PROSENSA'S PRO044 DATA PRESENTED AT WMS BY DR. NATHALIE GOEMANS

Here are top line notes from the PRO044 presentation at World Muscle Society, Oct 4.

Age of inclusion: 5-16 Mean age: 9

The drug was well tolerated.

They saw trace dystrophin and reverant fibers pre- treatment.

18 subjects, 9 cohorts… SubQ delivery: .5mg/kg, 1.5mg/kg, 5mg/kg, 8mg/kg, 10mg/kg, 12mg/kg

IV delivery: 1.5mg/kg, 5mg/kg, 12mg/kg

5 weekly injections, 13 week follow up

Increase in dystrophin after 5 weeks: SubQ delivery: 6 of 13 (46%), IV delivery: 6 of 8 (75%)

Higher skipping efficiency than drisapersen

Extension study will use 6-9mg/kg.

No information on timing of next studies were given.