Quoin Pharmaceuticals Gets FDA Approval for Second QRX003 Netherton Syndrome Study

Quoin Pharmaceuticals Ltd., a specialty pharmaceutical company focusing on rare and orphan diseases, has received FDA clearance to commence a new pivotal clinical study, CL-QRX003-002, aimed at treating Netherton Syndrome (NS) using their lead product, QRX003. This topical lotion, featuring a broad-spectrum serine protease inhibitor, is designed to counteract the excessive skin shedding linked to NS by significantly reducing the skin's hyperactivity of kallikreins. Earlier clinical data have shown that QRX003 promotes rapid and sustained skin healing, alleviates debilitating itching, and minimizes sleep disturbances following twice-daily application.

Building on promising results from previous studies where QRX003 was applied to 20% of each subject’s body surface area, Study CL-QRX003-002 will explore the application of QRX003 on up to 80% of the body surface area of 12-15 subjects. This increased application is intended to gather extensive safety and efficacy data, reflecting how the product would be used post-approval. Participants will continue with their off-label systemic therapy throughout the study. This study complements another pivotal study, CL-QRX003-003, conducted at Northwestern University by Dr. Amy Paller, where QRX003 is tested without the adjunct therapy.

The combination of data from these two studies is anticipated to furnish QRX003 with a comprehensive opportunity for regulatory approval both as a standalone treatment and in conjunction with other therapies. Quoin aims to recruit between 24 to 30 subjects across six U.S. and six international sites. Five U.S. sites are currently operational, and Quoin plans to open the sixth by June. International sites are also nearing readiness, with cohorts of participants prepared for enrollment, supporting accelerated recruitment. This strategic approach could enable Quoin to complete recruitment by the end of Q1 2026, with plans to file a New Drug Application (NDA) later that year.

Dr. Michael Myers, Quoin's CEO, highlighted the significance of FDA clearance for this study, stating that Quoin is uniquely positioned as the sole company conducting two late-stage whole-body pivotal clinical studies for NS. He expressed optimism that the combined data from Studies CL-QRX003-002 and CL-QRX003-003 will provide a robust safety and efficacy profile for QRX003, potentially paving the way for its approval as a groundbreaking treatment for NS.

Quoin Pharmaceuticals Ltd. is dedicated to developing therapies for rare and orphan diseases, including Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Scleroderma, and Epidermolysis Bullosa. Their pipeline includes four innovative products that could address numerous unmet medical needs within these indications.