Onchilles Pharma Announces IND Clearance for N17350, Advancing the First Next-Generation Cytotoxic Therapeutic Leveraging the ELANE Pathway into the Clinic

SAN DIEGO--(BUSINESS WIRE)--Onchilles Pharma, a private biotech company pioneering next-generation cytotoxic therapeutics that harness the ELANE pathway, today announced the U.S. Food and Drug Administration (FDA) clearance of its Investigational New Drug (IND) application for N17350 to initiate first-in-human clinical studies in patients with advanced solid tumors. N17350, the company’s first-in-class tumor-directed therapeutic candidate, leverages the ELANE pathway, an innate immune mechanism that selectively kills a wide range of cancer cells while preserving and activating the immune system.

“IND clearance for N17350 marks an important milestone for Onchilles and reflects years of rigorous work establishing the ELANE pathway as a clinically actionable mechanism to improve the treatment of cancer,” said Lev Becker, Ph.D., Co-Founder and Chief Scientific Officer of Onchilles Pharma. “From the beginning, this work was driven by fundamental observations in patients. What emerged was a mechanism that broadly kills cancer cells while preserving and activating immune cells, thereby transforming cancer cell death into long-lasting anti-tumor immunity. We now have the opportunity to evaluate in the clinic a differentiated, cancer-selective mechanism that overcomes limitations of current treatment paradigms.”

N17350 demonstrated consistent efficacy in preclinical models spanning dozens of cancer cell lines, patient-derived samples, and multiple in vivo models, including chemotherapy-resistant and immunologically “cold” tumors, where it induced immunogenic cancer cell death and drove CD8+ T cell–mediated immune activation. These findings support its evaluation in a broad first-in-human clinical study.

“The preclinical data supporting N17350 suggest a mechanism that kills tumors and simultaneously primes the immune system,” said Alain P. Algazi, M.D., Onchilles N17350 Clinical Advisory Board Chair and Director, Program Leader, UCSF Head and Neck Medical Oncology. “If this dual activity translates clinically, it could represent a meaningful advance for patients with tumors that are difficult to treat with existing cytotoxic or immunotherapy approaches.”

The first-in-human study will evaluate the safety, tolerability, and preliminary clinical activity of N17350 as a monotherapy in patients with advanced solid tumors, including melanoma, head and neck neoplasms, squamous cell carcinoma of skin, non-small-cell lung carcinoma, triple-negative breast neoplasms (ClinicalTrials.gov Identifier: NCT07339176). The trial is also designed to assess pharmacodynamic biomarkers associated with ELANE pathway engagement and immune activation, providing early clinical insight into the mechanism. Onchilles plans to initiate first-in-human dosing at multiple sites in the U.S. and Australia.

“N17350 is fundamentally different from traditional cytotoxic chemotherapies or immunotherapies,” said Court R. Turner, J.D., Co-Founder and Chief Executive Officer of Onchilles Pharma. “By leveraging the ELANE pathway, we are advancing a cancer-selective therapeutic approach designed to eliminate tumors as well as preserve and train the immune system to respond at the same time. As we move into clinical testing, we believe this approach has the potential to address unmet needs across many different tumor types.”

About Onchilles Therapeutic Programs Targeting the ELANE Pathway

At the core of this approach is the ELANE pathway, a unique cancer-selective killing mechanism that leverages a vulnerability shared by many cancer cell types: elevated histone H1 levels. By targeting the ELANE pathway and inducing immunogenic cancer cell death, N17350 and NEU-002 are designed to rapidly eliminate tumors while mobilizing an adaptive immune response, offering the potential for sustained anti-tumor immunity. N17350 and NEU-002 offer a unique approach to treating cancer regardless of their genetic makeup, anatomical origin, or immune status, positioning them as potential game-changers in cancer therapy.

About Onchilles Pharma

Onchilles Pharma is a global drug discovery and development company pioneering first-in-class cytotoxic therapies designed to selectively kill cancer cells while preserving and activating immune function. By harnessing the ELANE pathway, these next-generation therapeutics are designed to deliver potent and selective tumor cell destruction, overcoming the limitations of traditional chemotherapy and immunotherapy. The company’s pipeline includes N17350, a tumor-directed lead candidate in first-in-human studies, and the NEU-002 program for systemic delivery which extends the reach of N17350 to address all solid tumors. For more information, visit www.onchillespharma.com.