The U.S. Food and Drug Administration granted Breakthrough Therapy Designation to Terns Pharmaceuticals’ oral allosteric BCR::ABL1 inhibitor, TERN‑701, for adult patients with Philadelphia chromosome–positive chronic myeloid leukemia in the chronic phase who have failed two or more tyrosine‑kinase inhibitors. The designation, effective April 27 2026, is intended to accelerate the development and review of therapies that offer substantial improvement over existing treatments.
The designation is based on data from the ongoing Phase 1/2 CARDINAL trial, which showed encouraging rates of major and deep molecular response at week 24, including in patients with high baseline disease burden and prior exposure to allosteric TKIs. Safety data from the trial indicate that most adverse events were low‑grade, with a low incidence of severe events, reinforcing the FDA’s confidence in the drug’s safety profile.
For Terns, the Breakthrough status is expected to streamline regulatory interactions, shorten the time to pivotal trials and market entry, and strengthen the company’s competitive positioning against established CML therapies such as Novartis’ asciminib. The designation also signals strong confidence in TERN‑701’s potential to become a best‑in‑disease therapy for CML patients, especially those without the T315I mutation.
Terns reported a net loss of $23.9 million for Q1 2025, up from $22.4 million in Q1 2024, and an earnings per share of –$0.24 for Q4 2025, beating analyst estimates. The company’s cash, cash equivalents, and marketable securities totaled $334.3 million as of March 31 2025, a balance that is expected to support operations through 2028. The next earnings report is scheduled for May 14 2026.
Management highlighted the significance of the Breakthrough designation and the pending Merck acquisition. “There remains an urgent need for CML treatments that offer improved efficacy, safety, and tolerability over current therapies. This designation from the FDA supports the significant potential of TERN‑701 to be a best‑in‑disease therapy for CML patients and offer substantial improvement based on the faster, deeper responses compared to prior TKIs and encouraging safety and tolerability profile observed to date,” said Scott Harris, Chief Development and Operations Officer. “This Breakthrough Therapy Designation, along with the recent agreement for Merck to acquire Terns, has the potential to accelerate efforts to advance TERN‑701 to a pivotal trial and to patients. This is an exciting time for everyone involved in the TERN‑701 program. We are grateful to the investigators, patients and community advocates whose dedication and support have made these advancements possible,” added Amy Burroughs, Chief Executive Officer. “TERN‑701 showed highly encouraging safety with no dose limiting toxicities in dose escalation up to the maximum dose of 500 mg, linear pharmacokinetics with once daily dosing, and compelling molecular responses in patients with high CML disease burden who had responded poorly to multiple prior therapies including asciminib. The favorable safety profile and dose‑related increase in molecular responses with TERN‑701 allowed us to select doses at the top end of the dose range to take forward to dose expansion,” said Emil Kuriakose, Chief Medical Officer.
The Breakthrough Therapy Designation represents a pivotal regulatory milestone for Terns, positioning TERN‑701 as a promising new option for patients who have exhausted existing therapies. Coupled with the strategic partnership with Merck, the designation could accelerate the drug’s development timeline and broaden its commercial potential, while the company’s financial position provides a runway to pursue these objectives.
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