Latest news with #NethertonSyndrome
Yahoo
4 days ago
- Health
- Yahoo
Quoin Pharmaceuticals Releases Fourth Episode in NETHERTON NOW Video Series Featuring International Patient Advocate Mandy Aldwin-Easton
New Episode Highlights the Painful, Often Misunderstood Reality of Living with Netherton Syndrome and the Urgent Need for Effective Treatments ASHBURN, Va., Aug. 12, 2025 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) ('Quoin' or the 'Company'), a late clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, today announced the release of the fourth episode in its NETHERTON NOW video series. This installment features Mandy Aldwin-Easton, a Netherton Syndrome patient, and a widely recognized, passionate advocate for people living with rare diseases. Mandy serves as Medical and Communications Manager and Co-founder of the UK-based Ichthyosis Support Group and is President of the European Network for Ichthyosis. In the video, Mandy shares her personal journey living with Netherton Syndrome, from delayed diagnosis, severe, chronic pain, multiple hospitalizations to the profound long-term impact this disease has had on her life. 'One thing we don't talk about is how incredibly painful skin is when it doesn't work properly,' Mandy explains in the video. 'Imagine millions of paper cuts all over your body. It's just so highly unpredictable. The one thing anybody wants with a rare disease like Netherton Syndrome is hope—that better treatments are on the way.' Mandy's story underscores the severe physical and emotional toll of the disease. Despite having a highly supportive family and medical advocacy, she has experienced extreme flare-ups, prolonged hospitalizations, and a lack of targeted treatment options throughout her entire life. As an adult, she continues to navigate the complexities of a disease that many still fail to recognize as life-threatening. 'I couldn't go out because my skin was falling off me. It was so incredibly painful that it was just unbearable to walk,' she recalls. 'Even with hospital stays and treatments, no one really knew how to care for my skin.' 'Mandy has long been a pillar of the ichthyosis and Netherton Syndrome communities,' said Denise Carter, Co-Founder and Chief Operating Officer of Quoin Pharmaceuticals. 'Netherton Syndrome is not just a rare skin disease. It is a relentless and punishing condition marked by excruciating pain, debilitating flare-ups, recurrent infections, and emotional trauma. The toll on patients and their families is immense, not only physically and emotionally but also financially, requiring lifelong care, frequent hospitalizations, and ongoing interventions that place a heavy burden on families and healthcare systems. Beyond her personal resilience, Mandy's unwavering advocacy has made her a powerful voice for this underserved and often overlooked population. We are honored to feature her in our NETHERTON NOW series and remain steadfast in our mission to bring awareness and hope to those living with this devastating disease.' The release of this video comes as Quoin continues to advance its lead product candidate, QRX003, in multiple pivotal clinical trials for the treatment of Netherton Syndrome. Recently reported data from ongoing studies have shown highly encouraging improvements in key clinical outcomes. 'Mandy's story reflects the extraordinary strength and perseverance of patients who have endured a lifetime of suffering without access to an approved treatment,' said Dr. Michael Myers, Chief Executive Officer and Co-Founder of Quoin Pharmaceuticals. 'As we advance QRX003 through multiple late-stage pivotal trials, voices like Mandy's reinforce the urgency of our mission. Quoin is committed to delivering a meaningful treatment for the severely underserved Netherton Syndrome community and to driving forward research that addresses the significant unmet needs across a broader range of rare skin disorders.' The full video featuring Mandy Aldwin-Easton is available at: About QRX003QRX003 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, visit: About Quoin Pharmaceuticals Pharmaceuticals Ltd. is a late 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, SAM Syndrome, Palmoplantar Keratoderma, Scleroderma, Microcystic Lymphatic Malformations, Venous Malformations, Angiofibroma and others. For more information, visit or LinkedIn for updates. Cautionary Note Regarding Forward-Looking StatementsThe 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: bringing awareness and hope to those living with Netherton Syndrome, continuing to advance QRX003 through multiple late-stage pivotal trials, delivering a meaningful treatment for the Netherton Syndrome community, driving forward research that addresses the significant unmet needs across a broader range of rare skin disorders 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, SAM Syndrome, Palmoplantar Keratoderma, Scleroderma, , Microcystic Lymphatic Malformations, Venous Malformations, Angiofibroma 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; the Company's ability to pursue its regulatory strategy; the Company's ability to obtain regulatory approvals for commercialization of product candidates or to comply with ongoing regulatory requirements; the Company's ability to complete clinical trials on time and achieve desired results and benefits as expected, the Company experiencing unanticipated or higher than expected clinical trial costs; and other factors discussed in the Company's Annual Report on Form 10-K for the year ended December 31, 2024 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 Myers, Ph.D., CEOmmyers@ Investor Contact:PCG AdvisoryJeff Ramsonjramson@ 863-6341
Yahoo
24-06-2025
- Business
- Yahoo
Quoin Pharmaceuticals Announces FDA Grants Rare Pediatric Disease Designation for QRX003 in Netherton Syndrome
