ARIAD Initiates Submission Of New Drug Application For Brigatinib To The U.S. Food And Drug Administration Ahead Of Plan

ARIAD Pharmaceuticals, Inc. recently announced the initiation of a New Drug Application (NDA) submission for its investigational anaplastic lymphoma kinase (ALK) inhibitor, brigatinib, to the U.S. Food and Drug Administration (FDA). ARIAD is seeking U.S. marketing approval of brigatinib for patients with ALK+ non-small cell lung cancer (NSCLC) who are resistant to crizotinib. Brigatinib received Breakthrough Therapy designation from the FDA for the treatment of patients with ALK+ NSCLC whose tumors are resistant to crizotinib, and was granted orphan drug designation by the FDA for the treatment of ALK+ NSCLC. The Company is seeking accelerated approval for brigatinib from the FDA and plans to request a priority review of the application.

“We are ahead of our previously announced schedule for initiating the submission of our brigatinib NDA to the FDA and we are grateful to have breakthrough status from the FDA, which provides the opportunity to utilize the rolling submission process,” stated Paris Panayiotopoulos, president and chief executive officer of ARIAD. “If approved, we believe that brigatinib will become an important new medicine for ALK+ NSCLC patients who have become resistant or intolerant to prior crizotinib therapy and will offer additional hope to these patients and their families.”

ARIAD’s NDA is a rolling submission which will occur in three parts. The initial submission contains all nonclinical portions of the NDA and will be followed by submissions of the chemistry, manufacturing and controls (CMC) and clinical data. The rolling NDA submission is expected to be complete in the third quarter of 2016.

Phase 2 ALTA Results Presented at ASCO
Results from the ongoing Phase 2 ALTA trial of brigatinib reported at this year’s annual meeting of the American Society of Clinical Oncology earlier this month showed that, in patients on the 180 mg regimen (Arm B, n=110) with a median follow-up of 8.3 months (range 0.1—20.2), 54 percent achieved a confirmed objective response, the trial’s primary endpoint. In this arm, the median progression free survival (PFS) exceeded one year (12.9 months) in this post-crizotinib setting. Additionally, a confirmed intracranial objective response rate (ORR) of 67 percent (12/18) was achieved in patients with measurable brain metastases.

The most common treatment-emergent adverse events (TEAEs; ≥ 25% of all patients, [Arm B/A]), regardless of relationship to treatment, were nausea (40%/33%), diarrhea (38%/19%), cough (34%/18%), and headache (27%/28%). TEAEs, ≥ grade 3, occurring in ≥ 5 percent of all patients (Arm B/A), were increased blood creatine phosphokinase (9%/3%) and hypertension (6%/6%).

About Brigatinib
Brigatinib is an investigational, targeted cancer medicine discovered internally at ARIAD. It is in development for the treatment of patients with anaplastic lymphoma kinase positive (ALK+) non-small cell cancer (NSCLC) whose disease is resistant to crizotinib. Brigatinib is currently being evaluated in the global Phase 2 ALTA trial that is the basis for its initial regulatory review. ARIAD has also initiated the Phase 3 ALTA 1L trial to assess the efficacy of brigatinib in comparison to crizotinib. More information on brigatinib clinical trials, including the expanded access program (EAP) can be found here (http://www.ariad.com/research-development/brigatinib/).

About ARIAD
ARIAD Pharmaceuticals, Inc., headquartered in Cambridge, Massachusetts is focused on discovering, developing and commercializing precision therapies for patients with rare cancers. ARIAD is working on new medicines to advance the treatment of rare forms of chronic and acute leukemia, lung cancer and other rare cancers. ARIAD utilizes computational and structural approaches to design small-molecule drugs that overcome resistance to existing cancer medicines. For more information, visit http://www.ariad.com.