Quoin Pharmaceuticals’ Announces Positive Interim QRX003 Data for Netherton Syndrome

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Today, Quoin Pharmaceuticals announced additional positive interim data from its ongoing, open-label study evaluating QRX003 for the treatment of Netherton Syndrome.¹ Positive improvements were observed using the Investigator’s Global Assessment (IGA), Worst Itch Numeric Rating Scale (WINRS), and Modified Ichthyosis Area Severity Index (M-IASI).

Study CL-QRX003-002 is a randomized, double-blinded, vehicle-controlled study testing the QRX003 topical lotion versus a placebo in patients 14 years and older. The product was applied twice daily for 12 weeks. Before and after photographs were also taken and published on Quoin’s website.

At baseline, IGA was considered to be moderate with a WINRS score of 7 and a M-IASI score of 18. At the midpoint of the trial, disease severity improved to mild with a 4 on both the WINRS and the M-IASI. After 12 weeks, final results indicated a WINRS score of 2 and an M-IASI score of 3, resulting in an IGA of “almost clear.” Additionally, Patient Satisfaction Scoring was extremely positive with no reported adverse reactions.

“We are very excited to announce continued positive results from our ongoing Netherton Syndrome clinical studies. With the availability of photographic evidence that clearly demonstrates the profound change in skin appearance for this subject in our open-label study, we believe we are continuing to develop a growing body of evidence of the potential efficacy of QRX003 in Netherton Syndrome,” Michael Myers, PhD, CEO of Quoin Pharmaceuticals, said in a statement. “The marked improvement demonstrated across all measured endpoints is highly encouraging. Furthermore, and importantly, the patient satisfaction scores with QRX003 continue to be very positive, with no safety concerns observed.”¹

The study is being conducted under an open U.S. Investigational New Drug (IND) application, which was first approved by the US Food and Drug Administration (FDA) in December 2024.² This includes a whole-body trial, led by Amy Paller, MD, at Northwestern University, which aims to generate real-world data by administering QRX003 to a broader body surface area than in previous studies.

“[We] anticipate that clinical data from this study will become a key component of the data package we are assembling to support a New Drug Application (NDA) filing for QRX003 as potentially the first approved treatment for Netherton Syndrome,” Myers noted. “2025 is already off to an exciting start for Quoin and we look forward to providing additional updates throughout the year.”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 designed to address the underlying pathology of NS 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 NS, offering new hope to patients and their families.