Quoin Pharmaceuticals advances rare disease pipeline with two active trials

Published 29/07/2025, 16:18
Quoin Pharmaceuticals advances rare disease pipeline with two active trials

ASHBURN, Va. - Quoin Pharmaceuticals Ltd. (NASDAQ:QNRX), a micro-cap pharmaceutical company with a market capitalization of $4.8 million, is advancing its lead product QRX003 in two pivotal registrational clinical studies for Netherton Syndrome, according to a company statement released Tuesday. According to InvestingPro data, the company maintains a healthy balance sheet with more cash than debt, positioning it well for these clinical developments.

The studies are being conducted at sites across the U.S., Europe, and the Middle East, with each expected to enroll 12 to 16 subjects. Full recruitment is targeted for early to mid-Q1 2026. The company aims to file a New Drug Application in the second half of 2026. With a current ratio of 2.57, InvestingPro analysis shows the company’s liquid assets comfortably exceed its short-term obligations, providing financial flexibility for these clinical programs.

QRX003 is being tested as both a monotherapy and in conjunction with off-label systemic therapy in the respective studies. Clinical endpoints include Investigator’s Global Assessment, Ichthyosis Area Severity Index, pruritus measurements, and patient satisfaction scores.

The company also reported progress with its Peeling Skin Syndrome (PSS) program following positive initial data from a single-patient study in New Zealand. The patient showed improvement across key endpoints, including a two-grade improvement in the Investigator’s Global Assessment. Quoin plans to expand this study to include an additional 4 to 6 subjects.

Additionally, Quoin is advancing its topical rapamycin program for rare skin diseases including microcystic lymphatic malformations, venous malformations, and angiofibromas. The program utilizes two proprietary delivery technologies, with clinical testing anticipated to begin in the first half of 2026.

The company has discontinued development of QRX007 for Netherton Syndrome and QRX004 for Epidermolysis Bullosa to focus resources on its more advanced programs.

Quoin plans to self-commercialize QRX003 in the U.S., Western Europe, and Japan if approved, and has established nine commercial partnerships covering 61 additional countries. Based on InvestingPro Fair Value analysis, the stock appears undervalued despite facing near-term profitability challenges. Analysts have set price targets ranging from $30 to $350, reflecting significant potential upside if clinical trials succeed. For deeper insights into Quoin’s financial health and growth prospects, investors can access the comprehensive Pro Research Report, available exclusively to InvestingPro subscribers.

In other recent news, Quoin Pharmaceuticals has made significant advancements in its clinical programs. The U.S. Food and Drug Administration granted Rare Pediatric Disease Designation to Quoin’s lead asset, QRX003, for treating Netherton Syndrome. This follows the European Medicines Agency’s Orphan Drug Designation for the same treatment. The FDA also cleared Quoin to initiate a pivotal clinical study for QRX003, which will evaluate its efficacy in combination with off-label systemic therapy. Positive initial results from an ongoing clinical study for Pediatric Peeling Skin Syndrome using QRX003 showed significant improvement in patients’ skin conditions. In corporate developments, Quoin Pharmaceuticals announced that its Chief Financial Officer, Gordon Dunn, will depart following a mutual agreement. Mr. Dunn will remain in his position until a successor is appointed, and a separation agreement is expected to be finalized soon. These developments highlight Quoin’s ongoing efforts in advancing treatments for rare skin disorders.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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