2022-11-03 | NDAQ: BPMC | Press release

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CAMBRIDGE, Mass., November 3, 2022 /PRNewswire/ — Blueprint Medicines Corporation (NASDAQ: BPMC) today announced the acceptance of abstracts highlighting the long-term clinical results and impact on overall survival of AYVAKIT® (avapritinib) in patients with advanced systemic mastocytosis (SM), along with concrete evidence characterizing the burden of MS, for presentation at the 2022 American Society of Hematology (ASH) Annual Meeting in December. These abstracts were submitted before the usual deadline and posted 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 MS, 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 US claims-based analyzes describing patterns of disease progression and accumulation of severe symptoms over time in patients with non-advanced MS.

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

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

Oral presentation

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 and time: Sunday, December 11, 2022 of 4:30 p.m.6:00 PM ET

abstract number: 625

Location: Ernest N. Morial Convention Center, room 217-219

Poster presentations

Presentation title: Updated Analysis of the 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 and time: Saturday, December 10, 2022 of 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 the Landscape of KIT Gene Molecular Profiling in Systemic Mastocytosis: Real-World Insights from Selected European Countries

Session title: Granulocytes, Monocytes and Macrophages : Poster I

Session date and time: Saturday, December 10, 2022 of 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 and time: Sunday, December 11, 2022 of 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 US Population-Level Analysis Using a Health Claim-Based Dataset

Session title: Myeloproliferative syndromes: clinical and epidemiological: Poster II

Session date and time: Sunday, December 11, 2022 of 6:00 p.m.8:00 p.m. ET

abstract number: 3053

Location: Ernest N. Morial Convention Center, Hall D

About Blueprint Medications

Blueprint Medicines is a global precision therapy company inventing life-changing therapies for people with cancer and blood disorders. By applying a precise yet agile approach, we create drugs that selectively target genetic factors, with the goal of staying ahead through disease stages. Since 2011, we have leveraged our research platform, including our molecular targeting expertise and world-class drug design capabilities, to rapidly and reproducibly translate science into a wide range of precision therapies. Today, we provide approved medicines directly to patients in United States and Europe, and we are advancing several programs globally 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.

Caution 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 drug plans, strategies, timelines and expectations and current or future approved drug candidates from Blueprint Medicines. , including the results of ongoing clinical trials; expectations regarding the potential benefits of AYVAKIT in the treatment of patients with MS; and Blueprint Medicines’ strategy, goals and planned milestones, business plans and direction. The words “aim”, “may”, “will”, “could”, “should”, “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 words. ‘identification. All forward-looking statements contained in this press release are based on management’s current expectations and beliefs and are subject to a number of important risks, uncertainties and factors that may cause actual events or results to differ. differ materially from those expressed or implied by any forward-looking statement. statements contained in this press release, including, without limitation, the risks and uncertainties related to the impact of the COVID-19 pandemic on the business, operations, strategy, objectives and planned milestones of Blueprint Medicines, including Blueprint Medicines’ ongoing and planned research and discovery activities, ability to conduct ongoing and planned clinical trials, clinical delivery of current or future drug candidates, commercial delivery of current or future approved products, and launching, marketing and selling current or future approved products; Blueprint Medicines’ ability and plans to continue to establish and expand a commercial infrastructure, and to successfully launch, market and sell current or future approved products; delay in any ongoing or planned clinical trials or development of Blueprint Medicines’ current or future drug candidates; advancing Blueprint Medicines from multiple early-stage efforts; the ability of Blueprint Medicines to successfully demonstrate the safety and efficacy of its drug candidates and to obtain 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 releases 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 protections for any drug candidates it develops; 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 related costs; the success of Blueprint Medicines’ current and future collaborations, partnerships or licensing agreements; 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 more detail in the section titled “Risk Factors” in Blueprint Medicines’ filings with the Securities and Exchange Commission (SEC), including Blueprint Medicines’ most recent annual report. Blueprint Medicines on Form 10-K, as supplemented by its most recent Quarterly Report on Form 10-Q and any other filings Blueprint Medicines has made or may make with the SEC in the future. All forward-looking statements contained in this press release represent the views of Blueprint Medicines 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 expressly disclaims any obligation to update forward-looking statements.

Trademarks

Blueprint Medicines, AYVAKIT and associated logos are registered trademarks of Blueprint Medicines Corporation.

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