Dutch biotech firm ProQR Therapeutics has initiated a Phase Ib clinical trial (PQ-010-001) of QR-010, a new investigational RNA therapeutic designed to repair the genetic mutation in the mRNA of cystic fibrosis (CF) patients due to the DF508 mutation.
QR-010 is a RNA-based oligonucleotide designed to address the underlying cause of the disease by repairing the mRNA defect encoded by the DF508 mutation in the CFTR gene of these patients.
The 28-day randomised, double-blind, placebo-controlled Phase Ib trial will be conducted in 20 centres across the world.
The trial will evaluate the safety, tolerability and pharmacokinetics of single and multiple ascending doses of inhaled QR-010 in 64 CF patients carrying two copies (homozygotes) of the DF508 mutation.
ProQR CEO Daniel de Boer said: "We are proud to announce that our first clinical study of QR-010 is now open and actively enrolling.
"Since the foundation of the company three years ago our team has worked very hard towards this step in the development of a therapy for CF, and we are excited to have reached this important milestone."
As exploratory efficacy endpoints, the trial will also evaluate sweat chloride, weight gain, CFQ-R Respiratory Symptom Score and lung function, measured by FEV.
In this trial, QR-010 will be administered through inhalation for up to three times a week for a maximum period of four weeks.
The Phase Ib trial will be conducted in parallel with a proof-of-concept Nasal Potential Difference (NPD) study, which will begin enrolment of 16 CF patients that are either homo- or heterozygous for the DF508 mutation in third quarter of this year.
ProQR chief development officer Noreen Henig said: "The Phase Ib study and the NPD proof-of-concept study will provide a strong, early signal as to the therapeutic potential of QR-010."
CF, a genetic disease, affects an estimated 70,000 to 100,000 patients across the world and causes early morbidity and mortality, while the disease currently has no cure.
Image: "Clubbing" of the fingers is a classic feature of Cystic Fibrosis. Photo: courtesy of Jerry Nick, M.D.