Why Nightstar Is Thursday’s Top Biotech

By Chris Lange Updated Jan 11, 10:20PM EST · Published Jun 14, 1:36PM EDT

Follow 24/7 Wall St. on Google

This post may contain links from our sponsors and affiliates, and Flywheel Publishing may receive compensation for actions taken through them.

Why Nightstar Is Thursday’s Top Biotech — © Thinkstock

Nightstar Therapeutics PLC (NASDAQ: NITE) shares saw a massive gain on Thursday after the firm announced a crucial decision from the U.S. Food and Drug Administration (FDA). Essentially, the agency has granted Regenerative Medicine Advanced Therapy (RMAT) designation to NSR-REP1.

For some quick background, NSR-REP1 is the company’s lead product candidate and is currently in Phase 3 development for the treatment of choroideremia, a rare, degenerative, genetic retinal disorder that leads to blindness.

The designation for NSR-REP1 was based on clinical data supporting the maintenance and improvement of visual acuity from completed Phase 1/2 trials

The RMAT designation is an expedited program for the advancement and approval of regenerative medicine products. RMAT allows companies developing regenerative medicine and gene therapies to work more closely and frequently with the FDA, and grants all of the benefits of Breakthrough Therapy Designation, including eligibility for priority review, rolling review and accelerated approval.

[nativounit]

Dave Fellows, Chief Executive Officer, commented:

Receiving RMAT designation for NSR-REP1 highlights the potential of this gene therapy to maintain and improve visual acuity in choroideremia. This designation further underscores a recognition of the serious nature of choroideremia and the urgent need to develop new treatments for those affected by inherited retinal diseases that would otherwise lead to blindness. We look forward to working closely with the FDA to discuss the NSR-REP1 development program and to determine how we can accelerate the pathway for making NSR-REP1 available to choroideremia patients.

Shares of Nightstar were last seen up about 20% at $16.77, with a consensus analyst price target of $28.50 and a 52-week range of $10.01 to $24.93.

[recirclink id=469109]

[wallst_email_signup]

About the Author Chris Lange →

Chris Lange is a writer for 24/7 Wall St., based in Houston. He has covered financial markets over the past decade with an emphasis on healthcare, tech, and IPOs. During this time, he has published thousands of articles with insightful analysis across these complex fields. Currently, Lange's focus is on military and geopolitical topics.

Lange's work has been quoted or mentioned in Forbes, The New York Times, Business Insider, USA Today, MSN, Yahoo, The Verge, Vice, The Intelligencer, Quartz, Nasdaq, The Motley Fool, Fox Business, International Business Times, The Street, Seeking Alpha, Barron’s, Benzinga, and many other major publications.

A graduate of Southwestern University in Georgetown, Texas, Lange majored in business with a particular focus on investments. He has previous experience in the banking industry and startups.