Clinical Trials

Multiple clinical trials sponsored by Cassava Sciences, Inc. have evaluated simufilam (PTI-125) across Phase 1, Phase 2, and Phase 3 research, with recruitment statuses recorded as either completed or terminated. While Phase 2 and Phase 3 studies primarily focus on Alzheimer's disease, Phase 1 evaluations in healthy volunteers have investigated basic clinical pharmacology, including drug absorption, metabolism, excretion, and hepatic insufficiency.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT05575076 TERMINATED
Alzheimer Disease
Cassava Sciences, Inc.
 2022-11-07 PHASE3
NCT05352763 TERMINATED
Alzheimer's Disease
Cassava Sciences, Inc.
 2022-05-12 PHASE2
NCT05026177 TERMINATED
Alzheimer Disease
Cassava Sciences, Inc.
 2021-11-18 PHASE3
NCT06390410 COMPLETED
Hepatic Insufficiency
Cassava Sciences, Inc.
 2024-06-26 PHASE1
NCT04994483 COMPLETED
Alzheimer Disease
Cassava Sciences, Inc.
 2021-11-03 PHASE3
NCT06195319 COMPLETED
Absorption; Metabolism; Excretion
Cassava Sciences, Inc.
 2024-02-07 PHASE1
NCT04388254 COMPLETED
Alzheimer Disease
Cassava Sciences, Inc.
 2020-03-24 PHASE2
NCT04932655 COMPLETED
Healthy Volunteers
Cassava Sciences, Inc.
 2021-05-04 PHASE1
NCT04079803 COMPLETED
Alzheimer Disease
Cassava Sciences, Inc.
 2019-09-09 PHASE2

(data from https://clinicaltrials.gov, updated on 2025-05-23)

Check the Simufilam product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Simufilam interacts with altered filamin A to disrupt its pathogenic binding with alpha-7 nicotinic acetylcholine receptors and stabilize the actin cytoskeleton, thereby suppressing downstream hyperphosphorylation of tau protein and preventing amyloid-beta aggregation. By reversing neurotoxic signaling cascades and maintaining cytoskeletal integrity, simufilam aims to mitigate neurodegeneration and cognitive decline in clinical trial populations with Alzheimer's disease.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.