Moderna, Inc. · NASDAQ:MRNA
Moderna — Technical Presentation (January 2020)
18 slides from Moderna's technical presentation, Jan 10, 2020
Slide 1: CMV vaccine (mRNA-1647) 7-month interim analysis call
DNA mRNA Protein CMV vaccine (mRNA-1647) 7-month interim analysis call January 10, 2020
Slide 2: Forward-looking statements
Forward-lookingstatements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended including, but not limited to, the expected Phase 3 trial dosing regimen, the timing of the Phase 2 trial and the start of the Phase
Slide 3: Moderna's development pipeline
Moderna’s development pipeline (as of January 10, 2020) mRNA-1647 is the first mRNA infectious disease vaccine to start a phase 2 7 Prophylactic vaccine program s Positive 2 Cancer Phase 1 Data1 vaccine program s Chikungunya KRAS 3 Intratum oral im m uno- antibody Vaccine oncology program s CMV 1 Lo
Slide 4: CMV data generation to date
CMV data generation to date Month 0 (day 1) Month 2 Month 6 CMV phase 1 dosing regimen 3-month 7-month September interim interim Today 2019 R&D Day analysis analysis First interim analysis: Seronegative Second interim analysis: Neutralizing subjects successfully immunized antibody titers continued t
Slide 5: Congenital cytomegalovirus overview
Congenital cytomegalovirusoverview • Cytomegalovirus: CMV is a common CMV infection sequelae infection and is the leading cause of birth defects in the US Jaundice ‒ 0.65% of US newborns infected annually Microcephaly (~25,000 US newborns) Neonatal Hearing loss period Vision loss • Disease burden: S
Slide 6: Congenital CMV vaccine includes 6 mRNAs
Congenital CMV vaccine includes 6 mRNAs 5 encode the Pentamer, 6th encodes gB antigen Epithelial cells Fibroblasts Slide 6
Slide 7: CMV vaccine (mRNA-1647) phase 1 trial
CMV vaccine (mRNA-1647) phase 1 trial Key objective: ▪ To assess the safety, reactogenicity, and immunogenicity of different dose levels of mRNA-1647 Primary endpoint: Safety Secondary endpoints: ▪ Neutralizing antibodies against CMV infection of epithelial cells and fibroblasts ▪ Binding antibodies
Slide 8: Immunogenicity in CMV-seronegative participants, per-protocol set
Immunogenicity in CMV-seronegative participants, per-protocol set Neutralizing Antibodies Against Epithelial Cell Infection CMV-seropositive GMT benchmark = 5,9171 ▪ Neutralizing antibody titers against both epithelial cell and Phase B Phase C fibroblast infection continued to Placebo 30 μg 90 μg 18
Slide 9: Immunogenicity in CMV-seropositive participants, per-protocol set
Immunogenicity in CMV-seropositive participants, per-protocol set Neutralizing Antibodies Against Epithelial Cell Infection CMV-seropositive GMT benchmark = 5,9171 ▪ In seropositive subjects, the third vaccination boosted Phase B Phase C neutralizing antibody titers Placebo 30 μg 90 μg 180 μg Placeb
Slide 10: Solicited adverse reactions post 2nd vaccination
Solicited adverse reactions (AR) post 2nd vaccination, solicited safety set CMV Serostatus at Baseline CMV-seronegative CMV-seropositive Phase B Phase C Phase B Phase C Placebo 30 μg 90 μg 180 μg Placebo 300 μg Placebo 30 μg 90 μg 180 μg Placebo 300 μg Local ARs 11 (73%) 11 (79%) 12 (80%) 10 (91%) 9
Slide 11: Solicited adverse reactions post 3rd vaccination
Solicited adverse reactions (AR) post 3rd vaccination, solicited safety set CMV Serostatus at Baseline CMV-seronegative CMV-seropositive Placebo 30 μg 90 μg 180 μg Placebo 30 μg 90 μg 180 μg 1 (9%) 8 (73%) 7 (54%) 9 (75%) 8 (73%) 6 (60%) 6 (100%) Pain - Local ARs - - 1 (8) 1 (8) - - - 2 (15) 1 (8) 2
Slide 12: Durable immunogenicity demonstrated in seronegative initial cohort
Durable immunogenicity demonstrated in seronegative initial cohort Neutralizing antibodies against epithelial cell infection Seropositive Baseline GMT Slide 12
Slide 13: Additional boosting demonstrated in seropositive cohort
Additional boosting demonstrated in seropositive cohort Neutralizing antibodies against epithelial cell infection Seropositive Baseline GMT Slide 13
Slide 14: 7-month interim analysis summary
CMV vaccine (mRNA-1647) 7-month interim analysis summary • Safety: The vaccine was generally well-tolerated and there were no vaccine-related serious adverse events (SAEs). Safety and tolerability at the 300µg dose level was comparable to that observed at the 180µg dose level • In the CMV-seronegati
Slide 15: Phase 2 dose confirmation study
Phase 2 dose confirmation study • The phase 2 dose-confirmation study implements the intended phase 3 formulation of the mRNA-1647 vaccine, which contains the same proprietary lipid nanoparticle (LNP) used in the phase 1 study • US sites only • 3 dose levels (50µg, 100µg, 150µg); randomized, double-
Slide 16: Phase 3 preparations ongoing
Phase 3 preparations ongoing • Received Type C meeting feedback from FDA on primary endpoint: prevention of primary CMV infection in a population that includes women of childbearing age (WOCBA) • We believe we can achieve this objective with a <8,000 subject trial • Preparation and product manufactu
Slide 17: CMV is a blockbuster opportunity
CMV is a blockbuster opportunity • Estimated annual peak sales of $2-5 billion • Assuming GARDASIL2 like ASP; GM estimated to be +90%3 (EBIT margins of approximately 50%) 1 • NEJM phase 2 publication by Pass et al shows 50% vaccine efficacy with Sanofi’s vaccine targeting only the gB antigen • Moder
Slide 18: Our Mission
Our Mission To deliver on the promise of mRNA science to create a new generation of transformative medicines for patients. Slide 18