Shows Continued Execution Across its Pipeline
of Infectious Disease, Immuno-Oncology and Rare Disease
Programs
Ends Year With $1.7 Billion in Cash, Cash
Equivalents and Investments
Moderna, Inc. (Nasdaq: MRNA), a clinical stage biotechnology
company pioneering messenger RNA (mRNA) therapeutics and vaccines
to create a new generation of transformative medicines for
patients, today reported financial results for the fourth quarter
and full year of 2018 and highlighted pipeline progress since the
Company’s last corporate update in January.
New updates announced today include:
Infectious Diseases
- Moderna is preparing an IND for
submission to the US Food and Drug Administration (FDA) for a
follow-on Zika vaccine program (mRNA-1893); no further development
planned for its first Zika vaccine candidate (mRNA-1325)
Immuno-Oncology
- Randomized Phase 2 protocol submitted
to the FDA for personalized cancer vaccine (PCV) (mRNA-4157) study
in patients with resected melanoma
- IND opened for Phase 1 study of mRNA
encoding IL12 (MEDI1191) injected intratumorally in solid
tumors
Rare Diseases
- FDA grants Fast Track designation for
methylmalonic acidemia (MMA) program (mRNA-3704); IND opened for
Phase 1/2 study of pediatric patients
“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,” said Stéphane Bancel, Moderna’s
chief executive officer. “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 development candidates in its
portfolio, with 11 in clinical studies. Across Moderna's pipeline
more than 1,000 subjects have been enrolled in clinical studies.
The Company’s updated pipeline can be found at
www.modernatx.com/pipeline.
Summary of Recent Highlights by Modality
Prophylactic vaccines:
Moderna is developing vaccines against viral diseases where
there is unmet medical need – including complex vaccines with
multiple antigens for common diseases, as well as vaccines against
epidemic and pandemic threats to global public health.
- hMPV+PIV3 (mRNA-1653): In
February, Moderna announced positive data from a planned 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), two viruses that cause respiratory illnesses. It is a
combination vaccine that consists of two distinct mRNA sequences
encoding the fusion (F) proteins of hMPV and PIV3 formulated in
Moderna’s proprietary lipid nanoparticle (LNP) technology. Moderna
plans to advance mRNA-1653 into a Phase 1b study of pediatric
subjects.
- Zika Vaccine (mRNA-1893):
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 (BARDA) remains
committed to its grant of up to approximately $125 million for
development of a Zika vaccine.*
- Publication of Note: In
February, Moderna researchers published new data in the scientific
journal Molecular Therapy: Nucleic Acids that demonstrate how mRNA
vaccines delivered with a proprietary Moderna lipid nanoparticle
(LNP) show enhanced tolerability and comparable immunogenicity
relative to legacy LNPs.
Cancer Vaccines: These programs focus on stimulating
a patient’s immune system with antigens derived from tumor-specific
mutations to enable the immune system to elicit a more effective
antitumor response.
- Personalized Cancer Vaccine (PCV)
(mRNA-4157): In February, Moderna and Merck submitted a new
protocol to the FDA to commence a randomized Phase 2 study to
assess whether post-operative adjuvant therapy with mRNA-4157, in
combination with Merck’s PD-1 inhibitor KEYTRUDA®, improves
recurrence-free survival compared to KEYTRUDA alone. The study has
a primary endpoint of recurrence-free survival with a primary
analysis at 12 months and will be conducted with patients that have
had complete resection of cutaneous melanoma but remain at high
risk of recurrence.Moderna’s PCV is designed and manufactured
individually based on the DNA sequence of a patient’s tumor,
encoding for peptides containing mutations found in their cancer in
order to deliver multiple unique and personalized neoantigens in a
single vaccine. Moderna’s PCV now includes up to 34 neoantigens, up
from 20. Moderna has also fully operationalized its personalized
vaccine unit at its manufacturing site in Norwood, Mass., which is
expected to further enhance supply chain management and enable the
Company to accelerate manufacturing of individualized cancer
treatments for patients.
Intratumoral Immuno-oncology: These programs
aim to drive anti-cancer T cell responses by injecting mRNA
therapies directly into tumors.
- OX40L + IL23 + IL36γ (Triplet)
(mRNA-2752): mRNA-2752 has cleared dosing of the
first cohort of patients in the Phase 1 study and the dosing of a
second cohort has commenced. mRNA-2752, also known as the Triplet,
is an intratumoral injection comprising three mRNAs encoding for
OX40L + IL23 + IL36γ for the treatment of advanced or metastatic
solid tumor malignancies or lymphoma. The open-label, multi-center
study is evaluating the safety and tolerability of mRNA-2752 as a
single agent and in combination either with AstraZeneca’s
durvalumab or tremelimumab, and will assess anti-tumor activity,
protein expression in tumors and pharmacokinetics.
