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Quoin Pharmaceuticals Initiates Clinical Testing of QRX003 in Pediatric Netherton Syndrome Patient

• Quoin Pharmaceuticals has begun clinical testing of QRX003 in a young child with Netherton Syndrome at Children’s Health Ireland in Dublin. • This assessment marks the first evaluation of QRX003 in a pediatric patient, with plans to expand the study to include additional pediatric subjects in Spain. • QRX003 is Quoin's lead product, currently in late-stage clinical trials, showing promising results in adult Netherton Syndrome patients with no treatment-related adverse events. • The trial aims to broaden the data set for QRX003 and potentially lower the eligibility age for treatment, addressing the unmet need for approved therapies for Netherton Syndrome.

Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) has commenced clinical testing of its lead product, QRX003, in a young child diagnosed with Netherton Syndrome (NS). This rare, inherited genetic disorder currently lacks any approved treatments or a cure. The clinical assessment is taking place at Children’s Health Ireland in Dublin, under the guidance of Dr. Alan Irvine, a consultant dermatologist and professor at Trinity College Dublin.

Clinical Assessment Details

The clinical assessment, led by Dr. Alan Irvine, aims to evaluate the safety and efficacy of QRX003 in a pediatric patient. Dr. Irvine, a leading researcher in epithelial genetics, will oversee the evaluation. "We are pleased to announce this latest initiative for Quoin as we strive to generate the broadest and most diverse data set possible for QRX003 in Netherton Syndrome patients," said Dr. Michael Myers, Quoin CEO. The company hopes the data will facilitate lowering the eligibility age for treatment.

QRX003: A Potential Treatment for Netherton Syndrome

QRX003 is a topical lotion formulated with a proprietary delivery technology containing a broad-spectrum serine protease inhibitor. It is designed to perform the function of LEKTI, a protein absent in Netherton Syndrome patients, which leads to excessive skin shedding and a compromised skin barrier. The drug aims to normalize skin-shedding and strengthen the skin barrier.

Ongoing Clinical Trials and Expansion

QRX003 is currently being evaluated in two late-stage clinical trials under an open Investigational New Drug application with the U.S. Food and Drug Administration. Previous clinical data has shown promising results, with all evaluable subjects demonstrating improvement across several endpoints and no treatment-related adverse events. In addition to five open clinical sites in the United States, a sixth site is opening in Saudi Arabia, with plans for two additional sites in the United Kingdom. The company also plans to broaden the scope of the clinical assessment to include three additional pediatric subjects in Spain.

About Netherton Syndrome

Netherton Syndrome is a rare hereditary skin disorder caused by a mutation in the SPINK5 gene, leading to severe skin barrier defects, recurring infections, and a predisposition to allergies, asthma, and eczema. Patients often suffer from severe dehydration, chronic skin inflammation, and stunted growth. The absence of approved therapeutic treatments highlights the urgent need for effective therapies like QRX003.
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[1]
Quoin Pharmaceuticals Initiates Clinical Testing of Lead Product in Pediatric Netherton ...
markets.businessinsider.com · Nov 5, 2024

Quoin Pharmaceuticals initiates testing of QRX003 for Netherton Syndrome in a pediatric patient at Children’s Health Ire...

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