Moderna, Inc. · NASDAQ:MRNA
Moderna — 38th Annual J.P. Morgan Healthcare Conference (January 2020)
29 slides from Moderna's conference presentation, Jan 13, 2020
Slide 1: 38th Annual J.P. Morgan Healthcare Conference
DNA mRNA Protein 38th Annual J.P. Morgan Healthcare Conference Stéphane Bancel, CEO January 13th, 2020
Slide 2: Forward-looking statements
Forward-looking statements 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, statements concerning: clinical program next steps; clinical de-risking of its prophylactic vaccines
Slide 3: Moderna's development pipeline
Moderna’s development pipeline 1 KRAS 5 Prophylactic vaccine program s Positive Vaccine 2 Cancer vaccine program s Phase 1 Data2 Chikungunya IL-12 3 Intratum oral im m uno-oncology programs antibody 1 Localized regenerative therapeutics program CMV RSV (1172) vaccine vaccine 3 System ic secreted & c
Slide 4: Major events in four modalities in 2019
Major events in four modalities in 2019 CMV vaccine Prophylactic hMPV/PIV3 vaccines vaccine PCV Cancer vaccines Chikungunya antibody Systemic secreted & cell surface therapeutics MMA Systemic PA intracellular therapeutics Slide 4
Slide 5: Congenital cytomegalovirus overview
Congenital cytomegalovirus overview • Cytomegalovirus: CMV is a common infection and is the leading cause of birth defects in the US CMV infection sequelae ̶ 0.65% of US newborns infected annually (~25,000 US newborns) • Jaundice • Microcephaly • Disease burden: Significant impact on survivors, fami
Slide 6: CMV vaccine (mRNA-1647)
CMV vaccine (mRNA-1647) Immunogenicity from 7-month data confirms and builds on 3-month data • Safety1: ̶ Generally well-tolerated ̶ No vaccine-related serious adverse events • CMV-seronegative group at seven months (after third vaccination): ̶ Epithelial cell assay titers >10-fold higher than CMV-s
Slide 7: Phase 2 trial started; Phase 3 preparations ongoing for CMV vaccine
Phase 2 trial started; Phase 3 preparations ongoing for CMV vaccine (mRNA-1647) • 3 dose levels; randomized, double-blind, placebo-controlled • 252 seronegative & seropositive adults Phase 2 dose • Utilizes intended phase 3 formulation; same LNP used in phase 1 confirmation study • 12 U.S. sites • F
Slide 8: 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%) • NEJM phase 2 publication by Pass et al shows 50% vaccine efficacy with Sanofi’s vaccine targeting only the gB antigen • Moderna
Slide 9: Major events in four modalities in 2019
Major events in four modalities in 2019 CMV vaccine hMPV/PIV3 vaccine (mRNA-1653) Prophylactic hMPV/PIV3 • Positive interim phase 1 adult data vaccines vaccine • Generally well tolerated • Phase 1b age de-escalation study ongoing • Moderna owns the worldwide rights to mRNA-1653 PCV Cancer vaccines C
Slide 10: Major events in four modalities in 2019
Major events in four modalities in 2019 CMV vaccine Prophylactic hMPV/PIV3 Personalized Cancer Vaccine (mRNA-4157) vaccines vaccine • Positive interim phase 1 data reported at ASCO • Well tolerated at all dose levels studied with no dose limiting toxicities reported PCV • Neoantigens specific CD8+ T
Slide 11: Antibody against Chikungunya virus (mRNA-1944)
Major events in four modalities in 2019 CMV vaccine Prophylactic hMPV/PIV3 vaccines vaccine PCV Cancer vaccines Chikungunya antibody Systemic secreted & cell surface therapeutics MMA Systemic PA intracellular therapeutics Slide 11
Slide 12: Antibody against Chikungunya virus (mRNA-1944)
Antibody against Chikungunya virus (mRNA-1944) Protective antibody levels of >1µg/mL expected to endure at least 16 weeks at the middle dose of 0.3 mg/kg immunity 0.1 mg/kg 0.3 mg/kg 0.6 mg/kg Cohort (N=6) (N=6) (N=4) Cmax 2.0 7.9 10.2 (µg/mL) Cmax range 1.1-3.1 6.3-10.0 7.0-14.2 (µg/mL) Cmax 40.6%
Slide 13: Major events in four modalities in 2019
Major events in four modalities in 2019 CMV vaccine Prophylactic hMPV/PIV3 vaccines vaccine PCV Cancer vaccines Chikungunya antibody Systemic secreted & cell surface MMA (mRNA-3704) therapeutics • Actively recruiting patients in phase 1/2 • FDA fast track status MMA PA (mRNA- 3927) • Open IND System
Slide 14: mRNA as a potential new class of medicines
mRNA as a potential new class of medicines 1. Large product opportunity 2. Higher probability of technical success 3. Accelerated research and development timelines 4. Greater capital efficiency over time vs. recombinant technology Slide 14
Slide 15: Risk management is essential to building a new class of medicines
Risk management is essential to building a new class of medicines Personalized OX40L/ cancer IL-23/IL-36𝛾 CMV vaccine (triplet) vaccine VEGF-A (no LNP) Increasing biology risk KRAS cancer OX40L Zika vaccine Fabry vaccine MMA PA H10/H7 Chikungunya Influenza Antibody Vaccine Varying technology risk…
