Moderna, Inc. · NASDAQ:MRNA
Moderna — Q3 2019 Earnings Presentation
49 slides from Moderna's earnings presentation, Nov 6, 2019
Slide 1: Business Updates and Third Quarter Financial Results
DNA mRNA Protein Business Updates and Third Quarter Financial Results November 6, 2019
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, development candidate activities, f uture clinical
Slide 3: 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 3
Slide 4: 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 IL23+IL36 g vaccine (Triplet) VEGF-A (no LNP) OX40L Increasing biology risk CMV v accine Fabry MMA Flu Chikungunya v accines Antibody (H7, H10) Varying technology risk Localized Systemic Systemic Prophylacti
Slide 5: Two important positive clinical milestones in two modalities
Two important positive clinical milestones in two modalities recently presented at our September 12th R&D Day Cytomegalovirus (CMV) vaccine (mRNA-1647) • Positive interim phase 1 data • Successfully immunized seronegatives and boosted seropositives • Generally well tolerated • Phase 2 to start in th
Slide 6: Body of clinical data from Moderna's platform to date
Body of clinical data from Moderna’s platform to date • Moderna platform investments in research, manufacturing and clinical have enabled 16 investigational medicines to start clinical trials in the last 3.5 years • We have enrolled more than 1,400 Personalized cancer OX40L+ IL23+IL36g subjects and
Slide 7: Moderna named top employer by Science
Moderna named top employer by Science for the fifth year in a row • Moderna ranked one of the global biopharmaceutical industry’s top employers in Science and Science Careers’ 2019 Top Employers Survey • 5th consecutive year • Annual survey evaluates companies in the biopharmaceutical industry in ca
Slide 8: Gaining insights through research collaborations
Gaining insights through research collaborations • Research collaborations have been a key tenet of Moderna’s strategy with over 15 collaborations with leading universities and medical centers • In October, Moderna announced an mRNA immunotherapy research collaboration with Harvard University • Goal
Slide 9: Moderna's development pipeline
Moderna’s development pipeline 7 Prophylactic Positive vaccine program s Phase 1 Data1 2 Cancer vaccine program s Chikungunya 3 Intratum oral im m uno- antibody KRAS oncology program s CMV vaccine Vaccine 1 Localized regenerative therapeutics program PCV IL12 3 System ic secreted therapeutics progra
Slide 10: Progress by modality
Progress by modality Prophylactic Cancer Intratumoral Localized Systemic Systemic vaccines vaccines immuno-oncology regenerative secreted intracellular therapeutics therapeutics therapeutics Slide 10
Slide 11: Prophylactic vaccines
Prophylactic vaccines Six positive phase 1 readouts Clinical summary • Safety: >1,000 healthy volunteers enrolled in nine phase 1 vaccine trials, at dose levels up to 400µg ̶ Emerging safety and tolerability profile consistent with that of marketed adjuvanted vaccines • Progress: ̶ CMV (mRNA-1647):
Slide 12: Prophylactic vaccines: CMV and mRNA-1647 overview
Prophylactic vaccines CMV and mRNA-1647 overview Congenital cytomegalovirus (CMV) overview • CMV is a common human pathogen and member of the herpes family of viruses and is the most common infectious disease cause of birth defects in the U.S. • Disease burden: Birth defects occur in approximately 2
Slide 13: CMV vaccine (mRNA-1647) phase 1 interim analysis