ASHBURN, Va., June 24, 2025 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) ('Quoin' or the 'Company'), a late clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the Company's lead asset, QRX003, for the treatment of Netherton Syndrome. The designation reinforces the potential of QRX003 as a therapeutic candidate for a profoundly underserved pediatric population. It follows earlier regulatory recognition by the European Medicines Agency (EMA), which granted Orphan Drug Designation to QRX003 in May 2025. 'We are very pleased to announce the receipt of Rare Pediatric Disease Designation for QRX003 for Netherton Syndrome, a severe and underserved genetic disease,' said Dr. Michael Myers, Chief Executive Officer of Quoin Pharmaceuticals. 'The Quoin team is fully focused on completing our pivotal clinical studies and advancing QRX003 towards a New Drug Application as the first potential treatment for this terrible disease. We are highly encouraged by the promising efficacy data and clean safety profile seen to date.' The FDA's Rare Pediatric Disease Designation program is intended to encourage the development of new therapies for serious and life-threatening diseases that primarily affect individuals under 18 years of age. If a New Drug Application (NDA) for QRX003 is approved, upon reauthorization of the program Quoin may be eligible to receive a Priority Review Voucher (PRV), which can be redeemed to receive priority review for another marketing application or may be sold or transferred. About Netherton Syndrome in ChildrenNetherton Syndrome is a rare genetic disorder affecting approximately 1 in 200,000 newborns worldwide. Symptoms typically present at birth or in early infancy and persist throughout life. Affected infants often require prolonged hospitalization due to severe skin inflammation, dehydration, and increased risk of infection. The condition can be challenging to diagnose early, as it may resemble other dermatological or immunological disorders. Mortality in infancy is estimated at 10 to 20 percent, underscoring the serious clinical burden associated with the disease. About Quoin Pharmaceuticals Pharmaceuticals Ltd. is a late 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: or LinkedIn for updates. Cautionary Note Regarding Forward Looking StatementsThe 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: potential of QRX003 as a therapeutic candidate for a profoundly underserved pediatric population, completing the Company's pivotal clinical studies, advancing QRX003 towards a New Drug Application as the first potential treatment for Netherton Syndrome, if a New Drug Application for QRX003 is approved, upon reauthorization of the program, Quoin may be eligible to receive a Priority Review Voucher, 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 Company's ability to pursue its regulatory strategy; the Company's ability to obtain regulatory approvals for commercialization of product candidates or to comply with ongoing regulatory requirements; the Company's ability to complete clinical trials on time and achieve desired results and benefits as expected; and other factors discussed in the Company's Annual Report on Form 10-K for the year ended December 31, 2024 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 Myers, Ph.D., CEOmmyers@ Investor RelationsPCG AdvisoryJeff Ramsonjramson@ 863-6341Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
22-05-2025
- Business
- Yahoo
Quoin Pharmaceuticals Announces FDA Clearance to Initiate Second Pivotal Whole Body QRX003 Netherton Syndrome Clinical Study
Study CL-QRX003-002 will test whole-body application of QRX003 in conjunction with off-label systemic therapy in approximately 12-15 subjects. Study CL-QRX003-002 complements first whole-body pivotal study, CL-QRX003-003, being led by Dr. Amy Paller at Northwestern University and supported by up to six international sites that are expected to open, where subjects are being tested with QRX003 as monotherapy. Combined data from Studies CL-QRX003-002 and CL-QRX003-003 is anticipated to provide QRX003 with the broadest possible label opportunity post-approval as both monotherapy and adjunct therapy in conjunction with off-label systemic treatments. Five U.S. clinical sites have been opened, and six international sites are being opened. All currently have available cohorts of subjects ready to be enrolled into the studies facilitating accelerated recruitment. Quoin anticipates completion of recruitment into both pivotal studies by end of Q1 2026 and filing an NDA for regulatory approval later in the year. ASHBURN, Va., May 22, 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 FDA clearance to initiate a second Netherton Syndrome (NS) pivotal clinical study CL-QRX003-002 for QRX003. Quoin's lead product, QRX003, is a topical lotion formulated with a broad-spectrum serine protease inhibitor that has been shown to significantly downregulate the hyperactivity of the kallikreins in the skin responsible for the excessive skin shedding associated with this disease. Clinical data from Quoin's ongoing studies has demonstrated clear evidence of rapid, prolonged and almost complete skin healing following twice-daily application of QRX003 to the treatment areas along with the almost complete elimination of key symptoms such as chronic, debilitating pruritus and has facilitated zero nightly sleep disturbances. Study CL-QRX003-002 builds off of positive clinical data generated in Quoin's earlier stage open-label study where QRX003 was applied to the arms and lower legs or approximately 20% of each subject's body surface area (BSA). In this pivotal study, QRX003 will be applied to approximately 12-15 subjects twice daily to at least 80% of each of the subjects' BSA thereby generating substantially more safety and efficacy data per subject and will match how QRX003 is intended to be used if regulatory approval is obtained. All subjects recruited into this study are currently receiving off-label systemic therapy and will continue to remain on this treatment