FDA Gives Go-Ahead on Quoin Pharmaceuticals’ QRX003 Netherton Syndrome Study

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Quoin Pharmaceuticals announced today that the US Food and Drug Administration (FDA) has granted clearance to initiate a new clinical study for QRX003, a promising topical therapy for Netherton Syndrome.¹

The trial, led by Amy Paller, MD, at Northwestern University, aims to generate real-world data by administering QRX003 to a broader body surface area (BSA) than in previous studies.

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 designed to address the underlying pathology of NS by inhibiting kallikreins, serine proteases that contribute to the excessive skin shedding characteristic of the disease.

This upcoming study marks Quoin’s third clinical trial under its open Investigational New Drug (IND) application for QRX003. Unlike earlier trials, which restricted the lotion’s application to about 20% of the body, the new study will involve twice-daily application to over 80% of the participants’ BSA. This more extensive use aligns with potential real-world application scenarios if QRX003 receives regulatory approval.

Up to 8 participants with NS will be enrolled. QRX003 will be applied topically to more than 80% of the BSA over a duration of 12 weeks.

This trial’s design reflects a significant step forward, as it replicates how NS patients might use QRX003 in everyday life. Data generated from this study will supplement Quoin’s regulatory submission package and strengthen the evidence base for QRX003’s efficacy and safety.

Paller, the study's principal investigator, highlighted the importance of addressing the root cause of NS.

"The best kind of treatment, short of curative gene therapy, focuses on reversing the mechanism by which skin disease occurs," Paller said in a news release.¹ "Targeting kallikreins, which are thought to lead to the clinical manifestations of Netherton Syndrome, could be an ideal approach."

The initiation of this study comes on the heels of promising interim data from 2 ongoing QRX003 trials. These studies have demonstrated encouraging safety and efficacy outcomes, paving the way for the more comprehensive “whole body” approach in the upcoming trial.

Michael Myers, PhD, CEO of Quoin Pharmaceuticals, expressed optimism about the new trial’s potential.

"We are excited to advance this groundbreaking trial. By applying QRX003 to more than 80% of each subject’s body surface area, we’re taking an important step toward understanding its full potential in real-world settings," Myers said.¹ "This study underscores our commitment to delivering the first-ever approved treatment for this devastating disease."

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

Reference

1. Quoin Pharmaceuticals announces FDA clearance to initiate new QRX003 Netherton Syndrome clinical study. News release. Globe Newswire. December 19, 2024. Accessed December 19, 2024. https://www.globenewswire.com/news-release/2024/12/19/2999779/0/en/Quoin-Pharmaceuticals-Announces-FDA-Clearance-to-Initiate-New-QRX003-Netherton-Syndrome-Clinical-Study.html