The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to TERN-701, a new targeted drug for patients with Philadelphia chromosome–positive chronic myeloid leukemia who have already failed at least two prior treatments and do not carry the T315I mutation.
Results from the Phase 1/2 CARDINAL trial, presented at the ASH Annual Meeting, showed strong effectiveness. At the recommended dose, 80% of patients achieved a major molecular response, with most reaching this milestone within 24 weeks. All evaluable patients maintained their response for at least that duration, despite having received multiple prior therapies.
The drug also showed a favorable safety profile. Only 2% of patients stopped treatment side effects, which were mostly mild, including diarrhea, headache, and nausea. Severe blood-related side effects were uncommon.
These findings suggest TERN-701 could offer a more effective and tolerable option for patients with limited treatment choices, though further studies are needed.