Global Blood Therapeutics (GBT) is extending yesterday's breakout to a 2+ year high after the company received breakthrough therapy designation (BTD) for voxelotor, formerly known as GBT440, its oral drug for sickle cell disease.
The FDA selectively grants BTD to expedite the development and review of drugs that have demonstrated preliminary clinical evidence indicating the potential for substantial improvement over available therapy.
Sickle cell is a debilitating disease that is eventually fatal and affects people of African descent. There are no drugs on the market that really help SCD patients.
Voxelotor is the first treatment to ever receive BTD from the FDA for SCD.
The stock has taken off in response to the news because the company said the BTD was based in part on preliminary efficacy and safety data from Part A of the Phase 3 HOPE Study, which has yet to be reported.
The company plans on disclosing top-line data from Part A of the HOPE study in the first half of 2018, but the BTD clearly bodes well for that read out.
The Part A study will give the company more clues to set up a Phase 3 Part B of the HOPE study, which should report top line data in the first half of 2019.
The stock is back to levels not seen since late 2015, soon after the IPO. Recent data indicates that close to 20% of the 34 million share float.
Valuation is the big question at this juncture. Sickle cell has huge market potential. The company has a ~$2.4 billion market cap and ~$300 million in cash after its recent offering.