Blueprint Medicines Announces New Drug Discovery Program – 10th International Liver Cancer Association (ILCA) Annual Conference

Blueprint Medicines
Blueprint Medicines (PRNewsFoto/Blueprint Medicines)

Blueprint Medicines Announces New Drug Discovery Program Targeting PRKACA Kinase Fusions for the Treatment of Fibrolamellar Carcinoma at 10th International Liver Cancer Association (ILCA) Annual Conference

CAMBRIDGE, Mass., Sept. 9, 2016 /PRNewswire/ — Blueprint Medicines Corporation (NASDAQ: BPMC), a leader in discovering and developing highly selective kinase medicines for patients with genomically defined diseases, today disclosed a new drug discovery program targeting protein kinase cAMP-activated catalytic subunit alpha (PRKACA) fusions for the treatment of fibrolamellar carcinoma (FLC). The announcement was made during an oral presentation on September 8, 2016 at the 10th ILCA Annual Conference inVancouver, Canada on Blueprint Medicines’ efforts to develop targeted therapies for patients with hepatocellular carcinoma.

“Patients with liver cancers have historically suffered from a lack of genomically defined therapeutic options. Our new drug discovery program represents our commitment to patients with orphan cancers,” said Christoph Lengauer, Chief Scientific Officer of Blueprint Medicines. “The addition of this program is a testament to the strength of our scientists and genomics platform, as well as to the quality of our unique library of kinase inhibitors and continued momentum of our discovery efforts.”

FLC is a rare and distinct subtype of liver cancer that typically arises in young adults. Currently, there are no approved therapies, and surgery is the only available treatment option for some patients, but most patients inevitably progress. Research published in 2014 by the laboratory of Dr. Sanford Simon, Professor and Head of the Laboratory of Cellular Biophysics at The Rockefeller University in New York in Science (Honeyman J.N. et al., Detection of a recurrent DNAJB1-PRKACA chimeric transcript in fibrolamellar hepatocellular carcinoma) and Blueprint Medicines in Nature Communications(Stransky N. et al., The landscape of kinase fusions in cancer) identified PRKACA kinase fusions in FLC. Blueprint Medicines estimates that more than ninety percent of patients with FLC harbor the PRKACA fusion, which is the only known recurrent genomic event in FLC and is considered to be the driver gene of the disease. Given the high medical need and the opportunity for a potentially transformative therapeutic impact, Blueprint Medicines is developing drug candidates for the selective inhibition of PRKACA.

“FLC is a devastating disease, and patients are in great need of effective therapeutic options,” said Dr. Sanford Simon. “Targeting PRKACA represents a promising opportunity to develop innovative therapeutics that could make a difference in these patients’ lives.”

The unveiling of the PRKACA program expands Blueprint Medicines’ existing pipeline, which includes Phase 1 clinical programs for its drug candidates BLU-554, a selective inhibitor of FGFR4 for patients with hepatocellular carcinoma, and BLU-285, a selective inhibitor of both exon 17 mutant KIT and D842V mutant PDGFRα for patients with advanced systemic mastocytosis and unresectable, treatment-resistant gastrointestinal stromal tumors.  Blueprint Medicines expects to report preliminary data from the dose escalation portion of each of these Phase 1 clinical trials by the end of 2016.  Blueprint Medicines’ existing pipeline also includes preclinical programs for its drug candidate BLU-667, a selective inhibitor of RET activating mutations, fusions and predicted resistant mutants found in non-small cell lung cancer and thyroid cancer, a rare genetic disease program in collaboration with Alexion Pharma Holding and cancer immunotherapy programs in collaboration with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc.

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 estimated number of patients with FLC that harbor the PRKACA fusion; and the timing of clinical data or proof of concept for preclinical and clinical programs, including, without limitation, the timing and type of preliminary clinical data for Blueprint Medicines’ Phase 1 clinical trials for BLU-285 and BLU-554; and Blueprint Medicines’ strategy, business plans and focus. 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; Blueprint Medicines’ ability to develop and commercialize companion diagnostics for its current and future drug candidates, including a companion diagnostic for BLU-554 with Ventana Medical Systems, Inc. and a companion diagnostic for BLU-285 with QIAGEN Manchester Limited; and the success of Blueprint Medicines’ rare genetic disease collaboration with Alexion Pharma Holding and its cancer immunotherapy collaboration with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. These and other risks and uncertainties are described in greater detail in the section entitled “Risk Factors” in Blueprint Medicines’ Quarterly Report on Form 10-Q for the quarter ended June 30, 2016, as filed with the Securities and Exchange Commission (SEC) on August 9, 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.