Quoin Pharmaceuticals Announces Further Clinical Evidence of QRX003 Effectiveness in Netherton Syndrome
Quoin Pharmaceuticals (NASDAQ: QNRX) has announced additional clinical evidence regarding QRX003's effectiveness in treating Netherton Syndrome. After observing positive results during a 12-week treatment period, the company reported that all clinical benefits were completely reversed within 4 weeks of treatment discontinuation.
The clinical data shows that the first subject, who received twice-daily doses, experienced significant improvements across all measured endpoints during the treatment period. The Modified Ichthyosis Area of Severity Index (M-IASI) improved from 18 to 3, the Worst Itch Numeric Rating Scale (WINRS) decreased from 7 to 2, and the Investigator's Global Assessment (IGA) improved from Moderate to Almost Clear. However, these metrics reverted to baseline levels after treatment stopped.
The results indicate that chronic, whole-body treatment with QRX003 is necessary for maintaining positive clinical outcomes in Netherton Syndrome patients. Quoin currently has four clinical studies underway, with three under FDA IND applications, aiming to develop the first approved treatment for this condition.
Quoin Pharmaceuticals (NASDAQ: QNRX) ha annunciato ulteriori evidenze cliniche riguardanti l'efficacia di QRX003 nel trattamento della Sindrome di Netherton. Dopo aver osservato risultati positivi durante un periodo di trattamento di 12 settimane, l'azienda ha riportato che tutti i benefici clinici sono stati completamente invertiti entro 4 settimane dalla sospensione del trattamento.
I dati clinici mostrano che il primo soggetto, che ha ricevuto dosi due volte al giorno, ha sperimentato miglioramenti significativi in tutti i parametri misurati durante il periodo di trattamento. L'Indice di Severità della Cheratosi Modificato (M-IASI) è migliorato da 18 a 3, la Scala Numerica di Valutazione del Prurito Peggiore (WINRS) è diminuita da 7 a 2, e la Valutazione Globale dell'Investigator (IGA) è passata da Moderato a Quasi Chiaro. Tuttavia, queste metriche sono tornate ai livelli basali dopo l'interruzione del trattamento.
I risultati indicano che è necessario un trattamento cronico e completo con QRX003 per mantenere risultati clinici positivi nei pazienti con Sindrome di Netherton. Attualmente Quoin ha quattro studi clinici in corso, con tre sotto domande IND della FDA, con l'obiettivo di sviluppare il primo trattamento approvato per questa condizione.
Quoin Pharmaceuticals (NASDAQ: QNRX) ha anunciado evidencia clínica adicional sobre la efectividad de QRX003 en el tratamiento del Síndrome de Netherton. Después de observar resultados positivos durante un periodo de tratamiento de 12 semanas, la empresa informó que todos los beneficios clínicos se invirtieron completamente dentro de las 4 semanas posteriores a la interrupción del tratamiento.
Los datos clínicos muestran que el primer sujeto, quien recibió dosis dos veces al día, experimentó mejoras significativas en todos los puntos finales medidos durante el periodo de tratamiento. El Índice Modificado de Severidad de Ictiosis (M-IASI) mejoró de 18 a 3, la Escala Numérica de Evaluación del Picor Peor (WINRS) disminuyó de 7 a 2, y la Evaluación Global del Investigador (IGA) mejoró de Moderada a Casi Clara. Sin embargo, estas métricas volvieron a niveles basales después de que se detuvo el tratamiento.
Los resultados indican que se necesita un tratamiento crónico y corporal completo con QRX003 para mantener resultados clínicos positivos en pacientes con Síndrome de Netherton. Actualmente, Quoin tiene cuatro estudios clínicos en marcha, con tres bajo solicitudes IND de la FDA, con el objetivo de desarrollar el primer tratamiento aprobado para esta condición.
퀀 제약(Quoin Pharmaceuticals) (NASDAQ: QNRX)이 네더튼 증후군 치료를 위한 QRX003의 효과에 대한 추가 임상 증거를 발표했습니다. 12주 치료 기간 동안 긍정적인 결과를 관찰한 후, 회사는 치료 중단 후 4주 이내에 모든 임상 혜택이 완전히 역전되었다고 보고했습니다.
임상 데이터에 따르면, 하루 두 번의 용량을 받은 첫 번째 피험자는 치료 기간 동안 모든 측정된 지표에서 상당한 개선을 경험했습니다. 수정된 어류 각화증 중증도 지수(M-IASI)는 18에서 3으로 개선되었고, 최악의 가려움증 수치 평가(WINRS)는 7에서 2로 감소했으며, 연구자의 전반적 평가(IGA)는 중간에서 거의 맑음으로 개선되었습니다. 그러나 이러한 지표는 치료가 중단된 후 기준 수준으로 되돌아갔습니다.
