XYone Therapeutics Doses First Patient With XYA02 ADC

Canton, Massachusetts, U.S., September 29, 2026

XYA02 Targets MUC1-C in Solid Tumors

XYA02 is a novel MUC1-C-targeting ADC engineered with a proprietary antibody linked to an Exatecan payload. MUC1-C is a tumor-associated protein expressed across several solid tumor types, making it a potential target for cancer-directed therapies. According to XYone, MUC1-C remains anchored to tumor cells, while the MUC1-N domain can be shed from the cell surface, providing a scientific rationale for selectively targeting the MUC1-C domain with an ADC approach. The company is developing XYA02 as part of a broader oncology pipeline focused on MUC1-C-directed therapeutic modalities. The clinical program is designed to investigate XYA02 across multiple advanced solid tumors. The study includes patients with non-small cell lung cancer, ovarian cancer, gastric and gastroesophageal junction cancers, and colorectal cancer. Initial treatment will involve dose escalation, followed by additional evaluation in selected tumor types based on emerging safety, pharmacokinetic, biomarker and antitumor activity data. The registered study is identified as NCT07670312 and is planned as a Phase 1b/2, multicenter, non-randomized, open-label, multiple-dose first-in-human investigation.

Phase 1b/2 Trial Evaluates Safety and Activity

The first-patient dosing milestone initiates the clinical evaluation of XYA02 in humans. The study’s primary development objectives include establishing the safety and tolerability profile of the investigational ADC and identifying dose levels suitable for further clinical testing. Researchers will also evaluate pharmacokinetic and pharmacodynamic characteristics and look for preliminary evidence of antitumor activity. These early-stage findings will help guide decisions about subsequent development of XYA02 in specific cancer populations. The clinical trial is expected to enroll approximately 190 participants across advanced solid-tumor populations. The registered protocol covers MUC1-expressing advanced, relapsed or refractory solid tumors, including colorectal, gastric, gastroesophageal junction, pancreatic, ovarian and non-small cell lung cancers. The trial is being conducted in Australia, with participating research centers including facilities in Sydney and Melbourne, according to publicly available trial information.

XYone Advances MUC1-C Oncology Pipeline

XYone Therapeutics said the first patient dosing represents the company’s progression into the clinical-stage development of XYA02 following years of research focused on MUC1-C-targeted therapies. The company has also reported that the U.S. FDA granted Orphan Drug Designation for XYA02 in pancreatic cancer and gastric cancer, with additional applications pending for other indications. Orphan Drug Designation can provide regulatory development incentives for therapies intended for rare diseases or conditions.

However, XYA02 remains an investigational drug and has not been approved by the FDA, the Australian Therapeutic Goods Administration or any other regulatory authority. Its safety and efficacy have not yet been established. The ongoing Phase 1b/2 study will therefore be important for generating the initial human clinical data needed to determine whether the MUC1-C-targeting ADC can advance into later-stage development.

Source: XYone Therapeutics press release