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U301 is an investigational, next-generation formulation of treprostinil, a synthetic analog of prostacyclin (PGI2). Developed by United Therapeutics, U301 is specifically being evaluated for the treatment of pulmonary hypertension associated with heart failure with preserved ejection fraction (PH-HFpEF). The drug acts as a potent vasodilator of both pulmonary and systemic arterial vascular beds and inhibits platelet aggregation. By binding to the prostacyclin receptor (IP receptor), it increases intracellular cyclic adenosine monophosphate (cAMP), leading to smooth muscle relaxation. U301 is designed to provide a more convenient or optimized delivery profile compared to existing treprostinil formulations, such as Remodulin, particularly for the PH-HFpEF patient population where traditional vasodilators have historically shown limited efficacy.
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