On Jun 4, we issued an updated research report on Blueprint Medicines Corporation BPMC.
The company’s lead pipeline candidate, avapritinib, an inhibitor of KIT and PDGFRA proteins, is being evaluated in several late-stage studies for cancer indications. Earlier this week, Blueprint Medicines announced data from the registration-enabling NAVIGATOR study, which evaluated avapritinib for treating patients with PDGFRA Exon 18 mutant gastrointestinal stromal tumors (GIST) and the fourth-line GIST. The indications currently have no approved therapies.
The data was presented at the annual meeting of American Society of Clinical Oncology (ASCO) held in Chicago. The company plans to submit a new drug application (NDA) for avapritinib later this month and subsequently, file a regulatory application in Europe during third-quarter 2019 for the given indication.
Notably, Blueprint Medicines is also investigating avapritinib in other settings for GIST. The phase III VOYAGER program is evaluating the safety and efficacy of avapritinib compared with Bayer’s BAYRY Stivarga (regorafenib) on patients with the third or fourth-line GIST. The company also plans to initiate a global phase III precision medicine study, COMPASS-2L, to evaluate the safety and efficacy of avapritinib as compared to Pfizer’s PFE Sutent (sunitinib) for treating second-line GIST patients with pre-specified disease genotypes during the second half of 2019.
Other than GIST, Blueprint Medicines is pursuing a broad clinical development program for avapritinib across the advanced, indolent and smoldering forms of systemic mastocytosis (SM). Avapritinib is currently being evaluated under two studies, namely the phase I EXPLORER clinical study and the registration-enabling phase II PATHFINDER study for advanced SM. The company plans to submit an NDA for avapritinib for the advanced SM indication in the first quarter of 2020.
Apart from avapritinib, Blueprint Medicines has several pipeline candidates in early developmental stages.
BLU-667 in being studied in phase I ARROW study evaluating the previously-treated patients with receptor tyrosine RET-fusion non-small cell lung cancer (NSCLC) and RET-mutant medullary thyroid carcinoma (MTC). The company announced encouraging interim top-line data from the study in March 2019.
Blueprint Medicines also presented updated results from the registration-enabling ARROW analysis on BLU-667 for treating patients with RET-altered cancers at the annual meeting of ASCO this month. The company decides to submit an NDA for BLU-667 for the NSCLC indication in the first quarter of 2020 while the NDA for the MTC indication is expected to be filed during the first half of 2020.
By 2020, the company anticipates to have a couple of marketed products in the United States, such as avapritinib and BLU-667.
Other pipeline candidates in phase I development are BLU-554 for hepatocellular carcinoma HCC, which is a type of liver carcinoma and BLU-782 for fibrodysplasia ossificans progressiva, a severely disabling genetic disease caused by mutations in the ALK2 gene. The company aims to begin a phase II program on BLU-782 during the fourth quarter of 2019.
Notably, Blueprint Medicines presently has no approved product in its portfolio. Thus, an approval of avapritinib will lower the company’s heavy dependence on its partners for revenue generation. The company is currently in partnership with Roche RHHBY and CStone Pharmaceuticals for the development and commercialization of its pipeline candidates.
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