- IL12 (MEDI1191): An IND has been
opened for a Phase 1 study of mRNA encoding IL12 injected
intratumorally in advanced or metastatic solid tumors. Moderna's
strategic collaborator AstraZeneca will lead this open-label,
multi-center study of intratumoral injections of MEDI1191 alone and
in combination with a checkpoint inhibitor. Moderna provided the
preclinical data package to support the IND submission and will
provide clinical supply for this trial. MEDI1191 is an mRNA
encoding for IL12, a potent immunomodulatory cytokine, which aims
to enhance immune response in immunologically “cold” tumors.
- Publication of Note: In January,
Moderna announced the publication of pre-clinical data in the
scientific journal Science Translational Medicine that showed
local delivery of the Triplet (mRNA-2752) induced a broad immune
response and caused tumor regression in both injected lesions and
distant un-injected tumors in mice. When combined with checkpoint
inhibitors, mRNA-2752 was able to induce responses in tumor models
that are otherwise unresponsive to checkpoint inhibitors.
Localized Regenerative Therapeutics: These programs focus
on the potential for the localized production of proteins to be
used as a regenerative medicine for damaged tissues.
- Publication of Note: In
February, Moderna announced the publication of data from a Phase
1a/b study in Nature Communications showing the potential of mRNA
encoding for vascular endothelial growth factor A (VEGF-A) as a
regenerative therapeutic. When injected directly into the skin of
patients with diabetes mellitus, the mRNA encoding VEGF-A was well
tolerated, showed protein expression as demonstrated by
dose-dependent protein translation and demonstrated protein
pharmacology with evidence of increased blood flow. The data
supported advancement of AZD8601, which now is in an ongoing Phase
2a study led by AstraZeneca.
Systemic Secreted Therapeutics: In this modality, mRNA is
delivered systemically to create proteins that are secreted outside
the cell with the aim of producing pharmaceutically active proteins
with therapeutic effects across the human body.
- Antibody Against the Chikungunya
Virus (mRNA-1944): Dosing of the first 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. This is the first monoclonal antibody
encoded by mRNA to be dosed in a human and the first development
candidate from the Company’s systemic therapeutics modalities to
start clinical testing. Moderna announced the dosing of the first
patient in the study in February. mRNA-1944 encodes a fully human
IgG antibody originally isolated from B cells of a patient with a
prior history of potent immunity against chikungunya infection and
is composed of two mRNAs that encode the heavy and light chains of
this anti-chikungunya antibody within Moderna’s proprietary lipid
nanoparticle (LNP) technology. This formulation was developed by
Moderna and is utilized for IV delivery of each of its systemic
therapeutics, including its rare disease programs.
Systemic Intracellular Therapeutics: These programs
aim to deliver mRNA into cells within target organs as a
therapeutic approach for diseases caused by a missing or defective
protein.
- Methylmalonic Acidemia (MMA)
(mRNA-3704): The FDA has granted Fast Track designation for
mRNA-3704, the first for a Moderna investigational medicine.
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. The objectives of the study are to evaluate safety and
tolerability, assess the pharmacodynamic response and characterize
the pharmacokinetic profile of mRNA-3704. The program previously
received Rare Pediatric Disease Designation by
the FDA and Orphan Drug Designation by both
the FDA and the European Medicines Agency (EMA).
This is Moderna’s first rare disease program to advance into
clinical trials.
Information about each program in Moderna’s pipeline, including
those discussed in this press release, can be found on the investor
relations page of its website https://investors.modernatx.com/.
Fourth Quarter and Full Year 2018 Financial Results
- Cash Position: Cash, cash
equivalents and investments as of December 31, 2018 and December
31, 2017 were $1.7 billion and $0.9 billion, respectively.
- Net Cash Used in Operating
Activities: Net cash used in operating activities was $330.9
million for the year ended December 31, 2018 compared to $331.5
million for the year ended December 31, 2017.
- Cash Used for Purchases of Property
and Equipment: Cash used for purchases of property and
equipment was $105.8 million for the year ended December 31, 2018
compared to $58.4 million for the year ended December 31, 2017. Of
these amounts, cash disbursements specifically related to the
Norwood manufacturing facility were $94.5 million and $41.2 million
for the years ended December 31, 2018 and 2017, respectively.
The Norwood plant opened in July 2018.
