BURLINGAME, Calif., June 11, 2026
Enliven Therapeutics announced updated positive Phase 1 clinical data for ELVN-001, its investigational BCR::ABL1-targeted therapy for chronic myeloid leukemia (CML), alongside regulatory alignment with the U.S. Food and Drug Administration (FDA) on key design elements for the upcoming Phase 3 ENABLE-2 trial. The data, presented at the European Hematology Association (EHA) 2026 Congress, demonstrated encouraging molecular response rates and a favorable safety profile in heavily pretreated CML patients, supporting the advancement of ELVN-001 into pivotal-stage development later this year.
Strong Molecular Responses Observed Across Previously Treated CML Patients
The ongoing ENABLE Phase 1 trial enrolled 161 patients with relapsed, refractory, or intolerant CML who had previously received tyrosine kinase inhibitor (TKI) therapies. Among patients treated in the 80 mg once-daily Phase 1b cohort, ELVN-001 achieved an overall major molecular response (MMR) rate of 61%, with 48% of evaluable patients reaching MMR within 24 weeks. Across all Phase 1b participants who had received only one or two prior TKIs, response rates were even stronger, with 67% achieving overall MMR and 55% reaching MMR by week 24. Investigators also reported encouraging deep molecular response (DMR) rates, suggesting the potential for durable disease control in patients who have exhausted multiple treatment options. Importantly, efficacy remained consistent regardless of prior exposure to asciminib, a recently approved allosteric BCR::ABL1 inhibitor.
Favorable Safety Profile Supports Long-Term Treatment Potential
ELVN-001 continued to demonstrate a favorable safety and tolerability profile, a critical consideration for patients requiring long-term therapy. Most treatment-emergent adverse events were Grade 1 or Grade 2, while only 6% of patients discontinued treatment due to adverse events. At the recommended 80 mg once-daily dose, Grade 3 or higher adverse events were reported in 24% of patients, with thrombocytopenia being the only severe event occurring in more than 5% of participants. The safety findings reinforce ELVN-001’s highly selective design and support its potential as a differentiated next-generation ATP-competitive kinase inhibitor for CML.
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FDA Alignment Clears Path for ENABLE-2 Phase 3 Study
Enliven also announced a significant regulatory milestone following its End-of-Phase 1 meeting with the FDA. The agency agreed with the selection of 80 mg once daily as the recommended Phase 3 dose and supported the inclusion of patients who have received one or more prior TKIs in the planned ENABLE-2 pivotal trial. The Phase 3 study is expected to compare ELVN-001 against physician’s choice of approved ATP-competitive TKI therapies in previously treated CML patients. Enrollment is anticipated to begin during the second half of 2026, positioning ELVN-001 as a potential new treatment option for patients requiring effective and well-tolerated long-term disease management.
ELVN-001 Designed to Address Unmet Needs in Chronic Myeloid Leukemia
ELVN-001 is a highly selective small-molecule kinase inhibitor specifically designed to target the BCR::ABL1 gene fusion, the primary oncogenic driver of CML. The therapy was engineered to maintain activity against challenging resistance mutations, including T315I, while providing a complementary mechanism to allosteric inhibitors. With strong efficacy signals, manageable safety findings, and regulatory support for pivotal development, Enliven believes ELVN-001 has the potential to become a best-in-class therapy for patients with previously treated chronic myeloid leukemia.
Source: Enliven Therapeutics press release