Slide 16: mRNA technology validated by clinical data in two modalities
mRNA technology validated by clinical data in two modalities De-risked modalities Exploratory modalities Personalized OX40L/ cancer IL-23/IL-36𝛾 vaccine (triplet) VEGF-A Increasing biology risk CMV Chikungunya (no LNP) vaccine Antibody KRAS cancer OX40L vaccine MMA PA Varying technology risk…
Slide 17: mRNA technology validated by clinical data in two modalities
mRNA technology validated by clinical data in two modalities De-risked modalities mRNA advantages 1. Large product opportunity 2. High probability of CMV Chikungunya vaccine Antibody technical success 3. Accelerated research and development timelines 4. Greater capital efficiency over time vs. recom
Slide 18: mRNA technology validated by clinical data in two modalities
mRNA technology validated by clinical data in two modalities Therapeutic areas mRNA advantages 1. Large product opportunity 2. High probability of Infectious technical success Diseases 3. Accelerated research and development timelines 4. Greater capital efficiency over time vs. recombinant technolog
Slide 19: Moderna to expand development in its two de-risked modalities
Moderna to expand development in its two de-risked modalities Therapeutic areas mRNA advantages 1. Large product opportunity 2. High probability of Infectious Autoimmune technical success Diseases Diseases 3. Accelerated research and development timelines 4. Greater capital efficiency over time vs.
Slide 20: IL-2 (mRNA-6231) – long acting mutein
IL-2 (mRNA-6231) – long acting mutein mRNA-encoded IL-2 modified for the expansion of regulatory T cells • Modified IL-2 is longer acting and more selective for the high affinity IL-2 receptor which is expressed mainly by regulatory T cells • First intended subcutaneous administration of the LNP suc
Slide 21: PD-L1 (mRNA-6981)
PD-L1 (mRNA-6981) mRNA-encoded PD-L1 to send a tolerizing signal to immune cells • We intend to influence myeloid cells to provide additional co-inhibitory signals in the context of immune synapses by augmenting endogenous expression of PD-L1 • We believe that this tolerizing signal to lymphocytes m
Slide 22: Anticipated clinical next steps and catalysts
Anticipated clinical next steps and catalysts • CMV – Phase 2 immunogenicity data at 3-month IA, Phase 3 start Prophylactic • hMPV/PIV3 – Phase 1b seropositive age de-escalation immunogenicity data readout vaccines • RSV – Phase 1 safety and immunogenicity data readout • Zika – Phase 1 safety and im
Slide 23: Ability to invest ~$1.45 billion to create value
Ability to invest ~$1.45 billion to create value Cash and investments 1 at December 31, 2019 (unaudited) At December 31, 2019, we had $1.26 billion of cash, cash equivalents and investments Grant funding status as of December 31, 2019 (unaudited) Total additional funding available from grants is ~$1
Slide 24: Moderna in January 2020
Moderna in January 2020 1 4 10 10 Pipeline Preparing for In or preparing Ph 1 trials ongoing Positive Ph 1 readouts: Ph 3 for Ph 2 6 vaccines, PCV, OX40L, VEGF, anti-Chikungunya antibody 4 5 5 2 Development Programs in Vaccines for major unmet needs Immuno-Oncology Rare disease Autoimmune disease •
Slide 25: Moderna priorities for 2020
Moderna priorities for 2020 1 Execute on the development pipeline • Execution of CMV phase 2 and preparation for CMV phase 3 • Advance the 20 programs in development Slide 25
Slide 26: Moderna's development pipeline (as of January 13th, 2020)
Moderna’s development pipeline (as of January 13 th, 2020) KRAS 5 Prophylactic vaccine program s Positive Vaccine 2 Cancer vaccine program s Phase 1 Data1 Chikungunya IL-12 3 Intratum oral im m uno-oncology programs antibody 1 Localized regenerative therapeutics program CMV RSV (1172) vaccine vaccin
Slide 27: Moderna priorities for 2020
Moderna priorities for 2020 1 Execute on the development pipeline • Execution of CMV phase 2 and preparation for CMV phase 3 • Advance the 20 programs in development 2 New development candidates in the two clinically de-risked modalities • Prophylactic vaccines • Systemic secreted and cell surface t
Slide 28: Our mission
Our mission To deliver on the promise of mRNA science to create a new generation of transformative medicines for patients. Slide 28
Slide 29: mRNA as a potential new class of medicines
mRNA as a potential new class of medicines 1. Large product opportunity 2. Higher probability of technical success 3. Accelerated research and development timelines 4. Greater capital efficiency over time vs. recombinant technology Slide 29
Moderna — 38th Annual J.P. Morgan Healthcare Conference (January 2020) on 6ix