January 4, 2017: JPMorgan Chase initiates coverage of biotechnology firm Global Blood Therapeutics with an Overweight rating and a price target of $25. JPMorgan thinks GBT440 is a promising wholly owned asset targeting a broad and underserved market, as such current levels could provide an attractive entry point despite the relative lack of critical upcoming data.
GBT440-001 is a randomized, placebo-controlled, double-blind, single and multiple ascending dose study. This ongoing Phase 1/2 study is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of GBT440 in both healthy subjects and adults with SCD. The study is being conducted in three parts: Part A (single dose administration), Part B (multiple dose administration, daily for 15 days in healthy subjects and 28 days in SCD patients) and Part C (multiple dose administration, daily for 90 days in SCD patients). Some patients in Part C have taken GBT440 for up to 6 months.
Results presented at ASH showed:
– All 41 SCD patients receiving GBT440 for up to six months have demonstrated a profound and durable reduction in hemolysis (red blood cell destruction) as assessed by hemoglobin, reticulocytes, and/or bilirubin.
– All patients taking GBT440 showed profound and durable reductions in irreversibly sickled cells compared with those taking a placebo.
Results from Part C (dosing for at least 90 days) demonstrate that among the 13 GBT440-treated patients:
– Patients treated with GBT440 for at least 90 days showed a clinically significant increase in hemoglobin (greater than 1 g/dL increase) compared with 14 placebo patients (46 percent vs. 0 percent; p=0.006).
Patients treated with GBT440 had a sustained reduction in irreversibly sickled cells compared with placebo-treated patients (-76.6 percent vs. +9.7 percent; p<0.001).- GBT440 was well tolerated up to six months of dosing. The most common treatment-related adverse events were Grade 1/2 headache and gastrointestinal disorders and occurred at similar rates in the placebo and GBT440 arms. There were no drug-related serious or severe adverse events. No sickle cell crises events occurred in study participants while on GBT440. Exercise testing data showed normal tissue oxygen delivery (no change in oxygen consumption compared to placebo).
– Absorption, Metabolism and Excretion of GBT440 a Novel Hemoglobin S (HbS) Polymerization Inhibitor for the Treatment of Sickle Cell Disease (SCD), in Healthy Male Subjects (Abstract #2487)
– Results of a study evaluating the pharmacokinetics, metabolism, and excretion of GBT440 given orally to healthy subjects showed that the drug was completely excreted from the body, with a half-life of approximately three days. This is much shorter than the lifespan of a red blood cell (about 120 days) of a healthy subject, suggesting that the binding of GBT440 to hemoglobin is a reversible process. The data also suggests that the pharmacokinetics of GBT440 are unlikely to be affected in patients with renal disorders.
Global Blood Therapeutics Stock Chart
Global Blood Therapeutics, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering, developing and commercializing novel therapeutics to treat grievous blood-based disorders with significant unmet need.
GBT440 is Global Blood Therapeutics leading drug candidate which targets the underlying mechanism of red blood cell sickling and offers the potential to treat sickle cell disease (SCD) rather than only its symptoms.