Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation

Open-Label, Exploratory Study to Evaluate the Effects of QR-010 on Nasal Potential Difference in Subjects With CF With the ΔF508 CFTR Mutation

Exploratory proof of concept study to determine whether intranasal administration of QR-010 in subjects with cystic fibrosis, homozygous or compound heterozygous for the ΔF508 mutation, can increase the function of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR).

This is an open-label, multi-center, exploratory study to estimate the effect of intranasal administration of QR-010 on the nasal mucosa in the restoration of CFTR function, as measured by nasal potential difference (NPD), in the nasal epithelium of adult subjects with CF who are homozygous or compound heterozygous for the ΔF508 CFTR mutation.

Inclusion Criteria:

• Confirmed diagnosis of CF as defined by iontophoretic pilocarpine sweat chloride test (sweat chloride) of > 60 mmol/L
• Nasal potential difference (NPD) measurement at Screening consistent with CF
• Confirmation of CFTR gene mutations homozygous or compound heterozygous for the ΔF508 mutation
• Body mass index (BMI) of ≥ 18 kg/m2
• Non-smoking for a minimum of 2 years
• Stable lung function
• FEV1 ≥40% of predicted normal for age, gender, and height at Screening

Exclusion Criteria:

• Breast-feeding or pregnant
• Acute allergy or infection affecting nasal conditions not resolved within 14 days prior Screening
• Use of lumacaftor or ivacaftor
• Use of any investigational drug or device
• Hemoptysis