- Revenue: Total revenue was $35.4
million for the fourth quarter of 2018 compared to $91.9 million
for the fourth quarter of 2017. Total revenue was $135.1 million
for the year ended December 31, 2018 compared to $205.8 million for
the year ended December 31, 2017. The decreases in both periods
were mainly attributable to the termination of the Alexion
strategic alliance arrangement in October 2017, and a decrease in
grant revenue from the BARDA contract, primarily due to revisions
to the Zika program and a focus on preclinical studies of
mRNA-1893, the follow on to mRNA-1325. The decreases were partially
offset by increases in collaboration revenue from AstraZeneca and
Merck.
- Research and Development
Expenses: Research and development expenses were $150.4 million
for the fourth quarter of 2018 compared to $117.8 million for the
fourth quarter of 2017. Research and development expenses were
$454.1 million for the year ended December 31, 2018 compared to
$410.5 million for the year ended December 31, 2017. The increases
in both periods were primarily due to an increase in personnel
related cost, including stock-based compensation, mainly driven by
an increase in the number of employees supporting research and
development programs, an increase in consulting and outside
services, and an increase in facility and equipment related
costs.
- General and Administrative
Expenses: General and administrative expenses were $38.0
million for the fourth quarter of 2018 compared to $15.9 million
for the fourth quarter of 2017. General and administrative expenses
were $94.3 million for the year ended December 31, 2018 compared to
$64.7 million for the year ended December 31, 2017. The increases
in both periods were mainly attributable to increases in personnel
related costs, including stock-based compensation, primarily driven
by an increase in the number of employees, and consulting and
outside services costs, both of which were in support of public
company readiness.
- Net Loss: Net loss was $141.4
million for the fourth quarter of 2018 compared to $37.9 million
for the fourth quarter of 2017. Net loss was $384.7 million for the
year ended December 31, 2018 compared to $255.9 million for the
year ended December 31, 2017.
2019 Expected Cash Position
Cash, cash equivalents and investments at December 31, 2019 are
expected to be in the range of $1.15 billion to $1.20 billion.
Other Corporate Updates
- Moderna Added to Russell
Indexes: In February 2019, Moderna was selected for addition to
the Russell 1000® and Russell 3000® indexes as part of the Russell
Investments’ quarterly reconstitution, effective March 18, 2019.
FTSE Russell determines membership for its Russell U.S. Indexes
primarily by objective, market-capitalization rankings and style
attributes. Approximately $9 trillion in assets are benchmarked
against Russell U.S. Indexes.
- Company Management: Moderna
today announced the appointment of Lavina Talukdar, who will join
the Company in April as head of investor relations. Ms. Talukdar
joins Moderna from the Abu Dhabi Investment Authority (ADIA) where
she serves as senior portfolio manager. She was previously a
partner and research analyst at Lord Abbett and a senior research
analyst at MFS Investment Management.
Annual Company Events
Moderna today announced that its annual Science Day will take
place on May 7, 2019 in Cambridge, Mass. and its annual R&D Day
will take place on September 12, 2019 in New York City.
Investor Call and Webcast Information
Moderna will host a live conference call and webcast
at 8:00 a.m. ET on Wednesday, March 6, 2019. To access
the call, please dial 866-922-5184 (domestic) or 409-937-8950
(international) and refer to conference ID 8294495. A webcast of
the call will also be available under “Events & Presentations”
in the Investors section of the Moderna website at
https://investors.modernatx.com/. The archived webcast will be
available on Moderna’s website approximately two hours after the
conference call and will be available for 30 days following the
call.
About Moderna
Moderna is advancing messenger RNA (mRNA) science to create a
new class of transformative medicines for patients. mRNA medicines
are designed to direct the body’s cells to produce intracellular,
membrane or secreted proteins that can have a therapeutic or
preventive benefit and have the potential to address a broad
spectrum of diseases. Moderna’s platform builds on continuous
advances in basic and applied mRNA science, delivery technology and
manufacturing, providing Moderna the capability to pursue in
parallel a robust pipeline of new development candidates. Moderna
is developing therapeutics and vaccines for infectious diseases,
immuno-oncology, rare diseases and cardiovascular diseases,
independently and with strategic collaborators.
Headquartered in Cambridge, Mass., Moderna currently has
strategic alliances for development programs with AstraZeneca, Plc.
and Merck, Inc., as well as the Defense Advanced Research Projects
Agency (DARPA), an agency of the U.S. Department of Defense, and
the Biomedical Advanced Research and Development Authority (BARDA),
a division of the Office of the Assistant Secretary for
Preparedness and Response (ASPR) within the U.S. Department of
Health and Human Services (HHS). Moderna has been ranked in the top
ten of Science’s list of top biopharma industry employers for
the past four years. To learn more,
visit www.modernatx.com.
KEYTRUDA is a registered trademark of Merck Sharp & Dohme
Corp., a subsidiary of Merck & Co., Inc.
