PepGen Inc. · NASDAQ:PEPG
PepGen — Technical Presentation (November 2026)
26 slides from PepGen's technical presentation, Nov 10, 2026
Slide 1: FREEDOM2-DM1 10 mg/kg MAD Cohort Data Update
November 10, 2026 FREEDOM2-DM1 10 mg/kg MAD Cohort Data Update
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation is being made by PepGen Inc. (“PepGen,” “we,” “our,” “us”) and contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as “aims,” “anticip
Slide 3: Agenda
Agenda Paul Streck, MD, MBA Executive Vice President, Head of R&D FREEDOM2 Clinical Trial Design, Safety, Splicing, and Q&A James McArthur, PhD President and Chief Executive Officer Key Takeaways, Closing Remarks, and Q&A Noel Donnelly, MBA Chief Financial Officer Q&A Johanna Hamel, MD Associate Pro
Slide 4: PGN-EDODM1 MAD 10 mg/kg Cohort Reported Best-In-Class Splicing with Improvements
PGN-EDODM1 MAD 10 mg/kg Cohort Reported Best-In-Class Splicing with Improvements Across Myotonia, Mobility, and Grip Strength 1 2 3 4 Highest level of splicing correction reported in a multiple dose study 46.5% mean change in splicing at week 13 5 of 6 patients improved more than 20% natural variabi
Slide 5: PGN-EDODM1 Introduction, Study Design, Splicing, and PK
PGN-EDODM1 Introduction, Study Design, Splicing, and PK Paul Streck, MD, MBA Head of R&D
Slide 6: Myotonic Dystrophy Type 1 (DM1)
Sources: Neuroepidemiology (2022) 56 (3): 163–173., Neurology 2021 Feb 16;96(7):e1045-e1053 1Some estimates are as much as 5-fold higher prevalence of DM1: Brain (10 Oct 2026), awag250 CTG: cytosine-thymine-guanine; DMPK: dystrophia myotonica protein kinase Tara and Rhys, Living with DM1 Jubal and F
Slide 7: PGN-EDODM1 Designed to Target Pathogenic DMPK RNA Using EDO Technology
PGN-EDODM1 is Designed to Target Pathogenic DMPK RNA to Restore Splicing Using EDO Technology to Deliver to a Broad Range of Tissues EDO technology improves endosomal escape, resulting in a 98-fold increase in nuclear delivery EDO technology uses receptor independent peptide delivery for broad, mult
Slide 8: FREEDOM2: Phase 2 MAD Study Design
FREEDOM2: Phase 2 MAD Study Design 1The U.S. FDA placed a partial clinical hold on FREEDOM2-DM1, with no U.S. participants dosed in the FREEDOM2-DM1 study to date DSMB: data safety monitoring board; FU: follow-up; IV: intravenous; MAD: multiple-ascending dose; PBO: placebo; PK: pharmacokinetics; vHO
Slide 9: Baseline Demographics of FREEDOM2 Study Participants
Baseline Demographics of FREEDOM2 Study Participants 1Splicing index is measured on a scale of 0 to 1: mild = 0–0.40, moderate = 0.41–0.75, and severe = 0.76–1.00 (Provenzano M, et al. J Clin Invest. 2025;135:e185426) BMI, body mass index; Q4W, once every 4 weeks; SD, standard deviation Characterist
Slide 10: Consistent, Favorable Safety Profile of PGN-EDODM1 at 5 and 10 mg/kg
Any TEAE 7 (87.5) 8 (100) Mild Moderate Severe 4 (50.0) 3 (37.5) 0 (0.0) 4 (50.0) 4 (50.0) 0 (0.0) Any SAE 0 0 Any related SAE 0 0 Any AESI or dose-limiting toxicities 0 0 Any TEAE leading to study withdrawal 0 0 Any TEAE leading to death 0 0 Consistent, Favorable Safety Profile of PGN-EDODM1 at 5 a
Slide 11: Renal Biomarker Analysis Supports Favorable Safety Profile
Renal Biomarker Analysis Supports Favorable Safety Profile 1Magnesium: One participant showed a decrease in serum magnesium at a single timepoint at 10mg/kg 2Serum Creatinine: One participant at 10mg/kg at a single timepoint went above the upper limit of normal and returned to normal 24 hours later
Slide 12: Highest Splicing Correction Reported in a Multiple Dose Study
PGN-EDODM1: Highest Splicing Correction Reported in a Multiple Dose Study Demonstrates Robust Target Engagement 1 DM1 Splicing index at day 28 analysis from the single ascending dose (SAD) study 2 DM1 Splicing index at day 7 following the fourth dose from the multiple ascending dose (MAD) study 3Mis
Slide 13: Splicing Index Changes: 22-Gene Panel
