Blueprint Medicines Receives FDA Authorization to Advance Novel Drug Candidate into Clinical Trial for Systemic Mastocytosis

Blueprint Medicines

today announced that the U.S. Food and Drug Administration (FDA) accepted the Company's Investigational New Drug (IND) application to begin a Phase 1 clinical trial of BLU-285 in patients with advanced systemic mastocytosis (SM), a disorder of the mast cells. Blueprint Medicines "BLU-285 has the potential to be a highly targeted therapy for patients with severe forms of SM who lack effective treatment options," said Jeffrey Albers, Chief Executive Officer of Blueprint Medicines. "With the achievement of this milestone, we are simultaneously advancing multiple drug candidates into clinical trials for genomically defined subsets of patients in dire need of new therapies. We remain singularly focused on using our deep understanding of the genetic blueprint of cancer and other diseases driven by the abnormal activation of kinases to craft highly selective medicines aimed at improving patients' lives." BLU-285 is a potent and highly selective inhibitor of the KIT D816V mutant, the primary driver of disease in more than 94 percent of SM patients. SM is characterized by the abnormal accumulation of mast cells. In patients with advanced forms of the disease, mast cells accumulate in organs, such as the bone marrow, intestines, spleen and liver, compromising organ function. There are no approved targeted therapies for SM patients with KIT D816V-driven disease. In this Phase 1 trial, Blueprint Medicines plans to enroll approximately 60 patients with advanced SM, which includes aggressive SM with associated hematological non-mast cell disorders (SM-AHNMD) and mast cell leukemia (MCL), and other relapsed or refractory myeloid malignancies at multiple sites in Europe and the United States. The trial will test the safety and tolerability of escalating doses of BLU-285, with the goal of establishing a maximum tolerated dose (MTD), or a recommended dose if the MTD is not achieved. Additional study objectives include assessing early signs of biological activity using disease-specific biomarkers and clinical efficacy as measured by response rate and patient-reported outcomes. In July, Blueprint Medicines also announced the acceptance of INDs to advance BLU-285 into a Phase 1 trial for unresectable, treatment-resistant gastrointestinal stromal tumor (GIST) and BLU-554 into a Phase 1 trial for advanced hepatocellular carcinoma (HCC).