Pepgen Inc. · NASDAQ:PEPG
Pepgen — Investor Presentation (August 2026)
29 slides from Pepgen's investor presentation, August 2026
Slide 1: Company Presentation
Company Presentation August 2026
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation 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,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,
Slide 3: Leveraging EDO Platform to Drive Meaningful Impact for Patients
Leveraging EDO Platform to Drive Meaningful Impact for Patients PGN-EDODM1: Myotonic Dystrophy Type 1 OUR VISION • Best-in-class potential; selectively targets only pathogenic DMPK RNA • Favorable emerging safety profile and FREEDOM2 5 mg/kg results supportive of the ongoing dosing in 10 mg/kg MAD c
Slide 4: PepGen's EDO Platform Designed to Solve the Delivery Challenge of Oligonucleotides
PepGen’s EDO Platform Has Been Designed and Developed to Solve the Delivery Challenge of Oligonucleotides PGN-EDODM1 Delivery to DM1 Patient Myotubes EDO platform results in nuclear delivery of oligonucleotide therapeutics Red = oligo Immortalized myoblasts from a DM1 patient with 2600 CTG repeats w
Slide 5: EDO Technology Can Improve Endosomal Escape and Increase Nuclear Uptake up to 98-Fold
EDO Technology Can Improve Endosomal Escape and Has Been Shown to Increase Nuclear Uptake up to 98-Fold PMO Delivery in DM1 Cells 73x NUCLEAR UPTAKE 2.0 Nuclear uptake (pmol) 1.5 98x 1.0 55x 0.5 0.0 1 5 10 20 Concentration (nmol) Immortalized myoblasts from a healthy individual or a DM1 patient with
Slide 6: PGN-EDODM1: A Differentiated Drug with Best-in-Class Potential
PGN-EDODM1: A Differentiated Drug with Best-in-Class Potential 1 Differentiated Delivery Technology • Receptor-independent EDO peptide delivery • Designed to escape the endosome – unlike TfnR targeting 2 Differentiated Target • Selectively targets pathogenic RNA (CUG repeat in DMPK) • Demonstrated h
Slide 7: PGN-EDODM1 – Myotonic Dystrophy Type 1 (DM1)
PGN-EDODM1 – Myotonic Dystrophy Type 1 (DM1)
Slide 8: Myotonic Dystrophy Type 1 Overview and Unmet Medical Need
Myotonic Dystrophy Type 1 Overview and Unmet Medical Need Jubal, retired professor living with DM1 DM1 Overview • Caused by CTG expansion in the DMPK gene, resulting in pathogenic DMPK transcripts driving disease pathology • More than 110,000 patients affected in the U.S. and EU • Symptoms can devel
Slide 9: DM1's Broad, Multisystemic Symptoms Underscore the Need for a Disease-Modifying Therapy
DM1's Broad, Multisystemic Symptoms Underscore the Need for a Disease-Modifying Therapy DM1 SYMPTOMS CNS: fatigue, daytime sleepiness, difficulty concentrating, hypersomnia, Cardiovascular: learning difficulties cardiac conduction issues Speech and Swallowing: Gastrointestinal: dysphagia, aspiration
Slide 10: PGN-EDODM1 Blocking Approach Targets the Pathogenic CUGexp Repeats DMPK RNA
PGN-EDODM1 Blocking Approach Targets the Pathogenic CUGexp Repeats DMPK RNA PGN-EDODM1 binds selectively to the pathogenic DM1 is caused by pathogenic DMPK transcripts DMPK transcript Trapped Liberated MBNL1 is MBNL1 inactive and restores correct results in splicing mis-splicing • DM1 is caused by p
