Clinical TrialJun 16, 2026, 08:32 AM
Quoin Pharmaceuticals QRX003 Shows Positive Results in Pediatric Netherton Syndrome
AI Summary
Quoin Pharmaceuticals announced positive clinical results from its pediatric Netherton Syndrome (NS) Compassionate Use program for QRX003. Four of six enrolled patients, all under 10 years old, showed 'Improved' or 'Significantly Improved' status across key clinical endpoints. Notably, one patient achieved a full 5-grade improvement in IGA and remains clear at 15 months, while a 6-month-old infant saw significant reduction in emollient use and restored neutrophil levels after just 3 weeks. No treatment-related adverse events have been reported, and additional patients are enrolling.
Key Highlights
- 4 of 6 pediatric Netherton Syndrome patients showed 'Improved' or 'Significantly Improved' status with QRX003.
- One patient achieved a 5-grade improvement in Investigator's Global Assessment (IGA) from severe to clear, remaining clear at 15 months.
- Two patients saw at least a 4-grade improvement in pruritus, with one's pruritus completely eliminated.
- A 6-month-old infant reduced emollient use from 12 times/day to almost none after 3 weeks of QRX003 treatment.
- The 6-month-old infant's neutrophil levels were fully restored to normal after 3 weeks of QRX003 treatment.
- No treatment-related adverse events were reported for any of the six patients in the program.
- Additional pediatric patients are enrolling in the compassionate use program in June and September.
- Quoin plans a comprehensive data release, including from ongoing Phase 2 studies, in the coming months.
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