Quoin Pharmaceuticals Announces Highly Positive ‘Whole Body’ Clinical Data from Ongoing Pediatric Netherton Syndrome Study

Rhea-AI Summary

Quoin Pharmaceuticals (NASDAQ: QNRX) has reported significant clinical data from its ongoing Investigator Pediatric Netherton Syndrome (NS) study for QRX003. The first pediatric patient receiving whole-body application showed dramatic improvement after just two weeks of treatment.

Key findings include:

• Investigator's Global Assessment (IGA) improved from 5 to 1 on a 0-10 scale
• Pruritus (itch) score decreased from 7 to 1-2 on a 0-10 scale
• Patient discontinued previously required medications including antihistamines, glucocorticoids, and antivirals
• No antibiotics needed since treatment initiation
• Zero nightly sleep disturbances reported for the first time
• No adverse events observed with twice-daily whole-body application

The company plans to expand the study to include additional pediatric subjects in other countries.

Positive

• Dramatic IGA improvement from 5 to 1 after 2 weeks
• Significant reduction in pruritus score from 7 to 1-2
• Elimination of multiple medication requirements
• No adverse events reported
• Treatment appears to target root cause of disease

Negative

• Data to single patient only
• Only 2 weeks of treatment duration observed
• Results require validation in larger patient population

Insights

Clinical Research Analyst

Quoin Pharmaceuticals has reported remarkably positive clinical data from its pediatric Netherton Syndrome (NS) study that could represent a significant breakthrough for this rare genetic skin disorder that currently has no FDA-approved treatments.

The results from the first pediatric patient receiving whole-body application of QRX003 show dramatic improvement across multiple disease measures after just two weeks of treatment. The patient's Investigator's Global Assessment score improved from 5 to 1 (on a 0-10 scale), while debilitating itching (pruritus) decreased from 7 to 1-2. Perhaps most significantly, the patient discontinued multiple medications previously required for disease management and experienced zero sleep disturbances for the first time.

These findings are particularly noteworthy because they suggest QRX003 may be addressing the fundamental pathophysiology of Netherton Syndrome rather than merely treating symptoms. For a devastating condition characterized by severely compromised skin barrier function, chronic inflammation, and susceptibility to infections, a therapy targeting the root cause represents a potential paradigm shift.

From an investment perspective, these early results could significantly impact Quoin's prospects despite the obvious limitation of being from a single patient. The orphan drug market for rare genetic disorders frequently supports premium pricing and extended market exclusivity. With a market capitalization of just $2.5 million, positive clinical developments could dramatically alter Quoin's valuation as the program advances.

The planned expansion to additional pediatric patients in other countries will be important for validating these initial findings. If similar efficacy is demonstrated in a larger patient population, QRX003 could establish Quoin as a significant player in the rare dermatological disease space, potentially attracting partnership interest from larger pharmaceutical companies seeking to expand their rare disease portfolios.

Biotech Investment Specialist

Quoin's clinical data for QRX003 in pediatric Netherton Syndrome represents a potentially transformative development that could dramatically alter the company's trajectory. With a market capitalization of just $2.5 million, these positive results could catalyze significant valuation reassessment if further validated.

The dramatic improvement seen in this first patient - with near-complete resolution of visible skin symptoms, substantial reduction in itching, elimination of multiple medications, and resolution of sleep disturbances - suggests QRX003 may be effectively targeting the underlying pathophysiology of this rare genetic disorder. For context, Netherton Syndrome affects approximately 1 in 200,000 newborns and currently has no FDA-approved treatments, representing a classic orphan drug opportunity.

What makes these results particularly compelling from an investment standpoint is the potential for QRX003 to command premium pricing if approved. Therapies for ultra-rare genetic disorders with high unmet need frequently command annual prices exceeding $300,000 per patient. With orphan drug designation typically providing 7 years of market exclusivity in the US and 10 years in Europe, even a small patient population can generate substantial revenue.

The critical question for investors is whether these remarkable results from a single patient will translate to a broader population. The planned expansion to additional pediatric patients across multiple countries will be important for derisking the program. Additionally, investors should monitor Quoin's cash position and burn rate, as small biotechs often require additional financing to advance promising candidates through clinical development.

For a micro-cap biotech, positive clinical data in a rare disease with no approved treatments represents the type of asymmetric risk/reward opportunity that can deliver outsized returns if the initial promise is confirmed in larger studies. However, investors should remain cognizant that single-patient data, while encouraging, represents an extremely preliminary signal that requires substantial additional validation.