Slide 11: PGN-EDODM1 Reduced Nuclear Foci, Liberated MBNL1 and Corrected Mis-Splicing in Patient Cells
PGN-EDODM1 Reduced Pathogenic Nuclear Foci, Liberated MBNL1 and Corrected Mis-Splicing in Patient Cells with Long CUG Repeats Foci Reduction MBNL1 Liberation Mis-Splicing Correction Not Treated Not Treated Across multiple transcripts 69% correction 54% reduction in PGN-EDODM1 Treated PGN-EDODM1 Trea
Slide 12: Multiple Doses Led to Greater Splicing Correction and Myotonia Improvement in Preclinical Studies
Multiple Doses of PGN-EDODM1 Led to Greater Improvement in Splicing Correction and Myotonia vs Single Dose in Preclinical Studies Tissue Concentration Mis-Splicing Correction Correction of Myotonia Skeletal muscle Across multiple transcripts Pinch test 99% 120 68% 56 Correction of 76% myotonia 0 0%
Slide 13: PGN-EDODM1 – FREEDOM SAD Trial in DM1
PGN-EDODM1 – FREEDOM SAD Trial in DM1
Slide 14: FREEDOM: Phase 1 PGN-EDODM1 Single-Ascending Dose Study Design
FREEDOM SAD FREEDOM: Phase 1 PGN-EDODM1 Single-Ascending Dose Study Design Single Dose PGN-EDODM1 or Placebo (randomized 3:1) Baseline DSMB D28 Wk 16 15 mg/kg Biopsy Biopsy Biopsy n=8 or PBO Multinational, randomized, double-blind, placebo-controlled SAD study in patients Baseline DSMB D28 Wk 16 Dos
Slide 15: PGN-EDODM1 Produced Dose-Dependent Splicing Correction Following Single Dose
FREEDOM SAD PGN-EDODM1 Produced Dose-Dependent Best-in-Class Splicing Correction Following Single Dose Splicing Index Changes: 22-Gene Panel* at D28 % Mean change from baseline Improvement 3.3% 87.5% of participants -12.3% across all doses showed improved splicing -29.1% -53.7% Placebo1 5 mg/kg 10 m
Slide 16: ~11% of Untreated DM1 Patients Demonstrate >20% Splicing Improvement
Natural History ~11% of Untreated DM1 Patients Demonstrate >20% Splicing Improvement over a 2 to 3 Month Time Period Natural History Data in Untreated DM1 Patients* ~15% OF UNTREATED PATIENTS ~74% OF UNTREATED PATIENTS ~11% OF UNTREATED PATIENTS >20% ±20% >20% Worse Splicing Midrange Correction Spli
Slide 17: Higher Doses Associated with More Patients Achieving >20% Splicing Improvement
FREEDOM SAD Higher PGN-EDODM1 Doses Associated with Increased Number of Patients Achieving >20% Splicing Improvement4 >20% 40 Splicing Worsening 20 ±20% (% change from baseline) 0 Splicing Midrange Splice Index -20 -40 Placebo1 >20% 5 mg/kg SAD -60 Splicing Improvement 10 mg/kg SAD2 -80 -100 15 mg/k
Slide 18: PGN-EDODM1 Demonstrated >20% Splicing Correction in a Majority of Patients at ≥10 mg/kg
FREEDOM SAD PGN-EDODM1 Demonstrated >20% Splicing Correction in a Majority of DM1 Patients after a Single Dose ≥10 mg/kg ±20% >20% Splicing Midrange Correction 74% of Untreated Patients Splicing Improves (% Change from Baseline) Worsening Improvement +20% 0% −20% −50% −70% Placebo SAD: 5 mg/kg SAD:
Slide 19: Greater Than Dose-Proportional Increase in Muscle Tissue Concentration
FREEDOM SAD Robust, Greater Than Dose-Proportional Increase in Muscle Tissue Concentration Following Single Dose Muscle Tissue Concentration at Mean D28 TA muscle 300 Mean TA Muscle 160.6 Mean ± SE (ng/g) 160.6 Concentration (ng/g) 200 Concentration 100 44.1 44.1 13.7 13.7 0 5mg/kg 5 mg/kg 10mg/kg 1
