Summary
- Global Blood Therapeutics can be a “trick or treat” investment to shareholders.
- The stellar response of the single patient with SCD can either foretell positive phase 3 trial or upcoming failure.
- Voxelotor can potentially capture peak sales ranging from $2B to $3B.
- This idea was discussed in more depth with members of my private investing community, Integrated BioSci Investing. Become a member today >>
Global Blood Therapeutics (NASDAQ:GBT), a firm focusing on the innovation of medicine to treat blood disorders, made our featured presentation as the Halloween 2017 stock. Yesterday, the stock won our Bioscience Catalyst trade of the day, as it earned more than 10% profits for traders. The winning momentum extended into today’s trading: shares traded up $1.15 at $39.80 (for another 2.98% profits). As we previously mentioned, the new fortunes are due to the stellar pipeline development.
Accordingly, the firm reported robust response in the single patient (having moderate to severe symptoms associated with sickle cell disease) who was treated with lead molecule GBT-440 (voxelotor). While the data on a single patient can indicate favorable results for the phase 3 Hope trial, the limited info can be an overly optimistic presentation. That being said, we’ll dissect the arguments for Global Blood Therapeutics for readers to gauge whether the firm is a trick or treat this Halloween.
“Affecting millions of people worldwide, SCD is a major health concern.According to Statistics from the CDC showed that there are 100,000 Americans living with the said condition (which occurs in 1 out of every 365 African-Americans, and 1 out of every 16,300 Hispanic-Americans). Prominently, SCD genes will always remain in the population, because “carriers” (those having the diseased plus normal genes) have other survival benefits. Most surprisingly, there is one carrier out of 13 blacks.”
HbS (sickle hemoglobin or abnormal hemoglobin) in its deoxygenated state tends to bind together to form rods, thus leading to the deformation of red blood cell (“RBC”). Gradually, the deformed RBCs become stiff (and result in a reduction in the blood flow). In addition, those sickled cells are easily degraded (leading to inadequate blood level or anemia).
The combinations of anemia and flow reduction cause oxygen deprivation throughout the body, thereby leading to a hypoxic state. Ultimately, hypoxia induces the “sickle cell crisis” - a painful condition, requiring multiple hospital admissions and blood transfusion that, in and of itself, causes other complicating effects.
To serve the robust demand for better SCD treatment, voxelotor can come in handy by limiting the polymerizing (or clumping) of Hbs. With the ability to galvanize normal hemoglobin function (and to improve oxygen flow), the aforesaid drug can potentially ameliorate the dreaded adverse effects of SCD.
With the market cap of $1.74B and the potential blockbuster voxelotor to service the large SCD market - one that is in dire need for better treatment - Global Blood Therapeutics can give investors the gargantuan treat going beyond 2017. Alternatively, the investment can be tricky due to the uncertain outcomes of its key developing therapeutic, voxelotor.
You can join Integrated BioSci Investing community, where we provide consultation regarding the outcomes of the Hope trial for subscribers. Our service can help you achieve the investor’s edge in clinical trial forecasting.
