What happened

After the company reported that a phase 3 trial of its most advanced clinical-stage drug panned out, shares in Global Blood Therapeutics (NASDAQ:GBT) rallied 10.7% on Wednesday.

So what

Global Blood Therapeutics is developing Voxelotor, an oral, once-daily therapy for sickle cell disease (SCD), a life-threatening chronic disease that can starve organs of blood and cause stroke.

A man pumps his arms in the air.


Voxelotor aims to improve patient outcomes by boosting the amount of oxygen held by hemoglobin, the molecules inside red blood cells, so that they hold their normal shape and stop sickling. 

Today, management unveiled results from part A of a two-part study, and overall, the results were good. Specifically,  58% of Voxelotor patients receiving a 1,500 mg dose saw an increase of at least 1 gram per deciliter in hemoglobin at week 12. For comparison, only 9% of patients given a placebo saw such an improvement.

Part A of the study was intended to determine appropriate dosing for part B, which would enroll more patients; however, management discussed its data with the Food and Drug Administration on June 25, and it plans to continue discussions with the hopes of convincing regulators to consider an accelerated approval based on its findings so far.

Now what

About 100,000 Americans have SCD, which can reduce lifespan by 25 to 30 years, so there's a big unmet need for new SCD therapies. However, there's no guarantee the FDA will agree to an early review of Voxelotor.

Nevertheless, the data reported today suggests Voxelotor's ability to increase hemoglobin could translate into improved morbidity and mortality, and that's exciting news for patients, the company, and its investors. Management plans to update investors on its conversations with the FDA about accelerated approval by the end of this year.

If Voxelotor eventually secures an OK, it could move the needle for Global Blood Therapeutics. Treatment currently averages over $200,000 per year for adults with the most common genotype of SCD, so this has the potential to generate hundreds of millions in sales annually.