— HARBOR Part 1 trial data in indolent systemic mastocytosis showed elenestinib was well-tolerated with broad symptom improvement, supporting further development to expand and extend company’s SM franchise leadership —
CAMBRIDGE, Mass., Dec. 9, 2023 /PRNewswire/ — Blueprint Medicines Corporation (NASDAQ:BPMC) today announced data showcasing its commitment to advance the scientific understanding and treatment of systemic mastocytosis (SM) at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition being held December 9-12 in San Diego. Data that will be presented include results from the HARBOR Part 1 trial of elenestinib in indolent systemic mastocytosis (ISM) and analyses of real-world data highlighting the burden of and urgency to treat ISM.
“Blueprint Medicines has transformed the standard of care for advanced and indolent systemic mastocytosis with AYVAKIT® (avapritinib), and its proven and compelling clinical profile is redefining what well-controlled disease means for patients living with SM,” said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. “Building on the success of AYVAKIT and the clinical expertise amassed during its development, we are strategically advancing our investigational next-generation KIT D816V inhibitor, elenestinib, to expand and extend Blueprint Medicines’ SM franchise leadership over the long term.”
HARBOR Part 1 trial data in patients with ISM showed elenestinib was well-tolerated and clinically active at all dose levels tested, supporting further development. In patients treated with elenestinib, most adverse events (AEs) were Grade 1 or 2, and there were no discontinuations due to AEs. Elenestinib showed clinically meaningful symptom improvements as assessed by the validated Indolent Systemic Mastocytosis Symptom Assessment Form Total Symptom Score (ISM-SAF TSS), and rapid and profound reductions across multiple measures of mast cell burden.
At ASH, new data on the burden of disease highlight the urgency to treat patients with ISM. A real-world analysis of U.S. health claims data showed patients with ISM had lower survival compared to a matched population cohort (p<0.0001), and a model-based analysis assessed that the lifetime risk of progression from ISM to advanced SM was approximately 20 percent. In addition, a data presentation reports on a diagnostic tool to aid in the identification of patients with SM, which was developed based on a real-world analysis at The Quality Cancer Care Alliance (QCCA).
In total, Blueprint Medicines’ presence at ASH builds on over a decade of innovative research in the field of SM, and reflects the company’s ongoing leadership in transforming care for patients living with the disease.
ASH abstracts are listed below:
Oral Presentations
Presentation Title: Decreased Survival Among Patients with Indolent Systemic Mastocytosis: A Population-Level Retrospective Cohort Analysis Using Healthcare Claims Dataset
Session Title: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Rare Myeloproliferative Neoplasms: Unveiling Promising Pathways and Novel Therapies
Session Date & Time: Today, December 9 from 9:30 – 11:00 a.m. PT (12:30 – 2:00 p.m. ET)
Presentation Date & Time: Today, December 9 at 10:00 a.m. PT (1:00 p.m. ET)
Abstract Number: 75
Location: San Diego Convention Center, Ballroom 20AB
Presentation Title: Elenestinib, an Investigational, Next Generation KIT D816V Inhibitor, Reduces Mast Cell Burden, Improves Symptoms, and Has a Favorable Safety Profile in Patients with Indolent Systemic Mastocytosis: Analysis of the HARBOR Trial
Session Title: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Rare Myeloproliferative Neoplasms: Unveiling Promising Pathways and Novel Therapies
Session Date & Time: Today, December 9 from 9:30 – 11:00 a.m. PT (12:30 – 2:00 p.m. ET)
Presentation Date & Time: Today, December 9 at 10:15 a.m. PT (1:15 p.m. ET)
Abstract Number: 76
Location: San Diego Convention Center, Ballroom 20AB
Poster Presentation
Presentation Title: Development and Validation of a Diagnostic Tool for the Timely Diagnosis of Patients with Systemic Mastocytosis
Session Title: 906. Outcomes Research—Myeloid Malignancies: Poster II
Session Date & Time: Sunday, December 10 from 6:00 – 8:00 p.m. PT (9:00 – 11:00 p.m. ET)
Abstract Number: 3800
Location: San Diego Convention Center, Halls G-H
Publication-Only Abstract
Title: A Model of Cumulative Risk of Disease Progression Among Patients with Indolent Systemic Mastocytosis
Abstract Number: 6406
Copies of Blueprint Medicines data presentations from the ASH annual meeting will be available in the “Science—Publications and Presentations” section of the company’s website at www.BlueprintMedicines.com.
About AYVAKIT (avapritinib)
AYVAKIT (avapritinib) is a precision therapy approved by the U.S. Food and Drug Administration (FDA) for the treatment of three indications: adults with ISM, adults with advanced SM, including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL), and adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. For more information, visit AYVAKIT.com. This medicine is approved by the European Commission (AYVAKYT®) for the treatment of adults with ASM, SM-AHN or MCL, after at least one systemic therapy, and adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation. Please click here to see the full U.S. Prescribing Information for AYVAKIT, and click here to see the European Summary of Product Characteristics for AYVAKYT.
In November 2023, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion recommending the approval of AYVAKYT for the treatment of adult patients with ISM with moderate to severe symptoms inadequately controlled on symptomatic treatment.
To learn about ongoing or planned clinical trials, contact Blueprint Medicines at medinfo@blueprintmedicines.com or 1-888-BLU-PRNT (1-888-258-7768). Additional information is available at blueprintclinicaltrials.com or clinicaltrials.gov.
