October 2, 2014
By Mark Terry, BioSpace.com Breaking News Staff
Cambridge, Mass.-based Ariad Pharmaceuticals, Inc. , today announced that its cancer compound AP26113 had received Breakthrough Therapy designation by the U.S. FDA. The 2012 FDA Safety and Innovation Act (FDASIA) created the Breakthrough Therapy designation to help move along the development and review of new drugs that might be a significant improvement over available therapies for patients with life-threatening and serious diseases.
AP26113 is an oral inhibitor of anaplastic lymphoma kinase (ALK). Studies have indicated that abnormal expression of ALK is implicated in certain types of non-small cell lung cancer (NSCLC) and neuroblastomas. The compound was specifically designed to overcome mutation-based resistance, including the L1196M “gatekeeper” mutation, as well as other resistancemutations that are commonly observed in patients who relapsed after early response to cancer drug crizotinib.
The Breakthrough Therapy designation was granted based on positive results from an ongoing Phase 1/2 trial. The results were recently presented at the 2014 EuropeanCancer Congress. Of the 72 ALK+ NSCLC patients who could be evaluated for response, 72 percent (52) demonstrated an objective response. The response had a median duration of 49 weeks with a median progression-free survival (PFS) of 56 weeks. A subgroup analysis of 14 ALK+ NSCLC patients with active, untreated or progressing, brain metastases showed evidence of improvement on radiographs.
“We are very pleased that the FDA has granted Breakthrough Therapy designation to AP26113,” said Harvey Berger, chairman and CEO of ARIAD in a press release. “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-naïve patients.”
ARIAD is also headquartered in Lausanne, Switzerland, and focuses on developing cancer treatments, specifically chronic and acute leukemia, lung cancer and difficult-to-treat cancers. The company uses computational and structural approaches to design small-molecule drugs in an attempt to overcome resistance to existing chemotherapeutic compounds.
In early August ARIAD announced a deal with Israel-based Medison Pharma Ltd. to commercialize Iclusig (ponatinib) in Israel for patients with Philadelphia-positive (Ph+) leukemias such as Chronic Myelogeneous Leukemia (CML). The drug is approved in the U.S., although it has had a slightly checkered history. A study indicated patients on the drug had an increasing number of arterial thrombotic events, in other words, blood clots. The drug was briefly drawn off the market, its safety reevaluated, then returned to commercial sales with tougher safety warnings. However, since the drug is something of a last-shot for patients who have developed resistance to other treatments, and appears very effective with closer to a 60 percent response rate as compared to a 30 percent response rate as a third-line treatment compared to its peers, it would still seem to be a viable product.
