Data from two clinical studies of an investigational cystic fibrosis therapy were presented at the European Cystic Fibrosis Society (ECFS) conference, according to ProQR Therapeutics.

The drug, QR-010, is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of the disease by targeting the mRNA in CF patients that have the F508del mutation. The drug demonstrated safety and systemic uptake after a single dose through inhalation, according to oral and poster presentations of an ongoing Phase 1b study.

An oral presentation titled “QR-010, an investigational RNA therapeutic, improves CFTR activity in cystic fibrosis subjects homozygous for the F508del mutation” was presented on Friday June 9 by Steve Rowe, MD, professor of Pulmonary, Allergy and Critical Care Medicine at University of Alabama and Director of the Gregory Fleming James Cystic Fibrosis Research Center, and director of the CFF Therapeutics Development Network. A poster presentation by ProQR Therapeutics was also made on June 9 titled: “QR-010 via inhalation is safe, well-tolerated, and achieves systemic concentrations in a single ascending dose study in subjects with cystic fibrosis homozygous for the F508del CFTR mutation.”

“QR-010 is an innovative approach to restoring CFTR function in patients with CF due to the F508del mutation.  Last year, we demonstrated that QR-010 restores CFTR function as measured by a very specific assay, the nasal potential difference. Now we have shown that QR-010 can be detected in the blood following a single dose inhalation.  We believe these results support the potential that QR-010 can treat all manifestations of CF,” said Noreen R. Henig, MD, Chief Medical Officer of ProQR. “I am very pleased that enrollment of the Phase 1b study is expected to be completed this month and we are looking forward to unblinding the study and report the top-line data from this phase 1b trial.

More information is available on the ProQR website.