Quoin Pharmaceuticals reports complete reversal of clinical improvements in Netherton Syndrome patients after stopping QRX003 treatment.
Quiver AI Summary
Quoin Pharmaceuticals has announced new clinical data indicating the need for ongoing treatment with QRX003 for patients with Netherton Syndrome, as all positive effects observed during a 12-week treatment period were reversed within four weeks after stopping the medication. The data was collected from the first subject in an open-label study, demonstrating that the condition reverted to its baseline status after treatment was discontinued. This supports the company's assertion that QRX003 functions as a competitive broad-spectrum serine protease inhibitor crucial for maintaining clinical improvements. Quoin is moving forward with multiple clinical studies and aims to submit a New Drug Application for QRX003, which could potentially be the first approved treatment for Netherton Syndrome, a rare genetic skin disorder.
Potential Positives
- Further clinical evidence supports the potential efficacy of QRX003 in treating Netherton Syndrome, a rare and orphan disease.
- The press release highlights the necessity of ongoing treatment with QRX003 to maintain positive clinical effects, thus reinforcing the importance of the product in patient care.
- Quoin is actively conducting four clinical studies under an open Investigational New Drug (IND) application with the FDA, indicating progress towards regulatory approval.
- The company aims to submit a New Drug Application (NDA) for QRX003, potentially positioning itself to be the first to offer a treatment for Netherton Syndrome, addressing an unmet medical need.
Potential Negatives
- Complete reversal of clinical improvements after just four weeks post-treatment suggests that ongoing chronic treatment with QRX003 is necessary, which may hinder patient adherence and limit the product's attractiveness.
- The need for continuous treatment raises concerns about the long-term viability and sustainability of QRX003 as a therapeutic option, particularly for patients with rare diseases.
FAQ
What is QRX003?
QRX003 is a topical lotion designed to treat Netherton Syndrome by inhibiting serine proteases to improve skin barrier function.
How does QRX003 work?
It works by mimicking the function of the LEKTI protein, which helps regulate skin shedding and maintain skin barrier integrity.
What were the results of the QRX003 study?
The study showed positive clinical improvements after 12 weeks, but all benefits reversed four weeks post-treatment discontinuation.
What is Netherton Syndrome?
Netherton Syndrome is a rare genetic skin disorder leading to severe skin barrier defects, causing chronic inflammation and infections.
Are there any approved treatments for Netherton Syndrome?
No, currently there are no approved therapeutic treatments for Netherton Syndrome, making QRX003 a potentially groundbreaking option.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
$QNRX Insider Trading Activity
$QNRX insiders have traded $QNRX stock on the open market 17 times in the past 6 months. Of those trades, 17 have been purchases and 0 have been sales.
Here’s a breakdown of recent trading of $QNRX stock by insiders over the last 6 months:
- MICHAEL MYERS (Chief Executive Officer) has made 5 purchases buying 593,450 shares for an estimated $278,916 and 0 sales.
- DENISE P. CARTER (Chief Operating Officer) has made 5 purchases buying 593,291 shares for an estimated $278,737 and 0 sales.
- GORDON DUNN (Chief Financial Officer) has made 6 purchases buying 151,077 shares for an estimated $78,204 and 0 sales.
- ANTHONY JAMES CULVERWELL purchased 100,000 shares for an estimated $45,000
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$QNRX Hedge Fund Activity
We have seen 6 institutional investors add shares of $QNRX stock to their portfolio, and 5 decrease their positions in their most recent quarter.
Here are some of the largest recent moves:
- ALTIUM CAPITAL MANAGEMENT LP removed 103,127 shares (-100.0%) from their portfolio in Q3 2024, for an estimated $50,841
- VIRTU FINANCIAL LLC added 25,165 shares (+inf%) to their portfolio in Q3 2024, for an estimated $12,406
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Full Release
- Four Weeks After Discontinuation of Treatment with QRX003 Complete Reversal of Positive Clinical Improvements Across all Measured Endpoints Observed
- Data Supports QRX003 Mechanism of Action as a Competitive Broad Spectrum Serine Protease Inhibitor
ASHBURN, Va., Jan. 23, 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 further clinical evidence of the potential efficacy of QRX003 in Netherton Syndrome.
In December 2024 and January 2025, Quoin announced positive data from the mid-point and at completion of 12 weeks of testing the first subject dosed twice-daily in Quoin’s ongoing open-label study which demonstrated evidence of clear improvement across all measured clinical endpoints. Today Quoin is sharing additional data on that subject at 4 weeks post-discontinuation of treatment with QRX003. As the table below illustrates all of the positive clinical benefits observed after 12 weeks of testing with QRX003 were completely reversed by 4 weeks after discontinuation of treatment resulting in the subject’s disease state reverting to the baseline status observed prior to QRX003 treatment. These results strongly indicate that ongoing, chronic treatment with QRX003 is necessary for a continued positive clinical outcome in Netherton Syndrome patients.
Table 1: First Patient Data from Open Label Study Part B- Dosed Twice Daily with QRX003
End Point | Baseline |
6 weeks
(Treatment period midpoint) |
12 weeks
(End of treatment period) |
4 weeks post
discontinuation of treatment |
M-IASI* | 18 | 4 | 3 | 18 |
WINRS** | 7 | 4 | 2 | 8 |
IGA*** | Moderate | Mild | Almost Clear | Moderate |
*
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.
Quoin CEO, Dr. Michael Myers, said, “This data set provides the clearest clinical evidence to date that the active ingredient in QRX003 is a competitive broad spectrum serine protease inhibitor and that discontinuation of treatment with QRX003 results in a complete reversal of all clinical benefits derived from continued treatment with the product. The rapid reversal of all observed clinical benefits to baseline disease status after just 4 weeks of treatment discontinuation serves to further underscore that chronic, whole-body treatment with QRX003 is potentially required to maintain the positive clinical outcomes observed in our clinical study. These results increase our commitment to complete the clinical development of QRX003 as expeditiously as possible with a goal of making the product widely available to the Netherton community once approved.
With four clinical studies in Netherton subjects underway, three of which are being conducted under an open Investigational New Drug (IND) application with the FDA, we believe Quoin is assembling a broad and diverse clinical data package to support a New Drug Application (NDA) filing for QRX003 as potentially the first approved treatment for Netherton Syndrome.”
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://quoinpharma.com/pipeline/#trials
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, the potential efficacy of QRX003 as a treatment for Netherton Syndrome, the goal of making the product widely available to the Netherton community once approved, we believe Quoin is assembling a broad and diverse clinical data package to support a New Drug Application (NDA) filing for QRX003 as potentially the first approved treatment for Netherton 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
[email protected]
Investor Relations
PCG Advisory
Jeff Ramson
[email protected]
(646) 863-6341