결과는 네더튼 증후군 환자에게 긍정적인 임상 결과를 유지하기 위해 QRX003의 만성 전신 치료가 필요하다는 것을 나타냅니다. 현재 퀀은 FDA IND 신청 하에 세 개, 총 네 개의 임상 연구를 진행 중에 있으며, 이 질환에 대한 첫 번째 승인 치료제를 개발할 계획입니다.
Quoin Pharmaceuticals (NASDAQ: QNRX) a annoncé des preuves cliniques supplémentaires concernant l'efficacité de QRX003 dans le traitement du syndrome de Netherton. Après avoir observé des résultats positifs pendant une période de traitement de 12 semaines, l'entreprise a rapporté que tous les bénéfices cliniques avaient été complètement inversés dans les 4 semaines suivant l'arrêt du traitement.
Les données cliniques montrent que le premier sujet, qui a reçu des doses deux fois par jour, a connu des améliorations significatives dans tous les critères mesurés pendant la période de traitement. L'indice modifié de sévérité de l'ichthyose (M-IASI) est passé de 18 à 3, l'échelle de notation numérique du pire prurit (WINRS) est passée de 7 à 2, et l'évaluation globale de l'investigateur (IGA) est passée de modéré à presque clair. Cependant, ces métriques sont revenues aux niveaux de base après l'arrêt du traitement.
Les résultats indiquent que un traitement chronique et total avec QRX003 est nécessaire pour maintenir des résultats cliniques positifs chez les patients atteints du syndrome de Netherton. Quoin a actuellement quatre études cliniques en cours, dont trois sous demandes IND auprès de la FDA, visant à développer le premier traitement approuvé pour cette condition.
Quoin Pharmaceuticals (NASDAQ: QNRX) hat zusätzliche klinische Nachweise zur Wirksamkeit von QRX003 bei der Behandlung des Netherton-Syndroms bekannt gegeben. Nach positiven Ergebnissen während eines 12-wöchigen Behandlungszeitraums berichtete das Unternehmen, dass alle klinischen Vorteile innerhalb von 4 Wochen nach Behandlungsabbruch vollständig rückgängig gemacht wurden.
Die klinischen Daten zeigen, dass das erste Subjekt, das zweimal täglich Dosen erhielt, während des Behandlungszeitraums signifikante Verbesserungen in allen gemessenen Endpunkten erlebte. Der modifizierte Ichthyose-Schweregradindex (M-IASI) verbesserte sich von 18 auf 3, die schlechteste Juckreiz-Bewertungsskala (WINRS) sank von 7 auf 2, und die globale Beurteilung des Prüfers (IGA) verbesserte sich von mäßig auf fast klar. Diese Messwerte kehrten jedoch nach Behandlungsabbruch auf das Basisniveau zurück.
Die Ergebnisse deuten darauf hin, dass eine chronische, ganzkörperliche Behandlung mit QRX003 notwendig ist, um positive klinische Ergebnisse bei Patienten mit Netherton-Syndrom aufrechtzuerhalten. Quoin hat derzeit vier klinische Studien laufen, von denen drei unter IND-Anträgen der FDA stehen, mit dem Ziel, die erste zugelassene Behandlung für diese Erkrankung zu entwickeln.
- Clear clinical evidence of QRX003's effectiveness during treatment period
- Significant improvement in all measured clinical endpoints (M-IASI, WINRS, IGA) during treatment
- Four ongoing clinical studies, three under FDA IND applications
- Potential to become first approved treatment for Netherton Syndrome
- Complete reversal of clinical benefits within 4 weeks of treatment discontinuation
- Necessity for chronic, continuous treatment to maintain benefits
- Product requires whole-body application for effectiveness
Insights
The latest clinical data from Quoin Pharmaceuticals presents compelling evidence for QRX003's therapeutic potential in Netherton Syndrome, a rare genetic disorder with no currently approved treatments. The results demonstrate the drug's clear mechanism of action through three key metrics:
- The Modified Ichthyosis Area of Severity Index (M-IASI) improved from 18 to 3 during treatment, indicating significant reduction in skin symptoms, before reverting to 18 post-discontinuation
- The Worst Itch Numeric Rating Scale (WINRS) showed marked improvement from 7 to 2, returning to 8 after stopping treatment
- The Investigator's Global Assessment (IGA) progressed from Moderate to Almost Clear, then reverted to Moderate
The complete reversal of benefits post-discontinuation is actually a positive finding, as it provides strong validation of QRX003's mechanism of action as a serine protease inhibitor. This 'washout' effect is particularly valuable for regulatory submissions, as it demonstrates clear causality between treatment and clinical improvement.
