BTX-A51 phase 1 trial : US biotech company BioTheryX has initiated patient dosing in its first clinical program of its small molecule BTX-A51 in patients having relapsed/refractory acute myeloid leukemia (AML), and also in high-risk myelodysplastic syndrome patients.

The phase 1 clinical trial of BTX-A51 will assess the safety, pharmacokinetics, and tolerability of the oral multi-kinase inhibitor in the two conditions.

According to BioTheryX, BTX-A51 has the potential to block a specific leukemic stem cell target (CK1-alpha) along with super-enhancer targets (CDK7/CDK9), thereby preventing transcription of important oncogenic genes.

The small molecule developed by BioTheryX is said to have shown significant preclinical animal efficacy implying its use in the removal of AML stem cells and the potential for use in various malignancies.

Commenting on BTX-A51 phase 1 trial, David Stirling – CEO of BioTheryX said: “As I stated when the Investigational New Drug application for BTX-A51 was accepted by the FDA, the novel mechanism of BTX-A51 may become one of the most important new treatments for AML in the last 40 years, and has the potential to significantly improve the lives of AML patients and their families.”

With operations in New York and San Diego, BioTheryX is focused on developing new classes of compounds, which are based on multi-kinase inhibition and targeted protein degradation.

  Acute myeloid leukemia, BioTheryX, BTX-A51, David Stirling, Myelodysplastic syndrome, New York, San Diego, USA