Cellect(QNRX) Newsfilter·2025-02-27 12:30Core Viewpoint - Quoin Pharmaceuticals has announced highly positive clinical data from its ongoing Investigator Pediatric Netherton Syndrome study, showing significant improvements in patients' disease state after two weeks of treatment with QRX003, a topical lotion designed for rare skin disorders [1][4]. Clinical Data Summary - The study reported dramatic improvements in both key clinical endpoints: Investigator's Global Assessment (IGA) and Pruritus, with scores significantly reduced from baseline after two weeks of treatment [2]. - For the first pediatric patient, the IGA score improved from 7 at baseline to between 1-2 after treatment, indicating almost complete elimination of visual disease effects [4]. - The Pruritus score decreased from 5 to 1, reflecting a transition from "chronically debilitating itch" to a "highly tolerable and non-intrusive" level [4]. Patient Outcomes - Following treatment with QRX003, the patient has discontinued all previously required medications, including antihistamines, glucocorticoids, and antivirals, and has not needed antibiotics since treatment began [3][4]. - The patient is now experiencing zero nightly sleep disturbances for the first time, further indicating the treatment's potential efficacy [3][4]. Product Information - QRX003 is a topical lotion formulated with a proprietary delivery technology that contains a broad-spectrum serine protease inhibitor, aimed at normalizing skin-shedding processes and strengthening the skin barrier [7]. - Netherton Syndrome, the condition QRX003 targets, is a rare hereditary skin disorder caused by a mutation in the SPINK5 gene, leading to severe skin barrier defects and other complications [5][6]. Company Overview - Quoin Pharmaceuticals is a clinical-stage specialty pharmaceutical company focused on developing treatments for rare and orphan diseases, with a pipeline that includes products targeting various conditions, including Netherton Syndrome [8].