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Global Blood Therapeutics (Nasdaq: GBT) | Company Profile, History, Competitors

 

Global Blood Therapeutics (NASDAQ: GBT) is a biopharmaceutical company with a lead drug candidate, GBT440 (voxelotor), for the treatment of sickle cell anemia. GBT has exclusive worldwide rights to the drug.

As of 2017, Global Blood Therapeutics is undergoing a Phase 3 trial for its leading drug candidate GBT440. The drug is an oral prohylactic that is used to inhibit sickle cell disease.

GBT440 (Voxelotor)

Global Blood Therapeutics' lead drug candidate GBT440 (voxelotor) is a once-a-day oral prophylactic therapy to treat sickle sell disease. GBT440 is a hemoglobin modifier that increases affinity of the protein for oxygen, which inhibits sickle hemoglobin polymerization. This would then inhibit sickle cell disease, which is caused by the mutation of hemoglobin.

GBT440 is currently undergoing a Phase 3 trial called Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization (HOPE). The results are expected to be read out in 2019/2020.

Sickle Cell Disease

Sickle cell disease occurs in a birth rate of 300,000 newborns per year. In the United States, the number of existing sickle cell patients is between 70,000 to 100,000. Sickle cell disease is caused by a mutation in hemoglobin into sickle hemoglobin. This has a tendency to bind into long rods within blood cells that can cause potential blockage in small blood vessels. The disease, which begins in childhood, results in pain due to blocked bloodflow of organs, can lead to organ damage and an early death.

Risks

GGT440 is undergoing Phase 3 trials. Phase 3 data may not support prior Phase 2 data.

Management*\

Dr. Ted Love (CEO): Dr. Ted Love serves as CEO since 2014. Prior, he was EVP of R&D at Onyx Pharmaceuticals. He has also worked at Genentech where he saw the approvals of Herceptin, Xolair, Raptiva and Avastin. He graduated with a BA in molecular biology from Haverford College and an MD from Yale School of Medicine.

Jeffrey Farrow (CFO): Jeffrey Farrow serves as CFO since 2016. Prior, he was CFO of ZS Pharma and CFO of Horizon Therapeutics. He was also VP Finance at Evotec and Renovis. Early in his career, he was an auditor at KPMG.

Jung Choi (Chief Business Officer): Jung Choi serves as Chief Business and Strategy Officer since 2015. Prior, she was VP of Corporate Development at InterMune. She had also worked at Gilead Sciences, where she led the US commercialization of Hepsera. She graduated with a BA and MBA from Stanford University.

Dr. Josh Lehrer (SVP Clinical Development): Dr. Josh Lehrer serves as medical director since 2013. Prior, he led clinical development programs at Genentech. He graduated with a BA from Harvard College and M.Phil in Biological Sciences at Cambridge University.

Financials

 $ USD in millions    2013    2014 2015 2016 2017
 Revenues $0 $0 $0 $0 $0 
 Gross Profit $0 $0  $0  $0  $0 
 Operating Profit($16)($21)($46)($83)($119)
 Net Income($20)($24)($51)($83)($117)
 EPS
($1.73)($3.95)($2.48)($2.76)