On May 10, 2022 Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a clinical-stage precision oncology company designing and developing novel targeted therapies for cancer treatment, reported the U.S. Food and Drug Administration (FDA) granted an eighth regulatory designation, and third Breakthrough Therapy designation, to lead drug candidate repotrectinib (Press release, Turning Point Therapeutics, MAY 10, 2022, View Source [SID1234614122]).

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Breakthrough Therapy designation (BTD) was granted for the treatment of patients with ROS1-positive metastatic non-small cell lung cancer (NSCLC) who have been previously treated with one ROS1 tyrosine kinase inhibitor and who have not received prior platinum-based chemotherapy. The efficacy analyses supporting the BTD application included approximately 50 patients pooled from the Phase 1 and Phase 2 portions of the TRIDENT-1 study. Efficacy evaluations in Phase 2 patients were by physician assessment.

"We are excited to receive our third BTD and eighth overall FDA regulatory designation for repotrectinib in an indication where there are no approved targeted therapies," said Mohammad Hirmand, M.D., Chief Medical Officer. "We are encouraged by the continued momentum in TRIDENT-1 with enrollment targets achieved in cohorts EXP-1, EXP-4 and EXP-6. We look forward to continuing to progress repotrectinib toward registration with our first pre-NDA meeting with the FDA to discuss the topline data by blinded independent central review from the ROS1-positive advanced NSCLC cohorts of the TRIDENT-1 study expected later this quarter."

BTD is granted by the FDA to expedite the development and regulatory review of an investigational medicine that is intended to treat a serious or life-threatening condition. The criteria for BTD require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.

Repotrectinib was previously granted two BTDs in: ROS1-positive metastatic NSCLC patients who have not been treated with a ROS1 tyrosine kinase inhibitor, and patients with advanced solid tumors that have an NTRK gene fusion who have progressed following treatment with one or two prior TRK tyrosine kinase inhibitors, with or without prior chemotherapy, and have no satisfactory alternative treatments. Repotrectinib was also previously granted four Fast-Track designations in: ROS1-positive advanced NSCLC patients who are ROS1 TKI naïve; ROS1-positive advanced NSCLC patients who have been previously treated with one prior line of platinum-based chemotherapy and one prior ROS1 TKI; ROS1-positive advanced NSCLC patients pretreated with one prior ROS1 TKI without prior platinum-based chemotherapy; and NTRK-positive patients with advanced solid tumors who have progressed following treatment with at least one prior line of chemotherapy and one or two prior TRK TKIs and have no satisfactory alternative treatments. Repotrectinib was also granted an Orphan Drug designation in 2017.

Enrollment across all six cohorts of the study remains open and continues to progress steadily.