CMV vaccine (mRNA-1647) phase 1 interim analysis Immunogenicity in CMV-seronegative participants, per-protocol set Variable Neutralizing Antibodies Against Epithelial Cell Infection ▪ Seronegative subjects Placebo 30 μg 90 μg 180 μg successfully immunized to Baseline GMT 8 8 8 8 generate neutralizin
Slide 14: CMV vaccine (mRNA-1647) phase 1 interim analysis
CMV vaccine (mRNA-1647) phase 1 interim analysis Immunogenicity in CMV-seropositive participants, per-protocol set Variable Neutralizing Antibodies Against Epithelial Cell Infection ▪ Seropositive subjects Placebo 30 μg 90 μg 180 μg effectively boosted beyond Baseline GMT 8,169 3,614 5,634 5,700 lev
Slide 15: Most Common Systemic AEs
CMV vaccine (mRNA-1647) phase 1 interim analysis Solicited adverse events (AE) post 2nd 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 n=13 n=15 n=16 n=15 n=13 n=12 n=11 n=11 Local AEs - 11 (73%) 13
Slide 16: CMV vaccine (mRNA-1647) phase 1 interim analysis
CMV vaccine (mRNA-1647) phase 1 interimanalysis Durable immunogenicity demonstrated in initial cohort followed to one year Slide 16
Slide 17: CMV vaccine (mRNA-1647) phase 1 interim analysis: Summary
CMV vaccine (mRNA-1647) phase 1 interim analysis Summary • mRNA-1647 exhibited dose-related increase in neutralizing antibodies • Seronegative subjects successfully immunized to generate neutralizing titers against CMV • Seropositive subjects effectively boosted beyond levels seen in natural infecti
Slide 18: CMV vaccine (mRNA-1647): Next steps
CMV vaccine (mRNA-1647) Next steps • Phase 2 dose confirmation study to be initiated in the near term • N=252 adult females and males 18-40 yrs of age, both seronegative and seropositive status • 3 doses: 50 µg, 100 µg and 150 µg • 3:1 randomization • Dose selection after first interim analysis: saf
Slide 19: CMV is a blockbuster commercial opportunity
CMV is a blockbuster commercial opportunity Moderna owns global commercial rights to mRNA-1647 • Large unmet medical need with no approved vaccine • Indication expansion opportunity • Total addressable market could be as high as 70 million vaccinations per year • Focused sales effort: OBGYN and Pedi
Slide 20: PCV (mRNA-4157): Phase 1 and phase 2 studies ongoing
PCV (mRNA-4157): Phase 1 and phase 2 studies ongoing KRAS (mRNA-5671/Merck V941): Phase 1 study ongoing PCV (mRNA-4157) Phase 1 trial • Interim safety, tolerability immunogenicity data presented at ASCO 20191 • Continuing to enroll patients in phase 1 safety, tolerability and immunogenicity trial mo
Slide 21: Intratumoral immuno-oncology
Intratumoral immuno-oncology Three phase 1 programs in combination with checkpoint inhibitor ongoing • OX40L (mRNA-2416) ̶ Phase 1 study evaluating safety and tolerability of mRNA-2416 administered intratumorally in monotherapy and combination with durvalumab is ongoing with patients dosed in both a
Slide 22: Localized regenerative therapeutics
Localized regenerative therapeutics Program Preclinical Phase 3 and Modality Program # Phase 1 Phase 2 Moderna rights Indication dev elopment commercial Localized VEGF-A AZ to pay regenerative AZD8601 Myocardial ischemia milestones and therapeutics royalties Slide 22
Slide 23: Antibody against Chikungunya virus (mRNA-1944)
Antibody against Chikungunya virus (mRNA-1944) mRNA-1944 contains two mRNAs that encode for the heavy and light chains of CHKV-24 antibody, which may confer passive immunity Expected protective level in mice2 Slide 23
Slide 24: 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 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 (µg/mL) 1.1-3.1 6.3-10.0 7.0-14.2 Cmax 40.6% 18.2% 29.
