The US Food and Drug Administration (FDA) has awarded a Regenerative Medicine Advanced Therapy (RMAT) designation to Longeveron’s Lomecel-B for treating mild Alzheimer’s Disease.
An RMAT designation provides the benefits of both fast track and breakthrough designations, thereby, allowing for accelerated approval based on surrogate or intermediate endpoints. It also allows for multiple meetings with the FDA to expedite drug development.
Following the news, Longeveron stock was up by over 20% in pre-market trading today, compared to the market close on 9 July.
Lomecel-B is made up of human mesenchymal stem cells, derived from the bone marrow of healthy human donors. It is being evaluated as a treatment for hypoplastic left heart syndrome, and ageing-related frailty, in addition to Alzheimer’s disease.
Lomecel-B met the primary safety endpoint in a Phase II CLERMIND trial (NCT05233774), which enrolled 50 patients with mild Alzheimer’s. There were three reported serious events within 30 days of Lomecel-B administration, one in each of three different treatment dose groups, compared to none in the placebo group.
There were no incidences of hypersensitivity, notable changes in laboratory evaluations and electrocardiogram (EKG), or cases of amyloid-related imaging abnormalities (ARIA). Additionally, no clinically asymptomatic microhaemorrhages were noted on MRI. The therapy also improved cognitive functions and reduced neuroinflammation assessed by diffusion tensor imaging, an MRI imaging technique.
The global market for Alzheimer’s treatments is expected to be worth over $15.9bn in 2030, as per a GlobalData report. The field is dominated by amyloid-targeting therapies, with Eli Lilly’s Kisunla (donanemab) being the latest FDA-approved therapy in this class.
GlobalData is the parent company of Pharmaceutical Technology.
The news comes as a welcome reprieve for the company after it discontinued a Phase II trial of Lomecel-B in ageing-related frailty in Japan, in February. The decision was a ‘strategic move’ by the Longeveron to focus on developing Lomecel-B in hypoplastic left heart syndrome, a rare congenital heart defect.
The company is evaluating Lomecel-B in a Phase IIb ELPIS II trial (NCT04925024) in hypoplastic left heart syndrome patients under 12 years of age. The trial plans to recruit 38 participants, with enrolment expected to be completed by the end of the year.
Stem cell treatments are a relatively new area in the Alzheimer’s treatment space. Regeneration Biomedical is evaluating its autologous stem cell treatment, RB-ADSC, in mild-to-moderate Alzheimer’s in a Phase I trial (NCT05667649).
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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