Three additional patients have been added to Quoin Pharmaceuticals Ltd’s investigator-led pediatric Netherton Syndrome study, and a current pediatric patient in the ongoing study shows completely healed skin after nine months of “whole-body” application of QRX003.

QRX003 is a topical lotion formulated with a proprietary delivery technology that 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.

All three newly added subjects will receive twice-daily whole-body application of QRX003, initially for a period of 12 weeks before continuing in a long-term extension protocol until regulatory approval has been obtained. Two of the subjects reside in Austria, and the third lives in Ireland.

Study Update

At baseline, prior to initiation of treatment with QRX003, the patient’s skin had an Investigator’s Global Assessment (IGA) of 4, on a scale of 0–4 where 4 is the most severe score possible. At nine months, the IGA had materially improved to 0, and the subject’s skin is completely clear. In addition, the subject’s pruritus score changed from 5 at baseline to 0 at nine months. The subject continues to experience no nightly sleep disturbances without the need for any sedating medication, marking the first continuous period of uninterrupted sleep in their life.

Furthermore, since being treated with QRX003, the subject has continued to have no requirement for previously necessary medications such as antibiotics, antivirals, antihistamines, and glucocorticoids.

No adverse events have been reported to date after nine months of daily, whole-body treatment with QRX003.