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- Rectify Pharmaceuticals presented new preclinical data at EASL 2026 showing RTY-406 significantly improved disease-relevant endpoints in a mouse model of primary sclerosing cholangitis and demonstrated proof of mechanism in non-human primates. - RTY-406 is a dual-acting ABCB4/BSEP positive functional modulator that targets abnormal bile composition and impaired bile flow, two core pathophysiological drivers of PSC. - The drug candidate is currently in Phase 1 clinical development as a potential first-in-class disease-modifying therapy for PSC patients, a devastating disease with no approved therapies addressing underlying biology. - In preclinical studies, RTY-406 reduced markers of cholestasis, fibrosis, and inflammation while demonstrating dose-dependent improvements in liver function markers without evidence of liver toxicity.
- DEBRA Research gGmbH and the University Medical Center Groningen have established a dedicated clinical trial site for Epidermolysis bullosa (EB), strengthening research infrastructure for this rare genetic skin disorder. - The collaboration will enhance clinical trial capacity in a region with recognized expertise in EB diagnosis and care, led by Principal Investigator Dr. Marieke C. Bolling. - DEBRA Research is supporting the international COSEB project to standardize outcome measurements across EB studies, ensuring future trials generate robust and comparable data. - The partnership aims to accelerate therapeutic innovation for the global EB community by improving trial efficiency and supporting both company-sponsored and investigator-initiated studies.
