2017 Outlook:
2017 Outlook and Projected Future Milestones
Full results for the phase II efficacy and safety study of alidornase alfa expected to be announced during the first quarter of 2017.
Full results for the phase II efficacy and safety study of OPRX-106 expected to be announced during the second half of 2017.
Completion of enrollment in the phase III trial of pegunigalsidase alfa for the treatment of Fabry disease expected in 2017; interim data analysis anticipated in 2018 to support EMA and other regulatory filings outside of the United States.
If superiority to Fabrazyme is not yet achieved at that time, as shown in the interim data, the trial will continue for an additional year before reporting two-year data to support a U.S. regulatory filing.
If superiority to Fabrazyme is not yet achieved at that time, as shown in the interim data, the trial will continue for an additional year before reporting two-year data to support a U.S. regulatory filing.
Present, via two key opinion leader (KOL) presentations and a poster, pegunigalsidase alfa clinical data at the 13th Annual WORLD Symposium™ to be held in February 2017.
Present at the 5th Update on Fabry Nephropathy: Biomarkers, Progression and Treatment Opportunities in April 2017.
Present, at the 40th European Cystic Fibrosis Conference, full data from the Company’s phase II efficacy and safety study of alidornase alfa, in June 2017, and at the North American Cystic Fibrosis Conference in November 2017.
Potential partnership for alidornase alfa.
Potential collaboration for alidornase alfa with the Cystic Fibrosis Foundation.
Potential partnership for oral anti-TNF.
