Vertex Pharmaceuticals jolted Wall Street with a fresh batch of midstage data on one of its combo therapies against cystic fibrosis.
The FDA approved Novartis' ($NVS) cystic fibrosis treatment that fights bacterial infection via a powder solution inhaled from a handheld device, the first of its kind to do so.
Novartis notched an FDA approval on Friday for a dry-powder formulation of an antibiotic that fights a type of bacterial infection in patients with cystic fibrosis (CF).
Austin-based Savara Pharmaceuticals has rounded up $11.4 million in cash to fuel the upstart biotech through a key transition phase: taking its lead development program through a crucial proof-of-concept study that could leave the biotech on the threshold of a late-stage trial.
Pharmaxis has been dealt another blow by the FDA. The Australian company confirmed Tuesday morning that the regulatory agency has rejected its bid to market the cystic fibrosis drug Bronchitol for treatment in U.S. patients.
Cambridge, MA-based Vertex Pharmaceuticals says it nailed solid mid-stage data for VX-787, an experimental influenza drug in a new class of medications that promises to prevent the virus from replicating. And the biotech added that it will be in hot pursuit of a new collaboration on the drug.
Vertex Pharmaceuticals has set the stage for an ambitious Phase III program for its combo approach matching the experimental VX-809 and Kalydeco for cystic fibrosis. And if it can stick with the plan, Vertex will cut a short path directly to an approval for the groundbreaking combo.
Kala Pharmaceuticals received a grant from the Cystic Fibrosis Foundation to demonstrate that the company's mucosal-barrier-penetrating nanoparticle can do the same across bacterial biofilms, a symptom of cystic fibrosis.
At the same time Australian Pharmaxis inks a financing deal with NovaQuest Pharma Opportunities Fund III for up to $40 million, the company has gotten disappointing news from the FDA--its cystic fibrosis inhalant therapy received a negative recommendation from an FDA advisory committee for use in U.S. patients.
Australia's Pharmaxis is scheduled to make its case for a new cystic fibrosis therapy in front of an FDA advisory committee. But based on the agency staff review out earlier this week, investigators will likely get grilled about a high dropout rate in two key studies and the mixed efficacy data they delivered.