Global Blood Therapeutics (GBT), a biopharmaceutical company developing novel, small molecule therapeutics for the treatment of severe blood disorders, today announced the closing of a $48 million Series B financing. Proceeds from the financing will be used to advance the company's lead drug candidate, GBT440, for the treatment of sickle cell disease (SCD). GBT440 has shown promising results in preclinical testing and the company is initiating a Phase I/II clinical trial.
'We believe that our ongoing GBT440 development program has the potential to demonstrate early proof of concept as soon as the second half of 2015,' stated Ted W. Love, M.D., chief executive officer of GBT. 'This potential rapid pathway to early POC was instrumental in helping us secure participation from such a high quality investor syndicate.'
The Series B financing included investments from Wellington Management Company LLP, RA Capital, Deerfield Management, Sabby Capital, Perceptive Life Sciences, an affiliate of Cowen Group and one undisclosed blue chip public investment fund.
GBT440 is an oral, once daily dosing, direct-acting sickle hemoglobin (HbS) modifier for the chronic, prophylactic treatment of patients with sickle cell disease (SCD). The drug is a potential best-in-class disease modifier that works by increasing hemoglobin's affinity for oxygen. Since oxygenated hemoglobin does not polymerize, GBT440 blocks polymerization of HbS and the resultant sickling of red blood cells (RBCs). With the promise of restoring normal hemoglobin dynamics, GBT440 may be capable of preventing and halting the progression of SCD.
'GBT has all of the key elements required to be a leader in the development of novel drugs including an experienced management team with a strong track record of clinical and commercial success, innovative and exceptional science addressing a significant patient need, and promising data to support their therapeutic approach,' stated Deerfield's William Slattery. 'We believe GBT is uniquely positioned for success and we are excited to be an early investor in the company.'
This financing follows the company's presentation of new positive data for GBT440 at the 2014 American Society of Hematology (ASH) Conference last month in San Francisco. Findings reported in both oral and poster presentations highlighted the drug's ability to prevent sickling of RBCs, inhibit the polymerization of deoxygenated HbS, improve RBC deformability and reduce whole blood viscosity in preclinical SCD models. In addition, in a sickle cell disease mouse model, repeat oral dosing showed prolonged red blood cell half-life and decreased reticulocyte counts. These results demonstrate GBT440's potential to disrupt fundamental SCD pathophysiology and, in turn, halt the progression of the disease.
About Sickle Cell Disease (SCD)
Sickle cell disease (SCD) is an inherited disorder caused by a genetic mutation leading to formation of hemoglobin S (HbS). A primary and obligatory event in the molecular pathogenesis of SCD is the polymerization of deoxygenated HbS. This polymerization results in the red blood cell (RBC) sickling that causes the normally flexible RBCs to become rigid with a significantly shorter lifespan.
Sickled RBCs cannot adapt their shape to allow uninterrupted flow through capillaries (smaller blood vessels). Sickled cells, unlike flexible healthy RBCs, stack up against each other, blocking normal blood flow. As a consequence of the resulting vascular occlusion, patients with SCD suffer acute and chronic complications including unpredictable and recurrent episodes of severe pain, progressive organ damage, stroke and a shortened life expectancy.
About Global Blood Therapeutics
Global Blood Therapeutics (GBT) is a biopharmaceutical company developing novel, small molecule therapeutics for the treatment of severe blood disorders. The company is addressing serious, non-malignant blood-based conditions for which there are currently no effective cures and only limited therapeutic options. Lead drug candidate, GBT440, is a potentially disease-modifying therapeutic for patients with sickle cell disease. GBT440 has shown promising results in preclinical testing and the company is initiating a Phase I/II clinical trial. In addition to GBT440, the company is advancing pipeline research programs addressing hereditary angioedema (HAE) and hypoxic cardiopulmonary disorders.
To learn more, please visit: www.globalbloodtx.com.