Shares of Global Blood Therapeutics Inc. (NASDAQ:GBT), a clinical-stage biotech developing new drugs to treat blood-based disorders, surged 47.1% higher in January, according to data from S&P Global Market Intelligence. The FDA's decision to grant the company's lead candidate a Breakthrough Therapy designation was the main driving force behind last month's gain.
The Breakthrough Therapy designation is the FDA's favorite tool for speeding up development of drugs the agency feels have potential to truly make a difference for patients with serious ailments. The FDA designated Global Blood Therapeutics Inc.'s lead candidate, voxelotor, a Breakthrough Therapy for the treatment of sickle cell disease last month.
Breakthrough Therapy announcements don't usually lead to such dramatic stock price movements. This one carries extra weight because it's the first for sickle cell disease despite the presence of several promising sickle cell disease candidates in clinical-stage development at the moment.
As a once-daily tablet, voxelotor would have a huge convenience advantage over complex gene therapies in development for sickle cell disease from bluebird bio (NASDAQ:BLUE) and Crispr Therapeutics (NASDAQ:CRSP). Since Crispr hasn't even begun human testing with any candidates and Bluebird is still fine-tuning its approach, Global Blood Therapeutics could also gain a big first-to-market advantage.
Of course, voxelotor still needs to generate some winning data during the ongoing phase 3 Hope study. Part A of the trial will finally show us how well the drug performs against a placebo after a few months of treatment. Look for top-line data in the first half of the year.