SOUTH SAN FRANCISCO, CA, USA I June 20, 2016 I Global Blood Therapeutics, Inc. (GBT) (GBT), a biopharmaceutical company developing novel therapeutics for the treatment of grievous blood-based disorders with significant unmet needs, today announced it has initiated a Phase 2a study of GBT440, a hemoglobin modifier, in adults with idiopathic pulmonary fibrosis (IPF) and hypoxemia.
Hypoxemia (abnormally low levels of oxygen in the blood) can lead to tissue hypoxia (a deficiency in the amount of oxygen reaching the tissues), which is believed to play a key role in adverse patient outcomes in IPF. GBT440 has been shown in preclinical studies to increase oxygen saturation in hypoxic conditions. Because of this ability, GBT is developing GBT440 as a potential treatment for hypoxemia, associated with acute and chronic lung disorders, such as IPF, in which the lungs cannot supply adequate oxygen to the blood.
“Current treatment for IPF is targeted at slowing lung fibrosis but there has been no improvement in oxygenation and symptom burden of the disease. Supplemental oxygen therapy, which is used to treat the hypoxemia and its associated breathlessness, has local and systemic risks and poses a significant burden to patients. For patients with this fatal disease, there is a critical need for a therapeutic drug that can improve oxygenation without those risks and improve quality of life,” said Ted W. Love, M.D., chief executive officer of GBT. “Proof of concept has been established in three animal model studies suggesting that hemoglobin modification may increase oxygen saturation and improve oxygen delivery to tissues. Based on those data, we believe that GBT440 is a promising therapeutic option for IPF, and we are advancing the clinical development of this oral, once-daily compound with the initiation of this Phase 2a study.”
About the Phase 2a GBT440-006 Study
GBT440-006 is a randomized, double-blind, placebo-controlled study of GBT400 that is evaluating safety, tolerability, pharmacokinetics and effect on hypoxemia in patients with IPF. A total of 18 IPF patients will be enrolled and randomized to one of two doses of GBT440 or placebo administered orally once daily for 28 days. An additional 12 patients may be enrolled following an independent safety review for a total of up to 30 patients.
In addition to safety, tolerability and pharmacokinetics of GBT440, the change from baseline in oxygen saturation both at rest and after exercise will be explored.
About IPF
Idiopathic pulmonary fibrosis (IPF) is a fatal disease characterized by irreversible, progressive scarring of the lungs. As a patient’s lung scarring worsens, the lungs cannot properly move oxygen into the bloodstream 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 shortness of breath and destruction of healthy lung tissue, resulting in hypoxemia, tissue hypoxia, and ultimately organ dysfunction. Patients with IPF typically experience progressive worsening of lung function over time, requiring 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 restore normal hemoglobin function and increase oxygen uptake in the lungs, resulting in improved oxygen delivery to tissues.
About Global Blood Therapeutics
Global Blood Therapeutics, Inc. (GBT) 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 both healthy subjects and SCD patients in a randomized, placebo-controlled, double-blind Phase 1/2 clinical trial. In addition to GBT440 for the treatment of SCD, GBT is engaged in research and development activities targeted toward hypoxemic pulmonary disorders, including idiopathic pulmonary fibrosis (IPF), and hereditary angioedema (HAE). To learn more, please visit: www.globalbloodtx.com.
SOURCE: Global Blood Therapeutics
Post Views: 95
SOUTH SAN FRANCISCO, CA, USA I June 20, 2016 I Global Blood Therapeutics, Inc. (GBT) (GBT), a biopharmaceutical company developing novel therapeutics for the treatment of grievous blood-based disorders with significant unmet needs, today announced it has initiated a Phase 2a study of GBT440, a hemoglobin modifier, in adults with idiopathic pulmonary fibrosis (IPF) and hypoxemia.
Hypoxemia (abnormally low levels of oxygen in the blood) can lead to tissue hypoxia (a deficiency in the amount of oxygen reaching the tissues), which is believed to play a key role in adverse patient outcomes in IPF. GBT440 has been shown in preclinical studies to increase oxygen saturation in hypoxic conditions. Because of this ability, GBT is developing GBT440 as a potential treatment for hypoxemia, associated with acute and chronic lung disorders, such as IPF, in which the lungs cannot supply adequate oxygen to the blood.
“Current treatment for IPF is targeted at slowing lung fibrosis but there has been no improvement in oxygenation and symptom burden of the disease. Supplemental oxygen therapy, which is used to treat the hypoxemia and its associated breathlessness, has local and systemic risks and poses a significant burden to patients. For patients with this fatal disease, there is a critical need for a therapeutic drug that can improve oxygenation without those risks and improve quality of life,” said Ted W. Love, M.D., chief executive officer of GBT. “Proof of concept has been established in three animal model studies suggesting that hemoglobin modification may increase oxygen saturation and improve oxygen delivery to tissues. Based on those data, we believe that GBT440 is a promising therapeutic option for IPF, and we are advancing the clinical development of this oral, once-daily compound with the initiation of this Phase 2a study.”
About the Phase 2a GBT440-006 Study
GBT440-006 is a randomized, double-blind, placebo-controlled study of GBT400 that is evaluating safety, tolerability, pharmacokinetics and effect on hypoxemia in patients with IPF. A total of 18 IPF patients will be enrolled and randomized to one of two doses of GBT440 or placebo administered orally once daily for 28 days. An additional 12 patients may be enrolled following an independent safety review for a total of up to 30 patients.
In addition to safety, tolerability and pharmacokinetics of GBT440, the change from baseline in oxygen saturation both at rest and after exercise will be explored.
About IPF
Idiopathic pulmonary fibrosis (IPF) is a fatal disease characterized by irreversible, progressive scarring of the lungs. As a patient’s lung scarring worsens, the lungs cannot properly move oxygen into the bloodstream 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 shortness of breath and destruction of healthy lung tissue, resulting in hypoxemia, tissue hypoxia, and ultimately organ dysfunction. Patients with IPF typically experience progressive worsening of lung function over time, requiring 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 restore normal hemoglobin function and increase oxygen uptake in the lungs, resulting in improved oxygen delivery to tissues.
About Global Blood Therapeutics
Global Blood Therapeutics, Inc. (GBT) 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 both healthy subjects and SCD patients in a randomized, placebo-controlled, double-blind Phase 1/2 clinical trial. In addition to GBT440 for the treatment of SCD, GBT is engaged in research and development activities targeted toward hypoxemic pulmonary disorders, including idiopathic pulmonary fibrosis (IPF), and hereditary angioedema (HAE). To learn more, please visit: www.globalbloodtx.com.
SOURCE: Global Blood Therapeutics
Post Views: 95
