We issued an updated report on Global Blood Therapeutics, Inc. GBT on Sep 2.
The company’s lead product candidate is voxelotor (GBT440), an oral, once-daily treatment for sickle cell disease (SCD). SCD is a chronic, inherited blood disorder that affects hemoglobin. The company is evaluating voxelotor in a phase III HOPE study in adult and adolescent patients with SCD. Data from the study, presented in December 2018, demonstrated rapid, robust and sustained improvements in hemoglobin levels and measures of hemolysis, with a favorable safety and tolerability profile for both 1500 mg and 900 mg doses.
In the second quarter of 2019, the company announced a final agreement with the FDA on the design of the post-approval confirmatory study of voxelotor to demonstrate stroke risk reduction benefit of the drug, considering transcranial doppler (TCD) flow velocity as the primary endpoint.
Shares of the company have gained 12% year to date against the industry’s decline of 1.7%.
The FDA approved a rolling submission of voxelotor for the potential treatment of SCD. The company expects to submit an NDA in the second half of 2019.
In August 2018, the company entered an exclusive worldwide license agreement with drug giant Roche Holding AG RHHBY for the development and commercialization of inclacumab, a novel, fully human monoclonal antibody against P-selectin, as a treatment for vaso-occlusive crises (“VOC”) in patients with SCD.
However, Global Blood has no approved products in its portfolio yet. The company has only one candidate, voxelotor, which is in late-stage studies. If the company is unable to receive regulatory approval for the drug, its business will be significantly affected, as inclacumab is in early stages of development.
The company is also battling stiff competition. The FDA has accepted Novartis’ NVS Biologics License Application (BLA) for its investigational sickle cell medicine, crizanlizumab (SEG101), and granted Priority Review to the same. Pfizer PFE is also developing rivipansel for the treatment of VOC in patients with SCD.
Global Blood currently has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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