“Patients with CF feel and do better when the CFTR protein channel works more normally,” said Noreen R. Henig, chief development officer of ProQR, in a statement. “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. Our proof-of-concept NPD study did exactly that in CF patients homozygous for ∆F508; it demonstrated that CFTR protein channels are active in this cohort following administration of QR-010 as measured by the total chloride response. 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.”
Investors bid up the stock price 33% on Thursday in pre-market trading. And that followed an 11% spike on Wednesday.
Investigators at the RNA company explained that the proof-of-concept study evaluated the impact of QR-010 on the nasal potential difference (NPD) assay, a measure of CFTR function. The study enrolled 18 CF patients, 10 homozygous for the ΔF508 mutation and 8 compound heterozygous (one copy of the ΔF508 mutation and one copy of another cystic fibrosis disease-causing mutation). The primary endpoint for each cohort was the change from baseline in CFTR-mediated total chloride transport as measured by NPD.
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