On November 23, 2020 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported 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) (Free ASH Whitepaper) Annual Meeting and Exposition (Press release, Blueprint Medicines, NOV 23, 2020, View Source [SID1234571589]). 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.

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"The breadth of our upcoming ASH (Free ASH Whitepaper) 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 (Free ASH Whitepaper) conference website: View Source

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.