Slide 1: PGN-EDODM1 in Development for Myotonic Dystrophy Type 1
PGN-EDODM1 in Development for Myotonic Dystrophy Type 1 Jenny Shoskes, PharmD Associate Director, Clinical Development MDF Conference, May 2-3, 2025
Slide 2: PepGen is committed to transforming the lives of people with neuromuscular disorders
PepGen is committed to transforming the lives of people with severe neuromuscular and neurological disorders with our Enhanced Delivery Oligonucleotides (EDOs) Jubal and family 2
Slide 3: What is an Enhanced Delivery Oligonucleotide?
What is an Enhanced Delivery Oligonucleotide? Enhanced Delivery Oligonucleotide EDO peptide A peptide is a small piece of a protein EDO peptides are designed to deliver the active part of the drug into the cell PGN-EDODM1 Oligonucleotide An oligonucleotide is a small piece of genetic material This i
Slide 4: PGN-EDODM1 Mechanism of Action - Approach in DM1
PGN-EDODM1 Mechanism of Action - Approach in DM1 DM1 DM1 + PGN-EDODM1 Mutation makes tangles that MBNL1 – Proofreader to accurately stop use of the proofreader read the code PGN-EDODM1 disrupts the tangles, allows the proofreader to work MBNL1 1. 1. 2. 2. Abnormal splicing - Normal splicing- Proofre
Slide 5: The DM1 Community Helps to Inform our Clinical Trials
The DM1 Community Helps to Inform our Clinical Trials Sharing Advising us on Guiding us on Helping us perspectives the preferred which understand on inclusion order of the symptoms ways to and assessments matter most to reduce travel exclusion and time in them and planning criteria clinic to help bu
Slide 6: Overview of FREEDOM-DM1 and FREEDOM2-DM1 Trials
Overview of FREEDOM-DM1 and FREEDOM2-DM1 Trials Open in US, Canada and UK Multinational Study Initiated • Phase 1 Randomized, double-blind, placebo- • Phase 2 randomized, double-blind, placebo- controlled single ascending dose study in people controlled multiple ascending dose study in people with w
Slide 7: Select Inclusion and Exclusion Criteria for FREEDOM and FREEDOM2
Select Inclusion and Exclusion Criteria for FREEDOM and FREEDOM2 Inclusion Criteria: Exclusion Criteria: • Ages 18-60 (FREEDOM), 16-60 • Congenital DM1 (FREEDOM2) • Known history or presence of any clinically • Confirmed genetic testing of DM1 significant conditions that may interfere with study saf
Slide 8: FREEDOM-DM1 Information
FREEDOM-DM1 Information • Phase 1 study to explore if a single intravenous dose of the investigational drug, PGN-EDODM1, is safe and tolerable for people with DM1 compared to a placebo (a substance that has no active drug). • Planning to enroll 32 people with DM1 in US, Canada, and UK • Randomized 3
Slide 9: PGN-EDODM1 Selectively Targets Only Pathogenic DMPK to Correct RNA Mis-Splicing
PGN-EDODM1 Selectively Targets Only Pathogenic DMPK to Correct RNA Mis-Splicing Dose-dependent Dose-dependent Favorable increases in increase in drug emerging tissue evaluable people2 in safety profile1 mean splicing concentration correction following in people with observed in first two single dose
Slide 10: FREEDOM2-DM1 Information
FREEDOM2-DM1 Information • Phase 2 study to explore if multiple, intravenous doses of PGN- EDODM1 every 4 weeks is safe and tolerable for people with DM1 compared to a placebo (a substance that has no active drug) at different dose levels • Planning to enroll 24 people with DM1. Currently open in Ca
Slide 11: Enrolling FREEDOM-DM1 and FREEDOM2-DM1 Sites
Enrolling FREEDOM-DM1 and FREEDOM2-DM1 Sites Canada • CIUSSS du Saguenay-Lac-Saint-Jean* – Dr. Jean-Denis Brisson • University of Ottawa* – Dr. Hanns Lochmuller • University of Calgary – Dr. Gerald Pfeffer United States • Stanford University – Dr. Jacinda Sampson • University of California-Irvine –
Slide 12: Thank You and To Learn More
Thank You and To Learn More Clinical study Community Clinical site participants and and clinical staff and researchers and their families advisors investigators www.freedom-dm1.com www.freedom-dm2.com clinicaltrials.gov: NCT06204809 clinicaltrials.gov: NCT06667453 clinicaltrials@pepgen.com 12 Genera