Pepgen Inc. · NASDAQ:PEPG
Pepgen — Corporate Update (September 2022)
24 slides from Pepgen's corporate update, Sep 28, 2022
Slide 1: PepGen Reports Positive Data from Phase 1 Trial of PGN-EDO51 for DMD
PEPGEN REPORTS POSITIVE DATA FROM PHASE 1 TRIAL OF PGN-EDO51 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY James McArthur, Ph.D. Chief Executive Officer SEPTEMBER 28, 2022
Slide 2: Disclaimers
DISCLAIMERS This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “inte
Slide 3: Positive Data from PGN-EDO51 Ph1 Single Ascending Dose Trial
POSITIVE DATA FROM PGN-EDO51 PH1 SINGLE ASCENDING DOSE TRIAL *Comparative statements are based on cross-trial comparisons with publicly-available data for other exon skipping approaches that have been assessed following a single dose. PGN-EDO51 Ph1 HNV trial overview A double-blind, placebo-controll
Slide 4: PGN-EDO51: An Enhanced Delivery Oligonucleotide
PGN-EDO51: AN ENHANCED DELIVERY OLIGONUCLEOTIDE Efficient cellular uptake of oligo, including in cardiac and skeletal tissue PEPGEN’S ENHANCED DELIVERY PEPTIDES Next-generation delivery peptides; engineered with the goal of offering enhanced activity and improved tolerability EXON 51-SKIPPING OLIGON
Slide 5: PGN-EDO51 for Duchenne Muscular Dystrophy
PGN-EDO51 FOR DUCHENNE MUSCULAR DYSTROPHY Michelle Mellion, M.D. SVP Clinical Development
Slide 6: Completed Single Ascending Dose Ph1 Trial in Healthy Normal Volunteers
WE HAVE COMPLETED A SINGLE ASCENDING DOSE PH1 TRIAL OF PGN-EDO51 IN HEALTHY NORMAL VOLUNTEERS PH1 HEALTHY NORMAL VOLUNTEER (HNV) TRIAL SUMMARY Overview Study population: Healthy adult males (n = 32, 3:1 PGN-EDO51:placebo) Dosing: Single dose, i.v. administration Placebo control Biceps biopsies condu
Slide 7: Highest Levels of Oligo Delivery & Exon Skipping Observed
HIGHEST LEVELS OF OLIGO DELIVERY & EXON 51 SKIPPING OBSERVED, SUPPORTING FURTHER DEVELOPMENT OF PGN-EDO51 PH1 ENDPOINTS READOUTS OBTAINED Safety & tolerability Pharmacokinetics Tissue concentration (biceps) Target engagement Exon skipping (biceps) Generally well-tolerated Highest level of oligonucle
Slide 8: Highest Levels of Exon Skipping Observed in Humans Following a Single Dose
HIGHEST LEVELS OF EXON 51 SKIPPING OBSERVED IN HUMANS FOLLOWING A SINGLE DOSE Protocol PGN-EDO51-101: Phase 1, first in human, randomized double blind, placebo controlled single ascending dose study in healthy adult volunteers. Single dose of either PGN-EDO51 or Placebo were administered by IV infus
Slide 9: High, Persistent Tissue Concentrations of Oligonucleotide Observed
HIGH, PERSISTENT TISSUE CONCENTRATIONS OF OLIGONUCLEOTIDE WERE OBSERVED Protocol PGN-EDO51-101: Phase 1, first in human, randomized double blind, placebo controlled single ascending dose study in healthy adult volunteers. Single dose of either PGN-EDO51 or Placebo were administered by IV infusion at
Slide 10: Safety & Tolerability Summary
SAFETY & TOLERABILITY SUMMARY PGN-EDO51 WAS GENERALLY WELL-TOLERATED AT DOSES ASSESSED IN PH1 SAD TRIAL All participants completed the study; there were no discontinuations. The majority of treatment-emergent adverse events (TEAEs) were assessed as mild and resolved without any intervention. At 10 m
Slide 11: Majority of TEAEs Mild and Resolved Without Intervention
MAJORITY OF TEAEs MILD AND RESOLVED WITHOUT INTERVENTION; SUPPORTS PROGRESSION TO PH2a PATIENT TRIAL * No Grade 4 or 5 recorded; Protocol PGN-EDO51-101: Phase 1, first in human, randomized double blind, placebo controlled single ascending dose study in healthy adult volunteers. Single dose of either
