ProQR shares spike on positive PoC study for lead CF drug
“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.