Enliven Therapeutics (NASDAQ:ELVN) executives said the company is focused on advancing ELVN-001 into late-stage development for chronic myeloid leukemia, with regulatory interactions and trial initiation expected to be key events over the next year.
Speaking at TD Cowen’s Seventh Annual Oncology Innovation Summit, Chief Executive Officer Rick Fair said the company is preparing for its first Phase 3 trial in second-line-plus CML while building an organization capable of executing multiple pivotal trials and, potentially, supporting a commercial launch “just a few years away.”
ELVN-001 Data to Be Updated at EHA
Fair reviewed data from the company’s Phase 1b trial of ELVN-001 in CML, noting that the abstract for the upcoming European Hematology Association meeting was based on a December 2025 data cut previously disclosed in January.
He said the safety and tolerability profile showed a discontinuation rate due to adverse events of about 6% and dose reductions due to adverse events of less than 10%. Fair said Enliven views the drug’s tolerability as tied to its specificity for ABL1.
On efficacy, Fair cited achieved major molecular response, or MMR, rates of 38% in the more mature 80 mg group and 53% in the less mature 60 mg and 120 mg randomized cohort. He said all patients who entered the trial in MMR maintained that status. Cumulative MMR rates were about 47% and 69%, respectively, in those dose groups.
Fair cautioned that the difference between the groups was related to data maturity rather than dose response. He said the more mature “high 30” percentage is likely closer to the expected achieved MMR rate in the heavily pretreated, resistant patient population enrolled in the study.
The company also reported data in patients previously treated with asciminib. Fair said achieved MMR was 38% and cumulative MMR was 47% in that group, consistent with the overall study population. He noted that 92% of those patients had received at least three prior treatments, while 37% had received five or more prior treatments.
At EHA, Enliven plans to present updated safety and efficacy data from a March data cut, including about 20 additional enrolled patients and a similar number of additional efficacy-evaluable patients.
Phase 3 Planning Centers on Second-Line-Plus CML
Fair said Enliven plans to propose a second-line-plus randomized pivotal study against physicians’ choice of tyrosine kinase inhibitors. He said the company expects feedback from health authorities in the third quarter, including alignment on trial design and dose.
Enliven expects to take an 80 mg dose forward. Fair said 60 mg, 80 mg and 120 mg appeared similar from a safety and efficacy perspective in the Phase 1 study, but modeling suggests that 80 mg should provide full target coverage even for patients taking proton pump inhibitors.
The regulatory endpoint is expected to be MMR at 24 weeks, with a superiority comparison, though Fair said the exact timing remains subject to discussions with regulators because precedent differs across later-line and frontline CML settings.
Fair said the company also plans to seek guidance this year on requirements for a frontline CML trial, particularly the safety database needed to begin testing ELVN-001 in newly diagnosed patients. Enliven also plans to initiate a Phase 2 investigator-sponsored trial in frontline CML this year.
Company Positions ELVN-001 Against Allosteric Therapies
Fair contrasted ELVN-001 with TERN-701 and asciminib, saying ELVN-001 is a selective ATP-competitive inhibitor, while TERN-701 is an allosteric agent that he said appears chemically similar to asciminib, marketed as SCEMBLIX.
Fair said Enliven believes ELVN-001 could be used in sequence after allosteric therapies because it has a different mechanism and may address different resistance patterns. He said the company expects SCEMBLIX to be widely used in first- and second-line CML by the time Enliven enters the market, making the post-asciminib setting an important commercial opportunity.
He also said Enliven views ELVN-001 as potentially competitive in frontline CML based on its tolerability, efficacy, lack of food effect and lower potential for drug-drug interactions. However, he said competition in the frontline setting will depend on clinical profiles generated in future studies.
SCEMBLIX Launch Highlights CML Market Opportunity
Ben Hohl, Enliven’s chief financial officer and head of corporate development, said Novartis’ SCEMBLIX launch shows demand for new CML therapies. He cited Novartis’ reported first-quarter SCEMBLIX sales of $433 million and peak sales guidance above $4 billion.
“It shows that doctors and patients, they really do want new drugs, and they’re willing to prescribe new drugs,” Hohl said. “Just the CML market, it really is huge and can support multiple blockbuster drugs.”
Fair said Enliven does not expect Ascentage’s olverembatinib, which he described as a “me-too, me-better ponatinib,” to compete directly with ELVN-001. He said Enliven expects to compete for the first three lines of therapy with allosteric agents, while olverembatinib may replace ponatinib in fourth-line-plus CML if its profile holds up.
Cash Runway Extends Into 2029
On the company’s pipeline, Fair said Enliven remains “very, very focused” on ELVN-001 in CML. He also said the company has a program in Graves’ disease, but has not yet disclosed details because it has been challenging to identify a development candidate that meets the company’s target product profile.
Hohl said Enliven has roughly $450 million in cash and expects that balance to support operations into the first half of 2029. He said that runway should carry the company through topline pivotal data from the planned second-line-plus CML trial.
About Enliven Therapeutics (NASDAQ:ELVN)
Enliven Therapeutics is a clinical-stage biotechnology company focused on developing small-molecule therapies that harness induced proximity mechanisms to selectively target and degrade disease-causing proteins in cancer. Leveraging its proprietary Induced Proximity platform, the company designs molecular glues and related modalities to recruit endogenous cellular machinery for targeted protein degradation, with the goal of treating malignancies driven by so-called “undruggable” oncogenic factors.
The company’s pipeline comprises several early-stage programs directed at key oncogenic drivers across hematologic and solid tumor indications.
