ArQule Announces Issuance of U.S. Patent Covering Composition of Matter of Proprietary Reversible BTK Inhibitor, ARQ 531

BURLINGTON, Mass.--()--ArQule, Inc. (Nasdaq: ARQL) today announced that the U.S. Patent and Trademark Office has issued U.S. Patent Number 9,630,968. The patent claims composition of matter of ARQ 531. ArQule will be entitled to patent protection through December 2035 in the U.S. for the allowed claims. ARQ 531 is an investigational, orally bioavailable, potent and reversible inhibitor of both wild type and C481S-mutant Bruton’s tyrosine kinase (BTK). The company plans to begin a phase 1 clinical trial by the third quarter of 2017 with ARQ 531 in patients with B-cell malignancies who are refractory to other therapeutic options.

“This patent secures long lasting and foundational intellectual protection for ARQ 531,” said Peter Lawrence, President, Chief Operating Officer, and General Counsel at ArQule. “We continue to work to extend and strengthen all intellectual property related to chemical equity that has been generated through our multiple years of investment in the BTK discovery program.”

B-cell malignancies, like chronic lymphocytic leukemia, Waldenstrom’s macroglobulinemia, diffuse large B-cell lymphoma and mantle cell lymphoma are driven by BTK. The only approved BTK inhibitor, ibrutinib, is irreversible and makes a covalent bond with the C481 residue of the targeted protein. Although ibrutinib has demonstrated excellent responses in patients with elevated B-cell receptor signaling, clinical resistance has been observed, and the BTK C481S mutation is emerging as a predominant mechanism of resistance. As a reversible inhibitor, ARQ 531 does not require interaction with the C481 residue, a binding site essential for irreversible ibrutinib binding to BTK, thus positioning ARQ 531 as a targeted therapy for patients harboring C481S-mutant BTK who have developed resistance to irreversible BTK inhibitors.

About BTK and ARQ 531

ARQ 531 is an investigational, orally bioavailable, potent and reversible Bruton’s tyrosine kinase (BTK) inhibitor. Biochemical and cellular studies have shown that ARQ 531 inhibits both the wild type and C481S-mutant forms of BTK. The C481S mutation is a known emerging resistance mechanism for first generation irreversible BTK inhibitors. In preclinical studies ARQ 531 has demonstrated high oral bioavailability as well as good ADME, pharmacokinetic and metabolic properties. The company plans to initiate a phase 1 trial by the third quarter of 2017. BTK is a therapeutic target that has been clinically proven to inhibit B-cell receptor signaling in blood cancers.

About ArQule

ArQule is a biopharmaceutical company engaged in the research and development of targeted therapeutics to treat cancers and rare diseases. ArQule’s mission is to discover, develop and commercialize novel small molecule drugs in areas of high unmet need that will dramatically extend and improve the lives of our patients. Our clinical-stage pipeline consists of four drug candidates, all of which are in targeted, biomarker-defined patient populations, making ArQule a leader among companies our size in precision medicine. ArQule’s proprietary pipeline includes: ARQ 087, a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor (FGFR) family, in phase 2 for iCCA and in phase 1b for multiple oncology indications; ARQ 092, a selective inhibitor of the AKT serine/threonine kinase, in phase 1 for multiple oncology indications as well as ultra-rare Proteus syndrome, in partnership with the National Institutes of Health (NIH); ARQ 751, a next generation AKT inhibitor, in phase 1 for patients with AKT1 and PI3K mutations; and ARQ 761, a β-lapachone analog being evaluated as a promoter of NQO1-mediated programmed cancer cell necrosis, in phase 1/2 in multiple oncology indications in partnership with the University of Texas Southwestern Medical Center. In addition, we have advanced ARQ 531, an investigational, orally bioavailable, potent and reversible inhibitor of both wild type and C481S-mutant BTK, through toxicology testing and plan to initiate a phase 1 trial by the third quarter of 2017. ArQule’s current discovery efforts are focused on the identification and development of novel kinase inhibitors, leveraging the Company’s proprietary library of compounds. You can follow us on Twitter and LinkedIn.

This press release contains forward-looking statements regarding the Company’s patent protection for its BTK inhibitor, ARQ 531, including its current composition of matter patent and anticipated future patent filings, and also planned clinical trials regarding ARQ 531. These statements are based on the Company’s current beliefs and expectations, and are subject to risks and uncertainties that could cause actual results to different materially. For example, although the U.S. Patent and Trademark Office has granted composition of matter protection to ARQ 531 in the United States, similar foreign patent authorities have yet to act on the composition of matter patent filings for ARQ 531 and may not grant such protection. In addition, the U.S. Patent and Trademark Office and similar foreign patent authorities may not grant additional patents for ARQ 531. Moreover, third parties may successfully challenge the validity of our patents. Regarding the Company’s planned clinical trials with ARQ 531, problems may arise that could lead to the delays or discontinuation of clinical development, including safety and regulatory issues. For more detailed information on the risks and uncertainties associated with the Company’s patent, drug development and other activities, see the Company’s periodic reports filed with the Securities and Exchange Commission. The Company does not undertake any obligation to publicly update any forward-looking statements.

Contacts

ArQule, Inc.
Dawn Schottlandt, 781-994-0300
Sr. Director, Investor Relations/
Corp. Communications
www.arqule.com

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Contacts

ArQule, Inc.
Dawn Schottlandt, 781-994-0300
Sr. Director, Investor Relations/
Corp. Communications
www.arqule.com