ASHBURN, Va., June 16, 2026
Quoin Pharmaceuticals announced a positive clinical update from its ongoing pediatric Netherton Syndrome (NS) Compassionate Use Program, reporting encouraging treatment responses among children receiving its investigational topical therapy QRX003. The program currently includes six pediatric patients under the age of 10, with the youngest participant being only six months old. According to the company, four of the six enrolled patients were classified as either “Improved” or “Significantly Improved” across multiple key clinical endpoints. Importantly, no treatment-related adverse events have been reported among any participants, further supporting the therapy’s favorable safety profile as development progresses toward broader clinical evaluation.
QRX003 Demonstrates Meaningful Clinical Improvements in Children
Among the treatment responders, three patients achieved at least a one-grade improvement on the Investigator’s Global Assessment (IGA) scale, which measures disease severity from clear (0) to severe (4). One patient experienced a remarkable transformation, improving from severe disease at baseline to completely clear skin after nine months of treatment, with the benefit maintained through 15 months of therapy. Additional improvements were observed in pruritus (itching), a debilitating symptom of Netherton Syndrome. Two responding patients achieved at least a four-point reduction in itch severity, while one experienced complete elimination of pruritus. These results suggest QRX003 may have the potential to significantly improve both skin health and quality of life for affected children living with this rare genetic disorder.
Youngest Patient Shows Early Signs of Benefit After Three Weeks
One of the most notable observations came from the program’s youngest participant, a six-month-old infant who had received only three weeks of treatment at the time of assessment. Prior to enrollment, the infant required application of moisturizing emollients every two hours, totaling approximately twelve applications per day, due to severe skin barrier dysfunction. Following treatment with QRX003, the child’s skin condition improved to the extent that almost no emollient use was required on treated areas. Additionally, the infant entered the study with critically low neutrophil levels, increasing vulnerability to infections. After three weeks of treatment, neutrophil levels reportedly returned to normal ranges, providing an additional indication of potential therapeutic benefit. While longer-term follow-up is still pending, these early findings have generated significant interest within the ongoing program.
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Expanding Clinical Evidence Supports Future Development Plans
Netherton Syndrome is a rare inherited skin disorder caused by mutations in the SPINK5 gene, leading to the absence of the protective protein LEKTI and resulting in severe skin barrier defects, chronic inflammation, recurrent infections, and intense itching. There are currently no approved therapies available for the condition. QRX003 is designed to replace the function of LEKTI through a proprietary topical formulation containing a broad-spectrum serine protease inhibitor, helping normalize skin shedding and strengthen the skin barrier. Quoin has already enrolled an additional pediatric patient in June and expects two more children to enter the program by September, further expanding the dataset. The company plans to release a comprehensive clinical update from both the Compassionate Use Program and its ongoing Phase 2 studies in the coming months. With encouraging efficacy signals, a favorable safety profile, and growing patient experience, QRX003 continues to advance as a potential first approved treatment option for individuals living with Netherton Syndrome.
Source: Quoin Pharmaceuticals press release
