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Blueprint Medicines' NDA for Avapritinib on Track for GIST

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Blueprint Medicines Corporation (BPMC - Free Report) presented data from the NAVIGATOR study on its lead pipeline candidate, avapritinib, at the annual meeting of American Society of Clinical Oncology (ASCO), held in Chicago.

The registration-enabling NAVIGATOR study evaluated avapritinib for treating patients with PDGFRA Exon 18 mutant gastrointestinal stromal tumors (GIST) and the fourth-line GIST on two patient groups. The indications currently have no approved therapies. Data from the program showed that patients with PDGFRA Exon 18 mutant GIST achieved an objective response rate (ORR) of 86% while the median duration of response (DOR) was not reached. Meanwhile, patients with fourth-line GIST had an ORR of 22% while the median DOR was 10.2 months.

Based on the data presented, Blueprint Medicines 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.

Meanwhile, 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 - Free Report) Stivarga (regorafenib) on patients with the third or fourth-line GIST. The company also has an objective to initiate a global phase III precision medicine probe, COMPASS-2L, to evaluate the safety and efficacy of avapritinib as compared to Pfizer’s (PFE - Free Report) Sutent (sunitinib) for second-line GIST patients with pre-specified disease genotypes in the second half of 2019.

Shares of Blueprint Medicines have soared 57% so far this year versus the industry’s slip of 1.9%.

We would like to remind investors that other than GIST; Blueprint Medicines is pursuing a broad clinical development investigation on avapritinib across the advanced, indolent and smoldering forms of systemic mastocytosis (SM).

Avapritinib is currently being evaluated in two studies, namely the phase I EXPLORER clinical study and the registration-enabling phase II PATHFINDER study for advanced SM. The company looks forward to submit an NDA for avapritinib pertaining to the advanced SM indication in the first quarter of 2020.

Blueprint Medicines has no approved product in its portfolio at the moment. Thus, an approval of avapritinib will lower the company’s heavy dependence on its partners for revenue generation. The company currently has partnership with Roche (RHHBY - Free Report) and CStone Pharmaceuticals for the development and commercialization of its pipeline candidates.

Zacks Rank

Blueprint Medicines currently has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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