The clinical development strategy, with four concurrent studies including three under FDA IND, suggests an accelerated pathway to potential approval. For rare diseases like Netherton Syndrome, which affects approximately 1 in 200,000 newborns, the FDA often provides expedited review processes when clear efficacy is demonstrated.
The requirement for chronic, whole-body treatment presents both opportunities and challenges. While it ensures consistent revenue streams post-approval, it also necessitates robust long-term safety data and careful consideration of treatment costs. The company's focus on completing clinical development "expeditiously" aligns with the urgent unmet medical need in this patient population.
- Four Weeks After Discontinuation of Treatment with QRX003 Complete Reversal of Positive Clinical Improvements Across all Measured Endpoints Observed
- Data 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 efficacy of QRX003 in Netherton Syndrome.
In December 2024 and January 2025, Quoin announced positive data from the mid-point and at completion of 12 weeks of testing the first subject dosed twice-daily in Quoin’s ongoing open-label study which demonstrated evidence of clear improvement across all measured clinical endpoints. Today Quoin is sharing additional data on that subject at 4 weeks post-discontinuation of treatment with QRX003. As the table below illustrates all of the positive clinical benefits observed after 12 weeks of testing with QRX003 were completely reversed by 4 weeks after discontinuation of treatment resulting in the subject’s disease state reverting to the baseline status observed prior to QRX003 treatment. These results strongly indicate that ongoing, chronic treatment with QRX003 is necessary for a continued positive clinical outcome in Netherton Syndrome patients.
Table 1: First Patient Data from Open Label Study Part B- Dosed Twice Daily with QRX003
| End Point | Baseline | 6 weeks (Treatment period midpoint) | 12 weeks (End of treatment period) | 4 weeks post discontinuation of treatment |
| M-IASI* | 18 | 4 | 3 | 18 |
| WINRS** | 7 | 4 | 2 | 8 |
| IGA*** | Moderate | Mild | Almost Clear | Moderate |
*M-IASI: Modified Ichthyosis Area of Severity Index, a score used to assess the severity and extent of skin symptoms associated with ichthyosis. Lower scores indicate improvement.
**WINRS: Worst Itch Numeric Rating Scale, which measures the severity of itch on an 11-point scale (0 = no itch, 10 = worst imaginable itch).
***IGA: Investigator’s Global Assessment, which uses descriptive categories (e.g., clear, mild, moderate, severe) to evaluate the overall severity of Netherton Syndrome symptoms.
Quoin CEO, Dr. Michael Myers, said, “This data set provides the clearest clinical evidence to date that the active ingredient in QRX003 is a competitive broad spectrum serine protease inhibitor and that discontinuation of treatment with QRX003 results in a complete reversal of all clinical benefits derived from continued treatment with the product. The rapid reversal of all observed clinical benefits to baseline disease status after just 4 weeks of treatment discontinuation serves to further underscore that chronic, whole-body treatment with QRX003 is potentially required to maintain the positive clinical outcomes observed in our clinical study. These results increase our commitment to complete the clinical development of QRX003 as expeditiously as possible with a goal of making the product widely available to the Netherton community once approved.
With four clinical studies in Netherton subjects underway, three of which are being conducted under an open Investigational New Drug (IND) application with the FDA, we believe Quoin is assembling a broad and diverse clinical data package to support a New Drug Application (NDA) filing for QRX003 as potentially the first approved treatment for Netherton Syndrome.”
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://quoinpharma.com/pipeline/#trials
About Quoin Pharmaceuticals Ltd.
Quoin Pharmaceuticals Ltd. is a 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 comprises four products in development that collectively have the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa 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 a description 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,” “plan,” “anticipate,” “believe,” 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, the potential efficacy of QRX003 as a treatment for Netherton Syndrome, the goal of making the product widely available to the Netherton community once approved, we believe Quoin is assembling a broad and diverse clinical data package to support a New Drug Application (NDA) filing for QRX003 as potentially the first approved treatment for Netherton and Quoin’s products in development collectively having the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa 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 deliver a safe and effective treatment for Netherton Syndrome, its studies may not be successful or may not generate data which is sufficiently robust and comprehensive to an NDA filing for QRXOO3 as an approved treatment for Netherton Syndrome and other factors discussed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 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
FAQ
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