On May 25, 2022 Endeavor BioMedicines, a clinical-stage biotechnology company targeting the core drivers of terminal diseases including oncology and fibrosis, reported the first patient has been dosed in an open-label Phase 2 study evaluating ENV-101 (taladegib) in patients with advanced solid tumors harboring Patched-1 (PTCH1) loss of function mutations (Press release, Endeavor BioMedicines, MAY 25, 2022, View Source [SID1234615044]).

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"Dosing the first patient in the Phase 2 study of ENV-101 is a critical step for advancing medicines that treat cancer by attacking the genetic cause of disease," said John Hood, Ph.D., Co-Founder, CEO and Chairman of Endeavor BioMedicines. "The clinical effects of ENV-101, a small molecule inhibitor of the Hedgehog signaling pathway for the treatment of cancer have been evaluated in nearly 200 participants so far. ENV-101 has so far demonstrated exceptional ability to inhibit this oncogenic pathway and induce regression of tumors dependent on it. We hope this trajectory will continue as the drug advances through this next stage of clinical development."

The multi-center study employs a two-stage design to evaluate the efficacy and safety of ENV-101, a potent Hedgehog (Hh) pathway inhibitor, in patients with histologically or cytologically confirmed refractory advanced solid tumors characterized by loss of function (LOF) mutations in the PTCH1 gene. Endeavor expects to enroll 44 adults who are 18 and older in the first part of the study. Patients will receive 200 mg of ENV-101 once a day in one arm of the trial, and 300 mg of ENV-101 once a day in the other arm. The primary endpoint of the trial is Objective Response Rate (ORR) – comprised of Complete Response (CR) and Partial Response (PR), measured by Response Evaluation Criteria in Solid Tumors, Version 1.1 (RECIST 1.1) – as determined by an independent review through study completion. Confirmed CR or PR will be defined as a repeat assessment performed no less than 28 days after the criteria for response is first met.

Dr. Christopher Vaughn, M.D., Oncologist at Hematology Oncology Associates of Fredericksburg (HOAF) in Virginia, stated, "It is wonderful and exciting to bring potentially landscape-changing new therapeutic advances to our patient community. I fully appreciate the collaboration and support between the Endeavor team and HOAF’s research department. The Endeavor trial embodies the model for individualized therapy and for potentially better response rates with limited side effects for our patients."

"ENV-101 is for patients with tumor mutations in the Hedgehog pathway, which affect approximately 2% of all cancers, amounting to roughly 30,000-40,000 patients annually in the U.S. alone," said Dr. Srikanth Pendyala, Chief Medical Officer, Endeavor Biomedicines. "Mutations in this pathway can aberrantly activate downstream signaling resulting in proliferation, survival and metastasis of cancer cells. ENV-101 is intended to inhibit the activity of the Hedgehog pathway which may significantly benefit the lives of cancer patients with these mutations."

ENV-101: Targeting the Hedgehog Signaling Pathway in Oncology and Fibrosis

ENV-101 (taladegib), an orally available small molecule inhibitor of the Hedgehog signaling pathway, has already demonstrated preliminary clinical efficacy and safety in nearly 200 subjects enrolled across six completed studies. Initially targeted for a broad group of patients with basal cell carcinoma (BCC), Endeavor is now investigating precision therapy approaches for ENV-101 in multiple types of cancers driven by oncogenic driver mutations in PTCH1, as well as in idiopathic pulmonary fibrosis (IPF).

PTCH1 oncogenic driver mutations in the Hedgehog signaling pathway are found in approximately 2% of all cancers. Because of its prevalence across multiple types of cancer, Endeavor plans to enroll patients in a tumor agnostic study that includes any patient with oncogenic hedgehog mutations irrespective of tissue of origin. Endeavor is currently enrolling patients in an open label Phase 2 clinical trial in oncology (www.clinicaltrials.gov identifier NCT05199584).

For more information about our clinical trial please contact us at: [email protected] or call us at 858-727-3199.