First-in-class therapeutic, N17350, demonstrates potent and selective tumor killing in chemotherapy-naïve and chemotherapy-treated ovarian cancer patient-derived models

Onchilles Pharma, a private biotech company pioneering next-generation cytotoxic therapeutics that harness the ELANE pathway, today announced the presentation of new preclinical data for its lead drug candidate, N17350, at the American Association for Cancer Research^® Annual Meeting 2025 (“AACR”). The presentation highlights N17350’s superior and selective tumor-killing ability compared to standard-of-care chemotherapies in ovarian cancer models, including chemotherapy-resistant tumors, derived from patients.

This data builds on the growing body of evidence supporting N17350’s novel mechanism of action and its potential to become a next-generation cytotoxic therapeutic. Unlike traditional chemotherapies, which indiscriminately damage both cancer and healthy immune cells, N17350 leverages elevated levels of histone H1 isoforms found in cancer cells to selectively kill tumors while sparing healthy cells. The results show that N17350 maintains its efficacy across various tumor types and treatment histories.

“We are excited to share this compelling data at AACR, which further reinforces the potential of N17350 to address unmet needs across cancers, with the potential to make a significant impact in chemotherapy-resistant disease,” said Lev Becker, Ph.D., Founder and Chief Scientific Officer of Onchilles Pharma. “These results demonstrate the strength of leveraging the ELANE pathway to therapeutically target tumor heterogeneity and overcome limitations of current cytotoxic therapies.”

Highlights from the AACR Presentation Include:

• Broad Efficacy: N17350 was effective in killing cancer cells derived from treatment-naïve, neoadjuvant chemotherapy-treated (NACT), and recurrent ovarian tumors.
• Selective Cytotoxicity: While standard chemotherapies were equally toxic to both cancer and immune cells, N17350 preserved immune cell viability while inducing immunogenic cell death in cancer cells.
• In Vivo Superiority: In cell-derived xenograft (CDX) models from ovarian cancer patients, N17350 outperformed carboplatin in inducing tumor regression.
• No Resistance: N17350 maintains potency following repeat dosing and effectively kills chemotherapy-resistant cancer cells.

“These new data exemplify the power of the ELANE pathway, and we believe therapeutics such as N17350 could represent a fundamentally new class of cancer treatments,” said Court R. Turner, J.D., Co-Founder and Chief Executive Officer of Onchilles Pharma. “We remain focused on translating these compelling preclinical results into clinical benefit as we prepare to enter the clinic later this year.”

N17350 is currently advancing toward first-in-human clinical trials, with an initial focus on skin, head and neck, triple-negative breast, and lung cancers. These new data support its continued development and highlight its promise as a differentiated cytotoxic modality with potential applications across a broad range of solid tumors.

The AACR poster presentation, titled “N17350 outperforms cytotoxic agents in killing tumors of treatment-naïve and chemotherapy-treated ovarian carcinoma patients,” will be available in Poster Section 21 (Poster Board Number: 30, Presentation Number: 861) from 2pm to 5pm CT on Monday, April 28th. The poster will also be available to meeting attendees via the AACR virtual platform beginning at 1pm ET today.

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 targets an essential vulnerability shared by all cancer cells: elevated histone H1 levels. 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. 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.

Looking Ahead: Clinical Validation in 2025

Onchilles Pharma plans to initiate a Phase 1 trial of its lead candidate, N17350, at the end of 2025 to validate the ELANE pathway in humans. This trial will further explore the safety and efficacy of this innovative mechanism, representing a significant step toward realizing the potential of this next-generation therapeutic modality across many cancer types.

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 biologic drug candidates aimed at treating various cancers, such as head & neck, skin, breast, and lung cancers, with plans to begin first-in-human trials in 2025. For more information, visit www.onchillespharma.com.

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“These results demonstrate the strength of leveraging the ELANE pathway to therapeutically target tumor heterogeneity and overcome limitations of current cytotoxic therapies," said Lev Becker, Ph.D., Founder & Chief Scientific Officer of Onchilles Pharma.