Phase 1 Trial Data on RNA-based Therapy for CF to Be Presented at NACFC 2016

Phase 1 Trial Data on RNA-based Therapy for CF to Be Presented at NACFC 2016

ProQR Therapeutics recently announced that new data from an ongoing proof-of-concept clinical study, called PQ-010-002, evaluating the effect of an RNA-based therapy (QR-010) on cystic fibrosis patients, will be presented at the North American Cystic Fibrosis Conference (NACFC), taking place Oct. 27–29 in Orlando, Florida.

The company is developing RNA-based therapies to treat severe genetic disorders, including CF and Leber’s congenital amaurosis Type 10.

ProQR Therapeutics will also release preliminary data from a Phase 1b study (PQ-010-001) evaluating both the safety and tolerability of QR-010 at the Orlando conference. The study was performed using single-dose cohorts, and a multiple-dose cohort study is currently in the enrollment phase (NCT02532764).

The Phase 1 proof-of-concept study is being conducted over 28 days at five centers in the U.S. and Europe. It plans to enroll 16 patients with CF (with another 16 possibly added later), of whom eight are homozygous for the ΔF508 mutation (carrying two copies of the mutation in the CFTR gene), and eight are compound heterozygous (one copy of the ΔF508 mutation, and one other CF-causing mutation). Researchers will measure the activity of the CFTR protein in the nasal epithelium in CF patients (achieved by measuring total chloride transport in the nasal cavity), determining QR-010’s effect on CFTR function through the degree to which chloride transport is restored. At NAFC, researchers will present top-line data from the 16 treated patients.

“We are excited to announce data from our two ongoing clinical studies of QR-01,” Daniel de Boer, chief executive officer of ProQR, said in a press release. “In our pre-clinical models QR-010 showed an unprecedented restoration of CFTR function in the NPD [nasal potential difference] test. Because NPD in CF patients has positive predictive value for clinical benefit, we would see a positive outcome in this study as an important proof of efficacy.”

QR-010 is a therapeutic targeting the mRNA (an RNA subtype) in CF patients that have the ΔF508 mutation. By binding to the defective mRNA in the CFTR protein, QR-010 is expected to restore CFTR function. Self-administered, QR-010 is delivered through a small, hand-held aerosol delivery device, or nebulizer, directly into the patients’ lungs. QR-010 was granted orphan drug designation in the United States and the European Union.

PQ-010-002 is also currently enrolling patients at its sites in the U.S., France, and Belgium. More information is available through the study’s clinical website (NCT02564354).


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