Company Description
Blueprint Medicines Corporation (Nasdaq: BPMC) is a global, fully integrated biopharmaceutical company focused on inventing medicines for serious diseases in two core areas: allergy/inflammation and oncology/hematology. The company describes its approach as targeting the root causes of disease, using deep scientific knowledge in its focus areas and drug discovery expertise across multiple therapeutic modalities. Blueprint Medicines has a track record of success with two approved medicines, including AYVAKIT/AYVAKYT (avapritinib), which it is bringing to patients with systemic mastocytosis (SM) in the U.S. and Europe.
According to recent company disclosures, Blueprint Medicines operates as a commercial-stage biopharmaceutical company with established research, development and commercial capability and infrastructure. It generates product revenue from sales of AYVAKIT/AYVAKYT and advances a broad pipeline of programs ranging from early science to advanced clinical trials in mast cell diseases and solid tumors. The company is listed on the Nasdaq Global Select Market under the symbol BPMC.
Core therapeutic focus and disease areas
Blueprint Medicines states that it seeks to alleviate human suffering by solving important medical problems in allergy/inflammation and oncology/hematology. A central theme of its work is mast cell biology and diseases driven by abnormal mast cell activation. Systemic mastocytosis (SM) is highlighted as a rare disease driven in about 95 percent of cases by the KIT D816V mutation, leading to uncontrolled proliferation and activation of mast cells and chronic, severe, often unpredictable symptoms across multiple organ systems. The company notes that the vast majority of those affected have indolent systemic mastocytosis (ISM), while a minority have advanced SM subtypes such as aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL).
Blueprint Medicines is bringing AYVAKIT/AYVAKYT to people living with SM in the U.S. and Europe and is advancing additional programs in mast cell-mediated diseases, including chronic urticaria and other mast cell disorders. In oncology/hematology, the company references a pipeline that includes programs in breast cancer and other solid tumors vulnerable to CDK2 inhibition, as well as targeted protein degrader candidates in its CDK franchise.
AYVAKIT/AYVAKYT (avapritinib)
AYVAKIT (avapritinib) in the U.S. and AYVAKYT (avapritinib) in the EU are central to Blueprint Medicines' commercial portfolio. Company communications describe AYVAKIT/AYVAKYT as the first and only medicine approved by the U.S. Food and Drug Administration (FDA) to treat the root cause of systemic mastocytosis. It is FDA approved for adults with indolent SM (ISM), adults with advanced SM (including ASM, SM-AHN and MCL), and adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. In Europe, under the AYVAKYT brand, it is approved for adults with ISM with moderate to severe symptoms inadequately controlled on symptomatic treatment, adults with ASM, SM-AHN or MCL after at least one systemic therapy, and adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation.
The company reports that AYVAKIT is a potent and selective inhibitor of activated KIT and PDGFRA mutant kinases and is designed to bind and inhibit these proteins in diseases where mutations force them into an increasingly active state. Blueprint Medicines emphasizes long-term clinical data from trials such as PIONEER, PATHFINDER and EXPLORER, showing durable symptom improvements, quality-of-life benefits and survival outcomes in SM populations, as well as real-world evidence characterizing the burden of disease.
Pipeline: elenestinib, BLU-808 and CDK programs
Beyond its approved medicines, Blueprint Medicines highlights a pipeline of investigational therapies. Elenestinib is described as a next-generation, potent and highly selective KIT D816V inhibitor being studied in indolent systemic mastocytosis. The company has initiated the registration-enabling Phase 3 HARBOR trial of elenestinib in ISM, with the goal of advancing disease-modifying therapy within its SM franchise.
BLU-808 is an investigational, highly potent and selective oral wild-type KIT inhibitor developed using the company’s expertise in mast cell biology. Blueprint Medicines reports Phase 1 data in healthy volunteers showing BLU-808 was well-tolerated at all doses tested, with dose-dependent reductions in serum tryptase and pharmacokinetics supporting once-daily oral dosing. The company is initiating multiple proof-of-concept studies of BLU-808 in mast cell-mediated allergic and inflammatory diseases, including chronic urticaria, allergic rhinoconjunctivitis, allergic asthma and mast cell activation syndrome.
