Global Blood Focuses on Developing Lead Candidate Voxelotor

We initiated coverage on Global Blood Therapeutics GBT on Mar 28.

Global Blood Therapeutics’ lead product candidate is voxelotor (GBT440), an oral, once-daily treatment for sickle cell disease (SCD).

Shares of the company have increased 7.7% in the past year against the industry’s decline of 13.9%.

The company is evaluating voxelotor in a phase III HOPE study in adult and adolescent patients with SCD. It is also evaluating the safety and pharmacokinetics of single and multiple doses of voxelotor in a phase IIa study in adolescent and pediatric patients to treat SCD.

The company’s clinical studies on voxelotor are progressing fast. Data from the phase III HOPE study in adult and adolescent patients, 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. The company plans to present full data from the study in June 2019. However, with positive data in hand, Global Blood Therapeutics proposed an accelerated approval pathway to the FDA, using hemoglobin as the primary endpoint. The FDA has agreed to a rolling submission of its new drug application for the drug.

Global Blood Therapeutics plans to initiate a post-approval confirmatory study later in 2019 to demonstrate stroke risk reduction benefit of voxelotor using transcranial doppler (TCD) flow velocity as the primary endpoint.

The potential approval of voxelotor will be a huge boost for the company as the current treatment options for SCD are limited. We note that SCD is marked by red blood cell or RBC destruction, occluded blood flow and hypoxia, leading to anemia, stroke, multi-organ failure, severe pain crises and shortened patient life span. In clinical studies, it has been observed that voxelotor reduces markers of red blood cell destruction, leads to improvements in anemia and markers of tissue oxygenation, and reduces the number of sickled RBCs. As a result, the company is optimistic about voxelotor and its potential to treat this significantly unmet disease.

However, many companies are developing hematopoietic stem cell transplantation, gene therapies and gene editing therapies to treat SCD. bluebird bio BLUE is developing the LentiGlobin BB305 gene therapy and Novartis AG NVS is developing crizanlizumab, an anti-P-selectin monoclonal antibody for the treatment of vaso-occlusive crises (VOC)  in patients with SCD. Novartis expects to file a new drug application for crizanlizumab in the first half of 2019. Pfizer PFE is also developing rivipansel for the treatment of VOC in patients with SCD. So voxelotor, on approval, is expected to face significant competition.


Global Blood Therapeutics, Inc. Price


Global Blood Therapeutics, Inc. Price | Global Blood Therapeutics, Inc. Quote

Zacks Rank

Global Blood currently carries 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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