Shares of Blueprint Medicines BPMC rallied 18.2% on Monday driven by investor enthusiasm following the company’s announcement of its 2025 corporate outlook and growth strategy, highlighting plans to build on its established R&D and commercial expertise.
Blueprint’s commercial portfolio only comprises its lead drug Ayvakit (avapritinib), an inhibitor of KIT and PDGFRA proteins, which was initially approved in the United States and the EU to treat gastrointestinal stromal tumors in 2020. Later, the drug’s label was expanded by respective regulatory authorities in both geographies to treat advanced and indolent systemic mastocytosis (SM).
In the press release, BPMC has updated its projections for its SM franchise, driven by the strong global launch of Ayvakit, a significant increase in diagnosed SM patients, and new epidemiological data suggesting SM is more prevalent than previously estimated. The company now anticipates a peak revenue opportunity of $4 billion for the SM franchise, with Ayvakit alone expected to achieve $2 billion in annual revenues by 2030.
The company anticipates reporting its fourth-quarter and full-year 2024 results in February 2025. In October 2024, Blueprint projected Ayvakit product revenues in the range of $475-$480 million for the year, marking growth of more than 130% compared with 2023. The growth trajectory of Ayvakit sales is expected to continue rising in 2025.
Additionally, in 2025, the company will seek to achieve reimbursement of Ayvakit in 20 countries or more across the globe to ensure better patient access. In the past three months, BPMC shares have gained 15.9% against the industry’s 12.1% decline.
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BPMC Reports Upbeat Data From Its Phase I BLU-808 Study
Blueprint is conducting an early-stage study of BLU-808 to assess its unique potency and selectivity, aiming to develop a flexible treatment approach and optimize the benefit-risk profile for a range of mast cell-driven diseases. BLU-808 is an oral wild-type KIT inhibitor.
Per the data readout from the phase I single-ascending dose (SAD) and multiple-ascending dose (MAD) study, BLU-808 demonstrated dose-dependent serum tryptase responses, indicating mast cell target engagement across various dose levels. In the SAD cohorts, tryptase reductions were observed after a single dose, while the MAD cohorts showed rapid, robust and sustained tryptase reductions.
Additionally, the candidate was overall well tolerated across all tested doses in the study. All treatment-related adverse events were mild in severity and did not result in discontinuations or dose modifications. Detailed results will be presented at an upcoming medical conference.
Based on the encouraging phase I data readout, Blueprint is initiating proof-of-concept studies on BLU-808 across a broad therapeutic area, including chronic urticaria, allergic asthma, allergic rhinitis, allergic conjunctivitis and mast cell activation syndrome in 2025.
Other Developmental Programs in BPMC’s Clinical Pipeline
To further bolster its footprint in the SM market, Blueprint is evaluating elenestinib (BLU-263) — a next-generation KIT inhibitor — in the phase II/III HARBOR study to treat patients with indolent SM. In the latest press release, BPMC announced that it has initiated the registration-enabling phase III portion of the study. Site activations and enrollment for the study are currently ongoing and will continue throughout 2025.
Blueprint is accelerating the development of its CDK2 and CDK4 targeted protein degraders, focusing on their potential to achieve best-in-class profiles for treating breast cancer and other solid tumors. While the company is concluding the phase I study of its CDK2 inhibitor BLU-222 for breast cancer, it plans to shift investment away from this program. BPMC is actively exploring strategic partnerships to further expand its CDK franchise.
Blueprint Medicines Corporation Price and Consensus
Blueprint Medicines Corporation Price and Consensus
Blueprint currently carries a Zacks Rank #2 (Buy).
Some other top-ranked stocks from the sector are Castle Biosciences CSTL, CytomX Therapeutics CTMX and Halozyme Therapeutics HALO. Currently, CSTL and CTMX sport a Zacks Rank #1 (Strong Buy) each, while HALO carries a Zacks Rank #2 at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
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