Quoin Pharmaceuticals Announces Additional Positive Interim Data from Ongoing Open-Label Netherton Syndrome Clinical Study

Rhea-AI Summary

Quoin Pharmaceuticals announces additional positive interim data from its ongoing open-label Netherton Syndrome clinical study. The study involves QRX003, a treatment administered twice daily. After 12 weeks, the first subject showed significant improvements across all measured clinical endpoints:

• M-IASI: Reduced from 18 to 3 (lower scores indicate improvement)
• WINRS: Decreased from 7 to 2 (measures itch severity on an 11-point scale)
• IGA: Improved from 'Moderate' to 'Almost Clear'

Photographs demonstrating the subject's skin improvements are available on Quoin's website. No safety concerns were identified during the study. Patient satisfaction scores were highly positive and improved further after 12 weeks. Quoin CEO, Dr. Michael Myers, expressed excitement over the results, highlighting the potential efficacy of QRX003 and the absence of safety issues. The company is preparing for a 'whole body' clinical study at Northwestern University, with FDA clearance already obtained. Data from this study will support a New Drug Application for QRX003 as the first approved treatment for Netherton Syndrome. Quoin anticipates providing more updates throughout 2025.

Positive

• Significant improvements in clinical endpoints for the first subject in the QRX003 study.
• No safety concerns reported during the study.
• Positive patient satisfaction scores that improved over 12 weeks.
• FDA clearance obtained for a 'whole body' clinical study at Northwestern University.
• Potential efficacy of QRX003 demonstrated through photographic evidence.

Negative

• None.

Insights

Medical Research Analyst

The interim clinical data from Quoin's QRX003 trial for Netherton Syndrome demonstrates remarkable therapeutic efficacy. The 12-week results show substantial improvements across all key metrics: M-IASI score dropped from 18 to 3, indicating significant reduction in disease severity; WINRS decreased from 7 to 2, showing marked improvement in patient comfort; and IGA progressed from Moderate to Almost Clear.

Think of M-IASI like a temperature gauge for skin condition - the dramatic drop from 18 to 3 is similar to bringing a high fever down to near-normal levels. The sustained improvement over 12 weeks, coupled with positive patient satisfaction scores and clean safety profile, suggests QRX003 could become the first approved treatment for this rare genetic condition.

The progression to a 'whole body' study under an open IND at Northwestern University represents a important step toward NDA submission. For a rare disease with no approved treatments, these results could position QRX003 as a breakthrough therapy.

Financial Analyst

This clinical milestone significantly strengthens Quoin's market position in the rare disease space. With a market cap of just <money>$6.26M</money>, the company appears undervalued considering the potential market opportunity for the first approved Netherton Syndrome treatment.

The consistent positive data across multiple endpoints reduces development risk and increases the probability of successful commercialization. The orphan drug designation typically commands premium pricing and with no approved competitors, QRX003 could generate substantial revenue despite the small patient population.

The advancement toward an NDA filing represents a critical value inflection point. For context, orphan drugs can generate annual revenues ranging from <money>$100M</money> to over <money>$1B</money>, depending on pricing and patient population. The stock could see significant revaluation as the company progresses toward potential commercialization.

• Highly Positive Clinical Data on Completion of Testing for First Subject Dosed Twice Daily with QRX003 in Open-Label Study
• Demonstrated Clinical Benefits from QRX003 Observed Across All Measured Endpoints
• Before and After Dosing with QRX003 Photographs Available for Review On Website
• No Product Safety Concerns Identified in Any Quoin Netherton Study to Date

ASHBURN, Va., Jan. 06, 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 additional positive interim clinical data from one of its ongoing Netherton Syndrome clinical studies.

In December 2024, Quoin announced positive data from the first subject dosed twice-daily in Quoin’s ongoing open-label study after 6-weeks of dosing with QRX003, marking the midpoint of testing. The Company is now pleased to share clinical data for that subject upon completion of testing. The following table illustrates the clear improvements observed for this subject from baseline through 12 weeks of twice-daily dosing with QRX003 across all measured clinical endpoints.

