CAMBRIDGE, Mass.--(BUSINESS WIRE)--Oct. 2, 2014--
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
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
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
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.
Source: ARIAD Pharmaceuticals, Inc.
ARIAD Pharmaceuticals, Inc.
Liza Heapes, 617-621-2315