Chimeric Therapeutics (ASX:CHM) has achieved a significant regulatory milestone, announcing it has secured FDA Orphan Drug Designation (ODD) for its lead candidate, CHM CDH17, in the treatment of gastric cancer.
The designation, granted for rare diseases with high unmet need, provides a suite of regulatory and commercial incentives including market exclusivity, fee reductions and scientific guidance from major regulators. Chimeric said the decision underscores the therapeutic promise of its technology and represents a significant validation of its clinical trajectory.
The company said it plans to intensify its global regulatory strategy, prioritising submissions and engagement in the United States, European Union and China, where orphan pathways can significantly streamline development and commercialisation. It said these markets collectively represent the greatest opportunity to bring its treatment to patients while establishing the company as a competitive player in the global rare-disease landscape.
“This is a great step forward in the development of CHM CDH17 to serve patients with gastric cancer, where there is a significant unmet need,” said Dr Rebecca McQualter, CEO of Chimeric Therapeutics.
The Phase 1/2 trial (NCT06055439) of CHM CDH17 is a two-stage study designed to determine a recommended Phase 2 dose and evaluate its safety and objective response rate in patients with advanced colorectal cancer, gastric cancer, and gastrointestinal NETs. CHM CDH17 is a third-generation, novel CAR-T cell therapy that targets CDH17, a cancer biomarker associated with poor prognosis and metastases in the most common gastrointestinal tumours. The Phase 1 portion of this study is expected to enrol up to 15 patients and lead to dose selection and expansion with indication-specific Phase 2 cohorts.