Global Blood Therapeutics Soared 15% What Happened?
Shares of Global Blood Therapeutics (NASDAQ: GBT), a clinical-stage biopharmaceutical company that focuses its research and therapies on combating blood-based disorders, soared higher by more than 15 percent after Monday’s market close.
The company said in a press release on Monday that it reached an agreement with the U.S. Food and Drug Administration (FDA) concerning the design of its pivotal trial exploring its therapy called GBT440.
GBT440 is an oral, once a day therapy for patients suffering from sickle cell disease. The therapy is designed to increase a patient’s hemoglobin’s affinity for oxygen. The FDA has already granted GBT440 fast track designation and orphan drug designation and the therapy will now be evaluated in a Phase 3 clinical trial.
Global Blood Therapeutics confirmed in its press release that the FDA has given the necessary approval to commence a Phase 3 clinical trial called HOPE. The study will begin screening patients in December who have had at least one episode of vaso-occlusive crisis over the past year and top-line results are expected in the first half of 2019.
Part A of the HOPE study will compare two doses of GBT440, a 900 mg dose and 1500 mg dose versus a placebo. This trial will involve up to 150 adolescents and adults at sites located across the world.
Part B of the HOPE study will include 250 patients who will receive either GBT440 or a placebo as a continuation of Step A.
The primary endpoint of the study, that is the necessary objectives to be considered successful, will be to demonstrate that patients who receive GBT440 show a statistically significant increase in hemoglobin at 24 weeks of treatment compared to those who received a placebo.
“We are extremely pleased with our interactions and outcome of our pre-Phase 3 process with the FDA. We believe that this agreed-upon study design underscores our common goal to address the urgent unmet medical need for new disease-modifying preventative therapies for SCD and the importance of innovative regulatory strategies to bring new therapies to market to treat these patients,” said Ted W. Love, M.D., president and chief executive officer of GBT. “Solidification of the path forward for our pivotal study is an important milestone and we believe the HOPE Study design has a strong grounding in GBT440’s mechanism of action, including its potential to fundamentally modify the course of the disease by inhibiting sickle hemoglobin polymerization.”