Enliven Therapeutics said its lead drug candidate elvn-001 produced higher response rates in its phase 1 ENABLE study, while the company also secured alignment with the FDA on the dose and patient population for its planned phase 3 trial.
As of the March 10 cutoff, 161 patients had been enrolled across dose levels from 10 mg to 240 mg daily, up from earlier-stage enrollment. Median treatment duration reached 35 weeks, and 76% of patients remained on study.
The updated data showed a 54% overall major molecular response rate in the phase 1b group, based on 69 evaluable patients. In the 80 mg once-daily cohort, the overall major molecular response rate was 61% among 28 evaluable patients. The rate of achieved major molecular response was 40% in the overall phase 1b group and 48% in the 80 mg cohort. Maintained major molecular response was 100% in both groups.
Deep molecular response was reported in 22% of patients in the overall phase 1b group and 30% in the 80 mg once-daily cohort by 24 weeks.
Response rates were strongest in less heavily pretreated patients. Among patients with 1 to 2 prior unique TKIs, the achieved response rate was 55% in phase 1b and 60% in the post-asciminib subgroup. That compared with 32% for patients with 3 to 4 prior TKIs and 29% for those with 5 or more. In the 80 mg cohort, the overall response rate by 24 weeks was 61%, versus 55% in the broader phase 1b population.
Among patients previously treated with asciminib, response rates were similar to the broader group. Enliven said 62% of enrolled patients had prior asciminib exposure, and within that subgroup 93% had received three or more prior TKIs and 34% had received five or more.
Safety data showed 6% of patients discontinued because of adverse events. Grade 3 or higher treatment-emergent adverse events were reported in 53 of 158 patients, or 34%, overall. The most common were thrombocytopenia at 6%, neutropenia at 6% and lipase elevation at 6%.
At the 80 mg dose, which the company said is the biologically optimal dose, grade 3 or higher adverse events were reported in 15 of 62 patients, or 24%. Thrombocytopenia was the only grade 3 or higher event occurring in more than 5% of patients in that cohort.
Following its end-of-phase 1 meeting with the FDA, Enliven said 80 mg once daily was selected as the recommended phase 3 dose. The company also said the planned ENABLE-2 study is expected to enroll patients with CML previously treated with one or more TKIs and will compare elvn-001 with physician’s choice of an ATP-competitive TKI.
Enliven said ENABLE-2 is expected to begin in the second half of 2026. Today the company's shares have moved 11.4% to a price of $41.22. If you want to know more, read the company's complete 8-K report here.
