Moderna Announces Publication of Preclinical Data for Chikungunya Antibody Program

Published in Science Immunology, study shows mRNA encoding antibody
against chikungunya virus is well tolerated, results in linear
dose-dependent protein expression and provides 100 percent protection
against infection in animals

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 announced
the publication of preclinical data in Science Immunology, showing
that mRNA encoding a human monoclonal antibody against the chikungunya
virus delivered in a proprietary lipid nanoparticle (LNP) can protect
from infection by the virus in vivo. These data supported the
initiation of a Phase 1 program of mRNA-1944 against chikungunya virus
in January 2019 to evaluate the safety, tolerability, pharmacokinetics
and pharmacodynamics of escalating doses of mRNA-1944 via intravenous
infusion in healthy adults.

“These preclinical data demonstrate three critical features of our
therapeutic platform: first, that it was well-tolerated, with no
dose-limiting toxicities; second, we showed linear dose-dependent
pharmacology; and third, that the expressed protein was able to prevent
disease,” said Stephen Hoge, M.D., president at Moderna. “The mRNA and
delivery technologies presented here underpin our expansion into
systemic therapeutics in humans, first with mRNA-1944 against
chikungunya virus this past January, and soon with our first metabolic
rare disease program, mRNA-3704, for methylmalonic acidemia.”

The preclinical data published today showed that treatment with mRNA was
well tolerated at doses ranging from 0.3 mg/kg to 3.0 mg/kg in non-human
primates, with linear dose-dependent pharmacology, meaning that
increases in mRNA doses result in predictable and proportionate
increases in expressed antibody in the blood. Finally, in a mouse viral
challenge model for chikungunya virus infection, the mRNA-encoded
protein provided protection from arthritis, musculoskeletal tissue
infection and death. Extrapolating these findings from the preclinical
models suggests that persistence of antibody levels of at least 1
microgram per milliliter could be protective against chikungunya virus
infection in humans.

“Using the body’s own machinery to produce antibodies against
chikungunya by using mRNA may be a powerful way to combat the virus,”
said James Crowe Jr., MD, director of the Vanderbilt Vaccine Center. “We
are pleased that our early work contributed to these preclinical
findings and look forward to the results from the ongoing Phase 1
clinical study of the antibody against chikungunya virus.”

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, a mosquito-borne virus. It 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. The research and development of mRNA-1944 was financially
supported by the Defense Advanced Research Projects Agency (DARPA), an
agency of the U.S. Department of Defense, and the antibody was initially
identified by researchers at Vanderbilt University Medical Center.

A link to the publication, A Lipid-encapsulated mRNA Encoding a
Potently Neutralizing Human Monoclonal Antibody Protects Against
Chikungunya Infection
(Kose, et al.), can be found here.

Moderna currently has 21 mRNA development candidates in its portfolio
spanning infectious diseases, immuno-oncology, rare diseases and
cardiovascular disease, with 11 of these development candidates now in
the clinic. The Company’s pipeline can be found at www.modernatx.com/pipeline.
In the past three years, Moderna and collaborators have published more
than 26 peer-reviewed papers, with 13 in the last year alone.

About Chikungunya

Chikungunya is a mosquito-borne virus that poses a significant public
health problem in tropical and subtropical regions. The disease is
characterized by an acute onset of fever, rash, muscle pain, and
sometimes debilitating pain in multiple joints. There are currently no
effective therapies or approved vaccines to treat or prevent chikungunya
infection or disease, and effective mosquito control is challenging.
Currently, people infected with chikungunya are treated with
non-steroidal anti-inflammatory drugs to relieve some symptoms. In
addition to a systemic secreted antibody that could provide passive
immunity, Moderna is also exploring using mRNA to encode viral antigens
as a prophylactic vaccine against the chikungunya virus (mRNA-1388).

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 the Company 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; 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.

Special Note Regarding Forward-Looking Statements

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
suggestion that preclinical data for mRNA-1944 showing passive
immunization may lead to the prevention of infectious diseases like
chikungunya in humans and that mRNA-1944 may be well-tolerated and
delivered in a dose-dependent manner in humans; and the potential for
the human body to use its own machinery to produce antibodies against
Chikungunya by using mRNA to combat the virus. 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
Moderna’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: whether preclinical results of mRNA-1944 will be predictive of
any future clinical studies, including the ongoing Phase 1 clinical
study of mRNA-1944; whether mRNA-1944 will be shown to be unsafe or
intolerable during future preclinical or clinical studies; clinical
development is lengthy and uncertain, especially for a new class of
medicines such as mRNA, and therefore our clinical programs or
development candidates may be delayed, terminated, or may never advance;
no mRNA drug has been approved in this new potential class 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 class of medicines; and those risks and
uncertainties described under the heading “Risk Factors” 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
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.

Contacts

Moderna Contacts:

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

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