Surfaxin^® (lucinactant), our lead product, is the first precision-engineered, protein B-based agent that mimics the surface-active properties of human surfactant. Surfaxin has shown to remove inflammatory and infectious infiltrates from patients' lungs when used by lavage, or "lung wash," and replenish the vital surfactant levels in the lungs. Surfaxin is delivered in a bolus form through an endotracheal tube in premature infants, and as a proprietary lavage through a tube, called a bronchoscope, in full-term infants and adults.

Surfaxin, if approved, would be the world's next generation surfactant—significantly improving and expanding the current surfactant therapy for RDS and treating other neonatal intensive care unit (NICU) respiratory conditions for which the only treatment today is mechanical ventilation. The potential attributes of Surfaxin create an opportunity for improving critical care respiratory medicine—a market that could exceed $1.5 billion.

Respiratory Distress Syndrome (RDS) in Premature Infants

We have conducted a pivotal Phase 3 trial which enrolled 1,294 patients and was designed as a multinational, multicenter, randomized, masked, controlled, prophylaxis, event-driven, superiority trial to demonstrate the safety and efficacy of Surfaxin over Exosurf®, an approved, non-protein containing synthetic surfactant. Survanta®, a cow-derived surfactant and the leading surfactant used in the United States, served as a reference arm in the trial. Key trial results were assessed by an independent adjudication committee comprised of leading neonatologists and pediatric radiologists. This committee provided a consistent and standardized method for assessing critical efficacy data in the trial. An independent Data Safety Monitoring Board (DSMB) was responsible for monitoring the overall safety of the trial and no major safety issues were identified. 

We have also conducted a supportive, multinational Phase 3 clinical trial which enrolled 252 patients and was designed as a non-inferiority trial comparing Surfaxin to Curosurf®, a porcine (pig) derived surfactant and the leading surfactant used in Europe. This trial demonstrated the overall safety and non-inferiority of Surfaxin to Curosurf.

The results from both the landmark, pivotal Phase 3 clinical trial and supportive Phase 3 clinical trial of Surfaxin for the treatment of Respiratory Distress Syndrome in premature infants, formed the basis for the New Drug Application (NDA) filed with the United States FDA.  The FDA has provided Discovery with an Approvable Letter for Surfaxin.

Bronchopulmonary Dysplasia (BPD, also known as Chronic Lung Disease) in Premature Infants

We recently concluded a Phase 2 clinical trial of Surfaxin for the prevention and treatment of BPD in premature infants. This double-blind, controlled Phase 2 clinical trial was intended to enroll up to 210 very low birth weight premature infants born at risk for developing BPD. The study’s objective was to determine the safety and tolerability of administering Surfaxin as a therapeutic approach for the prevention and treatment of BPD. We plan to perform a comprehensive analysis of the clinical data from this trial, report results and submit these data for publication.

Bronchopulmonary Dysplasia (BPD), also known as Chronic Lung Disease, is a costly syndrome affecting premature infants. It is associated with surfactant deficiency and the prolonged use of mechanical ventilation or oxygen supplementation. Some premature babies are born with a lack of natural surfactant in their lungs. Without surfactant, the air sacs in the lungs collapse and are unable to absorb sufficient oxygen resulting in RDS. To treat RDS, babies require a surfactant usually within one hour of birth as well as mechanical ventilation to support the babies’ respiration. The lack of surfactant and use of mechanical ventilation may cause chronic injury and scarring of the lungs – resulting in BPD. Presently there are no approved drugs for the treatment of BPD. These babies suffer from abnormal lung development and typically have a need for respiratory assistance - oftentimes, for many months, as well as comprehensive care spanning years. It is estimated that the cost of treating an infant with BPD in the United States can approach $250,000 with approximately 50,000 infants developing BPD in the United States and Europe each year.

The FDA has granted Surfaxin Orphan Drug Designation and Fast Track Designation for this indication.

Surfactant Lavage for Acute Lung Injury and Acute Respiratory Distress Syndrome (ALI/ARDS) in Adults

We performed a Phase 2 clinical trial as an open-label, controlled, multi-center, international study utilizing Surfactant Lavage to treat ARDS in adults. Patients were randomized to receive either surfactant administered in high concentrations and large volumes via a bronchoscopic segmental lavage technique (lung wash), or the current standard of care (SOC), which is mechanical ventilation and other supportive therapies. Surfactant was delivered with a bronchoscope to each of the 19 segments of the lung and was intended to cleanse and remove inflammatory substances and debris from the lung, while leaving sufficient amounts of surfactant behind to help re-establish the lung’s capacity to absorb oxygen.

The results from the Phase 2 study indicated that the Surfactant Lavage exhibited a positive pharmacologic effect manifested as improved oxygenation. This was demonstrated by an acute increase in the P/F ratio after patients received Surfactant Lavage. There were no differences between the treatment groups and Standard of Care in all-cause mortality and the incidence rate of being alive and off mechanical ventilation at Day 28.

We, alone or with potential partners, will apply scientific and clinical observations generated from this trial towards the design of future trials to address this serious respiratory syndrome. These trials may determine whether earlier intervention and or administration of surfactant throughout the patient’s clinical course may further improve clinical outcomes.

The FDA has granted Fast Track Approval Status. Both the FDA and Europe's regulatory equivalent, the EMEA, have granted Orphan Drug Designation for this indication.

Selected Bibliography on Surfactant and ALI/ARDS