US-based Cassava Sciences has announced that oral therapy simufilam slowed cognitive decline by 38% versus placebo over six months in patients with mild to moderate Alzheimer’s disease in a Phase II study.
The company has announced positive topline results from its Cognition Maintenance Study (CMS). The CMS (NCT04994483) is a small proof-of-concept study designed to demonstrate the effects of drug versus placebo in a randomised withdrawal trial design. The study enrolled 157 patients with mild-to-moderate Alzheimer’s disease, a more advanced and difficult-to-treat stage of the illness.
In the Phase II, double-blind, placebo-controlled randomised study, all patients first received open-label simufilam 100mg for 12 months. Patients were then randomised 1:1, with one group receiving simufilam 100mg and the other receiving placebo for six months.
The pre-specified cognitive endpoint was mean change in Alzheimer’s Disease Assessment Scale-Cognitive (ADAS-Cog)11 scores over six months, drug versus placebo. ADAS-Cog11 is a cognitive subscale that measures language and memory. It is also useful for assessing the presence and progression of dementia.
Slowed decline of 38%
A slowed cognitive decline of 38% was reported in patients treated with simufilam for six months compared to placebo. The placebo arm declined 1.5 points on ADAS-Cog and at all measured timepoints. The simufilam arm declined 0.9 points on ADAS-Cog, a 38% difference in favour of the drug at month six. Drug effects favoured patients with mild disease.
Cassava president and CEO Remi Barbier said: “Patients started out taking open-label simufilam for 12 months prior to enrolling in the CMS. CMS patients on placebo were, in effect, withdrawn from simufilam for six months.
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“This placebo arm declined while the CMS arm randomised to simufilam improved. We believe the emerging separation of cognitive scores between these two arms represents a drug effect.”
How simufilam works
The pipeline drug acts as a filamin A inhibitor, binding to the filamin A protein with high affinity to prevent amyloid beta (42) toxic cascade. The candidate is yet to be approved by a regulatory authority.
GlobalData predicts a sales forecast of simufilam of $32m in 2029. GlobalData is the parent company of the Clinical Trials Arena.
Cassava Sciences is also evaluating simufilam tablets for Alzheimer’s disease dementia in two Phase III clinical studies. The Phase III programme is enrolling approximately 1,750 patients suffering from mild-to-moderate Alzheimer’s symptoms that meet other study eligibility criteria. Both upcoming studies have received a special protocol assessment (SPA) from the US Food and Drug Administration (FDA).
In May, Eli Lilly announced the results of the Phase III trial for their candidate Donanemab which slowed clinical decline by 35% compared to placebo and resulted in 40% less decline in the ability to perform activities of daily living.