ASHBURN, Va. — Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) , a specialty pharmaceutical company focused on rare and orphan diseases, today announced clearance from the U.S. Food and Drug Administration (FDA) to recruit teen subjects aged 14 years and older into its two ongoing clinical trials for QRX003, which is being developed as a potential treatment for Netherton Syndrome (NS). Both trials are being conducted under Quoin’s open Investigational New Drug Application (IND) for QRX003.
Dr. Michael Myers, Chief Executive Officer of Quoin, said, “We are very pleased to announce this exciting development, not just for Quoin’s clinical program, but for the Netherton community as a whole. We are frequently petitioned by parents and caregivers that teen subjects be allowed to participate in our clinical studies, given the severity of the disease and the absolute dearth of viable treatment options. Today, it is our privilege to announce that we have FDA clearance to do just that.
“This FDA clearance represents the very first time that non-adult Netherton subjects will be tested in clinical studies conducted under an open-IND and, as such, represents a very significant and important step forward for members of this community. Significantly, teens who are currently receiving off-label systemic therapy will be eligible to participate in our open-label study, while those who are not receiving such therapy may be recruited into the placebo controlled blinded study. This important feature not only widens the pool of eligible subjects but also eliminates the need for parents or caregivers to make difficult decisions about treatments these patients and loved ones are receiving. The inclusion of this patient population in our studies will be, we believe, a critical component of the development of a robust data set that could result in regulatory approval with a broad label as QRX003 is being tested both as monotherapy and in conjunction with off-label treatments.
“Quoin remains fully committed to the Netherton community, their families and treating physicians, and we are proud to be the only company actively dosing Netherton subjects in clinical studies conducted under an open IND. We look forward to continuing our mission to deliver what we hope will be the first safe and effective treatment for this debilitating disease,” concluded Dr. Myers
About Netherton Syndrome
Netherton Syndrome, a form of Ichthyosis, is a rare, hereditary skin disorder caused by a mutation in the SPINK5 gene (serine protease inhibitor, Kazal Type 5) that leads to severe skin barrier defects and recurring infections, as well as a pronounced predisposition to allergies, asthma, and eczema. Patients also often suffer from severe dehydration, chronic skin inflammation and stunted growth.
Currently, there is no cure for Netherton Syndrome, nor are there any approved therapeutic treatments.
About QRX003
QRX003 is a topical lotion, formulated with a proprietary delivery technology, and contains a broad- spectrum serine protease inhibitor, whose mechanism of action is intended to perform the function of a specific protein, called LEKTI. The absence of LEKTI in Netherton patients leads to excessive skin shedding resulting in a highly porous and compromised skin barrier. QRX003 is designed to lead to a more normalized skin shedding process and the formation of a stronger and more effective skin barrier.
About Quoin Pharmaceuticals Ltd.
Quoin Pharmaceuticals Ltd. is an emerging specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases. We are committed to addressing unmet medical needs for patients, their families, communities and care teams. Quoin’s innovative pipeline comprises three products in development that collectively have the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Epidermolysis Bullosa and others.
Contacts
Quoin Pharmaceuticals Ltd.
Michael Myers, Ph.D., CEO
[email protected]
Investor Relations
PCG Advisory
Stephanie Prince
[email protected]
(646) 863-6341