Blueprint Medicines Receives FDA Orphan Drug Designation for Novel Drug Candidate for Treatment of Hepatocellular Carcinoma

CAMBRIDGE, Mass., Sept. 30, 2015 /PRNewswire/ -- Blueprint Medicines (NASDAQ: BPMC) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its novel drug candidate BLU-554 for the treatment of hepatocellular carcinoma (HCC). BLU-554, an exquisitely selective inhibitor of fibroblast growth factor receptor 4 (FGFR4), is currently being evaluated in a Phase 1 clinical trial in patients with advanced HCC and cholangiocarcinoma.

"We believe BLU-554 represents a promising new approach for molecularly targeted therapy in HCC," said Jeffrey Albers, Chief Executive Officer of Blueprint Medicines. "Patients with advanced HCC have a poor prognosis, and there's a clear need for better treatment options. We are pleased that we continue to make progress toward our goal of delivering a highly targeted medicine to improve the lives of HCC patients."

Aberrantly activated signaling of FGFR4 may be a key driver in up to 30 percent of HCC patients, according to an analysis by Blueprint Medicines, and BLU-554 has been shown to have significant anti-tumor activity in preclinical models of HCC driven by aberrant FGFR4 signaling. Liver cancer is the second leading cause of cancer-related deaths worldwide, with HCC accounting for most liver cancers. In the United States, HCC is the fastest rising cause of cancer-related deaths, and over the past two decades, the incidence of HCC has tripled while the five-year survival rate has remained below 12 percent.

The FDA's Office of Orphan Drug Products grants orphan status to support development of medicines for safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. Orphan drug designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.

About Blueprint MedicinesBlueprint Medicines makes kinase drugs to treat patients with genomically defined diseases. Led by a team of industry innovators, Blueprint Medicines integrates a novel target discovery engine and a proprietary compound library to understand the blueprint of cancer and craft highly selective therapies. This empowers the Blueprint Medicines team to develop patient-defined medicines aimed at eradicating cancer.

Forward-Looking StatementsThis 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 for BLU-554 to provide clinical benefit to patients, the potential benefits of orphan drug designation for BLU-554 and the estimated number of HCC patients whose cancer tumors have aberrantly activated signaling of FGFR4. 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 planned clinical trials and/or development of Blueprint Medicines' drug product candidates, including BLU-554; Blueprint Medicines' ability to successfully demonstrate the efficacy and safety of its drug product candidates; the pre-clinical 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.

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SOURCE Blueprint Medicines