Pipeline

BCR-ABL Program: ELVN-001

ELVN-001 is a potent, highly selective, small molecule kinase inhibitor designed to specifically target the breakpoint cluster region – Abelson (“BCR-ABL”) gene fusion, the oncogenic driver for patients with chronic myeloid leukemia (“CML”). It targets the ATP-binding site of the ABL1 kinase domain and binds to a unique P-loop “folded-in” active conformation of ABL1, creating a narrow selectivity tunnel. 

Although the approval of BCR-ABL tyrosine kinase inhibitors (“TKIs”) changed prognosis of CML from an often fatal disease to a manageable chronic condition, patients still face several barriers that limit their ability to achieve durable responses, maintain long-term treatment success, and sustain a good quality of life. Patients can develop primary or secondary resistance to currently available TKIs, preventing them from achieving or maintaining the level of response required for long-term survival. In addition, the currently available ATP-competitive TKIs (e.g. nilotinib, dasatinib) have off-target activity that may lead to adverse events, potentially requiring dose reductions or treatment discontinuation. All approved TKIs are associated with drug-drug interactions that can affect the safety and efficacy of some medications, and most BCR-ABL TKIs must be taken either with food or on an empty stomach, making adherence more difficult. These factors underscore the significant global opportunity for therapies that can address these unmet needs. 

As a highly selective ATP-competitive inhibitor, ELVN-001 is differentiated from other ATP-competitive inhibitors in that it avoids off-target activity that may cause adverse events. With this potential additional safety margin, ELVN-001 may be optimally dosed to drive better efficacy. Additionally, ELVN-001 potentially represents a complementary option to allosteric BCR-ABL inhibitors (e.g., asciminib) due to its distinct mechanism of action and high selectivity which may play an increasingly important role in the standard of care for CML. Specifically, ELVN-001 was designed to have activity against emerging mutations known to confer resistance to allosteric TKIs, including asciminib. ELVN-001 was also designed to be a more attractive option for patients with comorbidities, on concomitant medications or desiring more freedom from stringent administration requirements. 

ELVN-001 is currently being evaluated in the Phase 1 ENABLE clinical trial in heavily pretreated patients with CML, and we plan to initiate ENABLE-2, a 2L+ Phase 3 pivotal trial in the second half of 2026. The most recent data is available on the Program Presentation & Publications section of the website. 

HER2 Program: ELVN-002

ELVN-002 is being evaluated in a Phase 1 trial as a monotherapy agent in people with HER2+ and HER2 mutant tumors. As part of that trial, we enrolled patients with HER2+ metastatic breast cancer (MBC) in an exploratory cohort in combination with ado-trastuzumab emtansine and patients with HER2+ NSCLC in an exploratory cohort in combination with trastuzumab deruxtecan. To learn more, please visit www.clinicaltrials.gov (NCT05650879).

ELVN-002 is also being evaluated in combination with trastuzumab with or without chemotherapeutic agents in people with HER2+ solid tumors. To learn more about the Phase 1 study, please visit www.clinicaltrials.gov (NCT06328738).

In May 2025, we made the decision to explore strategic alternatives for ELVN-002 so that we could prioritize ELVN-001. 

Additional Programs

Our drug development strategy is target and indication agnostic, rooted in validated biology and differentiated chemistry. Our pipeline reflects this approach, featuring medicines enabled by our expertise in complex medicinal chemistry to deliver new and improved treatments for patients. 

Program Presentations & Publications

Other Scientific Resources