Blueprint Medicines Showcases Broad Portfolio of Highly Selective Investigational Kinase Medicines with Presentations at Upcoming Medical Meetings

CAMBRIDGE, Mass., Sept. 24, 2018 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ:BPMC), a leader in discovering and developing targeted kinase medicines for patients with genomically defined diseases, today announced the presentation of new clinical and preclinical data for its investigational therapeutic candidates avapritinib, BLU-667 and BLU-782.

"Data presentations for avapritinib, BLU-667 and BLU-782 at important upcoming medical and scientific meetings reflect the power of our scientific platform to rapidly and reproducibly advance highly selective kinase medicines against diverse, difficult-to-drug targets," said Andy Boral, M.D., Ph.D., Chief Medical Officer of Blueprint Medicines. "The data we are presenting highlight the depth and breadth of our growing portfolio and represent continued progress across multiple clinical and preclinical programs."

Avapritinib: Gastrointestinal Stromal Tumors (GIST)

2018 European Society for Medical Oncology (ESMO) Congress
October 19-23, 2018; Munich, Germany

Poster Presentation Title: Correlation of ctDNA and response in patients with PDGFRα D842 GIST treated with avapritinib
Session Title: Breast cancer - early stage, locally advanced & metastatic, CNS tumours, Developmental therapeutics, Genitourinary tumours - prostate & non-prostate, Palliative care, Psycho-oncology, Public health policy, Sarcoma, Supportive care 
Session Date & Time: Monday, October 22, 2018 from 12:45 p.m. – 1:45 p.m. GMT (6:45 a.m. – 7:45 a.m. ET)
Presenter: Suzanne George, MD
Abstract Number: 5100

Trial in Progress poster: VOYAGER: An open-label, randomized, Phase 3 study of avapritinib vs. regorafenib in patients with locally advanced metastatic or unresectable gastrointestinal stromal tumor (GIST)
Session Title: Breast cancer - early stage, locally advanced & metastatic, CNS tumours, Developmental therapeutics, Genitourinary tumours - prostate & non-prostate, Palliative care, Psycho-oncology, Public health policy, Sarcoma, Supportive care 
Session Date & Time: Monday, October 22, 2018 from 12:45 p.m. – 1:45 p.m. GMT (6:45 a.m. – 7:45 a.m. ET)
Presenter: Sebastian Bauer, MD
Abstract Number: 4534

2018 Connective Tissue Oncology Society (CTOS) Annual Meeting
November 14-17, 2018; Rome, Italy

Oral Presentation Title: Avapritinib is Highly Active and Well-Tolerated in Patients with Advanced GIST Driven by Diverse Variety of Oncogenic Mutations in KIT and PDGFRA
Session Title: Session 3 – GIST
Session Date & Time: Thursday, November 15, 2018 from 4:00 p.m. to 6:00 p.m. GMT (10:00 a.m. to 12:00 p.m. ET)
Presenter: Michael Heinrich, MD
Abstract Number: 3027631

BLU-667: RET-Altered Solid Tumors

IASLC 19th World Conference on Lung Cancer
September 23-26, 2018; Toronto, Canada

Late-Breaking Oral Presentation Title: Activity of Osimertinib and the Selective RET Inhibitor BLU-667 in an EGFR-Mutant Patient with Acquired RET Rearrangement
Session Title: New Therapies and Emerging Data in ALK, EGFR and ROS1
Session Date & Time: Wednesday, September 26, 2018 at 1:30 to 3:00 p.m. ET
Presenter: Zosia Piotrowska, MD
Abstract Number: MA26.03

88^th Annual Meeting of the American Thyroid Association (ATA)
October 3-7, 2018; Washington D.C.

Oral Presentation Title: Clinical Activity of Selective RET Inhibitor, BLU-667, in Advanced RET-Altered Thyroid Cancers; Updated Results from the Phase 1 ARROW Study
Session Title: Translational Short Call Oral Abstracts
Session Date & Time: Saturday, October 6, 2018 from 8:50 a.m. – 9:50 a.m. ET
Presenter: Mimi I. Hu, MD
Abstract Number: 5

BLU-782: Fibrodysplasia Ossificans Progressiva

2018 American Society for Bone and Mineral Research (ASBMR) Annual Meeting
September 28 – October 1, 2018; Montreal, Quebec, Canada

Oral Presentation Title: BLU-782; a highly selective ALK2 inhibitor, designed specifically to target the cause of fibrodysplasia ossificans progressiva
Session Title: Plenary Oral 2 – Translational Highlights II
Session Date & Time:  Sunday, September 30, 2018 from 9:45 a.m. – 11:00 a.m. ET
Presenter: Andrew Garner, PhD
Presentation Number: 1078

About Blueprint Medicines

Blueprint Medicines is developing a new generation of targeted and potent kinase medicines to improve the lives of patients with genomically defined diseases. Its 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 multiple programs in clinical development for subsets of patients with gastrointestinal stromal tumors, hepatocellular carcinoma, systemic mastocytosis, non-small cell lung cancer, medullary thyroid cancer and other advanced solid tumors, as well as multiple programs in research and preclinical development. For more information, please visit www.blueprintmedicines.com.

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

Blueprint Medicines' ability to rapidly and reproducibly advance medicines. 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 BLU-554 for FGFR4-driven hepatocellular carcinoma, avapritinib for PDGFRα D842V-driven GIST and advanced systemic mastocytosis and BLU-667 for RET-driven non-small cell lung cancer; 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 June 30, 2018, as filed with the Securities and Exchange Commission (SEC) on August 1, 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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