Airway Therapeutics announced the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application to develop AT-100 (rhSP-D) as a preventive treatment for the serious respiratory disease BPD in very preterm babies.

Airway will initiate a Phase 1b randomized clinical trial to confirm the feasibility of intra-tracheal administration of AT-100 and its beneficial safety and tolerability profile. The Phase 1b trial will be followed by a randomized pivotal trial with preliminary results expected as soon as second half 2023.

"Preterm infants with BPD have higher mortality, and those who survive suffer from lifelong chronic consequences. With no approved treatments for BPD, AT-100 has potential to reduce incidence and severity of BPD, related short- and long-term consequences, and ultimately improve the survival of these vulnerable patients," said Marc Salzberg, M.D., CEO of Airway. "We are excited to advance this new therapy into the clinic with the potential to deliver novel therapies for patients in desperate need of new treatment options."

AT-100 is a novel human recombinant protein – an engineered version of an endogenous protein – that has been shown in preclinical studies to reduce inflammation and infection triggered in very preterm babies, by mechanical ventilation and oxygen support, and ultimately reduce the onset of lung damage.

Very preterm infants whose lungs are not fully developed must be intubated and ventilated to enable breathing. Although the mechanical ventilation preserves life, it can damage fragile lung tissue, causing inflammation and infection which can result in the arrest of lung development, and ultimately BPD.

Airway has filed an additional IND application with the FDA for AT-100 for treatment of COVID-19. In addition to its anti-inflammatory properties, preclinical studies of AT-100 have shown potential to inhibit SARS-CoV-2 replication and promote viral elimination. AT-100 may also reduce secondary infections in mechanically ventilated COVID-19 patients. This multidimensional approach differentiates AT-100 from other COVID-19 treatments in development.

AT-100 is a novel recombinant human protein rhSP-D, an engineered version of an endogenous protein that reduces inflammation and infection in the body while modulating the immune response to break the cycle of injury and inflammation. Airway is focused on advancing AT-100 for the prevention of BPD in very preterm born babies and as a therapeutic for seriously ill COVID-19 patients. AT-100's anti-inflammatory and anti-infective properties also make it a potential treatment for other respiratory diseases such as influenza, respiratory syncytial virus (RSV) and inflammatory diseases outside the lung. The FDA and European Medicines Agency have granted AT-100 Orphan Drug Designation.