Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients

The purpose of this study is to evaluate the safety, tolerability, and to determine the
pharmacokinetics of QR-010 administered via inhalation in adult homozygous for ΔF508 Cystic
Fibrosis.

Inclusion Criteria:

- Confirmed diagnosis of CF as defined by iontophoretic pilocarpine sweat chloride test
(sweat chloride) of > 60 mmol/L

- Confirmation of CFTR gene mutations homozygous for the ΔF508 mutation

- Body mass index (BMI) ≥ 17 kg/m2

- Non-smoking for a minimum of two years

- FEV1 ≥70% of predicted normal for age, gender, and height, at Screening

- Stable lung function

- Adequate hepatic and renal function

Exclusion Criteria:

- Breast‐feeding or pregnant

- Use of lumacaftor or ivacaftor

- Use of any investigational drug or device

- History of lung transplantation

- Hemoptysis