ArQule, Daiichi Sankyo announce decision to advance ARQ 197 into Phase 3 clinical trial for NSCLC

ArQule, Inc. (Nasdaq: ARQL) and Daiichi Sankyo Co., Ltd. (TSE 4568) today announced that they will move forward with a Phase 3 clinical trial of ARQ 197, a small molecule inhibitor of the c-Met receptor tyrosine kinase, in patients with non-small cell lung cancer (NSCLC). In connection with this decision, the sponsor company, Daiichi Sankyo, will file a Special Protocol Assessment (SPA) with the U.S. Food and Drug Administration (FDA) for a trial comparing ARQ 197 plus erlotinib against erlotinib plus placebo.

“ArQule and Daiichi Sankyo signed the ARQ 197 partnership in December 2008, and in less than two years, we are requesting an SPA for a Phase 3 trial with this potential first-in-class molecule.”

"The decision to advance ARQ 197 into Phase 3 clinical testing underscores the success of our partnership with Daiichi Sankyo," said Paolo Pucci, chief executive officer of ArQule. "ArQule and Daiichi Sankyo signed the ARQ 197 partnership in December 2008, and in less than two years, we are requesting an SPA for a Phase 3 trial with this potential first-in-class molecule."

"The efficacy observed among patients with NSCLC who received ARQ 197 provides us with encouraging evidence that ARQ 197 may be beneficial to this patient population. Based on these results, we are developing plans to support a Phase 3 clinical program and bring new hope to patients with this disease," said Dr. Kazunori Hirokawa, global head of R&D Unit, Daiichi Sankyo.

An SPA is an agreement with the FDA establishing the design, endpoints and statistical analysis of a clinical trial intended to provide the necessary data to support a New Drug Application (NDA). Following FDA review of the SPA, the two companies will implement the protocol for the Phase 3 trial and commence patient enrollment. Details regarding the trial design will be communicated at that time.

This decision follows the completion of a comprehensive review of clinical and pre-clinical data, including discussions with key opinion leaders and a meeting with the FDA following the recently completed Phase 2 trial. In this trial, treatment with ARQ 197 in combination with erlotinib showed promising overall survival and progression-free survival among patients with advanced, refractory NSCLC. Data from this trial related to overall survival and progression-free survival were statistically significant in patients with non-squamous cell histology when adjusted for imbalances in key prognostic factors.

Patients, physicians and other healthcare professionals seeking additional information regarding this trial and other trials involving ARQ 197 may call 1-800-373-7827.