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Isocyclosporin (DFL24498) is a small molecule dual inhibitor being developed by Dompé Farmaceutici S.p.A. for the treatment of atopic keratoconjunctivitis (AKC). It acts through a novel dual mechanism of action, targeting both the transient receptor potential cation channel mucolipin subfamily member 2 (TRPML2) and calcineurin. TRPML2 is a key mediator of immune cell activation, while calcineurin is a phosphatase that regulates T-cell mediated immune responses. By inhibiting these two critical pathways, isocyclosporin aims to reduce ocular inflammation. The drug is formulated as an eye drop solution designed for enhanced ocular solubility, which improves its bioavailability, distribution, and tolerability within the eye. It is currently being evaluated in Phase 3 clinical trials to demonstrate its efficacy and safety in improving the signs and symptoms of AKC compared to a vehicle control.
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