OXFORD, UK – March 24, 2025 – Pathios Therapeutics Limited (“Pathios”), a biotech company focused on the development of first-in-class therapies for cancer, today announced the appointment of Sumeet Ambarkhane, M.D., as Chief Medical Officer (CMO). A physician by training, Dr. Ambarkhane possesses more than 20 years of experience across academia and the biopharmaceutical industry in areas spanning clinical care, early through late-stage drug development, regulatory and commercialization. In his new role with Pathios, he will guide the advancement of the company’s novel therapeutic programs, including PTT-4256, its lead internally discovered, oral, highly potent, and selective small molecule inhibitor of GPR65, which is currently being evaluated in a first-in-human clinical trial in advanced solid cancers.
Dr. Ambarkhane most recently served as CMO for Alligator Bioscience, where he provided medical leadership and direction for company’s preclinical and clinical pipeline of tumour-directed immuno-oncology drugs targeting 4-1BB and CD40 receptors and played a pivotal role in advancing mitazalimab towards Phase 3 in pancreatic cancer. Prior to joining Alligator Bioscience, Dr. Ambarkhane served as Senior Global Program Medical Director at MorphoSys AG, leading the clinical development for its hemato-oncology programs, including tafasitamab (Monjuvi®/Minjuvi®). Under his leadership, Monjuvi was granted breakthrough therapy designation and subsequently an accelerated approval by the U.S. Food and Drug Administration, in addition to conditional approval by European Medicines Agency. His career also includes a tenure at UCB Pharmaceuticals in roles of increasing responsibility across geographies supporting the company’s immunology and neurology drug development portfolio. Dr. Ambarkhane holds a bachelor’s degree in medicine and surgery, as well as medical doctorate degrees in medicine and clinical pharmacology from Seth G.S. Medical College and King Edward Memorial Hospital, University of Mumbai in India.
“As we continue to make rapid progress translating our leadership on GPR65 antagonism into high-value development programs, we are delighted that such a talented and experienced CMO has joined our team. We look forward to benefiting from Sumeet’s extensive drug development expertise as we maintain our momentum with the ongoing clinical trial of PTT-4256 in solid tumours,” said Paul Higham, Chief Executive Officer of Pathios. “We welcome Sumeet to Pathios and look forward to an exciting and productive 2025 with a management team that possesses an impressive breadth of skill and experience.”
“In today’s versatile and dynamic ecosystem, it is rare to find an opportunity to work on a truly differentiated and promising approach to treating disease. Pathios Therapeutics has trailblazed research and validated genetic analysis related to GPR65 inhibition and is the first and only company positioned to bring its genetically validated protective effect to patients in the form of a novel immunotherapy,” said Dr. Ambarkhane. “I am eager to work alongside the high-calibre team of scientists, entrepreneurs and executives at Pathios to continue to advance this promising technology and ultimately deliver much-needed breakthroughs for people with cancer.”
Pathios is pursuing a first-of-its-kind therapeutic approach that leverages the therapeutic potential of GPR65 antagonism. The company’s lead program, PTT-4256, is a clinical-stage, first-in-class oral small molecule GPR65 inhibitor that targets a fundamental biological mechanism that is co-opted by all solid tumours to foster immunosuppressive polarization of immune cells. GPR65, an acid-sensing G protein-coupled receptor, 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. Inhibition of GPR65 has been shown to rapidly upregulate anti-tumour natural killer and T cell genes while downregulating pro-tumour myeloid genes.
Pathios’ internal human genetic analysis provides insights that reductions in GPR65 function are associated with significantly improved survival across a range of solid tumour types, positioning it as a unique immuno-oncology 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 company is currently evaluating PTT-4256 in the RAISIC-1 trial (Relief of Acidic Immune Suppression in Cancer), a modular multi-part, multi-arm open-label Phase 1/2 clinical study in patients with a range of advanced solid tumours.
In addition to cancer, there are strong genetic associations between GPR65 activity and a variety of other immunologically mediated diseases, offering Pathios the opportunity to expand its research and development into other high-value therapeutic areas with significant unmet need. In addition, the company is currently investigating the potential of GPR65 inhibition as a therapeutic approach for neuroinflammatory diseases, driven by a wealth of genetic data connecting GPR65 activity to Alzheimer’s disease, vascular dementia, and Parkinson’s disease, among others.
About Pathios Therapeutics
Pathios is a 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. 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. Pathios is headquartered at the Innovation Centre on Milton Park, a key science precinct south of Oxford, UK, where together with Greywolf Therapeutics and ThirtyFiveBio it is part of Oxford location of the unique Molecule to Medicine (MTM) drug discovery and development ecosystem.
For more information, please visit www.pathios.com
Contacts:
Pathios Therapeutics
Paul Higham, CEO
+44 1235 644 960
Tom McCarthy, Executive Chair of Pathios Therapeutics and MTM
+44 1235 644 960
Vida Strategic Partners (on behalf of Pathios Therapeutics)
Tim Brons
+1 646 319 8981