Slide 25: Antibody against Chikungunya virus (mRNA-1944)
Antibody against Chikungunya virus (mRNA-1944) mRNA-1944 driven protein expression results in functional antibody (CHKV-24) Serum neutralization activity 48 hr after mRNA-1944 administration • Neutralizing antibody titers 50 > 100 of Subjects 100 observed at all dose levels, indicating functional an
Slide 26: Antibody against Chikungunya virus (mRNA-1944): Summary of related adverse events
Antibody against Chikungunya virus (mRNA-1944) Summary of related adverse events Cohort Grade 1 Grade 2 Grade 3 Placebo (N=6) None None None • All AEs were transient and resolved mRNA-1944 spontaneously without 0.1 mg/kg Feeling of warmth, transient (1) None None treatment (N=6) mRNA-1944 • No serio
Slide 27: Antibody against Chikungunya virus (mRNA-1944): Translation from preclinical species to humans
Antibody against Chikungunya virus (mRNA-1944) Translation from preclinical species to humans CHKV-24 IgG concentration (µg/mL) Solid line = Median predicted Shaded area = 90% prediction interval Symbols = Individual participant observations Slide 27
Slide 28: Antibody against Chikungunya virus (mRNA-1944): Next steps
Antibody against Chikungunya virus (mRNA-1944) Next steps • mRNA-1944 will continue to be explored in two additional cohorts • One cohort with steroid premedication at the 0.6mg/kg dose • One cohort with two doses of 0.3mg/kg (without steroid premedication) administered one week apart Slide 28
Slide 29: Antibody against Chikungunya virus (mRNA-1944): Summary
Antibody against Chikungunya virus (mRNA-1944) Summary • Administration of mRNA-1944 resulted in dose-dependent increases in levels of antibody against Chikungunya (CHKV-24) • Neutralizing antibodies were observed at all dose levels, indicating functional antibody production by mRNA-1944 • None of t
Slide 30: Our modality strategy
Our modality strategy Personalized OX40L+ cancer IL23+IL36 vaccine (Triplet) VEGF-A (no LNP) OX40L Increasing biology risk CMV Fabry v accine MMA Flu Chikungunya v accines Antibody (H7, H10) Varying technology risk Localized Systemic Systemic Prophylactic Cancer Intratumoral regenerativ e secreted
Slide 31: MMA (mRNA-3704) and PA (mRNA-3927)
MMA (mRNA-3704): Multiple clinical trial sites actively recruiting PA (mRNA-3927): Open IND, plans to start phase 1/2 trial MMA (mRNA-3704) • Encodes for MUT enzyme, an intracellular protein that acts in the mitochondria of liver cells • Same proprietary lipid nanoparticle formulation as antibody ag
Slide 32: Anticipated clinical next steps and catalysts
Anticipated clinical next steps and catalysts • CMV – Phase 2 dose confirmation study start and immunogenicity data at 3 month IA Prophylactic • hMPV+PIV3 – Phase 1b seropositive age de-escalation immunogenicity data readout vaccines • RSV – Phase 1 safety and immunogenicity data readout • Zika – Ph
Slide 33: Third quarter 2019 financial results
Third quarter 2019 financial results (Unaudited) Balance Sheets September 30, December 31, 2019 2018 Cash, cash equivalents and investments 1 $ 1.34 billion $ 1.69 billion Statements of Cash Flows 9 months ended 9 months ended Sept. 30, 2019 Sept. 30, 2018 Net cash used in operating activities 2 $ 3
Slide 34: Q3 2019 cash balance and investments and grant funding status
Q3 2019 cash balance and investments 1 and grant funding status (Unaudited) Cash balance and investments1 at September 30, 2019 • At September 30, 2019, we had $1.34 billion of cash, cash equivalents and investments Grant funding status as of September 30, 2019 Total additional funding available fro
Slide 35: 2019 YE guidance update
2019 YE guidance update (Unaudited) Guidance Ended 2018 with cash and February 2019 May 2019 August 2019 November 2019 investments 1 of $1.69 billion Cash and investments 1 Reiterated Cash and investments 1 Reiterated at 12/31/19 expected to at 12/31/19 expected at be in the range of $1.15 approxima
Slide 36: 2020 guidance
2020 guidance Notable trends in 2020: • Cellscript in-license payment is non-recurring • Limited need for significant new facilities/capital expenditures • Advancement of development pipeline continues, but: • CMV phase 3 trial only requires pre-investment in 2020 • Partners fund significant develop
