ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) today reported financial results for the third quarter and nine months ended September 30, 2012 and provided an update on corporate developments.
“We are advancing the clinical development of ponatinib with the initiation of the global, Phase 3 EPIC trial in patients with newly diagnosed chronic myeloid leukemia (CML) and the start of a Phase 1/2 trial in Japan,” stated Harvey J. Berger, M.D., chairman and chief executive officer of ARIAD. “We also presented positive clinical proof-of-concept data on AP26113 and continue to advance its development. Importantly, we are now commercial-ready in the U.S. for the potential approval of ponatinib in the first quarter of 2013 and are building our infrastructure in Europe for anticipated ponatinib approval in the third quarter of next year.”
Ponatinib Clinical Development Progress
- The U.S. Food and Drug Administration (FDA) accepted for filing the New Drug Application (NDA) of ponatinib in patients with resistant or intolerant CML or Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL). The FDA granted ARIAD’s request for Priority Review of ponatinib and established an action date of March 27, 2013 under the Prescription Drug User Fee Act (PDUFA). The Company anticipates approval and commercial launch of ponatinib in the U.S. in the first quarter of 2013.
- ARIAD also submitted a Marketing Authorization Application (MAA) for ponatinib to the European Medicines Agency (EMA). ARIAD is seeking marketing approval in the European Union of ponatinib in adult patients with resistant or intolerant CML or Ph+ ALL. The Committee for Medicinal Products for Human Use (CHMP) granted ARIAD’s request for accelerated assessment of the MAA, potentially decreasing the regulatory review time. ARIAD anticipates approval of ponatinib in the E.U. in the third quarter of 2013.
- Approximately 100 patients with CML or Ph+ALL at 23 centers in the U.S. are now receiving ponatinib through an expanded access protocol. Forty of these patients are in the chronic-phase of the disease. Patients on this expanded access protocol could be eligible to transition to commercial use of ponatinib following its anticipated approval early next year. These U.S. patients are part of a broad, global expanded access program that includes more than 400 patients, some of whom are receiving ponatinib through compassionate-use programs.
- ARIAD will present follow-up data from the pivotal Phase 2 PACE trial at the upcoming American Society of Hematology (ASH) Annual Meeting that will be held in Atlanta, GA, December 8 to 11, 2012. The ASH presentation will include 12 months of available response rate and duration of response data from the patients enrolled in the trial.
- Patient enrollment is underway in the global, Phase 3 EPIC trial of ponatinib in patients with newly diagnosed CML. This trial compares ponatinib to imatinib and has a primary endpoint of major molecular response at 12 months. ARIAD anticipates full patient enrollment in the trial by end of 2013, and the study includes an interim analysis of the primary endpoint 12 months after half of the approximately 500 patients in the trial have been enrolled.
Ready for U.S. Commercialization of Ponatinib
- ARIAD is commercially ready in the U.S. with all account executives now hired and trained. In addition to the account executives, the U.S. commercial team consists of regional business directors, corporate account managers, global marketing and distribution leaders, managed markets and payer reimbursement experts, with support from medical affairs and medical information personnel.
- In Europe, ARIAD has made major strides in executing on its commercial plan with the initiation of early-access programs and implementation of pricing and reimbursement activities. The European supply chain has been established, and key hires for the European leadership team have been made. ARIAD is committed to being commercial-ready in Europe on July 1, 2013.
- Dosing continues in the ongoing Phase 1/2 clinical trial of AP26113, ARIAD’s investigational dual inhibitor of epidermal growth factor receptor (EGFR) and anaplastic lymphoma kinase (ALK). AP26113 is now being administered at a once-daily oral dose of 300 mg. Clinical investigators are enrolling patients at seven sites in the U.S., and additional sites will be opened shortly in Europe. Patients with non-small cell lung cancer (NSCLC) are now exclusively being enrolled in the trial.
- Phase 1 data presented at the recent European Society of Medical Oncology meeting provided compelling clinical evidence of the anti-tumor activity of AP26113 at multiple dose levels in patients with ALK-positive NSCLC and initial clinical evidence of anti-tumor activity in patients with EGFR-mutant NSCLC. Importantly, AP26113 showed activity in ALK-positive patients with brain metastases.
- Planning is actively underway for pivotal trial(s) of AP26113 to start in 2013 in ALK-positive NSCLC patients. ARIAD will also plan to begin a pivotal trial in patients with EGFR-mutant NSCLC who have failed prior EGFR inhibitor therapy, dependent on the clinical findings from the Phase 2 portion of the currently ongoing Phase 1/2 trial.