Onchilles Pharma Presents at AACR 2026 New Preclinical Data from the Systemically Delivered NEU-002 Program, Supporting Advancement Toward Development Candidate Selection
Onchilles Pharma, a privately held biotechnology company focused on advancing next-generation cancer therapeutics targeting the ELANE pathway, has unveiled new preclinical findings from its NEU-002 program at the prestigious American Association for Cancer Research Annual Meeting 2026. The data, presented in a scientific poster session, highlight the continued translational progress of NEU-002, an engineered therapeutic elastase platform designed for systemic administration. The results underscore the therapy’s ability to generate robust anti-tumor activity across multiple preclinical solid tumor models when delivered via both intravenous (IV) and intraperitoneal (IP) routes.
The announcement represents a significant milestone in the company’s strategy to expand the applicability of ELANE pathway-based therapies beyond localized tumor targeting toward a broader, systemically delivered approach. By doing so, Onchilles aims to address a critical unmet need in oncology: effectively treating solid tumors that are inaccessible or less responsive to localized delivery methods.
According to Court R. Turner, Co-Founder and Chief Executive Officer of Onchilles Pharma, the NEU-002 program was specifically designed to overcome limitations associated with tumor-directed delivery. He emphasized that the therapy seeks to extend the reach of ELANE-mediated cancer cell killing to a wider range of solid tumor indications that remain inadequately treated with current modalities. Importantly, he noted that the preclinical data presented at AACR demonstrate meaningful anti-tumor activity following both IV and IP administration, supporting the feasibility of a systemic treatment paradigm.
Turner further highlighted that NEU-002 is engineered not only to directly eliminate tumor cells but also to preserve and potentially enhance the immune system’s capacity to mount a durable anti-tumor response. This dual mechanism—combining direct cytotoxicity with immune activation—could position the therapy as a powerful tool for achieving long-term disease control, a goal that has remained elusive for many patients with advanced cancers.
The preclinical findings presented at AACR provide compelling evidence supporting the therapeutic potential of NEU-002. In studies using CT26 colon cancer models, candidates from the NEU-002 program demonstrated effective tumor biodistribution and significant anti-tumor activity following systemic administration. These results are particularly notable given the challenges typically associated with delivering biologically active enzymes like elastase in the systemic circulation, where they are often neutralized by endogenous inhibitors.
In a CT26 flank tumor model, intravenous administration of a NEU-002 candidate led to complete tumor clearance in all treated animals. This striking outcome was further reinforced by rechallenge experiments, in which the treated animals exhibited durable protection against subsequent tumor reintroduction. Such findings are indicative of long-term immune memory, suggesting that the therapy not only eliminates existing tumors but also primes the immune system to recognize and respond to future cancer threats.
In a separate CT26 visceral model, designed to mimic more advanced and disseminated disease, intraperitoneal administration of a NEU-002 candidate resulted in a significant reduction in ascites formation and overall tumor burden. These results highlight the potential applicability of NEU-002 in treating cancers that spread within the abdominal cavity, such as ovarian and gastrointestinal malignancies, where treatment options are often limited and prognosis remains poor.
A key innovation underlying the NEU-002 program lies in its molecular engineering. The therapy has been specifically designed to protect elastase activity from inactivation by circulating serine protease inhibitors, which typically limit the effectiveness of such enzymes when administered systemically. By overcoming this biological barrier, Onchilles has enabled the therapeutic to retain its functional activity in the bloodstream, thereby expanding its reach to tumors throughout the body.
These advances represent a critical step forward in translating the ELANE pathway from a localized therapeutic strategy into a systemic platform capable of addressing a wide spectrum of solid tumors. The company has indicated that two lead candidates from the NEU-002 program have been selected for further development. These candidates will undergo additional preclinical evaluation, including pharmacokinetic studies in non-human primates, to support the selection of a final development candidate and eventual progression into clinical trials.
At the core of Onchilles Pharma’s scientific approach is the ELANE pathway, a unique cancer-selective mechanism that exploits a shared vulnerability among many cancer cell types—namely, elevated levels of histone H1. By targeting this vulnerability, ELANE-based therapies can selectively induce cancer cell death while sparing normal cells. Moreover, the process of tumor cell destruction is designed to be immunogenic, meaning it stimulates the body’s immune system to recognize and attack cancer cells.
The company’s pipeline is anchored by N17350, its first-in-class clinical-stage candidate currently being evaluated in early human studies. This program represents the initial application of ELANE pathway targeting in a tumor-directed format. Building on this foundation, the NEU-002 program seeks to extend the same mechanism into a systemic delivery approach, thereby broadening its therapeutic reach.
Together, N17350 and NEU-002 embody a novel paradigm in cancer treatment—one that integrates targeted cytotoxicity with immune system activation. Unlike traditional chemotherapy, which often causes widespread damage to healthy tissues, or certain immunotherapies that rely heavily on pre-existing immune activity, ELANE-based therapies aim to directly kill cancer cells while simultaneously initiating a robust and adaptive immune response.
This approach has the potential to transcend many of the limitations associated with current cancer treatments. Because it targets a fundamental biological characteristic shared by diverse tumor types, the ELANE pathway may be effective regardless of a tumor’s genetic mutations, anatomical location, or immune environment. This universality positions Onchilles Pharma’s platform as a potentially transformative advancement in oncology.
Founded with a mission to redefine cancer treatment, Onchilles Pharma is focused on developing first-in-class cytotoxic therapies that are both highly selective and immunologically active. By leveraging cutting-edge protein engineering and a deep understanding of cancer biology, the company aims to deliver therapies that not only eradicate tumors but also provide lasting protection against recurrence.
The data presented at AACR 2026 mark an important step in this journey, demonstrating the feasibility and promise of systemic ELANE pathway targeting. As the NEU-002 program advances toward clinical development, it holds the potential to expand treatment options for patients with solid tumors and to contribute to a new era of precision oncology—one defined by therapies that are both potent and enduring in their effects.
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. Our pipeline is led by N17350, our first-in-class, clinical-stage program, followed by NEU-002, the second program that extends this approach with systemic delivery.
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 gamechangers 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 (ClinicalTrials.gov Identifier: NCT07339176), and the NEU-002 program for systemic delivery, extends the reach of N17350 to address all solid tumors.
Source Link:https://www.businesswire.com/
