Digital product blueprint group insiders2/26/2024 Three patients (5 percent) discontinued AYVAKIT due to treatment-related AEs, and most patients (84 percent) have remained on treatment as of the data cutoff date. The most common AEs (≥15 percent) were peripheral edema, periorbital edema, thrombocytopenia, anemia, neutropenia, diarrhea, nausea, vomiting and fatigue. Across multiple measures of mast cell burden, AYVAKIT showed profound reductions in serum tryptase, bone marrow mast cells, KIT D816V allele burden and spleen volume.Ĭonsistent with previously disclosed data, AYVAKIT was generally well-tolerated in 62 patients enrolled in the PATHFINDER trial, and most adverse events (AEs) were reported as Grade 1 or 2. Treatment with AYVAKIT resulted in robust improvements in patient-reported quality of life, based on the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire. In new patient-reported outcomes data, AYVAKIT showed a statistically significant reduction in total symptom score after 40 weeks (p<0.001), as measured by the Advanced Systemic Mastocytosis Symptom Assessment Form. These results show that responses deepened over time at a rate consistent with the EXPLORER trial.ĪYVAKIT led to robust and durable benefits across a number of additional clinical activity measures. ![]() The CRh rate was 19 percent, with a median time to CRh of 5.6 months. The median time to response was two months, and all responses were ongoing at a median follow-up of 10.4 months. Overall, 75 percent (95% CI: 57%, 89%) of patients had a confirmed response, which was defined as complete remission with full or partial recovery of peripheral blood counts (CR/CRh), partial remission or clinical improvement. Combined with Phase 1 EXPLORER trial results, these data support Blueprint Medicines' marketing applications in advanced SM under review in the U.S. In a pre-specified interim analysis from the PATHFINDER trial, 32 patients who primarily received a starting dose of 200 mg once daily were evaluable for response, as of a data cutoff date of June 23, 2020. We are currently on the precipice of our first potential approval in advanced SM, and we are committed to working closely with the SM community so we may meet the needs of patients as quickly as possible."ĪYVAKIT – Highlights from the Registrational Phase 2 PATHFINDER Trial For BLU-263, data in healthy volunteers showed a well-tolerated safety profile and support our plans to initiate the Phase 2/3 HARBOR study, which expands our development efforts into a broader population of patients with non-advanced SM. "In the PATHFINDER trial, AYVAKIT had high response rates consistent with EXPLORER trial data, reinforcing the profound clinical benefits that can be achieved by precisely targeting the underlying driver of disease. "Data reported at AACR reflect our commitment to transform treatment for patients living with systemic mastocytosis," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. Blueprint Medicines is developing AYVAKIT for advanced and non-advanced SM, and BLU-263 to further address the range of patient needs in non-advanced SM and other mast cell disorders. ![]() ![]() ![]() The presentations focus on registrational PATHFINDER trial data of AYVAKIT™ (avapritinib) in advanced SM, PIONEER Part 1 data highlighting the impact of AYVAKIT on skin manifestations of SM, and Phase 1 trial data for BLU-263, a next-generation KIT D816V inhibitor. CAMBRIDGE, Mass., Ap/PRNewswire/ - Blueprint Medicines Corporation (NASDAQ: BPMC) today announced that multiple presentations across the company's leading systemic mastocytosis (SM) program are being reported at the virtual American Association for Cancer Research (AACR) Annual Meeting 2021.
0 Comments
Leave a Reply.AuthorWrite something about yourself. No need to be fancy, just an overview. ArchivesCategories |