Emvododstat (PTC299) Clinical Trials

Size	Price	Qty
5mg	USD 390

Clinical Trials

Emvododstat has been evaluated in several Phase I, Phase II, and Phase III clinical trials targeting oncological, infectious, and genetic conditions, including acute myeloid leukemia, neurofibromatosis type II, metastatic breast cancer, HIV-related Kaposi's sarcoma, central nervous system tumors, and COVID-19 pneumonia. Sponsored by industry, defense, and academic entities—including PTC Therapeutics, the United States Department of Defense, the AIDS Malignancy Consortium, and the Pediatric Brain Tumor Consortium—these studies comprise both completed and terminated trials, alongside a completed Phase Ib study in metastatic breast cancer.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT04439071	TERMINATED	Pneumonia; COVID-19; Coronavirus	PTC Therapeutics	2020-07-09	PHASE2; PHASE3
NCT03761069	TERMINATED	Leukemia, Myeloid, Acute; AML	PTC Therapeutics	2018-10-29	PHASE1
NCT03761069	Terminated	Leukemia Myeloid Acute\|AML	PTC Therapeutics	2018-10-29	Phase 1
NCT01158300	COMPLETED	Brain and Central Nervous System Tumors	Pediatric Brain Tumor Consortium	2010-11	PHASE1
NCT00911248	TERMINATED	Neurofibromatosis 2	PTC Therapeutics	2009-07-31	PHASE2
NCT00508586	COMPLETED	Metastatic Breast Cancer	PTC Therapeutics	2007-11	PHASE1
NCT00704821	TERMINATED	Advanced Cancer	PTC Therapeutics	2008-07-03	PHASE1
NCT00686842	TERMINATED	Kaposi's Sarcoma	AIDS Malignancy Consortium	2008-09	PHASE1; PHASE2
NCT00911248	Terminated	Neurofibromatosis 2	PTC Therapeutics\|United States Department of Defense	2009-07-31	Phase 2
NCT00704821	Terminated	Advanced Cancer	PTC Therapeutics	2008-07-03	Phase 1

(data from https://clinicaltrials.gov, updated on 2023-06-26)

Check the Emvododstat (PTC299) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Emvododstat acts as a dual inhibitor that targets dihydroorotate dehydrogenase (DHODH) and post-transcriptionally suppresses VEGFA mRNA translation, thereby depleting intracellular pyrimidine nucleotide pools and blocking hypoxia-induced VEGF protein production. This combined biochemical blockade inhibits de novo pyrimidine biosynthesis and tumor angiogenesis to suppress abnormal cellular proliferation, providing therapeutic rationale for its evaluation in clinical trials for acute myeloid leukemia, solid tumors, and viral infections.

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>).