Rare Diseases: New development candidate
announced for glycogen storage disease type 1a (GSD1a), a rare
metabolic disorder; Company now has five rare disease programs in
its pipeline
Immuno-Oncology: Two personalized cancer
vaccine (PCV) abstracts to be presented at the 2019 ASCO Annual
Meeting
Infectious Diseases: Merck submitted an IND for
mRNA-1172, a more potent RSV vaccine development candidate;
development paused for first RSV candidate, mRNA-1777
Ended quarter with $1.55 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 first quarter of
2019 and provided business updates.
“We continue to execute against our corporate objectives as we
progress clinical studies across our development portfolio,
introduce a new mRNA rare disease development candidate and focus
on identifying additional modalities and disease targets,” said
Stéphane Bancel, Moderna’s chief executive officer. “We are excited
to pursue a treatment to potentially address the underlying cause
of glycogen storage disease type 1a, and we believe this candidate
also reflects the continued productivity of our mRNA platform. At
yesterday’s annual Science Day event, we presented new insights
into our mRNA and delivery science, including the potential
delivery of mRNA to white blood cells. While our team has
additional research to perform in this area, we look forward to
being able to bring new candidates into development as we continue
working to help patients with a wide range of serious
diseases.”
Moderna currently has 21 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.
- Respiratory syncytial virus
(RSV) vaccine (mRNA-1777 and mRNA-1172): Merck has filed
an investigational new drug (IND) application with the U.S. Food
and Drug Administration (FDA) and plans to run a Phase 1 study for
a follow-on development candidate (mRNA-1172), a vaccine for RSV
which has shown enhanced potency in preclinical studies and uses a
Merck proprietary formulation. 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.
- Varicella zoster virus (VZV)
vaccine (mRNA-1278): Based on an assessment of the commercial
opportunity, research priorities and other factors, Merck has
discontinued preclinical development of mRNA-1278, an
investigational vaccine for VZV (the virus that causes shingles).
Merck has returned rights to Moderna, and the Company will not
continue development at this time.
- Cytomegalovirus (CMV) vaccine
(mRNA-1647): The first three dose levels in Moderna’s
ongoing study of mRNA-1647 are fully enrolled, and the study is
currently enrolling subjects into the fourth (300μg) dose cohort.
The Phase 1 study is randomized, observer-blind and
placebo-controlled with the goal of evaluating the safety and
immunogenicity of mRNA-1647, a vaccine against the pentamer and gB
complexes of CMV.
- Presentation of note: Moderna
presented data from its Phase 1 studies of vaccines against viruses
that cause respiratory diseases at the European Society for
Pediatric Infectious Disease (ESPID) meeting held in Ljubljana,
Slovenia.
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
anti-tumor response.
- Personalized cancer vaccines (PCVs)
(mRNA-4157, NCI-4650): Two abstracts for Moderna PCV programs
were accepted for presentation at the 2019 American Society of
Clinical Oncology (ASCO) Annual Meeting. Moderna will present new
updates to clinical data from the Company’s Phase 1 study of
mRNA-4157, a PCV being studied alone in patients with resected
solid tumors and in combination with Merck’s pembrolizumab in
patients with unresectable solid tumors. The National Cancer
Institute (NCI) will also present data from its Phase 1 study of
PCV NCI-4650, a monotherapy for patients with advanced metastatic
cancers. The two abstracts are:
- Moderna: A Phase 1 multi-center study
to assess the safety, tolerability and immunogenicity of mRNA-4157
alone in patients with resected solid tumors and in combination
with pembrolizumab in patients with unresectable solid tumors.
Selected for a Poster Discussion, Saturday, June 1, 1:15 PM-2:45 PM
CST.
- NCI: Immunogenicity and tolerability of
personalized mRNA vaccine mRNA-4650 encoding defined neoantigens
expressed by the autologous cancer. Selected for a Poster Session,
Saturday, June 1, 8:00 AM-11:00 AM CST.
Moderna and NCI PCVs are 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. The NCI program uses Moderna’s mRNA technology but uses a
different neoantigen selection process and study design.
Intratumoral Immuno-Oncology: These programs
aim to drive anti-cancer T cell responses by injecting mRNA
therapies directly into tumors.
- OX40L (mRNA-2416): Moderna
continues to enroll patients in its Phase 1/2 trial evaluating
mRNA-2416, an intratumoral injection of mRNA encoding OX40L, for
the treatment of advanced relapsed/refractory solid tumor
malignancies and lymphomas and is also preparing to start
enrollment of a Phase 2 expansion cohort of the study in patients
with advanced ovarian carcinoma.
