A drug developer that recently reported positive phase 3 results for its cystic fibrosis treatment for a severe form of the genetic disorder is raising $40 million in equity, according to a Form D filing with the U.S. Securities and Exchange Commission.
So far it has raised more than half that amount, according to the filing dated May 29.
The target of Ataluren is nonsense mutation cystic fibrosis. Its name derives from an alteration in the genetic code that prematurely halts the development of an essential protein, in this case cystic fibrosis transmembrane conductance regulator protein or CFTR.
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South Plainfield, New Jersey-based PTC Therapeutics‘ drug Ataluren was given orphan drug status by the U.S. Food and Drug Administration and the European Commission to treat nonsense mutation cystic fibrosis and nonsense mutation Duchenne and Becker muscular dystrophy. The FDA has also granted Ataluren Subpart E designation, a status that expedites the development, evaluation and marketing for cystic fibrosis and dystrophinopathy, and fast track designation for the development of treatment for nonsense mutation dystrophinopathy, according to a statement on the company’s website.
Cystic fibrosis causes the body to produce thick, sticky secretions in the lungs, which makes it difficult for people with the disease to breathe and produces frequent lung affections.
The 48-week trial enrolled 238 patients, 6 years old and up at several different sites in North America, Europe and Israel. Patients were randomly given Ataluren or placebo, according to a press statement. Patients who completed the study were eligible to receive open-label Ataluren in an ongoing extension study. Topline data from the 48-week trial released last week was “promising” according to Dr. Michael Konstan, a principal investigator at University Hospitals Rainbow Babies & Children’s Hospital in Cleveland.
The FDA-approved a drug earlier this year that targets a specific mutation found in up to 5 percent of cystic fibrosis cases. Kalydeco was developed by Vertex Pharmaceuticals (NASDAQ:VRTX) and the Cystic Fibrosis Foundation to treat an amino acid mutation G551D.
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