Longeveron's Lomecel-B, a living cell product, has shown promising results in the Phase IIa CLEAR MIND clinical trial (NCT05233774) for the treatment of mild Alzheimer's Disease. The trial met key primary and secondary endpoints, demonstrating an overall slowing of disease worsening compared to placebo.
The findings, recently presented at the Alzheimer's Association International Conference (AAIC), highlight Lomecel-B's potential to address the underlying pathology of Alzheimer's Disease. The study enrolled 48 patients aged 60-85 with mild Alzheimer's, assessing the safety and efficacy of Lomecel-B versus placebo.
Safety and Efficacy
The CLEAR MIND trial established a favorable safety profile for Lomecel-B, with no incidence of hypersensitivity or infusion-related reactions. Furthermore, no cases of amyloid-related imaging abnormalities (ARIA) were reported.
Efficacy results included a statistically significant improvement relative to placebo in the Alzheimer's Disease Cooperative Study Activities of Daily Living (ADCS-ADL) at week 39. This suggests a positive impact on the daily functioning of patients with mild Alzheimer's.
Impact on Brain Volume
Lomecel-B demonstrated a reduction in brain volume loss in areas associated with Alzheimer's Disease, including the total brain volume (TBV), hippocampus, ventricles, and thalamus. Notably, there was a statistically significant improvement in left hippocampal volume compared to placebo. The treatment also led to a 20-30% reduction in left and right ventricular enlargement, respectively.
Expert Commentary
"As a medicinal signaling cell therapy that has multiple potential mechanisms of action to address inflammatory responses in the brain, Lomecel-B offers potential to address the underlying pathology of Alzheimer’s Disease without the limitations of previous therapies," said Wa’el Hashad, chief executive officer at Longeveron. He added that the company is encouraged by the safety profile and efficacy evidence, which support the differentiated therapeutic potential of Lomecel-B.
Trial Design and Patient Population
The Phase IIa CLEAR MIND trial was a randomized, placebo-controlled study involving 48 patients with mild Alzheimer's Disease. Participants had a Mini-Mental State Examination (MMSE) score of 18-24 and brain MRI and positron emission tomography (PET) scans consistent with Alzheimer's Disease. 36 patients received Lomecel-B, while 12 received a placebo.
Regulatory Status and Future Directions
The FDA has granted Lomecel-B both Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation for the treatment of mild Alzheimer’s Disease. Longeveron plans to meet with the FDA to review the data and discuss the future development path for Lomecel-B in Alzheimer’s Disease.
"We believe these results provide important validation of both the safety and therapeutic potential of Lomecel-B in the treatment of mild Alzheimer’s Disease and suggest, with further clinical evaluation, that this cellular therapy has the potential to have a positive impact on patients afflicted with this devastating disease," said Joshua Hare, MD, co-founder, chief science officer, and chairman at Longeveron.
