Amicus Therapeutics announces 3-in-3 strategy for the next generation ERTs Amicus Therapeutics provided its FY14 strategic outlook and financial guidance. Key Highlights include: Amicus announces "3-in-3" strategy for three next-generation Enzyme Replacement Therapies, or ERTs, expected to enter the clinic in next three years with lead programs in Fabry, Pompe and Mucopolysaccharidosis I; preclinical proof-of-concept data for next-generation ERTs for Fabry and Pompe to be featured at Lysosomal Disease Network WORLD Symposium and American College of Medical Genetics meetings in 1H14; data from global registration studies of migalastat HCl monotherapy for Fabry disease expected to include complete 12- and 24-month data from Study 011 in 2Q14 and 18-month clinical data from Study 012 in 2H14. Company also stated "As we enter 2014, Amicus is strongly positioned to continue our focus on advancing a pipeline of next-generation enzyme replacement therapies for patients with lysosomal storage diseases. "
Amicus Therapeutics sees current cash to fund operations into 2017 Amicus Therapeutics provided its full-year 2015 strategic outlook and financial guidance. The company sees its FY15 net cash spend expected to range between $73M-$83M. Current cash of $169.1M at December 31, 2014 is projected to fund operating plan into 2017. Amicus said migalastat monotherapy FDA interaction is planned in 1Q15 and an MAA submission is on track for mid-2015 to seek marketing approval for Fabry patients with amenable mutations. Initiation of longer-term Phase 2 Fabry co-administration study is anticipated in 2015 in support of Fabry franchise strategy to develop migalastat in combination with ERT for Fabry patients with non-amenable mutations. Next-generation Pompe ERT is set to enter clinic in 2H15 and additional details on Pompe program are to be highlighted at 33rd Annual J.P. Morgan Healthcare Conference, the company said.