Quoin Pharmaceuticals Announces Further Positive Clinical Data from Ongoing Pediatric Netherton Syndrome Study

Rhea-AI Summary

Quoin Pharmaceuticals (NASDAQ: QNRX) reported positive interim clinical data from its ongoing Investigator Pediatric Netherton Syndrome study of QRX003. The study demonstrated significant improvement in treated skin areas, with the subject's condition improving from 'Severe' to 'Mild' after six weeks of twice-daily treatment.

Due to these positive results, the treatment area has been expanded from 20% to the whole body surface area (BSA). No adverse events or safety concerns have been reported, consistent with other ongoing Netherton Syndrome clinical studies. The Investigator's Global Assessment (IGA) showed improvement from 'Severe' at baseline to 'Mild-Moderate' after 12 days, progressing to 'Mild' after 6 weeks.

Positive

• Clinical improvement from 'Severe' to 'Mild' in just 6 weeks
• Treatment area expanded from 20% to 100% body surface area
• No adverse events or safety concerns reported
• Rapid improvement shown within first 12 days of treatment

Negative

• data from single patient study
• Full efficacy and safety profile yet to be established

Insights

Medical Research Analyst

This interim clinical data release for QRX003 in pediatric Netherton Syndrome represents a significant therapeutic breakthrough. The improvement from "Severe" to "Mild" on the Investigator's Global Assessment (IGA) scale within just 6 weeks is remarkably rapid for a chronic skin condition. The decision to expand treatment from 20% BSA to whole body application, coupled with the absence of adverse events, strongly validates the drug's safety profile.

In the context of rare disease therapeutics, these results are particularly compelling. Netherton Syndrome, affecting approximately 1 in 200,000 newborns, has historically lacked effective treatments. The rapid improvement in skin condition suggests QRX003 may be addressing the fundamental pathophysiology of the disease, rather than just managing symptoms. The expansion to whole-body treatment could provide more comprehensive efficacy data, potentially strengthening the case for regulatory approval.

Financial Analyst

For a micro-cap biotech with a market cap of just $4.9M, these clinical results could be transformative. The positive data in both pediatric and adult populations positions QNRX favorably for potential accelerated approval pathways, given the unmet medical need in Netherton Syndrome. The expansion to whole-body treatment could expedite the path to pivotal trials.

The market opportunity, while niche, is significant due to orphan drug pricing potential. Similar rare disease treatments often command premium pricing of $200,000-500,000 annually per patient. With approximately 2,000-3,000 Netherton Syndrome patients in major markets, the peak sales potential could reach $500M-1B annually. The clean safety profile and rapid efficacy signals enhance the probability of regulatory success, which could attract partnership interest from larger pharmaceutical companies seeking to expand their rare disease portfolios.

• Continued Significant Improvement in Skin Appearance Observed in Study
• Subject’s Disease Classification Improved from “Severe” to “Mild” after 6 Weeks Dosing
• Treatment area expanded from 20% Body Surface Area to “Whole Body”
  

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

The Investigator Pediatric Study continues to demonstrate significant improvement in the skin area treated with QRX003 versus the non-treated area. At the mid-point of the testing period the Investigator’s Global Assessment (IGA) of the skin condition improved from “Severe” at baseline to “Mild” after six weeks of dosing twice a day with QRX003, indicating a very substantial improvement in a short period of testing. As a result of these positive results, the subject is being transitioned to having QRX003 applied to their whole body surface area (BSA) as opposed to the approximately 20% of their BSA that was being tested for the initial 6 weeks.

In addition, there have been no adverse events or safety concerns reported to date for this subject, which is consistent with observations from each of Quoin’s ongoing clinical studies in Netherton Syndrome subjects.

Quoin CEO, Dr. Michael Myers, said, “Earlier this week we shared positive data from our ongoing open label clinical study in subjects aged 14 years and older and provided photographic evidence of the improvement in that subject’s skin, which is accessible via this link. Today, we are very pleased to announce another positive update from our pediatric study across a number of clinical endpoints for the subject in this study. The significant improvement in the Investigator’s Global Assessment (IGA) from Severe at Baseline to Mild-Moderate after 12 days of dosing, and now to Mild after 6 weeks of dosing is truly remarkable, and we believe that the investigator’s decision to transition the subject to “whole body” treatment is an exciting step forward that will provide even more representative data on the potential safety and efficacy of QRX003 in Netherton Syndrome patients. We look forward to expanding this study to include additional pediatric subjects in other countries, and we believe each of these subjects may have an opportunity to move directly to whole body testing based on the positive nature of the results generated to date. With this study well underway and the adult “whole body” study cleared to proceed by the FDA, we are beginning to assemble what we hope will be very compelling clinical evidence that supports the potential of QRX003 as a safe and effective treatment for Netherton Syndrome patients.”

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 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, Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa 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,” “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 belief that the decision to transition the pediatric subject to “whole body” treatment will provide even more representative data on the safety and efficacy of QRX003 as a treatment for Netherton Syndrome; expanding the study to include other pediatric subjects in other countries, the belief that such subjects may may have an opportunity to move directly to whole body testing based on the results generated to date, beginning to assemble what we hope will be very compelling clinical evidence that supports the potential of QRX003 as a safe and effective treatment for Netherton Syndrome patients 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, Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa 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 its studies successfully generate data that is sufficiently robust and comprehensive to support an NDA filing for QRXOO3 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, 2023 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 latest clinical results for QNRX's QRX003 in pediatric Netherton Syndrome?

The latest results show improvement from 'Severe' to 'Mild' condition after 6 weeks of treatment, with no adverse events reported.

How much body surface area is now being treated in QNRX's pediatric Netherton study?

The treatment area has been expanded from 20% to whole body surface area due to positive results.

What is the safety profile of QRX003 in QNRX's pediatric Netherton study?

No adverse events or safety concerns have been reported to date in the pediatric study.

How quickly did QNRX's QRX003 show improvement in the pediatric Netherton study?

The treatment showed improvement from 'Severe' to 'Mild-Moderate' after just 12 days of dosing.

What is the dosing frequency of QRX003 in QNRX's pediatric Netherton study?

QRX003 is administered twice daily in the pediatric Netherton Syndrome study.