Health Care Professionals


Santhera is passionate about developing new treatments for patients with mitochondrial diseases. Our focus is on the development of treatments for neuromuscular and neuro-ophthalmological diseases that currently lack treatment options.

Our pipeline: POL6014

POL6014, a clinical stage selective inhibitor of human neutrophil elastase, has the potential to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases such as non-cystic fibrosis bronchiectasis (NCFB), alpha-1 antitrypsin deficiency (AATD) and primary ciliary dyskinesia (PCD).

POL6014 is a highly potent and selective inhibitor of human neutrophil elastase (hNE) and was shown to reach high concentrations in the lung when administered by inhalation via an optimized eFlow® nebulizer (PARI Pharma GmbH). A first-in-man Phase 1 study in healthy volunteers and a Single Ascending Dose (SAD) safety and tolerability Phase 1 study in CF patients have successfully been completed with POL6014. The drug candidate was well tolerated and safe with a favorable pharmacokinetic profile and strong elastase inhibition as previously shown in animal models.

Santhera has in-licensed the compound from Polyphor Ltd. and will assume the global development, regulatory filings and commercialization of POL6014.

For more information please see press release of February 15, 2018.