ProQR shares spike on positive PoC study for lead CF drug
Shares of the Dutch biotech ProQR Therapeutics $PRQR shot up this morning after it released upbeat results from an early proof-of-concept study for its cystic fibrosis drug QR-010.
“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.”
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