Quoin Pharmaceuticals Announces Initial Positive Clinical Data for QRX003 from Pediatric Peeling Skin Syndrome Study

Rhea-AI Summary

Quoin Pharmaceuticals (NASDAQ: QNRX) has reported positive initial clinical data from its ongoing Investigator Pediatric Peeling Skin Syndrome study for QRX003. After 12 weeks of treatment, the study showed significant improvements across multiple endpoints:

- Modified Ichthyosis Area Severity Index (M-IASI) improved from 36 to 12 - Investigator's Global Assessment (IGA) improved from severe (4) to mild (2) - Children's Dermatology Life Quality Index (CDQLI) improved from 19 to 11

The treatment was well-tolerated with no adverse events reported. Based on these positive results, the patient will continue treatment with QRX003, with another assessment scheduled after 24 weeks. This study may be the first formal clinical trial for Peeling Skin Syndrome, a rare genetic disease with no currently approved treatments.

Positive

• Clear improvement in patient's skin condition after 12 weeks of treatment
• Significant two-grade improvement in IGA from severe to mild
• No adverse events reported, demonstrating good tolerability
• Potential to be first approved treatment for Peeling Skin Syndrome
• Quality of life improvement demonstrated through CDQLI score reduction

Negative

• Study currently limited to only one pediatric patient
• Long-term efficacy and safety data not yet available

Insights

Clinical Research Physician specializing in Dermatology and Rare Diseases

QRX003 shows promising early results in treating Peeling Skin Syndrome with significant symptom reduction and no adverse events.

The initial clinical data from Quoin's pediatric Peeling Skin Syndrome study represents a significant therapeutic advance in this rare genetic disorder. The 12-week results demonstrate substantial improvements across multiple validated clinical endpoints. Most notably, the Modified Ichthyosis Area Severity Index (M-IASI) decreased dramatically from 36 to 12, indicating a 66% reduction in disease severity.

The Investigator's Global Assessment (IGA) showed a two-grade improvement from Severe (4) to Mild (2), which is considered clinically meaningful in dermatological conditions. This magnitude of improvement in just 12 weeks is particularly impressive for a chronic genetic skin disorder.

Equally important is the 8-point improvement in the Children's Dermatology Life Quality Index (CDLQI), decreasing from 19 to 11. This validated tool measures real-world impact on pediatric patients' daily lives, suggesting QRX003 is providing meaningful benefits to quality of life beyond just clinical measurements.

The clean safety profile with no reported adverse events is especially valuable in pediatric applications where treatment tolerability is crucial. This favorable risk-benefit profile supports the investigator's decision to continue treatment to the 24-week assessment.

What makes these results particularly noteworthy is the complete absence of approved treatments for Peeling Skin Syndrome. If QRX003 maintains this efficacy profile in larger studies, Quoin could potentially secure the first regulatory approval for this orphan indication, which typically comes with market exclusivity benefits and premium pricing potential for rare disease therapies.

• Clear Improvement in Patient’s Skin Appearance Observed in Study after 12 weeks Compared to Baseline
• Key endpoints including Investigator’s Global Assessment (IGA), Modified Ichthyosis Area Severity (M-IASI) and Children’s Dermatology Life Quality Index (CDLQI) all demonstrated improvement from baseline
• QRX003 is being well tolerated and no adverse events have been reported
• Patient is expected to continue to be treated with QRX003 to assess progress
• Company continues to advance QRX003 in its late-stage Netherton Syndrome clinical studies
  

ASHBURN, Va., May 14, 2025 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a late clinical stage, specialty pharmaceutical company focused on rare and orphan diseases, today announces positive initial clinical data from its ongoing Investigator Pediatric Peeling Skin Syndrome clinical study.

As the table below illustrates, after 12 weeks of treatment, clear evidence of skin healing in the area treated with QRX003 compared to baseline was observed.

End Point	Baseline	12 weeks (End of treatment period)
M-IASI*	36	12
IGA**	4 (Severe)	2 (Mild)
CDQLI***	19	11

*M-IASI: Modified Ichthyosis Area of Severity Index, a score used to assess the severity and extent of skin symptoms associated with ichthyosis. Lower scores indicate improvement.
**IGA: Investigator’s Global Assessment, which uses descriptive categories (e.g., clear, mild, moderate, severe) to evaluate the overall severity of disease symptoms.

