Pipeline
Advancing the First Selective Cytotoxic Therapies to the Clinic in a Broad Range of Solid Tumors
Onchilles’ lead programs—N17350 (NEU-001) for tumor-directed delivery and NEU-002 for intravenous administration—have the potential to transform cancer treatment by delivering broad tumor-killing capabilities with immune preservation, an unmet need not addressed by current cytotoxic agents or immunotherapies.
Onchilles Pharma’s N17350 (NEU-001, tumor-directed) and NEU-002 (IV-administered) are engineered to deliver potent, cancer-selective cytotoxicity. Unlike traditional chemotherapy, which indiscriminately destroys both cancerous and healthy tissues, these therapies specifically target cancer cells through the ELANE pathway, leveraging their inherent vulnerabilities to trigger powerful tumor regression.
Onchilles Pharma has built an extensive translational dataset spanning 11 distinct cancer types, over 50 genetically diverse cell lines, 41 patient samples, and more than 20 animal models – mirroring the key characteristics of the patients we plan to enroll in our upcoming Phase 1 clinical trial. This research demonstrates:
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Broad efficacy in CDX/PDX models derived from tumors resected from ovarian, breast, and colon cancer patients;
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Robust immune activation in immunologically cold, warm, and hot tumors, and
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Effectiveness against chemoresistant tumors and those that have progressed on checkpoint inhibitors
In addition, no resistance mechanism has emerged upon repeat dosing, reinforcing our confidence in translating these findings to patients.
Setting a New Benchmark in Oncology
Onchilles is advancing its pipeline in a broad spectrum of cancers, including head & neck, skin, breast, and lung, with first-in-human trials anticipated to begin in late 2025. These clinical studies will evaluate the potential of ELANE pathway-targeting therapeutics to eliminate tumors while preserving immune function, with a focus on patients who have developed resistance to chemotherapy or checkpoint inhibitors. By combining direct cancer cell killing with immune activation, Onchilles’ therapeutics could redefine the standard of care for a wide range of solid tumors.