Terns Pharmaceuticals (TERN) investor relations material

Study background and landscape

• TERN-701 is a next-generation oral allosteric BCR-ABL inhibitor for chronic myeloid leukemia (CML), designed to surpass current therapies in efficacy, safety, and convenience.

• CML remains a chronic disease with significant unmet needs, as 40% of patients switch therapies within five years due to efficacy or side effects.

• Allosteric TKIs like asciminib have improved efficacy and safety but still present challenges such as pancreatic toxicity, hypertension, and food-related dosing restrictions.

• TERN-701 was developed to address these limitations and potentially set a new standard in CML treatment.

Study design and patient population

• The CARDINAL phase I study enrolled over 85 heavily pretreated CML patients, predominantly in third-line or later settings, with high disease burden and prior TKI failures, including asciminib.

• Patients had a median of 3 prior TKIs, with 60% having received three or more prior therapies and 64% discontinuing their last TKI due to lack of efficacy.

• 38% had prior asciminib exposure, and 22% had prior ponatinib; most discontinued due to lack of efficacy.

• 87% of patients remained on treatment at a median duration of 6 months, with discontinuations mainly due to disease progression.

• CARDINAL is a global, multi-center Phase 1 trial with dose escalation (160–500 mg) and randomized dose expansion (320 mg and 500 mg QD).

Efficacy results

• At doses of 320 mg and above, TERN-701 achieved 75% MMR and 36% DMR by 24 weeks, with 62% achieving MR2.

• Across all doses, 64% achieved MMR by 24 weeks, with 29% achieving DMR and 59% MR2.

• High response rates were observed in difficult-to-treat subgroups, including those with prior asciminib, ponatinib, or ELVN-001 exposure.

• No patients lost MMR at data cutoff; responses were consistent across all baseline transcript levels and in patients with high disease burden.

• TERN-701 demonstrated activity in patients with resistance mutations, such as F317L, and in those who failed prior asciminib or ponatinib.