Quoin Reports Positive QRX003 “Whole Body” Pediatric Data for Netherton Syndrome

Image Credit: © Kate - stock.adobe.com

Positive “whole body” clinical data from the ongoing Investigator Pediatric Netherton Syndrome study was announced by Quoin Pharmaceuticals today.¹ A pediatric patient using QRX003 saw an improved disease state with visually healed skin and no adverse effects after just 2 weeks of twice-daily application.

Improvements in both of the trial’s clinical endpoints, the Investigator’s Global Assessment (IGA) and pruritus, were observed. At baseline, the participant had an average itch score of 5 and an IGA score of 7, on scales of 0-10. After 2 weeks of treatment, this was reduced to 1 and 1-2, respectively. More specifically, pruritus which was previously described as “chronically debilitating” is now “highly tolerable and non-intrusive.”

Thanks to the success of QRX003, the patient has discontinued all previously used medications including antihistamines, glucocorticoids, and antivirals. Additionally, no antibiotic therapy has been needed since treatment began. The participant is no longer experiencing issues with sleep and no adverse events have been reported thus far.

CEO of Quoin, Michael Myers, MD said in a statement, “While cautioning upfront that this data is from just a single patient over a short period of time, these truly exciting and groundbreaking results provide a snapshot of the potential efficacy of QRX003 as a chronic whole-body treatment of Netherton Syndrome. After just two weeks of treatment, a transformational life change has occurred for this first patient to receive whole-body application of QRX003...We believe that these results indicate that QRX003 is directly targeting the root cause of the disease and has the potential to provide Netherton Syndrome patients with the opportunity to have lives essentially free from the ravages of this terrible disease.”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.

This whole-body data supports the initial positive results of the comparative Investigator Pediatric Study that was announced earlier this year, in which only 20% of the body surface area was treated.²

“As we continue with our plans to expand this study to include additional pediatric subjects in other countries who would also receive whole body application of QRX003, we believe that the highly encouraging nature of the results generated to date for this patient could potentially bring hope and comfort to children and families whose lives are being so dramatically impacted by the consequences of this cruel disease,” Myers concluded.¹

References

1. Quoin Pharmaceuticals Announces Highly Positive ‘Whole Body’ Clinical Data from Ongoing Pediatric Netherton Syndrome Study. News release. Globe Newswire. February 27, 2025. Accessed February 27, 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

2. Quoin Pharmaceuticals Announces Further Positive Clinical Data from Ongoing Pediatric Netherton Syndrome Study. News release. Globe Newswire. January 14, 2025. Accessed February 27, 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