PepGen (PEPG) Company presentation summary

Vision and platform overview

• Focus on therapies addressing root causes of serious genetic neuromuscular and neurological diseases, aiming for meaningful functional improvement.

• EDO platform achieves superior nuclear delivery and uptake of therapeutic oligonucleotides, overcoming prior limitations.

• Strong cash runway projected into the second half of 2027, supporting ongoing clinical milestones.

• Research pipeline explores EDO's potential in additional genetic conditions, including Charcot-Marie-Tooth disease.

PGN-EDODM1 mechanism and preclinical data

• Selectively targets pathogenic DMPK RNA, addressing the underlying cause of myotonic dystrophy type 1 (DM1).

• Demonstrated highest rate of splicing correction in DM1 after a single dose, with robust nuclear uptake up to 98-fold higher than naked oligonucleotides.

• Preclinical studies showed multiple doses led to greater splicing correction and improved myotonia compared to a single dose.

• Cost-effective manufacturing due to short linear peptide design.

Clinical trial design and results

• FREEDOM Phase 1 SAD: Multinational, randomized, double-blind, placebo-controlled study with single IV doses (5, 10, 15 mg/kg) in DM1 patients.

• Dose-dependent, best-in-class splicing correction observed, with 87.5% of participants showing improvement.

• Muscle tissue concentration increased more than dose-proportionally, supporting effective delivery.

• FREEDOM2 Phase 2 MAD: Multiple IV doses (5, 10, 12.5 mg/kg) every 4 weeks for 12 weeks, with ongoing enrollment and open-label extension.