Onchilles Pharma Presents New Preclinical Data for the Systemically Delivered NEU-002 Program Targeting the ELANE Pathway at SITC 2024

Novel Intravenous Therapeutic Candidate Demonstrates Pan-Cancer Efficacy and Immunogenic Cell Death in Preclinical Models

Onchilles Pharma, a private biotech company pioneering pan-cancer therapeutics that leverage the ELANE pathway, today announced the presentation of new preclinical data from its NEU-002 program at the 39th Annual Meeting of the Society for Immunotherapy of Cancer (SITC). The event is being held virtually and at the George R. Brown Convention Center in Houston, Texas from November 6-10, 2024.

The ELANE pathway is a novel innate immune mechanism that enables potent and selective immunogenic cell death of cancer cells irrespective of immunotype, genotype, anatomical origin while sparing healthy tissue. Onchilles Pharma has leveraged this pathway to develop N17350 and NEU-002, two leading therapeutic candidates designed for intratumoral and intravenous (IV) administration, respectively. N17350 is on track to start Phase 1 clinical trials in 2025 to validate the ELANE pathway in head & neck, skin, breast, and lung cancers.

At SITC 2024, Onchilles Pharma presented new preclinical data on NEU-002, which allows targeting the ELANE pathway through systemic delivery. NEU-002 addresses the challenges of systemic delivery by overcoming inhibition from serine protease inhibitors and enhancing substrate specificity, thereby extending its potential to treat a broad range of solid tumor types that are well-suited for systemic administration.

Key Preclinical Data Highlights:

• Enhanced Specificity and Activity: NEU-002 retains full enzymatic activity in plasma and shows improved substrate specificity, effectively cleaving CD95, its therapeutic target, while sparing known off-target substrates.
• Selective Cancer Cell Killing: The NEU-002 candidates selectively induce immunogenic cell death in primary cancer cells derived from ovarian cancer patients, validating their selective killing properties in primary human tissue.
• Durable Responses: In preclinical studies using the CT26 tumor mouse model, NEU-002 treatment generated complete responses with resistance to tumor rechallenge, suggesting the potential for immune memory formation.

"The data presented today mark a significant advancement in the systemic targeting of the ELANE pathway," said Lev Becker, Ph.D., Scientific Founder and Chief Scientific Officer of Onchilles Pharma. "The ability to selectively target cancer cells while inducing a sustained immune response could provide durable clinical benefits across a wide range of tumor types."

Court R. Turner, J.D., Co-Founder & Chief Executive Officer, added, "N17350 and NEU-002 represent a transformative approach in oncology, with the potential to redefine the standard of care. We are excited about advancing N17350 into a Phase 1 trial in 2025, providing the clinical proof-of-concept for targeting the ELANE pathway and delivering innovative cancer treatments to patients."

About the NEU-002 Program and the ELANE Pathway

Onchilles Pharma's NEU-002 program stems from groundbreaking research by its scientific founder, Dr. Lev Becker, who identified the ELANE pathway as a novel innate immune mechanism where neutrophil elastase (ELANE) selectively kills cancer cells while sparing healthy cells. Published in Cell in 2021, these findings have laid the foundation for developing NEU-002, which is tailored for systemic IV delivery, and N17350, designed for intratumoral administration. By leveraging the ELANE pathway, these candidates offer a unique approach to treat cancer regardless of genetic background, anatomical origin, or immunotype, positioning them as potential game-changers in cancer therapy.

Looking Ahead: Clinical Validation in 2025

Onchilles Pharma plans to initiate a Phase 1 trial of its lead candidate, N17350, in 2025 to validate the ELANE pathway in humans. This trial will further explore the safety and efficacy of this innovative mechanism, representing a significant step toward realizing the potential of ELANE-based therapies.

About Onchilles Pharma

Onchilles Pharma is a global drug discovery and development company focused on harnessing the ELANE pathway to create next-generation, pan-cancer therapeutics. These first-in-class medicines hold the potential to revolutionize cancer treatment by selectively targeting tumors, independent of genetic background, anatomical origin, or immune status. The company's pipeline includes biologic drug candidates aimed at treating various cancers, such as head & neck, skin, breast, and lung cancers, with plans to begin first-in-human trials in 2025. For more information, visit www.onchillespharma.com.

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