Blueprint Medicines Announces New Data from Ongoing Phase 1 Clinical study of Avapritinib (BLU-285) in Patients with Advanced Systemic Mastocytosis Showing Evidence Strong Clinical Activity

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Blueprint Medicines Corporation (BPMC), an innovator in discovering and developing targeted kinase medicines for patients with genomically defined diseases, today announced new data from its ongoing Phase 1 medical study of avapritinib (formerly called BLU-285), a powerfulThe brand new data in the dose escalation part of the Phase 1 trial showed strong clinical activity no matter advanced SM subtype, prior treatment with midostaurin or presence of additional mutations.
Since the info cutoff date of October 4, 2017, the info showed a general response rate (ORR) of 72 percent and a disease control rate (DCR) of 100 percent in patients evaluable for response, in line with the International Working Group-Myeloproliferative Neoplasms Research and Treatment and European Competence Network on Mastocytosis (IWG-MRT-ECNM) consensus criteria. Since the info cutoff date, avapritinib was well-tolerated and a lot adverse events (AEs) reported by investigators were Grade A few. Furthermore, there are no discontinuations on account of treatment-related adverse events, and 30 of 32 patients remained on treatment which has a median treatment use of nine months. The data will be presented today within an oral presentation through the Plenary Scientific Session at the 59th American Society of Hematology Annual Meeting and Exposition (ASH) in Atlanta, Georgia.

Based on these data, Blueprint Medicines promises to engage global regulatory authorities inside the first half of 2018 to acquire input on registration pathways for avapritinib in patients with advanced SM and patients with indolent and smoldering SM. Susceptible to regulatory feedback, the corporation anticipates initiating a registration-enabling medical trial of avapritinib in patients with advanced SM within the first half of 2018 along with a dose escalation and proof-of-concept medical study of avapritinib in patients with indolent and smoldering SM inside the second half of 2018. Moreover, Blueprint Medicines is constantly enroll patients inside the expansion element of the ongoing Phase 1 medical study in patients with advanced SM with all the purpose of generating additional data in 2018.

“The new medical study results for avapritinib reported on the ASH plenary scientific session represent a thrilling milestone for the systemic mastocytosis community. The info showed an incredibly high response rate plus a favorable tolerability profile, suggesting avapritinib has the potential to transform treating this devastating rare disease,” said Daniel J. DeAngelo, M.D., Ph.D., Director of Clinical and Translational Research, Adult Leukemia at Dana-Farber Cancer Institute, Associate Professor of medication at Harvard School of medicine, and an investigator for the Phase 1 trial. “The data also provide strong evidence that selective inhibition of D816V mutant KIT, a disease-driver present in nearly all patients through the spectrum of SM, can be a potentially important treatment strategy that could offer patients improved outcomes. These data strongly support continued growth and development of avapritinib in a broad population of patients with SM.”

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