Analyst Conference Summary

biotechnology

conference date: March 6, 2019
for quarter ending: December 31, 2018 (fourth quarter 2018)

Forward-looking statements

Overview: Clinical stage mRNA company continues to advance its pipeline. This is the first quarter reported since Moderna had its initial public stock offering in December 2018.

Basic data (GAAP):

Revenue was $35.4 million, down from $91.9 million year-earlier. All revenue is from collaboration and grants.

Net income was negative $141.4 million, down from negative $37.9 million year-earlier.

EPS was not stated, nor were the number of shares outstanding. But a public source listed 324.4 million shares outstanding.

Guidance:

Cash at the end of 2019 is expected between $1.15 and $1.20 billion.

Conference Highlights:

Stephane Bancel, Moderna's CEO, said "Execution by our team has enabled us to make important pipeline progress so far this year. We now have two additional programs ready for Phase 2 clinical development, newly opened INDs for our first rare disease program and a fifth immuno-oncology program, dosed the first cohort in a study of our systemically delivered mRNA that encodes for a secreted monoclonal antibody, and recently reported positive interim Phase 1 data for a novel combination vaccine designed to protect against viruses that can cause severe respiratory diseases in children. We look forward to generating new clinical data for programs across our portfolio over the next 12-24 months. Our strong cash position enables us to focus on advancing investigational medicines in our pipeline, pursue new candidates within our existing modalities and continue to invest in our mRNA platform to discover new modalities and treatments for patients across a broader range of disease areas."

Moderna currently has 20 mRNA candidates, with 11 in clinical studies

Working on vaccines first was part of a risk management strategy.

In February Moderna announced positive data from an interim analysis of safety and immunogenicity from its Phase 1 study of mRNA-1653 in healthy adults. mRNA-1653 is designed to protect against human Metapneumovirus (hMPV) and Parainfluenza Virus Type 3 (PIV3), that cause respiratory illnesses. Moderna plans to advance mRNA-1653 into a Phase 1b study of pediatric subjects.

Moderna's follow-on Zika vaccine candidate, mRNA-1893, continues to progress toward an IND filing. There will be no further development of Moderna's first Zika candidate, mRNA-1325. The Biomedical Advanced Research and Development Authority remains committed to its grant of up to approximately $125 million for development.

In February Moderna and Merck submitted a new protocol to the FDA for a randomized Phase 2 study to assess whether post-operative adjuvant therapy with mRNA-4157, in combination with Merck's Keytruda, improves recurrence-free survival compared to Keytruda alone. The study will be conducted with patients that have had complete resection of cutaneous melanoma but remain at high risk of recurrence

mRNA-2752 has finished dosing of the first cohort of patients in the Phase 1 study and begun dosing of a second cohort. mRNA-2752, also known as the Triplet, is an intratumoral injection comprising three mRNAs encoding for OX40L + IL23 + IL36 for the treatment of advanced or metastatic solid tumor malignancies or lymphoma. mRNA-2416, OX40L only, amendment filed for Phase 2 cohort, had 2 patients with some regression in ovarian cancer in Phase 1.

An IND has been opened for a Phase 1 study of MEDI1191, encoding IL12, injected intratumorally in advanced or metastatic solid tumors with collaborator AstraZeneca. Will be studied as a monotherapy and in combination with a checkpoint inhibitor.

Moderna announced the publication of data from a Phase 1a/b study in Nature Communications showing the potential of mRNA encoding for VEGF-A (vascular endothelial growth factor A) as a regenerative therapeutic. The data supported advancement of AZD8601, which now is in an ongoing Phase 2a study led by AstraZeneca.

Dosing of the first Chikungunya Virus cohort has been completed in Moderna's Phase 1 study evaluating the safety and tolerability of escalating doses of mRNA-1944 via intravenous infusion in healthy adults.

(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 $1.69 billion.

Operating expense (GAAP) of $188.5 millon consisted of $150.4 million for R&D and $38.0 million for SG&A. Operating income was negative $153.0 million. $8.9 million interest income; $2.9 million other income. Income tax provision $0.2 million.

For the full year 2018 the net loss was $385 million and $331 million cash was used in operating activities. Cash used for capital expenditures was $106 million.

Q&A summary:

PCV, why chosen as indication? Trial design? Because in cancer drugs the effect tends to be greater in early lines of disease and the adjuvant setting. Expected to be true for melanoma. The likelihood of success is highest. Power is typical for a proof of concept study.

Is there a point where you need to pick between and 2416 and 2752? It is too early to predict which will do better in the trials. We are starting the Triplet at a lower dose, but that should not cause a significant delay.

Single v. multiple doses in rare diseases? We start with a single ascending dose for safety. Based on preclinical data, multiple doses should be as safe.

MMA timeline? No guidance, emphasis on safety. We will give regular enrollment updates.

PCV decision to move to Phase 2? Totallity of immunogenicity data, tolerability, safety at 1 mg dose.

PCV epitope spreading data? Antigen spreading was not a focus. The goal was to establish a dose against epitopes. More data in later trials.

hMPV+PIV3 study? Note the first exposure will be in sero-positives. There is a concern of disease exacerbation. The nice result in Phase 1 was reaching a plateau dose so soon. Still in design phase, need to sit down with regulators before releasing the full design.

Discussions with FDA have been positive, as with EU, the FDA has been helpful and has provided deep insights. We don't see any one area of being easier to get through the FDA.

Cap ex should be down this year v. 2018 because we spent so much on the Norwood plant buildout, but are not giving specific guidance.

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