APEPTICO GmbH, a biotechnology company based in Austria developing peptide drugs based on its PEPBASE discovery technology, has announced phase I clinical trial for its pulmonary sodium ion channel activator AP301. The orally inhaled drug candidate was safe and well-tolerated by all study participants. AP301 is being developed for the prevention and treatment of oedematous respiratory failure in patients suffering from lung infection, lung injury and lung transplantation.

The phase I single-centre clinical trial evaluated the safety, tolerability and pharmacokinetic profile of AP301 in an orally inhaled, double-blind, randomized, placebo-controlled, dose escalation study in 48 healthy male volunteers. AP301 was shown to be safe at all doses investigated, with no reports of serious adverse side effects.

AP301 is the first compound against respiratory failure caused by pulmonary oedema that activates lung oedema reabsorption and thus differs from the currently used anti-inflammatory treatment that often fails in patients with acute lung injury. The synthetic peptide AP301 activates alveolar liquid clearance (ALC) and prevents both endothelial and epithelial lung tissue from hyper-permeability as a result of microbial and viral lung infections. AP301 also prevents ischaemia reperfusion injury following lung transplantation in the lower respiratory tract.

“The successful completion of our phase I trial is an important step for APEPTICO’s drug development programme” said Dr Bernhard Fischer, CEO of APEPTICO. “We look forward to starting the phase II a trial with the aerosol formulation of AP301 in 2012, and are committed to building on our success with peptide drugs,” he added.

AP301 is a fully synthetic peptide molecule whose structure is based on the lectin-like domain of human Tumour Necrosis Factor alpha. The AP301 peptide is water soluble and can be administered into the lung by oral inhalation. AP301 is designed to activate the pulmonary epithelial sodium channel (ENaC) which results in an accelerated oedema clearance in the airspace. Comprehensive research and development conducted by APEPTICO has demonstrated that the AP301 peptide is effective to clear lung water in animal models of pulmonary permeability oedema, pneumonia, influenza virus lung infection, acute lung injury and lung transplantation. AP301 has received Orphan Drug Designation by the EMA and the US FDA for various indications.