reMYND announces positive Phase 2a clinical trial results for treatment of mild-to-moderate Alzheimer’s disease
reMYND, a biotech company developing treatments for neurodegenerative disorders like Alzheimer’s and Huntington's disease, announces promising results from its Phase 2a clinical trial investigating septin modulation in patients with mild-to-moderate Alzheimer’s disease. The trial participants demonstrated significant improvement on memory tests, marking an important milestone in the pursuit of effective Alzheimer’s disease treatments.
The Phase 2a trial assessed the safety, efficacy, and tolerability of REM0046127. This small molecule drug acts as a molecular glue, restoring the integrity of septin filaments in neurons. Septin filaments regulate calcium homeostasis, but are impaired in doing so in the context of Alzheimer's disease, leading to neurotoxic calcium concentrations and widespread cell death (as further detailed in reMYND’s publication in Science last May).
In Alzheimer’s mouse models, REM0046127 has a neuroprotective effect. It restores neuronal communication as well as cognition, and mitigates the brain pathology typically observed in Alzheimer’s brains—amyloid-beta plaques and tau tangles. That is why reMYND has now conducted a randomized, placebo-controlled, double-blind Phase 2a study to evaluate the safety, tolerability and pharmacodynamics of REM0046127 in a small group of participants with mild-to-moderate Alzheimer’s disease.
Participants were treated for 4 weeks with either placebo (4 participants) or different daily doses of REM0046127 (8 participants). Treated participants showed marked improvement in memory retrieval, as well as restoration of brain activity as measured by EEG, and increased dopamine levels in the cerebrospinal fluid.
“REM0046127 treatment for 28 days improved activity and function of neuronal pathways underlying memory in Alzheimer’s patients. Markers of the tau pathology typical for Alzheimer’s were also mitigated by the compound, indicating that restoration of septin filaments does not only confer fast symptomatic benefit, but also has potential disease-modifying effects,” said Gerard Griffioen, CSO at reMYND.
The trial was terminated early due to off-target effects that limited the therapeutic window of the investigational drug. Griffioen explains: “Our findings highlight the therapeutic potential of septin modulators for Alzheimer’s patients. The occurrence of off-target effects, however, warrants follow-up studies with an optimized lead compound to minimize side effects and ensure a more favorable therapeutic profile for long-term use.”
Following the encouraging efficacy results, reMYND plans to bring an improved molecule to the clinic next year. In doing so, the company is moving closer to a much-needed therapy for millions of Alzheimer’s patients across the globe.