Slide 20: PGN-EDODM1 Generally Well Tolerated, TEAEs Primarily Mild to Moderate
FREEDOM SAD PGN-EDODM1 Was Generally Well Tolerated, with TEAEs Primarily Mild to Moderate Across Dose Cohorts • Most frequent TEAEs: nausea, nasopharyngitis, Placebo Cohort 1 Cohort 2 Cohort 3 (n=6) 5 mg/kg 10 mg/kg 15 mg/kg Total and headache (n=24) N (events) (n=6) (n=6) (n=6) • No electrolyte-re
Slide 21: PGN-EDODM1 – FREEDOM2 MAD Trial in DM1
PGN-EDODM1 – FREEDOM2 MAD Trial in DM1
Slide 22: FREEDOM2: Phase 2 MAD Study Design
FREEDOM2 MAD FREEDOM2: Phase 2 MAD Study Design 4 Doses of PGN-EDODM1 or Placebo (randomized 3:1) DSMB Multinational, randomized, double- 12.5 mg/kg Safety Biopsy Biopsy or PBO FU blind, placebo-controlled, MAD study Recruiting n=8 open in Canada, UK, NZ, Australia 7d post final dose and South Korea
Slide 23: Favorable Emerging Safety Profile; No Increase in Toxicity with Multiple Doses at 5 mg/kg
FREEDOM2 MAD Favorable Emerging Safety Profile of PGN-EDODM1; No Increase in Toxicity with Multiple Doses at 5 mg/kg Summary of Treatment Emergent PGN-EDODM1 was Generally Well-Tolerated, 5 mg/kg (n=8) Adverse Events (TEAEs)1 n(%) with All AEs Mild or Moderate in Severity1 • All participants complet
Slide 24: FREEDOM2 5 mg/kg Splicing Correction
FREEDOM2 MAD FREEDOM2 5 mg/kg Splicing Correction 5 mg/kg Collective Splicing Data 5 mg/kg Individual Splicing Data Splicing FREEDOM2 5mg/kgAnalysis Mean splicing data FREEDOM2- 5mg/kg Individual Splicing data 20 100 Excluding (Mean % change from baseline ± SE) 10 80 70.8 notable splicing outlier %
Slide 25: FREEDOM2 5 mg/kg Myotonia (vHOT) Trends
FREEDOM2 MAD FREEDOM2 5 mg/kg Myotonia (vHOT): PGN-EDODM1 Shows Promising Middle Finger vHOT Trends at Lowest Dose vHOT Analysis 6 +2.5s 4 (52% CFB) • Excluding notable splicing 2 outlier, the active group remained below baseline (n=5) Improvement 0 • Splicing outlier demonstrated -2 22 sec differen
Slide 26: FREEDOM2 MAD at 10 & 12.5 mg/kg Potential to Build Upon Single-Dose Splicing Correction
FREEDOM SAD FREEDOM2 MAD at 10 & 12.5 mg/kg FREEDOM2 MAD has Potential to Build Upon Robust Single-Dose Splicing Correction ±20% >20% Splicing Midrange Correction 74% of Untreated Patients Splicing Improves (% Change from Baseline) Worsening Improvement +20% 0% −20% −50% −70% Placebo SAD 5 mg/kg SAD
Slide 27: Promising Safety, Splicing and vHOT Data in FREEDOM2 Lowest Dose
FREEDOM2 MAD Promising Safety, Splicing and vHOT Data in FREEDOM2 Lowest Dose – Supports Ongoing 10 mg/kg MAD Cohort SAFETY & • PGN-EDODM1 was generally well-tolerated; all AEs were mild or moderate in severity, with no SAEs or cumulative toxicity with repeat TOLERABILITY dosing observed SPLICING &
Slide 28: Summary of PGN-EDODM1 and FREEDOM Program
Summary of PGN-EDODM1 and FREEDOM Program 1 Differentiated Delivery Technology PHASE 2 FREEDOM2 MAD & OLE 2 Differentiated Target Promising Safety, Splicing and vHOT Data in FREEDOM2 Lowest Dose – Supports Ongoing 10 mg/kg MAD Cohort • Completed enrollment in the 10 mg/kg MAD cohort of FREEDOM STUDY
Slide 29: Thank you
' 9 Thank you