Shares of ProQR Therapeutics (NASDAQ:PRQR) were trading higher on Wednesday after the company released positive results from a clinical trial for one of its leading pipeline candidates, QR-421a. The biotech is also benefiting from bullish commentary from a Wall Street analyst. As of 1:07 p.m. EDT, ProQR stock was up by 8% after rising by as much as 13.5% earlier today.
QR-421a is an experimental treatment for Usher syndrome, a rare disorder that, among other things, can cause loss of vision. In a phase 1/2 clinical trial for QR-421a, the treatment was well tolerated by patients and produced improvement in several measures of vision, including visual acuity, visual field, and more.
Aniz Girach, chief medical officer of ProQR Therapeutics, said, "With just a single dose, QR-421a demonstrated clinical proof of concept with benefit observed in treated eyes compared to the untreated eyes in multiple concordant measures of vision."
Meanwhile, MP Securities analyst Liisa Bayko raised her price target on ProQR Therapeutics' stock to $35, up from $19. She also kept an outperform rating on the stock. According to the analyst, QR-421a seems increasingly promising after the clinical trial results that ProQR Therapeutics released. Bayko raised the probability of success of the QR-421a program on patients with Usher syndrome in her model to 60%, up from 20%. Note that ProQR Therapeutics is trading for $5.79 per share as of this writing, which means the $35 price target represents a significant upside.
ProQR Therapeutics plans to start two phase 2/3 studies for QR-421a by the end of the year. As is usual with biotech companies, ProQR could see its shares plunge if these clinical trials don't yield positive results, while the stock could skyrocket if the data indicates that QR-421a is safe and effective. Is ProQR Therapeutics' risk-reward profile attractive? In my view, the answer is no, but investors comfortable with above-average risk may reach a different conclusion.