Blueprint Medicines Corp (NASDAQ:BPMC) announced that it will present new preclinical data demonstrating the potential utility of BLU-285 in a subset of patients with acute myeloid leukemia (AML) characterized by a mutation in KIT Exon 17 during an oral presentation at the 2015American Society of Hematology (ASH) Annual Meeting and Exposition being heldDecember 5-8 in Orlando, Fla.

KIT Exon 17 mutations have been identified as drivers in multiple diseases, and the presence of these mutations greatly reduces the effective treatment options available to patients. BLU-285 is a potent and highly selective inhibitor of KIT Exon 17 and PDGFRα D842V mutants, which are drivers of disease in patients with gastrointestinal stromal tumors (GIST) and systemic mastocytosis (SM).

Blueprint Medicines is enrolling patients in a dose-escalation, Phase 1 clinical trial of BLU-285 for the treatment of unresectable, treatment-resistant GIST. Additionally, theU.S. Food and Drug Administration in September accepted Blueprint Medicines’Investigational New Drug application to begin a Phase 1 clinical trial of BLU-285 for the treatment of advanced SM, and Blueprint Medicines is in the process of initiating clinical sites for this trial.

In AML, a subset of patients with the t(8;21) translocation and an additional KIT Exon 17 mutation are at increased risk of recurrence compared to those without it, creating a strong need for new therapies to address their form of the disease.

In preclinical results to be presented by Blueprint Medicines at ASH, BLU-285 showed potent inhibition of KIT activity in an AML cell line with the N822K mutation in Exon 17, including reduced phosphorylation, downstream signaling and cellular proliferation. BLU-285 administration in a xenograft model harboring AML cells driven by the KIT Exon 17 mutation resulted in a reduction of disease burden compared to cytarabine-treated animals. All BLU-285-dosed mice showed tumor regression, and several showed complete disease clearance.

“We are encouraged by these preclinical results, which further strengthen our belief that BLU-285 may play an important role in multiple hematologic and solid tumor malignancies,” said Andy Boral, M.D., Ph.D., Senior Vice President of Clinical Development. “We continue to advance our clinical programs for BLU-285 while also exploring new therapeutic areas where targeting the KIT Exon 17 mutation may bring substantial clinical benefit.” (Original Source)

Shares of Blueprint Medicines closed yesterday at $21.21. BPMC has a 1-year high of $37.17 and a 1-year low of $18. The stock’s 50-day moving average is $21.81 and its 200-day moving average is $26.57.

Blueprint Medicines Corp is a biopharmaceutical company. It is focused on improving the lives of patients with genomically defined diseases driven by abnormal kinase activation.