ProQR Begins Enrollment in Clinical Trial Testing New Cystic Fibrosis Therapy

ProQR Begins Enrollment in Clinical Trial Testing New Cystic Fibrosis Therapy

ProQR therapeutics recently announced that they have begun enrolling patients for their open-label, exploratory trial PQ-010-002, which will evaluate the effect of their novel investigational therapeutic formulation QR-010 in ∆F508 homozygous (defective gene carrying two allelic copies) and compound heterozygous (defective gene carrying one copy of the ∆F508 mutation and one other disease causing mutation) cystic fibrosis (CF) patients. This trial will seek to estimate QR-010 efficacy after topical administration of the therapy in the nasal mucosa in order to improve the functionality of the Cystic Fibrosis Transmembrane Conductance Receptor (CFTR) protein, a defect in which is the pivotal cause of CF. Efficacy will be measured based on Nasal Potential Difference. NPD is a diagnostic test which measures how well sodium and chloride ions flow across the mucous membranes in the nose.

QR-010 is a unique, first-of-its-kind therapy that may be able to treat the root cause of CF, repairing the genetic mutation in the mRNA of the CFTR gene as a result of the ∆F508 mutation. The PQ-010-002 trial will be a 28-day study conducted in parallel with PQ-010-001 (the ongoing Phase 1b safety and tolerability study of QR-010 in CF patients homozygous for the ∆F508 mutation, where the drug is being delivered to the lungs via inhalation) across 5 sites located in the US and Europe that are experienced in conducting NPD measurements. At least 16 patients with either homozygous or compound heterozygous alleles of the ∆F508 mutation in the CFTR gene are likely to be enrolled and NPD, and sweat chloride measurements are expected to be conducted before and after topical administration of QR-010 on the nasal mucosa three times a week for four weeks.

Noreen R. Henig, MD, Chief Development Officer of ProQR, commented on the study, stating, “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.”

According to 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, “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.”

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