Terns Pharmaceuticals (TERN) The Citizens Life Sciences Conference 2026 summary

Key data and clinical insights

• TERN-701 demonstrated 2x-3x higher efficacy and a differentiated safety profile compared to other agents in chronic myeloid leukemia (CML), with a 75% major molecular response (MMR) rate at go-forward doses and over 30% deep molecular response rates in late-line patients.

• Safety profile showed low rates of cytopenias and absence of adverse events like hypertension or pancreatic toxicity, differentiating it from asciminib.

• Efficacy was maintained in patients refractory to asciminib and ponatinib, including those with challenging mutations, with rapid and deep responses observed.

• No food effect was observed, addressing a significant patient compliance issue seen with other TKIs, and the drug showed limited drug-drug interactions.

• Confidence intervals for response rates did not overlap with asciminib, supporting statistical differentiation and reproducibility of results.

Market landscape and unmet needs

• Imatinib transformed CML into a chronic disease, but over half of patients become refractory, and second-generation TKIs, while more effective, have notable safety trade-offs.

• Asciminib has gained significant frontline market share due to improved safety and tolerability, but efficacy remains similar to 2G-TKIs.

• Tolerability and efficacy are key drivers for switching therapies, not just resistance mutations.

• There is a substantial unmet need for safer, more effective therapies, especially for patients who are refractory to multiple lines of treatment.

Development plans and regulatory strategy

• Dose selection between 320 mg and 500 mg is ongoing, with analyses considering efficacy, safety, and response kinetics; FDA interaction for dose and registrational study design is expected midyear.

• The first registrational study will target second line plus patients against a 2G-TKI control, with plans to initiate a frontline study soon after, pending safety database adequacy.

• Frontline study endpoints will be MMR at 48 weeks, while second line plus will use MMR at 24 weeks for accelerated approval.

• A mutation-specific cohort has been added to the CARDINAL study to address patients with T315I and other mutations, with potential for label expansion.

• Funding from recent capital raise is expected to support development through commercialization and launch.