Enliven Reports 61% MMR in 80 mg Cohort, FDA OKs Phase 3 Design
ELVN•Enliven Therapeutics’ ELVN-001 achieved 61% major molecular response (MMR) at 24 weeks in the 80 mg once-daily cohort and 67% MMR across all Phase 1b patients previously treated with up to two TKIs. The FDA endorsed 80 mg QD dosing and 2L+ patient inclusion for the ENABLE-2 Phase 3 trial launching in H2 2026.
1. Updated Phase 1b Efficacy
ELVN-001 delivered 67% MMR by 24 weeks across all Phase 1b patients and 61% MMR specifically in the 80 mg once-daily cohort, with deep molecular responses achieved in 30% of the 80 mg group. Response rates held steady regardless of prior asciminib exposure, with higher efficacy in less heavily pretreated patients.
2. Tolerability and Safety Profile
Of 161 enrolled patients, discontinuations due to adverse events were limited to 6%, and 76% remain on treatment with a median duration of 35 weeks. Grade ≥3 treatment-emergent adverse events occurred in 34% overall and 24% at the 80 mg dose, with thrombocytopenia as the most common serious event.
3. FDA Alignment and Phase 3 Plans
Following the End-of-Phase 1 meeting, the FDA endorsed 80 mg QD as the recommended Phase 3 dose and agreed to enroll patients with one or more prior TKIs in the ENABLE-2 pivotal trial, slated to start in the second half of 2026.
