By Brady Huggett
Discovery Laboratories Inc. said it will begin a pivotal Phase III trial evaluating Surfaxin for the treatment of idiopathic respiratory distress syndrome in premature infants.
The trial will enroll about 1,500 patients, most likely from North America, South America and Europe, and is designed to support new drug applications to the FDA and regulatory authorities worldwide.
"The trials should take about a year," said Robert Capetola, CEO and president of Discovery Laboratories. "We should be able to file pretty soon after that."
Discovery's Surfaxin will be evaluated against available surfactant therapy in an active comparator trial, with the endpoint designed to show Surfaxin's superiority.
Capetola said there are 2 million babies born worldwide each year with idiopathic respiratory distress syndrome. These premature infants' genes that code for surfactant proteins are not turned on yet, and they are candidates for Surfaxin therapy. Enrolling 1,500 patients shouldn't be hard, Capetola said.
"We have constant surveillance and we are in touch with virtually every hospital in the United States," he said. "We visit all these hospitals and we look at birth rates and look at prematurity and we do an audit of their paper trail."
Surfaxin is a humanized, peptide-containing surfactant designed to lower surface tension at the air-water interface in the lungs. Compared to animal-derived surfactants, Surfaxin may be longer lasting. Discovery acquired exclusive worldwide rights to Surfaxin from Johnson & Johnson, of New Brunswick, N.J., and The Scripps Research Institute, of La Jolla, Calif.
Animal-derived surfactants are approved around the world for idiopathic respiratory distress syndrome, Capetola said, adding that all drugs for the syndrome are given the same way - a bolus down the trachea. For the Phase III trial, the bolus will be given half an hour after birth. Capetola would not disclose specific endpoints or how they would be measured, but said "modern day techniques" will be used to assess the patient.
Discovery has two other trials of Surfaxin under way in the United States: a pivotal Phase III trial in meconium aspiration syndrome in full-term newborns and a Phase II trial in acute respiratory distress syndrome in adults. Surfaxin also is under preclinical development for the treatment of asthma, chronic obstructive pulmonary disease, acute and chronic bronchitis and several other pulmonary diseases.
In January 2000, Discovery halted a Phase II/III Surfaxin trial for acute respiratory distress syndrome because of manufacturing issues. (See BioWorld Today, Jan. 28, 2000.)
Discovery's stock (NASDAQ:DSCO) dipped 9 cents Wednesday to close at $3.125. n