ProQR Therapeutics N.V. a company developing RNA medicines for the treatment of severe diseases including cystic fibrosis, recently announced that it has initiated the enrollment of patients into PQ-010-001, a Phase 1b clinical trial for QR-010. The compound is a new RNA-based therapeutic drug designed to restore the genetic mutation in the mRNA of patients with cystic fibrosis (CF) that have the DF508 mutation. The therapy could represent a future treatment that targets the underlying cause of CF.
“We are proud to announce that our first clinical study of QR-010 is now open and actively enrolling,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “Since the foundation of the company 3 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.”
“QR-010 is an innovative approach to target the underlying defect of CF. We are very pleased to participate in the first clinical trial for this novel compound,” said Professor Stuart Elborn of Queen’s University Belfast and past-President of the European Cystic Fibrosis Society.
PQ-010-001 is a double-blind, placebo-controlled, randomized, Phase 1b 28-day clinical trial that will be conducted across 20 research sites worldwide.
This is the first clinical trial assessing the tolerability, pharmacokinetics and safety of single-dose and multiple ascending doses of QR-010 (inhaled) in a population of 64 patients with CF who have two homozygotes copies of the DF508 mutation.
The clinical trial will assess the efficacy of the drug as well as weight gain, CFQ-R Respiratory Symptom Score, sweat chloride, and lung function examined through FEV1. QR-010 will be given to patients through inhalation for up to 3 times per week, for up to 4 weeks. In addition to this clinical trial, the company will also conduct a parallel proof-of-concept Nasal Potential Difference (NPD) trial, expected to begin enrollment of 16 patients with CF in last trimester of 2015 who are either homo- or heterozygous for the DF508 mutation.
“The Phase 1b study and the NPD proof-of-concept study will provide a strong, early signal as to the therapeutic potential of QR-010,” said Noreen R. Henig, MD, Chief Development Officer of ProQR.