A biopharmaceutical company with a cystic fibrosis drug in late-stage development has raised $30 million, according to a company statement. It was about $10 million less than the original funding target listed in a form D document with the U.S. Securities and Exchange Commission last month.
The target of PTC Therapeutics‘ small molecule drug Ataluren is nonsense mutation cystic fibrosis. Its name is derived 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. Although other companies are developing drugs to treat cystic fibrosis, none are looking at the subgroup that the South Plainfield, New Jersey biopharmaceutical company is, said PTC Therapeutics President Cl udia Hirawat in an e-mail.
Hirawat also clarified that the form D figure reflects the maximum limit of what could be raised in this round of financing, but it had only sought $30 million.
With cystic fibrosis the goal is to improve lung function and reduce pulmonary exacerbation. In a Phase 3 clinical study, Ataluren demonstrated trends in improvement in lung function when compared to a placebo. There were also trends in improvements in a secondary endpoint, pulmonary exacerbations, when compared to the placebo, Hirawat said in the e-mail..
Cystic fibrosis causes the body to produce thick, sticky secretions in the lungs that makes it difficult for people with the disease to breathe and produces frequent lung infections. Ataluren is also in phase 2b development for nonsense mutation Duchenne/Becker muscular dystrophy.
The 14-year-old company has raised at least $591 million to date: $213 million from venture capital, $260 million through collaborations with other pharmaceutical companies and $118 million through grants.