Amicus Therapeutics, JCR Pharmaceuticals, GlaxoSmithKline preclinical data

In mice, Amigal migalastat co-formulated with JR-051 significantly reduced globotriaosylceramide (GL-3) levels in the heart and kidneys vs. JR-051 alone. Details were not disclosed. Accumulation of GL-3 causes various symptoms of Fabry's disease, including pain, kidney failure and an increased risk of heart attack or stroke. Under an amended 2010 deal with GSK, Amicus is responsible for U.S. commercialization of the co-formulation of Amigal and JR-051 for which clinical trials are planned for next year (see BioCentury, Nov. 1, 2010). JCR granted GSK worldwide rights to enzyme replacement therapies, including JR-051, to treat lysosomal storage diseases in 2009. JR-051 is a recombinant human alpha galactosidase A enzyme. ...