What happened
Global Blood Therapeutics' (GBT) stock rose by more than 8.2% on Wednesday as of 2:40 p.m. ET on news that the company initiated a phase 2/3 clinical trial for its candidate called GBT601, which is being developed to treat sickle cell disease. The trial will be the biotech's fourth ongoing late-stage sickle cell disease therapy project, not to mention a trio of earlier-stage projects for the same indication.
So what
Global Blood Therapeutics already has one medicine approved to treat sickle cell disease called Voxelotor. GBT601 works with the same mechanism of action as Voxelotor, and it's intended to be a next-generation therapy that might be able to accomplish superior results with smaller doses. Plus, it could only require taking one pill per day, which is an improvement over the older drug.
Because the candidate isn't treading wholly uncharted ground in terms of how it operates, there's a good chance that the clinical trial will go smoothly, and that regulators will be more amenable to granting it approval if it's shown to be efficacious than they would be with a novel medicine.
Now what
The study is now enrolling as many as 60 patients with sickle cell disease. After the initial dose-finding studies of the phase 2 portion of the trial are complete, which is planned to take 12 weeks, the phase 3 portion will track the patients for 48 weeks, homing in on the ideal dose for treatment and comparing the drug's efficacy against a placebo.
Expect an interim update before the end of phase 2, potentially before the end of this year. If patients are responding to treatment more effectively than expected and without any serious adverse events or other mishaps, this biotech stock will probably soar.
