Pathios Therapeutics – Transforming Cancer Treatment through Human Genetics and Immune Innovation

Pathios Therapeutics is a leading UK-based biotechnology research company at the forefront of immuno-oncology innovation. Established in 2017, the company has built a strong reputation for combining advanced genetics, pharmacology, and immunology to redefine how cancers are understood and treated. By leveraging the body’s own immune responses through genetic insight, Pathios Therapeutics has emerged as a major contributor to the next generation of targeted cancer therapies.

Founded by Dr Tom McCarthy (Executive Chair) and Dr Stuart Hughes (Founding CEO and current Chief Scientific Officer), the company operates from its headquarters in Milton Park, Abingdon, Oxfordshire, United Kingdom. It can be contacted via +44 1235 644 960, and its official website is www.pathiostherapeutics.com.

Pathios Therapeutics: A Vision Rooted in Genetic Science

The central mission of Pathios Therapeutics is clear—to use human genetics to transform cancer outcomes. Unlike traditional oncology companies that primarily focus on killing cancer cells, Pathios Therapeutics targets the immune microenvironment surrounding tumours. This approach enhances the body’s natural ability to fight disease rather than simply suppressing it.

Their research focuses on GPR65, an acid-sensing G-protein coupled receptor (GPCR) found in immune cells. In tumour environments, acidity often causes immune cells to lose their ability to combat cancer. Pathios Therapeutics’ scientific breakthrough lies in inhibiting GPR65 to reverse this immunosuppression. This enables the immune system to recover its natural ability to destroy malignant cells, creating a paradigm shift in immuno-oncology.

Scientific Focus: Understanding GPR65 and the Tumour Microenvironment

The Science Behind GPR65

The tumour microenvironment is typically acidic, and this acidity suppresses the immune system’s response. Pathios Therapeutics discovered that the receptor GPR65 plays a critical role in this process. When activated by acidity, it alters the function of immune cells—particularly T-cells and macrophages—turning them into supporters of tumour growth rather than fighters against it.

By developing selective inhibitors that block this receptor, Pathios aims to restore proper immune function within tumours. This not only enhances immune system performance but also increases the effectiveness of other treatments, such as checkpoint inhibitors and immunotherapies.

The Lead Candidate: PTT-4256

The company’s flagship drug candidate, PTT-4256, is a potent, orally active small-molecule inhibitor of GPR65. It represents the first clinical programme in the world targeting this specific receptor. PTT-4256 is currently undergoing Phase 1/2 clinical trials under the study name RAISIC-1, focusing on advanced solid tumours.

If successful, PTT-4256 could represent a revolutionary treatment pathway—one that strengthens the immune system’s natural defences rather than replacing them. This scientific foundation positions Pathios Therapeutics as a pioneer in acid-sensing GPCR research, an area largely unexplored in human immunology until now.

Innovation in Action: The Role of Human Genetics

Using Genetic Insights to Drive Drug Discovery

Pathios Therapeutics’ research is deeply rooted in human genetic data. Their investigations revealed that certain genetic variants of GPR65—such as the I231L mutation—are linked with improved cancer survival rates. This finding reinforces the company’s hypothesis that inhibiting GPR65 may lead to better therapeutic outcomes for patients.

By combining genomics, transcriptomics, and functional immunology, Pathios builds a strong, data-driven understanding of how the immune system behaves in acidic conditions. This genetic-based approach not only improves treatment precision but also reduces the risks associated with trial-and-error drug development.

Broad Potential Beyond Oncology

While Pathios Therapeutics’ primary focus is cancer, the company also sees potential applications of GPR65 inhibition in neuroinflammatory and neurodegenerative diseases. Research into cell types such as microglia suggests that GPR65 plays a role in conditions like Alzheimer’s and Parkinson’s disease. Thus, Pathios may eventually expand its pipeline into broader immunological and neurological therapies.

Corporate Structure and Partnerships

Leadership and Founders

The company’s leadership reflects decades of experience in biotechnology and pharmaceutical innovation.

• Dr Tom McCarthy, Co-Founder and Executive Chair, brings deep experience in building biotech companies through the M:M Bio ecosystem, which integrates flexible infrastructure and expert guidance.

• Dr Stuart Hughes, Founding CEO and current Chief Scientific Officer, has been instrumental in shaping the scientific direction of the company, focusing on immune regulation and molecular biology.

Together, they have assembled a team of world-class researchers, pharmacologists, and clinical strategists to drive innovation from concept to clinic.

Investors and Strategic Alliances

Pathios Therapeutics is backed by a strong consortium of investors, including Canaan Partners, Brandon Capital Partners, and Bristol Myers Squibb. These investors bring not only capital but also strategic expertise in global drug development and commercialisation.

Additionally, the company is part of the M:M Bio ecosystem, which offers a collaborative framework that allows biotech start-ups to grow efficiently while maintaining scientific integrity and operational agility.

Funding and Milestones

In April 2024, Pathios announced the first close of its Series B funding round, raising approximately US$25 million. This funding, led by Bristol Myers Squibb, is aimed at supporting the clinical advancement of PTT-4256. The capital will also help accelerate preclinical programmes in neuroinflammatory research.

In November 2024, the company achieved another significant milestone by dosing the first patient in its Phase 1/2 trial. Earlier, in 2023, Pathios received a £567,000 grant from Innovate UK to explore GPR65 inhibition in brain tumour models. These milestones highlight a clear trajectory toward clinical validation and global relevance.

Pathios Therapeutics Headquarters and Operations

Pathios operates from Milton Park, Abingdon, Oxfordshire, one of the UK’s most prominent biotechnology and life-science hubs. The company’s official address is 99 Park Drive, Milton, Abingdon, OX14 4RY, United Kingdom.

Milton Park provides access to a dynamic community of innovation-driven companies, world-class laboratory facilities, and close proximity to the University of Oxford’s research network. This strategic location enhances collaboration opportunities and talent acquisition across the UK’s growing biotech landscape.

Future Outlook: Transforming the Immuno-Oncology Landscape

Pathios Therapeutics is on a clear mission to redefine cancer therapy through immune restoration. By focusing on the underlying biology of immune dysfunction rather than the tumour alone, the company introduces a fundamentally new therapeutic philosophy.

As its GPR65 inhibitor advances through clinical stages, Pathios could potentially open new doors for personalised cancer medicine. The use of genetic markers to guide therapy design may improve treatment accuracy and patient survival rates. Moreover, its exploratory work in neuroinflammation demonstrates a long-term vision that goes beyond oncology.

With continued investor confidence, a solid scientific foundation, and a leadership team driven by innovation, Pathios Therapeutics stands poised to become a global pioneer in immunogenetic therapies.

FAQs

Conclusion: A New Era in Cancer Research

Pathios Therapeutics exemplifies how science, genetics, and human ingenuity can converge to reshape medicine. Founded in 2017 and based in Milton, Oxfordshire, this company has already achieved remarkable progress within a short span.

By combining deep human genetic research, cutting-edge pharmacology, and strong industry partnerships, Pathios is crafting a future where the immune system becomes humanity’s greatest ally against cancer and other complex diseases. The company’s unwavering focus on GPR65 inhibition, powered by data-driven discovery, could well mark the dawn of a new era in precision oncology.

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