* This project has been funded in whole or in part with
Federal funds from the Department of Health and Human Services;
Office of the Assistant Secretary for Preparedness and Response;
Biomedical Advanced Research and Development Authority, under
Contract No. HHSO100201600029C.
Forward Looking Statement
This press release 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: the planned next steps in the Company’s pipeline
programs and specifically including, but not limited to, statements
regarding the Company’s plans regarding a Phase 1/2 study of
mRNA-3704 for methylmalonic acidemia (MMA); plans to initiate a
Phase 1 study of mRNA-1893, a Zika vaccine; plans by AstraZeneca to
initiate a Phase 1 clinical trial for MEDI1191 an mRNA for IL12,
following the opening of the filed IND; plans to initiate a Phase
1b study of mRNA-1653, a combination vaccine against hMPV and PIV3;
and the Company’s cash, cash equivalents, and investments at
December 31, 2019. In some cases, forward-looking statements can be
identified by terminology such as “will,” “may,” “should,”
“expects,” “intends,” “plans,” “aims,” “anticipates,” “believes,”
“estimates,” “predicts,” “potential,” “continue,” or the negative
of these terms or other comparable terminology, although not all
forward-looking statements contain these words. The forward-looking
statements in this press release are neither promises nor
guarantees, and you should not place undue reliance on these
forward-looking statements because they involve known and unknown
risks, uncertainties, and other factors, many of which are beyond
the Company’s control and which could cause actual results to
differ materially from those expressed or implied by these
forward-looking statements. These risks, uncertainties, and other
factors include, among others: preclinical and clinical development
is lengthy and uncertain, especially for a new category of
medicines such as mRNA, and therefore our preclinical programs or
development candidates may be delayed, terminated, or may never
advance to or in the clinic; no mRNA drug has been approved in this
new potential category of medicines, and may never be approved;
mRNA drug development has substantial clinical development and
regulatory risks due to the novel and unprecedented nature of this
new category of medicines; and those risks and uncertainties
described under the heading “Risk Factors” and those described in
Moderna’s Prospectus filed with the U.S. Securities and
Exchange Commission (SEC) on December 7, 2018 and in
subsequent filings made by Moderna with the SEC,
which are available on the SEC's website
at www.sec.gov. Except as required by law, Moderna disclaims
any intention or responsibility for updating or revising any
forward-looking statements contained in this press release in the
event of new information, future developments or otherwise. These
forward-looking statements are based on Moderna’s current
expectations and speak only as of the date hereof.
MODERNA, INC.
CONDENSED CONSOLIDATED STATEMENTS OF
OPERATIONS
(Unaudited, in thousands)
Three Months Ended December
31,
Years EndedDecember 31,
2018 2017 2018 2017
Revenue: Collaboration revenue $ 32,816 $ 88,416 $ 122,512 $
176,974 Grant revenue 2,605 3,488 12,556
28,851 Total revenue 35,421 91,904 135,068
205,825 Operating expenses: Research and development
150,429 117,827 454,082 410,459 General and administrative 38,023
15,905 94,252 64,722 Total operating
expenses 188,452 133,732 548,334 475,181
Loss from operations (153,031 ) (41,828 ) (413,266 )
(269,356 ) Interest income 8,894 3,783 27,023 15,235 Other income
(expense), net 2,879 (70 ) 1,835 (1,875 ) Loss before
provision for (benefit from) income taxes (141,258 ) (38,115 )
(384,408 ) (255,996 ) Provision for (benefit from) income taxes 168
(171 ) 326 (80 ) Net loss $ (141,426 ) $ (37,944 ) $
(384,734 ) $ (255,916 )
MODERNA, INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
AND STATEMENTS OF CASH FLOWS DATA
(Unaudited, in thousands)
December 31, 2018 2017
Cash, cash equivalents and investments $ 1,694,417 $ 901,880 Total
assets 1,962,149 1,084,489 Total liabilities 431,908 459,193 Total
stockholders’ equity (deficit) 1,530,241 (551,365 )
Years
Ended December 31, 2018 2017 Net cash used in
operating activities $ (330,865 ) $ (331,484 ) Cash used for
purchases of property and equipment (1) (105,766 ) (58,401 )
(1) Includes $94.5 million and $41.2 million for the years ended
December 31, 2018 and 2017, respectively, related to our Norwood
manufacturing facility.
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version on businesswire.com: https://www.businesswire.com/news/home/20190306005193/en/
Media:Jason GlashowHead of Corporate
Communications617-674-5648jason.glashow@modernatx.com
Investors:Lorence KimChief Financial
Officer617-209-5849lorence.kim@modernatx.com
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