BOSTON, March 10, 2025--(BUSINESS WIRE)--PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, 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.
Oral Presentations
Title: CONNECT1-EDO51: A 12-Week Open-Label Phase 2 Study to Evaluate PGN-EDO51 Safety and Efficacy in People with Duchenne Amenable to Exon 51 Skipping Session: Clinical Trial Updates Date & Time: Wednesday, March 19th at 8:45-9:00 a.m. CT Presenter: Paul Streck, MD, MBA, Executive Vice President, R&D
Title:FREEDOM-DM1: A Phase 1, Placebo-Controlled Single Ascending Dose Study to Evaluate PGN-EDODM1 in People with Myotonic Dystrophy Type 1 (DM1) Session: Clinical Trial Updates Date & Time: Wednesday, March 19th at 12:45-1:00 p.m. CT Presenter: Jane Larkindale, DPhil, Vice President, Clinical Science
Poster Presentations
Posters will be displayed beginning at 6:00-8:00 p.m. CT on Sunday, March 16th through Tuesday, March 18th in the conference exhibit hall.
Title: Mechanistic Characterization of Enhanced Delivery Oligonucleotide (EDO) Platform Poster Number: #P21 Presenter: Jane Larkindale, DPhil, Vice President, Clinical Science
Title: Nonclinical Data for PGN-EDODM1 Demonstrated Mechanistic and Meaningful Activity for the Potential Treatment of DM1 Poster Number: #P48 Presenter: Ashling Holland, PhD, Director, Research & Preclinical Development
Title: Single- and Repeat-Dose Nonclinical Data for PGN-EDO51 Demonstrated Favorable Pharmacology and Safety Profiles for the Treatment of DMD Poster Number: #P49 Presenter: Ashling Holland, PhD, Director, Research & Preclinical Development
Title: FREEDOM-DM1: A Phase 1, Placebo-Controlled Single Ascending Dose Study to Evaluate PGN-EDODM1 in People with Myotonic Dystrophy Type 1 (DM1) Poster Number: #O45 Presenter: Jennifer Shoskes, PharmD, Associate Director, Clinical Development
Title: CONNECT1-EDO51: A 12-Week Open-Label Phase 2 Study to Evaluate PGN-EDO51 Safety and Efficacy in People with Duchenne Amenable to Exon 51 Skipping Poster Number: #O74 Presenter: Bassem Morcos, MD, Medical Director, Clinical Development
Following the conference, the presentations presented at the MDA Clinical & Scientific Conference will be available on the Investors page of PepGen’s website under Scientific Publications.
About PGN-EDODM1
PGN-EDODM1, PepGen's investigational candidate in development for the treatment of DM1, utilizes the Company's proprietary EDO technology to deliver a therapeutic oligonucleotide that is designed to restore the normal splicing function of MBNL1, a key RNA splicing protein. PGN-EDODM1 is designed to directly address the deleterious effects of cytosine-uracil-guanine (CUG) repeat expansion in the DMPK transcripts which sequester MBNL1, by binding to the pathogenic CUG trinucleotide repeat expansion present in the DMPK transcripts, disrupting the binding between the CUG repeat expansion and MBNL1. We believe this mechanism will allow the DMPK transcripts to continue performing its normal function within the cell, while also liberating MBNL1 to correct downstream mis-splicing events. We believe that this innovative therapeutic approach has considerable advantages over oligonucleotide modalities that rely on knockdown or degradation of the DMPK transcripts. The FDA has granted PGN-EDODM1 both Orphan Drug and Fast Track Designations for the treatment of patients with DM1.
About PGN-EDO51
PGN-EDO51, PepGen's investigational candidate in development for the treatment of DMD, utilizes the Company's proprietary EDO technology to deliver a therapeutic oligonucleotide that is designed to target the root cause of this devastating disease. PGN-EDO51 is designed to skip exon 51 of the dystrophin transcript, an established therapeutic target for approximately 13% of DMD patients, thereby aiming to restore the open reading frame and enabling the production of a truncated, yet functional dystrophin protein. The FDA has granted PGN-EDO51 both Orphan Drug and Rare Pediatric Disease Designations for the treatment of patients with DMD amenable to an exon-51 skipping approach.
About PepGen
PepGen is a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen’s EDO platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, we are generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases.
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