FDA Recognizes Lomecel-B Cell Therapy for Alzheimer Disease Ahead of New Data
Longeveron will present updated data from the phase 2a CLEAR MIND study at AAIC 2024 at the end of July.
Wa’el Hashad
Credit: California Life Sciences
The FDA has granted both Fast Track and Regenerative Medicine Advanced Therapeutic (RMAT) Designations to Longeveron's Lomecel-B investigational allogeneic medicinal signaling cell (MSC) therapy for the potential treatment of patients with Alzheimer disease (AD).1,2
“Fast Track designation is another important milestone for Longeveron and Lomecel-B™, which, along with the recent granting of RMAT designation, recognizes the critical need to quickly advance novel, safe and effective investigational treatments for AD, which has a devastating impact on patients and their families,” Wa’el Hashad, Chief Executive Officer, Longeveron, said in a statement.1 “AD is a neurodegenerative disorder that leads to progressive memory loss and death, and its tremendous impact on our aging population is exacerbated by a lack of therapeutic options that slow disease progression or improve cognitive function. We believe that Lomecel-B™, which demonstrated an overall slowing/prevention of disease worsening compared to placebo in the CLEAR MIND Phase 2a clinical trial, has the potential to become an important treatment option for physicians and patients, and we look forward to sharing the latest data with the Alzheimer’s Disease research and patient communities at AAIC 2024.”
Longeveron previously announced topline results from the phase 2a CLEAR MIND trial (NCT05233774) that demonstrated that it had met its primary end point of safety, with slowing of disease worsening in 50 patients aged 60-85 years old with a diagnosis of mild AD. At the end of the 39-week treatment period, investigators reported no new safety concerns, in addition to no cases of amyloid-related imaging abnormalities, clinically asymptomatic microhemorrhages on MRI, or notable changes in laboratory evaluations and electrocardiogram.3
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“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,” Joshua Hare, cofounder, Chief Science Officer and Chairman of the Board, Longeveron, said in another statement.2
Investigators also observed statistically significant improvements in the Composite AD Score (CADS) after 39 weeks in the 25 x 106 cells (25M) x 1 dose (P = .091) group vs placebo and for the pooled intervention groups (25M x 1 dose, 25M x 4 doses, 100 x 106 cells [100M] x 4 doses; P = .099). CAD combines the AD Assessment Scale- Cognitive subscale 13, AD Cooperative Study-Activities of Daily Living (ADCS-ADL), Clinical Dementia Rating-Sum of Boxes, and left hippocampal volume. For the 25M x 1 dose group, treated patients experienced a statistically significant slowing of disease progression in left hippocampal volume (P = .015) relative to placebo.3
“These study results with Lomecel-B™ are encouraging,” Jeffrey Cummings, MD, Vice Chair of Research, UNLV Department of Brain Health, said in a statement about the topline results.3 “The study met its primary safety endpoint and is supported by lack of deterioration in cognitive or atrophy signals. The efficacy observations are encouraging, and these results should be used as a foundation for further studies.”
Longeveron will present updated data from CLEAR MIND at the 2024 Alzheimer’s Association International Conference, July 28 to August 1, in Philadelphia, Pennsylvania.1 The company is also evaluating Lomecel-B for the potential treatment of aging-related frailty and hypoplastic left heart syndrome, the former indicationf or which a phase 2b completed has been completed and the latter for which is being evaluated in phase 2b study.