- OX40L + IL23 + IL36γ (Triplet)
(mRNA-2752): Moderna continues to dose a second cohort
(0.5 mg) of patients in its ongoing Phase 1 study evaluating the
safety and tolerability of mRNA- 2752 for the treatment of advanced
or metastatic solid tumor malignancies or lymphoma. mRNA-2752 is an
investigational mRNA immuno-oncology therapy that encodes a novel
combination of three immunomodulators designed to activate the
immune system to recognize and eradicate tumors that are resistant
to checkpoint inhibitors. It is being studied both as a single
agent and in combination with AstraZeneca’s durvalumab or
tremelimumab.
- IL12 (MEDI1191): At the American
Academy of Cancer Research (AACR) Annual Meeting in March,
Moderna’s strategic collaborator AstraZeneca shared preclinical
data that supported advancing MEDI1191 into a Phase 1 clinical
study. MEDI1191 is an mRNA encoding for IL12, a potent
immunomodulatory cytokine, which aims to enhance immune response in
immunologically “cold” tumors. AstraZeneca is now leading an
open-label, multi-center study of intratumoral injections of
MEDI1191 alone and in combination with a checkpoint inhibitor.
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): Moderna has completed enrollment of the
second dose level cohort (0.3 mg/kg) of its 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
secreted therapeutics modality to start clinical testing. The
formulation used for mRNA-1944 is also utilized in Moderna’s MMA
program.
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): Site initiation activities are ongoing for the
Phase 1 study of mRNA-3704, Moderna’s program for the rare
metabolic disease MMA.
- Propionic acidemia (PA)
(mRNA-3927): The European Commission (EC) has adopted the
recommendation from the Committee for Orphan Medicinal Products for
orphan drug designation for mRNA-3927, a development candidate for
propionic acidemia (PA).Additionally, enrollment continues in the
Company’s global natural history study of MMA and PA (MaP study).
This is a global, multi-center, non-interventional study for
patients with confirmed diagnosis of MMA due to methylmalonyl-CoA
mutase (MUT) deficiency or PA and is designed to identify and
correlate clinical and biomarker endpoints for these
disorders.
- Glycogen storage disease type 1a
(GSD1a) (mRNA-3745): 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, due to mutations within the enzyme glucose
6-phosphatase (G6Pase), leading to life-threatening hypoglycemia
and long-term liver and kidney damage. Patients with this disease
incur metabolite buildup associated with hepatomegaly (enlarged
livers), which can lead to benign and malignant liver tumors.
mRNA-3745 is an IV-administered mRNA encoding G6Pase enzyme,
designed to restore deficient or defective intracellular enzyme
activity. This is expected to increase blood glucose levels, while
decreasing levels of uric acid, lactic acid and triglycerides. In a
mouse model, Moderna has shown the ability to improve hypoglycemia
and other metabolic abnormalities associated with GSD1a, and mice
treated with G6Pase mRNA showed a dose-dependent improvement in
fasting glycemia and a reduction in both serum triglycerides and
liver weight. There are approximately 6,500 GSD1a patients in the
United States and the European Union. Disease management requires a
strict diet to maintain blood glucose levels with some patients
requiring a liver transplant. Preclinical data from this program
were shared at the 22nd Annual Meeting of the American Society of
Gene & Cell Therapy (ASGCT).
- Other systemic therapeutic
preclinical research: Also at ASGCT 2019, Moderna and academic
collaborators gave oral presentations and shared data from seven
preclinical animal studies using mRNA-based therapies for several
rare diseases including: ornithine transcarbamylase deficiency
(OTC) and maple syrup urine disease (MSUD) (in collaboration with
the Gene Therapy Program at the Perelman School of Medicine,
University of Pennsylvania); arginase-1 (ARG1) deficiency (in
collaboration with the University of California, Los Angeles
Department of Surgery and Department of Molecular and Medical
Pharmacology); factor VIII deficiency (hemophilia A) (in
collaboration with the Seattle Children’s Research Institute);
adult-onset type II citrullinemia (CTLN2); and progressive familial
intrahepatic cholestasis type 3 (PFIC3). These programs are early
research initiatives and presently are not Moderna development
candidates.
- Publication of note: In March,
Moderna published data in the American Journal of Human Genetics
that showed preclinical proof-of-concept for administering mRNA
encoding human ɑ-Gal, across species, as a potential systemic mRNA
therapy for the treatment of Fabry disease.
Information about each development candidate 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/.
First Quarter 2019 Financial Results
- Cash Position: Cash, cash
equivalents and investments as of March 31, 2019 and
December 31, 2018 were $1.55 billion and $1.69 billion,
respectively.
- Net Cash Used in Operating
Activities: Net cash used in operating activities was $143.9
million for the three months ended March 31, 2019 compared to
$111.4 million for the three months ended March 31, 2018. Net
cash used in operating activities includes $22.0 million and $25.0
million in the first quarter of 2019 and 2018, respectively, of
in-licensing payments to Cellscript, LLC and its affiliate,
mRNA RiboTherapeutics, Inc., to sublicense certain patent rights.
