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Quoin Pharmaceuticals Announces Highly Positive ‘Whole Body' Clinical Data from Ongoing Pediatric Netherton Syndrome Study
GlobeNewswire News Room· 2025-02-27 12:30
Core Viewpoint - Quoin Pharmaceuticals has announced highly positive clinical data from its ongoing Investigator Pediatric Netherton Syndrome study, showing significant improvement in patients' disease state after two weeks of treatment with QRX003, a topical lotion [1][4]. Group 1: Clinical Data and Results - Dramatic improvement in the patient's disease state was observed, with clear visual evidence of skin healing after just two weeks of whole-body application of QRX003 [1]. - Both key clinical endpoints, Investigator's Global Assessment (IGA) and Pruritus, showed highly significant clinical improvements from baseline after two weeks of treatment [2]. - The patient's IGA score improved from 7 at baseline to between 1-2 after treatment, indicating almost complete elimination of the visual effects of the disease [4]. - The Pruritus score decreased from 5 to 1, reflecting a significant reduction in itch severity [2][4]. - The patient has discontinued previously required medications, including antihistamines, glucocorticoids, and antivirals, and has not needed antibiotics since treatment began [3][4]. Group 2: Patient Impact - The patient is now experiencing zero nightly sleep disturbances for the first time in her life, further indicating the potential efficacy of QRX003 [3][4]. - The results suggest that QRX003 may directly target the root cause of Netherton Syndrome, providing patients with a chance to live free from the disease's severe impacts [4]. Group 3: Company Overview and Future Plans - Quoin Pharmaceuticals is a clinical-stage specialty pharmaceutical company focused on developing treatments for rare and orphan diseases, including Netherton Syndrome [8]. - The company plans to expand the study to include additional pediatric subjects in other countries, aiming to further validate the encouraging results observed [4].
Quoin Pharmaceuticals Announces Highly Positive 'Whole Body' Clinical Data from Ongoing Pediatric Netherton Syndrome Study
Newsfilter· 2025-02-27 12:30
Core Viewpoint - Quoin Pharmaceuticals has announced highly positive clinical data from its ongoing Investigator Pediatric Netherton Syndrome study, showing significant improvements in patients' disease state after two weeks of treatment with QRX003, a topical lotion designed for rare skin disorders [1][4]. Clinical Data Summary - The study reported dramatic improvements in both key clinical endpoints: Investigator's Global Assessment (IGA) and Pruritus, with scores significantly reduced from baseline after two weeks of treatment [2]. - For the first pediatric patient, the IGA score improved from 7 at baseline to between 1-2 after treatment, indicating almost complete elimination of visual disease effects [4]. - The Pruritus score decreased from 5 to 1, reflecting a transition from "chronically debilitating itch" to a "highly tolerable and non-intrusive" level [4]. Patient Outcomes - Following treatment with QRX003, the patient has discontinued all previously required medications, including antihistamines, glucocorticoids, and antivirals, and has not needed antibiotics since treatment began [3][4]. - The patient is now experiencing zero nightly sleep disturbances for the first time, further indicating the treatment's potential efficacy [3][4]. Product Information - QRX003 is a topical lotion formulated with a proprietary delivery technology that contains a broad-spectrum serine protease inhibitor, aimed at normalizing skin-shedding processes and strengthening the skin barrier [7]. - Netherton Syndrome, the condition QRX003 targets, is a rare hereditary skin disorder caused by a mutation in the SPINK5 gene, leading to severe skin barrier defects and other complications [5][6]. Company Overview - Quoin Pharmaceuticals is a clinical-stage specialty pharmaceutical company focused on developing treatments for rare and orphan diseases, with a pipeline that includes products targeting various conditions, including Netherton Syndrome [8].
