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Audax Therapeutics is developing inhaled small molecule agonists of EPAC1 (Exchange Protein directly Activated by cAMP 1) for the treatment of respiratory and vascular diseases, including idiopathic pulmonary fibrosis (IPF) and pulmonary arterial hypertension (PAH). EPAC1 is a guanine nucleotide exchange factor that mediates cAMP signaling independently of protein kinase A. Activation of EPAC1 has been shown to suppress inflammation, inhibit interleukin-6 (IL-6) activity, and modulate pathways involving the NLRP3 inflammasome, TGFβ, and JAK/STAT, which are critical in fibrotic and vascular remodeling processes. These compounds are specifically formulated for inhaled delivery to target the lung tissue directly, aiming to maximize therapeutic efficacy while minimizing systemic side effects. The program is currently in the preclinical stage of development.
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