Analyst Conference Summary

biotechnology

conference date: November 6, 2019 @ 5:00 AM Pacific Time
for quarter ending: September 30, 2019 (third quarter, Q3 2019)

Forward-looking statements

Overview: Two positive results from Phase I trials. Moderna had its initial public stock offering in December 2018. Moderna has lots of cash, but is burning through it quickly.

Basic data (GAAP):

Revenue was $17.0 million, up sequentially from down from $13.1 million, and down from $41.8 million year-earlier. All revenue is from collaboration and grants.

Net income was negative $123.2 million, up sequentially from negative $135.1 million, and down from negative $80.3 million year-earlier.

EPS was not stated, but calculates to negative $0.37, sequentially from not stated, and down from not stated year-earlier. It is strange that the company does not report EPS.

Guidance:

Expects to end the year with about $1.2 billion in cash and equivalents. In 2020, Moderna net cash used for operating activities and capital expense to be between $490 million and $510 million.

Conference Highlights:

Stephane Bancel, Moderna's CEO, said "The positive Phase 1 cytomegalovirus vaccine results announced this quarter represent an important step toward the prevention of congenital CMV infections. Our mRNA technology platform has now generated six positive infectious disease vaccine clinical readouts. Additionally, the recent positive Phase 1 chikungunya antibody results help de-risk a delivery technology shared by our rare disease programs, further validating our approach into new areas where mRNA medicines have the potential to treat a wide range of diseases. We ended the quarter with a strong cash balance of $1.34 billion and up to $187 million in additional untapped grant funding, giving us up to $1.5 billion to invest in the Company moving forward. We expect investment levels in 2020 to be approximately $500 million, similar to 2019."

Moderna currently has 21 mRNA candidates, with 13 now in clinical studies

Working on vaccines first was part of a risk management strategy and continues to prioritize risk management.

Moderna's Phase 1 study evaluating the safety and tolerability of escalating doses of mRNA-1944 for Chikungunya Virus had positive interim results.

Moderna announced a multi-year mRNA immunotherapy research collaboration with Harvard University to explore fundamental immunological processes and identify potential therapeutic opportunities.

In q2 2019 the first subject was dosed in the Phase 1, mRNA-1172 or Merck V172, RSV vaccine (Respiratory syncytial virus ) study. As a result, further development of mRNA-1777 has been paused and next steps will be determined based on data from the new mRNA-1172 Phase 1 study.

The OX40L (mRNA-2416) Phase 2 expansion cohort in ovarian cancer will focus only on the combination with durvalumab (Imfinzi).

Moderna's Phase 1 study of mRNA-1647, Cytomegalovirus (CMV) vaccine, is fully enrolled and reported positive interim results in Q3. The study is randomized, observer-blind and placebo-controlled. 25,000 newborns infected in US each year, has proven difficult to vaccinate against. 6 RNA sequences are included in 1647.

The first participant 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 first subject was dosed for 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. 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.

Moderna has selected a new development candidate for the rare inherited metabolic disease GSD1a. 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.

(mRNA-3704): The FDA has granted Fast Track designation for mRNA-3704 for MMA (Methylmalonic Acidemia). Moderna now has an open IND and is preparing to begin a Phase 1/2 open-label, multi-center, multiple ascending dose study of mRNA-3704 in pediatric patients with isolated MMA due to MUT enzyme deficiency.

Cash ended the quarter at $1.34 billion, down sequentially from $1.44 billion. Cash used in operations was $107 million. Capital expense was $7 million.

Operating expense (GAAP) of $ millon consisted of $ million for R&D and $ million for SG&A. Operating income was negative $ million. $ million interest income; $ million other expense. Income tax benefit $ million.

Q&A summary:

Progress in MMA enrollment? Not yet dosed first subject. FDA wanted first 3 subjects 12 and above. By the time a severe MMA child is 12 they have usually had a liver transplant, so ineligible. Those without liver transplants typically have kidney damage, so also ineligible. Now we believe the FDA will lower the age to 8.

Chikungunya data timeline? Healthy volunteer trial. We are working to get the next cohorts enrolled.

CMV path forward? The Phase 2 is about 250 subjects, mainly to confirm the dose. The time should be quick, based on a 3-month endpoint. Will need to get regulatory guidance before launching a Phase 3 trial, then it should take a couple of years of follow up.

Merck enhanced RSV vaccine? You will know as soon as Merck lets us know.

Merck is running the KRAS study, so they will report the data.

8601 with AstraZeneca? They designed a very informative pharmacology trial. Challenge is the half life of the isotope used. They have been adding sites with this capability. Once they enroll the subjects it is six months to the endpoing.

Phase 3 will focus on sero negatives because it is hard to display good results with sero positives. But we want safety and tolerability in the label for the whole population.

PA dosing strategy? Similar to MMA, based on preclinical data. Once every two or three weeks. Lots of biomarkers to look at, we will narrow that, it is not as clear for PA as it is for MMA.

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