Pepgen Inc. · NASDAQ:PEPG
Pepgen — Investor Presentation (March 2023)
63 slides from Pepgen's investor presentation, Mar 27, 2023
Slide 1: Empowering Oligonucleotide Therapeutics: Company Presentation March 2023
EMPOWERING OLIGONUCLEOTIDE THERAPEUTICS COMPANY PRESENTATION MARCH 2023
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: PepGen's EDO Technology Addresses Delivery Challenges Limiting Oligonucleotide Therapeutics
PEPGEN’S EDO TECHNOLOGY IS DESIGNED TO ADDRESS THE DELIVERY CHALLENGES THAT LIMIT OLIGONUCLEOTIDE THERAPEUTICS THE CHALLENGE THE EDO SOLUTION Unconjugated oligonucleotides are not readily distributed to muscle, and are not efficiently taken up into cells and the nucleus Our EDO platform is engineere
Slide 4: Delivery Platform Has the Power to Unlock Oligonucleotide Potential
EXONDYS 51® (eteplirsen) – Sarepta Therapeutics Approved 2016, 2022 sales: $512M**** WE BELIEVE THAT OUR DELIVERY PLATFORM HAS THE POWER TO UNLOCK THE THERAPEUTIC POTENTIAL OF OLIGONUCLEOTIDES * Clinical data included in drug label (FDA). **Source: Sarepta MOMENTUM study update, 20 and 30 mg/kg coho
Slide 5: The Power of EDOs: Enhanced Delivery Oligonucleotides
ENHANCED DELIVERY OLIGONUCLEOTIDES Efficient cellular uptake of oligos 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 THERAPEUTIC OLIGONUCLEOTIDE Genetic
Slide 6: A Next-Generation Oligonucleotide Delivery Platform to Transform Patient Outcomes
Our Enhanced Delivery Oligonucleotide (EDO) platform is engineered to offer enhanced therapeutic activity and improved tolerability, with greater skeletal, diaphragm and cardiac muscle penetrance A NEXT-GENERATION OLIGONUCLEOTIDE DELIVERY PLATFORM WITH THE POTENTIAL TO TRANSFORM PATIENT OUTCOMES DMD
Slide 7: 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 REGISTRA-TIONAL* PGN-EDO51 Duchenne muscular dystrophy Exon 51 PGN-EDODM1 Myotonic dystrophy type 1 DMPK PGN-EDO53 Duchenne muscular dystrophy Exon 53 PGN-EDO45 Duchenne muscula
Slide 8: Anticipate Initiating Three Patient Clinical Trials in 2023, Readouts in 2024
ANTICIPATE INITIATING THREE PATIENT CLINICAL TRIALS IN 2023, WITH CLINICAL READOUTS EXPECTED IN 2024 Clinical plans are subject to alignment with regulatory authorities CONNECT1-EDO51: Ph2 open-label MAD study in patients (planned initiation 1H23) Initial dystrophin, exon skipping and safety data an
Slide 9: PepGen: Experienced Team of Company Builders, Scientists, and Clinicians
NOEL DONNELLY (CFO) PEPGEN: EXPERIENCED TEAM OF COMPANY BUILDERS, SCIENTISTS, AND CLINICIANS JAMES MCARTHUR, PhD (CEO & President) JOSH RESNICK, MD (Director) HEIDI HENSON (Director) Management team Board of Directors* * Plus Dr. McArthur (Director). NIELS SVENSTRUP, PhD (SVP Chemistry & Manufacturi
Slide 10: PGN-EDO51 for Duchenne Muscular Dystrophy
PGN-EDO51 FOR DUCHENNE MUSCULAR DYSTROPHY
Slide 11: Duchenne Muscular Dystrophy Is a Debilitating, Progressive Muscle-Wasting Disease
ROOT CAUSE OF DISEASE EXON 51 PATIENT POPULATION* EXON 51 THERAPEUTIC LANDSCAPE PEPGEN’S TREATMENT APPROACH Caused by mutations in the dystrophin gene Absence of dystrophin leads to muscle degeneration ~2,000 (US) ~3,200 (EEA) ~700 (JP) Exondys51® approved in US on the basis of <1% dystrophin restor
