Benzinga | Dec 6, 2021 04:06PM EST

Nature Medicine Publishes Results From Two Registration Studies Of AYVAKIT Showing Sustained Benefits In Patients With Advanced Systemic Mastocytosis

Blueprint Medicines Corporation (NASDAQ:BPMC) today announced two Nature Medicine publications on the registration-enabling EXPLORER and PATHFINDER trials of AYVAKIT(r) (avapritinib), highlighting its robust efficacy and safety datasets in advanced systemic mastocytosis (Advanced SM). The publications feature overall response, patient-reported outcomes and survival data, which reinforce the durable activity of AYVAKIT. Additional new analyses underscore the treatment's impact regardless of patient subgroup or disease pathology. Based on these data, AYVAKIT was approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with advanced SM in June 2021.

SM is a rare hematologic disorder driven by the KIT D816V mutation in nearly all cases. In advanced SM, the median overall survival with previously available treatment options ranges from less than six months to approximately 3.5 years, depending on the subtype.1

"Targeting the primary genetic driver of systemic mastocytosis, avapritinib has shown rapid and durable improvements in mast cell burden, patient-reported symptoms and quality of life, with a generally well-tolerated safety profile," said Daniel DeAngelo, M.D., Ph.D., Chief of the Division of Leukemia at Dana-Farber Cancer Institute, lead and senior author on the EXPLORER and PATHFINDER trial publications, respectively. "I am highly encouraged by the survival benefits observed with this therapy, including in patients with mast cell leukemia who have a particularly poor prognosis. The depth of activity achieved by avapritinib, including molecular remission of KIT D816V, sets a new benchmark for the treatment of advanced systemic mastocytosis."

"The data published today in Nature Medicine comprise the largest therapeutic dataset ever reported in advanced systemic mastocytosis, encompassing two studies enrolling approximately 150 patients with up to four years of follow-up, and reflecting our deep commitment to pioneer new science and improve outcomes for patients living with this devastating disease," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines.

In addition, Blueprint Medicines announced plans to report four SM data presentations, including an oral presentation on the EXPLORER trial, at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta on December 11-14, 2021. These datasets further illustrate the significant impact of AYVAKIT in advanced SM, showcase Blueprint Medicines' clinical leadership in SM and reflect the company's ongoing collaboration with the community to improve patient care.