Analyst Conference Summary

biotechnology

conference date: May 7, 2020 @ 5:00 AM Pacific Time
for quarter ending: March 31, 2020 (first quarter, Q1 2020)

Forward-looking statements

Overview: Rapid pipeline developing and an astonishing amount of funding.

Basic data (GAAP):

Revenue was $8.4 million, down sequentially from down from $14.1 million, and down from $16.0 million year-earlier. All revenue is from collaboration and grants.

Net income was negative $124.2 million, flat sequentially from negative $124.2 million, and up from negative $132.6 million year-earlier.

EPS (diluted) was negative $0.35, down sequentially from negative $0.37, and down from negative $0.40 year-earlier.

Guidance:

Conference Highlights:

Stephane Bancel, Moderna's CEO, said "The imminent Phase 2 study start is a crucial step forward as we continue to advance the clinical development of mRNA-1273, our vaccine candidate against SARS-CoV-2. With the goal of starting the mRNA-1273 pivotal Phase 3 study early this summer, Moderna is now preparing to potentially have its first BLA approved as soon as 2021. We are accelerating manufacturing scale-up and our partnership with Lonza puts us in a position to make and distribute as many vaccine doses of mRNA-1273 as possible, should it prove to be safe and effective. We also are continuing to progress our development pipeline and invest in our future. We are very pleased with Vertex's decision, based on our preclinical progress, to extend our strategic collaboration working to develop the technology to allow for delivery of mRNA in the lung."

Moderna currently has 24 mRNA candidates, with 12 now in clinical studies. The goal of the next phase of development is to submit multiple BLAs to the FDA.

Raised $550 million cash after the quarter ended, in Feb. 2020.

In Q1 2020 positive interim results announced from Phase 1 Zika vaccine candidate (mRNA-1893) study.

The first clinical-grade batch of novel coronavirus vaccine (mRNA-1273) shipped to the NIH for use in Phase 1 study in the U.S. in Q1 2020; delivered from Company’s cGMP facility in 42 days from sequence selection. Adding cohorts to Phase 1 trial. Near starting the Phase 2 study. Phase 3 study could start summer of 2020. Working on creating manufacturing capacity for one million doses per year, with a partner working on expanding that to 1 billion per year. Barda award (up to $483 million) for development is not cash up front, but will reimburse expenses. Has another $180 million in grant funding.

Cytomegalovirus (CMV) vaccine mRNA-1647 Phase 2 study enrollment is near completion, with interim data expected in Q3 2000. The Phase 1 study reported positive interim results in Q3 1019. 25,000 newborns infected in US each year, has proven difficult to vaccinate against. 6 RNA sequences are included in 1647. Estimates annual peak sales would be $2 to $5 billion if the vaccine is approved and adopted globally.

Three new vaccines were announced in February 2020: mRNA-1345 for pediatric RSV; mRNA-1189 for EBV; and mRNA-1273 for SARS-CoV-2.

The OX40L (mRNA-2416) Phase 2 expansion cohort in ovarian cancer will focus only on the combination with durvalumab (Imfinzi). Interim Phase 1/2 data was positive, as reported at AACR.

The collaboration with Vertex for cystic fibrosis was extended through August 2021.

In 2019 the first participant was dosed in Phase 1b age de-escalation study of hMPV+PIV3 vaccine (mRNA-1653). This is a Phase 1b study of pediatric subjects protect against human Metapneumovirus (hMPV) and Parainfluenza Virus Type 3 (PIV3).

Moderna's follow-on Zika vaccine candidate, mRNA-1893, continues to progress toward an IND filing. The FDA granted Fast Track designation for this Zika vaccine. The Biomedical Advanced Research and Development Authority remains committed to its grant of up to approximately $125 million for development.

The randomized Phase 2 study to assess whether post-operative adjuvant therapy with mRNA-4157, in combination with Merck's Keytruda, improves recurrence-free survival in adjuvant cutaneous melanoma compared to Keytruda alone continues. The Phase 2 patients are at 34 neoantigens, more than the Phase 1 trial used.

The Phase 1/2 MMA (mRNA-3704) study is actively recruiting patients following a protocol amendment expanding the eligibility criteria to patients 8 years and older

The Phase 1 KRAS cancer vaccine (mRNA-5671 or Merck V941) study continues. About 20% of human cancers are driven by mutated KRAS. 5671 codes for the most common mutations.

The Phase 1 study of mRNA encoding IL12 (MEDI1191) injected intratumorally continues. Collaborator is AstraZeneca

The FDA granted Fast Track designation to mRNA-3927 for PA ; study start-up is ongoing for the open-label, multi-center Phase 1/2 study of multiple ascending doses of mRNA-3927 in pediatric patients with PA in the U.S.

Inherited metabolic disease GSD1a results in a buildup of glycogen in tissues and an inability to regulate glucose, leading to life-threatening hypoglycemia and long-term liver and kidney damage. mRNA-3745 is an IV-administered mRNA encoding G6Pase enzyme, designed to restore deficient or defective intracellular enzyme activity.

Three clinical trial sites are open and actively recruiting patients for the Phase 1/2 open-label, dose escalation study evaluating mRNA-3704 for the treatment of MMA, or Methylmalonic acidemia.

AZD8601, which now is in an ongoing Phase 2a study led by AstraZeneca.

First patient enrolled in Phase 1/2 MMA (mRNA-3704) study; actively recruiting patients at U.S. sites following a protocol amendment expanding the first cohort eligibility criteria to patients 8 years and older. The FDA previously granted Fast Track designation for mRNA-3704 for MMA (Methylmalonic Acidemia).

Cash ended the quarter at $1.72 billion, up sequentially from $1.26 billion. In Q1 2020 $550 million more was raised with a share offering. $106 million cash used in operating activities. $6 million used for cap ex.

Operating expense (GAAP) of $139 millon consisted of $115 million for R&D and $24 million for SG&A. Operating income was negative $131 million. $8 million interest income; $1 million other expense. Income tax $0 million.

Q&A summary:

50 mic dose for COVID, v. other doses? 50 mic is our current best guess. But no decision, there are balancing factors. The immune response is usually measured by neutralizing antibodies. The emerging preclinical data is consistent with our antibody hypothesis.

Phase 3 COVID design? Any pivotal trial needs to be placebo controlled and have sufficient subjects. You have to predict the attack rate; if less are exposed than planned, you don't know if the vaccine worked. Will need to talk to NAIAD, NIH, and FDA. Timeline depends on those decisions, not made yet. Probably not a binary event, but emergence of data. Endpoints could be COVID-19 disease symptoms and infection per se.

Emerging mutations? So far none are expected to interfere with the antibody spike protein, which our vaccine codes for. If a mutant needing addressing does emerge, our platform allows for a fast response. We would not need to do the entire Phase 1 through 3 testing for the new version.

CMV update? We remain on track. We have vaccinated over 70% with second dose. With this larger Phase 2 study we will be powered to validate safety. We are on track to start a Phase 3 trial.

Oldest age in new cohorts of patients? High risk patients? In Phase 2 there is no upper limit for the over 55 age group. We need to get responsibly towards high risk patients, but not in Phase 2. We will do that in Phase 3, carefully.

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