On Thursday, shares of leading gene therapy company uniQure QURE are skyrocketing, up over 50% in morning trading after it announced that it will launch a pivotal study next year testing AMT-061, the company's promising gene therapy candidate to treat patients with hemophilia B.

AMT-061 has been advanced after several meetings with the FDA and the European Medicines Agency (EMA). The therapy combines an AAV5 vector with the factor IX (FIX)-Padua gene variant, which contains a protein with a single amino acid substitution and has shown an eight-nine-fold increase in FIX activity compared to wild-type FIX protein in preclinical testing.

AMT-061 is also structurally identical to uniQure's therapy AMT-060, which has already been tested in 10 patients but contains two nucleotide substitutions in the coding sequence for FIX

"Our mission in hemophilia B has always been to develop the safest and most effective gene therapy with the broadest application to patients. We believe AMT-061 moves us closer to this goal, as it has the potential to provide optimized clinical and tolerability benefits to nearly all severe and moderately severe patients with hemophilia B," stated Matthew Kapusta, chief executive officer of uniQure.

Hemophilia B, also known as FIX deficiency or Christmas disease, is a genetic disorder that is caused by missing or defective factor IX, a clotting protein; it is passed down from parents to children, but roughly one-third of cases are caused by a spontaneous mutation. According to the CDC, hemophilia occurs in approximately 1 in 5,000 live births, while about 20,000 people live with the disorder in the U.S.

The FDA agreed that AMT-061 should be included under the existing Breakthrough Therapy designation and Investigational New Drug (IND), while the EMA agreed to label the therapy under PRIME.

The trial will be open-label and evaluate a single dose of AMT-061, and the primary endpoint will be the prevention of bleedings. UniQure said that patients will serve as their own control, with baselines to be established during a six-month observational lead-in phase.

Today's Stocks from Zacks' Hottest Strategies

It's hard to believe, even for us at Zacks. But while the market gained +18.8% from 2016 - Q1 2017, our top stock-picking screens have returned +157.0%, +128.0%, +97.8%, +94.7%, and +90.2% respectively.

And this outperformance has not just been a recent phenomenon. Over the years it has been remarkably consistent. From 2000 - Q1 2017, the composite yearly average gain for these strategies has beaten the market more than 11X over. Maybe even more remarkable is the fact that we're willing to share their latest stocks with you without cost or obligation. See Them Free>>

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report

uniQure N.V. (QURE): Free Stock Analysis Report

To read this article on Zacks.com click here.

Zacks Investment Research

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.