Full Press Release Details
Top-line Data from Phase 3 VOYAGER Trial
The VOYAGER trial evaluated the efficacy and safety of avapritinib (N=240) versus regorafenib (N=236) in patients with third- or fourth-line GIST.
Avapritinib showed a median PFS of 4.2 months compared to 5.6 months for regorafenib. The difference in median PFS between the avapritinib and regorafenib groups was not statistically significant. The overall response rate was 17 percent for the avapritinib group and 7 percent for the regorafenib group.
Avapritinib was generally well-tolerated with most adverse events reported as Grade 1 or 2. Top-line safety results were consistent with previously reported data, and no new safety signals were observed.
Additional analyses of the VOYAGER trial results are ongoing, and Blueprint Medicines plans to present the data at a future medical meeting.
Financial Guidance
Based on its current operating plans, Blueprint Medicines continues to expect that its existing cash, cash equivalents and investments together with anticipated product revenues but excluding any additional potential option fees, milestone payments or other payments under its collaboration or license agreements, will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the second half of 2022. Anticipated savings from the discontinuation of further development of avapritinib in non-PDGFRA exon 18 mutant GIST indications is expected to offset any previously forecast revenues from those indications through 2022.
As of December 31, 2019 , Blueprint Medicines had cash, cash equivalents and investments of $548.0 million . In addition, Blueprint Medicines received $308.4 million in estimated net proceeds from its January 2020 follow-on public offering.
Conference Call Information
Blueprint Medicines will host a live webcast today beginning at 8:00 a.m. ET to discuss the top-line data from the VOYAGER trial. To access the live call, please dial (855) 728-4793 (domestic) or (503) 343-6666 (international), and refer to conference ID 2207088. A webcast of the conference call will be available in the Investors & Media section of Blueprint Medicines' website at http://ir.blueprintmedicines.com . The archived webcast will be available on Blueprint Medicines' website approximately two hours after the conference call and will be available for 30 days following the call.
About the VOYAGER Trial
VOYAGER was a global, open-label, randomized, Phase 3 trial designed to evaluate the efficacy and safety of avapritinib versus regorafenib in patients with third- or fourth-line GIST. Patients were randomized 1:1 to receive either avapritinib (300 mg once daily dosing) or regorafenib (160 mg once daily dosing for three out of every four weeks) at multiple sites in the United States , Canada , European Union, Australia and Asia . The primary efficacy endpoint was PFS by blinded, independent central radiology review, based on modified Response Evaluation Criteria in Solid Tumors version 1.1 (mRECIST 1.1 criteria) for GIST. For more information about the VOYAGER trial, please visit www.clinicaltrials.gov (ClinicalTrials.gov Identifier: NCT03465722).
About AYVAKIT (avapritinib)
Avapritinib is not approved for the treatment of any other indication in the U.S. or any other jurisdiction by the FDA or any other health authority.
Blueprint Medicines is developing avapritinib globally for the treatment of advanced, smoldering and indolent systemic mastocytosis (SM). The FDA granted Breakthrough Therapy Designation to avapritinib for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia.
Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of avapritinib and certain other drug candidates in Mainland China, Hong Kong , Macau and Taiwan . Blueprint Medicines retains development and commercial rights for avapritinib in the rest of the world.
About Blueprint Medicines
Cautionary Note Regarding Forward-Looking Statements
SOURCE Blueprint Medicines Corporation