Splicing Index Changes: 22-Gene Panel2 4 of 6 participants at 10 mg/kg showed improved splicing greater than 50% PGN-EDODM1 Splicing Correction Exceeds Natural Splicing Variability1 in 5 of 6 Participants at 10 mg/kg DM1 Splicing index at day 7 following the fourth dose from FREEDOM2-DM1 clinical st
Slide 14: Repeated Dosing of PGN-EDODM1 Increased Muscle Tissue Levels
Repeated Dosing of PGN-EDODM1 Increased Muscle Tissue Levels 28d or 7d indicates days following the only dose (SAD) or last dose (MAD 4 dose study) 1SAD 5mg/kg group reflects n=4 above the lower limit of quantification (LLOQ) and SAD 10 mg/kg group reflects n=5 due to unavailability of biopsy tissue
Slide 15: FREEDOM2 Key Functional Outcomes
FREEDOM2 Key Functional Outcomes Johanna Hamel, MD University of Rochester
Slide 16: In DM1, Muscle Dysfunction Drives Multisystemic Impact for Patients
In DM1, Muscle Dysfunction Drives Multisystemic Impact for Patients DM1 Most Prevalent Symptoms1 1Christopher Project Reference Group. The myotonic dystrophy experience: a North American cross-sectional study. Muscle Nerve. 2019;59(4):457-464. doi:10.1002/mus.26420. Tara and Rhys, Living with DM1
Slide 17: Early and Sustained Improvement in Myotonia at 10 mg/kg
Early and Sustained Improvement in Myotonia at 10 mg/kg Myotonia measured by video Hand Opening Time (vHOT) FREEDOM2 10 mg/kg data plotted two ways: Left panel shows change from baseline over time; Right panel shows change from baseline as a bar graph with mean and standard error of the mean vHOT an
Slide 18: vHOT vs CLCN1 Splicing at 10 mg/kg
vHOT vs CLCN1 Splicing at 13 Weeks Splicing Correction of CLCN1, a Muscle Chloride Channel, was Associated with Improved Myotonia at 10 mg/kg Data illustrate direction only and do not represent a statistical correlation 1Charlet-B. et al. Mol. Cell, 2002, Vol. 10, 35-44 DSI: DM1 splicing index; Q4W:
Slide 19: Hand Grip Strength Analysis
Hand Grip Strength Analysis Hand Grip Change From Baseline (max PPN) FREEDOM2 10 mg/kg data plotted two ways: Left panel shows change from baseline over time; Right panel shows change from baseline as a bar graph with mean and standard error of the mean PPN: percent predicted normal; SE: standard er
Slide 20: Timed Up and Go Analysis
Timed Up and Go Analysis TUG Change From Baseline (sec) FREEDOM2 10 mg/kg data plotted two ways: Left panel shows change from baseline over time; Right panel shows change from baseline as a bar graph with mean and SEM Week 12 was not collected for this endpoint; SE: standard error Q4W: One dose ever
Slide 21: 10-Meter Walk/Run Test Analysis
10-Meter Walk/Run Test Analysis 10MWRT Change From Baseline (sec) FREEDOM2 10 mg/kg data plotted two ways: Left panel shows change from baseline over time; Right panel shows change from baseline as a bar graph with mean and SEM Week 12 was not collected for this endpoint; SE: standard error Q4W: One
Slide 22: Conclusion
Conclusion James McArthur, PhD CEO
Slide 23: Highest Reported Splicing Correction in a Multidose Study and Functional Improvement
Highest Reported Splicing Correction in a Multidose Study and Improvement in Mobility, Myotonia, and Strength, with Favorable Safety Profile Observed at 10 mg/kg 10 mg/kg n=6, placebo pooled n=4; Percent improvement values are shown as week 13 for splicing, and week 16 for all other endpoints. Perce
Slide 24: Next Steps for PGN-EDODM1 Development
Next Steps for PGN-EDODM1 Development DLT: Dose limiting toxicities; SAE: Serious adverse event FREEDOM2 AND FREEDOM-OLE Cohort 3 (12.5 mg/kg) is fully enrolled, participants have received up to 3 doses FREEDOM2 and OLE studies are expected to support End of Phase 2 meeting in H2 2027 H1 2027: FREED
Slide 25: Question and Answer Session
Question and Answer Session Paul Streck, MD, MBA Executive Vice President, Head of R&D James McArthur, PhD President and Chief Executive Officer Noel Donnelly, MBA Chief Financial Officer Johanna Hamel, MD Associate Professor of Neurology, Pathology and Laboratory Medicine University of Rochester
Slide 26: Thank you
g Thank you