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Blueprint Medicines is a fully integrated global biopharmaceutical company that invents life-changing medicines across two strategic focus areas of allergy/inflammation and oncology/hematology. Our approach combines deep scientific expertise, modality-agnostic drug discovery capabilities, and a proven track record of R&D success. Currently, we are commercializing our approved medicine AYVAKIT/AYVAKYT® (avapritinib) in the U.S. and Europe, as well as in other regions of the world through partnerships.

News release details

Blueprint Medicines Announces Data Presentations Highlighting Significant Progress in Advancing RET-Altered Cancer and Systemic Mastocytosis Programs

May 14, 2020 at 9:00 AM EDT

CAMBRIDGE, Mass., May 14, 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 present updated clinical data for pralsetinib in RET-altered cancers and avapritinib in systemic mastocytosis.

"The upcoming presentations reflect our commitment to bring transformative therapies to patients by selectively targeting genomic drivers of disease," said Andy Boral, M.D., Ph.D., Chief Medical Officer of Blueprint Medicines. "The data support the rapid clinical advancement of pralsetinib and avapritinib, with multiple global regulatory submissions under review or planned in 2020. These abstracts reinforce the consistent clinical activity shown by our drug candidates across broad patient populations with RET-altered cancers and systemic mastocytosis."

Updated clinical data will be presented in the American Society of Clinical Oncology 2020 (ASCO20) Virtual Scientific Program, at the European Academy of Allergy and Clinical Immunology (EAACI) Digital Congress and at the Virtual Edition of the 25th European Hematology Association (EHA25) Annual Congress. Accepted abstracts are listed below. Abstracts are expected to be available on the EAACI conference website ( at the start of the congress, and are now available on the following conference websites: and The following presentations will be available to registered participants of each virtual meeting at its conference website.

ASCO20 Virtual Scientific Program
May 29-31, 2020

Oral Presentation

Presentation Title: Clinical activity of the RET inhibitor pralsetinib (BLU-667) in patients with RET fusion+ solid tumors
Session Title: Drug Development for Rare Mutations: The Opportunity to Unite and Conquer
Session Date & Time (Scheduled Broadcast): Sunday, May 31, 2020 from 10:30 a.m.12:00 p.m. ET
Abstract Number: 109

Poster Discussion Presentation

Presentation Title: Registrational dataset from the Phase I/II ARROW trial of pralsetinib (BLU-667) in patients (pts) with advanced RET fusion+ non-small cell lung cancer (NSCLC)
Presentation Date & Time (On-Demand): Friday, May 29, 2020 beginning at 8:00 a.m. ET
Session Title: Lung Cancer—Non-Small Cell Metastatic
Abstract Number: 9515

Poster Presentation

Presentation Title: AcceleRET Lung: A Phase III study of first-line pralsetinib in patients (pts) with RET-fusion+ advanced/metastatic non-small cell lung cancer (NSCLC)
Presentation Date & Time (On-Demand): Friday, May 29, 2020 beginning at 8:00 a.m. ET
Session Title: Lung Cancer—Non-Small Cell Metastatic
Abstract Number: TPS9633

EAACI Digital Congress
June 6-8, 2020

Late Breaking Oral Presentation

Presentation Title: Avapritinib reduced cutaneous symptoms and mast cell (MC) burden in patients (pts) with indolent systemic mastocytosis (ISM) in the PIONEER study
Presentation Date & Time (On-Demand): Saturday, June 6, 2020 beginning at 9:00 a.m. CEST (3:00 a.m. ET)
Session Title: Skin Diseases: What Is New?
Abstract Number: 1832

EHA25 Virtual Congress
June 11-14, 2020

Poster Presentations

Presentation Title: Avapritinib induces responses in patients (pts) with advanced systemic mastocytosis (AdvSM), regardless of prior midostaurin therapy 
Presentation Date & Time (On-Demand): Friday, June 12, 2020 beginning at 8:30 a.m. CEST (2:30 a.m. ET)
Session Title: Myeloproliferative Neoplasms - Clinical
Abstract Number: EP1079

Presentation Title: Results from PIONEER: a randomized, double-blind, placebo-controlled, Phase 2 study of avapritinib in patients with indolent systemic mastocytosis
Presentation Date & Time (On-Demand): Friday, June 12, 2020 beginning at 8:30 a.m. CEST (2:30 a.m. ET)
Session Title: Myeloproliferative Neoplasms - Clinical
Abstract Number: EP1082

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 one FDA-approved precision therapy and are currently advancing multiple investigational medicines in clinical development, along with a number of research programs. For more information, visit 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, plans and timelines for submitting marketing applications for avapritinib and pralsetinib and, if approved, commercializing avapritinib for additional indications or pralsetinib; the potential benefits of Blueprint Medicines' current and future approved drugs or drug candidates in treating patients; 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 the launch, marketing and sale of current or future approved products; the delay of any current or planned clinical trials or the development of Blueprint Medicines' drug candidates or licensed product candidate; 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 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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