BioTheryX announced the U.S. Food and Drug Administration (FDA) has cleared BioTheryX's investigational new drug application (IND) for BTX-A51, an oral multi-kinase inhibitor, for the treatment of patients with relapsed/refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). BioTheryX anticipates dosing of first patients to begin during the third quarter of this year.

BTX-A51 is a small molecule, multi-kinase inhibitor that 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 This molecule has demonstrated remarkable preclinical animal efficacy implying the eradication of AML stem cells, and the potential for use in multiple malignancies.

"The acceptance of our IND is a major milestone in transitioning BioTheryX from a pre-clinical to a clinical-stage biotechnology company," said David Stirling, Ph.D., CEO of BioTheryX. "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. In short, BTX-A51 seeks to address a truly unmet medical need in very novel ways."

In addition to the multi-kinase program described above, BioTheryX's other technology platform is in the field of protein homeostasis, also known as targeted protein degradation. This technology uses 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 pre-clinical assets include a large and growing library of novel small molecule, cereblon-binding targeted protein degraders, which BioTheryX has termed Protein Homeostatic Modulators (PHM®). 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.

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