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FDA Clears Quoin Pharmaceuticals to Employ Teens for Netherton Syndrome Clinical Trials

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Teens to be included in both placebo and open label studies for QRX003, a potential treatment for Netherton syndrome.

Medical research and clinical trials. Image Credit: Adobe Stock Images/The Gentleman

Image Credit: Adobe Stock Images/The Gentleman

Today, Quoin Pharmaceuticals announced that the FDA has given approval to include teenagers aged 14 years and older in two ongoing clinical trials for QRX003, a potential treatment for Netherton syndrome (NS). According to a press release, this marks the first time that teen patients with NS will have the opportunity to participate in clinical trials under an Investigational New Drug Application (IND). Additionally, the studies will accommodate teens currently on off-label systemic therapy, with the focus of generating comprehensive data for QRX003's development.1

“We are very pleased to announce this exciting development, not just for Quoin’s clinical program, but for the Netherton community as a whole,” said Michael Myers, BSc, PhD, CEO, Quoin, in a press release. “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.”

At the end of last year, the FDA provided clearance for numerous protocol amendments for the QRX003 clinical trials. Resulting from positive clinical data reports and a superior safety profile, Quoin stated that implementation of these protocol amendments has the potential to provide even more full-bodied data, offering a faster approval.2

“We are extremely pleased to announce clearance of our clinical trial optimization plan for QRX003 in NS. Armed with positive initial data and a clean safety profile to date, we believe that these protocol amendments could ultimately result in the generation of a highly compelling data set which could support regulatory filings and approval for QRX003 as the first treatment for this terrible disease. These latest developments underscore Quoin’s continued commitment to delivering a safe and effective treatment for this very underserved patient population,” said Myers, in an additional release.

QRX003 was first cleared for testing towards an IND application in April 2022. According to the company, it is a topical lotion, formulated with a proprietary delivery technology, and contains a broad-spectrum serine protease inhibitor, with a mechanism of action that is intended to perform the function of a specific protein, called LEKTI.3

NS is a rare disorder that impacts the skin, hair, and immune system, with symptoms typically presenting at birth, including red and scaly skin; outbreaks of rashes; thin and fragile hair; and immune reactions, including hay fever, asthma, itchy skin, and eczema. Babies diagnosed with the condition may grow slowly and have issues gaining weight.4

“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,” finished Myers, in the latest release.

References

1. Quoin Pharmaceuticals Announces FDA Clearance to Recruit Teen Subjects into Both Ongoing Netherton Syndrome Clinical Studies. GlobeNewswire. March 4, 2024. Accessed March 4, 2024. https://www.globenewswire.com/news-release/2024/03/04/2839532/0/en/Quoin-Pharmaceuticals-Announces-FDA-Clearance-to-Recruit-Teen-Subjects-into-Both-Ongoing-Netherton-Syndrome-Clinical-Studies.html

2. Quoin Pharmaceuticals Announces FDA Clearance of Clinical Optimization Plan for QRX003 for Netherton Syndrome. Quoin Pharma. December 13, 2023. Accessed March 4, 2024. https://investors.quoinpharma.com/news-releases/news-release-details/quoin-pharmaceuticals-announces-fda-clearance-clinical

3. Quoin Will Initiate Clinical Testing in 1H 2022. Quoin Pharma. April 25, 2022. Accessed March 4, 2024. https://investors.quoinpharma.com/news-releases/news-release-details/quoin-pharmaceuticals-announces-fda-clearance-ind-its-lead-asset

4. National Center for Advancing Translational Sciences. Netherton Syndrome. Webpage. Updated February 2024. Accessed March 4, 2024. https://rarediseases.info.nih.gov/diseases/7182/netherton-syndrome

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