Apilimod mesylate Clinical Trials

Size	Price	Qty
5mg	USD 147
25mg	USD 490

Clinical Trials

Multiple clinical trials sponsored by entities such as Synta Pharmaceuticals, OrphAI Therapeutics, and the National Institute of Allergy and Infectious Diseases have evaluated apilimod mesylate across diverse inflammatory, infectious, neurodegenerative, and oncological conditions. These Phase 1 and Phase 2 studies, along with expanded access protocols, assessed therapeutic efficacy, safety, and pharmacokinetics in Crohn's disease, COVID-19, C9ORF72-associated amyotrophic lateral sclerosis, frontotemporal dementia, relapsed non-Hodgkin lymphoma, and chronic lymphocytic leukemia. Recruitment for these trials has concluded, with statuses recorded 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 mesylate product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Apilimod mesylate selectively inhibits the lipid kinase PIKfyve and suppresses interleukin-12 (IL-12) and interleukin-23 (IL-23) production, thereby disrupting endolysosomal trafficking and pro-inflammatory signaling pathways. This cellular disruption impairs endosomal entry mechanisms and pathogenic cytokine-driven immune responses, establishing the biological foundation for its clinical evaluation in Crohn's disease, COVID-19, neurodegenerative disorders, 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>).