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Global Blood Therapeutics Announces Publication of Preclinical GBT440 Results in British Journal of Haematology that Support Sickle Cell Disease (SCD) Program
"One promising strategy for preventing red blood cell sickling and subsequently modifying sickle cell disease over the long term involves inhibiting polymerization of HbS in red blood cells. This can be achieved by increasing the proportion of oxygenated HbS in those cells," said
Results of the preclinical studies showed that in vitro, GBT440 dose-dependently increased the affinity of HbS for oxygen, delayed polymerization of HbS and reduced the number of sickled red blood cells (RBCs) in whole blood from SCD patients. Additionally, in an animal model of SCD, GBT440 inhibited
"Our preclinical work has developed a foundation of evidence that GBT440 is a potent inhibitor of the polymerization of HbS. We continue to build on these data with our ongoing Phase 1/2 study, which has shown that GBT440 was well tolerated over 90 days of dosing, and that all SCD patients who received multiple doses of GBT440 exhibited improvements in one or more clinical markers of hemolysis and anemia," said
About Sickle Cell Disease (SCD)
SCD is a chronic, inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, which results in the formation of abnormal hemoglobin known as HbS. In its deoxygenated state, HbS has a propensity to polymerize, or bind together into long, rigid rods within a
GBT is developing GBT440 as an oral, once-daily therapy for patients with SCD. GBT440 works by increasing hemoglobin's affinity for oxygen. Since oxygenated HbS does not polymerize, GBT believes that GBT440 blocks polymerization and the resultant sickling of RBCs. With the potential to restore normal hemoglobin function and improve oxygen delivery, GBT440 may be capable of modifying the progression of SCD.
GBT is also developing GBT440 for the potential treatment of hypoxemic pulmonary disorders, including idiopathic pulmonary fibrosis (IPF). Emerging data suggest that hemoglobin modifiers such as GBT440 have the potential to restore normal hemoglobin function and increase oxygen uptake in the lungs, resulting in improved oxygen delivery to tissues.
Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended and Section 21E of the Securities Exchange Act of 1934, as amended. We intend these forward-looking statements, including statements regarding the therapeutic potential of GBT440 and its potential to change the clinical course of SCD, the potential for once-daily oral dosing of GBT440, our plans to initiate a pivotal trial in adults with SCD and the timing thereof, to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. We can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved, and furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, the risks that our clinical and preclinical development activities may be delayed or terminated for a variety of reasons, that regulatory authorities may disagree with our clinical development plans or require additional studies or data to support further clinical investigation of our product candidate, and that drug-related adverse events may be observed in later stages of clinical development, along with those set forth in our Annual Report on Form 10-K for the fiscal year ended
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Myesha Edwards (investors), Global Blood Therapeutics, 650-351-4730, firstname.lastname@example.org; Julie Normart (media), BrewLife, 415-946-1087, email@example.com