplastic left heart syndrome (HLHS) (Phase 2b on-going). To the Company's knowledge based on publicly available information, Lomecel-B™ is the first cellular therapeutic candidate to receive RMAT designation for Alzheimer’s Disease.
“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,” said Joshua Hare, Co-founder, Chief Science Officer and Chairman of the Board at Longeveron. “Alzheimer’s Disease is a neurodegenerative disorder that leads to progressive memory loss and death and currently has very limited therapeutic options. In the CLEAR MIND Phase 2a clinical trial, Lomecel-B™ demonstrated an overall slowing/prevention of disease worsening compared to placebo. 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.”
Longeveron previously announced top-line results for the CLEAR MIND Phase 2a clinical trial on October 5, 2023, and reported additional clinical data and imaging biomarker results on December 20, 2023. Full study results for CLEAR MIND will be reported in a Featured Research Oral Presentation at the 2024 Alzheimer’s Association International Conference (AAIC) on July 28, 2024.
“We are thrilled to receive the RMAT designation for Lomecel-B™ for the treatment of mild Alzheimer’s Disease and we 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, M.D., Chief Medical Officer at Longeveron.
The RMAT designation is the fourth special regulatory designation received by Longeveron for Lomecel-B™. The Lomecel-B™ HLHS program has received three separate and distinct FDA designations: Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation.
“We are happy to receive this important RMAT designation as it continues to validate the work we have done so far and allows us to have the important dialogue with the FDA to advance our work and potentially bring this investigational therapeutic option to the many patients suffering from Alzheimer’s Disease,” said Wa’el Hashad, Chief Executive Officer and Board Member at Longeveron.
Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline regenerative medicine products, including cell therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition. Similar to Breakthrough Therapy designation, RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, and potential priority review of the biologics license application (BLA) if Priority Review designation is granted following BLA submission.
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