PGN-EDODM1 / PepGen - LARVOL DELTA

An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE) (clinicaltrials.gov) - P2 | N=48 | Not yet recruiting | Sponsor: PepGen Inc

New P2 trial • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy

FREEDOM-DM1: Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (clinicaltrials.gov) - P1 | N=24 | Active, not recruiting | Sponsor: PepGen Inc | Trial primary completion date: Jun 2025 ➔ Oct 2025 | Recruiting ➔ Active, not recruiting

Enrollment closed • Trial primary completion date • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy

FREEDOM-DM1: Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (clinicaltrials.gov) - P1 | N=32 | Recruiting | Sponsor: PepGen Inc | Trial completion date: Apr 2025 ➔ Oct 2025

Trial completion date • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy

PepGen Announces Presentations at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference (Businesswire) - "PepGen Inc...today announced that the Company will be presenting two oral presentations as well as five posters at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, being held March 16-19, 2025 in Dallas, TX. The oral presentations feature data from the ongoing CONNECT1-EDO51 clinical trial in Duchenne muscular dystrophy (DMD) and from the ongoing FREEDOM-DM1 clinical trial in myotonic dystrophy type 1 (DM1), which the Company recently announced."

P1 data • P2 data • Preclinical • Duchenne Muscular Dystrophy • Myotonic Dystrophy

PepGen Announces Positive Initial Results, Including Robust Splicing Correction, from Ongoing FREEDOM-DM1 Trial in Patients with DM1 (Businesswire) - P1 | N=32 | FREEDOM-DM1 (NCT06204809) | Sponsor: PepGen Inc | "Mean splicing correction from evaluable participants was 12.3% and 29.1% at 5 mg/kg (n=6) and at 10 mg/kg (n=4)1,2, respectively, as measured by the 22-gene panel3 at 28 days post-dosing. Dose-dependent increase in muscle tissue concentrations of PGN-EDODM1 was observed at 5 mg/kg (n=6) and at 10 mg/kg (n=5)1 at day 28. While single-dose studies have not demonstrated improved functional outcomes in DM1 patients, the Company collected data from these cohorts and believes these data showed positive early trends in some functional outcome measures...There were no adverse events related to electrolytes or renal biomarkers. Most of the treatment emergent adverse events were mild or moderate in severity...The Company expects to report results from the FREEDOM 15 mg/kg cohort in the second half of 2025 and from the FREEDOM2 5 mg/kg cohort in the first quarter of 2026."

P1 data • P2 data • Myotonic Dystrophy

FREEDOM2-DM1: A Clinical Study of PGN-EDODM1 in People with Myotonic Dystrophy Type 1 (clinicaltrials.gov) - P2 | N=24 | Recruiting | Sponsor: PepGen Inc | Not yet recruiting ➔ Recruiting

Enrollment open • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy

FREEDOM2-DM1: A Clinical Study of PGN-EDODM1 in People with Myotonic Dystrophy Type 1 (clinicaltrials.gov) - P2 | N=24 | Not yet recruiting | Sponsor: PepGen Inc

New P2 trial • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy

FREEDOM-DM1: Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants with Myotonic Dystrophy Type 1 (clinicaltrials.gov) - P1 | N=32 | Recruiting | Sponsor: PepGen Inc | N=24 ➔ 32

Enrollment change • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy

Nonclinical Data for PGN-EDODM1 Demonstrated Nuclear Delivery, Mechanistic and Meaningful Activity for the Potential Treatment of DM1 (New Directions 2024) - No abstract available

PepGen Receives U.S. FDA Fast Track Designation for PGN-EDODM1 for the Treatment of Myotonic Dystrophy Type 1 (GlobeNewswire) - "PepGen Inc...announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to PGN-EDODM1, an investigational candidate for the treatment of myotonic dystrophy type 1 (DM1)....PGN-EDODM1 is currently being evaluated in the ongoing FREEDOM-DM1 Phase 1 clinical trial for the treatment of people living with DM1. The Company expects to report initial data from the trial in 2024."

Fast track designation • P1 data • CNS Disorders • Muscular Atrophy • Myotonic Dystrophy

PGN-EDODM1 Single- and Repeat-Dose Nonclinical Data Indicated Mechanistic and Meaningful Activity for Potential Treatment of Myotonic Dystrophy Type 1 (MDA 2024) - "Currently, there are no approved therapies for DM1. Nonclinical pharmacology studies with PGN-EDODM1 showed considerable therapeutic potential on splicing and myotonia correction. Nonclinical data warrants clinical evaluation in the Phase 1 single-ascending dose study FREEDOM-DM1 in adults with DM1."

Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy

FREEDOM-DM1: Phase 1 Study Design to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 for Myotonic Dystrophy Type 1 (MDA 2024) - "The Phase 1 study FREEDOM-DM1 will inform continued development of PGN-EDODM1 targeting the central cause of DM1."

Clinical • P1 data • PK/PD data • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy

FREEDOM-DM1: Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (clinicaltrials.gov) - P1 | N=24 | Recruiting | Sponsor: PepGen Inc

New P1 trial • Genetic Disorders • Muscular Dystrophy • Myotonic Dystrophy