Quoin Pharmaceuticals Launches First Episode of "NETHERTON NOW" Video Series to Raise Awareness for Netherton Syndrome
Newsfilter· 2025-02-25 12:30
Core Viewpoint - Quoin Pharmaceuticals is launching a video series titled "Living with Netherton" to raise awareness about Netherton Syndrome, a rare genetic disease, coinciding with Rare Disease Day on February 28, 2025 [1][2][8]. Company Initiatives - The "Living with Netherton" series is part of the broader NETHERTON NOW campaign aimed at increasing understanding and advocacy for Netherton Syndrome [2][5]. - Future episodes will continue to highlight patient and family experiences, emphasizing the need for awareness and treatment options [4]. Patient Experience - The first episode features Carmon McTigue, whose son faced a long and challenging journey to diagnosis, ultimately revealing the lack of approved treatments for Netherton Syndrome [3][5]. - The family's story illustrates the severe impacts of the disease, including multiple hospitalizations and complications such as MRSA [3][6]. Disease Overview - Netherton Syndrome is caused by mutations in the SPINK5 gene, leading to severe symptoms including excessive skin shedding and a high risk of complications [6][10]. - Up to 20% of infants born with Netherton Syndrome do not survive, highlighting the urgent need for increased awareness and research [6]. Product Development - Quoin Pharmaceuticals is focused on developing treatments for rare diseases, with its lead candidate QRX003 currently in four clinical trials [7][11]. - QRX003 is a topical lotion designed to improve skin barrier function in patients with Netherton Syndrome [10].
Quoin Pharmaceuticals Launches First Episode of “NETHERTON NOW” Video Series to Raise Awareness for Netherton Syndrome
Globenewswire· 2025-02-25 12:30
Core Viewpoint - Quoin Pharmaceuticals is launching a video series titled "Living with Netherton" to raise awareness about Netherton Syndrome, a rare genetic disease, coinciding with Rare Disease Day on February 28, 2025 [1][2][8] Company Initiatives - The "Living with Netherton" series is part of the broader NETHERTON NOW campaign aimed at increasing understanding and advocacy for Netherton Syndrome [2][5] - Future episodes will continue to highlight patient and family experiences, emphasizing the need for awareness and treatment options [4] Patient Experience - The first episode features Carmon McTigue, whose son faced a long and challenging journey to diagnosis, ultimately revealing the lack of approved treatments for Netherton Syndrome [3][5] - The story illustrates the severe impacts of the disease, including multiple hospitalizations and complications such as MRSA [3][6] Disease Overview - Netherton Syndrome is caused by mutations in the SPINK5 gene, leading to severe symptoms including excessive skin shedding and a high risk of complications [6][10] - Up to 20% of infants born with Netherton Syndrome do not survive, highlighting the urgent need for increased awareness and research [6] Product Development - Quoin's lead candidate, QRX003, is currently in four clinical trials, with recent data suggesting its potential efficacy as a treatment for Netherton Syndrome [7][10] - QRX003 is designed to improve skin barrier function and normalize skin shedding in patients [10] Community Engagement - The NETHERTON NOW website serves as a resource hub for patients and families, offering educational materials and updates on research advancements [9][12]
Quoin Pharmaceuticals Launches 'NETHERTON NOW' Campaign to Raise Awareness of Netherton Syndrome, a Rare Genetic Disease with No Approved Treatment or Cure
Newsfilter· 2025-02-04 12:30
Core Viewpoint - Quoin Pharmaceuticals has launched the "NETHERTON NOW" campaign to raise awareness about Netherton Syndrome, a genetic disease with significant misdiagnosis rates and high mortality among affected infants [1][4]. Company Overview - Quoin Pharmaceuticals Ltd. is a clinical-stage specialty pharmaceutical company focused on developing treatments for rare and orphan diseases, including Netherton Syndrome [9]. - The company is conducting four ongoing clinical studies to evaluate the safety and efficacy of its product QRX003 for Netherton Syndrome [3]. Netherton Syndrome - Netherton Syndrome is characterized by severe skin shedding, red scaly skin, and complications such as dehydration, infections, asthma, and allergies [2][7]. - The condition is caused by a mutation in the SPINK5 gene, leading to significant challenges for patients and their families [6]. QRX003 Product Development - QRX003 is a topical lotion designed to improve skin barrier function and normalize skin shedding, addressing the core issues of Netherton Syndrome [8]. - Recent clinical data has shown promising results for QRX003, with photographic evidence demonstrating significant skin improvement after 12 weeks of treatment [3]. Campaign Objectives - The "NETHERTON NOW" campaign aims to increase public awareness, foster advocacy, and provide educational resources about Netherton Syndrome [4][5]. - The campaign also serves as a platform for patients and families to share experiences and connect with one another [3]. Future Directions - Quoin is preparing for a "whole body" clinical study of QRX003, which has received FDA clearance, marking a significant step in the product's development [3]. - The company is committed to building a robust data package to support regulatory approval in the US, EU, and other countries [3].
