Quoin Pharmaceuticals Announces Additional Positive ‘Whole Body’ Clinical Data from Ongoing Pediatric Netherton Syndrome Study and Approval to Initiate Testing of a Second Pediatric Patient

Rhea-AI Summary

Quoin Pharmaceuticals (NASDAQ: QNRX) has reported additional positive clinical data from its ongoing pediatric Netherton Syndrome (NS) study of QRX003. After 6 weeks of whole-body treatment, the first patient's skin remains almost completely healed, demonstrating treatment durability.

Key outcomes include:

• Elimination of previously required medications (antibiotics, antivirals, antihistamines, glucocorticoids)
• Almost complete elimination of pruritus (itching)
• Zero nightly sleep disturbances without sedating medication
• No adverse events reported after 6 weeks

Based on these positive results, Quoin has received approval to begin whole-body QRX003 treatment on a second pediatric NS patient, with testing expected to commence within weeks.

Insights

Biotech Clinical Trials Analyst

Quoin's latest clinical update represents a significant efficacy milestone for QRX003 in treating Netherton Syndrome (NS), a rare genetic skin disorder. The continued healing of the first pediatric patient's skin after 6 weeks demonstrates sustained therapeutic effect - crucial for chronic rare disease treatments. Most compelling is the complete elimination of pruritus (severe itching), typically the most debilitating NS symptom requiring heavy sedation.

The multi-dimensional efficacy profile is particularly noteworthy: QRX003 has enabled this patient to discontinue all previously required medications (antibiotics, antivirals, antihistamines, glucocorticoids), suggesting the drug addresses fundamental disease mechanisms rather than merely providing symptomatic relief. The clean safety profile after 6 weeks of whole-body application significantly derisks the developmental pathway.

The regulatory approval to expand testing to a second pediatric patient represents important clinical validation and development momentum. For rare disease therapeutics, each successful patient case carries outsized significance given the patient population. This development pathway appears to be following an accelerated pediatric study model that could potentially expedite the timeline to market.

While early-stage with patient data, these results suggest QRX003 could become a transformative treatment addressing the root cause of NS rather than just managing symptoms - the holy grail in rare disease therapeutics where current options are severely

Orphan Drug Market Specialist

Quoin's QRX003 clinical progress represents significant commercial potential in the orphan drug landscape. Netherton Syndrome (NS) exemplifies the ultra-rare disease market dynamic where successful treatments can command premium pricing despite small patient populations. The comprehensive symptom resolution (skin healing, pruritus elimination, medication discontinuation) positions QRX003 as potentially disease-modifying rather than merely palliative - a critical distinction that typically justifies higher reimbursement rates.

The durability of effect over 6 weeks suggests QRX003 could become a maintenance therapy requiring consistent long-term administration, creating a recurring revenue stream. The clean safety profile is particularly valuable as it could support broad labeling without significant restrictions, maximizing the addressable patient population.

From a regulatory perspective, the expansion to multiple patients demonstrates a methodical development approach. With orphan drug designation, Quoin would benefit from regulatory incentives including market exclusivity and potentially accelerated approval pathways if this efficacy profile continues.

The quality-of-life improvements (elimination of sleep disturbances, medication cessation) will be powerful endpoints for both regulatory submissions and payer negotiations. Orphan drug pricing often correlates directly with measurable improvements in functional outcomes and reduced healthcare resource utilization, both of which appear evident in this case. The upcoming expansion to additional patients will be critical to validate these findings across a broader population.

• Continued Clear Visual Evidence of Almost Completely Healed Skin After 6 weeks Treatment
• High Durability of Treatment Effect of QRX003 with Continuous Daily Dosing
• Patient Continues Not to Require Previously Necessary Medications
• With Patient’s Pruritus Almost Completely Eliminated, Patient Continues to Experience Zero Nightly Sleep Disturbance
• No Adverse Events Reported from 6 Weeks of Whole Body Treatment with QRX003
• Approval Received to Initiate Whole Body Testing of a Second Pediatric Patient

ASHBURN, Va., April 02, 2025 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a late clinical stage, specialty pharmaceutical company focused on the development and commercialization of therapeutic products that treat rare and orphan diseases, today announces additional highly positive clinical data from its ongoing Investigator Pediatric Netherton Syndrome (NS) study. After 6 weeks of continued whole body application of QRX003, the subject’s skin remains almost completely healed demonstrating the durability of ongoing daily treatment with the product. In addition, the patient has continued to have no requirement for previously necessary medications such as antibiotics, antivirals, antihistamines and glucocorticoids. Importantly, with the patient’s pruritus or itch almost completely eliminated, she continues to experience zero nightly sleep disturbances without the need for any sedating medication, marking the first continuous period of uninterrupted sleep in the patient’s life. No adverse events have been reported to date after 6 weeks of whole body treatment with QRX003.

