Under terms of the global licensing agreement between the two Swiss companies, Santhera will develop, handle regulatory filings and market the investigative drug and derivate compounds.
Supported by positive preclinical and clinical data, Santhera plans to launch a multiple-ascending dose trial in European CF patients during the second half of 2018.
“With the licensing of POL6014, Santhera is broadening its clinical stage pipeline in rare diseases with the option to address multiple pulmonary indications,” Santhera CEO Thomas Meier said in a press release, adding that the company will initiate discussions with U.S. and European regulators on eventual approval of the therapy.
POL6014 is a highly potent, selective inhibitor of human neutrophil elastase (hNE), which is secreted by neutrophils during chronic inflammation. Excessive hNE degrades elastase and collagen tissue structural elements, contributing to lung tissue damage, increased mucus hyper-secretion and inflammation.
The new hNE inhibitor is directly administered to the lungs by inhalation via PARI Pharma’s optimized eFlow nebulizer.
Results of a Phase 1 clinical trial in healthy volunteers and CF patients show that POL6014 is safe and well tolerated, with no serious adverse events. Early data indicate that the drug may effectively prevent elastase activity in the sputum of CF patients.
In addition, results indicate that POL6014 holds therapeutic potential for other neutrophilic diseases such as non-cystic fibrosis bronchiectasis, alpha-1 antitrypsin deficiency and primary ciliary dyskinesia.
“We are very excited to be licensing this program to Santhera, a specialty pharmaceutical company highly committed to the development and commercialization of orphan drugs with a strong and experienced team in the field of rare diseases and in respiratory trials,” Polyphor CEO Giacomo Di Nepi said in a press release. “There is a high unmet medical need for the treatment of chronic neutrophilic lung inflammations.”
The U.S.-based Cystic Fibrosis Foundation funded initial clinical development of POL6014.