Blueprint Medicines Receives FDA Authorization to Proceed with Clinical Trials for Two Drug Candidates

- FDA accepts INDs for BLU-554 in hepatocellular carcinoma and BLU-285 in unresectable, treatment-resistant GIST -

- Company on track to achieve key milestone of initiating multiple Phase 1 trials in mid-2015 -

Jul 13, 2015, 08:05 ET from Blueprint Medicines

CAMBRIDGE, Mass., July 13, 2015 /PRNewswire/ -- Blueprint Medicines (NASDAQ: BPMC) today announced that the U.S. Food and Drug Administration (FDA) accepted the Company's Investigational New Drug (IND) applications to begin Phase 1 clinical trials for its two lead drug candidates: BLU-554 for the treatment of advanced hepatocellular carcinoma (HCC) and cholangiocarcinoma, and BLU-285 for unresectable, treatment-resistant gastrointestinal stromal tumor (GIST).  Blueprint Medicines is also on track to file an IND for BLU-285 in systemic mastocytosis.

"We are delighted to reach this important milestone in the development of our two lead drug candidates to meet the urgent needs of patients facing a poor prognosis and few, if any, viable treatment options," said Jeffrey Albers, Chief Executive Officer of Blueprint Medicines. "This achievement is a testament to our team's ability to execute on our stated goal of simultaneously advancing multiple programs into clinical trials. Our powerful drug discovery engine continues to consistently generate highly selective kinase inhibitors against previously unaddressed genomic drivers of disease."

BLU-554 in Advanced HCC and Cholangiocarcinoma

BLU-554 is a potent and selective inhibitor of fibroblast growth factor receptor 4 (FGFR4).  Aberrant signaling of FGFR4 is a disease driver in up to 30 percent of HCC patients and may also play a role in a subset of patients with cholangiocarcinoma, a related cancer of the biliary duct.  HCC is the most prevalent form of liver cancer.  Liver cancer is the second-leading cause of cancer-related deaths worldwide.

In this Phase 1 trial, Blueprint Medicines expects to enroll 50 patients with advanced, unresectable HCC and an additional 10 patients with advanced, unresectable cholangiocarcinoma.  The trial will enroll patients at multiple centers in the U.S., EU and Asia.

The trial will test the safety and tolerability of escalating doses of BLU-554, with the goal of establishing a maximum tolerated dose (MTD), or a recommended dose if the MTD is not achieved. Once the MTD or recommended dose has been established, HCC and cholangiocarcinoma patients with aberrant FGFR4 signaling will be enrolled in disease-specific expansion cohorts. Additional study objectives include the assessment of early signs of biological and clinical activity using disease-specific biomarkers and response rate measurement according to Response Evaluation Criteria In Solid Tumors (RECIST).

BLU-285 in Unresectable and Treatment-Resistant GIST

BLU-285 is a potent and selective inhibitor of KIT Exon 17 and PDGFR-alpha D842V mutants, which are key disease drivers of metastatic and treatment-resistant GIST.  GIST is the most common form of sarcoma of the gastrointestinal tract.  Patients with the KIT Exon 17 and PDGFR-alpha D842V mutants are not adequately treated with existing therapies.

In this Phase 1 trial, Blueprint Medicines plans to enroll 60 patients with GIST or other relapsed or refractory solid tumors at multiple sites in the U.S., EU and Asia.  The trial will test the safety and tolerability of escalating doses of BLU-285, with the goal of establishing an MTD, or a recommended dose if the MTD is not achieved. Once the MTD or recommended dose has been established, patients with treatment-resistant GIST and PDGFR-alpha D842V mutant GIST will be enrolled in expansion cohorts. Additional study objectives include assessing early signs of biological activity using disease-specific biomarkers and clinical efficacy as measured by response rate by RECIST modified for GIST.

About Blueprint Medicines

Blueprint 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 Statements Various statements in this release concerning Blueprint Medicines' future expectations, plans and prospects, including without limitation, statements regarding Blueprint Medicines' ability to craft highly selective kinase inhibitors and deliver targeted medicines for patients with genomically defined cancers and statements concerning the planned Phase 1 clinical trials for BLU-554 and BLU-285, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. In particular, it should be noted that the data on BLU-554 and BLU-285 is early-stage and may not be replicated in clinical trials. Any forward-looking statements in this press release are based on management's current expectations of future events and are subject to a number of risks and uncertainties. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, the risk of delay of any planned clinical trials and/or development of Blueprint Medicines' drug product candidates, including BLU-554 and BLU-285. Blueprint Medicines' ability to successfully demonstrate the efficacy and safety of its drug product candidates, the pre-clinical and clinical results for its drug product candidates, which may not support further development of such drug product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials, obtaining, maintaining and protecting intellectual property, Blueprint Medicines' ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties, competition from others developing products for similar uses, Blueprint Medicines' ability to manage operating expenses, Blueprint Medicines' ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Blueprint Medicines' dependence on third parties for various functions, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Blueprint Medicines' Quarterly Report on Form 10-Q for the quarter ended March 31, 2015, as well as discussions of potential risks, uncertainties and other important factors in Blueprint Medicines' subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent Blueprint Medicines' views only as of today 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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