Quoin Pharmaceuticals Announces Positive Interim Data from Two Ongoing Netherton Syndrome Clinical Studies

Rhea-AI Summary

Quoin Pharmaceuticals (NASDAQ: QNRX) has reported positive interim data from two ongoing Netherton Syndrome clinical studies for its QRX003 treatment. In the first study, a subject receiving twice-daily dosing showed significant improvements after six weeks: Modified Ichthyosis Area of Severity Index (MIASI) reduced from 18 to 4, Investigator's Global Assessment (IGA) improved from 'moderate' to 'mild', and pruritus assessment decreased from 7 to 4 on the Worst Itch Numeric Rating Scale.

In the second study, a pediatric patient showed marked improvement after just 12 days of treatment, with IGA classification improving from 'severe' to 'mild-moderate'. No safety concerns were reported in either study.

Positive

• MIASI score significantly improved from 18 to 4 after 6 weeks of treatment
• Disease severity (IGA) improved from 'moderate' to 'mild' in adult study
• Pruritus assessment reduced from 7 to 4 on the WINRS scale
• Pediatric study showed rapid improvement from 'severe' to 'mild-moderate' in just 12 days
• No safety concerns or adverse events reported in both studies

Negative

• sample size in both studies affects data reliability
• Results are only interim, not final study conclusions

Insights

Medical Research Analyst

The interim clinical data for QRX003 in Netherton Syndrome shows promising efficacy signals. The MIASI score reduction from 18 to 4 in the twice-daily dosing cohort represents a significant 77.8% improvement in disease severity. The improvement in IGA from 'moderate' to 'mild' and WINRS itch score decrease from 7 to 4 further validate the drug's potential therapeutic benefit.

Particularly noteworthy is the rapid response in the pediatric study, where improvement from 'severe' to 'mild-moderate' was observed in just 12 days. This accelerated response could be important for pediatric patients who often face more severe manifestations of the disease. The absence of safety concerns across both studies strengthens the drug's risk-benefit profile.

Financial Analyst

For a micro-cap biotech with a market cap of just $3.28M, these positive interim results could be transformative. Netherton Syndrome, being a rare genetic disorder, represents a potentially lucrative market with competition. The dual-pronged clinical progress in both adult and pediatric populations expands the potential market opportunity.

The rapid efficacy signals, particularly in the pediatric study, could accelerate the pathway to potential regulatory approval. Strong safety data combined with meaningful efficacy metrics across multiple endpoints strengthens the commercial viability of QRX003. Given the company's small market cap, positive clinical progression could trigger significant value appreciation.

• Clinical Data from First Subject Dosed Twice Daily with QRX003 in Open Label Study Is Positive at Mid-point of Testing
• Significant Improvement in Skin Appearance Observed in Pediatric Study after Only 12 Days of Dosing with QRX003

ASHBURN, Va., Dec. 18, 2024 (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 two of its ongoing Netherton Syndrome clinical studies.

Data from the first subject being dosed twice-daily in Quoin’s ongoing open label study is suggestive of clinical efficacy across a number of measured endpoints after six weeks of dosing with QRX003, which is the midpoint of testing. At baseline, prior to dosing, the subject’s Modified Ichthyosis Area of Severity Index (MIASI) was 18. Following six weeks of dosing with QRX003, the subject’s MIASI had been reduced to 4. In addition, the Investigator’s Global Assessment (IGA) of disease severity prior to dosing classified the subject as ‘moderate’. After six weeks of dosing with QRX003, the IGA for the subject was classified as ‘mild’. The subject’s pruritus or itch assessment at baseline was 7 out of a maximum of 11 based on the Worst Itch Numeric Rating Scale (WINRS) and was reduced to 4 at the treatment midpoint. Finally, the patient satisfaction scores across assessed metrics were highly positive. No safety concerns were reported for the subject during this initial testing period.

In addition, after the initial 12 days of dosing in Quoin’s ongoing 12-week Investigator Pediatric Study, a significant improvement was observed in the skin area treated with QRX003 versus the non-treated area. Specifically, at baseline prior to dosing with QRX003, the IGA assessment of the subject’s skin was classified as ‘severe’. After 12 days of treatment with QRX003, this was improved to ‘mild-moderate’, representing a very rapid improvement in skin appearance. There have been no adverse events or safety concerns reported to date.

Quoin CEO, Dr. Michael Myers, said, “While cautioning that this is interim clinical data from a very limited number of subjects, we are very pleased to announce these early results from two of our ongoing Netherton Syndrome clinical studies. Having previously announced positive data for subjects dosed once-daily for 12 weeks in our ongoing open-label study, we are excited to share initial data for the first subject dosed twice-daily to reach six weeks of dosing in this study. Although clear improvements were observed across all four measured endpoints, the reduction in MIASI from 18 at baseline to just 4 after six weeks of dosing with QRX003 is particularly noteworthy. Similarly, the reduction in pruritus severity from 7, out of a maximum of 11, to 4 after six weeks of dosing with QRX003 is also promising.

“Furthermore, it is very encouraging to observe such a clear improvement in skin condition in the investigator pediatric study, after just 12 days of being treated with QRX003. The change from an IGA classification of severe prior to dosing to mild-moderate after such a short period of time is highly encouraging as we seek to recruit additional pediatric subjects in Spain and the United Kingdom. We remain steadfast in our commitment to develop a safe and effective treatment for the Netherton Syndrome community.”

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,” “look forward to,” 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 development of a safe and effective treatment for the Netherton Syndrome community and Quoin’s belief that its products in development 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. 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 clinical studies may not generate the results anticipated, the Company ability to recruit additional pediatric subjects, or the clinical studies not generating data which is sufficiently robust and comprehensive to support an NDA filing and the Company’s ability to obtain regulatory approvals. More detailed information about the risks and uncertainties affecting the Company is summarized 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:
Investor Relations
PCG Advisory
Jeff Ramson
646-863-6893
jramson@pcgadvisory.com

FAQ

What are the key results from QNRX's Netherton Syndrome trials in December 2023?

The trials showed significant improvements in patients treated with QRX003, including MIASI score reduction from 18 to 4, improved IGA ratings from 'moderate' to 'mild' in adults, and from 'severe' to 'mild-moderate' in pediatric patients.

How effective is QRX003 in treating pediatric Netherton Syndrome patients?

Initial results show QRX003 is effective in pediatric patients, with significant skin improvement observed after just 12 days of treatment, improving from 'severe' to 'mild-moderate' on the IGA scale.

What improvements were seen in QNRX's twice-daily dosing study of QRX003?

After six weeks of twice-daily dosing, the patient showed MIASI reduction from 18 to 4, IGA improvement from 'moderate' to 'mild', and pruritus reduction from 7 to 4 on the WINRS scale.

Are there any safety concerns with QNRX's QRX003 treatment?

No safety concerns or adverse events were reported in either the adult or pediatric studies during the testing period.