*** CDQLI: The Children’s Dermatology Life Quality Index is a validated clinical tool designed for children aged 4-15 that is used to measure the impact of their skin disease on a child’s quality of life in terms of symptoms, leisure activities, sleep, school, personal relationship and treatment. The scale for the CDQLI is 0-30.

QRX003 is being well tolerated by the patient and no adverse events have been reported. As a result of these positive initial results, the patient is expected to continue to be treated with QRX003 with a further clinical assessment by the investigator scheduled after 24 weeks of treatment.

Quoin CEO, Dr. Michael Myers, said, “We are very pleased to announce such positive initial data across a number of clinical endpoints for the pediatric subject in this study, which we believe may be the first formal clinical study for this disease. The two grade improvement, which is accepted as being clinically meaningful in both the Investigator’s Global Assessment (IGA) (severe to mild) and the M-IASI (moderate to clear) after 12 weeks of QRX003 application is very encouraging. We fully support the investigator’s decision to continue treatment. In addition, the validated CDQLI questionnaire results to date indicate that the pediatric subject in the study is experiencing a distinct positive improvement in their quality of life as a result of ongoing treatment with QRX003. Consistent with what we are observing in our Netherton Syndrome studies, QRX003 is well tolerated with no adverse events reported. We look forward to expanding this study to include additional pediatric subjects in other countries and to advancing the clinical development of the product for this disease. There is currently no approved treatment or cure for peeling skin syndrome and there are no clinical studies listed on clinicaltrials.gov as actively recruiting and dosing subjects, thus presenting a further opportunity for Quoin to potentially achieve the first regulatory approval for another rare genetic disease. We are also continuing to advance QRX003 in our late-stage Netherton Syndrome clinical studies."

About Peeling Skin Syndrome (PSS)
Generalized inflammatory peeling skin syndrome (PSS) is a rare autosomal recessive genodermatosis caused by loss-of-function disease-causing variants of the corneodesmosin gene (CDSN), resulting in excessive shedding of the superficial layers of the epidermis. Patients generally suffer from a variety of conditions including severe pain and chronic pruritus (itch). There is currently no approved treatment for PSS, and patients try to manage symptoms using over-the-counter emollients.

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 patient continuing to be treated with QRX003 to assess progress; Quoin’s Pediatric Peeling Skin Syndrome clinical study being the first formal clinical study for this disease, expanding the study to include additional pediatric subjects in other countries, advancing the clinical development of a product for Peeling Skin Syndrome, achieving the first regulatory approval for another rare genetic disease, advancing QRX003 in Quoin’s late-stage Netherton Syndrome clinical studies 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 pursue its regulatory strategy; the Company’s ability to obtain regulatory approvals for commercialization of product candidates or to comply with ongoing regulatory requirements; the Company’s ability to complete clinical trials on time and achieve desired results and benefits as expected; 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:

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

FAQ

What are the initial results of Quoin Pharmaceuticals' (QNRX) QRX003 trial for Peeling Skin Syndrome?

After 12 weeks of treatment, QRX003 showed significant improvements with M-IASI score improving from 36 to 12, IGA score improving from severe to mild, and CDQLI score improving from 19 to 11, with no adverse events reported.

How did QRX003 affect the quality of life in pediatric patients with Peeling Skin Syndrome?

The Children's Dermatology Life Quality Index (CDQLI) improved from 19 to 11, indicating a distinct positive improvement in the patient's quality of life during treatment.

What makes QNRX's QRX003 trial significant for Peeling Skin Syndrome treatment?

This may be the first formal clinical study for Peeling Skin Syndrome, a condition with no currently approved treatments, potentially positioning Quoin to achieve the first regulatory approval for this rare genetic disease.

What are the safety results of QRX003 in the Peeling Skin Syndrome trial?

QRX003 was well-tolerated by the patient with no adverse events reported during the 12-week treatment period.

What are the next steps for QNRX's QRX003 Peeling Skin Syndrome study?

The patient will continue treatment with a further clinical assessment scheduled after 24 weeks, and the company plans to expand the study to include additional pediatric subjects in other countries.