Blueprint's (BPMC) Systemic Mastocytosis Study Data Impresses

Blueprint Medicines Corporation (NASDAQ:BPMC) announced encouraging initial data from the dose-finding part 1 of phase II PIONEER study on its lead candidate avapritinib for treating patients with indolent systemic mastocytosis (SM). The candidate was well-tolerated at all dose levels tested and no patients discontinued treatment due to serious adverse side effects.

SM is a rare form of disease wherein mast cells accumulate in internal tissues and organs such as liver, spleen, bone marrow and small intestines.

Data from the study showed that treatment with once daily avapritinib (25 mg, 50 mg or 100 mg) led to rapid and robust reductions in serum tryptase, an enzyme released from mast cells when those are activated.

The results will be released at the annual meeting of American Society of Hematology held in Orlando, FL.

Blueprint Medicines plans to initiate patient screening in registration-enabling part 2 portion of the PIONEER study for indolent SM in the first half of 2020.

Shares of Blueprint Medicines were up 5.3% following the aforementioned news. In fact, the stock has surged 45.6% so far this year, outperforming the industry’s increase of 6.6%.

Blueprint Medicines is pursuing a broad clinical development program for avapritinib across the advanced, indolent and smoldering forms of SM. The candidate is currently being evaluated in three ongoing, registration-enabling studies for SM, namely the EXPLORER, the PATHFINDER and the PIONEER.

The company plans to submit a new drug application (NDA) for avapritinib in the first quarter of 2020 for the advanced SM indication.

The NDA will be based on response data from approximately 55 patients and safety data pertaining to roughly 100 patients from the EXPLORER and the PATHFINDER studies.

Meanwhile, Blueprint Medicines is evaluating avapritinib in several late-stage studies for some cancer indications. In August 2019, the FDA accepted the NDA for avapritinib to treat patients with PDGFRA Exon 18 mutant gastrointestinal stromal tumors (GIST), regardless of any prior therapy, and the fourth-line GIST.

The FDA has set an action date of Feb 14, 2020 for both indications.

Additionally, Blueprint Medicines is evaluating the safety and efficacy of avapritinib compared with Bayer’s (OTC:BAYRY) Stivarga (regorafenib) in the phase III VOYAGER study for treating patients with third- and fourth-line GIST. The company plans to file a supplemental NDA for avapritinib to the FDA in the second half of 2020 for addressing the third-line GIST.

We remind investors that Blueprint Medicines has no approved product in its portfolio at the moment. Revenues generated by the company are all related to its partnerships with Roche (OTC:RHHBY) and CStone Pharmaceuticals for the development and commercialization of its pipeline candidates. A potential approval of avapritinib will lower the company’s heavy dependence on its partners for revenues and thus help boost its growth prospects.

Zacks Rank & Key Pick

Blueprint Medicines currently carries a Zacks Rank #3 (Hold). A better-ranked stock in the biotech sector is Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) , which sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Vertex’s earnings estimates have been revised 5% upward for 2019 and 10.6% for 2020 over the past 60 days. The stock has surged 34.7% year to date.

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