Blueprint Medicines' (BPMC) Aykavit sNDA Gets FDA Priority Tag
Blueprint Medicines Corporation BPMC announced that the FDA has accepted its supplemental new drug application (sNDA) seeking label expansion for Ayvakit, Blueprint’s only marketed drug to treat adults’ indolent Systemic Mastocytosis (SM). The FDA has granted priority review to the sNDA with a Prescription Drug User Fee Act (PDUFA) date of May 22, 2023.
Aykavit, a kinase inhibitor, is already approved in the United States for the treatment of adults with advanced SM, which includes patients with aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL). Indolent SM is a form of non-advanced SM. Aykavit is also approved to treat adults with unresectable or metastatic gastrointestinal stromal tumors (GIST). Aykavit is also approved in Europe for the treatment of adults with ASM, SM-AHN, or MCL and metastatic GIST.
The sNDA for indolent SM was based on positive top-line results from the global PIONEER study conducted by Blueprint. Management reported that Aykavit achieved the primary as well as all key secondary endpoints in the PIONEER study. The study showed that Ayvakit led to statistically significant reductions in patient-reported symptoms and all measures of mast cell burden studies. The drug was generally well tolerated and met a safety profile supporting chronic treatment. If the company receives approval for expanded use in indolent SM, Blueprint will become the first company with approved therapies for patients which will provide a major boost to the company’s revenues.
In the past year, the shares of Blueprint have declined 35.7% compared with the industry’s decline of 6.9%.
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SM is a rare disease that is caused by KIT D816V mutation in most cases. It is a result of abnormal growth of mast cells in the human body resulting in chronic, severe, and often unprecedented symptoms in multiple organs. According to demographics, most patients suffer from non-advanced (indolent or smoldering) SM. Popular symptoms for such non-advanced SM include anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue, and bone pain despite treatment being treated with multiple symptom-driven therapies. This significantly degrades the quality of life of patients and with no approved therapies for the treatment of non-advanced SM, there remains a huge unmet medical need.
We remind investors that Ayvakit faces competition for the GIST indication from Deciphera Pharmaceutical’s DCPH Qinlock, which is approved for advanced GIST. Deciphera received FDA approval for Qinlock (ripretinib), for the treatment of adult patients with advanced GIST, who have received prior treatment with three or more kinase inhibitors back in May 2020.
DCPH is also working to expand the label of Qinlock for the larger commercial opportunity in second-line GIST in its phase III Intrigue study and reported statistically significant data, recently.
Zacks Rank and Stocks to Consider
Blueprint Medicines currently has a Zacks Rank #4 (Sell).
Some better-ranked stocks in the same industry are 89BIO, Inc. ETNB and Adicet Bio, Inc. ACET, both carrying a Zacks Ranks #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
In the past 90 days, the estimate for 89BIO’s 2022 loss per share has narrowed from $3.34 to $2.65. During the same period, the loss estimate per share for 2023 has narrowed from $3.59 to $2.59. In the past year, the shares of 89BIO have risen by 28.1%.
ETNB delivered an average earnings surprise of 10.08%, beating estimates in three out of the trailing four reported quarters.
In the past 90 days, the estimate for Adicet Bio’s 2022 loss per share has narrowed from $1.63 to $1.54. During the same period, the loss estimate per share for 2023 has narrowed from $2.25 to $2.09. In the past year, the shares of Adicet have decreased by 31.9%.
ACET delivered an average earnings surprise of 54.24% for the trailing four quarters, having beaten estimates twice and missing the same twice.
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