PepGen Publishes Presentation on FREEDOM2-DM1 10 mg/kg Clinical Trial Results

PUBT · 3d ago
PepGen Publishes Presentation on FREEDOM2-DM1 10 mg/kg Clinical Trial Results
  • PepGen published its FREEDOM2-DM1 10 mg/kg presentation, reporting Phase 2 results for PGN-EDODM1 in myotonic dystrophy type 1.
  • Mean splicing correction reached 46.5% at week 13 among six treated patients, versus 6.8% with placebo.
  • Hand-opening time improved by 4.3 seconds at week 13 and 3.9 seconds at week 16 in the 10 mg/kg group.
  • All adverse events were mild or moderate, with no serious adverse events or treatment discontinuations in the study.
  • The 12.5 mg/kg cohort is fully enrolled, with participants having received up to three doses.


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