Skip to main content

A phase 1 study of BTX-A51, a multi-kinase inhibitor, in patients with AML or high-risk MDS




TAP Partner

Project Term: November 16, 2010 - TBD

In November 2010, LLS made an equity investment in BioTheryX originally to support the development of a promising LLS-funded project with Toronto-based University Health Network and is currently supporting "A First-In-Human, Open-Label, Escalating Multiple-Dose Study to Evaluate the Safety, Toxicity, and Pharmacokinetics of BTX-A51 Capsules in Patients With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome." BTX-A51 appears to block a specific leukemic stem cell target (CK1-alpha) as well as super enhancer targets (CDK7/CDK9) preventing transcription of key oncogenic genes. BTX-A51 has demonstrated preclinical animal efficacy implying the eradication of AML stem cells and the potential for use in multiple malignancies. A clinical study of BTX-A51 in patients with relapsed or refractory AML or high-risk MDS is currently enrolling.

Lay Abstract

For additional information, please visit

Therapy Acceleration Program
To All Projects