QRX003 Shows Promise in Pediatric Patients with Netherton Syndrome, Quoin Announces

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More data has been announced from Quoin Pharmaceuticals regarding positive results from the Investigator Pediatric Study QRX003 trial for the treatment of Netherton Syndrome.¹ Just last week, Quoin shared promising interim results from its ongoing, open-label study in patients 14 years of age and older.²

The comparative study saw significant improvements in skin appearance based on all clinical endpoints versus the area not treated with QRX003. At the midpoint of the trial, disease severity improved from “Severe” at baseline to “Mild,” according to the Investigator’s Global Assessment (IGA). These results occurred after 6 weeks of testing in which patients used QRX003 twice a day. A change in IGA to the “Mild-Moderate” distinction was noted as early as 12 days of dosing.

With these results, investigators plan to transition to a whole-body treatment evaluation, which aims to generate more real-world data. QRX003 will be applied to patients’ whole body surface area rather than the 20% surface area that was tested in this initial study. In terms of the therapy’s safety, no adverse events have been reported thus far, which is consistent with Quoin’s existing trials for Netherton Syndrome.

In a press release, Quoin CEO, Michael Myers, MD, said, “The significant improvement in the Investigator’s Global Assessment (IGA) from Severe at Baseline to Mild-Moderate after 12 days of dosing, and now to Mild after 6 weeks of dosing is truly remarkable, and we believe that the investigator’s decision to transition the subject to “whole body” treatment is an exciting step forward that will provide even more representative data on the potential safety and efficacy of QRX003 in Netherton Syndrome patients.”¹

Netherton Syndrome is a severe genetic skin condition caused by mutations in the SPINK5 gene, leading to excessive skin shedding and compromised skin barrier function. Current treatment options are limited and primarily focus on symptomatic relief.

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. If successful, QRX003 could become the first FDA-approved treatment for Netherton Syndrome, offering new hope to patients and their families.

“We look forward to expanding this study to include additional pediatric subjects in other countries, and we believe each of these subjects may have an opportunity to move directly to whole body testing based on the positive nature of the results generated to date,” Myers added. “With this study well underway and the adult “whole body” study cleared to proceed by the FDA, we are beginning to assemble what we hope will be very compelling clinical evidence that supports the potential of QRX003 as a safe and effective treatment for Netherton Syndrome patients.”¹

References

1. Quoin Pharmaceuticals Announces Further Positive Clinical Data from Ongoing Pediatric Netherton Syndrome Study. News release. Globe Newswire. January 14, 2025. Accessed January 14, 2025. https://www.globenewswire.com/news-release/2025/01/14/3009328/0/en/Quoin-Pharmaceuticals-Announces-Further-Positive-Clinical-Data-from-Ongoing-Pediatric-Netherton-Syndrome-Study.html

2. Quoin Pharmaceuticals Announces Additional Positive Interim Data from Ongoing Open-Label Netherton Syndrome Clinical Study. News Release. Globe Newswire. January 6, 2025. Accessed January 14, 2025. https://www.globenewswire.com/news-release/2025/01/06/3004620/0/en/Quoin-Pharmaceuticals-Announces-Additional-Positive-Interim-Data-from-Ongoing-Open-Label-Netherton-Syndrome-Clinical-Study.html