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pp-137

Tablets

Dossier type
CTD
Dossier status
Under development
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Hypertrophic cardiomyopathy (obstructive and non-obstructive HCM) Clinical stage: Phase 2 Modality: Small molecule Mechanism / Target: Selective cardiac β-myosin heavy chain (MYH7) inhibitor — reduces hypercontractility of cardiomyocytes by decreasing actin-myosin cross-bridge formation Route / form: Oral tablet (once daily) Differentiation: Potentially wider therapeutic window for LVEF vs mavacamten; designed to avoid systolic dysfunction; next-generation profile in validated HCM target class
Manufacturer #39169

Clinical-stage muscle disease company developing precision myosin modulators. Targets skeletal and cardiac muscle diseases driven by abnormal sarcomere function. Wholly owned pipeline including Phase 3 asset for rare muscular dystrophy.
 

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