Apilimod (STA-5326) Clinical Trials

Size	Price	Qty
5mg	USD 147
25mg	USD 490

Clinical Trials

Several clinical trials have evaluated Apilimod across autoimmune, infectious, neurodegenerative, and oncological indications. Early investigations included a Phase 2 trial in Crohn's disease sponsored by Synta Pharmaceuticals Corp., NIAID, and the NIH, while subsequent Phase 1 and Phase 2 studies sponsored by OrphAI Therapeutics addressed non-Hodgkin lymphoma, chronic lymphocytic leukemia, COVID-19, and C9ORF72-associated amyotrophic lateral sclerosis, alongside an expanded access protocol for frontotemporal dementia. Across the trial portfolio, recruitment statuses are reported as completed or no longer available.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT05163886	COMPLETED	Amyotrophic Lateral Sclerosis; ALS	OrphAI Therapeutics	2021-12-23	PHASE2
NCT04446377	COMPLETED	COVID-19 Disease	OrphAI Therapeutics	2020-07-15	PHASE2
NCT02594384	COMPLETED	Lymphoma, Non-Hodgkin; Leukemia, Chronic Lymphocytic	OrphAI Therapeutics	2015-10	PHASE1
NCT00234741	Completed	Crohn''s Disease	Synta Pharmaceuticals Corp.\|National Institute of Allergy and Infectious Diseases (NIAID)\|National Institutes of Health (NIH)	2005-11	Phase 2

(data from https://clinicaltrials.gov, updated on 2025-06-22)

Check the Apilimod (STA-5326) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Apilimod selectively binds to and inhibits the lipid kinase PIKfyve while suppressing interleukin-12 (IL-12) and interleukin-23 (IL-23) expression, thereby disrupting phosphoinositide-regulated endolysosomal trafficking and downstream pro-inflammatory signaling cascades. This inhibition alters intracellular vesicular transport and attenuates pathological cytokine release, providing a strong rationale for its clinical investigation in inflammatory disorders such as Crohn's disease, viral infections like COVID-19, neurodegenerative diseases, and hematologic malignancies.

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>).