Cassava Sciences, Inc. (NASDAQ: SAVA) is a clinical-stage biotechnology company headquartered in Austin, Texas, focused on developing novel therapeutics for Alzheimer's disease and other neurological disorders. The company's lead candidate, simufilam, represents a novel approach targeting the filamin A protein, which is involved in synaptic function and neuronal survival[1].
Founded in 2004, Cassava Sciences has dedicated its research to understanding the underlying of Alzheimer's disease pathogenesis. The company's approach differs from traditional amyloid-targeting strategies, focusing instead on restoring synaptic function through modulation of filamin A^2].
| Program | Target/Mechanism | Indication | Phase | Status |
|---|---|---|---|---|
| Simufilam (PTI-125) | Filamin A modifier | Alzheimer's Disease | Phase 3 | Recruiting |
| Simufilam | Filamin A modifier | Alzheimer's Disease | Phase 3 | Active |
| PTI-125-DS | Filamin A modifier | Alzheimer's Disease | Phase 2 | Completed |
Simufilam is a small molecule drug that binds to an altered form of the filamin A protein found in Alzheimer's disease brains. This protein plays a critical role in maintaining synaptic structure and function. In Alzheimer's disease, filamin A becomes modified, leading to disrupted synaptic signaling and neuronal dysfunction^3].
Mechanism of Action:
Rationale:
The filamin A target represents a novel mechanism distinct from amyloid and tau approaches:
Clinical Development:
Phase 2 Results:
The Phase 2b randomized, double-blind, placebo-controlled study showed:
Phase 3 Program (RECLAIM-Alz):
Filamin A is a large actin-binding protein that organizes the cytoskeleton and serves as a scaffold for numerous signaling . In Alzheimer's disease:
Key Findings:
Cassava Sciences has developed biomarker assays to support clinical development:
| Biomarker | Purpose | Status |
|---|---|---|
| p-tau181 | Tau pathology marker | Validated |
| Neurofilament light chain | Neurodegeneration | Validated |
| Synaptic | Synaptic health | In development |
| Partner | Focus Area | Status |
|---|---|---|
| NIA | Government funding | Active |
| Academic collaborators | Research | Multiple |
| CRO partners | Clinical trials | Active |