Quoin Files US Patent Applications for Rare Disease Treatments

Image Credit: © dermnetnz.org

Today, Quoin Pharmaceuticals announced its filing of US patent applications for novel topical therapies to treat rare diseases like Netherton Syndrome.¹ The formulations are also targeted for orphan conditions like Peeling Skin Syndrome, SAM Syndrome, palmoplantar keratoderma, and severe atopic dermatitis. There are currently no treatments for Netherton Syndrome or Peeling Skin Syndrome that are approved by the US Food and Drug Administration (FDA).

This is Quoin’s third patent application for Netherton Syndrome. Quoin’s lead product, QRX003, is currently being investigated through 4 global clinical studies, 3 of which are under an open Investigational New Drug (IND) application with the FDA. QRX003 is being tested in both adult and pediatric patients, and positive clinical data from 2 open label studies are available. According to Quoin, they are the only company actively recruiting and testing patients in multiple clinical trials that are being conducted under an open IND.

“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,” Michael Myers, MD, Chief Executive Officer of Quoin, said in a statement.²

Netherton Syndrome is a severe genetic skin condition caused by mutations in the SPINK5 gene. It leads to excessive skin shedding and compromised skin barrier function. Current treatment options are limited and primarily focus on symptomatic relief.

QRX003 is a topical lotion designed to address the underlying pathology of Netherton Syndrome by inhibiting kallikreins, serine proteases that contribute to the excessive skin shedding characteristic of the disease. If successful, QRX003 could become the first FDA-approved treatment for Netherton Syndrome, offering new hope to patients and their families.

“While we are fully focused on completing clinical testing for QRX003 in NS, we are also taking multiple steps to ensure that we have the broadest and most extensive patent protection around the product for this disease as well for other rare skin disease applications that we are pursuing or intend to pursue. If granted, this application would provide broad patent protection for our product until 2045,” Myers said.

The company has also recently filed patent applications for novel topical formulations of rapamycin (sirolimus) both in the US and internationally, to treat diseases such as microcystic lymphatic malformations, venous malformations, and angiofibromas.³

References

1. Quoin Pharmaceuticals Files U.S. Patent Application for Novel Topical Formulations to Treat Netherton Syndrome and Other Skin Diseases. News release. Globe Newswire. Published March 25, 2025. Accessed March 25, 2025. https://www.globenewswire.com/news-release/2025/03/25/3048611/0/en/Quoin-Pharmaceuticals-Files-U-S-Patent-Application-for-Novel-Topical-Formulations-to-Treat-Netherton-Syndrome-and-Other-Skin-Diseases.html

2. Quoin pharmaceuticals announces further clinical evidence of QRX003 effectiveness in Netherton syndrome. News release. BioSpace. Published January 23, 2025. Accessed March 25, 2025. https://www.biospace.com/press-releases/quoin-pharmaceuticals-announces-further-clinical-evidence-of-qrx003-effectiveness-in-netherton-syndrome

3. Quoin Pharmaceuticals Files U.S. and International Patent Applications for Novel Topical Rapamycin Formulations to Treat Rare Disease Indications. News release. Globe Newswire. March 4, 2025. Accessed March 25, 2025. https://www.globenewswire.com/news-release/2025/03/04/3036382/0/en/Quoin-Pharmaceuticals-Files-U-S-and-International-Patent-Applications-for-Novel-Topical-Rapamycin-Formulations-to-Treat-Rare-Disease-Indications.html