Interim Data on QRX 003 for Netherton Syndrome

Data from the first subject being dosed twice-daily in Quoin’s ongoing open label study is suggestive of clinical efficacy across a number of measured endpoints after six weeks of dosing with QRX 003, which is the midpoint of testing.

At baseline, prior to dosing, the subject’s Modified Ichthyosis Area of Severity Index (MIASI) was 18. Following six weeks of dosing with QRX 003, the subject’s MIASI had been reduced to 4. In addition, the Investigator’s Global Assessment (IGA) of disease severity prior to dosing classified the subject as ‘moderate’. After six weeks of dosing with QRX 003, the IGA for the subject was classified as ‘mild’. The subject’s pruritus or itch assessment at baseline was 7 out of a maximum of 11 based on the Worst Itch Numeric Rating Scale (WINRS) and was reduced to 4 at the treatment midpoint. Finally, the patient satisfaction scores across assessed metrics were highly positive. No safety concerns were reported for the subject during this initial testing period.

In addition, after the initial 12 days of dosing in Quoin’s ongoing 12-week Investigator Pediatric Study, a significant improvement was observed in the skin area treated with QRX 003 versus the non-treated area. Specifically, at baseline prior to dosing with QRX 003, the IGA assessment of the subject’s skin was classified as ‘severe’. After 12 days of treatment with QRX 003, this was improved to ‘mild-moderate’, representing a very rapid improvement in skin appearance. There have been no adverse events or safety concerns reported to date.

Quoin CEO, Dr. Michael Myers, said, “While cautioning that this is interim clinical data from a very limited number of subjects, we are very pleased to announce these early results from two of our ongoing Netherton Syndrome clinical studies. Having previously announced positive data for subjects dosed once-daily for 12 weeks in our ongoing open-label study, we are excited to share initial data for the first subject dosed twice-daily to reach six weeks of dosing in this study. Although clear improvements were observed across all four measured endpoints, the reduction in MIASI from 18 at baseline to just 4 after six weeks of dosing with QRX003 is particularly noteworthy. Similarly, the reduction in pruritus severity from 7, out of a maximum of 11, to 4 after six weeks of dosing with QRX 003 is also promising.

“Furthermore, it is very encouraging to observe such a clear improvement in skin condition in the investigator pediatric study, after just 12 days of being treated with QRX 003. The change from an IGA classification of severe prior to dosing to mild-moderate after such a short period of time is highly encouraging as we seek to recruit additional pediatric subjects in Spain and the United Kingdom. We remain steadfast in our commitment to develop a safe and effective treatment for the Netherton Syndrome community.”