Slide 1: CONNECT1-EDO51: A 12-Week Open-Label Phase 2 Study of PGN-EDO51
CONNECT1-EDO51: A 12-Week Open-Label Phase 2 Study to Evaluate PGN-EDO51 Safety and Efficacy in People with Duchenne Amenable to Exon 51 Skipping Michelle Mellion1, Hugh McMillan2, Nicolas Chrestian3, Hernan Gonorazky4, Colleen O'Connell5, Sarah Vacca1, Mark Peterson1, Bassem Morcos1, Sejal Batra1,
Slide 2: Conflict of Interest
Conflict of Interest • I am a full-time employee of PepGen • I receive compensation, stock and benefits from PepGen 2
Slide 3: PepGen's EDO Technology Engineered to Optimize Tissue Delivery and Nuclear Uptake
PepGen’s Enhanced Delivery Oligonucleotide (EDO) Technology is Engineered to Optimize Tissue Delivery and Nuclear Uptake of Therapeutic Oligonucleotides PepGen’s EDO: Up to 25X Higher Nuclear Uptake of Oligonucleotide vs PMO PGN-EDO51 is PepGen’s investigational clinical candidate for the treatment
Slide 4: CONNECT1: Designed to Establish Proof-of-Concept and Inform CONNECT2-EDO51
CONNECT1*: Designed to Establish Proof-of-Concept and Inform CONNECT2-EDO51 Clinical Trial Design • Open label clinical trial in Canada • DMD patients (n=10) with exon 51 skippable mutation • Ages ≥ 8 years • Ambulatory and non-ambulatory Endpoints • Safety and tolerability • Dystrophin • Muscle tis
Slide 5: CONNECT1 5 mg/kg: Safety Profile
CONNECT1 5 mg/kg: Safety Profile1 MAD Period n (%) • All treatment emergent adverse events (TEAEs) Any TEAEs, n (%) 3 (100) were mild and resolved Related to study drug 1 (33.3) • Related TEAE was mild (abdominal pain, flatulence) • No discontinuations, dose modifications or dose • Mild 1 (33.3) int
Slide 6: PGN-EDO51 Produced High Exon Skipping and Promising Dystrophin Increase
PGN-EDO51 Produced High Levels of Mean Exon Skipping and Promising Dystrophin Increase over Short Treatment Duration and with Few Doses Total (unadjusted) Dystrophin 4 doses over 3 months MHC: Myosin Heavy Chain, BL: Baseline, SEM: Standard Error of the Mean 6
Slide 7: Dosing Continues in Cohort 2 at 10 mg/kg
Dosing Continues in Cohort 2 at 10 mg/kg1 • The emerging safety profile is favorable • At 10 mg/kg, a total of 7 doses have been administered • All treatment-related AEs were mild and have resolved without treatment • Asymptomatic mild hypomagnesemia in one patient was observed and treated with oral
Slide 8: CONNECT1 Key Takeaways
CONNECT1 Key Takeaways • PGN-EDO51 has a favorable safety profile to date1 • All participants at 5 mg/kg demonstrated increased dystrophin production and exon skipping at just 3 months and after 4 doses • Results support that our EDO technology delivers high levels of oligonucleotides to the nucleus
Slide 9: Thank you!
Thank you! All the study participants and their families The clinical investigators and their teams • Dr. Hugh McMillan, MD, MSc, FRCPC Children's Hospital of Eastern Ontario (CHEO) • Dr. Hernan Gonorazky, MD, CSCN The Hospital for Sick Children (SickKids) • Dr. Nicolas Chrestian, MD, FRCPC, CSCN CH