Blueprint Medicines highlights long-term clinical and real-world data for Ayvakit at EHA and EAACI 2025

Blueprint Medicines Corporation (NASDAQ: BPMC) presented new long-term data at the 2025 European Hematology Association (EHA) and European Academy of Allergy and Clinical Immunology (EAACI) congresses, demonstrating sustained efficacy and a favorable safety profile for AYVAKIT/AYVAKYT (avapritinib) in systemic mastocytosis (SM). The American biotech company reinforced Ayvakit’s role as a disease-modifying therapy across both indolent (ISM) and advanced SM, based on data drawn from multiple trials and large real-world cohorts.

The announcements mark a decade-long scientific collaboration by Blueprint Medicines with clinicians and patient communities. Data were sourced from its pivotal PIONEER, PATHFINDER, and EXPLORER trials, as well as from the PRISM real-world burden of disease study. These findings not only showcase Ayvakit’s durability across treatment settings but also help redefine the therapeutic standard for a complex and historically underserved rare disease.

What long-term data from PIONEER trial says about Ayvakit in indolent SM

The three-year follow-up data from the PIONEER trial confirmed that Ayvakit consistently improved symptom domains including gastrointestinal, dermatologic, and neurocognitive manifestations in ISM patients. These outcomes translate to meaningful quality-of-life gains that align with broader health status indicators recognized by allergists and immunologists. Ayvakit maintained a strong safety profile, with only 3 percent of patients discontinuing due to treatment-related adverse events (TRAEs), most of which were low-grade and manageable.

In addition to physical symptom relief, patients reported enhanced well-being measured by both disease-specific tools like the Mastocytosis Quality of Life (MC-QoL) questionnaire and global health instruments. This new evidence supplements two-year data published in The Journal of Allergy and Clinical Immunology: In Practice earlier in May 2025, and supports the continued expansion of Ayvakit’s label and global reimbursement pathways.

PATHFINDER and EXPLORER trials confirm survival gains in advanced systemic mastocytosis

Blueprint Medicines also shared updated survival results from the PATHFINDER and EXPLORER trials, which evaluated Ayvakit in advanced SM. Notably, Ayvakit delivered extended overall survival across all risk groups classified by the Revised Mutation-Adjusted Risk Score (MARS-R)—a validated model developed in collaboration with University Hospital Mannheim.

Compared to historical real-world cohorts treated with midostaurin from the German Registry on Disorders of Eosinophils and Mast Cells (GREM), Ayvakit-treated patients achieved substantially improved survival outcomes. The data underscore the drug’s clinical superiority in a disease known for its poor prognosis and limited treatment options.

These survival benefits have catalyzed a shift in physician attitudes, as shared by Blueprint’s Chief Medical Officer Becker Hewes, M.D. According to Dr. Hewes, positive real-world experiences with Ayvakit are influencing clinical judgment, with physicians increasingly open to initiating disease-modifying treatment earlier and extending treatment durations across prognostic subgroups.

PRISM study reveals real-world systemic burden of ISM

Findings from the PRISM study—one of the largest real-world analyses of systemic mastocytosis—offered crucial context to the trial-based results. Patients with ISM reported profound disruptions in physical function, emotional well-being, social participation, and daily lifestyle, including limitations in work and academic settings, and behavioral adjustments to avoid known symptom triggers.

This study adds critical patient-centric validation to Ayvakit’s role, emphasizing the need for early therapeutic intervention in SM, beyond symptom management alone. Blueprint Medicines has framed PRISM as a foundational evidence base in advancing the standard of care and bolstering access discussions in regulatory and payer settings.

Sentiment analysis: Blueprint Medicines stock trends, analyst views, and investment signals

Blueprint Medicines Corporation (NASDAQ: BPMC) is currently trading near $128, slightly off its 52-week high and well above medium-term moving averages. The Relative Strength Index (RSI) stands near 77, indicating potential short-term overbought conditions. However, institutional sentiment remains largely bullish due to robust Ayvakit revenues, pipeline strength, and a pending acquisition by Sanofi.

Buy-side analysts maintain a predominantly positive outlook, with price targets ranging between $129 and $135. Ayvakit’s year-over-year revenue growth of 61 percent and updated full-year guidance of $700–720 million have been positively received. While the biotech firm remains unprofitable on a trailing twelve-month basis, its ~$900 million cash reserve offers operational runway and flexibility for pipeline investment.

TipRanks and other technical platforms maintain a “Positive” sentiment on BPMC stock, while several brokerage firms—including TD Cowen and Citizens JMP—have shifted to Hold due to near-term valuation ceilings ahead of the Sanofi deal closure.

For long-term investors, the buy case remains intact, driven by Ayvakit’s sustained commercial expansion and the depth of Blueprint Medicines’ oncology pipeline. However, short-term traders may opt to wait for a retracement or confirmation of regulatory milestones tied to the Sanofi acquisition.

Institutional positioning and market activity

U.S. institutional investor flows remain moderate, with no sharp inflow or outflow trends reported in the immediate term. Due to the lack of FII/DII differentiation in U.S. equity markets, detailed fund breakdowns are not available. However, sentiment inferred from volume trends and analyst commentary suggests modest accumulation by healthcare-focused funds.

Recent downgrades from some analysts appear to reflect caution related to valuation plateauing near deal premium levels. Conversely, bullish coverage continues to cite the scale of Ayvakit’s commercial success and Blueprint’s strategic R&D execution.

Future outlook: What lies ahead for Ayvakit and Blueprint Medicines

Looking forward, Blueprint Medicines is expected to pursue global expansion for Ayvakit, especially in emerging and underserved markets. The company has indicated interest in advancing Ayvakit into earlier lines of therapy and exploring new indications informed by PRISM and MARS-R insights. Further publications and real-world registry updates may also support label extensions and pricing negotiations in Europe and Asia.

Regulatory watchers are also tracking the trajectory of Sanofi’s proposed $9+ billion acquisition of Blueprint Medicines, which may close in late 2025 subject to antitrust and governance approvals. If completed, the deal could bring expanded commercial reach and clinical trial infrastructure to accelerate Ayvakit’s penetration and future portfolio developments.

In the context of the 2025 EHA and EAACI meetings, Blueprint’s position as a rare disease leader appears reaffirmed. Ayvakit is increasingly being seen not just as a high-performing product, but as a platform-defining success in precision oncology.