Image Source: The Economic Times
Company Thesis
Global Blood Therapeutics (GBT) is a mid-cap biotech company currently investigating potential drug candidates in hematology. The company's flagship product, Voxelotor, is an oral, once-daily therapy that modulates hemoglobin’s affinity for oxygen, which is hypothesized to inhibit hemoglobin polymerization in patients with sickle cell disease and has recently been approved by the FDA.
Despite demonstrating statistical significance in Phase 3, critics have argued the drug does not possess clinical significance as its is unclear whether patients treated with Voxelotor demonstrated reductions in number of vaso-occlusive crises.