Blueprint Medicines said its drug Ayvakit (avapritinib) failed to meet its primary endpoint of an improvement in progression-free survival compared to regorafenib in a phase 3 study of patients with gastrointestinal-stromal tumors (GIST), rare tumors of the GI tract.
Photo: Jeff Albers, CEO of Blueprint Medicines
Shares in Blueprint fell more than 15 percent on the news.
The company said it plans to continue to commercialize Ayvakit in the United States for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and seek marketing approval for avapritinib for the treatment of this patient population in additional geographies.
The European Commission is expected to decide on Blueprint’s marketing authorization application for the treatment of adults with PDGFRA D842V mutant GIST in the third quarter of 2020. Based on the top-line phase 3 VOYAGER data, the company plans to discontinue further development of avapritinib in GIST beyond PDGFRA exon 18 mutant GIST.
Ayvakit is a kinase inhibitor approved by the U.S. Food and Drug Administration for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. AYVAKIT is the first precision therapy approved to treat a genomically defined population of patients with GIST and the only highly active treatment for PDGFRA exon 18 mutant GIST. The FDA granted Breakthrough Therapy designation to avapritinib for the treatment of unresectable or metastatic GIST harboring the PDGFRA D842V mutation.
“While we are disappointed by the outcome of the VOYAGER trial, we are deeply grateful to the patients, investigators and clinical site staff who contributed to the completion of this global study,” said Jeff Albers, CEO of Blueprint Medicines. “We hope these data will reveal important insights to improve the scientific understanding of the disease and inform future innovations in GIST, and we are committed to sharing the results at a future medical meeting.”
The VOYAGER trial evaluated the efficacy and safety of avapritinib versus regorafenib in patients with third- or fourth-line GIST.
Avapritinib showed a median progression free survival of 4.2 months compared to 5.6 months for regorafenib. The difference in median progression free survival 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.
Based on its current operating plans, Blueprint Medicines expects to have enough money 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.
Blueprint is also 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.
Author: Rare Daily Staff
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