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PEPR112 is a small molecule aryl hydrocarbon receptor (AhR) antagonist being developed by Peptris Technologies for the treatment of Duchenne Muscular Dystrophy (DMD). Identified through the company's proprietary pepAI platform, PEPR112 is a repurposed, FDA-approved oral generic drug that acts as an utrophin up-regulator. This therapeutic approach is intended to offset the muscle wasting and weakness caused by dystrophin mutations by promoting the expression of utrophin, a structural and functional analog of dystrophin. In preclinical studies using the D2-Mdx mouse model, PEPR112 demonstrated dose-dependent improvements in muscle function and strength, as well as reductions in tissue inflammation and fibrosis. As an existing generic with an established safety profile in pediatric populations (ages 3 to 16), PEPR112 is positioned as a potentially accelerated and cost-effective treatment option for DMD compared to current standards of care.
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