Quoin Pharmaceuticals Gets FDA Approval for QRX003 Netherton Syndrome Trial

Quoin Pharmaceuticals Ltd., a company specializing in rare and orphan diseases, has received FDA approval to begin a new clinical study focused on Netherton Syndrome (NS) for its product QRX003. This topical lotion is formulated with a broad-spectrum serine protease inhibitor, which targets kallikreins in the skin, enzymes believed to cause excessive skin shedding seen in Netherton Syndrome.

The forthcoming study will be spearheaded by Dr. Amy Paller from Northwestern University. The study aims to enroll up to eight participants, who will apply QRX003 twice daily to more than 80% of their body surface area over a 12-week span. This contrasts with earlier studies by Quoin, where QRX003 was administered to only about 20% of the body surface area, focusing on areas like the arms and lower legs. The new study's design seeks to replicate real-world usage conditions if QRX003 gains approval, marking its most extensive application in a clinical environment to date. The data generated will contribute to a robust data package, potentially supporting regulatory approval for QRX003 as a treatment for NS.

Dr. Amy Paller emphasized the treatment's potential, stating that targeting kallikreins is an optimal strategy for addressing the symptoms of Netherton Syndrome, short of gene therapy solutions. The company's CEO, Dr. Michael Myers, expressed enthusiasm about the FDA's clearance, highlighting that this study represents a significant step forward in the development of QRX003. He noted that the study's real-world setting will play a crucial role in future filings. This is Quoin's third study concerning NS conducted under their open Investigational New Drug application, demonstrating their dedication to developing the most comprehensive data set possible to support the approval of an effective treatment for this challenging condition.

Quoin Pharmaceuticals Ltd. operates as a clinical-stage specialty pharmaceutical company, focusing on therapeutic products for rare and orphan diseases. They are dedicated to meeting unmet medical needs of patients and supporting their families, communities, and care teams. The company's pipeline consists of four products in development, designed to address a variety of rare conditions including Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Scleroderma, and Epidermolysis Bullosa.

By pushing forward with this extensive study, Quoin Pharmaceuticals aims to provide a viable treatment option for those suffering from Netherton Syndrome. If successful, QRX003 could become the first approved treatment specifically targeting this condition, potentially improving the quality of life for patients affected by this debilitating disease. In collaboration with Dr. Paller and her team, Quoin aspires to gather critical data that will support their mission to provide groundbreaking treatments for rare diseases.

The new study marks an important stride in Quoin’s journey to deliver effective solutions for rare diseases, reinforcing its commitment to innovation and patient care. With FDA approval now secured, the company is poised to further its research efforts and contribute to the field of rare disease treatment with potentially life-changing impacts for those living with Netherton Syndrome and other related conditions.