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Blog Coverage ProQR Cystic Fibrosis Drug Meets Primary End Point

LONDON, UK / ACCESSWIRE / October 28, 2016 / Active Wall St. blog coverage looks at the headline from ProQR Therapeutics N.V. (NASDAQ: PRQR) as the company presented on October 27th, 2016, clinical data showing for the first time that its experimental drug QR-010 improves the function of a key protein that is defective in patients with cystic fibrosis (CF). Register with us now for your free membership and blog access at:

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Cystic Fibrosis

Cystic fibrosis is the most common fatal inherited disease in the Western world and affects an estimated 70,000 to 100,000 patients worldwide. There is no cure for CF. Disease manifestations lead to a shortened life expectancy with a median age of death of 27 years. Although over 1,900 CF-causing gene mutations have been identified, approximately 70% of all CF patients are affected by the DF508 mutation.

The Results

ProQR Therapeutics announced that clinical study PQ-010-002, a proof-of-concept study of nasal potential difference (NPD), demonstrated that QR-010 restored CFTR function in a cohort of homozygous ∆F508 CF patients. The company stated that the study met its primary endpoint in this cohort as measured by a change in total chloride response following 4 weeks of treatment with QR-010. In the compound heterozygous ∆F508 cohort, no meaningful difference was found.

ProQR also announced that clinical study PQ-010-001 completed all four single-dose cohorts. PQ-010-001 is a placebo-controlled Phase 1b study in subjects with CF homozygous for ∆F508. QR-010 was observed to be safe and well-tolerated in all four single dose cohorts. The multiple dose cohorts in this study are on-going and top line safety, tolerability and exploratory efficacy data from this study are expected in mid-2017.

"Patients with CF feel and do better when the CFTR protein channel works more normally. Our important first step in helping patients with CF was to demonstrate that QR-010 could restore CFTR function in patients with CF due to ∆F508, the most common mutation. Having achieved this major step, we have increased confidence in QR-010's potential to make a meaningful clinical impact for patients and will move forward with an aggressive development plan," said Noreen R. Henig, M.D., Chief Development Officer of ProQR.

What is PQ-010-002 study?

PQ-010-002 was an open-label, proof-of-concept study evaluating the effect of QR-010 on the nasal potential difference (NPD) assay, an important measurement of CFTR function. The study was conducted in 5 NPD specialized centers in the US and Europe. QR-010 was applied topically to the nasal mucosa 12 times over a period of 4 weeks. Primary endpoint for each cohort was the change from baseline in CFTR...