PepGen Announces Presentations at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference
PepGen (Nasdaq: PEPG) announced its upcoming participation at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference in Dallas, featuring two oral presentations and five posters. The presentations will showcase data from two significant clinical trials:
1. The CONNECT1-EDO51 Phase 2 study evaluating PGN-EDO51 for Duchenne muscular dystrophy (DMD)
2. The FREEDOM-DM1 Phase 1 study of PGN-EDODM1 for myotonic dystrophy type 1 (DM1)
The conference, scheduled for March 16-19, 2025, will include poster presentations on the Enhanced Delivery Oligonucleotide (EDO) Platform, nonclinical data for both drug candidates, and detailed information about both clinical trials. All presentations will be made available on PepGen's website after the conference.
PepGen (Nasdaq: PEPG) ha annunciato la sua prossima partecipazione alla Conferenza Clinica e Scientifica dell'Associazione Distrofia Muscolare 2025 a Dallas, con due presentazioni orali e cinque poster. Le presentazioni mostreranno dati provenienti da due importanti studi clinici:
1. Lo studio di fase 2 CONNECT1-EDO51 che valuta PGN-EDO51 per la distrofia muscolare di Duchenne (DMD)
2. Lo studio di fase 1 FREEDOM-DM1 di PGN-EDODM1 per la distrofia miotonica di tipo 1 (DM1)
La conferenza, programmata per il 16-19 marzo 2025, includerà presentazioni di poster sulla Piattaforma di Oligonucleotide a Consegna Potenziata (EDO), dati non clinici per entrambi i candidati farmaceutici e informazioni dettagliate su entrambi gli studi clinici. Tutte le presentazioni saranno rese disponibili sul sito web di PepGen dopo la conferenza.
PepGen (Nasdaq: PEPG) anunció su próxima participación en la Conferencia Clínica y Científica de la Asociación de Distrofia Muscular 2025 en Dallas, que contará con dos presentaciones orales y cinco carteles. Las presentaciones mostrarán datos de dos ensayos clínicos significativos:
1. El estudio de fase 2 CONNECT1-EDO51 que evalúa PGN-EDO51 para la distrofia muscular de Duchenne (DMD)
2. El estudio de fase 1 FREEDOM-DM1 de PGN-EDODM1 para la distrofia miotónica tipo 1 (DM1)
La conferencia, programada para el 16-19 de marzo de 2025, incluirá presentaciones de carteles sobre la Plataforma de Oligonucleótidos de Entrega Mejorada (EDO), datos no clínicos para ambos candidatos a fármacos e información detallada sobre ambos ensayos clínicos. Todas las presentaciones estarán disponibles en el sitio web de PepGen después de la conferencia.
PepGen (Nasdaq: PEPG)는 댈러스에서 열리는 2025 근육병협회 임상 및 과학 회의에 참여할 예정이며, 두 개의 구두 발표와 다섯 개의 포스터를 선보일 것입니다. 발표에서는 두 가지 중요한 임상 시험의 데이터를 보여줄 것입니다:
1. 듀셰네 근육병(DMD)을 위한 PGN-EDO51을 평가하는 CONNECT1-EDO51 2상 연구
2. 미오토닉 근육병 1형(DM1)을 위한 PGN-EDODM1의 1상 연구인 FREEDOM-DM1
2025년 3월 16일부터 19일까지 예정된 이 회의에서는 향상된 전달 올리고뉴클레오타이드(EDO) 플랫폼, 두 약물 후보에 대한 비임상 데이터 및 두 임상 시험에 대한 자세한 정보가 포함된 포스터 발표가 있을 것입니다. 모든 발표는 회의 후 PepGen의 웹사이트에서 제공될 예정입니다.
PepGen (Nasdaq: PEPG) a annoncé sa prochaine participation à la Conférence Clinique et Scientifique de l'Association de Dystrophie Musculaire 2025 à Dallas, avec deux présentations orales et cinq affiches. Les présentations mettront en avant des données de deux essais cliniques significatifs :
1. L'étude de phase 2 CONNECT1-EDO51 évaluant PGN-EDO51 pour la dystrophie musculaire de Duchenne (DMD)
2. L'étude de phase 1 FREEDOM-DM1 de PGN-EDODM1 pour la dystrophie myotonique de type 1 (DM1)
La conférence, prévue du 16 au 19 mars 2025, comprendra des présentations d'affiches sur la plateforme d'oligonucléotides à délivrance améliorée (EDO), des données non cliniques pour les deux candidats médicaments et des informations détaillées sur les deux essais cliniques. Toutes les présentations seront mises à disposition sur le site web de PepGen après la conférence.
PepGen (Nasdaq: PEPG) gab seine bevorstehende Teilnahme an der 2025 Konferenz der Muskulären Dystrophie Vereinigung für Klinische und Wissenschaftliche Forschung in Dallas bekannt, die zwei mündliche Präsentationen und fünf Poster umfasst. Die Präsentationen werden Daten aus zwei bedeutenden klinischen Studien zeigen:
1. Die CONNECT1-EDO51 Phase 2 Studie zur Bewertung von PGN-EDO51 bei der Duchenne-Muskeldystrophie (DMD)
2. Die FREEDOM-DM1 Phase 1 Studie zu PGN-EDODM1 bei der myotonen Dystrophie Typ 1 (DM1)
Die Konferenz, die vom 16. bis 19. März 2025 stattfindet, wird Posterpräsentationen zur Enhanced Delivery Oligonucleotide (EDO) Plattform, nichtklinische Daten für beide Arzneikandidaten und detaillierte Informationen zu beiden klinischen Studien umfassen. Alle Präsentationen werden nach der Konferenz auf der Website von PepGen verfügbar sein.
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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
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.
For more information, please visit PepGen.com. Follow PepGen on LinkedIn and X.
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Investor
Dave Borah, CFA
SVP, Investor Relations and Corporate Communications
dborah@pepgen.com
Media
Julia Deutsch
Lyra Strategic Advisory
Jdeutsch@lyraadvisory.com
Source: PepGen Inc.
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