OUR MEDICINES

Santhera is passionate about medical science and about developing new medical treatments for patients with rare diseases. Our focus is on the development of treatments for neuromuscular and pulmonary diseases that currently lack treatment options.

Our pipeline: lonodelestat

Lonodelestat (previously known as POL6014), a highly potent and selective peptide inhibitor of human neutrophil elastase (hNE), is in development for the treatment of cystic fibrosis. Neutrophil elastase is an enzyme associated with tissue inflammation, leading to degradation of the lung tissue in cystic fibrosis and several other pulmonary diseases.

Lonodelestat has EU orphan drug designations (ODD) for the treatment of CF as well as for AATD and PCD in both EU and US.

Santhera obtained the worldwide, exclusive rights from Polyphor AG to develop and commercialize lonodelestat in CF and other diseases.

Lonodelestat in development

In preclinical studies lonodelestat was effective in animal models of neutrophil activation in lung tissue and of acute lung injury (ALI) [1, 2]. Currently available clinical data demonstrated that single and multiple doses (Phase 1b) of lonodelestat when administered by inhalation via an optimized eFlow® nebulizer (PARI Pharma GmbH) can lead to high drug concentrations within the lung, resulting in inhibition of hNE in sputum of patients, an enzyme associated with lung tissue inflammation [3]. The Phase 1b study further confirmed the tolerability of lonodelestat after treatment of up to four weeks in patients with CF. Lonodelestat may also show therapeutic benefit for a range of neutrophilic pulmonary diseases with high medical need such as non-CF bronchiectasis (NCFB), alpha-1 antitrypsin deficiency (AATD), chronic obstructive pulmonary disease (COPD), acute respiratory distress syndrome (ARDS) or primary ciliary dyskinesia (PCD). Lonodelestat has EU orphan drug designations (ODD) for the treatment of CF as well as for AATD and PCD in both EU and US.

Santhera acknowledges the support of the Cystic Fibrosis Foundation (CFF) by providing funding for the conduct of the Phase 1a and 1b safety trials with lonodelestat.

For more information please see:
Santhera Announces Positive Results with Lonodelestat in Early Phase Cystic Fibrosis Trial (March 1, 2021)
Santhera Announces Publication of Phase I Clinical Data with POL6014 in Journal of Cystic Fibrosis (September 19, 2019)
Santhera Obtains Worldwide Exclusive License from Polyphor to Develop and Commercialize Clinical Stage Candidate for Cystic Fibrosis and Other Pulmonary Diseases (February 15, 2018)

References

1.	Sellier Kessler O et al. Effect of POL6014, a potent and selective inhaled neutrophil elastase inhibitor, in a rat model of lung neutrophil activation. Am J Respir Crit Care Med 2018; 197: A2988
2.	Lagente V et al. A novel protein epitope mimetic (PEM) neutrophil elastase (NE) inhibitor, POL6014, inhibits human NE-Induced acute lung injury in mice. Am J Respir Crit Care Med 2009; 179: A5668
3.	Barth P et al. Single dose escalation studies with inhaled POL6014, a potent novel selective reversible inhibitor of human neutrophil elastase, in healthy volunteers and subjects with cystic fibrosis. J Cyst Fibros 2020; 19: 299-304