CAMBRIDGE, Mass., Jan. 4, 2019 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced "2020 Blueprint," a two-year strategy to launch Blueprint Medicines' global commercial business. Under this strategy, by the end of 2020, the company expects to have two marketed products and at least four additional marketing applications pending in the United States and Europe. In addition, Blueprint Medicines today announced recent milestones and key goals through 2020 across its differentiated portfolio of investigational precision therapies.

"Our vision is to rapidly evolve Blueprint Medicines into the leading global precision therapy company, with a robust scientific platform reproducibly designing innovative compounds and an effective and nimble commercial organization delivering a portfolio of important medicines to patients worldwide," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "As we enter the new year, we are at the precipice of this transformation with the planned submission of our first new drug application for our lead therapeutic candidate avapritinib and growing momentum in building our commercial capabilities. Combined with seven ongoing or planned registration-enabling clinical trials for avapritinib and BLU-667, we believe Blueprint Medicines is well-positioned to quickly capitalize on this first potential regulatory approval and realize our goal of becoming a portfolio-based commercial-stage company."

"2020 Blueprint" Global Business Strategy

The "2020 Blueprint" strategy establishes a path to transform Blueprint Medicines into a global commercial enterprise focused on delivering a portfolio of precision therapies to patients with cancer and rare diseases. Under this strategy, Blueprint Medicines anticipates achieving the following by the end of 2020:

• 2 marketed products in the United States and 1 marketed product in Europe
• 4 additional marketing applications pending in the United States and Europe
• 6 therapeutic candidates in global clinical development
• 8 research programs that leverage strategic areas of focus

"Our '2020 Blueprint' strategy extends to our ongoing research efforts, where we are advancing multiple programs designed to complement our scientific and clinical expertise and planned commercial profile," said Marion Dorsch, Ph.D., Chief Scientific Officer of Blueprint Medicines. "Under this strategy, we have three research areas of focus: franchise opportunities in gastrointestinal stromal tumors, systemic mastocytosis and lung cancer where we believe our understanding of mutational drivers gives us an advantage; cancer immunotherapy under our ongoing collaboration with Roche; and novel genetic drivers that we characterize and target with our differentiated scientific platform."

Recent Portfolio Milestones and Key Goals through 2020

RECENT MILESTONES:

Blueprint Medicines today announced:

• Top-line results from the Phase 1 NAVIGATOR clinical trial of avapritinib in patients with advanced gastrointestinal stromal tumors (GIST), as of a data cutoff date of November 16, 2018. These data will be used to support the submission of a planned new drug application (NDA) to the U.S. Food and Drug Administration (FDA) in the first half of 2019 for the treatment of patients with PDGFRA Exon 18 mutant GIST, which primarily includes PDGFRα D842V GIST, and fourth-line GIST. The primary endpoints for registration are objective response rate (ORR) and duration of response (DOR) based on central radiology and modified Response Evaluation Criteria in Solid Tumors version 1.1 (mRECIST 1.1) criteria.
• In 43 patients with PDGFRA Exon 18 mutant GIST treated with a starting dose of 300 or 400 mg once daily (QD), the ORR was 86 percent (one response pending confirmation). Median DOR was not reached.
• In 111 patients with fourth-line GIST treated with a starting dose of 300 or 400 mg QD, the ORR was 22 percent (one response pending confirmation). Median DOR was 10.2 months.
• Top-line safety results were consistent with those previously reported. Avapritinib was well-tolerated, and most adverse events (AEs) reported by investigators were Grade 1 or 2. Across all doses (n=237), only 23 patients (9.7 percent) discontinued treatment with avapritinib due to treatment-related AEs.
• Two registration-enabling clinical trials for avapritinib in systemic mastocytosis (SM) are now underway. The first clinical site recently opened for the Phase 2 PIONEER clinical trial for patients with indolent and smoldering SM, and initial patient screening for this trial is anticipated in January 2019. In addition, patient dosing has been initiated in the Phase 2 PATHFINDER clinical trial for patients with advanced SM.
• As part of the collaboration with CStone Pharmaceuticals, the China National Medicinal Products Administration has approved an investigational new drug (IND) application for the ongoing global Phase 1 trial of BLU-554 in advanced hepatocellular carcinoma. The companies expect to initiate patient enrollment in the trial by the middle of 2019.
• The FDA has cleared an IND application for BLU-782, a selective ALK2 inhibitor in development for patients with fibrodysplasia ossificans progressiva (FOP), and the company plans to initiate a Phase 1 clinical trial in healthy volunteers in the first quarter of 2019.

