ProQR Therapeutics N.V.'s stock price was up more than 66% on Sept. 5 after the Cambridge, Mass.-based biotechnology company said its medicine was effective in treating a rare inherited eye disease.
The company's stock price was trading at $13.20 per share in pre-market trading Sept. 5, 2018, up 66.04% from the previous day's close.
An interim analysis of the company's early stage clinical trial of QR-110 showed the therapy demonstrated rapid and sustained improvement in vision in patients with Leber's congenital amaurosis 10, or LCA 10, due to a specific gene mutation.
QR-110 was well-tolerated in patients with no serious adverse events in the phase 1/2 trial of the therapy. Improvement in visual function was observed as early as two months after treatment and was maximal and stable by three months and thereafter, the company said in a statement.
ProQR said that LCA10, which typically leads to childhood blindness, has no available treatment options.
ProQR said it will stop enrollment in the current trial and progress to a phase 2/3 trial, which will be called Illuminate and is expected to start in the first half of 2019.