In oncology, Blueprint Medicines is advancing CDK2 and CDK4 targeted protein degraders and has evaluated the CDK2 inhibitor BLU-222 in a Phase 1 dose escalation study. The company indicates it is prioritizing investment in CDK targeted protein degraders for breast cancer and other solid tumors and engaging potential partners for its CDK franchise.
Business model and operations
Blueprint Medicines describes itself as a fully integrated company, encompassing discovery, clinical development and commercialization. Its business model combines product revenue from its approved medicines with continued investment in research and development across its pipeline. Company disclosures highlight AYVAKIT/AYVAKYT net product revenues as the primary revenue source and reference a strategy of balancing investment in innovation with financial discipline, aiming to achieve a self-sustainable financial profile.
The company emphasizes its established presence among specialists such as allergists, dermatologists and immunologists, particularly in the context of systemic mastocytosis and related mast cell diseases. It also references collaborations, such as an arrangement under which AYVAKIT/AYVAKYT is marketed in China by CStone Pharmaceuticals with tiered percentage royalties on sales, and an equity investment in IDRx, Inc. that generated proceeds upon that company’s acquisition by GSK.
Corporate developments and acquisition by Sanofi
On June 2, 2025, Sanofi and Blueprint Medicines announced an agreement under which Sanofi will acquire Blueprint Medicines. The transaction terms include a cash payment per share and a contingent value right tied to milestones for BLU-808. The announcement states that, subject to customary closing conditions, Sanofi expects to complete the acquisition in the third quarter of 2025. Following completion, a wholly owned subsidiary of Sanofi is expected to merge with and into Blueprint Medicines, with Blueprint becoming part of Sanofi’s rare disease and immunology portfolio.
Subsequently, on July 28, 2025, Blueprint Medicines filed a Form 15 (15-12G) with the U.S. Securities and Exchange Commission to terminate registration of its common stock under Section 12(g) of the Securities Exchange Act of 1934 and suspend its duty to file certain periodic reports. The filing notes that the approximate number of holders of record as of the certification date was one. This indicates that Blueprint Medicines’ reporting obligations as an independent public company have been suspended following the acquisition-related corporate actions.
Regulatory and governance context
Before the Form 15 filing, Blueprint Medicines’ common stock traded on the Nasdaq Global Select Market under the symbol BPMC. An 8-K filed on June 20, 2025, describes the company’s annual meeting of stockholders held on June 18, 2025, at which stockholders elected Class I directors, approved a non-binding advisory vote on executive compensation and ratified the appointment of the company’s independent registered public accounting firm for the fiscal year ending December 31, 2025.
With the subsequent Form 15 filing, Blueprint Medicines has terminated registration of its common stock under Section 12(g) and suspended certain reporting duties, consistent with its transition into a company with a single holder of record in connection with the Sanofi acquisition.
Position within pharmaceutical preparation manufacturing
Within the broader pharmaceutical preparation manufacturing industry, Blueprint Medicines is characterized in its own disclosures as focusing on therapies that “potently and selectively target known drivers of disease,” particularly in mast cell-driven conditions and targeted oncology. Rather than emphasizing broad, non-specific treatments, the company’s programs concentrate on genetically or biologically defined disease drivers such as KIT and PDGFRA mutations, and on mast cell activation pathways.
By concentrating on genomically and biologically defined subsets of patients and leveraging its mast cell biology expertise, Blueprint Medicines aims to develop therapies that address the underlying mechanisms of disease. Its portfolio in systemic mastocytosis and related mast cell disorders, alongside its CDK-targeted oncology programs, reflects this strategy.
Investment and research considerations
For those researching BPMC as a stock symbol, it is important to note that Blueprint Medicines has entered into an acquisition agreement with Sanofi and has filed a Form 15 to terminate registration of its common stock and suspend certain reporting obligations. This means BPMC’s status as an independently traded security has changed in connection with the transaction. Historical financial information, product revenue trends for AYVAKIT/AYVAKYT, and details on the pipeline can be found in the company’s prior earnings releases and regulatory filings.
From a research perspective, Blueprint Medicines’ disclosures highlight several themes: the burden and natural history of systemic mastocytosis; long-term safety and efficacy data for AYVAKIT/AYVAKYT; the potential of elenestinib as a next-generation KIT D816V inhibitor; and the broad applicability of BLU-808 across mast cell-mediated allergic and inflammatory diseases. These areas are central to understanding the company’s scientific and commercial trajectory prior to its acquisition by Sanofi.