Slide 12: Absence of Dystrophin Drives the Pathologies Observed in DMD
AN ABSENCE OF THE DYSTROPHIN PROTEIN DRIVES THE PATHOLOGIES OBSERVED IN PEOPLE WITH DMD Acts as a shock absorber to protect muscle cells from mechanical stress DMD patients produce little or no dystrophin In the absence of this critical protein, muscles cells are no longer protected from contractile
Slide 13: PGN-EDO51 Engineered to Transform Treatment of DMD Amenable to Exon 51 Skipping
PGN-EDO51 WAS ENGINEERED TO TRANSFORM THE TREATMENT OF DMD AMENABLE TO EXON 51 SKIPPING * 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 in humans, and on both head-to-head an
Slide 14: Preclinical Data
PRECLINICAL DATA
Slide 15: EDO Platform Activity in DMD Evaluated in Multiple Preclinical Models
THE ACTIVITY OF OUR EDO PLATFORM IN DMD HAS BEEN EVALUATED IN MULTIPLE PRECLINICAL MODELS Species Key readouts observed Non-GLP pharmacology studies Patient cells PGN-EDO51 DMD patient High levels of exon 51 skipping Non-GLP pharmacology studies Single & repeat dose PGN-EDO23 mdx Normalization of se
Slide 16: mdx Mice: Single Dose of PGN-EDO23 Normalized Creatine Kinase
MDX MICE: A SINGLE DOSE OF PGN-EDO23 WAS OBSERVED TO NORMALIZE CREATINE KINASE, A MARKER OF MUSCLE DAMAGE Protocol: peptide-PMO conjugate and a saline control were administered intravenously (IV) to mdx and WT mice; serum creatine kinase measured 7 days after injection. Mean ± SD; **** = p≤0.0001; n
Slide 17: mdx Mice: Significant Increase in Dystrophin with Repeat Dosing
EXON SKIPPING DYSTROPHIN Biceps, 30 mg/kg, Q4W Biceps, 30 mg/kg, Q4W MDX MICE: SIGNIFICANT INCREASE IN DYSTROPHIN OBSERVED WITH REPEAT DOSING Protocol: mdx mice were dosed 2, 3 or 4 doses, with 4-week intervals between doses. Tissue samples were collected 4 weeks post-each dose at time points indica
Slide 18: NHP: Higher Single Dose Exon Skipping for PGN-EDO51 vs R6G-PMO
NHP: MARKEDLY HIGHER SINGLE DOSE EXON SKIPPING LEVELS OBSERVED FOR PGN-EDO51 COMPARED TO R6G-PMO Protocol: PGN-EDO51 and R6G-PMO were administered to NHP by IV infusion over 30 min at the dose indicated (n=3). Biopsies taken 7 days post single dose. Study not powered for statistical significance. Da
Slide 19: NHP: Q2W Repeat Dose Exon Skipping Levels Above 70%
73.4% NHP: Q2W REPEAT DOSE EXON SKIPPING LEVELS OF >70% OBSERVED IN SKELETAL MUSCLES AT 30 MG/KG Protocol: PGN-EDO51 and R6G-PMO were administered to NHP by IV infusion over 30 min at the doses indicated (n=3). Q2W, three doses administered, saline control. Tissues were harvested 7 days after final
Slide 20: NHP: Exon Skipping Accumulated with Q4W Repeat Dose
RT-PCR ddPCR (droplet digital PCR) NHP: EXON SKIPPING LEVELS ACCUMULATED WITH Q4W REPEAT DOSE ADMINISTRATION OF PGN-EDO51 BY BOTH RT-PCR AND ddPCR NHP protocol: Single (30 min) or repeat (60 min) IV doses with PGN-EDO51 were performed in male NHP. For repeat evaluation, NHP received 4 doses with 4-w
Slide 21: Ph1 Clinical Data
PH 1 CLINICAL DATA
Slide 22: HV: Completed Single Ascending Dose Ph1 Trial of PGN-EDO51
