Santhera Pharmaceuticals has announced the publication of data from two Phase 1 studies of its POL6014 inhaled human neutrophil elastase (hNE) inhibitor in the Journal of Cystic Fibrosis. According to the company, inhalation of nebulized POL6014 resulted in a rapid reduction of hNE in the sputum of cystic fibrosis patients, with high lung concentrations of POL6014 and minimal systemic exposure.
The two single ascending dose studies evaluated inhaled POL6014 in 48 healthy volunteers and 24 CF patients. Doses given to healthy volunteers ranged from 20 mg to 960 mg, with doses up to 480 mg being well tolerated. CF patients received doses of 80 mg, 160 mg or 320 mg. Levels of POL6014 in the sputum of CF patients at 3 and 24 hours after inhalation were found to be 1000 times higher than plasma levels.
Santhera Chief Medical Officer and Head of Development Kristina Sjöblom Nygren said, “We are encouraged by these early data in healthy volunteers and patients with cystic fibrosis. POL6014 brings new promise to current CF treatment which, despite recent developments, is still lacking a solution to lung tissue inflammation, a cause of pulmonary exacerbations. In addition, inhalation of POL6014 shows a strong advantage over the oral route as it delivers the drug directly to the lung with a low systemic burden. As an innovative inhibitor of human neutrophil elastase, POL6014 could have potential in a range of other pulmonary diseases.”
POL6014, which Santhera licensed from Polyphor in February 2018, has received orphan drug designation in the EU for the treatment of CF, alpha-1 antitrypsin deficiency (AATD), and primary ciliary dyskinesia (PCD), the company said. A Phase 1b/2a multiple ascending dose study in patients with CF is underway.
Read the Santhera press release.