Slide 37: Key candidates across modalities are funded by partners
Key candidates across modalities are funded by partners Program Preclinical Phase 3 and Modality ID # Phase 1 Phase 2 Moderna rights Indication dev elopment commercial mRNA-1172 Respiratory syncytial virus (RSV) vaccine Merck to pay milestones and royalties mRNA-1777 Respiratory syncytial virus (RSV
Slide 38: Closing Remarks
Closing Remarks joes moderna
Slide 39: Moderna in November 2019: Foundations in Development
Moderna in November 2019 10 Pipeline 4 12 Positive Ph 1 readouts : 6 In or preparing for Ph 2 Ph 1 trials ongoing vaccines, PCV, OX40L, VEGF, anti- Chikungunya antibody 4 5 5 in Development Vaccines for major unmet Immuno-Oncology Rare Disease Programs needs • MMA – Ph 1 actively recruiting • PCV in
Slide 40: Moderna in November 2019: Foundations in Development
Moderna in November 2019 10 Pipeline 4 12 Positive Ph 1 readouts : 6 In or preparing for Ph 2 Ph 1 trials ongoing vaccines, PCV, OX40L, VEGF, Anti- Chikungunya antibody 4 5 5 in Development Vaccines for major unmet Immuno-Oncology Rare Disease Programs needs • MMA – Ph 1 actively recruiting • PCV in
Slide 41: 4 infectious vaccine pipeline programs with a major unmet medical need
4 infectious vaccine pipeline programs with a major unmet medical need Development Targeted indication Currently Potential candidate marketed peak sales product mRNA-1647 Prevention of CMV No Multi-billion CMV vaccine infection mRNA-1172 Prevention of RSV No Multi-billion mRNA-1777 infection in the
Slide 42: Moderna in November 2019: Foundations in Development
Moderna in November 2019 10 Pipeline 4 12 Positive Ph 1 readouts : 6 In or preparing for Ph 2 Ph 1 trials ongoing vaccines, PCV, OX40L, VEGF, anti- Chikungunya antibody 4 5 5 in Development Vaccines for major unmet Immuno-Oncology Rare Disease Programs needs • MMA – Ph 1 actively recruiting • PCV in
Slide 43: 5 I/O programs aimed at improving checkpoint inhibitor activity
5 I/O programs aimed at improving checkpoint inhibitor activity Program Trial Design Patients enrolled in combination arm mRNA-4157 RCT: Keytruda + PCV vs. Keytruda Yes PCV phase 2 monotherapy mRNA-4157 Phase 1: PCV + Keytruda in Yes PCV phase 1 adjuvant and metastatic settings mRNA-2416 Phase 1: OX
Slide 44: Moderna in November 2019: Foundations in Development
Moderna in November 2019 10 Pipeline 4 12 Positive Ph 1 readouts : 6 In or preparing for Ph 2 Ph 1 trials ongoing vaccines, PCV, OX40L, VEGF, anti- Chikungunya antibody 4 5 5 in Development Vaccines for major unmet Immuno-Oncology Rare Disease Programs needs • MMA – Ph 1 actively recruiting • PCV in
Slide 45: 5 rare disease pipeline programs with unmet medical needs and low-biology risk targets
5 rare disease pipeline programs with unmet medical needs and low-biology risk targets Program Targeted disease Currently Regulatory designations indication Marketed product mRNA-3704 MMA No FDA: orphan drug designation, rare pediatric disease designation, Fast Track designation, EMA orphan drug des
Slide 46: Moderna priorities for 2019-2020
Moderna priorities for 2019-2020 1 Execute on the development pipeline • 20 programs in development • Focus on demonstrating human proof of concept ̶ Major progress made with phase 1 CMV and Chikungunya antibody programs New development candidates in existing modalities 2 • Nominated new rare diseas
Slide 47: Moderna 3Q19 Highlights
Moderna 3Q19 Highlights • Announced positive phase 1 clinical data from our ̶ CMV vaccine (mRNA-1647) ̶ Antibody against Chikungunya virus program (mRNA-1944) • We have up to $1.5 billion to invest in the company between our cash and grants. • We expect to invest around $500 million in the company i
Slide 48: Our Mission
Our Mission To deliver on the promise of mRNA science to create a new generation of transformative medicines for patients. Slide 48
Slide 49: Save the Date: Manufacturing and Digital Day
Save the Date Manufacturing and Digital Day Afternoon of Wednesday March 4, 2020