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Endeavor BioMedicines Begins Tumor-Agnostic Phase II Trial Targeting Hedgehog Pathway

NEW YORK – Endeavor BioMedicines said Wednesday that the first patient with a solid tumor bearing a loss of function mutation in Patched-1 (PTCH1) has received its Hedgehog pathway inhibitor, taladegib, in a Phase II trial.

The tumor-agnostic, open-label trial underway will assess objective response rate in 44 advanced solid tumor patients receiving either a 200 mg or 300 mg dose of taladegib once a day in the first part of the study. Researchers will expand enrolment in the second stage of the trial based on their findings in the first stage. All patients in the study will have histologically or cytologically confirmed solid tumors with PTCH1 loss of function mutations.

Tumor mutations in the Hedgehog pathway occur in around 2 percent of all cancers and dysregulate downstream signaling, which allows cancer cells to proliferate and survive. Endeavor Chief Medical Officer Srikanth Pendyala said in a statement that between 30,000 and 40,0000 US cancer patients with such mutations may be eligible for a drug like taladegib each year. 

According to San Diego-based Endeavor, in prior human studies, taladegib had a better safety profile than comparable therapeutics in its class, and advanced cancer patients with a variety of tumor types responded to its drug at all doses.

Endeavor recently closed a $101 million Series B financing round to support advancement of its precision oncology pipeline, including taladegib and ULK1/2 inhibitor ENV-201. The company is also testing taladegib in idiopathic pulmonary fibrosis, and it is exploring ENV-201 as a therapy for KRAS-mutated tumors that are resistant to standard therapies.