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CS1 is an innovative formulation of valproic acid (VPA) that functions as an HDAC (histone deacetylase) inhibitor working through epigenetic modulation. It is being developed as a treatment for pulmonary arterial hypertension (PAH), a rare cardiovascular disease. In preclinical cardiovascular disease models, CS1 has shown disease-modifying potential through reverse pathological remodeling, as well as anti-fibrotic, anti-inflammatory, pulmonary pressure-reducing, and anti-thrombotic effects. The drug's unique efficacy profile aligns with the underlying mechanisms of PAH, positioning it to address the unmet need for more effective treatment options. CS1 has received Orphan Drug Designation from the FDA in March 2020 and Orphan Medicinal Product Designation from the European Commission in August 2024.
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