ArQule Presents Results From Phase 1b Expansion Study of ARQ 092 at the 2015 European Cancer Conference

Preliminary Results of the Phase 1b Expansion Cohort Show Four Partial Responses in Targeted Patient Populations

BURLINGTON, Mass., Sept. 28, 2015 (GLOBE NEWSWIRE) -- ArQule, Inc. (NASDAQ:ARQL) today announced preliminary results for the ongoing phase 1b expansion study in oncology for ARQ 092, an orally available selective pan-AKT inhibitor. The results were presented at the 2015 ESMO European Cancer Congress on September 27th, 2015.

Thus far 25 patients have been treated in the study, and four partial responses (PRs) have been observed. These include two patients with the AKT1 E17K mutation and one patient with the PIK3CA H1047R mutation. Mutation status for a fourth patient could not be determined. Of the four PRs, two were observed in follicular lymphoma, one in breast cancer and one in endometrial cancer. The poster presentation can be accessed in the “Investor and Media” section of our website,, under “Recent Data Presentations.”

The phase 1b study follows completion of the phase 1a dose escalation portion of the trial that enrolled 82 patients and tested various dosages and dose regimens. In the phase 1a trial after one PR was observed in a patient with chronic lymphocytic lymphoma, the study was expanded to phase 1b with a focus on lymphoma, endometrial cancer and tumors harboring AKT (AKT1 E17K) or PI3K (PIK3CA H1047R) mutations.

“The preliminary results of the phase 1b expansion cohort are very encouraging,” said Dr. Brian Schwartz, Head of Research and Development and Chief Medical Officer at ArQule. “The observation of four PRs in the 25 patients we have enrolled thus far validate our strategy to focus the trial on specific cancers with the AKT and PI3K mutations. We will continue to explore ARQ 092 in both cancer and rare diseases harboring these mutations.”

ArQule continues to enroll patients in the phase 1b study while assessing next steps for ARQ 092 in oncology. In parallel, ArQule collaborators at the National Institute of Health (NIH) received approval of the Investigational New Drug (IND) application to begin a phase 1 trial with ARQ 092 in Proteus syndrome, a rare disease that is caused by a mutation of the AKT1 gene.

About ARQ 092 and the AKT Pathway

ARQ 092 is an orally available, selective small molecule inhibitor of the AKT 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 092, the lead compound in ArQule’s AKT program, has completed Phase 1a clinical testing and has advanced into Phase 1b expansion testing in cohorts of patients with endometrial cancer, lymphoma and tumors harboring either AKT or PI3K mutations. A number of next-generation compounds in the Company’s AKT program are in early to late stages of pre-clinical development.

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, in Phase 2 and Phase 3 clinical development, is tivantinib (ARQ 197), an oral, selective inhibitor of the c-MET receptor tyrosine kinase. The Company’s pipeline includes: ARQ 092, designed to inhibit the AKT serine/threonine kinase, and ARQ 087, a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor (FGFR) family. ArQule’s current discovery efforts are focused on the identification of novel kinase inhibitors, leveraging the Company’s proprietary library of compounds.

This press release contains forward-looking statements regarding the Company’s clinical trials with ARQ 092. 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 and early stage clinical trial results, including in Proteus syndrome, does not ensure that later stage or larger scale clinical trials will be successful. For example, ARQ 092 may not demonstrate promising therapeutic effect; in addition, it may not demonstrate appropriate safety profiles in 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 or its partners, including the National Institutes of Health, 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.

Contacts: Dawn Schottlandt Sr. Director, Investor Relations/ Corp. Communications (781) 994-0300

Source:ArQule, Inc.