HV: WE HAVE COMPLETED A SINGLE ASCENDING DOSE PH1 TRIAL OF PGN-EDO51 IN HEALTHY NORMAL VOLUNTEERS PH1 HEALTHY VOLUNTEER (HV) 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 conducted
Slide 23: HV: Highest Levels of Oligo Delivery and Exon Skipping Observed
HV: 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 oligon
Slide 24: HV: Highest Levels of Exon Skipping Observed in Humans After Single Dose
HV: 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 i
Slide 25: HV: High, Persistent Tissue Concentrations of Oligonucleotide Observed
HV: 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 infusio
Slide 26: Safety & Tolerability Summary: PGN-EDO51 Generally Well-Tolerated
SAFETY & TOLERABILITY SUMMARY HV: PGN-EDO51 WAS GENERALLY WELL-TOLERATED IN PH1 SAD TRIAL At 10 mg/kg: All participants (n = 6) completed the study with no discontinuations. All related treatment-emergent adverse events (TEAEs) were assessed as mild and resolved without any intervention. Serum cysta
Slide 27: HV: TEAEs Mild and Resolved Without Intervention
HV: TEAEs MILD AND RESOLVED WITHOUT INTERVENTION AT SELECTED CLINICALLY-RELEVANT DOSES Healthy Volunteers (HV) with ≥1 AE, n (%) Placebo (n=8) Cohort A: 1 mg/kg (n=6) Cohort B: 5 mg/kg (n=6) Cohort C: 10mg/kg (n=6) Cohort D: 15 mg/kg (n=6) PGN-EDO51 Total (n=24) Any AE 4 (50) 4 (66.7) 2 (33.3) 5 (83
Slide 28: NHP Repeat Dose: 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. Shown as mean ± SD; n = 3 per group. Stud
Slide 29: Ph2 Clinical Plans
PH 2 CLINICAL PLANS
Slide 30: Two Ph2 DMD MAD Patient Studies Anticipated to Open in 2023
2Q: First HV dosed in Ph1 trial 3Q: Ph1 data: Highest single dose levels of exon skipping & oligo delivery* PGN-EDO51 generally well-tolerated 4Q: Completion of Ph2-enabling tox studies 1H: Initiation of CONNECT1-EDO51 Ph 2 patient study in Canada 2H: Initiation of CONNECT2-EDO51 Ph2 global patient
Slide 31: Two Ph2 MAD Studies Support Readout in 2024 and Potential Accelerated Approval
TWO PH2 MAD STUDIES SUPPORT CLINICAL DATA READOUT ANTICIPATED IN 2024 AND POTENTIAL ACCELERATED APPROVAL CONNECT1-EDO51 Ph2 open-label MAD study in patients (planned initiation 1H23) Initial dystrophin, exon skipping and safety data anticipated in 2024 CONNECT2-EDO51 Ph2 randomized, double-blind, pl
Slide 32: Marked Increase in Exon Skipping in NHP with Q4W Repeat Dosing
Q4W HV SINGLE DOSE NHP SINGLE VS REPEAT PATIENT REPEAT DOSE Biceps, 10 mg/kg, ddPCR Biceps, 20 mg/kg, ddPCR Studies initiating in 2023 MARKED INCREASE IN EXON SKIPPING OBSERVED IN NHP WITH Q4W REPEAT DOSING REGIMEN 2.5% 34.9% 14x 1.1% 1.4% We believe this NHP data supports potential for meaningful i
Slide 33: PGN-EDODM1 for Myotonic Dystrophy Type 1 (DM1)
PGN-EDODM1 FOR MYOTONIC DYSTROPHY TYPE 1 (DM1)
Slide 34: Myotonic Dystrophy Type 1 Is a Progressive, Debilitating Neuromuscular Disorder
ROOT CAUSE OF DISEASE PATIENT POPULATION** THERAPEUTIC LANDSCAPE PEPGEN’S TREATMENT APPROACH Due to a CTG repeat expansion mutation in the DMPK gene Leads to downstream dysregulation of a broad set of proteins ~40,000 (US) ~75,000 (EEA) ~15,000 (JP) No approved disease-modifying therapeutics Standar
Slide 35: PGN-EDODM1 Engineered to Liberate MBNL1 and Correct Mis-Splicing
