Quoin Announces Positive QRX003 Data for Pediatric Peeling Skin Syndrome

Image Credit: © Dr AS Vidya, Kerala, India

Today, Quoin Pharmaceuticals shared positive investigator data from its ongoing pediatric study of QRX003 for Peeling Skin Syndrome.¹ Overall skin healing was observed in a single patient after just 12 weeks of treatment. The therapy has been well tolerated by the patient, with no adverse events. With this positive data, the patient will continue being treated with QRX003 for at least up to 24 weeks.

Researchers utilized the Modified Ichthyosis Area of Severity Index (M-IASI), the Investigator’s Global Assessment (IGA), and the Children’s Dermatology Life Quality Index (CDQLI) to evaluate efficacy. In all measurements, lower scores indicate improvement. The M-IASI score dropped significantly from 36 at baseline to 12 at the conclusion of the trial. The IGA score reduced from 4, indicating severe disease, to 2, indicating mild disease. CDQLI score, which assesses symptoms, leisure and activities, sleep, school, and personal relationships, improved from 19 to 11.

In a press release, Quoin CEO, Michael Myers, MD, said, “We are very pleased to announce such positive initial data across a number of clinical endpoints for the pediatric subject in this study, which we believe may be the first formal clinical study for this disease. The two grade improvement, which is accepted as being clinically meaningful in both the Investigator’s Global Assessment (IGA) (severe to mild) and the M-IASI (moderate to clear) after 12 weeks of QRX003 application is very encouraging. We fully support the investigator’s decision to continue treatment. In addition, the validated CDQLI questionnaire results to date indicate that the pediatric subject in the study is experiencing a distinct positive improvement in their quality of life as a result of ongoing treatment with QRX003.”¹

Generalized inflammatory peeling skin syndrome is a rare, autosomal recessive condition due to disease-causing variants of the corneodesmosin gene. As superficial layers of the epidermis shed excessively, patients experience chronic pain and pruritus. There are currently no therapies approved by the US Food and Drug Administration (FDA) for this disease, leading patients to turn to over-the-counter emollient treatments. Quoin filed a US patent application for novel topical therapies and rare disease treatment in March.²

QRX003 is a topical lotion designed to address the underlying pathology of Netherton Syndrome by inhibiting kallikreins, serine proteases that contribute to the excessive skin shedding characteristic of the disease. It is also being developed for the treatment of Netherton Syndrome, which has demonstrated positive whole-body results in pediatric patients, according to a press release from Quoin earlier this year.³

“Consistent with what we are observing in our Netherton Syndrome studies, QRX003 is well tolerated with no adverse events reported. We look forward to expanding this study to include additional pediatric subjects in other countries and to advancing the clinical development of the product for this disease. There is currently no approved treatment or cure for peeling skin syndrome and there are no clinical studies listed on clinicaltrials.gov as actively recruiting and dosing subjects, thus presenting a further opportunity for Quoin to potentially achieve the first regulatory approval for another rare genetic disease. We are also continuing to advance QRX003 in our late-stage Netherton Syndrome clinical studies,” Myers said in a statement.¹

References

1. Quoin Pharmaceuticals Announces Initial Positive Clinical Data for QRX003 from Pediatric Peeling Skin Syndrome Study. News release. Globe Newswire. Published May 14, 2025. Accessed May 14, 2025. https://www.globenewswire.com/news-release/2025/05/14/3081028/0/en/Quoin-Pharmaceuticals-Announces-Initial-Positive-Clinical-Data-for-QRX003-from-Pediatric-Peeling-Skin-Syndrome-Study.html

2. Quoin Pharmaceuticals Files U.S. Patent Application for Novel Topical Formulations to Treat Netherton Syndrome and Other Skin Diseases. News release. Globe Newswire. Published March 25, 2025. Accessed May 14, 2025. https://www.globenewswire.com/news-release/2025/03/25/3048611/0/en/Quoin-Pharmaceuticals-Files-U-S-Patent-Application-for-Novel-Topical-Formulations-to-Treat-Netherton-Syndrome-and-Other-Skin-Diseases.html

3. Quoin Pharmaceuticals Announces Highly Positive ‘Whole Body’ Clinical Data from Ongoing Pediatric Netherton Syndrome Study. News release. Globe Newswire. February 27, 2025. Accessed May 14, 2025. https://www.globenewswire.com/news-release/2025/02/27/3033744/0/en/Quoin-Pharmaceuticals-Announces-Highly-Positive-Whole-Body-Clinical-Data-from-Ongoing-Pediatric-Netherton-Syndrome-Study.html