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Press Release

Global Blood Therapeutics Announces Publication of Preclinical Results Supporting GBT440 Program for Hypoxemic Pulmonary Disorders

Posted on: 14 Sep 16

SOUTH SAN FRANCISCO, Calif., Sept. 14, 2016 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT), a biopharmaceutical company developing novel therapeutics for the treatment of grievous blood-based disorders with significant unmet need, today announced publication of preclinical data supporting the development of GBT440 for the treatment of hypoxemic pulmonary disorders, including idiopathic pulmonary fibrosis (IPF), in Physiological Reports. To date, GBT has established preclinical proof of concept for GBT1118, a hemoglobin modifier and analog of GBT440, in three different animal models of hypoxia. Data from these models support the potential beneficial effects of hemoglobin modification as a promising therapeutic strategy to treat hypoxemia (abnormally low levels of oxygen in the blood) associated with chronic fibrotic lung disorders, such as IPF.

“Shortness of breath and worsening hypoxia are hallmark clinical features of IPF, and currently, there is no commercially available drug to resolve these devastating symptoms. New treatments are needed that can potentially improve patients’ quality of life,” said Ted W. Love, M.D., president and chief executive officer of GBT. “These data support our belief that hemoglobin modifiers can improve hypoxemia and potentially delay the progression of pulmonary fibrosis. Based on this preclinical proof of concept data, we recently initiated a Phase 2a trial of GBT440 in IPF patients and anticipate data from this trial in the second half of 2017.”

The published study is the first to suggest that hemoglobin modifiers, such as GBT440, have the potential to increase oxygen uptake in the lungs, resulting in improved oxygen delivery to tissues. To assess whether pharmacological modification of hemoglobin-oxygen affinity could improve hypoxemia associated with lung fibrosis, researchers evaluated GBT1118 in a bleomycin-induced mouse model of hypoxemia and fibrosis. After pulmonary fibrosis and hypoxemia were induced, GBT1118 was administered for eight days. Hypoxemia was determined by monitoring arterial oxygen saturation, while the severity of pulmonary fibrosis was assessed by histopathologic evaluation and determination of collagen and leukocyte levels in bronchoalveolar lavage fluid (BALF). Treatment with GBT1118:

  • Restored arterial oxygen saturation to near normal levels
  • Reduced collagen levels in BALF and inflammatory cell infiltration, and
  • Reduced lung fibrosis by approximately 50 percent.

About IPF
Idiopathic pulmonary fibrosis (IPF) is a fatal disease characterized by irreversible and progressive scarring of the lungs. As a patient’s lung scarring worsens, the lungs cannot properly facilitate oxygen absorption into the blood and, as a result, the body’s tissues do not get the oxygen they need. The cause of IPF is unknown and there is no cure. IPF inevitably causes progressive shortness of breath and destruction of lung tissue, resulting in worsening hypoxemia, tissue damage, and ultimately organ dysfunction. Progressive worsening of lung function ultimately necessitates the use of supplemental oxygen and frequent hospitalizations in the late stages of the disease.

IPF typically affects individuals over the age of 50, and the median survival after diagnosis is approximately 2 to 3 years.

About GBT440
GBT is developing GBT440 as an oral, once-daily therapy for patients with sickle cell disease (SCD) and for the potential treatment of hypoxemic pulmonary disorders, including IPF. GBT440, a hemoglobin modifier, works by increasing hemoglobin’s affinity for oxygen. Emerging data suggest that hemoglobin modifiers such as GBT440 have the potential to augment hemoglobin function and increase oxygen uptake in the lungs, restoring oxygen delivery to tissues.

About Global Blood Therapeutics
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. GBT is developing its lead product candidate, GBT440, as an oral, once-daily therapy for sickle cell disease (SCD) and is currently evaluating GBT440 in a Phase 1/2 study in both healthy subjects and adults with SCD and a Phase 2a study in adolescents with SCD. GBT is also investigating GBT440 for the treatment of hypoxemic pulmonary disorders in an ongoing Phase 2a study in patients with idiopathic pulmonary fibrosis. Additionally, GBT is also engaged in research and development activities with an oral kallikrein inhibitor for the prevention of hereditary angioedema attacks. To learn more, please visit:

Forward-Looking Statements
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 in IPF, other hypoxemic disorders and SCD, our ability to conduct, and to generate data from, our Phase 2a study of GBT440 in patients with IPF and the timing of these events, 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 GBT440 may not provide the clinical benefits that we anticipate, along with those set forth in our Annual Report on Form 10-K for the fiscal year ended December 31, 2015 and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2016, as well as discussions of potential risks, uncertainties and other important factors in our subsequent filings with the U.S. Securities and Exchange Commission. Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.


Last updated on: 15/09/2016

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