FDA Clears IND for Tumor-Directed Therapeutic Candidate in Solid Tumors

N17350, a first-in-class biologic, is the subject of an investigational new drug (IND) application cleared by the FDA for the treatment of patients with advanced solid tumors, according to a press release from the developer, Onchilles Pharma. The clearance enables the company to initiate a first-in-human phase 1/2 trial to evaluate the safety, tolerability, and preliminary activity of the novel agent, which leverages a previously untapped immune pathway.

“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,” Lev Becker, PhD, co-founder and chief scientific officer of Onchilles Pharma, stated in the press release.¹ “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 can evaluate in the clinic a differentiated, cancer-selective mechanism that overcomes limitations of current treatment paradigms.”

As a first-in-human study, clinical efficacy data for N17350 are not yet available; however, preclinical findings supporting the IND application suggest a potent and selective antitumor mechanism. N17350 is designed to mimic the natural immune surveillance ELANE pathway. The ELANE pathway is a unique cancer-selective killing mechanism that leverages elevated histone H1 levels.

Preclinical models demonstrated that the agent could induce rapid cancer cell death across a broad range of solid tumor types, regardless of the tumor’s underlying genetic mutations or existing resistance to standard-of-care therapies. This broad-spectrum potential position N17350 as a possible treatment for a variety of high-unmet-need cancers.

“The preclinical data supporting N17350 suggest a mechanism that kills tumors and simultaneously primes the immune system,” added Alain P. Algazi, MD, the director and program leader of Head and Neck Medical Oncology at UCSF.¹ “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.” Algazi is also the Onchilles N17350 Clinical Advisory Board Chair.

The phase 1/2 trial (NCT07339176) is an open-label, dose finding and dose expansion study designed to characterize the safety and preliminary antitumor activity of N17350.² The trial will be conducted in 2 distinct parts. Part 1, the dose finding phase, focuses on identifying the maximum tolerated dose and the recommended phase 2 dose. Part 2, the expansion phase, will further evaluate the safety and efficacy of the agent at the identified therapeutic dose in specific tumor cohorts.

The treatment regimen involves the administration of N17350 via intravenous infusion, including a dose range from 1 mg/ml to 4 mg/ml, on a standardized schedule, every second week for 8 or 12 weeks.

Eligibility criteria require participants to have advanced or metastatic solid tumors that have progressed despite standard curative or palliative therapies. Tumor types included are melanoma, head and neck neoplasms, squamous cell carcinoma of the skin, non–small cell lung cancer, and triple-negative breast cancer. Patients also had an ECOG performance status of 0 or 1, adequate recovery from prior therapy, and adequate hematologic, hepatic, and renal function, as defined by laboratory parameters.

Exclusion criteria include patients with history of solid organ transplant, a history of significant cardiovascular disease, or those who have received prior systemic anticancer therapy within 21 days or five half-lives before the first dose.

The primary end points of the study are the incidence of dose-limiting toxicities and the overall safety and tolerability profile of N17350, including the frequency and severity of adverse events. Secondary end points include the characterization of pharmacokinetic parameters, such as the area under the curve, maximum plasma concentration, and half-life. The study will also evaluate preliminary antitumor activity as a secondary objective, utilizing the objective response rate and duration of response according to RECIST v1.1 criteria.

The study will specifically look for infusion-related reactions and potential immune-mediated toxicities, given the agent’s role in modulating innate immune responses. The initiation of this phase 1 study marks the start of a clinical journey to determine if N17350 can translate its promising preclinical activity into a clinical benefit for patients with advanced cancer.

“N17350 is fundamentally different from traditional cytotoxic chemotherapies or immunotherapies,” Court R. Turner, JD, co-founder and chief executive officer of Onchilles Pharma, added.¹ “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.”

References

1. Onchilles Pharma announces IND clearance for N17350. News release. Onchilles Pharma. January 2, 2026. Accessed February 3, 2026. https://tinyurl.com/mu45az68
2. Intratumoral N17350 in Advanced Solid Tumors (OP-NEU-101). ClinicalTrials.gov. Updated January 14, 2026. Accessed February 3, 2026. https://tinyurl.com/2mjevj33