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INN-04-01 is a small molecule Janus kinase (JAK) inhibitor being developed by Innox Pharma for the treatment of inflammatory myopathies, including dermatomyositis and polymyositis. The drug is a repositioned formulation of tofacitinib, which functions by inhibiting the JAK-STAT signaling pathway. By blocking the activity of Janus kinases (primarily JAK1 and JAK3), INN-04-01 modulates the signaling of various pro-inflammatory cytokines involved in the pathogenesis of autoimmune muscle and skin diseases. It is currently in Phase 2 clinical development (e.g., NCT05241600) as a potential therapy for patients with refractory inflammatory myopathies where current standard-of-care treatments are often inadequate.
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