Blueprint Medicines to Present Preclinical Data on RET Inhibitors in an Oral Presentation at American Association for Cancer Research Annual Meeting

Mar 17, 2016, 08:00 ET from Blueprint Medicines

CAMBRIDGE, Mass., March 17, 2016 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ: BPMC), a leader in discovering and developing highly selective kinase medicines for patients with genomically defined diseases, today announced that it will present preclinical data highlighting the identification of potent and selective RET inhibitors, including its development candidate BLU-667, at the American Association for Cancer Research (AACR) annual meeting, taking place April 16-20 in New Orleans.

Activating RET kinase fusions and mutations have emerged as important drivers of disease in a variety of cancers, including non-small cell lung cancer (NSCLC) and papillary and medullary thyroid cancers.  RET kinase fusions are found in 1-2% of lung adenocarcinomas, and RET activating mutations have been identified in approximately 50% of medullary thyroid cancers and approximately 10% of papillary thyroid cancers.  In addition, Blueprint Medicines has identified RET fusions at low frequency in several other cancers including colon and breast.  Several approved multi-kinase inhibitors with RET activity have been evaluated in clinical trials. However, to date, clinical activity attributable to RET inhibition has been uncertain for these drugs, likely due to insufficient inhibition of the target and limitations of off-target toxicities.

In preclinical studies to be presented at AACR, Blueprint Medicines' RET inhibitors showed potent inhibition of RET activity in RET fusion positive lung adenocarcinoma and papillary thyroid cancer cell lines as well as medullary thyroid cancer cell lines driven by an activating RET point mutation.  Administration of Blueprint Medicines' RET inhibitors in RET-driven lung cancer and thyroid cancer in vivo xenograft models resulted in tumor regression with high levels of sustained RET and downstream pathway inhibition at well tolerated doses.  In addition, Blueprint Medicines' RET inhibitors retained in vitro and in vivo activity against RET resistant mutants that Blueprint Medicines predicts may arise in patients, providing a potential opportunity for a meaningful and durable clinical response in RET-driven disease.  Blueprint Medicines plans to file an IND for BLU-667 for the treatment of patients with RET-driven tumors by the end of 2016.

Oral Presentation Details:

  • Date & Time: Monday, Apr 18, 2016, 3:05 PM - 3:20 PM
  • Presentation Title: The development of potent, selective RET inhibitors that target both wild-type RET and prospectively identified resistance mutations to multi-kinase inhibitors
  • Abstract Number: 2641
  • Location: New Orleans Theater A, Morial Convention Center

About Blueprint Medicines

Blueprint Medicines is developing a new generation of highly selective and potent kinase medicines to improve the lives of patients with genomically defined diseases. The Company's approach is rooted in a deep understanding of the genetic blueprint of cancer and other diseases driven by the abnormal activation of kinases. Blueprint Medicines is advancing three programs in clinical development for subsets of patients with gastrointestinal stromal tumors, hepatocellular carcinoma and systemic mastocytosis, as well as multiple programs in research and preclinical development. For more information, please visit

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the potential inability of approved multi-kinase inhibitors to sufficiently inhibit RET and Blueprint Medicines' ability to develop potential treatments for RET-driven disease, including, without limitation, treatments that provide more meaningful and durable clinical response. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks and uncertainties related to the delay of any current or planned clinical trials or the development of Blueprint Medicines' drug product candidates, including BLU-285 and BLU-554; Blueprint Medicines' advancement of multiple early-stage efforts; Blueprint Medicines' ability to successfully demonstrate the efficacy and safety of its drug product candidates; the preclinical and clinical results for Blueprint Medicines' drug product candidates, which may not support further development of such drug product candidates; and actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in Blueprint Medicines' Annual Report on Form 10-K for the year ended December 31, 2015, as filed with the Securities and Exchange Commission (SEC) on March 11, 2016, and other filings that Blueprint Medicines may make with the SEC in the future. Any forward-looking statements contained in this press release represent Blueprint Medicines' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Blueprint Medicines explicitly disclaims any obligation to update any forward-looking statements.

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SOURCE Blueprint Medicines