BioMarin dropping BMN 195
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) discontinued development of BMN 195 (formerly SMT C1100) after data from a Phase I trial to treat Duchenne muscular dystrophy (DMD) showed that the small molecule utrophin up-regulator did not achieve plasma concentrations required to increase utrophin expression. The company said it continues to believe that utrophin up-regulation is a viable approach to treat DMD. BioMarin received exclusive, worldwide rights to BMN 195 from Summit Corp. plc (LSE:SUMM) under a 2008 deal.
BioMarin also reported preliminary data from an ongoing open-label, Phase II trial of PEG-PAL in 23 patients. Six of seven patients receiving PEG-PAL for at least four weeks had phenylalanine levels of less than 600 umol/L for at least three weeks. PEG-PAL is a pegylated recombinant phenylalanine ammonia lyase (PAL) enzyme. ...