Onchilles Pharma, a private biotech innovator focused on next-generation cytotoxic therapeutics, has announced that the US FDA has cleared its Investigational New Drug (IND) application for N17350, paving the way for first-in-human clinical studies in patients with advanced solid tumors.

 

N17350 is the company’s first-in-class tumor-directed therapeutic, designed to harness the ELANE pathway, an innate immune mechanism that selectively kills cancer cells while simultaneously 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, 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.”

 

Preclinical studies showed that N17350 effectively killed cancer cells across dozens of cell lines, patient-derived samples, and in vivo models, including chemotherapy-resistant and immunologically “cold” tumors. The drug not only induced cancer cell death but also stimulated CD8+ T cell–mediated immune activation, supporting its broad evaluation in humans.

 

“The preclinical data supporting N17350 suggest a mechanism that kills tumors and simultaneously primes the immune system,” said Alain P Algazi, 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 upcoming first-in-human trial will assess the safety, tolerability, and preliminary clinical activity of N17350 as a monotherapy in advanced solid tumors—including melanoma, head and neck cancers, squamous cell carcinoma of the skin, non-small-cell lung carcinoma, and triple-negative breast cancers (ClinicalTrials.gov Identifier: NCT07339176). 

 

The study will also measure pharmacodynamic biomarkers tied to ELANE pathway engagement and immune activation. Onchilles plans to begin dosing at multiple sites in the US and Australia.

 

“N17350 is fundamentally different from traditional cytotoxic chemotherapies or immunotherapies,” said Court R. Turner, 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.”