Terns Pharmaceuticals receives FDA Breakthrough Therapy Designation for TERN-701 in chronic myeloid leukaemia
Tuesday, April 28, 2026
Terns Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to TERN-701 for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia (Ph+ CML) in the chronic phase. The designation applies to patients without the T315I mutation who have previously been treated with two or more tyrosine kinase inhibitors (TKIs).
TERN-701 is an oral, allosteric BCR::ABL1 inhibitor being developed to address limitations in current CML treatments, particularly in patients who have not responded well or cannot tolerate existing therapies.
Breakthrough Therapy Designation is intended to speed up the development and review of medicines for serious conditions where there is a clear unmet medical need. To qualify, early clinical data must indicate that the treatment may offer a meaningful improvement over available options.
The designation is supported by findings from the ongoing Phase 1/2 CARDINAL trial, which is evaluating TERN-701 in patients with CML who have previously received at least one TKI and experienced treatment failure, inadequate response or intolerance. The study has shown promising results, including encouraging rates of major molecular response and deep molecular response at 24 weeks.
Positive responses were also observed in patients with a high disease burden and in those who had undergone multiple prior treatments, including earlier use of allosteric TKIs. The safety profile has been favourable so far, with most treatment-related side effects reported as low grade and a low number of severe adverse events or treatment discontinuations.
The designation is expected to support further clinical development of TERN-701 and may help accelerate its progress towards later-stage trials and potential availability for patients.
Source: globenewswire.com
