Terns Pharmaceuticals (TERN) TD Cowen 46th Annual Health Care Conference summary

Industry landscape and unmet needs

• Chronic myeloid leukemia (CML) has become a chronic disease with multiple approved therapies, but significant unmet needs remain in both efficacy and safety, especially for younger patients who may require lifelong treatment.

• Allosteric inhibitors like asciminib are gaining traction due to improved safety profiles and comparable efficacy to second-generation TKIs, with asciminib now holding about 25% frontline market share.

• Only 15%-20% of patients achieve treatment-free remission, highlighting the need for therapies that drive deeper and faster molecular responses.

• Resistance mutations are not the primary driver of therapy failure in CML; target coverage and tolerability are more critical factors.

• Physicians increasingly prioritize safety and tolerability, as these factors directly impact efficacy and long-term outcomes.

Clinical development and data highlights

• Phase I CARDINAL data for TERN-701 showed promising efficacy: 75% achieved major molecular response (MMR), 36% deep molecular response (DMR), and 62% MR2 in expansion cohorts.

• As of December, 85 patients were enrolled, with ongoing expansion at 320 mg and 500 mg doses; dose selection will be based on exposure-safety and exposure-efficacy analyses, targeting at least 20 patients per dose for six months.

• Plans are in place to meet with the FDA mid-year to discuss pivotal trial designs for both second-line-plus and frontline settings, aiming for rapid progression to pivotal studies.

• A mutation cohort at 500 mg is enrolling to assess activity in resistant populations, with potential for label expansion based on results.

• Safety profile is favorable, with hematologic adverse events most common but lower than other therapies; only one patient discontinued due to adverse events.

Differentiation and competitive positioning

• TERN-701 demonstrates higher target coverage and potency on certain resistance mutations compared to asciminib, with preclinical and early clinical data supporting activity in highly refractory patients.

• No food effect observed, simplifying dosing compared to asciminib.

• Confidence intervals for efficacy at 320 mg and above do not overlap with asciminib, suggesting meaningful differentiation.

• TERN-701 is expected to compete primarily in first and second line, targeting both generics and asciminib.

• Combination with active site inhibitors is not a current priority due to strong monotherapy responses and concerns about added toxicity.