ProQR Therapeutics has announced that dosing of cystic fibrosis (CF) patients in its Phase 1b clinical trial of QR-010 has been completed.
The PQ-010-001 study is a Phase 1b, 28-day, randomised, double-blind, placebo-controlled safety and tolerability trial, conducted in patients that have CF.
A total of four dose levels were studied: 6.25, 12.5, 25 and 50mg of QR-010 in solution per dose administered via inhalation. The study design consisted of eight cohorts of eight patients for a total of 64 patients. In each cohort, six patients received QR-010 and two patients received placebo. In cohorts one to four, a single dose of QR-010 was administered, and in cohorts five to eight 12 doses of QR-010 were administered over a four-week period.
The Phase 1b study is a first-in-human trial designed to primarily assess safety, tolerability and pharmacokinetics of QR-010. A number of exploratory efficacy endpoints are also being assessed including sweat chloride, weight gain, change in CFQ-R Respiratory Symptom Score and FEV1.
Noreen Henig, chief medical officer at ProQR, said: “QR-010 has the potential to be an innovative RNA [ribonucleic acid] therapy for patients with CF due to the F508del mutation. Completion of the Phase 1b study is an important step in development, and builds upon the preclinical data and positive PQ-010-002 study where QR-010 demonstrated a direct effect on restoring CFTR function.
“We are grateful to the patients and the medical community who participated in this early trial. ProQR is committed to creating meaningful RNA therapies for patients with CF.”