PGN-EDODM1 WAS ENGINEERED TO LIBERATE MBNL1 FROM DMPK-CUGexp TOXIC FOCI AND CORRECT MIS-SPLICING PGN-EDODM1 DEVELOPMENT DATA SUMMARY Correction of mis-splicing observed in preclinical models with long and short CTG repeats Reduction of toxic foci and liberation of MBNL1 observed in patient cells In
Slide 36: DM1 Is a Multi-Systemic Disease with Significant Impact on Quality of Life
DM1 IS A MULTI-SYSTEMIC DISEASE THAT HAS A SIGNIFICANT IMPACT ON QUALITY OF LIFE We believe that a potential therapeutic approach with a broad biodistribution profile may allow for the treatment of such multi-systemic pathologies Musculoskeletal: Myotonia (a temporary inability to relax a muscle aft
Slide 37: Oligo-Based Modalities for DM1 Focus on Two Mechanistic Approaches
OLIGO-BASED THERAPEUTIC MODALITIES FOR DM1 ARE FOCUSED ON TWO DISTINCT MECHANISTIC APPROACHES DM1 PATHOLOGY DMPK KNOCKDOWN DMPK COMPETITION DMPK transcript CUG repeat hairpin loops bind MBNL1 and form cross-linked foci Expanding toxic foci trap more MBNL1 ASO / siRNA degrade DMPK transcript to reduc
Slide 38: PGN-EDODM1 Differentiated Over Other Approaches for DM1
PGN-EDODM1 IS DIFFERENTIATED OVER OTHER APPROACHES AS A POTENTIAL TREATMENT FOR DM1 KEY ADVANTAGES OF OUR PGN-EDODM1 APPROACH PGN-EDODM1 targets MBNL1 binding to DMPK transcripts This may provide greater tolerability – no degradation of DMPK or risk of haploinsufficiency Avoids potential disconnect
Slide 39: Pharmacology of PGN-EDODM1 Evaluated in Multiple Preclinical Models
THE PHARMACOLOGY OF PGN-EDODM1 HAS BEEN EVALUATED IN MULTIPLE PRECLINICAL MODELS Species Key readouts observed Non-GLP pharmacology studies Patient cells PGN-EDODM1 DM1 patient Reduction in nuclear foci, liberation of MBNL1 Correction of downstream transcript mis-splicing Single dose PGN-EDODM1 HSAL
Slide 40: PGN-EDODM1 Activity Observed Across a Wide Range of CTG Repeats
PGN-EDODM1 ACTIVITY HAS BEEN OBSERVED IN PRECLINICAL MODELS WITH A WIDE RANGE OF CTG REPEATS DM1 patients CTG REPEAT LENGTH DM1 patient cells: 2,600 CTG repeats Reduction of nuclear foci and correction of downstream transcript mis-splicing observed HSALR mouse model: 220 – 250 CTG repeats Correction
Slide 41: Foci Reduction In Vitro: PGN-EDODM1 Reduced Pathogenic Nuclear Foci
FOCI REDUCTION IN VITRO: PGN-EDODM1 REDUCED PATHOGENIC NUCLEAR FOCI Immortalized myoblasts from healthy individual or DM1 patient with 2600 CTG repeats were cultured then differentiated for 4 days into myotubes. Treatment with PMO or peptide-PMO conjugates at concentrations given. Cells were harvest
Slide 42: Foci Reduction & Liberation of MBNL1 In Vitro
FOCI REDUCTION & LIBERATION OF MBNL1 IN VITRO: PGN-EDODM1 LIBERATED FOCI-BOUND MBNL1 Immortalized myoblasts from healthy individual or DM1 patient with 2600 CTG repeats were cultured then differentiated for 4 days into myotubes. Cells were treated with PGN-EDODM1 and harvested for analysis 24h after
Slide 43: Mis-Splicing Correction In Vitro
MIS-SPLICING CORRECTION IN VITRO: PGN-EDODM1 CORRECTED DOWNSTREAM TRANSCRIPT MIS-SPLICING EVENTS Immortalized myoblasts from healthy individual or DM1 patient with 2600 CTG repeats were cultivated then differentiated for 4 days. Treatment with PGN-EDODM1 at concentrations given. Cells were harvested
