Blueprint Medicines' (BPMC) Ayvakit Meets Goal in PIONEER Study

·4-min read

Blueprint Medicines Corporation BPMC announced top-line data from part 2 of the PIONEER study, which evaluated its marketed drug, Ayvakit (avapritinib) for treating patients with non-advanced systemic mastocytosis (SM).

Data from the registrational part 2 of the PIONEER study showed that treatment with Ayvakit led to clinically meaningful and highly significant improvements across the primary and all key secondary endpoints, including patient-reported symptoms and measures of mast cell burden.

Per the company, the study investigating Ayvakit plus best available care versus placebo plus best available care (control arm), met the primary endpoint with a highly significant difference in the mean change in total symptom score (TSS) at 24 weeks.

While the Ayvakit arm had a reduction of 15.6 points in mean TSS at 24 weeks, the control arm had a reduction of 9.2 points in mean TSS at 24 weeks. The results did not match investors’ expectations, hence the stock fell following the announcement of the news.

Ayvakit had a favorable safety profile compared to the control arm.

Blueprint Medicines plans to submit a supplemental new drug application (sNDA) for Ayvakit for treating non-advanced SM in the fourth quarter of 2022, based on the above data.

A type II variation marketing authorization application for Ayvakit is expected to be filed to the European Medicines Agency next year.

Shares of Blueprint Medicines have plunged 42.9% so far this year compared with the industry’s decrease of 17.7%.

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Blueprint Medicines’ lead drug, Ayvakit is approved for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumors (“GIST”), harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations in the United States. The European Commission has granted conditional marketing authorization to Ayvakyt (brand name of Ayvakit in Europe) as a monotherapy for the treatment of adult patients with unresectable/metastatic GIST harboring the PDGFRA D842V mutation.

In June 2021, the FDA approved Ayvakit for a new indication — advanced systemic mastocytosis (“SM”) — a rare and debilitating disease.

In March 2022, the European Commission approved Ayvakyt as a monotherapy for the treatment of adult patients with aggressive SM, including SM with an associated hematological neoplasm as well as mast cell leukemia, after at least one systemic therapy.

In the first six months of 2022, Ayvakit/Ayvakyt generated sales worth $52.3 million, reflecting a significant increase year over year. Potential label expansion into additional indications will make Ayvakit eligible to treat a broader patient population and is likely to drive sales further in 2022 and beyond.

Zacks Rank & Stocks to Consider

Blueprint Medicines currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector include Achilles Therapeutics plc ACHL, Atara Biotherapeutics, Inc. ATRA and ORIC Pharmaceuticals, Inc. ORIC, all carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Achilles Therapeutics’ loss per share estimates narrowed 6% for 2022 and 8.6% for 2023 in the past 60 days.

Earnings of Achilles Therapeutics surpassed estimates in three of the trailing four quarters and missed on the remaining occasion. ACHL delivered an earnings surprise of 12.45%, on average.

Atara Biotherapeutics’ loss per share estimates narrowed 26.9% for 2022 and 25.6% for 2023 in the past 60 days.

Earnings of Atara Biotherapeutics surpassed estimates in three of the trailing four quarters and missed on the other occasion. ATRA delivered an earnings surprise of 4.83%, on average.

ORIC Pharmaceuticals’ loss per share estimates narrowed 0.8% for 2022 and 14.8% for 2023 in the past 60 days.

Earnings of ORIC Pharmaceuticals surpassed estimates in three of the trailing four quarters and missed on the remaining occasion. ORIC delivered an earnings surprise of 8.85%, on average.


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