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: http://www.activewallst.com/register/.

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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 mediated total chloride transport as measured by NPD.

In the per-protocol population of subjects homozygous for the ΔF508 mutation meeting the pre-specified inclusion criteria (n=7), the average change from baseline in NPD at day 26 was statistically significant, -4.1 mV (p=0.0389). This finding was supported by a change in sodium channel activity and other sensitivity analyses of the NPD measurements, all pointing to strong evidence of restoration of CFTR activity.

In subjects’ compound heterozygous for the ΔF508 mutation, the average change from baseline in NPD was not significantly different at day 26.

The PQ-010-001 Study

PQ-010-001 is a Phase 1b randomized, double-blind, placebo-controlled, dose-escalation 28-day study currently enrolling patients in more than 20 centers in North America and Europe. This study evaluates the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of inhaled QR-010 in a total of 64 CF patients homozygous for the ΔF508 mutation. Exploratory efficacy endpoints in the multiple dose cohorts include sweat chloride, weight gain, CFQ-R Respiratory Symptom Score and lung function, measured by FEV1. The single dose portion of the study consisting of 4 cohorts has been completed. No dose-limited toxicity was observed up to the highest dose tested. The dose escalating multiple-dose study is currently enrolling cohort 6 and top line results are expected to be available in mid-2017.

What is QR-010?

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 DF508 mutation. The DF508 mutation is a deletion of three of the coding base pairs, or nucleotides, in the CFTR gene, which results in the production of a misfolded CFTR protein that does not function normally. QR-010 is designed to bind to the defective CFTR mRNA and to restore CFTR function. QR-010 is designed to be self-administered via an optimized eFlow® Nebulizer (PARI Pharma GmbH). QR-010 has been granted orphan drug designation in the United States and the European Union and fast-track status by the FDA. The QR-010 project has received funding from the European Union’s Horizon 2020 research and innovation program under grant agreement No 633545.

The latest results paves the way for a phase II trial and with solid results the company is optimistic moving ahead into the said trial on the front foot.

Stock Performance

ProQR Therapeutics’ share price finished yesterday’s trading session at $6.15, flat for the day. A total volume of 3.14 million shares exchanged hands, which was higher than the 3 months average volume of 38.63 thousand shares. Shares of the company have advanced 25.77% and 11.21% in the last three months and past six months, respectively. The stock currently has a market cap of $142.99 million.

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