On January 16, 2020 BioTheryX, Inc., a clinical stage biotechnology company creating new classes of compounds based on multi-kinase inhibition and targeted protein degradation, reported the initiation of patient dosing in its first clinical program (Press release, BioTheryX, JAN 16, 2020, View Source [SID1234553296]). The Phase 1 study of BTX-A51, a small molecule, oral multi-kinase inhibitor will evaluate the safety, pharmacokinetics and tolerability of BTX-A51 in patients with relapsed/refractory AML, as well as high risk myelodysplastic syndrome patients.
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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 remarkable preclinical animal efficacy implying the eradication of AML stem cells and the potential for use in multiple malignancies.
"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," said David Stirling, Ph.D., CEO of BioTheryX.
In addition to its multi-kinase inhibition program, BioTheryX’s other technology platform is in the field of targeted protein degradation. This technology utilizes the body’s own protein disposal system to selectively degrade and remove disease-causing proteins. It has potential applicability to a very broad range of disease targets, including a wide range of targets that have to date been considered "undruggable."
In this area, BioTheryX’s preclinical assets include a large and growing library of novel, small molecule, orally available, cereblon-binding targeted protein degraders which BioTheryX has termed Protein Homeostatic Modulators ("PHMs"). These IP-protected compounds are biologically active against a number of high value therapeutic targets in oncology, inflammation and other diseases. In addition to the therapeutic potential of these "molecular glue" molecules in their own right, these compounds also have a broad range of molecular orientations when bound to cereblon, providing a new level of structural control in the creation of bifunctional chimeric molecules that degrade high-value targets with great specificity. Recognizing this potential, BioTheryX has created a library of PHM-linked, biologically active chimeric molecules, including several that degrade the oncogenic targets of BTX-A51, thus dovetailing BioTheryX’s two major programs.