PepGen (PEPG) investor relations material

Vision and platform

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

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

• Strong cash runway projected into Q4 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 rate of splicing correction ever reported in DM1 after a single dose.

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

• FREEDOM2 trial cleared in South Korea, Australia, and New Zealand; enrollment active in Canada, UK, and South Korea.

Disease background and unmet need

• DM1 is caused by CTG expansion in the DMPK gene, affecting over 110,000 patients in the U.S. and EU.

• Symptoms are multisystemic, impacting CNS, muscle, respiratory, cardiovascular, gastrointestinal, and reproductive systems.

• No approved therapies currently address the underlying cause of DM1.