Blueprint Medicines To Present Broad Range of Data Reinforcing Commitment to Advance Patient Care in Systemic Mastocytosis at 62nd ASH Annual Meeting and Exposition

CAMBRIDGE, Mass., Nov. 23, 2020 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced plans to report data presentations highlighting the medical needs in systemic mastocytosis (SM) and the broad potential of AYVAKIT™ (avapritinib) across multiple SM patient populations at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition. AYVAKIT is designed to potently and selectively target D816V mutant KIT, the primary driver of SM, and is being developed for the treatment of advanced and non-advanced forms of the disease.

"The breadth of our upcoming ASH presentations highlights our commitment to fundamentally change the way systemic mastocytosis is diagnosed and treated across a broad population of patients impacted by this disease," said Andy Boral, M.D., Ph.D., Chief Medical Officer at Blueprint Medicines. "These presentations reflect our collaborative efforts with the systemic mastocytosis community to better understand the burden of disease, advance important strategies for facilitating diagnosis and establish new methods for measuring treatment response."

The accepted abstracts are listed below and available on the ASH conference website: https://www.hematology.org/meetings/annual-meeting.

Oral Presentation

Presentation Title: Pure Pathologic Response Is Associated with Improved Overall Survival in Patients with Advanced Systemic Mastocytosis Receiving Avapritinib in the Phase I EXPLORER Study
Session Title: Myeloproliferative Syndromes: Clinical: Translational Science in MPN—Hitting the Mark
Session Date & Time: Sunday, December 6, 2020 at 10:15 a.m. PT (1:15 p.m. ET)
Abstract Number: 345

Poster Presentations

Presentation Title: Patient Reported Outcomes Among Systemic Mastocytosis (SM) Patients in Routine Clinical Practice: Results from the TouchStone Survey
Session Title: Outcomes Research—Non-Malignant Conditions: Poster I
Session Date & Time: Saturday, December 5, 2020 from 7:00 a.m. - 3:30 p.m. PT (10:00 a.m. - 6:30 p.m. ET)
Abstract Number: 1638

Presentation Title: Results from PIONEER: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of Avapritinib in Patients with Indolent Systemic Mastocytosis
Session Title: Myeloproliferative Syndromes: Clinical: Poster I
Session Date & Time: Saturday, December 5, 2020 from 7:00 a.m. - 3:30 p.m. PT (10:00 a.m. - 6:30 p.m. ET)
Abstract Number: 1248

Presentation Title: Increased Detection of KIT D816V Mutation in Peripheral Blood Samples from Patients with Indolent Systemic Mastocytosis (ISM) in the Phase 2 PIONEER Study Using a High Sensitivity Droplet Digital (dd) PCR Assay Compared with Next Generation Sequencing (NGS)
Session Title: Hodgkin Lymphoma and T/NK Cell Lymphoma—Clinical Studies: Poster III
Session Date & Time: Monday, December 7, 2020 from 7:00 a.m. - 3:30 p.m. PT (10:00 a.m. - 6:30 p.m. ET)
Abstract Number: 3004

Presentation Title: Perceptions of Patient Disease Burden and Management Approaches of Systemic Mastocytosis By Healthcare Providers: Results from the TouchStone SM Survey
Session Title: Health Services Research—Non-Malignant Conditions: Poster III
Session Date & Time: Monday, December 7, 2020 from 7:00 a.m. - 3:30 p.m. PT(10:00 a.m. - 6:30 p.m. ET)
Abstract Number: 3403

Presentation Title: PIONEER Part 2: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib in Indolent Systemic Mastocytosis
Session Title: Hodgkin Lymphoma and T/NK Cell Lymphoma—Clinical Studies: Poster III
Session Date & Time: Monday, December 7, 2020 from 7:00 a.m. - 3:30 p.m. PT (10:00 a.m. - 6:30 p.m. ET)
Abstract Number: 2988

About AYVAKIT (avapritinib)

AYVAKIT (avapritinib) is a kinase inhibitor approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. For more information, visit www.AYVAKIT.com. This medicine is approved in Europe under the brand name AYVAKYT® for the treatment of adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation.

AYVAKIT is not approved for the treatment of any other indication, including SM, in the U.S. by the FDA or in Europe by the European Commission, or for any indication in any other jurisdiction by any other health authority.

Blueprint Medicines is developing AYVAKIT globally for the treatment of advanced and indolent SM. The FDA granted breakthrough therapy designation to AYVAKIT for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia.

Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of AYVAKIT in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for AYVAKIT in the rest of the world.

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 have two approved precision therapies and are currently advancing multiple investigational medicines in clinical and pre-clinical development, along with a number of earlier-stage 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, timelines and expectations for interactions with the FDA and other regulatory authorities; expectations regarding the potential benefits of AYVAKIT in treating patients with SM; and Blueprint Medicines' strategy, goals and anticipated milestones, business plans and focus. The words "aim," "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 impact of the COVID-19 pandemic to Blueprint Medicines' business, operations, strategy, goals and anticipated milestones, including Blueprint Medicines' ongoing and planned research and discovery activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Blueprint Medicines' ability and plans in establishing a commercial infrastructure, and successfully launching, marketing and selling current or future approved products, including AYVAKIT and GAVRETO™ (pralsetinib); Blueprint Medicines' ability to successfully expand the approved indications for AYVAKIT and GAVRETO or obtain marketing approval for AYVAKIT and GAVRETO in additional geographies in the future; the delay of any current or planned clinical trials or the development of Blueprint Medicines' current or future drug candidates; 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, partnerships or licensing arrangements. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in Blueprint Medicines' filings with the Securities and Exchange Commission (SEC), including Blueprint Medicines' most recent Annual Report on Form 10-K, as supplemented by its most recent Quarterly Report on Form 10-Q 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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Blueprint Medicines, AYVAKIT, AYVAKYT, GAVRETO and associated logos are trademarks of Blueprint Medicines Corporation.

SOURCE Blueprint Medicines Corporation

http://www.blueprintmedicines.com

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