Positive Phase 2 Clinical Data With Tivantinib in Hepatocellular Carcinoma to be Highlighted in Oral Presentation at 201

WOBURN, Massachusetts and TOKYO, May 17, 2012 /PRNewswire/ --

ArQule, Inc. and Daiichi Sankyo, Co., Ltd. (TSE 4568) today announced that an oral presentation at the Annual Meeting of the American Society of Clinical Oncology (ASCO) will feature Phase 2 trial data with tivantinib as a single agent investigational second-line treatment in hepatocellular carcinoma (HCC). ArQule announced that this randomized, double-blind study met its primary endpoint in January and will now present the full results from this trial, including positive data in the pre-defined c-MET high population. Additional clinical data with tivantinib will be featured in two poster discussions and two general poster sessions. Abstracts of these presentations with tivantinib have been published on http://www.asco.org.

"These findings represent the first randomized data reported with an investigational c-MET inhibitor administered as a single agent second-line treatment in HCC," said Paolo Pucci, chief executive officer of ArQule. "They clearly define c-MET high patients as a biological subgroup for potential targeted therapy with tivantinib. The robust statistical significance achieved in this trial reflects the anti-cancer activity of tivantinib alone and expands its therapeutic potential."

HCC Trial Summary: c-MET high patients

Data from the HCC trial demonstrated a statistically significant improvement in time-to-progression (HR=0.43, log rank p-value=0.03), accompanied by significant improvements in progression-free survival and disease control rate among second-line patients with c-MET high tumors who were treated with tivantinib. In addition, overall survival data were observed favoring tivantinib-treated patients in this population. Efficacy was similar in the two tivantinib dosing subgroups (360 milligrams twice daily and 240 milligrams twice daily), with less frequent neutropenia in the lower dose.

Previously announced top-line data from the HCC trial demonstrate that treatment with tivantinib produced a statistically significant 56 percent improvement in TTP in the intent-to-treat (ITT) population by central radiology review, the primary endpoint (HR = 0.64, log rank p-value = 0.04) in this trial. Adverse events were reported at similar rates in the treatment and placebo arms, except for a higher incidence of fatigue and hematologic events, including neutropenia and anemia, in tivantinib-treated patients. The incidence of hematologic events declined following dose reduction of tivantinib from 360 milligrams twice daily to 240 milligrams twice daily.

The schedule of this and other presentations of data with tivantinib is provided below. All times are Central Daylight Time.

        
        Oral Presentation
        Date and time: Saturday, June 2, 2012, 5:00 PM - 5:15 PM
        Abstract number: 4005
        Poster title: Tivantinib (ARQ 197) versus placebo in patients (Pts) with hepatocellular
         carcinoma (HCC) who failed one systemic therapy: Results of a randomized controlled
         phase II trial (RCT)
        Presenter: Lorenza Rimassa, MD
        Location: E Hall D1
        Poster Discussion Sessions
        Date and time: Saturday, June 2, 2012, 8:00 AM - 12:00 PM
        Abstract number: 4545
        Poster title: Safety and efficacy of MET inhibitor tivantinib (ARQ 197) combined with
         sorafenib in patients (pts) with renal cell carcinoma (RCC) from a phase 1 study
        Poster board # 24
        Presenter: Igor Puzanov, MD
        Location: E450a
        Date and time: Saturday, June 2, 2012, 1:15 PM - 5:15 PM
        Abstract number: 8519
        Poster title: Safety and efficacy of MET inhibitor tivantinib (ARQ 197) combined with
         sorafenib in patients (pts) with NRAS wild-type or mutant melanoma from a phase 1
         study
        Poster board # 8
        Presenter: Julie A. Means-Powell, MD
        Location: E450b
        General Poster Session
        Date and time: Monday, June 4, 2012, 8:00 AM - 12:00 PM
        Abstract number: 4117
        Poster title: Safety and efficacy of MET inhibitor tivantinib (ARQ 197) combined with
         sorafenib in patients (pts) with hepatocellular carcinoma (HCC) from a phase 1 study
        Poster board # 50D
        Presenter: Robert E. Martell, MD, PhD
        Location: S Hall A2
        Date and time: Monday, June 4, 2012, 8:00 AM - 12:00 PM
        Abstract number: 4082
        Poster title: A phase II study of tivantinib monotherapy in patients with previously
         treated advanced or recurrent gastric cancer
        Poster board # 46A
        Presenter: Kei Muro, MD
        Location: S Hall A2

Tivantinib is currently in Phase 3 development and has not yet been approved for any indication.

About ArQule

ArQule is a biotechnology company engaged in the research and development of next-generation, small-molecule cancer therapeutics. The Company's targeted, broad-spectrum products and research programs are focused on key biological processes that are central to human cancers. ArQule's lead product candidate, in Phase 2 and Phase 3 clinical development together with development and commercialization partner, Daiichi Sankyo, Co. Ltd, is tivantinib, an oral, selective inhibitor of the c-MET receptor tyrosine kinase. The Company's pipeline consists of ARQ 621, designed to inhibit the Eg5 kinesin motor protein, and ARQ 736, designed to inhibit the RAF kinases. ArQule's current discovery efforts, which are based on the ArQule Kinase Inhibitor Platform (AKIP(TM)), are focused on the identification of novel kinase inhibitors that are potent, selective and do not compete with ATP (adenosine triphosphate) for binding to the kinase.

http://www.ArQule.com

About Daiichi Sankyo

The Daiichi Sankyo Group is dedicated to the creation and supply of innovative pharmaceutical products to address the diversified, unmet medical needs of patients in both mature and emerging markets. While maintaining its portfolio of marketed pharmaceuticals for hypertension, hyperlipidemia, and bacterial infections, the Group is engaged in the development of treatments for thrombotic disorders and focused on the discovery of novel oncology and cardiovascular-metabolic therapies. Furthermore, the Daiichi Sankyo Group has created a "Hybrid Business Model," which will respond to market and customer diversity and optimize growth opportunities across the value chain. For more information, please visit http://www.daiichisankyo.com.

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