Cystic Fibrosis: Update Bulletin [August 2017]

This update bulletin edition presents US and EU key opinion leader (KOL) views on recent developments in the cystic fibrosis (CF) space. Topics covered include; Anthera Pharmaceuticals announcing that it has commenced screening in Europe in the Phase III non-inferiority RESULT study comparing the pancreatic enzyme replacement therapy (PERT) Sollpura to Pancreaze for exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF); Protalix BioTherapeutics’ announcing new positive Phase II trial results for Protalix BioTherapeutics’ AIR DNase (alidornase alfa; PRX-110) which were presented at the 40th European Cystic Fibrosis Society Conference in Seville, Spain in June 2017; and Verona Pharma initiating a Phase II pharmacokinetic trial in the US for RPL554, the company’s a first-in-class, inhaled, long-acting, mixed phosphodiesterase (PDE) 3/4 inhibitor, following the acceptance of an Investigational New Drug application (IND) by the US FDA.

Business Questions:

• How do KOLs rate the potential for an additional pancreatic enzyme replacement therapy in CF?
• What are KOLs’ opinions on the upcoming Phase III result study of Sollpura?
• If approved, what are the chances that Sollpura will be successful at gaining market share?
• What will AIR DNase need to show in order to be considered as a competitor to Pulmozyme?
• When could AIR DNase make it to market and what will it need to show in order to get there?
• What are KOLs’ thoughts on the development of RPL554 and to what extent do they consider it to be a viable product in CF?
• What safety concerns do KOLs have about RPL554?
• If approved, how would RPL554 be incorporated into the treatment paradigm for CF?