Quoin Pharmaceuticals Announces Additional Positive Clinical Data from Open-Label, Single-Arm Clinical Trial in Netherton Syndrome
Available Clinical Data from Six Subjects Demonstrates Well Defined Efficacy Signals Across a Number of Study Endpoints
No Safety Concerns Observed in the Study to Date
ASHBURN, Va., Oct. 24, 2023 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a specialty pharmaceutical company focused on rare and orphan diseases, announces additional positive clinical data from its ongoing open-label study evaluating the safety and efficacy of QRX003 as a potential treatment for Netherton Syndrome (NS).
This study, which is being conducted under Quoin’s open Investigational New Drug (IND) application, is an open-label, single-arm trial that is evaluating 10 NS patients dosed with QRX003 over a twelve week period. All subjects in the study are continuing to receive off-label systemic therapy for the duration of the trial.
Of the available data from six evaluable subjects, five demonstrated a well-defined positive improvement in pruritus, or itch, with those five subjects reporting absent or negligible pruritus on completion of dosing with QRX003 based on the endpoint scoring system. The sixth subject’s pruritus was effectively unchanged on completion of dosing with QRX003. In the Investigator assessed skin scoring system, all six patients experienced an improvement in skin appearance, with three of the six subjects demonstrating improvement throughout the study, while for the other three subjects, signs of improvement were exhibited at various points throughout the dosing period. Importantly, all of the 6 subjects indicated a positive impression of QRX003 across a number of key metrics.
The initial safety data across all patients is highly supportive of further product development with no reported treatment related adverse events impacting the study.
Quoin CEO, Dr. Michael Myers, said, “While acknowledging that this is still early stage data, we are very pleased to announce today additional positive results from our ongoing open-label study in Netherton Syndrome. In August of this year, we announced positive data from the first subject, who completed the 12-week dosing period in the study, and I’m now delighted to provide a further positive update for an additional five subjects.
“The results for pruritus are particularly encouraging, as this often causes significant distress for Netherton patients. In addition, there are well defined efficacy signals across the other evaluated endpoints including skin appearance and the subjects’ own impression of how QRX003 performed throughout the study, indicating that QRX003 may have the potential to become an effective treatment for Netherton Syndrome. Furthermore, the absence of any safety concerns from the study to date is a positive indicator for the ongoing clinical development of the product.
“With these results in hand, we are moving into an optimization phase for this study as well as our ongoing double blinded clinical trial and we look forward to providing updated information on this, at the appropriate time.
“Netherton Syndrome is a devastating and sometimes fatal rare disease. Quoin is fully committed to delivering what may have the potential to become the first approved treatment option to this underserved patient population.”
The active ingredient in QRX003 is a broad-spectrum serine protease inhibitor, whose mechanism of action is intended to down-regulate the hyperactivity of skin kallikreins, leading to a more normalized rate of skin shedding. If proven to be safe and effective, long term daily application of QRX003 could lead to the development of a more normally functioning skin barrier and a significant improvement in the quality of life of Netherton patients.
For more information about Quoin’s clinical trials in Netherton Syndrome, 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.
About Netherton Syndrome
Netherton Syndrome is a rare and sometimes fatal skin disease for which there is no approved treatment, and no cure. It is caused by a mutation of the SPINK5 gene which leads to uncontrolled skin shedding, resulting in a highly porous and ineffective skin barrier. Symptoms are present at birth and include red, scaly skin. Other symptoms include outbreaks of red, circular scaly rashes, thin, fragile hair (bamboo hair), and immune reactions such as hay fever, asthma, severe pruritus (itchy skin), and eczema. Dehydration and infection are common and can be serious or fatal. Babies tend to grow slowly and have poor weight gain. Netherton Syndrome is inherited in an autosomal recessive pattern.
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. 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. More detailed information about the risks and uncertainties affecting the Company is contained under the heading "Risk Factors" included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 that the Company filed with the SEC. One should not place undue reliance on these forward-looking statements, which speak only as of the date on which they were made. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. 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.
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