PepGen Touts Encouraging Early-Stage Data From Duchenne Program – PepGen (NASDAQ:PEPG)

PepGen Touts Encouraging Early-Stage Data From Duchenne Program - PepGen (NASDAQ:PEPG)

  • PepGen Inc PEPG announced results from its completed Phase 1 healthy volunteer trial of PGN-EDO51, the company’s lead product candidate for Duchenne Muscular Dystrophy (DMD) patients whose mutations are amenable to an exon 51 skipping approach.
  • The trial met its primary endpoint providing evidence that PGN-EDO51 was generally well tolerated at pharmacologically relevant doses.
  • All participants completed the trial; there were no discontinuations.
  • Most treatment-emergent adverse events (TEAEs) were assessed as mild and resolved without intervention. At 10 mg/kg, there was only Grade 1 (mild) adverse events (AEs).
  • At 15 mg/kg, there were mild, transient, reversible changes in kidney biomarkers that resolved without intervention in all but one volunteer who received IV hydration. This event was recorded as a non-life-threatening serious adverse event (SAE).   
  • Transient mild (Grade 1) to moderate (Grade 2) hypomagnesemia was observed in two participants at the 15 mg/kg dose and did not require any intervention.
  • Serum cystatin C, the recommended biomarker to assess renal function in DMD, showed minimal change at the highest dose.
  • PGN-EDO51 exhibited the highest levels of oligonucleotide delivery and exon skipping in a clinical study following a single dose compared to publicly available clinical data for other exon 51 skipping approaches.
  • Price Action: PEPG shares are up 15.4% at $6.00 during the premarket session on the last check Wednesday.

Written by biedex markets