Onchilles Pharma
Now enrolling · Phase 1/2 study of intratumoral N17350 in advanced solid tumors · NCT07339176

Next-Generation Cytotoxic Therapeutics That Stimulate the Immune System by Selectively Killing Cancer Cells

Onchilles Pharma is developing first-in-class therapeutic elastases that harness the ELANE pathway. The result is broad, cancer-selective tumor killing that spares immune cells and activates systemic anti-tumor immunity.

12
distinct cancer types
30+
genetically diverse cell lines
19+
animal models
55
patient samples
0
resistance observed on repeat dosing

Preclinical data. N17350 is investigational.

The ELANE Pathway Unlocks a New Approach to Selective Cancer Killing

At the core of this approach is the ELANE pathway, a unique cancer-selective killing mechanism that targets altered histone H1 biology, an essential vulnerability shared by all cancer cells. By targeting the ELANE pathway and inducing immunogenic cancer cell death, N17350 (NEU-001) and NEU-002 are designed to rapidly eliminate tumors while mobilizing an adaptive immune response, offering the potential for sustained anti-tumor immunity.

Targeting the ELANE pathway enables the selective eradication of cancer cells regardless of their genetic makeup, anatomical origin, or immune status. Onchilles has rigorously validated this approach across 12 distinct cancer types, over 30 genetically diverse cell lines, and more than 19 animal models. This body of evidence supported the rapid clinical advancement of ELANE pathway-targeting therapies, now in first-in-human testing, as a fundamentally new class of cancer-selective cytotoxic treatments.

Mitochondrion with the histone H1 and death domain complex bound to its surface, from the Onchilles mechanism-of-action animation
Onchilles Pharma headquarters, Alexandria GradLabs, San Diego

About Onchilles Pharma

Onchilles Pharma is a clinical-stage biotechnology 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, targeted therapies, and immunotherapy.

The company's lead program, N17350 (NEU-001), a tumor-directed cytotoxic agent, is in the clinic. OP-NEU-101, a Phase 1/2 open-label, dose-finding and expansion study of intratumoral N17350 in advanced solid tumors, is enrolling at sites in the US and Australia (NCT07339176). Its intravenous counterpart, NEU-002, continues through preclinical development. With an extensive translational dataset validating its approach, Onchilles is leading the way in developing safer, more effective cytotoxic therapies that kill tumors specifically, spare healthy tissue, and activate the immune system for long-term anti-cancer immunity.

Illustration of a cancer cell with therapeutic elastase molecules gathering inside it

Onchilles Pharma is eliminating the trade-off between tumor-killing efficacy and immune preservation with a novel class of cytotoxic therapeutics that selectively destroy cancer cells to activate systemic anti-tumor immunity.

A New Class of Selective Cytotoxic Therapeutics with Unique Immune System Activation

Onchilles Pharma has established a leadership position in targeting the ELANE pathway, translating this approach into two proprietary molecules, N17350 (NEU-001) for intratumoral delivery and NEU-002 for systemic delivery. Both have the potential to transform cancer treatment by delivering broad tumor-killing capabilities while preserving and activating immune function. No current cytotoxic agent or immunotherapy meets this need.

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