Onchilles Pharma Unveils New Preclinical Data for NEU-002 Targeting ELANE at SITC 2024

Onchilles Pharma, a private biotechnology company, revealed new preclinical findings from its NEU-002 program during the 39th Annual Meeting of the Society for Immunotherapy of Cancer (SITC), held virtually and in Houston, Texas, from November 6-10, 2024. The company focuses on developing pan-cancer therapeutics that utilize the ELANE pathway. This pathway is a unique innate immune mechanism that promotes the death of cancer cells without affecting healthy tissue, irrespective of the cancer’s type, genetic background, or location.

Onchilles Pharma has developed two leading therapeutic candidates, N17350 and NEU-002, based on the ELANE pathway. N17350 is designed for direct injection into tumors, while NEU-002 is formulated for intravenous (IV) administration. The company is preparing to start Phase 1 clinical trials for N17350 in 2025, targeting cancers such as head & neck, skin, breast, and lung cancers, to validate the ELANE pathway.

The preclinical data presented at the SITC meeting showcased NEU-002's ability to target the ELANE pathway systemically. NEU-002 aims to address the challenges associated with systemic delivery by overcoming restraints from serine protease inhibitors and enhancing substrate specificity. This improvement potentially allows NEU-002 to treat a wider range of solid tumors suitable for systemic administration.

Highlights from the preclinical data include:

1. Enhanced Specificity and Activity: NEU-002 maintains its full enzymatic function in plasma and demonstrates improved specificity towards its therapeutic target, CD95, while avoiding off-target substrates.
2. Selective Cancer Cell Killing: NEU-002 induces selective immunogenic cell death in primary cancer cells from ovarian cancer patients, confirming its targeted killing capability in human tissues.
3. Durable Responses: Preclinical studies in the CT26 tumor mouse model showed that NEU-002 treatment led to complete responses and resistance to tumor rechallenge, indicating potential for immune memory formation.

Dr. Lev Becker, the Scientific Founder and Chief Scientific Officer of Onchilles Pharma, emphasized the importance of these findings, noting that the ability to selectively target cancer cells while initiating a sustained immune response could offer durable clinical benefits across various tumors. Court R. Turner, Co-Founder and Chief Executive Officer, highlighted the transformative impact of N17350 and NEU-002, expressing enthusiasm for advancing N17350 into clinical trials in 2025 to provide clinical proof of concept for targeting the ELANE pathway.

The NEU-002 program originated from Dr. Lev Becker’s research, which identified the ELANE pathway as a novel mechanism where neutrophil elastase (ELANE) can selectively kill cancer cells while sparing healthy ones. These findings, published in 2021, laid the groundwork for developing NEU-002 for systemic IV delivery and N17350 for direct tumor injection. By targeting the ELANE pathway, these candidates offer a unique treatment approach that can be effective regardless of the cancer’s genetic background or location, positioning them as potential game-changers in oncology.

Looking ahead, Onchilles Pharma plans to launch a Phase 1 trial for N17350 in 2025 to verify the ELANE pathway's effectiveness in humans. This trial will further investigate the safety and efficacy of this innovative therapeutic mechanism, marking a significant step towards realizing the potential of ELANE-based treatments.

Onchilles Pharma is dedicated to discovering and developing drugs that exploit the ELANE pathway to create advanced, pan-cancer therapies. These pioneering medicines have the potential to revolutionize cancer treatment by selectively targeting tumors without regard to genetic background, anatomical origin, or immune status. The company's development pipeline includes biologic drug candidates for various cancers, including head & neck, skin, breast, and lung cancers, with plans to commence human trials in 2025.