Genome&Company "Approval for Change in Clinical Phase 2 Plan of 'GEN-001' for Bile Duct Cancer"

"Expectations for Ensuring Efficacy of Combination Therapy"

Genome&Company announced on the 2nd that it has received approval for the amendment of the domestic Phase 2 clinical trial plan (IND) for the microbiome immuno-oncology drug 'GEN-001' targeting cholangiocarcinoma patients.

Genome&Company received approval for the domestic Phase 2 clinical trial plan (IND) for the combination therapy of GEN-001 and Merck's 'Keytruda' in patients with advanced refractory cholangiocarcinoma in November last year. Subsequently, in April, the company submitted an application for approval of changes to the Ministry of Food and Drug Safety.

The change in the clinical trial plan was made following the approval last year of the combination therapy of the immuno-oncology drug 'Imfinzi' and the chemotherapy drugs 'Gemcitabine and Cisplatin (GemCis)' as the first-line standard treatment for cholangiocarcinoma, after additional consultations with Merck, the company explained.

With the change in clinical design, Cohort 3 was added to the existing two cohorts. The added study will evaluate a triple combination therapy of GEN-001, Keytruda, and 'mFOLFOX (fluorouracil, leucovorin, oxaliplatin)'. This is because there is currently no second-line standard treatment for cholangiocarcinoma, and mFOLFOX is mainly prescribed for patients who did not respond to the first-line standard treatment. The purpose is to confirm the efficacy and safety of GEN-001 by conducting the triple combination therapy including mFOLFOX.

Cohort 3 will be conducted on 42 cholangiocarcinoma patients who did not respond to the first-line standard treatment. GEN-001 will be administered to 21 patients initially, and based on the interim analysis results, the decision will be made whether to recruit an additional 21 patients. The Phase 2 clinical trial of GEN-001 for cholangiocarcinoma will involve up to 148 patients, with the first patient dosing scheduled for the second half of this year.

Jisoo Bae, CEO of Genome&Company, said, "Genome&Company changed the clinical design through consultations with Merck in accordance with the revised cholangiocarcinoma treatment guidelines last year," adding, "We will devote all efforts to research and development to produce positive results in the Phase 2 trial so that the combination therapy of GEN-001 can establish itself as the second-line standard treatment for cholangiocarcinoma."