Zatolmilast (BPN14770) Clinical Trials

Size	Price	Qty
2mg	USD 159
5mg	USD 317

Clinical Trials

Zatolmilast (BPN14770) is evaluated across multiple Phase 1 through Phase 3 clinical trials sponsored by organizations including Tetra Discovery Partners, Shionogi, and the National Institute of Mental Health. Primary indications under investigation include Fragile X Syndrome, Alzheimer's disease, depression, and PPP2R5D neurodevelopmental disorder (Jordan's Syndrome), alongside Phase 1 assessments of brain target occupancy, safety, and pharmacokinetics in healthy participants and individuals with hepatic or renal impairment. Recruitment statuses for these studies encompass completed, active not recruiting, enrolling by invitation, and withdrawn.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT06717438	ACTIVE_NOT_RECRUITING	Jordan's Syndrome; PPP2R5D Neurodevelopmental Disorder	Shionogi	2025-05-13	PHASE2
NCT05367960	ACTIVE_NOT_RECRUITING	Fragile X Syndrome	Tetra Discovery Partners	2022-11-01	PHASE3
NCT07012005	COMPLETED	Renal Impairment	Shionogi	2025-08-25	PHASE1
NCT07018492	COMPLETED	Hepatic Impairment	Shionogi	2025-08-30	PHASE1
NCT05163808	COMPLETED	Fragile X Syndrome	Tetra Discovery Partners	2022-03-29	PHASE2; PHASE3
NCT07011992	COMPLETED	Healthy Participants	Shionogi	2025-07-18	PHASE1
NCT05358886	COMPLETED	Fragile X Syndrome	Tetra Discovery Partners	2022-11-01	PHASE3
NCT05367960	Enrolling by invitation	Fragile X Syndrome	Tetra Discovery Partners	2022-11-01	Phase 3
NCT03569631	COMPLETED	Fragile X Syndrome; FXS; Fra(X) Syndrome	Tetra Discovery Partners	2018-07-09	PHASE2
NCT04044781	WITHDRAWN	To Determine Brain Target Occupancy of BPN14770	Tetra Discovery Partners	2020-01-10	PHASE1
NCT03861000	COMPLETED	Depression	National Institute of Mental Health (NIMH)	2019-03-12	PHASE1; PHASE2
NCT03817684	COMPLETED	Alzheimer's Disease	Tetra Discovery Partners	2019-04-30	PHASE2
NCT03030105	COMPLETED	Alzheimer Disease	Tetra Discovery Partners	2017-01	PHASE1
NCT02840279	COMPLETED	Alzheimer's Disease	Tetra Discovery Partners	2016-06	PHASE1
NCT02648672	COMPLETED	Alzheimer's Disease	Tetra Discovery Partners	2015-12	PHASE1

(data from https://clinicaltrials.gov, updated on 2026-06-09)

Check the Zatolmilast (BPN14770) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Zatolmilast acts as a selective allosteric inhibitor of phosphodiesterase 4D, specifically targeting PDE4D3 and PDE4D7 with IC50 values of 7.4 nM and 7.8 nM to suppress the enzymatic hydrolysis of cyclic adenosine monophosphate and restore downstream intracellular signaling cascades. This elevation of intracellular cyclic adenosine monophosphate promotes synaptic plasticity and neuronal network function, providing the biological mechanism for treating cognitive and neurodevelopmental deficits in clinical trial conditions such as Fragile X Syndrome and 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>).