Pepgen Inc. · NASDAQ:PEPG
Pepgen — Corporate Update (March 2026)
17 slides from Pepgen's corporate update, Mar 30, 2026
Slide 1: FREEDOM2-DM1 5 mg/kg MAD Cohort Data Update
March 2026 FREEDOM2-DM1 5 mg/kg MAD Cohort Data Update
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,” “on track,” “anticipates,” “believes,” “could,” “estimates,” “expects,”
Slide 3: Agenda
Agenda James McArthur, PhD President and Chief Executive Officer Key Takeaways, Platform, Closing Remarks, and Q&A Paul Streck, MD, MBA Head of R&D FREEDOM2 Clinical Trial Design, Clinical Data, and Q&A
Slide 4: Myotonic Dystrophy Type 1 Overview and EDO Platform
Myotonic Dystrophy Type 1 Overview and EDO Platform
Slide 5: Safety and efficacy results are supportive of the ongoing dosing in 10 mg/kg MAD cohort
Safety and efficacy results are supportive of the ongoing dosing in 10 mg/kg MAD cohort PGN-EDODM1 5 mg/kg MAD Lowest Dose Demonstrates Promising Safety, Splicing and vHOT Company on track to report clinical data from 10 mg/kg multiple dose cohort in 2H 2026 Data cutoff date: 04 March 2026 AE: Adver
Slide 6: DM1 is Caused by Pathogenic CUG Repeats in DMPK RNA
DM1 is Caused by Pathogenic CUG Repeats in DMPK RNA DM1 is caused by pathogenic DMPK transcripts Approximately 50% of DMPK transcripts are pathogenic while the remaining DMPK transcripts are normal1 Pathogenic DMPK transcripts containing cytosine-uracil-guanine (CUG) repeat sequences form hairpin lo
Slide 7: PGN-EDODM1 Blocking Approach Targets Only the Pathogenic DMPK RNA
PGN-EDODM1 Blocking Approach Targets Only the Pathogenic DMPK RNA PGN-EDODM1 is engineered to bind selectively to the pathogenic CUG repeat expansion present in DMPK transcript This reduces the ability of these CUG repeats to form hairpin loops and sequester RNA splicing proteins, including MBNL1, i
Slide 8: FREEDOM SAD Study: Dose-Dependent Best-in-Class Splicing Correction Following Single Dose
FREEDOM SAD Study: PGN-EDODM1 Produced Dose-Dependent Best-in-Class Splicing Correction Following Single Dose 1.Missing samples due to unavailability of biopsy tissue or sample outside of assay window. 2.One subject at 10 mg/kg biopsy was not collected at day 28 due to pseudoaneurysm in connection w
Slide 9: FREEDOM2-DM1 5 mg/kg MAD Cohort Overview and Results
FREEDOM2-DM1 5 mg/kg MAD Cohort Overview and Results
Slide 10: FREEDOM2 Phase 2 MAD Study Design
FREEDOM2 Phase 2 MAD Study Design DSMB: data safety monitoring board; FU: follow-up; IV: intravenous; MAD: multiple-ascending dose; PBO: placebo; PK: pharmacokinetics; vHOT: video hand opening test; OLE: open label extension * Dose dependent on recommendations of DSMB **The U.S. FDA recently placed
Slide 11: FREEDOM2: Demographics and Baseline Characteristics for 5 mg/kg MAD Cohort
5 mg/kg (n=8) Mean (SD) or n (%) Age (years) 32.5 (6.0) Female, n (%) 2 (25.0) BMI (kg/m2) 24.8 (5.0) Splicing Index 69.2 (17.3) vHOT – mean, middle finger (sec) 10.3 (8.3) CTG Repeats 603 (301) FREEDOM2: Demographics and Baseline Characteristics for 5 mg/kg MAD Cohort
Slide 12: Favorable Emerging Safety Profile of PGN-EDODM1; No Increase in Toxicity with Multiple Doses
Any TEAE 7 (87.5) Mild Moderate Severe 4 (50.0) 3 (37.5) 0 (0.0) Any SAE 0 Any related SAE 0 Any AESI or dose-limiting toxicities 0 Any TEAE leading to study withdrawal 0 Any TEAE leading to death 0 Favorable Emerging Safety Profile of PGN-EDODM1; No Increase in Toxicity with Multiple Doses Summary
Slide 13: FREEDOM2 5 mg/kg Splicing Correction
FREEDOM2 5 mg/kg Splicing Correction* - Mean splicing correction of 7.3% with PGN-EDODM1 (n=6) - Excluding notable splicing outlier, mean splicing correction of 22.9% (n=5) *Data cutoff date: 04 March 2026 **One patient’s muscle tissue concentration reading was pending at cutoff date. Splicing Anal
Slide 14: FREEDOM2 5 mg/kg Myotonia (vHOT): Promising Middle Finger vHOT Trends at Lowest Dose
FREEDOM2 5 mg/kg Myotonia (vHOT): PGN-EDODM1 Shows Promising Middle Finger vHOT Trends at Lowest Dose Improvement Improvement CFB: Change from baseline; SE: Standard error Data cutoff date: 04 March 2026 +2.5s (52% CFB) -2.95s (23% CFB) +2.5s (52% CFB) -3.4s (33% CFB) vHOT Analysis Excluding Splicin
Slide 15: Promising Safety, Splicing and vHOT Data in FREEDOM2 Lowest Dose
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 mild-to-moderate, no SAEs, and no signs of cumulative toxicity No kidney related TEAEs SPLICING & vHOT: Strong splicing correction at lowe
Slide 16: Question and Answer Session
Question and Answer Session James McArthur, PhD President and Chief Executive Officer Paul Streck, MD, MBA Head of R&D Noel Donnelly, MBA Chief Financial Officer
Slide 17: Thank you
@ Thank you