Pipeline

GBT440 for Hypoxemia

Our preclinical investigation of GBT440 demonstrated the compound’s ability to increase the oxygen affinity of hemoglobin. This mechanism has important therapeutic potential for the treatment of hypoxemia (low levels of oxygen in the blood) in patients with idiopathic pulmonary fibrosis (IPF), a condition in which the lungs cannot supply adequate oxygen to the blood.

To date, we have established proof of concept for GBT440 analogs in three different animal models of hypoxia. Emerging data suggest that hemoglobin modifiers such as GBT440 have the potential to enable increased oxygen uptake in the lungs, resulting in improved oxygen delivery to tissues.

About 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.

Currently, people with IPF are treated with supplemental oxygen therapy. Supplemental oxygen therapy is a well-established treatment for IPF, but is associated with a significant burden on the patients’ quality of life due to demands of the delivery equipment that ultimately can lead to significantly impaired mobility and psychosocial decline. A drug that has the potential to improve oxygenation could fill a significant unmet medical need.

Clinical Trials

We have two ongoing Phase 2a studies in adults with idiopathic pulmonary fibrosis (IPF) – one in people who become hypoxemic on exertion and one in people who are dependent on oxygen when resting, called ZEPHYR. We also have a Phase 1 clinical trial, called Basecamp, which is evaluating the physiologic effects of GBT440 in fit healthy volunteers under hypoxic conditions and exercise conditions that maximally stress the function of the heart and lungs.

Click here to access information on the Phase 2a study in adults with IPF who have low blood oxygen levels when they exercise  on ClinicalTrials.gov.

Click here to access information on ZEPHYR, the Phase 2a study in adults with IPF who need oxygen while resting on ClinicalTrials.gov.

Click here to access information on Basecamp, the Phase 1 study in healthy volunteers on ClinicalTrials.gov.

Abstracts & Presentations

American Thoracic Society (ATS) International Conference (2016)

Pulmonary Fibrosis Foundation (PFF) Summit (2015)

American Heart Association Scientific Sessions (2015)

Abstract: http://www.abstractsonline.com/pp8/#!/3795/presentation/41971