TERN-701 Gains FDA Orphan Drug Designation in Chronic Myeloid Leukemia
The BCR-ABL tyrosine kinase inhibitor TERN-701 has been granted an orphan drug designation from the FDA for the treatment of chronic myeloid leukemia.
White blood cells in leukemia, AI Generative: © Катерина Євтехова - stock.adobe.com
- TERN-701 has gained an orphan drug designation from the FDA for the treatment of patients with chronic myeloid leukemia (CML).
- The oral, potent, allosteric BCR-ABL tyrosine kinase inhibitor (TKI) aims to target the BCR-ABL myristoyl pocket.
- Evaluation of TERN-701 is currently underway in the phase 1 CARDINAL trial (NCT05367700).
The FDA has granted TERN-701 an orphan drug designation for the treatment of CML.1
TERN-701, an allosteric BCR-ABL TKI, is currently undergoing evaluation in the phase 1 CARDINAL trial. The agent is designed to target the ABL myristoyl pocket and is being developed for the treatment of patients with CML.2
“CML is a serious leukemia that requires chronic, lifelong treatment,” said Emil Kuriakose, MD, chief medical officer of Terns, in a press release.1 “Allosteric TKIs are a novel class of inhibitors that are highly selective and have demonstrated significantly improved clinical efficacy, safety and tolerability compared to prior generation active-site TKIs. Orphan drug designation for TERN-701 underscores the FDA’s recognition of the unmet need for people living with CML and Terns’ commitment to developing new treatment options.”
Based on emerging clinical data, the starting dose of TERN-701 appears safe and clinically active.3
About the Phase 1 Cardinal Study of TERN-701
In October 2023, the phase 1 CARDINAL trial of TERN-701 was initiated in the United States following the clearance of an investigational new drug application by the FDA. The ongoing phase 1 trial in China evaluating TERN-701 supports the study design of the phase 1 CARDINAL trial.
In the global, multicenter, open-label trial, investigators will evaluate the safety, pharmacokinetics, and efficacy of TERN-701 in participants with previously treated CML. The CARDINAL study includes 2 parts. The first part will utilize a dose-escalation design where experts will evaluate TERN-701 in 24 to 36 adult patients with CML. These patients will be included in 1 to 5 dose cohorts. Then, part 2 of the study will be a dose-expansion portion where investigators will examine the recommended dose from the first part of the study.
Enrollment on the study is open to patients aged 18 or older with disease that is in chronic phase, has confirmed BCR-ABL or Philadelphia chromosome positivity, and those who have experienced treatment failure or poor response to a minimum of 1 second-generation TKI such as nilotinib (Tasigna), dasatinib (Sprycel), or bosutinib (Bosulif).
Patients who are intolerant to prior TKI treatment, including asciminib (Scemblix), were eligible to enroll. Patients must also have an ECOG performance status of 0 to 2 and a life expectancy of 12 weeks or more.
