Terns Pharmaceuticals, Inc. receives U.S. Food and Drug Administration Breakthrough Therapy Designation for TERN-701 in previously treated Chronic Myeloid Leukemia based on Phase 1/2 data.
Written By: Vennela Reddy, BPharm
Reviewed By: Pharmacally Editorial Team
Terns Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to its investigational drug TERN-701 for adult patients with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in the chronic phase, without the T315I mutation, who have received two or more prior tyrosine kinase inhibitors (TKIs).
This designation, aimed at expediting development for drugs showing substantial improvement over available therapies, is based on preliminary clinical evidence.
Amy Burroughs, Chief Executive Officer of Terns, said: “The Breakthrough Therapy Designation, along with Merck’s planned acquisition of Terns, could accelerate TERN-701’s progression into pivotal trials and support its advancement to patients. We acknowledge the invaluable contributions of investigators, patients, and the broader community.”
TERN-701 is an oral, allosteric BCR::ABL1 inhibitor. The designation is supported by data from the ongoing Phase 1/2 CARDINAL trial (NCT06163430) in patients with treatment failure, suboptimal response, or intolerance to prior TKIs.
At 24 weeks, the trial showed encouraging rates of major and deep molecular responses, including in heavily pretreated patients and those with high baseline disease burden. Responses were also seen in patients previously treated with allosteric TKIs. The safety profile is favorable, with most adverse events low-grade, low incidence of severe events, and minimal treatment discontinuations.
Scott Harris, Chief Development and Operations Officer at Terns, added: “There remains a need for CML treatments with superior efficacy, safety, and tolerability. The FDA’s designation reflects TERN-701’s potential to deliver deeper, faster responses with an encouraging safety profile compared to existing TKIs.”
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About the Writer
Vennela Reddy, B Pharm is a pharmacy graduate with a keen interest in clinical research, pharmacovigilance, and medical writing, with a growing focus on publishable and scientific content development. In her words, she is passionate about translating complex medical data into clear, evidence-based communication.