Quoin Pharmaceuticals Reports Positive Interim Data from Netherton Syndrome Studies

20 December 2024
Quoin Pharmaceuticals Ltd., a specialty pharmaceutical company dedicated to rare and orphan diseases, has announced promising interim results from ongoing clinical trials involving QRX003 for Netherton Syndrome. Based in Ashburn, Virginia, Quoin is currently conducting two key studies that illustrate the potential efficacy of QRX003.

The first study involves an open-label trial where the first patient received QRX003 twice daily. After six weeks, significant improvements were noted across various clinical measures. Initially, the patient's Modified Ichthyosis Area of Severity Index (MIASI) was recorded at 18. Following the six-week treatment period, this index had dramatically decreased to 4. Furthermore, the Investigator’s Global Assessment (IGA) of disease severity improved from a classification of 'moderate' to 'mild'. The patient also reported a reduction in pruritus severity, dropping from 7 to 4 on the Worst Itch Numeric Rating Scale (WINRS). Notably, patient satisfaction was high, and no safety issues were reported during this phase.

In parallel, Quoin is conducting a 12-week Investigator Pediatric Study. Results from the first 12 days of dosing indicate a rapid enhancement in skin condition for the treated area compared to the untreated one. Initially, the IGA rated the skin as 'severe', but after the short treatment period with QRX003, it improved to 'mild-moderate'. Similar to the open-label study, there have been no adverse effects or safety concerns reported so far.

Dr. Michael Myers, CEO of Quoin Pharmaceuticals, expressed cautious optimism about these interim findings. While acknowledging the limited scope of data, he emphasized the significance of the improvements observed, particularly the reduction in MIASI and pruritus severity in the open-label study. The pediatric study's swift skin improvement also offers encouragement as the company seeks to enroll more young participants from Spain and the UK.

Quoin Pharmaceuticals is committed to developing treatments for conditions like Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Scleroderma, and Epidermolysis Bullosa. With a pipeline of four products in development, the company aims to address unmet medical needs in these rare and orphan disease areas. Their ongoing efforts underscore a commitment to providing safe and effective therapies for patients, families, and healthcare providers.

These developments reflect Quoin's dedication to advancing solutions for challenging medical conditions. The company's work on QRX003 is a significant step toward offering a hopeful alternative for individuals affected by Netherton Syndrome, and the preliminary data from these studies offer a promising outlook for the future.

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