BioTheryX Announces the Initiation of Patient Dosing in a First-in-Man Phase 1 Clinical Trial of BTX-A51, a Multi-Kinase Inhibitor

Study is evaluating the safety/tolerability of BTX-A51 in patients with relapsed/refractory acute myeloid leukemia (AML), as well as high risk myelodysplastic syndrome patients.

News provided by

Biotheryx, Inc.

Jan 16, 2020, 09:00 ET

CHAPPAQUA, N.Y., Jan. 16, 2020 /PRNewswire/ -- BioTheryX, Inc., a clinical stage biotechnology company creating new classes of compounds based on multi-kinase inhibition and targeted protein degradation, today announced the initiation of patient dosing in its first clinical program. 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.

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.

About BioTheryX

BioTheryX was founded by the drug development team behind the remarkable IMiDs® franchise of compounds (the most successful class of anti-cancer drugs in the world), and is applying its extensive and proven commercial experience to deliver efficacious therapies to patients with unmet medical needs. Our lead drug candidates are orally available small molecules for the treatment of cancers and inflammatory diseases.

Cautionary Note Regarding Forward-Looking Statements

This press release contains certain "forward-looking statements" within the meaning of the "safe harbor" provisions of the United States Private Securities Litigation Reform Act of 1995, including statements regarding the development and regulatory status of our product candidates, including the timing of our clinical trial for BTX-A51, and other statements which involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding our strategy, future operations, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "estimate," expect," "intend," "may," "might," "plan," "propose," "predict," "project," "forecast," "target," "potential," "will," "would," "could," "should," "continue," "and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These forward-looking statements are not guarantees of future performance, conditions or results, and involve a number of known and unknown risks, uncertainties, assumptions and other important factors, many of which are outside BioTheryX's control, that could cause actual results or outcomes to differ materially from those discussed in the forward-looking statements. We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements we make as a result of various risks and uncertainties, including but not limited to: whether we will be able to complete our Phase 1 clinical trial for BTX-A51, or file INDs/complete clinical trials for other product candidates on our expected timelines, or at all, whether our cash resources will be sufficient to fund our foreseeable and unforeseeable operating expenses and capital expenditure requirements or market any of our products after successful clinical trials and other important factors. The forward-looking statements contained in this press release reflect our current views with respect to future events, and we assume no obligation to update any forward-looking statements except as required by applicable law. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this release. BioTheryX does not undertake any obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.