Blueprint Medicines Shares Fall After Topline Results From Late-Stage Neoplastic Disorder Study

Blueprint Medicines BPMC announced positive top-line results from the Part 2 of PIONEER clinical trial of AYVAKIT (avapritinib) in patients with non-advanced systemic mastocytosis (SM).

The Part 2 of the PIONEER trial was designed to evaluate the efficacy and safety of AYVAKIT (25 mg once-daily dosing; N=141) versus control (N=71) over 24 weeks of treatment.

The trial demonstrated clinically meaningful and highly significant improvements across the primary and all key secondary endpoints, including patient-reported symptoms and objective measures of mast cell burden.

Becker Hewes, Chief Medical Officer, commented: "The PIONEER results showcase Blueprint Medicines' dedication to advancing the promise of precision therapy for patients with significant medical needs. AYVAKIT has the potential to be the first approved medicine for non-advanced SM, and the only treatment that would address the genetic root cause across advanced and non-advanced forms of the disease.”

With these top-line data in hand, the company plans to submit a supplemental new drug application (sNDA) to the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2022.

The company is also planning for a submission of a type II variation marketing authorization application to the European Medicines Agency (EMA) anticipated in 2023.

The detailed data readout from the PIONEER trial is expected to be presented at an upcoming medical meeting.

Price Action : Blueprint Medicines shares are trading around 22 percent down at $53.50 on Wednesday during pre-market session.