PTC124 to Be Tested at Higher Doses in DMD

PTC Therapeutics of South Plainfield, N.J., will test a higher dose of its experimental compound PTC124 in approximately 12 boys with Duchenne muscular dystrophy (DMD) caused by a premature stop codon mutation. The drug is designed to coax muscle cells to ignore these mutations and make functional dystrophin.

In October, the company announced that, in a trial of 26 boys with DMD, the drug appeared safe and well tolerated. Results showed significant decreases in serum creatine kinase (CK), which leaks out of damaged muscle tissue, and increases in dystrophin.

In the higher-dose study, boys will take the drug at 20 milligrams per kilogram of body weight at breakfast and lunch and 40 milligrams per kilogram at dinner for 28 days. They’ll be followed closely for an additional 28 days and periodically after that.

Participants must have a documented premature stop codon mutation in the dystrophin gene, be at least 5 years old and meet other study criteria. Contact Kerri Donnelly at PTC Therapeutics at (908) 222-7000, ext. 112, or kdonnelley@ptcbio.com.