Pathios Therapeutics Announces Dosing of First Patient in Phase 1/2 Clinical Study of GPR65 Inhibitor PTT-4256 in Patients with Advanced Solid Cancers

Company's First-in-Class, Oral, Highly Potent, and Selective Small Molecule Inhibitor of GPR65 Designed to Reverse Low pH-Induced Immunosuppression in the Acidic Tumour Microenvironment

MELBOURNE, Australia and OXFORD, United Kingdom, Nov. 20, 2024 /PRNewswire/ -- Pathios Therapeutics ("Pathios"), a biotech company focused on the development of first-in-class therapies for cancer, today announced 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. PTT-4256 is an internally discovered, oral, highly potent, and selective small molecule inhibitor of the pH-sensing GPR65.

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.

About Pathios Therapeutics
Pathios is a clinical stage drug discovery and development company focused on translating innovative science into new medicines. Pathios was founded by a team of experienced biotech and pharmaceutical industry professionals, entrepreneurs, and clinicians. The company is focused on developing small molecule inhibitors of the pH-sensing G protein-coupled receptor GPR65 to counteract the GPR65 mediated immunosuppressive polarization of immune cells, present in most cancers. To date, Pathios has raised a total of US$58M including support from the leading venture capital firms, Canaan and Brandon Capital, as well as a strategic investment from Bristol Myers Squibb.  The Pathios UK team is headquartered at the Innovation Centre on Milton Park, a key science precinct south of Oxford, UK, where together with Grey Wolf Therapeutics and ThirtyFiveBio it is part of the Oxford "pod" of this unique MTM drug discovery and development ecosystem.

For more information, please visit www.pathios.com.

Contacts:
Paul Higham, CEO
 +44 1235 644 960
[email protected]

Pathios Therapeutics
Tom McCarthy, Executive Chair
+44 1235 644 960
[email protected]

Vida Strategic Partners (on behalf of Pathios Therapeutics)
Tim Brons
+1 646 319 8981
[email protected]

SOURCE Pathios Therapeutics