PepGen (PEPG) Company presentation summary

Vision and platform overview

• Aims to develop therapies addressing the root cause of serious genetic neuromuscular and neurological diseases, focusing on functional improvement.

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

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

• Research pipeline explores applications in additional genetic conditions, including Charcot-Marie-Tooth disease.

PGN-EDODM1 mechanism and preclinical data

• Selectively targets pathogenic DMPK RNA in myotonic dystrophy type 1 (DM1), aiming for best-in-class efficacy.

• Demonstrated up to 98-fold increase in nuclear uptake and robust splicing correction in preclinical DM1 models.

• Multiple doses in animal models led to greater splicing correction and improved myotonia compared to a single dose.

• Reduced toxic nuclear foci and liberated MBNL1, correcting mis-splicing in patient-derived cells.

Clinical trial design and results

• FREEDOM Phase 1 SAD: Multinational, randomized, double-blind, placebo-controlled study with single IV doses at 5, 10, and 15 mg/kg.

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

• Robust, greater than dose-proportional increase in muscle tissue concentration following single dose.

• FREEDOM2 Phase 2 MAD: Multiple IV doses every 4 weeks, with key endpoints including safety, PK, splicing correction, and functional assessments.