Michael Myers, M.D., CEO of the Quoin pharmaceutical company describes Netherton Syndrome as a devastating skin disorder that at times may be fatal. In a recent article published in BioSpace, the doctor announced that Quoin’s first patient received dosing in the ongoing trial that evaluates the efficacy and safety of QRX003 to treat Netherton Syndrome. The study has been conducted through a United States Investigational New Drug Application. It assesses two separate doses of QRX003, one is a topical lotion and the other is a vehicle lotion that provides bulk to facilitate the administering of the lotion.
Doctor Myers further stated that the dosing of Quoin’s first patient and clearance of its second site to begin testing are positive steps for the company and community. He mentioned that their six sites should be opened shortly.
About Netherton Syndrome
Netherton Syndrome, which is rare (less than 5000 U.S. patients), affects the hair, skin, and immune system. Other symptoms may include immune reactions such as asthma, eczema, or itchy skin. Infection and dehydration occur often and may be serious.
The disease is caused by a disruption in the SPINK5 gene. The syndrome is inherited and its diagnosis is based on symptoms plus genetic tests and clinical examination.
Testing is conducted for twelve weeks with test materials applied to predetermined locations on the body. Various clinical endpoints are decided after consultation with the FDA and are assessed during the study.
The second study was announced in October of 2022 and has been cleared by the USFDA. This study will evaluate ten Netherton patients who are receiving off-label treatment. The study is scheduled to run for twelve weeks and be held at the sites used for the first study.
Dr. Myers believes that QRX003 may eventually be used primarily for Netherton Syndrome patients. The lotion down-regulates the hyperactivity of skin Kallikreins which are a subgroup of serine proteases that regulate functions such as blood pressure and heart rate. This leads to more normalized shedding of a patient’s skin.
If QRX003 proves to be effective as well as safe then the daily application may lead to a normal skin barrier plus improved quality of life for Netherton patients.
Quoin Pharmaceuticals
Quoin Pharmaceuticals is a specialty company with a focus on developing therapeutics to treat orphan and rare diseases. Quoin is committed to meeting the medical needs of patients, families, care teams, and communities.
