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IKT-001 is an investigational small molecule prodrug of imatinib mesylate being developed by Inhibikase Therapeutics for the treatment of Pulmonary Arterial Hypertension (PAH). It functions as a tyrosine kinase inhibitor targeting platelet-derived growth factor receptors (PDGFRα and PDGFRβ) and c-KIT, which are type III receptor tyrosine kinases involved in the pathological vascular remodeling and aberrant cellular signaling characteristic of PAH. IKT-001 is specifically engineered to improve the gastrointestinal tolerability profile compared to standard imatinib, potentially allowing for the delivery of therapeutic doses required to modify the course of the disease. The FDA has indicated that IKT-001 can be considered a New Molecular Entity (NME) and is eligible for orphan drug exclusivity. The drug is currently advancing into a pivotal Phase 3 clinical evaluation known as the IMPROVE-PAH trial.
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