ArQule to Present Clinical and Preclinical Data for ARQ 751 at the 30th EORTC/AACR/NCI Symposium

Three poster presentations highlighting clinical data from ARQ 751-101 and preclinical data on ARQ 751 in combination with other agents

BURLINGTON, Mass.--(BUSINESS WIRE)--Nov. 8, 2018-- ArQule, Inc. (Nasdaq: ARQL) today announced that it will be presenting clinical and preclinical data on the company’s next generation AKT inhibitor, ARQ 751, in three poster presentations at the 30th EORTC/AACR/NCI Symposium to be held from November 13 to 16, 2018 in Dublin, Ireland.

About ARQ 751
ARQ 751 is an orally bioavailable, selective small molecule inhibitor of the AKT serine/threonine kinase. The AKT pathway when abnormally activated is implicated in multiple oncogenic processes such as cell proliferation and apoptosis. This pathway has emerged as a target of potential therapeutic relevance for compounds that inhibit its activity, which has been linked to a variety of cancers as well as to select non-oncology indications. ARQ 751 is currently in a Phase 1 study in adult patients with refractory and/or metastatic tumors that harbor genetic alterations along the AKT pathway.

About Miransertib
Miransertib (ARQ 092) is an orally available, selective, pan-AKT (protein kinase B) inhibitor that potently inhibits AKT1, 2 and 3 isoforms. Dysregulation of AKT has been implicated in a variety of rare overgrowth diseases and cancers; however, there are currently no approved inhibitors of AKT. AKT inhibitors, either as single agent or combination therapy, show significant promise in molecularly defined patient populations. Miransertib is currently in a Phase 1/2 company-sponsored study for PIK3CA-Related Overgrowth Spectrum (PROS), a Phase 1 study for ultra-rare Proteus syndrome conducted by the National Institutes of Health (NIH/NHGRI), and a Phase 1b study in combination with the hormonal therapy, anastrozole, in patients with advanced endometrial cancer with AKT and PI3K mutations. Miransertib has been granted Rare Pediatric Disease Designation and Fast Track Designation by the U.S. Food and Drug Administration (FDA), as well as Orphan Designation by the FDA and European Medicines Agency in the rare overgrowth disease, Proteus syndrome.

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 five 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 pipeline includes: ARQ 531, an orally bioavailable, potent and reversible inhibitor of both wild type and C481S-mutant BTK, in Phase 1 for patients with B-cell malignancies refractory to other therapeutic options; Miransertib (ARQ 092), a selective inhibitor of the AKT serine/threonine kinase, in a Phase 1/2 company-sponsored study for Overgrowth Diseases, in a Phase 1 study for ultra-rare Proteus syndrome conducted by the National Institutes of Health (NIH), and in Phase 1b in combination with the hormonal therapy, anastrozole, in patients with advanced endometrial cancer; ARQ 751, a next generation AKT inhibitor, in Phase 1 for patients with AKT1 and PI3K mutations; Derazantinib, a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor (FGFR) family, in a registrational trial for iCCA; 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. ArQule’s current discovery efforts are focused on the identification and development of novel kinase inhibitors, leveraging the Company’s proprietary library of compounds.

Forward Looking Statements
This press release contains forward-looking statements regarding the planned clinical development of miransertib and ARQ 751. 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 differ materially. Positive information about early clinical results does not ensure that later-stage clinical trials will be successful. For example, miransertib and ARQ 751 may not demonstrate sufficient therapeutic effect in man; in addition, neither drug candidate may exhibit an adequate safety profile in planned or later stage or larger scale clinical trials as a result of known or as yet unanticipated side effects. The results achieved in later stage trials may not be sufficient to meet applicable regulatory standards or to justify further development. Problems or delays may arise during clinical trials or in the course of developing, testing or manufacturing miransertib and ARQ 751 that could lead the Company to discontinue development. Even if later stage clinical trials are successful, unexpected concerns may arise from subsequent analysis of data or from additional data. Obstacles may arise or issues may be identified in connection with review of clinical data with regulatory authorities. Regulatory authorities may disagree with the Company's view of the data or require additional data or information or additional studies. In addition, we are utilizing diagnostic tests to identify patients in the Phase 1/2 trial with miransertib in PROS diseases and in the Phase 1 trial with ARQ 751 in patients with AKT and PI3K mutations.We expect to utilize diagnostic tests in other clinical trials with miransertib and ARQ 751. We or our collaborators may need to develop and register these or other diagnostic tests as companion diagnostics with the FDA. We or our collaborators may encounter difficulties in developing and obtaining regulatory approval for companion diagnostics, including issues relating to access to certain technologies, selectivity/specificity, analytical validation, reproducibility, or clinical validation. Any delay or failure by our collaborators or us to develop or obtain regulatory approval of companion diagnostics could delay or prevent approval of our product candidates. Drug development involves a high degree of risk. Only a small number of research and development programs result in the commercialization of a product. For more detailed information on the risks and uncertainties associated with the Company's 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.

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Source: ArQule, Inc.

Corporate Contact:
ArQule, Inc.
Marc Schegerin, M.D.
Senior Vice President, Head of Strategy, Finance and Communication
ir@arqule.com
www.ArQule.com
or
Media Contact:
LifeSci Public Relations
Allison Blum, Ph.D., 646-627-8383
Allison@lifescipublicrelations.com