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Press Release

Advancements in ArQule’s Proprietary Pipeline to be Highlighted at the 2016 American Society of Hematology Annual Meeting

Posted on: 03 Nov 16

ArQule, Inc. (Nasdaq: ARQL) today announced that preclinical data will be presented on two molecules from its proprietary pipeline at the 2016 American Society of Hematology (ASH) Annual Meeting in San Diego, California, December 3 - 6, 2016.

A poster presentation summarizing preclinical studies of ARQ 531 in Chronic Lymphocytic Leukemia (CLL) will be presented by The Ohio State University investigators. ARQ 531 is an investigational, orally bioavailable, potent and reversible Bruton's tyrosine kinase (BTK) inhibitor.

An oral presentation of preclinical studies with ARQ 092 in Sickle Cell Disease will be presented by ArQule’s collaborator, the University of Illinois College of Medicine. ARQ 092 is an orally available, selective pan-AKT inhibitor.

The data reinforce the strength and depth of ArQule’s proprietary pipeline while warranting further exploration of both molecules in their respective indications. The company remains on track to submit an Investigational New Drug (IND) application for ARQ 531 in early 2017. ARQ 092 is currently being tested in a phase 1b trial for AKT driven oncology malignancies and in a phase 1 trial in the ultra-rare Proteus syndrome indication in collaboration with the National Institutes of Health (NIH).

Details, including times and locations, of the ASH Annual Meeting presentations can be found below. Abstracts are available online at

ARQ 092 in Sickle Cell Disease
Title: Specific inhibition of AKT with ARQ 092, an orally-available selective allosteric AKT inhibitor, attenuates acute vaso-occlusive events in sickle cell disease
Presenter: Dr. Jaehyung Cho, University of Illinois College of Medicine
Session Date: Saturday, December 3, 2016
Oral Session Name: Hemoglobinopathies, Excluding Thalassemia - Basic and
Translational Science: Mechanisms of Vaso-Occlusion
Session Time: 2:00 PM - 3:30 p.m. PT
Oral Presentation Time: 2:45 p.m. PT
Location: San Diego Convention Center, Room 7AB

ARQ 531 in Chronic Lymphocytic Leukemia
Title: The Bruton’s Tyrosine Kinase (BTK) Inhibitor ARQ 531 Effectively Inhibits Wild Type and C481S Mutant BTK and Is Superior to Ibrutinib in a Mouse Model of Chronic Lymphocytic Leukemia
Presenter: Sean Reiff, The Ohio State University
Session: Date: Sunday, December 4, 2016
Poster Session Name: CLL: Therapy, excluding Transplantation: Poster II
Poster Viewing Time: 9:00 a.m. - 8:00 p.m. PT
Poster Presentation Time: 6:00 p.m. - 8:00 p.m. PT
Location: San Diego Convention Center, Hall GH

About the AKT Pathway and ARQ 092

ARQ 092 is an orally bioavailable, selective small molecule inhibitor of the AKT kinases. 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 092, the lead compound in ArQule’s AKT program, has completed phase 1a clinical testing and has fully enrolled a phase 1b trial including cohorts of patients with endometrial cancer, lymphomas and tumors harboring either AKT or PI3K mutations. It is also in a phase 1 trial being conducted by the NIH for Proteus syndrome, a rare over-growth disease from the PROS family. Collaborators are exploring, in preclinical testing, other indications for ARQ 092 including Sickle Cell Disease. In mid-2016 the company initiated a phase 1 clinical trial with ARQ 751, a next generation AKT inhibitor, in cancers with AKT mutations.

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 531inhibits 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. ARQ 531 has high oral bioavailability as well as good ADME, pharmacokinetic and metabolic properties. The company plans to file an IND for ARQ 531 in early 2017. BTK is a therapeutic target that has been clinically proven to inhibit B-cell receptor signaling in hematological cancers.

About ArQule

ArQule is a biopharmaceutical company engaged in the research and development of targeted therapeutics to treat cancers and rare diseases. Our 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 lead product, in phase 3 clinical development, is tivantinib (ARQ 197), an oral, selective inhibitor of the c-MET receptor tyrosine kinase, for second-line treatment of hepatocellular carcinoma in partnership with Daiichi Sankyo in the West and Kyowa Hakko Kirin in Asia. 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. ArQule’s current discovery efforts are focused on the identification and development of novel kinase inhibitors, leveraging the Company’s proprietary library of compounds. For more information, please follow us on Twitter and LinkedIn.

This press release contains forward-looking statements regarding planned and on-going clinical trials with ARQ 531 and ARQ 092, respectively. 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 pre-clinical results or early-stage clinical trials does not ensure that later stage or larger scale clinical trials will be successful. For example, ARQ 092 and ARQ 531 may not demonstrate promising therapeutic effect; in addition, ARQ 531 may not exhibit an adequate safety profile in ongoing toxicology testing, and both it and ARQ 092 may not demonstrate appropriate safety in planned, current 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 these compounds 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. Drug development involves a high degree of risk. Only a small number of research and development programs result in the commercialization of a product. Positive pre-clinical data may not be supported in later stages of development. Furthermore, ArQule may not have the financial or human resources to successfully pursue drug discovery in the future. 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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Last updated on: 03/11/2016

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