ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) today announced that its investigational cancer medicine, AP26113, has received Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with anaplastic lymphoma kinase positive (ALK+) metastatic non-small cell lung cancer (NSCLC) who are resistant to crizotinib. This designation is based on results from the ongoing Phase 1/2 trial that show sustained anti-tumor activity of AP26113 in patients with ALK+ NSCLC, including patients with active brain metastases. “We are very pleased that the FDA has granted Breakthrough Therapy designation to AP26113,” stated Harvey J. Berger, M.D., chairman and chief executive officer of ARIAD. “We are encouraged by the clinical data on AP26113 that were presented recently at the European Cancer Congress, particularly in patients whose tumor had spread to the brain. We are focused on accelerating patient enrollment in the ongoing ALTA trial and on planning a front-line trial of AP26113 in treatment-naive patients.” Phase 1/2 Data Updated clinical data from the Phase 1/2 trial of AP26113 were recently shared at the 2014 European Cancer Congress. A total of 137 patients have been enrolled in the trial in the United States and Europe. Objective responses were observed in ALK+ NSCLC patients, and responses were observed in patients who were either TKI-naïve or resistant to crizotinib. Of the 72 ALK+ NSCLC patients evaluable for response, 52 (72%) demonstrated an objective response. The median duration of response was 49 weeks, and the median progression-free survival (PFS) was 56 weeks. In a subgroup analysis, 10 of 14 (71%) ALK+ NSCLC patients with active, untreated or progressing, brain metastases had evidence of radiographic improvement in those metastases. Of the seven evaluable TKI-naïve ALK+ NSCLC patients treated with AP26113, all demonstrated an objective response, including two complete responses (CR). The most common adverse events (AEs), regardless of treatment relationship and including all grades, were nausea (45%), diarrhea (37%), and fatigue (37%). Adverse events, grade 3 or higher, occurring in three or more patients were dyspnea (4%), increased lipase (4%), hypoxia (4%), fatigue (3%), alanine aminotransferase (ALT) increased (2%) and amylase increased (2%). Serious AEs, all causality, occurring in three or more patients were dyspnea (7%), pneumonia (5%), hypoxia (4%), neoplasm progression (4%), pyrexia (2%) and pulmonary embolism (2%).
The 2012 Food and Drug Administration Safety and Innovation Act (FDASIA) established the Breakthrough Therapy designation to expedite the development and review of new drugs with preliminary clinical evidence demonstrating that they may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases. The Breakthrough Therapy designation is a distinct status from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met. As of September 3, 2014, FDA listed 213 total requests for Breakthrough Designation, and 61 requests were granted. Approximately 41% of the designated products are in cancer. ( http://www.focr.org/breakthrough-therapies).About Non-Small Cell Lung Cancer and ALK Non-small cell lung cancer (NSCLC) is the most common form of lung cancer, accounting for approximately 85 percent of the estimated 228,190 new cases of lung cancer diagnosed each year in the United States, according to the American Cancer Society. Anaplastic lymphoma kinase (ALK) was first identified as a chromosomal rearrangement in anaplastic large-cell lymphoma (ALCL). Genetic studies indicate that abnormal expression of ALK is a key driver of certain types of NSCLC as well. Since ALK is generally not expressed in normal adult tissues, it represents a highly promising molecular target for cancer therapy. Approximately three to eight percent of patients with NSCLC have the ALK gene mutation. About ARIAD ARIAD Pharmaceuticals, Inc., headquartered in Cambridge, Massachusetts and Lausanne, Switzerland, is an integrated global oncology company focused on transforming the lives of cancer patients with breakthrough medicines. ARIAD is working on new medicines to advance the treatment of various forms of chronic and acute leukemia, lung cancer and other difficult-to-treat cancers. ARIAD utilizes computational and structural approaches to design small-molecule drugs that overcome resistance to existing cancer medicines. For additional information, visit http://www.ariad.com or follow ARIAD on Twitter ( @ARIADPharm). This press release contains “forward-looking statements” including, but not limited to, statements relating to preclinical and clinical data for AP26113 and its development plan. Forward-looking statements are based on management's expectations and are subject to certain factors, risks and uncertainties that may cause actual results, outcome of events, timing and performance to differ materially from those expressed or implied by such statements. These risks and uncertainties include, but are not limited to, preclinical data and early-stage clinical data that may not be replicated in later-stage clinical studies, the costs associated with our research, development, manufacturing and other activities, the initiation, conduct, timing and results of pre-clinical and clinical studies of our product candidates, the adequacy of our capital resources and the availability of additional funding, and other factors detailed in the Company's public filings with the U.S. Securities and Exchange Commission. The information contained in this press release is believed to be current as of the date of original issue. The Company does not intend to update any of the forward-looking statements after the date of this document to conform these statements to actual results or to changes in the Company's expectations, except as required by law.