Why Genome & Co. changes p2 trial IND for bile duct cancer drug?

Genome & Company is about to revise its strategy in developing a biliary tract cancer treatment candidate, GEN-001. The company plans to confirm the treatment effect of its combined use with cytotoxic chemotherapies by reflecting on the cancer’s secondary treatment trend.

In a public filing last Friday, the company said it has submitted applications for changing its investigational new drug (IND) plan for the domestic phase 2 clinical trial of GEN-001 to the Ministry of Food and Drug Safety.

Last November, Genome & Co. won the regulatory nod for the phase 2 clinical trial to treat progressive refractory bile duct cancer patients in Korea. GEN-001 is a microbiome-based immunotherapy the company is developing. Initially, Genome & Co. planned to evaluate its safety, drug resistance, and anti-tumor activity through a combined administration with MSD’s immune gate inhibitor Keytruda (pembrolizumab).

Through the IND change, the company plans to add one more cohort to the phase 2 clinical trial. Aside from the combo therapy with Keytruda, Genome & Co. will explore the safety and efficacy of the combined use of GEN-001, Keytruda, and mFORFOX (fluorouracil/leucovorin/oxaliplatin), anticancer chemotherapy.

The company targets up to 140 subjects and conducts the trial for about three years, beginning in August and continuing through October 2060.

Seoul National University Hospital and seven other institutions will be the sites.

The company will divide the phase 2 clinical trial into two substages to confirm its recommended doses of combined administration with Keytruda – part 1 is for safety introduction, and part 2 is the main clinical stage. The primary endpoint is the dose-limiting toxicity (DLT) occurrence rate and overall response rate (ORR).

“Secondary standard treatment has not been established for bile duct cancer patients. However, mFORFOX is most widely prescribed for patients who have failed in the standard primary treatment,” a company official said.

He went on to say, “In the upcoming trial, the company would add cohort 3 to be administered with pembrolizumab, mFORFOX, and GEN-001 to confirm GEN-001’s safety and efficacy when combined with pembrolizumab and explore its safety and efficacy when combined with both pembrolizumab and mFORFOX.