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PepGen to Advance PGN-EDODM1 Into Highest Dose Cohort in Phase 2 FREEDOM2-DM1 Study Following DSMB Review

PEPG
  • An independent Data and Safety Monitoring Board (DSMB) recommended advancing PepGen's Phase 2 FREEDOM2-DM1 study to the highest multiple ascending dose cohort of 12.5 mg/kg for PGN-EDODM1.
  • The DSMB also recommended escalating the dose in the open-label extension (OLE) study from 5 mg/kg to 10 mg/kg.
  • No changes to the study protocol were recommended, & six of eight participants from the 5 mg/kg FREEDOM2 cohort have enrolled in the OLE, bringing total OLE enrollment to 16 participants.
  • PGN-EDODM1 remains generally well-tolerated with no serious adverse events, dose-limiting toxicities, or cumulative toxicity reported across the 5 mg/kg & 10 mg/kg levels.
  • Seven of eight participants in the fully enrolled 10 mg/kg FREEDOM2 cohort have completed dosing, & no treatment discontinuations were reported in either FREEDOM2 or the OLE.
  • PepGen expects to report results from the 10 mg/kg FREEDOM2 cohort in November 2026, with data from the 12.5 mg/kg cohort expected in the first half of 2027.
  • Plans include engaging with regulators at an end of Phase 2 meeting in early 2027 to discuss the registrational program, & an OLE study update is expected by early January 2027.
  • PGN-EDODM1 utilizes PepGen's proprietary EDO technology to restore normal splicing function of the MBNL1 protein by disrupting pathogenic CUG repeat expansions in DMPK transcripts.
  • The U.S. FDA has granted PGN-EDODM1 Orphan Drug & Fast Track Designations for DM1 treatment, & the EMA has granted Orphan Designation.