By Lisa Seachrist
WASHINGTON — Discovery Laboratories Inc. has initiated a pivotal Phase II/III trial for its lead product, Surfaxin, a synthetic lung surfactant, as a treatment for acute respiratory distress syndrome (ARDS).
In addition, the FDA told the Doylestown, Pa.-based company earlier this year the drug would qualify for priority review when the company files for approval because it treats an unmet medical need. The only approved surfactant is harvested from cow lungs and used to treat premature infants.
"We feel that it is time that some of these animal harvested [surfactants] be replaced with modern drugs," said Robert Capetola, president and CEO of Discovery Laboratories. "We are very, very happy with the progress of Surfaxin so far."
Surfaxin is a synthetic lung surfactant containing both peptides and lipids which was invented at The Scripps Research Institute in La Jolla, Calif.
The product contains the peptide sinapultide (KL4) which is modeled after the human surfactant protein B (SP-B). SP-B is thought to aid in the spreading and stability of lung surfactant over the interior of the lung. Surfactant functions by regulating the surface tension of the air-liquid emulsion found in the lungs and preventing the alveoli — air sacs — from collapsing.
The 405-patient pivotal trial of Surfaxin will test the drug's ability to treat ARDS from direct causes — severe difficulty in breathing requiring mechanical ventilation as a result of damage to and inflammation of the lung's epithelia caused by diseases and injuries such as pneumonia, aspiration of gastric contents, inhalation of noxious substances, contusions and near drowning. Currently, there is no approved drug to treat ARDS.
The study will be a randomized, multicenter trial involving approximately 40 centers. Patients who develop ARDS from direct causes will be treated with either Surfaxin or the current standard of care, which is mechanical ventilation and blood pressure support.
Treated patients will receive Surfaxin via a bronchoscopic lavage — a lung wash with Surfaxin — in an attempt to wash away the noxious products of inflammation and restore the compromised surfactant.
The company expects the trial to be concluded in 18 to 24 months. The primary endpoint of the study is survival and withdrawal from mechanical ventilation 28 days after the beginning of treatment. Secondary endpoints will include mortality and number of days free from mechanical ventilation at 28 days.
In addition to ARDS, the company is developing Surfaxin for the treatment of meconium aspiration syndrome (MAS) seen in newborns and idiopathic respiratory distress syndrome seen in premature infants. The company currently has Surfaxin in a Phase II trial in MAS, which is expected to complete enrollment this fall.
"We have decided to focus on ARDS and MAS because it is a wide open market right now," Capetola said.
Discovery Laboratories' stock (NASDAQ:DSCO) closed Tuesday at $3.25 up $0.25. *