Table 1: First Patient Data from Open Label Study Part B- Dosed Twice Daily with QRX003

End Point	Baseline	6 weeks	12 weeks
M-IASI*	18	4	3
WINRS**	7	4	2
IGA***	Moderate	Mild	Almost Clear

*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.
**WINRS: Worst Itch Numeric Rating Scale, which measures the severity of itch on an 11-point scale (0 = no itch, 10 = worst imaginable itch).
***IGA: Investigator’s Global Assessment, which uses descriptive categories (e.g., clear, mild, moderate, severe) to evaluate the overall severity of Netherton Syndrome symptoms.

Photographs illustrating the improvements in the subject’s skin appearance can be accessed via the link below and are available on Quoin’s website.
https://quoinpharma.com/pipeline/#trials

In addition, the patient satisfaction scores across multiple assessed metrics which were highly positive after 6 weeks of testing demonstrated even further improvement after 12 weeks. No safety concerns were reported for the subject throughout the study.

Quoin CEO, Dr. Michael Myers, said, “We are very excited to announce continued positive results from our ongoing Netherton Syndrome clinical studies. With the availability of photographic evidence that clearly demonstrates the profound change in skin appearance for this subject in our open-label study, we believe we are continuing to develop a growing body of evidence of the potential efficacy of QRX003 in Netherton Syndrome. The marked improvement demonstrated across all measured endpoints is highly encouraging. Furthermore, and importantly, the patient satisfaction scores with QRX003 continue to be very positive, with no safety concerns observed.

We are eagerly awaiting the initiation of our ‘whole body’ clinical study, which will be conducted by Dr. Amy Paller at Northwestern University. We previously announced FDA clearance to proceed with this study under an open Investigational New Drug (IND) application in December and anticipate that clinical data from this study will become a key component of the data package we are assembling to support a New Drug Application (NDA) filing for QRX003 as potentially the first approved treatment for Netherton Syndrome. 2025 is already off to an exciting start for Quoin and we look forward to providing additional updates throughout the year.”

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 a description 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,” “plan,” “anticipate,” “believe,” 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, - potential efficacy of QRX003 as a treatment for Netherton Syndrome, await the initiation of our ‘whole body’ clinical study, which will be conducted by Dr. Amy Paller at Northwestern University, anticipate that clinical data from the whole body study will become a key component of the data package the Company is assembling to support a New Drug Application (NDA) filing for QRX003 as potentially the first approved treatment for Netherton Syndrome,), 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, its studies may not be successful or may not generate data which is sufficiently robust and comprehensive to 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 results from Quoin Pharmaceuticals' Netherton Syndrome study?

The latest results show significant improvements in clinical endpoints for the first subject dosed twice daily with QRX003, including reductions in M-IASI and WINRS scores and an improvement in IGA.

What is QRX003 and its significance in Netherton Syndrome?

QRX003 is a treatment under study by Quoin Pharmaceuticals for Netherton Syndrome. Recent interim data shows significant clinical improvements and no safety concerns.

What improvements were observed in the QRX003 study for Netherton Syndrome?

Improvements included a reduction in M-IASI from 18 to 3, a decrease in WINRS from 7 to 2, and an IGA improvement from 'Moderate' to 'Almost Clear' after 12 weeks.

Were there any safety concerns in the QRX003 Netherton Syndrome study?

No safety concerns were identified for the subject throughout the 12-week study period.

What are the next steps for QRX003 in treating Netherton Syndrome?

The next step is a 'whole body' clinical study at Northwestern University, which has received FDA clearance. Data from this study will support a New Drug Application for QRX003.

How did patient satisfaction scores change during the QRX003 study?

Patient satisfaction scores were highly positive after 6 weeks and showed even further improvement after 12 weeks of dosing with QRX003.