The U.S. Food and Drug Administration (FDA) has cleared a second Netherton Syndrome (NS) pivotal clinical study from Quoin Pharmaceuticals Ltd.
Quoin’s lead product, QRX003, is a topical lotion formulated with a broad-spectrum serine protease inhibitor that has been shown to significantly downregulate the hyperactivity of the kallikreins in the skin responsible for the excessive skin shedding associated with NS.
Clinical data from Quoin’s ongoing studies has demonstrated clear evidence of rapid, prolonged and almost complete skin healing following twice-daily application of QRX003 to the treatment areas along with the almost complete elimination of key symptoms such as chronic, debilitating pruritus and has facilitated zero nightly sleep disturbances.
Study CL-QRX003-002 builds off of clinical data generated in Quoin’s earlier stage open-label study where QRX003 was applied to the arms and lower legs or approximately 20% of each subject’s body surface area (BSA).
In the new pivotal study, QRX003 will be applied to approximately 12-15 subjects twice daily to at least 80% of each of the subjects’ BSA thereby generating substantially more safety and efficacy data per subject and will match how QRX003 is intended to be used if regulatory approval is obtained.
All subjects recruited into this study are currently receiving off-label systemic therapy and will continue to remain on this treatment for the duration of the study.
Study CL-QRX003-002 complements Quoin’s other pivotal study, CL-QRX003-003, which is being conducted by Amy Paller, MD, of Northwestern University in Chicago, IL, where QRX003 is being administered as monotherapy and subjects do not receive off-label systemic therapy. Quoin anticipates that combined both studies will recruit between 24-30 subjects in six U.S. sites and up to six international sites.
Five of the U.S. sites are already open and the sixth is expected to be opened in June. All international sites are at an advanced stage of preparation for opening.
Each of the U.S. and international sites has available cohorts of subjects that are ready to participate in both studies facilitating a rapid pace of recruitment which could enable Quoin to achieve its target of fully completing recruitment before the end of Q1 2026 and to file an NDA for regulatory approval later in the year.
“With FDA clearance to conduct this study, Quoin is the only company conducting two late-stage whole-body pivotal clinical studies in Netherton Syndrome patients,” says Quoin CEO Dr. Michael Myers in a news release. “We believe that the combined data generated from Study CL-QRX003-003, where QRX003 is being administered as monotherapy and this study, CL-QRX003-002, where QRX003 is being tested in conjunction with off-label systemic therapy, has the potential to generate a highly comprehensive safety and efficacy data package that could support the regulatory approval of a treatment for this dreadful disease.”
