Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome

Inclusion Criteria:

• Subject is a male or non-pregnant female at least 14 years of age.

• Females must be post-menopausal , surgically sterile , or use an effective method ofbirth control , for the duration of the study and for 3 months following completionof treatment. Women of childbearing potential (WOCBP) must have a negative urinepregnancy test (UPT) at Visit 1/Screening and Visit 2/Baseline.

• Subject has a clinical diagnosis of NS and agrees to genetic testing at Visit 1/Screening for confirmation of NS diagnosis if the subject does not have testresults confirming a SPINK5 mutation.

• Subject has NS lesions in the Treatment Area (i.e., arms or lower legs).

• Subject is in good general health and free of any disease state or physicalcondition that might impair evaluation of NS or exposes the subject to anunacceptable risk by study participation.

Exclusion Criteria:

• Subject is pregnant, lactating, or is planning to become pregnant during the study.

• Subject has any skin pathology in the Treatment Area or condition that, couldinterfere with the evaluation of the test article or requires use of interferingtopical, systemic, or surgical therapy.

• Subject has Fridericia-corrected QT ≥ 450 ms for males or ≥ 470 ms for females

• Subject has active cancer of any type excluding non-melanoma skin cancer outside ofthe Treatment Area.

• Subject has diabetes of any type, except non-insulin dependent diabetes mellitusthat is reasonably controlled.

• Subject has evidence of active infection during screening, or serious infectionwithin 30 days prior to Visit 2/Baseline.

• Subject has known human immunodeficiency virus, hepatitis B or C virus, or active orlatent tuberculosis.

• Subject has used ultraviolet phototherapy within the Treatment Area within 4 weeksprior to Visit 2/Baseline.

• Subject has used systemic prescription treatment for NS within 4 weeks prior toVisit 2/Baseline.

• Subject has used systemic biologic therapy for NS.

• Subject has used topical prescription treatment in the Treatment Area within 2 weeksprior to Visit 2/Baseline.

• Subject has used any topical bland moisturizers/emollients in the Treatment Areawithin 24 hours prior to Visit 2/Baseline.

• Subject is currently enrolled in an investigational drug, biologic, or device study.

• Subject has used an investigational drug, biologic, or device treatment within 30days prior to Visit 2/Baseline.