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.

• Cash runway extends into the second half of 2027.

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

PGN-EDODM1 and disease focus

• PGN-EDODM1 selectively targets pathogenic DMPK RNA in myotonic dystrophy type 1 (DM1), showing best-in-class potential.

• No approved therapies currently address the underlying cause of DM1, which affects over 115,000 patients in the US and EU.

• PGN-EDODM1 binds to CUG repeat expansions in DMPK RNA, liberating MBNL1 and restoring correct splicing.

• Demonstrated highest rate of splicing correction ever reported in DM1 after a single dose.

Preclinical and clinical data

• EDO technology increases nuclear uptake of oligonucleotides up to 98-fold compared to naked oligos.

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

• Phase 1 FREEDOM study showed dose-dependent, best-in-class splicing correction, with 87.5% of participants showing improvement.

• Robust, greater than dose-proportional increases in muscle tissue concentration observed after a single dose.