Quoin Pharmaceuticals Launches ‘NETHERTON NOW’ Campaign to Raise Awareness of Netherton Syndrome, a Rare Genetic Disease with No Approved Treatment or Cure
Globenewswire· 2025-02-04 12:30
Core Viewpoint - Quoin Pharmaceuticals Ltd. has launched the "NETHERTON NOW" campaign to raise awareness about Netherton Syndrome, a genetic disease with significant misdiagnosis rates and high mortality risk among infants [1][4]. Company Overview - Quoin Pharmaceuticals is a clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, with a commitment to addressing unmet medical needs [9]. - The company is developing therapeutic products, including QRX003, aimed at treating Netherton Syndrome and other rare conditions [9]. Netherton Syndrome Insights - Netherton Syndrome is characterized by severe skin issues, including excess skin shedding, red scaly skin, and a high risk of dehydration and infections [2][7]. - The condition is caused by a mutation in the SPINK5 gene, leading to significant physical, emotional, and social challenges for patients and their families [6][4]. Clinical Development - Quoin is conducting four ongoing clinical studies to evaluate the safety and efficacy of QRX003 in patients with Netherton Syndrome [3]. - Recent clinical data has shown promising results for QRX003, with photographic evidence demonstrating significant skin improvement after 12 weeks of treatment [3]. - A "whole body" clinical study has been approved by the U.S. Food and Drug Administration and will be led by Dr. Amy Paller at Northwestern University [3]. Campaign Objectives - The "NETHERTON NOW" campaign aims to increase public awareness, foster advocacy, and provide educational resources about Netherton Syndrome [4][5]. - The campaign also seeks to address the long diagnostic delays faced by patients and the lack of approved treatments [4]. Product Information - QRX003 is a topical lotion designed to improve skin barrier function and normalize skin shedding, addressing the core issues of Netherton Syndrome [8]. - The product utilizes proprietary delivery technology to act as a serine protease inhibitor, compensating for the absence of the LEKTI protein in affected patients [8].
Quoin Pharmaceuticals Announces Further Clinical Evidence of QRX003 Effectiveness in Netherton Syndrome
Newsfilter· 2025-01-23 13:30
Four Weeks After Discontinuation of Treatment with QRX003 Complete Reversal of Positive Clinical Improvements Across all Measured Endpoints ObservedData 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 e ...
Quoin Pharmaceuticals Announces Further Positive Clinical Data from Ongoing Pediatric Netherton Syndrome Study
Globenewswire· 2025-01-14 13:30
Continued Significant Improvement in Skin Appearance Observed in StudySubject’s Disease Classification Improved from “Severe” to “Mild” after 6 Weeks Dosing Treatment area expanded from 20% Body Surface Area to “Whole Body” ASHBURN, Va., Jan. 14, 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 positive interim clinical data from its ongoing Investigator Pedia ...
PRISM MarketView Spotlights Quoin Pharmaceuticals CEO Dr. Michael Myers as Specialty Pharma Shares News of Highly Positive Interim Data in Most Advanced, Comprehensive Netherton Syndrome Trials
Newsfilter· 2025-01-07 19:29
NEW YORK, Jan. 07, 2025 (GLOBE NEWSWIRE) -- PRISM MarketView has released an exclusive interview with Quoin Pharmaceuticals Ltd. (NASDAQ:QNRX) CEO Dr. Michael Myers, who shares the company's notable interim data in its ongoing Netherton Syndrome clinical trials. Quoin, a clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, is addressing unmet medical needs for those with Netherton Syndrome (NS), a rare and sometimes fatal skin disease for which there is currently no approved ...
Quoin Pharmaceuticals Announces Additional Positive Interim Data from Ongoing Open-Label Netherton Syndrome Clinical Study
Globenewswire· 2025-01-06 13:30
Highly Positive Clinical Data on Completion of Testing for First Subject Dosed Twice Daily with QRX003 in Open-Label StudyDemonstrated Clinical Benefits from QRX003 Observed Across All Measured EndpointsBefore and After Dosing with QRX003 Photographs Available for Review On WebsiteNo 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 pharmace ...