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Blueprint Medicines Up on Positive Top-Line Data From SM Study

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Shares of Blueprint Medicines Corporation (BPMC - Free Report) were up 13% on Tuesday after the company announced positive top-line data from the phase I EXPLORER study and the phase II PATHFINDER study, which evaluated its lead drug Ayvakit (avapritinib) for the treatment of patients with advanced systemic mastocytosis (SM), a rare, debilitating disease.

In fact, the stock has rallied 9% so far this year compared with the industry’s increase of 0.6%.


Data from both the EXPLORER study and the registration-enabling PATHFINDER study showed that treatment with Ayvakit led to profound reductions in mast cell burden, high overall response and complete remission rates as well as durable clinical benefits including prolonged median overall survival (OS) in the patient population. The drug was generally well-tolerated and reinforced its safety profile at the once daily 200 mg dose.

Notably, in the EXPLORER study, the overall response rate (ORR) was 76% and the median duration of response (DOR) was 38.3 months while 36% patients had a complete remission with full or partial recovery of peripheral blood counts (CR/CRh). The PATHFINDER study had an ORR of 75% with a median duration of response not reached while 19% patients had a CR/CRh.

Blueprint Medicines plans to submit a supplemental NDA to the FDA for Ayvakit in the fourth quarter of 2020 to treat patients with advanced SM.

We note that in January 2020, Ayvakit was approved by the FDA for treating unresectable or metastatic gastrointestinal stromal tumor (GIST), harboring a PDGFRA exon 18 mutation including PDGFRA D842V mutations in adults. Following this nod, Ayvakit became the first precision therapy to have won the FDA nod for treating genomically-defined patients with GIST, a rare form of cancer.

In the first six months of 2020, Ayvakit generated sales worth $9.1 million. The drug is also under review in Europe with a decision expected by the end of this month. A potential approval in the region should boost Ayvakit’s sales in the quarters ahead. Potential label expansion of the drug for the SM indication should also drive its prospects.

Zacks Rank & Stocks to Consider

Blueprint Medicines currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the healthcare sector include Emergent BioSolutions Inc. (EBS - Free Report) , Horizon Therapeutics Public Limited Company (HZNP - Free Report) and QIAGEN N.V. (QGEN - Free Report) , all presently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Emergent’s earnings estimates have been revised 55.5% upward for 2020 and 37.3% for 2021 over the past 60 days. The stock has soared 86.7% year to date.

Horizon Therapeutics’ earnings estimates have moved 49.7% north for 2020 and 49.6% for 2021 over the past 60 days. The stock has surged 115.9% year to date.

QIAGEN’s earnings estimates have been revised 10.6% upward for 2020 and 22.5% for 2021 over the past 60 days. The stock has soared 48.9% year to date.

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