PepGen (PEPG) corporate 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 for myotonic dystrophy type 1 (DM1)

• Selectively targets pathogenic DMPK RNA, showing best-in-class potential and favorable emerging safety profile.

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

• Regulatory clearance received in South Korea, Australia, and New Zealand; enrollment open in Canada, UK, and South Korea.

• Orphan Drug & Fast Track Designation in the U.S.; Orphan Drug Designation in the EU.

Mechanism of action and preclinical data

• PGN-EDODM1 binds selectively to pathogenic DMPK transcripts, reducing toxic RNA foci and liberating MBNL1 to restore correct splicing.

• In patient cells, treatment led to a 54% reduction in toxic foci and 69% correction of mis-splicing.

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