Lomecel-B becomes first cell therapy to receive RMAT designation for the treatment of mild Alzheimer’s.
Shares in longevity biotech Longeveron jumped significantly this week after the company announced that its investigational therapy for the treatment of mild Alzheimer’s has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the US FDA. According to the company, this marks the first time a cellular therapeutic candidate has received RMAT designation for Alzheimer’s.
The FDA’s RMAT designation aims to expedite the development and review of promising regenerative medicine products. To qualify, a therapy must show preliminary clinical evidence of its potential to treat, modify, reverse, or cure a serious or life-threatening condition and address unmet medical needs.
Longeveron’s Lomecel-B is an allogeneic medicinal signaling cell therapy derived from the bone marrow of young, healthy adult donors – a so-called “living cell product.” The therapy is being explored for multiple conditions, including Alzheimer’s, aging-related frailty and hypoplastic left heart syndrome (HLHS).
Longeveron CEO Wa’el Hashad said the RMAT designation allows the company to have “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.”
In a Phase 2a clinical trial for mild Alzheimer’s, Lomecel-B demonstrated positive outcomes, showing a significant dose-response improvement in cognitive function. Patients treated with Lomecel-B exhibited statistically significant improvements in the Montreal Cognitive Assessment (MOCA) and Mini-Mental State Examination (MMSE) scores compared with the placebo group, and caregivers reported an enhanced quality of life for those receiving the treatment.
“In the CLEAR MIND Phase 2a clinical trial, Lomecel-B demonstrated an overall slowing/prevention of disease worsening compared to placebo,” said Joshua Hare, co-founder and Chairman of Longeveron. “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.”
In the MRI biomarker study, Lomecel-B was shown to counteract brain volume loss in areas typically affected by Alzheimer’s. The therapy was associated with a 49% reduction in whole brain volume loss and significant reductions in left hippocampal volume loss, as well as decreases in left and right ventricular enlargement by 20% and 33%, respectively. Neuroinflammation levels were lower in all Lomecel-B doses compared with the placebo, and cerebral blood flow was improved.
The full results of the CLEAR MIND study will be presented at the 2024 Alzheimer’s Association International Conference later this month.
