Global Blood Therapeutics (GBT) Announces Publication of Positive GBT440 Preclinical Data in HP Disorders

September 14, 2016 4:10 PM EDT

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Global Blood Therapeutics, Inc. (GBT) (Nasdaq: GBT) 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.

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