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EPAC1 partial agonists are small molecule compounds developed by Audax Therapeutics for the treatment of Pulmonary Arterial Hypertension (PAH). These agents function as partial agonists of the Exchange Protein directly Activated by cAMP 1 (EPAC1), a protein that mediates various cellular responses to the second messenger cAMP. By partially activating EPAC1, these compounds are designed to modulate several key signaling pathways implicated in the pathogenesis of PAH, including the NLRP3 inflammasome, Transforming Growth Factor beta (TGFβ), and the JAK/STAT pathway. This multi-targeted downstream modulation is intended to reduce inflammation, fibrosis, and vascular remodeling. The program is currently in the hit-to-lead phase of preclinical development.
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