36th Annual J.P. Morgan Healthcare Conference Therapeutics JPM 2018...Slide 2 This presentation does...

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Page 1: 36th Annual J.P. Morgan Healthcare Conference Therapeutics JPM 2018...Slide 2 This presentation does not constitute an offer to sell to, or a solicitation of an offer to buy from,

Slide 1

36th Annual J.P. Morgan Healthcare Conference!"#$"%&'()*+'',-.(

© 2018 Moderna Therapeutics

Page 2: 36th Annual J.P. Morgan Healthcare Conference Therapeutics JPM 2018...Slide 2 This presentation does not constitute an offer to sell to, or a solicitation of an offer to buy from,

Slide 2

This presentation does not constitute an offer to sell to, or a solicitation of an offer to buy from, anyone in any state or in any other jurisdiction, whether under the Securities Act of 1933, as amended, or otherwise.

Various statements in this presentation concerning the future expectations of Moderna Therapeutics, Inc. (together with its affiliates, the “Company”), plans and prospects, including without limitation, the Company’s views with respect to the potential for mRNA therapeutics, its expectations with respect to timing of regulatory filings and the reporting of initial data from any clinical trial(s), the potential therapeutic opportunities for mRNA, its expectations regarding its product strategies, and its plans regarding commercialization of mRNA therapeutics constitute forward-looking statements. Words such as "expects," "plans," "potential," "target," "continue" and variations of these words or similar expressions are intended to identify forward-looking statements. Such statements are based upon the information available to us now and are subject to change. The Company will not necessarily inform you of such changes. These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, the Company’s ability to discover and develop novel drug candidates and delivery approaches, successfully demonstrate the efficacy and safety of its drug candidates, the pre-clinical and clinical results for its product candidates, which may not support further development of product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials, obtaining, maintaining and protecting intellectual property, the Company’s ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties, obtaining regulatory approval for products, competition from others using technology similar to the Company’s and others developing products for similar uses, the Company’s ability to manage operating expenses, the Company’s ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, the Company’s dependence on third parties for development, manufacture, marketing, sales and distribution of products, the outcome of litigation, and unexpected expenditures. In addition, any forward-looking statements represent the Company’s views only as of today and should not be relied upon as representing its views as of any subsequent date. The Company explicitly disclaims any obligation to update any forward-looking statements.

© 2018 Moderna Therapeutics

Legal Notices

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Slide 3

The evolution of understanding diseases over the last 100 years

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© 2018 Moderna Therapeutics

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Slide 4 © 2018 Moderna Therapeutics

mRNA is the software of life

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Page 5: 36th Annual J.P. Morgan Healthcare Conference Therapeutics JPM 2018...Slide 2 This presentation does not constitute an offer to sell to, or a solicitation of an offer to buy from,

Slide 5 © 2018 Moderna Therapeutics

Our vision for Moderna since day 1:a very broad platform, enabling many drug modalities

mRNA platform

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Page 6: 36th Annual J.P. Morgan Healthcare Conference Therapeutics JPM 2018...Slide 2 This presentation does not constitute an offer to sell to, or a solicitation of an offer to buy from,

Slide 6 © 2018 Moderna Therapeutics

Our vision for Moderna since day 1:each modality will over time enable many drugs

mRNA platform

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Slide 7

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We have been relentlessly executing on that vision

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Slide 8

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Slide 9Slide 9

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We have been relentlessly executing on that vision and now have 19 DCs, 10 of which are in clinical trials

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Slide 10

• 19 Development Candidates, of which 10 are in clinical trials̶ 1st therapeutic moving to Phase 2 with AZ̶ Multiple vaccines with positive human readouts in Phase 1

• Team continues to strengthen and expand̶ 600 team members in Cambridge, MA̶ 3rd year in a row as a top 10 employer in Science survey̶ 2 biotech/pharma veterans joined the executive team: Dr. John Mendlein (ex Atyr) and Juan Andres (ex NVS)̶ 2 experienced board members joined: Dr. Moncef Slaoui (ex GSK) and Stephen Berenson (ex JPM)

• Continue to invest in infrastructure and digital to enable our scale-up and growth̶ Robotics in place to produce up to 1,000 mRNAs per month for drug discovery̶ Current Cambridge GMP suites are running at full capacity̶ Norwood fully-integrated GMP site on track for operations in summer 2018 (built to scale to 100+ GMP lots/yr)̶ SAP already implemented in finance and manufacturing

• Strong balance sheet to continue to invest: $900 mm and no debt̶ 2017 investment - gross opex and capex >$450 mm

• Strong patent estate (70+ issued patents) plus licenses from Harvard and UPenn

Moderna in January 2018

© 2018 Moderna Therapeutics

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Slide 11 © 2018 Moderna Therapeutics

