April 9, 2013

Cambridge, MA, and Washington, April 8, 2013 – ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) today announced the presentation of preclinical data on ponatinib (Iclusig™) , at the American Association for Cancer Research (AACR) Annual Meeting 2013, in Washington.  In separate studies, ponatinib is shown to potently inhibit RET, a clinically proven oncogenic driver of medullary thyroid cancer (MTC) and non-small cell lung cancer (NSCLC), and FGFR, which is commonly mutated in endometrial, lung and other cancers.  

The first preclinical study, “Ponatinib is a highly-potent inhibitor of activated variants of RET found in MTC and NSCLC,” shows that ponatinib inhibits naturally occurring activating mutants of RET found in MTC and NSCLC at clinically achievable plasma concentrations.  The potency of ponatinib was found to substantially exceed that of other approved tyrosine kinase inhibitors with RET activity.  These results provide strong support for the clinical evaluation of ponatinib in patients with RET-driven cancers. 

“The unique design features that allow ponatinib to overcome mutation-based resistance in CML, including the gatekeeper mutation, also apply to its activity against its other tyrosine kinase targets including RET, FGFR and KIT,” stated Timothy P. Clackson, Ph.D., president of research and development and chief scientific officer at ARIAD.  “These preclinical data strongly support moving forward with evaluating ponatinib in RET-positive cancers, and we look forward to the start of a Phase 2 investigator-sponsored trial in patients with RET-positive NSCLC in this second quarter of 2013.” 

In the second preclinical study, “Ponatinib potently inhibits the activity of mutant variants of FGFR commonly found in endometrial, lung and other cancers,” the ability of ponatinib to inhibit a broad panel of naturally occurring mutant variants of FGFR1, 2, 3, and 4 was evaluated.  Ponatinib potently blocked a variety of mutant variants of FGFR, with particularly promising activity against mutant variants of FGFR2 that have been observed in endometrial and squamous cell carcinomas (SCCs) of the lung.  Specifically, ponatinib inhibits the eight mutants that make up more than 90 percent of the mutations observed in endometrial cancer patients, and six mutants observed in SCC at clinically achievable plasma concentrations.  Ponatinib also shows encouraging activity against FGFR1/3 fusion proteins found in glioblastoma multiforme.  These data support clinical evaluation of ponatinib in FGFR-driven cancers, including a Phase 2 investigator-sponsored trial of ponatinib in patients with SCCs that is currently underway and enrolling patients.   



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 or follow ARIAD on Twitter (@ARIADPharm).  

This press release contains “forward-looking statements” including, but not limited to, updates on preclinical developments for our product candidates. 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 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.




Membership Deliverables

MassBio Membership gives you:

Purchasing power-MassBio negotiates a broad-based collection of services on behalf of member companies and passes the savings on to its members. 

Networking and professional development-MassBio organizes and executes numerous conferences, committee meetings, and networking events throughout the year.

MassBio Signature Events-Attend our Investors Forum, Innovator Roundtables, Pharma Days, our Golf Classic Fundraiser, Policy Leadership Breakfast, and many others.

Public policy & Advocacy-As the state's leading advocate for our life science super cluster, we fight for public policy initiatives and tax incentives in Massachusetts and Washington, D.C., that will enable biotechnology companies to do their best work.

Economic development-MassBio provides expert advice and guidance to help companies find suitable locations and resources as they move to and grow in the Bay State. We make Massachusetts a better place for your business.

Visibility/Resources-Members are listed on a searchable directory that includes a profile and link to the company's website. We highlight your company's latest news by posting to our MassBio member news page. In addition, members can post job openings on our website free!

Educational incentives-We promote education, workforce development and lifelong learning through the MassBioEd Foundation. The foundation customizes training programs for companies and educational institutions and promotes science in the classroom through BioTeach. MassBio members receive a discount on courses offered by the foundation.