MedPath

Elenestinib

Generic Name
Elenestinib
Drug Type
Small Molecule
Chemical Formula
C27H29FN10O
CAS Number
2505078-08-8
Unique Ingredient Identifier
6FBM4CP6RK
Background

Elenestinib is a selective KIT inhibitor.

Blueprint Medicines Announces Positive BLU-808 Phase 1 Data and 2025 Growth Strategy

• Blueprint Medicines projects a $4 billion peak revenue opportunity for its systemic mastocytosis (SM) franchise, driven by AYVAKIT's success and updated SM prevalence estimates. • Phase 1 trial results for BLU-808, an oral wild type KIT inhibitor, showed a wide therapeutic window and significant tryptase reductions, supporting its potential in allergic and inflammatory diseases. • The company anticipates achieving $2 billion in AYVAKIT revenue by 2030 and is initiating proof-of-concept studies for BLU-808 in various allergic and inflammatory conditions. • Blueprint Medicines has initiated a Phase 3 trial of elenestinib in indolent systemic mastocytosis (ISM) and is advancing CDK2 and CDK4 targeted protein degraders for breast cancer.

Avapritinib Demonstrates Long-Term Benefits in Advanced Systemic Mastocytosis

• Avapritinib (AYVAKIT) shows significant survival benefits in treatment-naïve patients with advanced systemic mastocytosis (SM) compared to midostaurin in real-world outcomes. • Data from the PATHFINDER trial indicates that avapritinib leads to sustained improvements in bone density for advanced SM patients with low bone mass. • Ultra-sensitive KIT testing identifies previously undetected KIT mutations in indolent systemic mastocytosis (ISM) patients, suggesting a higher disease prevalence. • A predictive model using machine learning distinguishes between advanced SM and ISM with high accuracy, validated by an independent dataset from Dana-Farber Cancer Institute.
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