PepGen Touts Encouraging Early-Stage Data From Duchenne Program

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.