KEY GOALS:

Blueprint Medicines today announced the following key goals and anticipated milestones through 2020:

Avapritinib: advanced gastrointestinal stromal tumors

• Submit an NDA to the FDA for PDGFRA Exon 18 mutant GIST and fourth-line GIST in the first half of 2019.
• Present additional data from the Phase 1 NAVIGATOR trial in the first half of 2019.
• Complete enrollment of the Phase 3 VOYAGER trial in third- or fourth-line GIST in the second half of 2019.
• Initiate the Phase 3 COMPASS-2L precision medicine trial in second-line GIST in the second half of 2019.
• Submit an NDA to the FDA for third-line GIST in 2020.

Avapritinib: systemic mastocytosis

• Present updated data from the Phase 1 EXPLORER trial in advanced SM in the first half of 2019.
• Present initial data from the Phase 2 PIONEER trial in indolent and smoldering SM in the second half of 2019.
• Complete enrollment of the Phase 2 PATHFINDER trial in advanced SM in the second half of 2019.
• Submit an NDA to the FDA for advanced SM in 2020.

BLU-667: RET-altered non-small cell lung cancer (NSCLC), medullary thyroid cancer (MTC) and other solid tumors

• Present updated data from the Phase 1 ARROW trial in RET-altered cancers in the first half of 2019.
• Complete enrollment of previously treated NSCLC and MTC cohorts in the Phase 1 ARROW trial in the first half of 2019.
• Initiate a Phase 3 trial in first-line RET-fusion NSCLC in the second half of 2019.
• Initiate a Phase 2 combination trial of BLU-667 and osimertinib in treatment-resistant, EGFR-mutant NSCLC harboring an acquired RET alteration in the second half of 2019.
• Submit an NDA to the FDA for second-line RET-fusion NSCLC and second-line RET-mutant MTC in the first half of 2020.

BLU-554: advanced hepatocellular carcinoma

• Enroll the first patient in China in the ongoing global Phase 1 trial of BLU-554 under the collaboration with CStone Pharmaceuticals by the middle of 2019.
• Initiate a Phase 1 combination trial of BLU-554 and CS-1001, CStone Pharmaceuticals' anti-PD-L1 inhibitor, in China in the second half of 2019.

BLU-782: fibrodysplasia ossificans progressiva

• Initiate a Phase 1 trial in healthy volunteers in the first quarter of 2019.
• Initiate a Phase 2 trial in patients with FOP in the first half of 2020.

Research portfolio

• Provide an update on the company's robust and diverse research portfolio, including disclosure of up to two new targets, at a Blueprint Medicines Research and Development day in 2019.
• Nominate at least one new wholly-owned discovery program in 2019.

About Blueprint Medicines

Blueprint Medicines is a precision therapy company striving to improve human health. With a focus on genomically defined cancers, rare diseases and cancer immunotherapy, we are developing transformational medicines rooted in our leading expertise in protein kinases, which are proven drivers of disease. Our uniquely targeted, scalable approach empowers the rapid design and development of new treatments and increases the likelihood of clinical success. We are currently advancing four investigational medicines in clinical development, along with multiple research programs. For more information, visit www.BlueprintMedicines.com and follow us on Twitter (@BlueprintMeds) and LinkedIn.

Cautionary Notes 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 plans and timelines for the development of avapritinib, BLU-554, BLU-667 and BLU-782; the potential benefits of Blueprint Medicines' current and future drug candidates in treating patients; Blueprint Medicines' "2020 Blueprint" strategy, key goals and anticipated milestones through 2020; plans and timelines for marketed products and marketing applications in the United States and Europe, therapeutic candidates in clinical development and research programs; 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 candidates, including avapritinib, BLU-554, BLU-667 and BLU-782; Blueprint Medicines' advancement of multiple early-stage efforts; Blueprint Medicines' ability to successfully demonstrate the safety and efficacy of its drug candidates; the preclinical and clinical results for Blueprint Medicines' drug candidates, which may not support further development of such drug candidates; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials; Blueprint Medicines' ability to develop and commercialize companion diagnostic tests for its current and future drug candidates, including companion diagnostic tests for avapritinib for PDGFRα D842V-driven GIST, BLU-554 for FGFR4-driven hepatocellular carcinoma and BLU-667 for RET-driven NSCLC; the success of Blueprint Medicines' current and future collaborations, including its cancer immunotherapy collaboration with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. and its collaboration with CStone Pharmaceuticals. 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 September 30, 2018, as filed with the Securities and Exchange Commission (SEC) on October 30, 2018, and any other filings that Blueprint Medicines has made or 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. Except as required by law, Blueprint Medicines explicitly disclaims any obligation to update any forward-looking statements.

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SOURCE Blueprint Medicines

Investor and Media Contacts: Kristin Hodous, 617-714-6674, khodous@blueprintmedicines.com; Jim Baker, 617-844-8236, jbaker@blueprintmedicines.com