Events Details
Terns Pharmaceuticals Receives Breakthrough Therapy for TERN-701 in Chronic Myeloid Leukemia (CML)
Tuesday, April 28, 2026
Terns Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to TERN-701, an investigational oral allosteric BCR::ABL1 inhibitor.
The designation applies to the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia (Ph+ CML) in the chronic phase, without the T315I mutation, who have previously been treated with two or more tyrosine kinase inhibitors (TKIs).
The designation highlights the ongoing need for improved treatment options in CML, particularly for patients who do not respond adequately to existing therapies or experience intolerance. TERN-701 is being developed as a potential alternative with improved efficacy and tolerability compared to currently available treatments.
Breakthrough Therapy Designation is intended to speed up the development and review of medicines for serious conditions where early clinical evidence suggests a meaningful improvement over existing therapies.
The designation for TERN-701 is supported by data from the ongoing Phase 1/2 CARDINAL clinical trial. The study includes patients with CML who have previously received at least one TKI and experienced treatment failure, suboptimal response, or intolerance. Early results have shown promising clinical activity, including notable rates of major molecular response and deep molecular response at 24 weeks.
Responses have also been observed in patients with a high disease burden and those who have undergone multiple prior treatments, including allosteric TKIs. In addition, the treatment has demonstrated a manageable safety profile, with most adverse events reported as low grade and a low rate of severe side effects or treatment discontinuation.
The development of TERN-701 is expected to progress further, supported by recent strategic developments involving the company.
Source: ternspharma.com
