PGN-EDO51 / PepGen - LARVOL DELTA

CONNECT1-EDO51: A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (clinicaltrials.gov) - P2 | N=7 | Terminated | Sponsor: PepGen Inc | Trial completion date: Apr 2027 ➔ Aug 2025 | Active, not recruiting ➔ Terminated | Trial primary completion date: Apr 2025 ➔ Aug 2025; PGN-EDO51 development terminated by Sponsor

Trial completion date • Trial primary completion date • Trial termination • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

CONNECT2-EDO51: A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - P2 | N=0 | Withdrawn | Sponsor: PepGen Inc | N=16 ➔ 0 | Trial completion date: Apr 2030 ➔ May 2025 | Suspended ➔ Withdrawn | Trial primary completion date: Apr 2028 ➔ May 2025

Enrollment change • Trial completion date • Trial primary completion date • Trial withdrawal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

CONNECT2-EDO51: A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - P2 | N=16 | Suspended | Sponsor: PepGen Inc | Trial completion date: Apr 2029 ➔ Apr 2030 | Recruiting ➔ Suspended | Trial primary completion date: Apr 2027 ➔ Apr 2028

Trial completion date • Trial primary completion date • Trial suspension • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular 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

CONNECT1-EDO51: A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (clinicaltrials.gov) - P2 | N=7 | Active, not recruiting | Sponsor: PepGen Inc | Recruiting ➔ Active, not recruiting | N=11 ➔ 7 | Trial completion date: Nov 2027 ➔ Apr 2027 | Trial primary completion date: Nov 2025 ➔ Apr 2025

Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

PepGen Announces Update to Phase 2 CONNECT2-EDO51 Study in Patients with DMD (Businesswire) - "PepGen Inc...announced its voluntary decision to temporarily pause the Phase 2 CONNECT2-EDO51 study of PGN-EDO51 in patients with Duchenne muscular dystrophy (DMD) until the Company can review results from the 10 mg/kg cohort in the ongoing Phase 2 CONNECT1-EDO51 study."

Trial status • Trial suspension • Duchenne Muscular Dystrophy

PepGen Reports Fourth Quarter and Year-End 2024 Financial Results and Recent Corporate Highlights (Businesswire) - "Phase 2 CONNECT1-EDO51 Open-Label MAD Clinical Trial of PGN-EDO51: The 10 mg/kg cohort is fully enrolled (n=4) and participants in the 5 mg/kg cohort (n=3) are continuing in the long-term extension phase of the study. The Company expects to report clinical data from the 10 mg/kg cohort in the third quarter of 2025."

P2 data • Duchenne Muscular Dystrophy

CONNECT2-EDO51: A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (clinicaltrials.gov) - P2 | N=16 | Recruiting | Sponsor: PepGen Inc

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

CONNECT1-EDO51: A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (clinicaltrials.gov) - P2 | N=11 | Recruiting | Sponsor: PepGen Inc | Trial completion date: May 2027 ➔ Nov 2027 | Trial primary completion date: Mar 2025 ➔ Nov 2025

Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Single- and Repeat-Dose Nonclinical Data for PGN-EDO51 Demonstrated Favorable Pharmacology and Safety Profiles for the Treatment of DMD (New Directions 2024) - No abstract available

Duchenne Muscular Dystrophy

CONNECT1-EDO51: A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (clinicaltrials.gov) - P2 | N=10 | Recruiting | Sponsor: PepGen Inc | Trial completion date: Mar 2025 ➔ May 2027

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

CONNECT1-EDO51 and CONNECT2-EDO51: Phase 2 Study Designs to Evaluate Safety and Efficacy for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (MDA 2024) - "CONNECT1-EDO51 is an open-label trial in Canada and CONNECT2-EDO51 is a multinational, randomized, placebo-controlled trial designed to support development of PGN-EDO51 for the treatment of DMD. Based on nonclinical data, monthly repeat PGN-EDO51 dosing may result in accumulation of skipped transcripts and dystrophin production, which would be anticipated to result in clinical efficacy."

Clinical • P2 data • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Three Novel Enhanced Delivery Oligonucleotide Candidates for Duchenne Muscular Dystrophy (DMD) Mediated High Levels of Exon 53, 45, and 44 Skipping (MDA 2024) - "Combined, the nonclinical data support further development of PGN-EDO53, PGN-EDO45, PGN-EDO44. These EDOs, along with PGN-EDO51 (another DMD candidate), have the potential, if successful, to address 35% of the DMD population amenable to exon skipping."

Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Single- and Repeat-Dose Nonclinical Data for PGN-EDO51 Demonstrated Potential for the Treatment of Duchenne Muscular Dystrophy (DMD) (MDA 2024) - "PGN-EDO51 was generally well tolerated at clinically relevant doses in GLP repeat-dose toxicology studies. Combined, the nonclinical data showed significant accumulation of exon skipping and dystrophin protein after repeat doses in the mdx mouse model and accumulation of exon skipped transcripts in monkeys. These data suggest repeat PGN-EDO51 dosing every 4 weeks may result in dystrophin accumulation, which may potentially support a clinically meaningful benefit in people with DMD amenable to exon 51 skipping."

Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

CONNECT1-EDO51: A Study Of PGN-EDO51 In Participants With Duchene Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (clinicaltrials.gov) - P2 | N=10 | Recruiting | Sponsor: PepGen Inc | Not yet recruiting ➔ Recruiting | Initiation date: Oct 2023 ➔ Jan 2024

Enrollment open • Trial initiation date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

CONNECT1-EDO51: A Study Of PGN-EDO51 In Participants With Duchene Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (clinicaltrials.gov) - P2 | N=10 | Not yet recruiting | Sponsor: PepGen Inc

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