Blueprint Medicines Announces Data Presentations at ASCO and EHA Meetings from Registration-Enabling Clinical Trials of Avapritinib and BLU-667 Across Multiple Patient Populations

CAMBRIDGE, Mass., May 16, 2019 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced the presentation of updated clinical data for its potent and highly selective investigational medicines avapritinib and BLU-667.

At the 2019 American Society of Clinical Oncology (ASCO) Annual Meeting and 24^th Congress of the European Hematology Association (EHA), Blueprint Medicines plans to present:

• Data from the expansion portion of the registration-enabling ARROW trial of BLU-667 in patients with RET-altered non-small cell lung cancer (NSCLC) and thyroid cancers
• Data from the registration-enabling NAVIGATOR trial of avapritinib in patients with PDGFRA Exon 18 mutant and fourth-line gastrointestinal stromal tumors (GIST), which will be used for planned marketing applications in the United States and Europe
• Data from the registration-enabling EXPLORER trial of avapritinib in patients with advanced systemic mastocytosis (SM)

"Our upcoming presentations highlight data supporting our plans for multiple marketing applications in the United States and Europe over the next 18 months and demonstrate continued progress on our '2020 Blueprint' strategy," said Andy Boral, M.D., Ph.D., Chief Medical Officer of Blueprint Medicines. "The datasets reflect the rapid development of avapritinib and BLU-667, including in four indications that have received Breakthrough Therapy Designation from the FDA. These presentations for avapritinib and BLU-667 highlight the breadth and speed of development of our portfolio, as well as our commitment to rapidly deliver new precision therapies to cancer patients with limited or no effective treatment options."

The accepted abstracts are listed below and are now available online on the ASCO and EHA conference websites, respectively: https://am.asco.org/ and https://ehaweb.org/congress/eha24/key-information/.

2019 American Society of Clinical Oncology (ASCO) Annual Meeting
May 31-June 4, 2019; Chicago, Illinois

Oral Presentation

Presentation Title: Clinical activity and tolerability of BLU-667, a highly potent and selective RET inhibitor, in patients (pts) with advanced RET-fusion+ non-small cell lung cancer (NSCLC)
Session Title: Lung Cancer—Non-Small Cell Metastatic
Session Date & Time: Monday, June 3, 2019 from 8:00 a.m. – 11:00 a.m. CT (9:00 a.m. – 12:00 p.m. ET)
Abstract Number: 9008

Poster Discussion Presentations

Presentation Title: Activity and tolerability of BLU-667, a highly potent and selective RET inhibitor, in patients with advanced RET-altered thyroid cancers
Session Title: Head and Neck Cancer
Poster Session Date & Time: Saturday, June 1, 2019 from 1:15 p.m. – 4:15 p.m. CT (2:15 p.m. – 5:15 p.m. ET)
Poster Discussion Session Date & Time: Saturday, June 1, 2019 from 4:30 – 6:00 p.m. CT (5:30 p.m. – 7:00 p.m. ET)
Abstract Number: 6018

Presentation Title: Clinical activity of avapritinib in ≥ fourth line (4L+) and PDGFRA Exon 18 gastrointestinal stromal tumors (GIST)
Session Title: Sarcoma
Poster Session Date & Time: Saturday, June 1, 2019 from 8:00 a.m. – 11:00 a.m. CT (9:00 a.m. – 12:00 p.m. ET)
Poster Discussion Session Date & Time: Saturday, June 1, 2019 from 3:00 p.m. – 4:30 p.m. CT (4:00 p.m. – 5:30 p.m. ET)
Abstract Number: 11022

24^th Congress of the European Hematology Association (EHA)
June 13-16, 2019; Amsterdam, The Netherlands

Oral Presentation

Presentation Title: Avapritinib, a potent and selective inhibitor of KIT D816V, induces complete and durable responses in patients (pts) with advanced systemic mastocytosis (AdvSM)
Session Title: New Agents in MPN
Presentation Date & Time: Saturday, June 15, 2019 from 12:00 p.m. – 12:15 p.m. CEST (6:00 a.m. – 6:15 a.m. ET)
Abstract Number: S830

Investor Event and Webcast Information

Blueprint Medicines will host an investor event on Monday, June 3, 2019 beginning at 6:30 p.m. CT (7:30 p.m. ET) in Chicago to provide a portfolio update, including a review of updated clinical data from the ongoing ARROW trial of BLU-667 in patients with RET-altered cancers and the ongoing registration-enabling NAVIGATOR trial in patients with PDGFRA Exon 18 mutant and fourth-line GIST. The event will be webcast live and can be accessed under the "Investors & Media—Events & Presentations" section of Blueprint Medicines' website at www.BlueprintMedicines.com. A replay of the webcast will be available approximately two hours after the event and will be available for 30 days following the event.

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 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 plans and timelines for submitting marketing applications for avapritinib and BLU-667 in the United States and Europe, including expectations related to the data for such submissions; and Blueprint Medicines' strategy, goals and anticipated milestones, 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-667, BLU-554 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 and gain approval of its drug candidates on a timely basis, if at all; 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; and 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 period ended March 31, 2019, as filed with the Securities and Exchange Commission (SEC) on May 9, 2019, 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.

SOURCE Blueprint Medicines Corporation

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