Investors & Media

Transformative science, targeted medicines

We are working to advance a deep pipeline of precision therapies, designed to allow patients to live longer, better lives. Our approach combines world-leading expertise in protein kinases with sound execution and an intense focus on discovery.

News release details

Blueprint Medicines Highlights Advances in Preclinical Discovery Efforts

November 9, 2015 at 4:16 PM EST

CAMBRIDGE, Mass., Nov. 9, 2015 /PRNewswire/ -- Blueprint Medicines (NASDAQ: BPMC), a leader in discovering and developing highly selective kinase medicines for patients with genomically defined diseases, today provided an update on certain of its preclinical discovery efforts in oncology and rare genetic diseases, in addition to reporting its financial results for the third quarter ended September 30, 2015.

Blueprint Medicines

"We are excited about the ways in which we continue to leverage our novel library of kinase inhibitors to advance our drug discovery pipeline and target kinases as drivers of disease," said Christoph Lengauer, Ph.D., Chief Scientific Officer of Blueprint Medicines. "During the third quarter of 2015, we presented preclinical data validating the advancement of BLU-554 into clinical development and identifying PRKACA as a novel target for therapeutic intervention. In addition, we have predicted resistance mutations in NTRK, some of which have recently been clinically observed by others, and we are advancing compounds that inhibit NTRK and resistant mutants into preclinical safety studies. We have also entered into a collaboration with the Children's Hospital of Eastern Ontario seeking to identify new therapeutic opportunities for rare genetic diseases."

Oncology—Preclinical Discovery Highlights

At the 9th Annual Conference of the International Liver Cancer Association (ILCA) in September, Blueprint Medicines presented new preclinical data on BLU-554, one of Blueprint Medicines' lead drug candidates, and DNAJB1-PRKACA, a recurrent fusion in certain hepatocellular carcinoma (HCC) patients.

  • The new preclinical data presented by Blueprint Medicines at ILCA further validate the advancement of BLU-554 into clinical development. BLU-554 is an exquisitely selective inhibitor of fibroblast growth factor receptor 4 (FGFR4). The new data show that BLU-554 demonstrated anti-tumor activity in preclinical models of HCC with aberrant FGFR4 signaling. Blueprint Medicines is currently enrolling patients in a dose-escalation, Phase 1 clinical trial of BLU-554 for the treatment of advanced HCC and cholangiocarcinoma.
  • In addition, Blueprint Medicines continues to pursue research on a novel drug target for fibrolamellar hepatocellular carcinoma (FL-HCC). FL-HCC is a rare subtype of liver cancer that arises in adolescents and young adults. Preclinical data presented by Blueprint Medicines at ILCA indicate that more than 90 percent of patients with FL-HCC have the DNAJB1-PRKACA fusion, which is likely a driver of FL-HCC. The new data highlight the role of PRKACA in FL-HCC and its potential as a novel target for therapeutic intervention.

In addition, utilizing its proprietary platform, Blueprint Medicines has predicted resistance mutations in the neurotrophic tyrosine receptor kinase (NTRK).  Recently, some of these mutations were clinically observed by others in tumors that developed resistance after initially responding to an NTRK inhibitor.[1]Blueprint Medicines is advancing compounds that inhibit NTRK and resistant mutants into preclinical safety studies. Research published by Blueprint Medicines scientists in the September 10, 2014 edition of Nature Communications(Stransky N. et al., The landscape of kinase fusions in cancer) suggests NTRK  fusion events may drive tumorigenesis in multiple cancers, with a total of 23 NTRK1, NTRK2 and NTRK3 fusions identified across nine tumor types.

Rare Genetic Diseases—Preclinical Discovery Highlights

Continuing its focus on rare genetic diseases, Blueprint Medicines is currently collaborating with the Children's Hospital of Eastern Ontario (CHEO). Blueprint Medicines and CHEO are seeking to identify potential therapeutic opportunities for rare genetic diseases utilizing Blueprint Medicines' proprietary compound library, which will allow Blueprint Medicines to initiate development of future programs in rare genetic diseases.

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 Blueprint Medicines' discovery and development plans or programs, including without limitation for NTRK, FL-HCC and rare genetic diseases. 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' 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' Quarterly Reports on Form 10-Q for the quarters ended March 31, 2015 and June 30, 2015, as filed with the Securities and Exchange Commission (SEC) on June 11, 2015 and August 10, 2015, 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.

[1]   Russo M, Misale S, Wei G et al. Acquired resistance to the TRK inhibitor entrectinib in colorectal cancer [published online November 6, 2015]. Cancer Discov. 2015. doi: 10.1158/2159-8290. Accessed November 8, 2015.


To view the original version on PR Newswire, visit:

SOURCE Blueprint Medicines

Investor Relations: Hannah Deresiewicz, Stern Investor Relations, Inc., 212-362-1200,; Media Relations: Naomi Aoki, Ten Bridge Communications, Inc., 617-283-4298,