Quoin CEO Dr. Michael Myers, said, “As we continue to monitor the progress of this first patient to receive whole body application with QRX003, we are very pleased to announce that after 6 weeks of dosing we are seeing continued positive improvement and the patient’s skin is almost completely healed now. The patient continues to remain off previously needed medications including antihistamines, antivirals, and glucocorticoids, and she has not needed any antibiotics in the 6 weeks since the initiation of dosing with QRX003. Very importantly also, with her pruritus almost completely eliminated, the patient is still experiencing zero nightly sleep disturbances without the need for any sedating medications. For a majority of Netherton Syndrome patients, the continuous presence of chronically debilitating pruritus is the worst symptom of this disease and it severely impacts their ability to sleep or to even sit still for any period of time. Often heavy sedation is required to allow patients to sleep and sit still, which in turn has a major negative impact on their quality of life. The almost complete healing of the skin and the elimination of this chronically debilitating pruritus provides strong clinical evidence that QRX003 is successfully tackling the root causes of this disease and not merely providing symptomatic relief. Furthermore, I am very pleased to report that no adverse events have been reported after six full weeks of whole-body application of QRX003. Based on these positive results, approval has been received to initiate whole body application of QRX003 to a second pediatric Netherton patient. Testing is expected to commence in just a few weeks and we look forward to providing updates on both patients progress as we continue with our plans to further expand this study to include additional pediatric subjects.”

About Netherton Syndrome
Netherton Syndrome, a form of Ichthyosis, is a rare hereditary skin disorder caused by a mutation in the SPINK5 gene (serine protease inhibitor, Kazal Type 5) that leads to severe skin barrier defects and recurring infections, as well as a pronounced predisposition to allergies, asthma, and eczema. Patients often suffer from severe dehydration, chronic skin inflammation and stunted growth. Currently, there is no cure for Netherton Syndrome, nor are there any approved therapeutic treatments.

About QRX003
QRX003 is a topical lotion formulated with a proprietary delivery technology that contains a broad-spectrum serine protease inhibitor, whose mechanism of action is intended to perform the function of a specific protein called LEKTI. The absence of LEKTI in Netherton patients leads to excessive skin shedding, resulting in a highly porous and compromised skin barrier. QRX003 is designed to promote a more normalized skin-shedding process and the formation of a stronger and more effective skin barrier. For more information about Quoin’s current clinical trials please visit: https://www.nethertonsyndromeclinicaltrials.com/

About Quoin Pharmaceuticals Ltd.
Quoin Pharmaceuticals Ltd. is a late clinical-stage specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases. We are committed to addressing unmet medical needs for patients, their families, communities and care teams. Quoin’s innovative pipeline comprises four products in development that collectively have the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, SAM Syndrome,  Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa, Microcystic Lymphatic Malformations, Venous Malformations, Angiofibroma and others. For more information, visit: www.quoinpharma.com or LinkedIn for updates.

Cautionary Note Regarding Forward Looking Statements
The Company cautions that statements in this press release that are not descriptions of historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words referencing future events or circumstances, such as “expect,” “intend,” “hope,” “plan,” “potential,” “anticipate,” “look forward,” “believe,” “may,” and “will,” among others. All statements that reflect the Company’s expectations, assumptions, projections, beliefs, or opinions about the future, other than statements of historical fact, are forward-looking statements, including, without limitation, statements relating to: the potential efficacy of QRX003 as a treatment for Netherton Syndrome; testing expected to commence in a few weeks on a second pediatric patient; continue with our plans to further expand this study to include additional pediatric subjects; and Quoin’s products in development collectively having the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, SAM Syndrome,  Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa, Microcystic Lymphatic Malformations, Venous Malformations, Angiofibroma and others. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon the Company’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties including, but not limited to, the Company’s ability to deliver a safe and effective treatment for Netherton Syndrome; whether the Company’s studies successfully generate data that is sufficiently robust and comprehensive to support an NDA filing for QRX003 as an approved treatment for Netherton Syndrome; and other factors discussed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024 and in other filings the Company has made and may make with the SEC in the future. One should not place undue reliance on these forward-looking statements, which speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as may be required by law.

For further information, contact:
Quoin Pharmaceuticals Ltd.
Michael Myers, Ph.D., CEO
mmyers@quoinpharma.com

Investor Relations
PCG Advisory
Jeff Ramson
jramson@pcgadvisory.com
(646) 863-6341

FAQ

What are the key results from QNRX's 6-week Netherton Syndrome trial?

The first pediatric patient showed almost completely healed skin, eliminated need for previous medications, experienced no sleep disturbances, and reported no adverse events after 6 weeks of whole-body QRX003 treatment.

How effective is QRX003 in treating pruritus in Netherton Syndrome patients?

QRX003 has shown significant efficacy in almost completely eliminating pruritus, allowing the patient to experience uninterrupted sleep without sedating medication for the first time.

What medications were eliminated during QNRX's QRX003 treatment?

The patient no longer required antibiotics, antivirals, antihistamines, and glucocorticoids that were previously necessary.

What is the next step in QNRX's Netherton Syndrome clinical trial?

Quoin has received approval to begin whole-body QRX003 treatment on a second pediatric patient, with testing expected to start within weeks.