Core Viewpoint - Quoin Pharmaceuticals Ltd. has made significant advancements in 2024, particularly in the clinical development of QRX003 for Netherton Syndrome, showcasing promising clinical data and expanding its pipeline with new patent filings for topical rapamycin formulations [2][4][5]. Recent Accomplishments - The company filed U.S. and international patent applications for novel topical rapamycin formulations targeting microcystic lymphatic malformations, venous malformations, and angiofibromas, utilizing its proprietary Invisicare® delivery technology [4]. - Positive clinical data from a pediatric Netherton Syndrome study indicated that the first patient experienced transformational improvement after two weeks of whole-body application of QRX003, leading to the discontinuation of previously required medications [5]. - The launch of the 'NETHERTON NOW' awareness campaign aimed to raise awareness about Netherton Syndrome and provide educational resources for the community [6]. Clinical Development Highlights - Quoin received FDA clearance for a new whole-body clinical study for QRX003, which will evaluate the safety and efficacy of the treatment on over 80% of the body surface area in Netherton Syndrome patients [10]. - Interim data from ongoing studies showed significant clinical responses, with no adverse events reported, reinforcing the effectiveness of QRX003 [11][8]. Financial Highlights - As of December 31, 2024, Quoin had approximately $14.1 million in cash, cash equivalents, and marketable securities, expected to fund operations into the second quarter of 2026 [15]. - The net loss for the quarter ended December 31, 2024, was approximately $2.3 million, compared to $2.0 million for the same quarter in 2023, while the annual net loss was $9.0 million, up from $8.7 million in 2023 [15][19].

Core Viewpoint - Quoin Pharmaceuticals Ltd. is set to release its financial results for Q4 and the full year of 2024 on March 13, 2025, which will include operational updates and financial highlights [1][2]. Company Overview - Quoin Pharmaceuticals Ltd. is a late clinical-stage specialty pharmaceutical company focused on developing and commercializing therapeutic products for rare and orphan diseases [3]. - The company aims to address unmet medical needs for patients and their communities [3]. - Quoin's pipeline includes four products in development targeting various rare and orphan indications such as Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Scleroderma, and Epidermolysis Bullosa [3].

Core Viewpoint - Quoin Pharmaceuticals Ltd. has filed U.S. and International patent applications for novel topical rapamycin formulations aimed at treating rare diseases, including microcystic lymphatic malformations, venous malformations, and angiofibromas, utilizing its proprietary Invisicare delivery technology [1][3]. Company Overview - Quoin Pharmaceuticals Ltd. is a clinical-stage specialty pharmaceutical company focused on developing treatments for rare and orphan diseases, with a pipeline that includes products targeting conditions such as Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Scleroderma, and Epidermolysis Bullosa [9]. Product Development - The company is advancing its lead product, QRX003, which is in late-stage clinical testing as a potential treatment for Netherton Syndrome, and believes it could be the first approved treatment for this condition [2][3]. - Quoin is currently enrolling patients in three clinical trials under an open Investigational New Drug (IND) application for QRX003 [4]. Technology and Innovation - The Invisicare delivery technology is designed to enhance the penetration of rapamycin into the dermis, potentially improving clinical efficacy compared to other topical formulations that have underperformed [2][3]. - The company plans to submit IND applications for at least two additional target indications this year and to initiate clinical development promptly [3][7]. Target Indications - The company is focusing on microcystic lymphatic malformations, venous malformations, and angiofibromas as potential indications for its novel topical formulations [1][7]. - Microcystic lymphatic malformations occur in 1 in 6000 to 16,000 patients, while venous malformations have an incidence of 1 to 2 in 10,000 and a prevalence of 1% [5][6].

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].

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].

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].

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]

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].

Multi-pronged media campaign to raise awareness about the disease and its impact on patients, family members and caregivers.Interactive, stand-alone NETHERTON NOW website launched.Quoin's QRX003 product is on track to obtain the first regulatory approval for Netherton Syndrome. ASHBURN, Va., Feb. 04, 2025 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ:QNRX) (the "Company" or "Quoin"), a clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, has launched the "NETHERTON N ...

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 ...