Slide 44: HSALR Mouse Displays Molecular and Functional DM1 Phenotype
HSALR MOUSE DISPLAYS MOLECULAR AND FUNCTIONAL DM1 PHENOTYPE Source: Mankodi et al., SCIENCE 2000. DM1 ASSOCIATED ABNORMALITIES Skeletal muscle specific CUGexp MBNL1 sequestration in the nucleus Downstream mis-splicing events Myotonia (CTG)n 220-250 HSA gene 3’ UTR HSALR mouse Myotonia in hind legs R
Slide 45: HSALR: >68% Correction of Mis-Splicing and Complete Reversal of Myotonia
HSALR: PGN-EDODM1 ACHIEVED >68% CORRECTION OF MIS-SPLICING AND COMPLETE REVERSAL OF MYOTONIA AT 30 MG/KG Protocol: PGN-EDODM1 was administered IV to HSALR mice at 30 mg/kg (n=8) against a saline control (n=16) and wild-type (WT) saline control (n=8). Myotonia assessed, tissues harvested 2 weeks post
Slide 46: HSALR: Splicing Correction Translated to Phenotypic Improvement
HSALR: SPLICING CORRECTION TRANSLATED TO PHENOTYPIC IMPROVEMENT OF DM1 MICE TREATED WITH PGN-EDODM1 Protocol: PGN-EDODM1 was administered intravenously (IV) to WT and HSALR mice at 50 mg/kg (n=4-16); myotonia assessed two weeks post-administration. UNTREATED HSALR TREATED HSALR
Slide 47: HSALR: Single Dose Led to Durable Splicing Improvements Through 24 Weeks
HSALR: SINGLE DOSE TREATMENT OF PGN-EDODM1 LED TO DURABLE IMPROVEMENTS IN SPLICING THROUGH 24 WEEKS Protocol: PGN-EDODM1 was administered intravenously (IV) to WT and HSALR mice at 30 mg/kg; gastrocnemius muscle harvested 2 (n=8), 12 (n=8) or 24 (n=5) weeks post-administration; graph plotted as mean
Slide 48: Human PGN-EDO51 Tissue Concentrations Comparable to HSALR Mouse Model
HUMAN: PGN-EDO51 TISSUE CONCENTRATIONS WERE COMPARABLE TO THOSE ACHIEVED IN HSALR MOUSE MODEL WITH PGN-EDODM1 Protocol: PGN-EDODM1 was administered IV to HSALR mice at 30 (n=8) mg/kg against a wild-type (WT) saline control (n=8). Tissue concentration in the gastrocnemius muscle assessed by HPLC. Rob
Slide 49: DMPK Transcript Levels: Steric Blocking Mechanism Did Not Target DMPK for Degradation
DMPK TRANSCRIPT LEVELS IN VITRO + NHP: OUR STERIC BLOCKING MECHANISM OF ACTION WAS NOT OBSERVED TO TARGET DMPK FOR DEGRADATION In vitro: Immortalized myoblasts from DM1 patient with 2600 CTG repeats were differentiated for 4 days to myotubes and treated for 24h hours with PGN-EDODM1 from 1-20 µM. NT
Slide 50: HSALR: No Impact Observed on Other Transcripts Containing >10 CUG Repeats
TRANSCRIPT LEVELS IN HSALR MOUSE (QUADS) HSALR: NO IMPACT OBSERVED ON OTHER TRANSCRIPTS CONTAINING >10 CUG REPEATS Protocol: PGN-EDODM1 was administered once intravenously (IV) to HSALR mice at 30 mg/kg; quadriceps muscle harvested 2, 12 or 24 weeks post-administration; graph plotted as mean ± SD; n
Slide 51: NHP Single-Dose GLP Toxicology Studies Dosed Through 90 mg/kg
NHP SINGLE-DOSE GLP TOXICOLOGY STUDIES NHP: IND-ENABLING STUDIES DOSED THROUGH 90 MG/KG, RESULTS SUPPORT ANTICIPATED INITIATION OF PATIENT TRIAL IN 1H23 PGN-EDODM1 SUMMARY OF FINDINGS PGN-EDODM1 was well-tolerated through 90 mg/kg No adverse effects on kidney function No adverse effects on liver fun
