Unesbulin (PTC596) Clinical Trials

Size	Price	Qty
2mg	USD 179
5mg	USD 357

Clinical Trials

Several clinical trials have evaluated Unesbulin (PTC596) across Phase 1, Phase 2, and Phase 3 development for advanced solid tumors, high-grade serous ovarian cancer, leiomyosarcoma, and pediatric brain malignancies, such as diffuse intrinsic pontine glioma and high-grade glioma. Initiated by academic institutions and industry sponsors including PTC Therapeutics, Nationwide Children's Hospital, and the University of Oklahoma, study statuses range from completed early-phase safety evaluations to active ongoing protocols and a terminated pivotal trial.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT05269355	TERMINATED	Leiomyosarcoma	PTC Therapeutics	2022-05-23	PHASE2; PHASE3
NCT03605550	ACTIVE_NOT_RECRUITING	High Grade Glioma; Diffuse Intrinsic Pontine Glioma	Nationwide Children's Hospital	2018-08-01	PHASE1
NCT03761095	COMPLETED	Leiomyosarcoma	PTC Therapeutics	2019-03-13	PHASE1
NCT03206645	COMPLETED	Ovarian Cancer	University of Oklahoma	2017-08-28	PHASE1
NCT03605550	Active not recruiting	High Grade Glioma\|Diffuse Intrinsic Pontine Glioma	Nationwide Children''s Hospital\|PTC Therapeutics	2018-08-01	Phase 1
NCT02404480	COMPLETED	Cancer	PTC Therapeutics	2016-01	PHASE1

(data from https://clinicaltrials.gov, updated on 2025-06-13)

Check the Unesbulin (PTC596) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Unesbulin (PTC596) functions as a second-generation BMI-1 inhibitor that accelerates the degradation of the polycomb group protein BMI-1, leading to the downstream depletion of the anti-apoptotic protein MCL-1. This targeted depletion induces p53-independent mitochondrial apoptosis, driving cytotoxic action against chemoresistant cancer stem cells to suppress tumor growth in clinical indications such as leiomyosarcoma and high-grade gliomas.

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>).