Moderna Reports First Quarter 2019 Financial Results and Provides Business Updates

BusinessWire

5 years ago

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

CAMBRIDGE, Mass.–(BUSINESS WIRE)–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 22^nd
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		)

Contacts

Media:
Jason Glashow
Head of Corporate Communications
617-674-5648
jason.glashow@modernatx.com

Investors:
Lavina Talukdar
Head of Investor Relations
617-209-5834
lavina.talukdar@modernatx.com

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