Drug Profile

PTT 4256

Alternative Names: PTT-4256

Latest Information Update: 24 Jul 2026

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At a glance

Originator Pathios Therapeutics
Class Antineoplastics; Small molecules
Mechanism of Action GPR65 protein inhibitors

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes

Highest Development Phases

Phase I/II Solid tumours

Most Recent Events

29 May 2026 Efficacy and adverse event data from a phase I/II trial in Solid tumours presented at the 62^nd Annual Meeting of the American Society of Clinical Oncology (ASCO-2026)
04 Nov 2024 Phase-I/II clinical trials in Solid tumours (Late-stage disease, Metastatic disease, Second-line therapy or greater) in Australia (PO) (NCT06634849)
18 Apr 2024 Pathios Therapeutics plans clinical trial in Solid tumor (PO) by the end of 2024

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