Review by modality

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Slide 12

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The prophylactic vaccines modality is generating clinical dataNine DCs. Seven programs in the clinic

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Slide 13

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Slide 14 © 2018 Moderna Therapeutics

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Slide 15

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N2GL formulation – a Moderna platform success in 2017 to enable the Liver modality

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Slide 20

mRNA-3704 for Methylmalonic Acidemia (MMA)codes for MUT enzyme in the mitochondria

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© 2018 Moderna Therapeutics

Page 22: 36th Annual J.P. Morgan Healthcare Conference Therapeutics JPM 2018...Slide 2 This presentation does not constitute an offer to sell to, or a solicitation of an offer to buy from,

Slide 22

mRNA-3927 for Propionic Acidemia (PA) comprises2 mRNAs in the vial (for PCCA and PCCB proteins)

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© 2018 Moderna Therapeutics

Page 23: 36th Annual J.P. Morgan Healthcare Conference Therapeutics JPM 2018...Slide 2 This presentation does not constitute an offer to sell to, or a solicitation of an offer to buy from,

Slide 23

We are continuing to gain momentum19 development candidates nominated, with 10 in the clinic

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© 2018 Moderna Therapeutics

Page 24: 36th Annual J.P. Morgan Healthcare Conference Therapeutics JPM 2018...Slide 2 This presentation does not constitute an offer to sell to, or a solicitation of an offer to buy from,

Slide 24 © 2018 Moderna Therapeutics

January 2018 development pipeline – a molecular view19 Development Candidates, with 10 in clinical trials

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Page 25: 36th Annual J.P. Morgan Healthcare Conference Therapeutics JPM 2018...Slide 2 This presentation does not constitute an offer to sell to, or a solicitation of an offer to buy from,

Slide 25

Human studies for 10 programs underwayPromising human datasets emerging from first wave of programs Therapeutic Area (Modality)

DevelopmentCandidate (“DC”) Lead Indication /

TargetGLP

ToxicologyIND or CTA

Filed Phase 1 Phase 2

Infectious Diseases: Commercial Vaccines(Prophylactic Vaccines)

mRNA-1647 Moderna CMV

mRNA-1653 Moderna HMPV+PIV3

mRNA MRK-1777 Merck Undisclosed

mRNA MRK-V213 Merck Undisclosed

Infectious Diseases:Public Health Vaccines(Prophylactic Vaccines)

mRNA-1440 Moderna Influenza H10 n/a*

mRNA-1851 Moderna Influenza H7 n/a*

mRNA-1325 Moderna / BARDA Zika

mRNA-1893# Moderna Zika

mRNA-1388 Moderna / DARPA Chikungunya

Immuno-Oncology(Therapeutic Vaccines)

mRNA-4157 Moderna / Merck Personalized Cancer Vaccine

mRNA-5671 Moderna KRAS

Immuno-Oncology(Intratumoral I/O)

mRNA-2416 Moderna OX40L

mRNA-2752 Moderna OX40L+IL23+IL36γ Ongoing

mRNA-2905 AstraZeneca / Moderna IL12 Ongoing

CV Diseases(Localized; Liver)

mRNA AZD-8601 AstraZeneca VEGF-A

mRNA AZD-7970 AstraZeneca / Moderna Relaxin

Rare DiseasesmRNA-3704 Moderna Methylmalonic

Acidemia

mRNA-3927 Moderna Propionic Acidemia

Infectious Diseases mRNA-1944 Moderna / DARPA Chikungunya Ab Ongoing

Started: Nov ‘16

Fully enrolled

Started: Aug ‘17

Met 1°/2° endpoints

Fully enrolled

Started: Dec ‘16

Started: Aug ‘17

Started: Nov ‘17

Started: Dec ‘17

Started: Nov ‘17

Started: Jan ‘18

© 2018 Moderna Therapeutics

Page 26: 36th Annual J.P. Morgan Healthcare Conference Therapeutics JPM 2018...Slide 2 This presentation does not constitute an offer to sell to, or a solicitation of an offer to buy from,

Slide 26

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Page 27: 36th Annual J.P. Morgan Healthcare Conference Therapeutics JPM 2018...Slide 2 This presentation does not constitute an offer to sell to, or a solicitation of an offer to buy from,

Slide 27

• Execute on our 19 DCs currently in the pipeline – initiate several phase 2 trials

• Create important new DCs in our existing 5 modalities, with a priority on rare diseases

• Continue to build the infrastructure to deliver on the promise of our broad mRNA platform

• Continue to invest in mRNA science and technology. It is still only the beginning

2018-2019 Priorities

© 2018 Moderna Therapeutics