Quoin Pharmaceuticals to Initiate Clinical Study for Peeling Skin Syndrome
Quoin Pharmaceuticals (NASDAQ: QNRX) has announced plans to initiate a clinical study in New Zealand to evaluate QRX003 for Peeling Skin Syndrome (PSS), a rare autosomal disease with no approved treatment. The study will focus on a pediatric patient, with the company actively exploring additional clinical sites in other countries.
QRX003, Quoin's most advanced pipeline product, is already being tested in two late-stage clinical trials for Netherton Syndrome (NS) in the United States under an FDA-approved IND. The company aims to expand QRX003's development into PSS, leveraging potential similarities in the diseases' mechanisms. This move aligns with Quoin's strategy to broaden QRX003's applications in rare and orphan diseases.
Quoin Pharmaceuticals (NASDAQ: QNRX) ha annunciato piani per avviare uno studio clinico in Nuova Zelanda per valutare QRX003 per la Sindrome da Pelle Desquamata (PSS), una rara malattia autosomica senza trattamento approvato. Lo studio si concentrerà su un paziente pediatrico, con l'azienda che sta attivamente esplorando siti clinici aggiuntivi in altri paesi.
QRX003, il prodotto più avanzato nel pipeline di Quoin, è già in fase di sperimentazione in due studi clinici di fase avanzata per la Sindrome di Netherton (NS) negli Stati Uniti sotto un IND approvato dalla FDA. L'azienda mira ad espandere lo sviluppo di QRX003 nella PSS, sfruttando le potenziali somiglianze nei meccanismi delle malattie. Questa mossa è in linea con la strategia di Quoin di ampliare le applicazioni di QRX003 in malattie rare e orfane.
Quoin Pharmaceuticals (NASDAQ: QNRX) ha anunciado planes para iniciar un estudio clínico en Nueva Zelanda para evaluar QRX003 para el Síndrome de Piel Descamativa (PSS), una enfermedad autosómica rara sin tratamiento aprobado. El estudio se centrará en un paciente pediátrico, mientras la empresa explora activamente sitios clínicos adicionales en otros países.
QRX003, el producto más avanzado en el pipeline de Quoin, ya se está probando en dos ensayos clínicos de etapa avanzada para el Síndrome de Netherton (NS) en los Estados Unidos bajo un IND aprobado por la FDA. La empresa busca expandir el desarrollo de QRX003 hacia la PSS, aprovechando las posibles similitudes en los mecanismos de las enfermedades. Este movimiento está alineado con la estrategia de Quoin de ampliar las aplicaciones de QRX003 en enfermedades raras y huérfanas.
퀘인 제약(나스닥: QNRX)은 뉴질랜드에서 박리 피부 증후군 (PSS)에 대한 QRX003 임상 연구를 시작할 계획을 발표했습니다. 이는 승인된 치료법이 없는 희귀 자가소질 질환입니다. 연구는 소아 환자에 초점을 맞추며, 회사는 다른 국가에서 추가 임상 사이트를 적극적으로 탐색하고 있습니다.
QRX003은 퀘인 제약의 가장 앞선 파이프라인 제품으로, 미국에서 FDA 승인 IND에 따라 네더톤 증후군 (NS)에 대한 두 가지 후기 임상 시험에서 이미 시험되고 있습니다. 회사는 질병 메커니즘의 유사성을 활용하여 QRX003의 개발을 PSS로 확장하는 것을 목표로 하고 있습니다. 이러한 움직임은 희귀 및 고아 질환에서 QRX003의 적용을 확대하고자 하는 퀘인 제약의 전략과 일치합니다.
Quoin Pharmaceuticals (NASDAQ: QNRX) a annoncé des plans pour initier une étude clinique en Nouvelle-Zélande afin d'évaluer QRX003 pour le syndrome de peau épluchée (PSS), une maladie autosomique rare sans traitement approuvé. L'étude se concentrera sur un patient pédiatrique, l'entreprise explorant activement d'autres sites cliniques dans d'autres pays.
QRX003, le produit le plus avancé de Quoin, est déjà en cours d'essai dans deux études cliniques de phase avancée pour le syndrome de Netherton (NS) aux États-Unis sous un IND approuvé par la FDA. L'entreprise vise à étendre le développement de QRX003 vers le PSS, en tirant parti de similitudes potentielles dans les mécanismes des maladies. Ce mouvement s'inscrit dans la stratégie de Quoin d'élargir les applications de QRX003 dans les maladies rares et orphelines.
Quoin Pharmaceuticals (NASDAQ: QNRX) hat Pläne angekündigt, eine klinische Studie in Neuseeland zu initiieren, um QRX003 für das Peeling-Skin-Syndrom (PSS) zu bewerten, eine seltene autosomale Krankheit ohne genehmigte Behandlung. Die Studie konzentriert sich auf einen pädiatrischen Patienten, während das Unternehmen aktiv weitere klinische Standorte in anderen Ländern untersucht.
QRX003, das am weitesten fortgeschrittene Produkt in Quoins Pipeline, wird bereits in zwei späten klinischen Studien für das Netherton-Syndrom (NS) in den Vereinigten Staaten unter einem von der FDA genehmigten IND getestet. Das Unternehmen zielt darauf ab, die Entwicklung von QRX003 auf PSS auszuweiten, indem es mögliche Ähnlichkeiten in den Mechanismen der Krankheiten nutzt. Dieser Schritt passt zu Quoins Strategie, die Anwendungen von QRX003 bei seltenen und Waisenkankheiten zu erweitern.
