Rare and Orphan Diseases
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Quoin Pharmaceuticals Announces Achievement of Topical Rapamycin Target Loadings for Two Proprietary Delivery Technologies
Globenewswire· 2025-11-11 13:30
Core Insights - Quoin Pharmaceuticals Ltd. has successfully achieved target loading concentrations of 4% and 5% for its topical rapamycin delivery technologies, which are set to advance into clinical trial and stability batch manufacturing by Q4 2025, with clinical testing expected to begin in the first half of 2026 [1][2] Company Overview - Quoin Pharmaceuticals Ltd. is a late 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, and various vascular malformations [3] Product Development - The company has developed two proprietary delivery systems for rapamycin, achieving a 4% w/w loading in a topical formulation and a 5% w/w loading in a dermal patch system, which may offer competitive advantages over existing formulations [1][2] - Initial clinical indications for testing include Microcystic Lymphatic Malformations and Venous Malformations, both of which currently lack FDA-approved treatments [1][2] Clinical and Financial Milestones - Quoin plans to initiate formal clinical development for its identified indications and has recently completed a capital raise to fund ongoing studies, including pivotal studies for Netherton Syndrome [2]
Quoin Pharmaceuticals Announces Initial Positive Clinical Data for QRX003 from Pediatric Peeling Skin Syndrome Study
Globenewswire· 2025-05-14 11:30
Core Insights - Quoin Pharmaceuticals Ltd. announced positive initial clinical data from its ongoing Investigator Pediatric Peeling Skin Syndrome clinical study, showing significant skin healing after 12 weeks of treatment with QRX003 [1][4]. Clinical Study Results - After 12 weeks of treatment, the Modified Ichthyosis Area of Severity Index (M-IASI) score improved from 36 to 12, indicating a significant reduction in skin symptoms [2]. - The Investigator's Global Assessment (IGA) score improved from 4 (Severe) to 2 (Mild), demonstrating a clinically meaningful improvement [2][5]. - The Children's Dermatology Life Quality Index (CDQLI) score decreased from 19 to 11, reflecting a positive impact on the patient's quality of life [2][5]. Treatment Tolerance and Future Plans - QRX003 has been well tolerated by the patient, with no adverse events reported, and the patient is expected to continue treatment with further assessments scheduled after 24 weeks [4][5]. - The company plans to expand the study to include additional pediatric subjects in other countries and advance the clinical development of QRX003 for Peeling Skin Syndrome [5][6]. Company Overview - Quoin Pharmaceuticals Ltd. is focused on developing therapeutic products for rare and orphan diseases, with a pipeline that includes treatments for various conditions such as Netherton Syndrome and Peeling Skin Syndrome [8].