#USA #Cambridge #AYVAKIT #Blueprint Medicines #avapritinib

Blueprint Medicines Highlights Long-Term Efficacy of AYVAKIT®/AYVAKYT® at 2025 EHA and EAACI Congresses

Blueprint Medicines Corporation (Nasdaq: BPMC) recently made waves in the medical community by presenting groundbreaking data at the 2025 European Hematology Association (EHA2025) Congress and the European Academy of Allergy and Clinical Immunology (EAACI) Congress.

The data reflects over ten years of alliance with healthcare experts and advocates dedicated to enhancing treatment options for systemic mastocytosis (SM). AYVAKIT®/AYVAKYT® (avapritinib) is positioned as the standard and steadfast choice for both indolent and advanced forms of SM. The importance of addressing the underlying causes of this disease is underscored by recent findings on the real-world burden that patients face while managing their condition.

Dr. Becker Hewes, Chief Medical Officer at Blueprint Medicines, shared insights on the extensive patient data gathered from the PIONEER, PATHFINDER, and EXPLORER trials. These studies have showcased long-term benefits of AYVAKIT treatment, with follow-up periods reaching an impressive five years for indolent systemic mastocytosis (ISM) and up to 6.5 years for advanced SM.

"AYVAKIT has transformed clinical outcomes for patients with systemic mastocytosis, demonstrating sustained disease control and potential for prolonged survival," Dr. Hewes stated.

Key Findings from EHA2025 and EAACI Congress

The presentations covered a broad array of findings:

PIONEER Study Insights

The data from the PIONEER study illustrated robust improvements in patient-reported symptoms over an impressive timeframe of 144 weeks. They highlighted significant advancements in overall quality of life across various measures reflective of real-world challenges faced by patients. Patients reported improved experiences across skin, gastrointestinal, and neurocognitive symptom domains.

Common treatment-related adverse events (TRAEs) were minimal, with a low discontinuation rate of only 3%. More importantly, the safety profile remained well-tolerated after a median exposure of three years. In fact, patients demonstrated the capacity to remain on treatment for as long as five years, further affirming AYVAKIT's favorable outcome.

PATHFINDER and EXPLORER Studies

Moving to advanced SM, AYVAKIT has shown significant survival benefits, validating its status as an essential treatment option. When indirectly compared to other therapies, AYVAKIT enabled improvements across all prognostic categories, leveraging the Revised Mutation-Adjusted Risk Score (MARS-R) as a new tool for assessing survival risks.

Data generated in partnership with the University Hospital Mannheim confirmed MARS-R's role in aiding healthcare providers in making personalized care decisions, providing them with more clarity on the treatment processes.

PRISM Study Findings

The PRISM study is one of the most extensive analyses focusing on the challenges faced by patients with ISM. Across varying severities of the disease, patients voiced the significant impact SM had on their lives, affecting their physical, social, and emotional wellbeing. Everyday activities such as work and social interactions were frequently hindered, illustrating the need for comprehensive treatment approaches.

Further Presentations

Blueprint Medicines presented several additional sessions during the congresses:

• - EHA2025

- Oral Presentation: The MARS-R Tool for Predicting Overall Survival in Advanced Systems Mastocytosis.
- Poster Presentation: Blood-Based Proteomics Insights from the PIONEER Trial.
- Poster Presentation: Machine Learning Models in Distinguishing Indolent from Advanced SM.

• - EAACI Congress

- Flash Talk: Long-Term Analysis of AYVAKIT's Risk-Benefit Profile in Indolent SM.
- Flash Talk: Insights from the Socio-Emotional Impact of SM via the PRISM Survey.
- Poster Presentation: Updates from the Ongoing Phase 2/3 Study of Elenestinib for ISM.

For more detailed findings, please refer to the company's official publications available on their website.

Understanding Systemic Mastocytosis

Systemic mastocytosis (SM), a rare disease driven predominantly by the KIT D816V mutation, poses considerable challenges due to the uncontrolled proliferation of mast cells that lead to unpredictable and severe symptoms across multiple organ systems. While many patients present with indolent forms, advanced SM represents a serious threat, often linked with organ damage and significantly reduced survival rates.

AYVAKIT stands out as the first and only FDA-approved treatment addressing the root cause of various SM forms, marking a crucial evolution in management strategies for this complex disorder.

Conclusion

Blueprint Medicines continues to take strides in the realm of systemic mastocytosis, empowering clinicians and patients alike through groundbreaking data sharing and research collaborations. With AYVAKIT leading the charge in transforming treatment landscapes, the company reinforces its commitment to tackle significant medical challenges effectively.