- Expansion of QRX003 clinical testing to Peeling Skin Syndrome, a new indication
- Potential to achieve first regulatory approval for PSS treatment
- Ongoing late-stage clinical trials for Netherton Syndrome in the US
- Strategic alignment with company's goal to expand into other rare diseases
- None.
The initiation of a clinical study for Peeling Skin Syndrome (PSS) using QRX003 is a significant development in the rare disease space. PSS, like Netherton Syndrome (NS), lacks approved treatments, making this study potentially groundbreaking. The expansion of QRX003's application from NS to PSS demonstrates the versatility of the drug's mechanism and Quoin's strategic approach to rare disease treatment.
However, it's important to note that this is an early-stage, investigator-led study with only one pediatric patient identified. While promising, the scope means results may not be immediately generalizable. The company's active evaluation of additional clinical sites globally could accelerate research, but also increases operational complexity and costs.
From a scientific perspective, the overlapping presentation of PSS and NS suggests a potential for QRX003's efficacy, but this needs to be confirmed through rigorous clinical testing. The lack of other active clinical trials for PSS on clinicaltrials.gov underscores both the unmet medical need and the potential first-mover advantage for Quoin in this indication.
This clinical study initiation represents a strategic expansion of Quoin Pharmaceuticals' pipeline, potentially increasing the company's market opportunity. By targeting multiple rare diseases with a single product (QRX003), Quoin is maximizing its R&D investment and diversifying risk.
However, investors should note that rare disease drug development is high-risk and capital-intensive. The company's focus on orphan diseases means smaller patient populations, which can lead to challenges in patient recruitment and potentially revenue potential if approved.
Financially, while this news doesn't immediately impact Quoin's bottom line, it could enhance the company's valuation by expanding its potential market. The lack of approved treatments for PSS presents a significant market opportunity if QRX003 proves successful. However, the long timeline for drug development and approval in rare diseases means any financial benefits are likely years away.
Investors should closely monitor the company's cash position and burn rate as it expands its clinical programs, as additional funding may be required to support these initiatives.
Peeling Skin Syndrome is a rare autosomal disease with no approved treatment or cure
Initial clinical site and pediatric patient identified in New Zealand
Company actively evaluating opening additional clinical sites in other countries
ASHBURN, Va., Aug. 06, 2024 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”) a clinical stage, specialty pharmaceutical company focused on developing and commercializing novel treatments for rare and orphan diseases, today announced the planned initiation of an investigator-led clinical study in New Zealand to evaluate the safety and efficacy of QRX003 in a pediatric patient with Peeling Skin Syndrome (PSS). QRX003 is Quoin’s most advanced pipeline product and is currently being evaluated in two late stage clinical trials in the United States as a potential treatment for Netherton Syndrome (NS). Both studies are being conducted under an open Investigational New Drug (IND) application with the Food and Drug Administration (FDA).
“We are excited to expand QRX003’s development into this second indication, peeling skin syndrome, where it is believed the mechanism of action of our product could also provide a benefit for this devastating disease. Given the overlapping nature of how PSS presents, patients with the disease are known to have been previously misdiagnosed as having NS. Currently there are no clinical studies listed for peeling skin syndrome on clinicaltrials.gov as actively recruiting and dosing subjects and there is no approved treatment or cure, presenting a further opportunity for Quoin to achieve the first regulatory approval for another rare genetic disease,” stated Michael Myers, CEO, Quoin Pharmaceuticals. “The planned initiation of this study represents the execution of a key pillar of Quoin’s strategy to expand the clinical testing of QRX003 into other rare and orphan disease indications and we are actively assessing additional opportunities beyond this one.”
QRX003 is a unique “whole body, whole life” topical lotion that targets the vicious circle of skin inflammation and barrier disruption.
Quoin is conducting two ongoing clinical trials evaluating QRX003 for the treatment of Netherton Syndrome. For more information about the trials, please visit: https://www.nethertonsyndromeclinicaltrials.com/.
About Peeling Skin Syndrome (PSS)
Generalized inflammatory peeling skin syndrome (PSS) is a rare autosomal recessive genodermatosis caused by loss-of-function disease-causing variants of the corneodesmosin gene (CDSN), resulting in excessive shedding of the superficial layers of the epidermis. Patients generally suffer from a variety of conditions including severe pain and chronic pruritis (itch). There is currently no approved treatment for PSS, and patients manage symptoms using over-the-counter emollients.
About QRX003
QRX003 is a topical lotion, formulated with a proprietary delivery technology, and 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 lead to a more normalized skin shedding process and the formation of a stronger and more effective skin barrier.
About Quoin Pharmaceuticals Ltd.
Quoin Pharmaceuticals Ltd. is an emerging 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 three 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, Epidermolysis Bullosa and others. For more information, go to: www.quoinpharma.com.
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, statements relating to the Company’s expected cash runway, the belief that the data set from both clinical studies could potentially be sufficiently robust and comprehensive to support an NDA filing, without the need for any additional clinical studies in Netherton subjects, and the belief that certain protocol changes has enhanced the potential for a successful outcome and Quoin’s 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, 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 may need to raise additional funds sooner than planned, the clinical studies may not generate data which is sufficiently robust and comprehensive to support an NDA filing and the Company’s ability to obtain regulatory approvals. More detailed information about the risks and uncertainties affecting the Company is summarized 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
What is Quoin Pharmaceuticals (QNRX) planning to study for Peeling Skin Syndrome?
Where is Quoin Pharmaceuticals (QNRX) conducting the Peeling Skin Syndrome study?
What other conditions is Quoin Pharmaceuticals (QNRX) studying QRX003 for?
Is there an approved treatment for Peeling Skin Syndrome that Quoin Pharmaceuticals (QNRX) is targeting?
