By M. Marin
NASDAQ:QNRX
READ THE FULL QNRX RESEARCH REPORT
Ongoing studies demonstrate evidence of benefits of QRX003
Quoin Pharmaceuticals (NASDAQ:QNRX) has attained several milestones recently, including extending its cash runway into 2027, attaining Orphan Drug Designation for QRX003 in Netherton Syndrome in the US & EU as it generates positive data from the ongoing studies it is conducting, streamlining product pipeline, and hiring a new CFO to support expected upcoming commercialization activities.
Recent measures to support development and commercialization
- Extended cash runway into 2027
- Generated positive data from ongoing studies
- Streamlined product pipeline
- New CFO to support commercialization activities
- Launched Netherton Syndrome awareness campaign
- Attained Orphan Drug Designation for QRX003 in Netherton Syndrome
- Achieved target loading concentrations for 2 topical rapamycin delivery technologies
- Reported positive interim data, recruited 3 new patients in investigator led pediatric NS study
The company’s lead asset, QRX003, is being evaluated for the treatment of Netherton Syndrome (NS), pediatric NS, and Peeling Skin Syndrome (PSS). QRX003 is a topical lotion with a broad-spectrum serine protease inhibitor that has demonstrated the ability to significantly downregulate the hyperactivity of the kallikreins in the skin that are responsible for the excessive skin shedding that is associated with NS and other dermatological diseases. QNRX has active Netherton Syndrome (NS) studies that are being conducted concurrently under an FDA open Investigational New Drug (IND) application to assess QRX003 for treatment of NS.
Quoin expects NDA submission in 2026
Last month, the FDA granted Orphan Drug Designation (ODD) to QRX003 for the treatment of NS. Previously, the European Medicines Agency (EMA) granted QRX003 Orphan Drug Designation in May 2025. The FDA designation provides certain tax credits for qualified clinical testing, waiver or partial payment of FDA application fees, and seven years of market exclusivity, if approved, among other benefits. The FDA had previously granted QRX003 Rare Pediatric Disease (RPD) Designation for the treatment of NS.
The company believes these designations reinforce the potential of QRX003 as a therapeutic candidate as it advances clinical studies towards a potential New Drug Application (NDA) for QRX003 as the first approved treatment for NS. The potential for an expedited regulatory pathway is supported by the significant increase in approvals of products to treat rare and orphan diseases in recent years.
Demonstrating evidence of benefits of QRX003; currently no NS treatments are approved
Quoin is currently conducting two pivotal clinical studies for QRX003 in Netherton Syndrome in the U.S., Europe, and the Middle East. Full enrollment is expected in early to mid-1Q26.
Clinical data thus far 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.
There are no approved NS treatments currently, and Quoin is optimistic about QRX003’s prospects to be the first approved treatment for NS. Current standard of care seeks to manage the associated symptoms and complications of NS, but the NIH notes that “[t]here is no specific treatment” for Netherton syndrome. The overall patient population is fairly sizable. Quoin estimates that there are about 6,000-7,000 patients in the U.S. and EU suffering with Netherton syndrome. Data is not readily available, and these estimates are within the range of other published estimates. The National Organization for Rare Disorders (NORD) indicates that the actual number of people suffering from Netherton syndrome might exceed the number of reported cases because it is often undiagnosed.
Moving forward with Rapamycin & advancing pediatric Netherton Syndrome study
In addition to studying QRX003 for NS, Quoin is evaluating other assets and recently announced that the target loading concentrations for its two topical rapamycin delivery technologies have been achieved: 1) a rapamycin loading concentration of 4% w/w for Quoin’s proprietary topical formulation, and 2) a higher rapamycin concentration of 5% w/w has been formulated in a proprietary dermal patch system. Initial clinical indications for which there currently are no FDA-approved treatments or cures that Quoin has identified as targets include Microcystic Lymphatic Malformations and Venous Malformations, among others.
Quoin also has recruited three additional patients - two in Austria and one in Ireland - in its investigator-led pediatric NS study who will all receive twice-daily whole-body application of QRX003, initially for a period of twelve weeks before continuing in a long-term extension protocol until/if regulatory approval is granted. Quoin believes data from these additional subjects will add to its growing clinical database supporting QRX003 as a potential treatment for NS. The company expects clinical data from these subjects will continue to demonstrate positive clinical outcomes of QRX003 in NS without adverse events, representing a continuation of positive data generated thus far.
Following 9 months of continued whole body application of QRX003, positive clinical data from the first pediatric patient in the study demonstrates that the subject’s skin remains completely healed, which QNRX notes shows the durability of ongoing daily treatment with QRX003. Prior to initiation of treatment with QRX003, the subject’s skin had a baseline Investigator’s Global Assessment (IGA) of 4 (the most severe score possible on a scale of 0-4).
SUBSCRIBE TO ZACKS SMALL CAP RESEARCH to receive our articles and reports emailed directly to you each morning. Please visit our website for additional information on Zacks SCR.
DISCLOSURE: Zacks SCR has received compensation from the issuer directly, from an investment manager, or from an investor relations consulting firm, engaged by the issuer, for providing research coverage for a period of no less than one year. Research articles, as seen here, are part of the service Zacks SCR provides and Zacks SCR receives payments totaling a maximum fee of up to $50,000 annually for these services provided to or regarding the issuer. Full Disclaimer HERE.