Slide 12: NHP Repeat Dose Study: Kidney Biomarker Elevations Reduced After First Dose
IN NHP REPEAT DOSE STUDY, KIDNEY BIOMARKER ELEVATIONS WERE REDUCED AFTER FIRST DOSE OF PGN-EDO51 PD = pre-dose. Protocol: PGN-EDO51 was administered to NHP by IV infusion over 30 min at a given dose level (n=3). Q2W, three doses administered, saline control. Tissues were harvested 7 days after final
Slide 13: Completed Ph1 HNV Trial; On Track to Initiate DMD Patient Trial in 1H23
PEPGEN HAS COMPLETED A PH1 HNV TRIAL FOR PGN-EDO51; ON TRACK TO INITIATE DMD PATIENT CLINICAL TRIAL IN 1H23 2022 2023 2024 Anticipated milestones 2Q: First HNV dosed in Ph1 trial 3Q: Ph1 clinical safety, oligo delivery & exon skipping data 4Q: Completion of Ph2a-enabling tox studies 1H: Initiation o
Slide 14: PGN-EDODM1 for Myotonic Dystrophy Type 1
PGN-EDODM1 FOR MYOTONIC DYSTROPHY TYPE 1 (DM1) Jaya Goyal, Ph.D. EVP Research & Preclinical Development
Slide 15: PGN-EDODM1: An Enhanced Delivery Oligonucleotide
PGN-EDODM1: AN ENHANCED DELIVERY OLIGONUCLEOTIDE Efficient cellular uptake of oligo, including in cardiac and skeletal tissue and the CNS PEPGEN’S ENHANCED DELIVERY PEPTIDES Next-generation delivery peptides; engineered with the goal of offering enhanced activity and improved tolerability STERIC BLO
Slide 16: PGN-EDODM1 Corrected Molecular and Functional DM1 Phenotype in HSALR Mouse
PGN-EDODM1 CORRECTED MOLECULAR AND FUNCTIONAL DM1 PHENOTYPE IN HSALR MOUSE Protocol: PGN-EDODM1 was administered IV to HSALR mice at doses (mg/kg): 10, 20, 30 (n=8), 50 (n=4) against a saline control (n=16) and wild-type (WT) saline control (n=8). Myotonia assessed, tissues harvested 2 weeks post-ad
Slide 17: Human PGN-EDO51 Tissue Concentrations Comparable to HSALR Mouse Model
HUMAN PGN-EDO51 TISSUE CONCENTRATIONS WERE COMPARABLE TO THOSE ACHIEVED IN HSALR MOUSE MODEL Protocol: PGN-EDODM1 was administered IV to HSALR mice at doses (mg/kg): 10, 20, 30 (n=8), 50 (n=4) against a wild-type (WT) saline control (n=8). Tissue concentration assessed by HPLC. Robust mis-splicing c
Slide 18: On Track to Initiate a DM1 Patient Clinical Trial in 1H23
PEPGEN IS ON TRACK TO INITIATE A DM1 PATIENT CLINICAL TRIAL IN 1H23 2022 2023 2024 Anticipated milestones 2Q: NHP dose range-finding study 2H: IND-enabling studies 1H: Initiation of Ph1/2 DM1 patient clinical trial Safety and splicing data in DM1 patients (Ph1/2) Overview We believe oligonucleotide
Slide 19: Conclusion
CONCLUSION James McArthur, Ph.D. Chief Executive Officer
Slide 20: Scalable EDO Technology Designed to Enable Broad Portfolio
SCALABLE EDO TECHNOLOGY DESIGNED TO ENABLE BROAD PORTFOLIO PROGRAM INDICATION TARGET DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 NEXT ANTICIPATED MILESTONE PGN-EDO51 Duchenne muscular dystrophy Exon 51 1H23 Ph2a patient clinical trial initiation PGN-EDODM1 Myotonic dystrophy type 1 DMPK 1H23 Ph1/2
Slide 21: We Are Committed to Serving the DMD Community
WE ARE COMMITTED TO SERVING THE DMD COMMUNITY Source: https://www.cureduchenne.org/cure/exon-skipping/. Exon 53: PGN-EDO53 Next anticipated milestone: NHP exon skipping data (2H22) Exon 45: PGN-EDO45 Next anticipated milestone: Candidate nomination (2H22) DMD EXON SKIPPING PATIENT SUB-POPULATIONS Pe
Slide 22: The Future of PepGen
THE FUTURE OF PEPGEN PGN-EDO51 (DMD exon 51) Ph1 HNV trial showed: Highest level of single-dose exon skipping and oligo delivery observed in a clinical trial* PGN-EDO51 was generally well-tolerated Anticipate initiation of patient clinical trials for DMD & DM1 Anticipate clinical POC in two indicati
Slide 23: Q&A
Q&A
Slide 24: Thank You
THANK YOU