U.S. and International Sites participating in QRX003 NS study, Expanding to study QRX003 for additional indications and in younger patient populations
Quoin Pharmaceuticals (NASDAQ:QNRX) provided a business update last week. The company implemented a number of measures during the quarter to move lead asset QRX003 forward. For instance, Quoin initiated testing of the safety and efficacy of QRX003 in a pediatric patient with Netherton Syndrome (NS). NS is a rare inherited genetic disease for which there is currently no approved treatment or cure. This initiative addresses multiple QNRX’s objectives, including studying QRX003 in pediatric patient populations. Treating pediatric patients is important, as early treatment is critical to managing the disease. The company also commenced measures to expand clinical activities on QRX003 internationally. Separately, management raised its stake in QNRX shares, which we view as a positive indicator of its optimism about the company’s prospects.
Key takeaways
QNRX initiated assessment of QRX003 in an NS pediatric patient, consistent with 1) plans to extend patient populations eligible to participate in clinical studies and 2) contributing to the growing database to support potential regulatory approval and subsequent commercialization of QRX003
Quoin expects to add three additional pediatric patients in Spain shortly
With two active late stage clinical trials in the U.S. for NS being conducted concurrently under an open FDA IND application, QNRX is expanding the study to international sites
Quoin has identified a center in Saudi Arabia as the 1st international location (and 6th overall site) participating in the study
Quoin plans to open two additional clinical sites in the U.K.
The CEO and COO increased their investment in QNRX ADSs, which we view positively
QNRX is expanding QRX003 optionality, studying it in a pediatric patient with PSS
Assessing assets for multiple indications is a core objective that can spread costs over a wider base
Initiating QRX003 assessment in pediatric patient consistent with plans to expand populations eligible to participate in clinical study & expand database for potential regulatory approval…
The company expects the results of the clinical assessment of QRX003 in a young child with NS to contribute to its database to support potential regulatory approval and subsequent commercialization of QRX003. QNRX is also optimistic that the data from this assessment will facilitate its ability to lower the eligibility age for its clinical activities around NS. QRX003 is currently being evaluated for the treatment of NS in two active late-stage studies that are being conducted concurrently in the U.S. in patients 14 years or older under an open IND (Investigational New Drug) application with the FDA.
Two active late stage clinical trials in the U.S. for NS running concurrently under open IND
Quoin believes it is the only development company conducting dual NS studies concurrently under an open IND application. With both trials utilizing the same investigators and clinical sites, Quoin expects to attain substantial operational synergies and cost savings. The company is optimistic that opening international sites will accelerate overall recruitment activities and diversify the patient population being tested. In addition to five open U.S. clinical sites, the company has identified a center in Saudi Arabia as the first international location and sixth overall site participating. The Saudi Arabia center is currently treating several NS patients who will now be eligible to participate in Quoin’s studies. In addition, Quoin also announced the planned opening of two additional clinical sites in the U.K. that NS treat patients who will potentially be eligible for recruitment.
Both the U.K. and Saudi Arabia sites will operate under the open IND application. Quoin is also preparing for the opening of additional sites in other Western European countries and is concluding a feasibility study in multiple Eastern European countries. The targeted European territories have cohorts of NS patients available to participate in the study.
C-suite executives increased their investment in QNRX
Moreover, during the quarter, Quoin’s CEO and COO purchased additional QNRX ADSs. In early September 2024, CEO Michael Myers purchased 37,894 ADSs at prices ranging from $0.619 to $0.7974 per ADS. During the same period, COO Denise Carter purchased 37,735 ADSs at prices ranging from $0.6253 to $0.7965 per ADS. We view it positively when insiders invest in shares and believe it reflects their confidence in the company’s outlook.
QRX003 - Potential to Become 1st Approved Treatment for Netherton Syndrome
Quoin is optimistic about QRX003’s prospects to be the first approved treatment for NS. There are no approved NS treatments currently and 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.
Clinical study in Peeling Skin Syndrome
Moreover, QNRX is also launching a clinical study of QRX003 in New Zealand in a pediatric patient with PSS. Peeling Skin Syndrome (PSS) to evaluate its safety and efficacy. Peeling Skin Syndrome is a rare autosomal disease that has no approved treatment or cure. Quoin believes it has the opportunity to achieve the first regulatory approval for PSS. Most forms of PSS present at birth or during infancy, according to NORD. Symptoms include shedding or peeling of the outermost layer of the skin.
Assessing assets for multiple indications can help spread costs over expanded base, broaden target markets upon potential commercialization …
The company’s strategy is to design products to treat multiple indications. Reflecting this strategy, QNRX intends to study QRX003 for several conditions and build a database supporting its efficacy and safety as a treatment for a range of dermatological conditions. Earlier this month the company announced plans to begin a clinical study of QRX003 in Peeling Skin Syndrome (PSS), a rare autosomal disease that has no approved treatment, to evaluate the safety and efficacy of QRX003 in New Zealand in a pediatric patient with PSS. Most forms of PSS present at birth or during infancy, according to NORD. Symptoms include shedding or peeling of the outermost layer of the skin. Quoin believes it has the opportunity to achieve the first regulatory approval for PSS.
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