ProQR Therapeutics, a Leiden, NL based biotech company focused on development of drugs to treat severe genetic disorders, recently announced that its PQ-010-002 clinical trial is open for patient enrollment. The study’s primary endpoint is the estimation of the effect of topical administration of QR-010 on the nasal mucosa in the restoration of CFTR function, as measured by Nasal Potential Difference (NPD).
QR-010 is an experimental therapy designed to treat people with cystic fibrosis that have at least one copy of the ΔF508 mutation. In seventy percent of cystic fibrosis patients, the disease is caused by the ΔF508 mutation in the CFTR gene. QR-010 is unique in that it is designed to treat the underlying defect in CF, restoring normal function of the CFTR protein and halting progression of the disease.
PQ-010-002 is an open-label, exploratory clinical trial that is assessing the effect of QR-010 in ∆F508 homozygous and compound heterozygous cystic fibrosis (CF) patients.
“We are excited to announce that the second clinical study of QR-010 is now open and enrolling,” said Noreen R. Henig, MD, Chief Development Officer of ProQR. “This study is an important proof-of-concept study that will test the activity of QR-010 in the treatment of CF. In animal models of CF, QR-010 showed the ability to restore CFTR mediated NPD to normal or wild-type levels. Repeating the same test in individuals with CF will provide an important first signal of the therapeutic potential of QR-010.”
PQ-010-002 is a 28-day clinical trial that is being conducted across 5 European and US clinical sites experienced in performing NPD measurements.
NPD is a CF diagnostic test which is also used to examine the therapeutic benefit of investigational drug agents. A total of 16 CF patients (either homozygous or compound heterozygous for the ∆F508 mutation), are taking part in the study. Assessments of NPD and sweat chloride will be conducted prior and following local treatment in the nose with QR-010 three times per week for a period of four weeks.
“RNA-based therapeutics are a novel approach to the treatment of the gene mutations that cause CF. The preclinical nasal potential difference studies of QR-010 in the mouse models of CF are quite compelling. The proof-of-concept study of QR-010 will support the understanding of QR-010’s impact on CFTR function in patients with CF with the common ∆F508 CFTR mutation,” said John P. Clancy, Professor of Pediatrics and Research Director, Division of Pulmonary Medicine, Cincinnati Children’s Hospital and a member of the Cystic Fibrosis Foundation Therapeutic Development Network’s leadership team.
PQ-010-002 will be conducted at the same time as PQ-010-001 as an ongoing Phase 1b tolerability and safety clinical trial of QR-010 in patients with CF who are homozygous for the ∆F508 mutation. The trial will also evaluate QR-010 pharmacokinetics and preliminary clinical efficacy outcomes.
“We are very proud of our teams that have moved QR-010 from an interesting idea to two enrolling clinical studies in just three years,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “We are committed to bringing important new therapies to patients with CF and these studies are important steps in understanding the potential of QR-010.”