NEWS: (No Negative Findings) PepGen Announces Positive Data from Low-Dose Cohort of PGN-EDO51 in Ongoing CONNECT1-EDO51 Phase 2 Clinical Trial for Treatment of Duchenne Muscular Dystrophy
Positive
▪️PGN-EDO51 achieved mean exon skipping levels of 2.15% after three months at 5 mg/kg dose
▪️Mean muscle-adjusted dystrophin level increased by 0.70% from baseline
▪️Mean absolute dystrophin level increased by 0.26% from baseline
▪️The 5 mg/kg dose was well-tolerated with no discontinuations or dose reductions
▪️All patients from the 5 mg/kg cohort continued to the long-term extension phase
▪️Dosing of the second cohort at 10 mg/kg is ongoing
▪️PGN-EDO51 achieved mean exon skipping levels of 2.15% after three months at 5 mg/kg dose
▪️Mean muscle-adjusted dystrophin level increased by 0.70% from baseline
▪️Mean absolute dystrophin level increased by 0.26% from baseline
▪️The 5 mg/kg dose was well-tolerated with no discontinuations or dose reductions
▪️All patients from the 5 mg/kg cohort continued to the long-term extension phase
▪️Dosing of the second cohort at 10 mg/kg is ongoing
Negative
▪️None.
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