Quoin Pharmaceuticals Ltd. announced that the first patient has been dosed in its ongoing clinical trial evaluating the safety and efficacy of QRX003 as a treatment for Netherton Syndrome. Quoin's first clinical trial is a randomized, double blinded, vehicle-controlled study which is being conducted under a U.S. Investigational New Drug (IND) Application and is assessing two different doses of QRX003 topical lotion versus a vehicle lotion in Netherton patients. The test materials are applied once daily over a twelve-week period, to pre-designated areas of the patient's body.

Based on discussions with the U.S. Food and Drug Administration (FDA), a number of different clinical endpoints will be assessed in the study. In October 2022, Quoin announced it plans to initiate a second study assessing its QRX003 topical lotion in Netherton patients who are currently receiving off-label treatment including systemic therapy for symptomatic relief. This second study, which has now been cleared by the FDA to initiate clinical testing, is an open-label, non-placebo controlled trial that will evaluate 10 Netherton patients over a twelve week period.

The study, which will run concurrently with Quoin's ongoing clinical trial, will be conducted at the same sites and by the same clinical investigators as the first study, thereby optimizing the potential for a rapid recruitment of patients. The active ingredient in QRX003 is a broad-spectrum serine protease inhibitor, whose mechanism of action is intended to down-regulate the hyperactivity of skin kallikreins, leading to a more normalized rate of skin shedding. If proven to be safe and effective, long term daily application of QRX003 could lead to the development of a more normally functioning skin barrier and a significant improvement in the quality of life of Netherton patients.