Blueprint Medicines to Present New Data Reinforcing Clinical Leadership in Systemic Mastocytosis at 2022 ASH Annual Meeting

CAMBRIDGE, Mass., Nov. 3, 2022 /PRNewswire/ -- Blueprint Medicines Corporation BPMC today announced the acceptance of abstracts highlighting long-term clinical outcomes and impact on overall survival of AYVAKIT® (avapritinib) in patients with advanced systemic mastocytosis (SM), as well as real-world evidence characterizing the burden of SM, for presentation at the 2022 American Society of Hematology (ASH) Annual Meeting in December. These abstracts were submitted by the regular abstract deadline and posted online by ASH today.

Key highlights include an oral presentation on the PATHFINDER trial, which reinforces the transformative impact of AYVAKIT as a first-line treatment in advanced SM, and a poster presentation on the EXPLORER trial, which highlights survival benefits after nearly four years of patient follow-up in advanced SM. Additional data include U.S. claims-based analyses describing patterns of disease progression and the accumulation of severe symptoms over time in patients with non-advanced SM.

In addition, Blueprint Medicines has submitted a late-breaking abstract on registrational data from the PIONEER trial of AYVAKIT in patients with non-advanced SM for presentation at the ASH Annual Meeting. If accepted, the late-breaking abstract will be posted online by ASH on November 22, 2022.

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

Presentation Title: Avapritinib as First-Line Therapy in Patients with Advanced Systemic Mastocytosis: Efficacy and Safety from the PATHFINDER Clinical Study
Session Title: Myeloproliferative Syndromes: Clinical and Epidemiological: Towards Personalized Medicine in Myeloproliferative Neoplasms and Mastocytosis: New and Repurposed Drugs for Unmet Clinical Needs
Session Date & Time: Sunday, December 11, 2022 from 4:30 p.m. – 6:00 p.m. ET
Abstract Number: 625
Location: Ernest N. Morial Convention Center, Room 217-219

Presentation Title: An Updated Analysis on Safety and Efficacy of Avapritinib in Patients with Advanced Systemic Mastocytosis from the EXPLORER Clinical Study: Long-term Efficacy and Safety
Session Title: Myeloproliferative Syndromes: Clinical and Epidemiological: Poster I
Session Date & Time: Saturday, December 10, 2022 from 5:30 p.m. – 7:30 p.m. ET
Abstract Number: 1710
Location: Ernest N. Morial Convention Center, Hall D

Presentation Title: NGS Testing Practices and Molecular Profile Landscape of the KIT Gene in Systemic Mastocytosis: Real-World Insights from Selected European Countries
Session Title: Granulocytes, Monocytes, and Macrophages: Poster I
Session Date & Time: Saturday, December 10, 2022 from 5:30 p.m. – 7:30 p.m. ET
Abstract Number: 1090
Location: Ernest N. Morial Convention Center, Hall D

Presentation Title: AZURE: A Phase 1/2 Study of BLU-263 as Monotherapy and in Combination with Azacitidine in Patients with Advanced Systemic Mastocytosis
Session Title: Myeloproliferative Syndromes: Clinical and Epidemiological: Poster II
Session Date & Time: Sunday, December 11, 2022 from 6:00 p.m. – 8:00 p.m. ET
Abstract Number: 3058
Location: Ernest N. Morial Convention Center, Hall D

Presentation Title: Patterns of Disease Progression in Patients with Systemic Mastocytosis: A U.S. Population-Level Analysis Using Health Claims-Based Dataset
Session Title: Myeloproliferative Syndromes: Clinical and Epidemiological: Poster II
Session Date & Time: Sunday, December 11, 2022 from 6:00 p.m. – 8:00 p.m. ET
Abstract Number: 3053
Location: Ernest N. Morial Convention Center, Hall D

Blueprint Medicines is a global precision therapy company that invents life-changing therapies for people with cancer and blood disorders. Applying an approach that is both precise and agile, we create medicines that selectively target genetic drivers, with the goal of staying one step ahead across stages of disease. Since 2011, we have leveraged our research platform, including expertise in molecular targeting and world-class drug design capabilities, to rapidly and reproducibly translate science into a broad pipeline of precision therapies. Today, we are delivering approved medicines directly to patients in the United States and Europe, and we are globally advancing multiple programs for systemic mastocytosis, lung cancer and other genomically defined cancers, and cancer immunotherapy. For more information, visit www.BlueprintMedicines.com and follow us on Twitter (@BlueprintMeds) and LinkedIn.

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, strategies, timelines and expectations for Blueprint Medicines' current or future approved drugs and drug candidates, including the results of ongoing clinical trials; 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 continuing to establish and expand a commercial infrastructure, and successfully launching, marketing and selling current or future approved products; 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 timing and results of preclinical and clinical studies for Blueprint Medicines' drug candidates, which may not support further development of such drug candidates or may impact the timing of data publications or regulatory submissions; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials; Blueprint Medicines' ability to obtain, maintain and enforce patent and other intellectual property protection for any drug candidates it is developing; Blueprint Medicines' ability to develop and commercialize companion diagnostic tests for any of its current and future drug candidates; Blueprint Medicines' ability to successfully expand its operations and scientific platform and the costs thereof; the success of Blueprint Medicines' current and future collaborations, partnerships or licensing arrangements; and Blueprint Medicines' ability to realize the anticipated benefits of its succession planning and leadership development and transitions. 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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