Quoin Pharmaceuticals Announces Positive Clinical Update from Ongoing Pediatric Netherton Syndrome Compassionate Use Program
Four of Six Patients Participating in the Compassionate Use Program Were Classified as ‘Improved' or ‘Significantly Improved' from Baseline Assessment Across Key Clinical Endpoints
- All Six Patients Are Younger than 10 Years of Age, with the Youngest Being Just 6 Months Old
- Duration of Treatment To-Date Ranges from 3 Weeks to 15 Months
- No Treatment Related Adverse Events Reported for All Six Patients
- Additional Pediatric Patients Enrolling in Program in June and September
- Quoin Plans a Comprehensive Data Release, including from Ongoing Phase 2 Studies, in the Coming Months
ASHBURN, Va., June 16, 2026 (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 a positive clinical update from its ongoing pediatric Netherton Syndrome (NS) Compassionate Use program.
Six pediatric NS patients are currently enrolled in the program, all under 10 years of age, with the youngest being just 6 months old. Four of the six children in the study were classified as ‘Improved' or ‘Significantly Improved' from baseline across key clinical endpoints at the time of this release.
Three of the four treatment responders achieved a minimum of a one-grade improvement from baseline according to an Investigator's Global Assessment (IGA) (Scale 0-clear to 4-severe), with one patient achieving a full 5-grade improvement from 4 (severe) at baseline to 0 (clear) at nine months, and the patient continues to remain clear at 15 months. Of these three responders, two achieved at least a 4-grade improvement from baseline for pruritus (scale 0-10), with one patient's pruritus being completely eliminated. The third responder started with pruritus of 1 and remained at 1 during treatment.
The fourth, and youngest patient responder is 6 months old and has only been on treatment for 3 weeks. Quoin is still waiting for the 6 week safety and efficacy data, however, at baseline, the patient required the application of emollients to their skin 12 times a day, every 2 hours. After 3 weeks of dosing with QRX003, as a result of improvements in the infant's skin, the parents are using almost no emollient on the treated body sites. In addition, prior to receiving QRX003, the infant's neutrophil levels were extremely low putting the infant at significant risk of infection complications. After just 3 weeks of treatment with QRX003, these levels have been fully restored to normal levels, which is yet another clear indication of a positive treatment effect.
Two of the six patients have not achieved any improvement to date, though for one of these the duration of treatment has been less than 8 weeks and the patient remains on treatment. An additional patient was enrolled into the program on June 15th and two others are scheduled to begin treatment by the end of September. Quoin believes the information generated from this growing cohort of pediatric patients could provide valuable supportive evidence of QRX003's longer term safety and efficacy in this key patient population.
There have been no reports of treatment related adverse events for any of the six patients in the program.
Quoin plans a comprehensive release of clinical data from this program and from its ongoing Phase 2 studies in the coming months.
Quoin CEO Dr. Michael Myers said, "We are thrilled to provide this very significant update from the six NS children currently enrolled in our QRX003 Compassionate Use Program. As NS is a genetic disease, the efficacy of our product on children is extremely important to ascertain. With a remarkable 4 out of 6 children in the program responding positively and showing clear signs of improvement across key clinical endpoints when treated with QRX003, we are encouraged and remain optimistic about the potential for the product to become the first approved treatment for this disease. The shortest duration of treatment at just 3 weeks for a 6-month-old infant has already greatly reduced the burden of care for the child's parents by eliminating previously required application of emollients to the child's skin every 2 hours for the areas treated by QRX003. In addition, the patient who has been treated with QRX003 the longest, now at 15 months, continues to have fully healed skin, zero pruritus and no nightly sleep disturbances resulting in a complete life transformation. With another pediatric patient enrolled in the program on June 15th and two more scheduled by the end of September, we believe we have the opportunity to develop comprehensive evidence of how QRX003 performs in a real world setting for this key patient population. We are looking forward to providing a more comprehensive update from this program as well as from our ongoing Phase 2 studies in the coming months."
About Netherton Syndrome
Netherton Syndrome, a form of Ichthyosis, is a rare hereditary skin disorder caused by a mutation in the SPINK5 gene (serine protease inhibitor, Kazal Type 5) that leads to severe skin barrier defects and recurring infections, as well as a pronounced predisposition to allergies, asthma, and eczema. Patients often suffer from severe dehydration, chronic skin inflammation and stunted growth. Currently, there is no cure for Netherton Syndrome, nor are there any approved therapeutic treatments.
About QRX003
QRX003 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 please visit: https://www.nethertonsyndromeclinicaltrials.com/
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 is focused on two key platform products, QRX003 and QRX009, that collectively have the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Pachyonychia Congenita, Gorlin Syndrome and Tuberous Sclerosis Complex, microcystic lymphatic malformations, venous malformations, angiofibromas and others. For more information, visit: www.quoinpharma.com or LinkedIn for updates.
Cautionary Note Regarding Forward Looking Statements
The 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: enrolling additional patients into the Company's ongoing pediatric NS Compassionate Use program by the end of September; the information generated from this cohort of pediatric patients potentially providing valuable supportive evidence of QRX003's longer term safety and efficacy in this key patient population; plans for a comprehensive release of clinical data from the program and from Quoin's ongoing Phase 2 studies in the coming months; the potential for QRX003 to become the first approved treatment for Netherton Syndrome; the opportunity to develop comprehensive evidence of how QRX003 performs in a real world setting for pediatric NS patients; 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, Pachyonychia Congenita, Gorlin Syndrome, Tuberous Sclerosis Complex, microcystic lymphatic malformations, venous malformations, angiofibromas 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, 2025 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 Ltd.
Michael Myers, Ph.D., CEO
mmyers@quoinpharma.com
Investor Relations
PCG Advisory
Jeff Ramson
jramson@pcgadvisory.com
(646) 863-6341