After 2019, we have no further in-licensing payment obligation to
Cellscript and its affiliate.
- Cash Used for Purchases of Property
and Equipment: Cash used for purchases of property and
equipment was $7.6 million for the three months ended
March 31, 2019 compared to $31.9 million for the three months
ended March 31, 2018.
- Revenue: Total revenue was $16.0
million for the three months ended March 31, 2019 compared to
$29.0 million for the three months ended March 31, 2018. On
January 1, 2019, we adopted Accounting Standards Codification (ASC)
Topic 606, Revenue from Contracts with Customers (ASC
606), using the modified retrospective transition method applied to
those contracts which were not completed as of January 1, 2019. The
total revenue decrease was mainly attributable to the decrease in
collaboration revenue from all of our strategic alliances,
particularly AstraZeneca and Merck, largely driven by the adoption
of ASC 606. Total revenues under the previous revenue recognition
standard would have been $37.9 million for the first quarter of
2019.
- Research and Development
Expenses: Research and development expenses were $130.6 million
for the three months ended March 31, 2019 compared to $90.1
million for the three months ended March 31, 2018. The
increase was primarily due to an increase in personnel related
costs, including stock-based compensation, mainly driven by an
increase in the number of employees supporting research and
development programs, an increase in clinical trial and
manufacturing costs, an increase in lab supplies and materials for
our preclinical studies and clinical trials, and an increase in
consulting and outside services costs.
- General and Administrative
Expenses: General and administrative expenses were $27.3
million for the three months ended March 31, 2019 compared to
$16.3 million for the three months ended March 31, 2018. The
increase was mainly attributable to an increase 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 related to operating as
a publicly traded company.
- Net Loss: Net loss was $132.7
million for the three months ended March 31, 2019 compared to
$72.4 million for the three months ended March 31, 2018.
Other Corporate Updates
- Moderna Annual Science Day: On
May 7, Moderna hosted its annual Science Day, which featured
presentations from Stephen Hoge M.D., president and Melissa
Moore Ph.D., chief scientific officer of Moderna’s mRNA research
platform, and focused on some of the Company’s latest advances in
basic and applied mRNA science. This included improvements in the
potency and delivery of potential mRNA medicines, and a new
research program focused on delivery of mRNA to the immune system.
The archived webcast of Science Day is available under “Events
& Presentations” on the Investors section of
the Moderna website at
https://investors.modernatx.com/ and
will be available there for approximately 30 days.
- Company Management:
- Moderna’s mRNA platform chief
scientific officer, Dr. Melissa Moore, was recently elected to the
American Academy of Arts and Sciences.
- Moderna’s chief human resources
officer, Annie Drapeau, left the Company in April to return to a
human resources leadership role in the technology industry.
Investor Call and Webcast Information
Moderna will host a live conference call and webcast
at 8:00 a.m. ET on Wednesday, March 8, 2019. To access
the call, please dial 866-922-5184 (domestic) or 409-937-8950
(international) and refer to conference ID 8273939. 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.
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: mRNA-3745 as a potential treatment for GSD1a; the
continued productivity of the Company’s mRNA platform; and plans by
AstraZeneca to initiate a Phase 1 clinical trial for MEDI1191 an
mRNA for IL12. 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 most recent Annual Report on Form 10-K filed with
the U.S. Securities and Exchange
Commission (SEC) 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 March 31, 2019 2018 Revenue:
Collaboration revenue $ 14,115 $ 27,460 Grant revenue 1,910
1,579 Total revenue 16,025 29,039 Operating
expenses: Research and development 130,575 90,124 General and
administrative 27,283 16,317 Total operating expenses
157,858 106,441 Loss from operations (141,833 )
(77,402 ) Interest income 10,972 5,209 Other expense, net (287 )
(156 ) Loss before benefit from income taxes (131,148 ) (72,349 )
Benefit from income taxes (24 ) — Net loss $ (131,124 ) $
(72,349 )
MODERNA, INC. CONDENSED
CONSOLIDATED BALANCE SHEETS AND STATEMENTS OF CASH FLOWS DATA
(Unaudited, in thousands) March 31,
December 31, 2019 2018 Cash, cash equivalents
and investments $ 1,546,583 $ 1,694,417 Total assets 1,806,207
1,962,149 Total liabilities 360,174 431,908 Total stockholders’
equity 1,446,033 1,530,241
Three Months
Ended March 31, 2019 2018 Net cash used in
operating activities $ (143,927 ) $ (111,385 ) Cash used for
purchases of property and equipment (7,595 ) (31,909 )
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190508005278/en/
Media:Jason GlashowHead of Corporate
Communications617-674-5648jason.glashow@modernatx.com
Investors:Lavina TalukdarHead of Investor
Relations617-209-5834lavina.talukdar@modernatx.com
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