Vertex today announced positive phase III clinical trial results for VX-770 in cystic fibrosis, a drug that I had previously written about.
I don’t see any point in rehashing the press release, but the data is good news for the company and Cystic Fibrosis patients.
One of the exciting things about the biotechnology industry is its ability to innovate and translate developments in basic science into potential new drugs.
I previously wrote about denufosol in cystic fibrosis (CF), a disease that affects about 30,000 people in the United States and 70,000 worldwide. The disease is characterized by the accumulation of mucus that leads to bacterial overgrowth and chronic lung infections. Mucus cannot be removed from the lung in CF due to abnormal mucociliary transport resulting from impaired epithelial chloride secretion and sodium hyperabsorption. This is now known to be due to defective cystic fibrosis transmembrane regulator (CFTR) protein. A good overview of this can be found in the 2006 New England Journal of Medicine Editorial by Felix Ratjen, “Restoring Airway Surface Liquid in Cystic Fibrosis.”
Thanks to Adam Feuerstein of TheStreet for breaking the news this morning, that shares in Inspire Pharmaceuticals (NASDAQ: ISPH) have plunged following the announcement of negative data from the phase 3, TIGER-2 clinical trial for denufosol in cystic fibrosis.
According to the Chief Medical Officer at Inspire Pharma: ”The analysis of the primary endpoint, key secondary endpoints and select subgroup populations in TIGER-2 indicates an absence of meaningful treatment benefit in this patient population.”
I wrote a blog post last week about denufosol and the hope that this drug offered to cystic fibrosis patients despite the uncertainty about its clinical effectiveness.
Inspire Pharmaceuticals (NASDAQ:ISPH), a North Carolina based biopharmaceutical company that focuses on products for ophthalmic and pulmonary diseases, recently announced positive results from their phase 3 trial (TIGER-1) of denufosol tetrasodium in patients with Cystic Fibrosis (CF).
Cystic Fibrosis is a genetic disorder that can lead to death as a result of pulmonary complications from airway obstruction, bronchial thickening and accumulation of mucous. Lung function tests are widely used in the diagnosis, treatment and management of patients with CF. Measurement of FEV1 (Forced Expiratory Volume in 1 second) is regarded as the best predicator of mortality. As the disease progresses and the lungs become more obstructed, FEV1 decreases.