Slide 52: Clinical Plans
CLINICAL PLANS
Slide 53: Anticipate Advancing to the Clinic in 2023
2Q: NHP dose range-finding study 3Q: Clinical potential supported by PGN-EDO51 trial 2H: Ph1-enabling studies 1H: Initiation of FREEDOM-DM1 Ph1 SAD DM1 patient trial DM1 patient data: Functional assessments Correction of mis-splicing Safety Initiation of Ph2 MAD DM1 patient trial BUILDING ON A ROBUS
Slide 54: FREEDOM-DM1 Ph1 Study Anticipated to Open in 1H23, Patient Data in 2024
FREEDOM-DM1 PH1 STUDY ANTICIPATED TO OPEN IN 1H23, WITH PATIENT DATA IN 2024 FREEDOM-DM1: PHASE 1 Single ascending dose PHASE 2 Multiple ascending dose Clinical plans are subject to alignment with regulatory authorities To be conducted in DM1 patients Randomized, double-blind, placebo-controlled tri
Slide 55: EDO Pipeline
rg PepGen EDO PIPELINE
Slide 56: Lead Program Targets Largest Exon Skipping Patient Population in DMD
PEPGEN’S LEAD PROGRAM TARGETS LARGEST EXON SKIPPING PATIENT POPULATION IN DMD Source: https://www.cureduchenne.org/cure/exon-skipping/. * Comparative statements are based on cross-trial comparisons with publicly-available data for other exon skipping approaches that have been assessed following a si
Slide 57: PGN-EDO53: Single-Dose Exon Skipping Almost 7x Higher Than Comparator
PGN-EDO53: SINGLE-DOSE EXON SKIPPING LEVELS ALMOST 7X HIGHER THAN FOR R6G-PMO53 COMPARATOR Protocol: PGN-EDO53 and R6G-PMO53 were administered to NHP by IV infusion over 60 min (n=3). Q4W, three doses administered, PBS control. Biopsies taken 5 - 7 days after first and second administration; termina
Slide 58: PGN-EDO45: High, Dose-Dependent Exon Skipping in Human Myoblasts
PGN-EDO45: HIGH, DOSE-DEPENDENT LEVELS OF EXON 45 SKIPPING WERE OBSERVED IN WILD-TYPE HUMAN MYOBLASTS Protocol: WT human myoblasts were differentiated, treated for 48 hours with PGN-EDO45 or R6G-PMO45 and then evaluated for exon 45 skipping levels by RT-PCR. Data is presented as mean ± SD of 4 biolo
Slide 59: PGN-EDO44: High, Dose-Dependent Exon Skipping in Human Myoblasts
PGN-EDO44: HIGH, DOSE-DEPENDENT LEVELS OF EXON 44 SKIPPING WERE OBSERVED IN WILD-TYPE HUMAN MYOBLASTS WT human myoblasts PGN-EDO44 PGN-EDO44 dose Analysis Protocol: WT human myoblasts were differentiated, treated for 48 hours with PPMOs and then evaluated for exon 44 skipping levels by RT-PCR. Data
Slide 60: Current Manufacturing Capabilities Support Planned Trials and Commercialization
CURRENT MANUFACTURING CAPABILITIES DESIGNED TO SUPPORT ALL PLANNED CLINICAL TRIALS AND COMMERCIALIZATION HIGHLIGHTS: Fully synthetic manufacturing process; no cell-based steps Product and intermediates are readily characterized Research to date suggests product has robust stability Multiple cGMP bat
Slide 61: Conclusion
rg PepGen CONCLUSION
Slide 62: The Future of PepGen
2023 2024 THE FUTURE OF PEPGEN PGN-EDO51 DMD Exon 51 PGN-EDODM1 DM1 Pipeline Highest level of single-dose exon skipping & oligo delivery in humans* Differentiated approach with robust preclinical dataset Five neuromuscular disease candidates in pipeline Work underway to leverage EDO platform to expa
Slide 63: Thank You
rg PepGen THANK YOU