On November 20, 2024 Pathios Therapeutics ("Pathios"), a biotech company focused on the development of first-in-class therapies for cancer, reported dosing of the first patient in the initial module of a Phase 1/2 clinical trial evaluating PTT-4256, the company’s investigational first-in-class GPR65 inhibitor (Press release, Pathios Therapeutics, NOV 20, 2024, View Source [SID1234648522]). PTT-4256 is an internally discovered, oral, highly potent, and selective small molecule inhibitor of the pH-sensing GPR65.
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The RAISIC-1 trial (Relief of Acidic Immune Suppression in Cancer) is a modular multi-part, multi-arm open-label study being conducted in Australia. The study’s first module is designed to evaluate the safety, tolerability, preliminary efficacy and pharmacokinetics of PTT-4256 monotherapy in patients with a range of advanced solid tumours. Subsequent modules will explore the potential of PTT-4256 in specific tumour types, including in combination with different standards of care.
"The transition of our first-of-its-kind GPR65 Inhibitor in immunooncology into the clinic represents a significant milestone for our company and is a validation of the promise of this approach and of the hard work of the entire Pathios team," said Paul G. Higham, Chief Executive Officer of Pathios. "This multi-module trial has been carefully designed to provide a range of valuable data on PTT-4256 that will be critical to our continued clinical advancement of the program. In addition to important safety, tolerability and pharmacokinetic findings, this clinical trial includes investigating the activity of this novel target and mechanism in humans for the first time through measuring disease response and exploring links to biomarkers designed to elucidate the consequences of target engagement."
Pathios is pursuing this novel immunooncology approach which focuses on counteracting the immunosuppressive polarization of immune cells, blocking a ubiquitous cancer immune evasion pathway through the targeting of GPR65, an acid sensing a G protein-coupled receptor that has been shown to foster immunosuppressive polarization of immune cells. GPR65 is exclusively expressed on immune cells and is associated with driving the immunosuppressive immune cell phenotype in the tumour microenvironment (TME) that prevents immune-mediated killing of cancer cells.
Pathios’ human genetic analysis demonstrates that reductions in GPR65 function are associated with significantly improved survival across a range of solid tumour types, positioning it as a unique immunooncology target for therapeutic intervention. Preclinical data for PTT-4256 have demonstrated that the compound possesses excellent drug-like properties and impressive monotherapy anti-tumour activity.
The RAISIC-1 trial is being run by the Australian-based team at Pathios Therapeutics Pty Ltd, in partnership with key Australian clinical trial sites and collaborators, contract research organisations and M:M Bio Pty Ltd, part of the global Molecule to Medicine (MTM) ecosystem of companies. The study has been approved by the applicable Australian Ethics Committees and is being conducted under the Australian Therapeutic Goods Administration’s (TGA’s) Clinical Trial Notification (CTN) scheme. It has been registered on the World Health Organisation recognised clinical trial registry ClinicalTrials.gov with trial identifier NCT06634849.