• Groundbreaking data from first whole body application of QRX003 underscores potential efficacy
• Transformational improvement in disease state after just 2 weeks of treatment
• Clear visual evidence of skin healing observed
• Improvement has eliminated need for previously required medications
• Patient is now experiencing zero nightly sleep disturbance for the first time
• No adverse events reported from whole body treatment

ASHBURN, Va., Feb. 27, 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 highly positive clinical data from its ongoing Investigator Pediatric Netherton Syndrome (NS) study. Dramatic improvement was observed in the patient’s disease state with clear visual evidence of skin healing observed after just two weeks of whole-body application of QRX003. No adverse events have been reported to date.

Both key clinical endpoints, Investigator’s Global Assessment (IGA) and Pruritus or itch, demonstrated highly significant clinical improvements from baseline after two weeks of treatment with QRX003 on patient’s whole body.

Table 1: Results for First Pediatric Patient Receiving QRX003 ‘Whole-Body’ Application

Endpoint*	Baseline	Two Week
Investigator Global Assessment	7	1-2
Pruritus	5	1

*Both IGA and Pruritus scores based on a 0-10 scale.

Furthermore, following treatment with QRX003, the patient has now discontinued previously required medications including all antihistamines, glucocorticoids and antivirals. Importantly, the patient has not needed treatment with any antibiotics since the whole-body application of QRX003 was initiated. In addition, the patient is now experiencing zero nightly sleep disturbances for the first time in her life. No adverse events have been reported to date.

Quoin CEO Dr. Michael Myers, said, “While cautioning upfront that this data is from just a single patient over a short period of time, these truly exciting and groundbreaking results provide a snapshot of the potential efficacy of QRX003 as a chronic whole-body treatment of Netherton Syndrome. After just two weeks of treatment, a transformational life change has occurred for this first patient to receive whole-body application of QRX003. At baseline, the Investigators Global Assessment, or IGA, for the patient’s skin was at 5 on a scale of 0-10. After two weeks of treatment with QRX003, the IGA was at 1, representing an almost complete elimination of the visual effects of the disease on the patient’s skin. In addition, her pruritus, which at baseline was described as “chronically debilitating itch” and a 7 on of a scale of 0-10, had significantly dropped to a “highly tolerable and non-intrusive” score of between 1 and 2 after only two weeks of treatment with QRX003. Even more encouraging is the fact that the patient has been able to discontinue ongoing treatment with a variety of previously required medications including antihistamines, antivirals, glucocorticoids and she has not needed any antibiotics since the initiation of dosing with QRX003. We believe that these results indicate that QRX003 is directly targeting the root cause of the disease and has the potential to provide NS patients with the opportunity to have lives essentially free from the ravages of this terrible disease. In addition, the patient is now, for the first time, experiencing zero nightly sleep disturbances, again providing additional evidence of the potential efficacy of QRX003. Finally, we are very pleased to report that no adverse events have been recorded after two weeks of twice-daily, whole-body application of QRX003. As we continue with our plans to expand this study to include additional pediatric subjects in other countries who would also receive whole body application of QRX003, we believe that the highly encouraging nature of the results generated to date for this patient could potentially bring hope and comfort to children and families whose lives are being so dramatically impacted by the consequences of this cruel disease.”

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,” “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; QRX003 directly targeting the root cause of the disease, QRX003 having the potential to provide NS patients with the opportunity to live lives essentially free from the ravages of this terrible disease, expanding the study to include additional pediatric subjects in other countries who would also receive whole body application of QRX003, 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 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, 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 key clinical results of QNRX's QRX003 treatment for Netherton Syndrome?

After 2 weeks, IGA improved from 5 to 1, and pruritus score decreased from 7 to 1-2. Patient discontinued previous medications with no adverse events reported.

How effective is QRX003 in reducing medication dependency for Netherton Syndrome patients?

The first patient discontinued all antihistamines, glucocorticoids, antivirals, and hasn't needed antibiotics since starting QRX003 treatment.

What improvements in sleep quality were observed in the QNRX Netherton Syndrome trial?

The patient reported zero nightly sleep disturbances for the first time in their life after QRX003 treatment.

What are the safety findings from QNRX's whole-body application of QRX003?

No adverse events were reported after two weeks of twice-daily, whole-body application of QRX003.

What are the next steps for QNRX's Netherton Syndrome treatment study?

Quoin plans to expand the study to include additional pediatric subjects in other countries for whole-body QRX003 treatment.