In December, Global Blood Therapeutics (GBT) unveiled trial data for voxelotor, a sickle cell disease (SCD) treatment that it believes is good enough to secure an early go-ahead from regulators. The potential for an early approval, however, may hinge on new data the company is scheduled to reveal at the 24th European Hematology Association (EHA) Congress in an oral presentation on Friday, June 14.
The updated results could send shares soaring if it strengthens the argument for an accelerated approval, or it could cause a steep sell-off if it underwhelms. Here's what you should know about voxelotor and what data to focus on Friday.
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An important need for new options
SCD, a genetic disorder that causes painful complications and stroke, takes a big toll on patients.
There are over 100,000 SCD patients in the U.S., plus another 60,000 in Europe. The disease shortens life spans by 20 years or more, despite spending on treatment that can total in the hundreds of thousands of dollars per year.
If eventually approved by regulators, Global Blood Therapeutics' voxelotor could become the first disease-modifying drug available. By boosting oxygen in hemoglobin, voxelotor may prevent red blood cells from forming their sickle shape, reducing anemia and vaso-occlusive crisis (VOC), a debilitating condition in which sickled red blood cells obstruct blood circulation.
So far, so good
The data reported in December showed that a 1,500 mg daily oral dose of voxelotor increased hemoglobin by at least 1 gram per deciliter in 65% of patients age 12 and older at 24 weeks. Only 10% of patients who received a placebo had a similar improvement.
If that improvement prevents red blood cells from being destroyed faster than they're made, then it may prevent organ damage caused by inadequate oxygen due to anemia, while also lowering the risk of VOCs.
That thinking was backed up by researchers noting numerically fewer VOC events and a lower per-person-per-year rate of VOC occurrence in patients participating in its trial.
What to watch
The December data only included results from 154 patients participating in the company's phase 3 trial. On Friday, we could get results from an additional 118 patients.
For shares to head higher, investors will want additional conviction that voxelotor can disrupt this market. A stable or improving proportion of patients with 1 gram per deciliter improvement is key, but even more needle-moving could be any data presented on the secondary endpoint evaluating voxelotor's ability to reduce VOCs.
The stakes for investors are high. Voxelotor is Global Blood Therapeutics' only drug in development. And in the future, voxelotor could face competition from gene therapies, including more than one under development at bluebird bio.
If voxelotor's data is solid, then it could assuage any fears over approval and diminish some of the competitive threat. But if its update disappoints, then shares could drop substantially. Because of the hit-or-miss nature of this data, investors should pay close attention on Friday.