for the duration of the study. Study CL-QRX003-002 complements Quoin's other pivotal study, CL-QRX003-003, which is being conducted by Dr. Amy Paller, of Northwestern University, where QRX003 is being administered as monotherapy and subjects do not receive off-label systemic therapy. Quoin anticipates that combined both studies will recruit between 24-30 subjects in 6 U.S. sites and up to 6 international sites. Five of the U.S. sites are already open and the sixth is expected to be opened in June. All international sites are at an advanced stage of preparation for opening. Each of the U.S. and international sites has available cohorts of subjects that are ready to participate in both studies facilitating a rapid pace of recruitment which could enable Quoin to achieve its target of fully completing recruitment before the end of Q1 2026 and to file an NDA for regulatory approval later in the year. Quoin CEO, Dr. Michael Myers, added, 'I am very pleased to announce yet another important and significant milestone for Quoin. With FDA clearance to conduct this study, Quoin is the only company conducting two late-stage whole-body pivotal clinical studies in Netherton Syndrome patients. We believe that the combined data generated from Study CL-QRX003-003, where QRX003 is being administered as monotherapy and this study, CL-QRX003-002, where QRX003 is being tested in conjunction with off-label systemic therapy, has the potential to generate a highly comprehensive safety and efficacy data package that could support the regulatory approval of a treatment for this dreadful disease.' About Quoin Pharmaceuticals Pharmaceuticals Ltd. is a late 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: or LinkedIn for updates. Cautionary Note Regarding Forward Looking StatementsThe 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: study CL-QRX003-002 testing the whole-body application of QRX003 in conjunction with off-label systemic therapy in approximately 12-15 subjects, six international sites opening to support study CL-QRX003-003 testing subjects with QRX003 as monotherapy, combined data from Studies CL-QRX0003-002 and CL-QRX003 providing QRX003 with the broadest possible label opportunity post-approval as both monotherapy and adjunct therapy in conjunction with off-label systemic treatments, available cohorts of subjects being ready to be enrolled into the studies facilitating accelerated recruitment at the clinical sites, completing recruitment into both pivotal studies by end of Q1 2026, filing an NDA for regulatory approval later in the year, combined both studies recruiting between 24-30 subjects in 6 US sites and up to 6 international sites, opening a sixth US site in June, 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 Company's ability to pursue its regulatory strategy; the Company's ability to obtain regulatory approvals for commercialization of product candidates or to comply with ongoing regulatory requirements; the Company's ability to complete clinical trials on time and achieve desired results and benefits as expected; and other factors discussed in the Company's Annual Report on Form 10-K for the year ended December 31, 2024 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 Myers, Ph.D., CEOmmyers@ Investor RelationsPCG AdvisoryJeff Ramsonjramson@ 863-6341
Yahoo
06-05-2025
- Business
- Yahoo
Quoin Pharmaceuticals to Announce First Quarter 2025 Financial Results on Tuesday, May 13, 2025
Quoin Pharmaceuticals, Inc. ASHBURN, Va., May 06, 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 announced that it plans to release its financial results for the first quarter ended March 31, 2025, before the market opens on Tuesday, May 13, 2025. The announcement will include an operational update highlighting key achievements from the quarter, recent accomplishments, and financial highlights. About Quoin Pharmaceuticals Ltd. Quoin Pharmaceuticals Ltd. is a late 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, go to: For further information, contact: Quoin Pharmaceuticals Ltd. Michael Myers, Ph.D., CEO mmyers@ Investor Relations PCG Advisory Jeff Ramson jramson@ (646) 863-6341
Yahoo
30-04-2025
- Business
- Yahoo
Quoin Pharmaceuticals Regains Compliance with Nasdaq Minimum Bid Price Rule
ASHBURN, Va., April 30, 2025 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the 'Company' or 'Quoin'), a late clinical stage, specialty pharmaceutical company focused on the development and commercialization of therapeutic products that treat rare and orphan diseases, today announces that it has received written notice from the Listing Qualifications Department of The Nasdaq Stock Market LLC ('Nasdaq') stating that the Company has regained compliance with the minimum bid price requirement under Nasdaq Listing Rule 5550(a)(2). The notification from Nasdaq confirms that for the last 13 consecutive business days, the closing bid price of the Company's ordinary shares was at or above $1.00 per share. Accordingly, Nasdaq considers the matter closed. About Quoin Pharmaceuticals Pharmaceuticals Ltd. is a late 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, SAM Syndrome, Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa, Microcystic Lymphatic Malformations, Venous Malformations, Angiofibroma and others. For more information, visit: or LinkedIn for updates. Cautionary Note Regarding Forward Looking StatementsThe 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: 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, SAM Syndrome, Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa, Microcystic Lymphatic Malformations, Venous Malformations, Angiofibroma 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 the Company's 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, 2024 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 Myers, Ph.D., CEOmmyers@ Investor RelationsPCG AdvisoryJeff Ramsonjramson@ 863-6341Sign in to access your portfolio
