Up to half of all cystic fibrosis patients have fungal infections in their lungs caused by a spore-forming mold named Aspergillus fumigatus. Many of those patients, particularly children and young adults, will develop an allergic reaction to the fungus, Bronchopulmonary Aspergillosis (ABPA), according to the Cystic Fibrosis Foundation. Coughing, wheezing and other symptoms can be severe.
Current treatments are oral drugs, including itraconazole. The problem is that high doses are needed to get enough of the drug to the lungs. That causes severe side effects that must be managed and monitored — including liver toxicity.
A more promising strategy is to deliver the itraconazole directly to the lungs by breathing it in. That’s the approach taken by Lexington, Mass-based pharmaceutical company Pulmatrix, Inc. (NASDAQ: PULM). Pulmatrix’s innovation was creating small, dense particles that can be easily inhaled, and combining them with drugs such as itraconazole.
“We’ve urgently needed a way to deliver antifungal drugs directly to the lungs,” says Stanford University cystic fibrosis expert Richard Moss M.D. “Pulmatrix’s PUR1900, using the inhaled dry particle technology, meets that need. It could offer major benefits to cystic fibrosis patients with fungal infections in their lungs.”
That’s why the U.S. Food & Drug Administration has granted the company’s request for orphan drug status for PUR1900, explaining that the designation “is based on a plausible hypothesis that [the] drug may be clinically superior to the same drug that is already approved.”
While PUR1900 has the potential to improve the lives of cystic fibrosis patients, especially children and young adults, it also could bring significant revenues for Pulmatrix. The estimated cystic fibrosis market alone is tens of thousands of patients a year, and the drug could also find much larger markets treating millions of other pulmonary fungal infections and other immunocompromised patients, making Pulmatrix a strong investment opportunity.