Please ensure Javascript is enabled for purposes of website accessibility

Here's Why ProQR Therapeutics N.V. Is Skyrocketing Today

By Brian Feroldi - Updated Sep 5, 2018 at 12:52PM

You’re reading a free article with opinions that may differ from The Motley Fool’s Premium Investing Services. Become a Motley Fool member today to get instant access to our top analyst recommendations, in-depth research, investing resources, and more. Learn More

Shares jump after the company reports favorable clinical news. Here's what investors need to know.

What happened

After reporting data from a phase 1/2 clinical trial, shares of ProQR Therapeutics N.V. (PRQR 4.97%), a clinical-stage biotech focused on RNA medicines that treat rare genetic diseases, rose 70% as of 12:13 p.m. EDT on Wednesday.

So what

ProQR reported results from its phase 1/2 trial that is testing its compound called QR-110 as a hopeful treatment for LCA10, which is a genetic mutation that usually leads to childhood blindness.

Doctors giving thumbs up

Image source: Getty Images.

Here are the key takeaways from the study:

  • Use of QR-110 led to improvements in vision of the majority of patients when measured using visual acuity and the mobility course performance tests. Specifically, about 60% of patients showed a clinically meaningful response to the drug after three months of treatment. A secondary study also showed that the efficacy effects of the drug persisted beyond three months. 
  • The drug was well-tolerated by study participants, and no serious adverse events were reported.
  • A pivotal phase 2/3 trial is set to start in the first half of 2019.

Commenting on the study results, Dr. David Rodman, an executive vice president of R&D at ProQR, stated:

We observed a clinically meaningful improvement in vision in the treated eye as measured by both mechanistic and potential registration endpoints. Consistent with predictions based on our patient derived optic-cup models, improvement in visual function was observed as early as two months after treatment and was maximal and stable by three months and thereafter. 

Given the news, it isn't hard to figure out why shareholders are having a great day.

Now what

Today's update should provide investors with increased confidence that QR-110 might be the real deal. However, it's also important to remember that great phase 1/2 clinical news doesn't always repeat itself in larger late-stage trials. Given the risk, I'm content to root for ProQR's success from the safety of the sidelines.

Invest Smarter with The Motley Fool

Join Over 1 Million Premium Members Receiving…

  • New Stock Picks Each Month
  • Detailed Analysis of Companies
  • Model Portfolios
  • Live Streaming During Market Hours
  • And Much More
Get Started Now

Stocks Mentioned

ProQR Therapeutics N.V. Stock Quote
ProQR Therapeutics N.V.
$0.67 (4.97%) $0.03

*Average returns of all recommendations since inception. Cost basis and return based on previous market day close.

Related Articles

Motley Fool Returns

Motley Fool Stock Advisor

Market-beating stocks from our award-winning service.

Stock Advisor Returns
S&P 500 Returns

Calculated by average return of all stock recommendations since inception of the Stock Advisor service in February of 2002. Returns as of 05/22/2022.

Discounted offers are only available to new members. Stock Advisor list price is $199 per year.

Premium Investing Services

Invest better with The Motley Fool. Get stock recommendations, portfolio guidance, and more from The Motley Fool's premium services.