mRNA-6231 encodes a long-acting selective IL-2
to preferentially expand regulatory T cells that suppress immune
activity in autoimmune diseases
mRNA-6981 encodes PD-L1 to treat autoimmune
disease, initially to be developed in autoimmune hepatitis
Company to expand pipeline of innovative
vaccines in the near term based on clinical success of infectious
disease vaccine portfolio to date
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 that it has entered a new autoimmune
therapeutic area. Building on the clinical validation of systemic
delivery of mRNA provided by data from its antibody against the
chikungunya virus (mRNA-1944) program, this new therapeutic area
will include autoimmune and inflammatory diseases. The Company also
announced that it will expand its pipeline of innovative vaccines
in the near term, following six positive Phase 1 clinical trial
readouts from its infectious disease portfolio and the initiation
of a Phase 2 study for its CMV vaccine (mRNA-1647).
Moderna’s pipeline is organized into six modalities based on
similar mRNA technologies, delivery technologies and manufacturing
processes. The Company’s approach is to leverage early programs
within a modality to generate clinical data and insights that
reduce the technology risk of subsequent programs and accelerate
the expansion of the pipeline in that modality. Today’s
announcement reflects the Company’s belief that recent positive
Phase 1 data from its infectious disease vaccine portfolio,
including its complex CMV vaccine, and chikungunya antibody program
have de-risked its prophylactic vaccines and systemic therapeutics
modalities. As a result, Moderna intends to bring new development
candidates forward within these two areas.
“2019 was an inflection point for Moderna with significant
clinical advances resulting from our investments in science and
manufacturing capabilities. The positive Phase 1 results from our
CMV vaccine and chikungunya antibody programs validate our approach
and help clinically de-risk the delivery technologies for our
prophylactic vaccines and systemic therapeutics modalities. Based
on these learnings, we are excited to enter a new therapeutic area
in autoimmune disease and announce two new development candidates,”
said Stéphane Bancel, Moderna’s chief executive officer. “We are
entering 2020 with clear priorities, a strong cash balance and a
talented team of employees focused on achieving our mission. With
our CMV vaccine, we are preparing for our first pivotal Phase 3
study and we look forward to announcing additional new development
candidates in our two clinically de-risked modalities, prophylactic
vaccines and systemic therapeutics.”
Mr. Bancel will present an update on the Company and its
pipeline of mRNA development programs on Monday, January 13, 2020
at 4:30 p.m. PT at the 38th Annual J.P. Morgan Healthcare
Conference in San Francisco. The presentation will be followed by a
question and answer session. A live webcast of both the
presentation and question and answer session will be available
under “Events & Presentations” in the investors section of
Moderna’s website at investors.modernatx.com. A replay of the
webcast will be archived on Moderna’s website for 30 days following
the presentation.
Moderna currently has 21 mRNA development candidates in its
portfolio with 13 in clinical studies. Across Moderna’s pipeline,
more than 1,500 participants have been enrolled in clinical
studies. The Company’s updated pipeline can be found at
www.modernatx.com/pipeline.
About Moderna’s New Autoimmune Therapeutic Area
Autoimmune diseases are characterized by immune activation in
response to antigens normally present in the body, reflecting a
loss of tolerance. Within this therapeutic area, the Company is
developing two potential medicines, mRNA-6231 and mRNA-6981,
designed to engage peripheral tolerance pathways to dampen
autoimmune activation and help restore immune homeostasis, thereby
reducing autoimmune pathology.
mRNA-6231 is an mRNA encoding for a long-acting IL-2
mutein designed to preferentially activate and expand the
regulatory T cell population, dampening the immune response.
mRNA-6981 is an mRNA encoding for PD-L1 and is designed
to augment cell surface levels of PD-L1 on myeloid cells, providing
co-inhibitory signals to self-reactive lymphocytes.
As an initial step to addressing a range of autoimmune
indications, the Company plans to conduct a Phase 1 study of
mRNA-6231 in healthy adult volunteers and intends to pursue
proof-of-concept with mRNA-6981 in a Phase 1 study in type 1
autoimmune hepatitis (AIH), a condition that involves liver
inflammation and can lead to cirrhosis and liver failure. The Phase
1 study of mRNA-6231 will be the first clinical demonstration of
subcutaneous administration of this delivery technology.
Both of these new autoimmune development candidates share the
same delivery technology as mRNA-1944, the antibody against
chikungunya, reducing technology risk. The autoimmune therapeutic
area is Moderna’s fifth therapeutic area, in addition to infectious
diseases, immuno-oncology, rare diseases and cardiovascular
diseases.
Program Updates 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 threats to global public
health.
Serious infections transmitted from mother to baby
- Cytomegalovirus (CMV) vaccine (mRNA-1647): Moderna
recently announced positive seven-month interim data after the
third and final vaccination from the Phase 1 study of mRNA-1647, as
well as dosing of the first participant in the Phase 2
dose-confirmation study of mRNA-1647. Manufacturing and planning
are underway for the pivotal Phase 3 study designed to evaluate the
efficacy of mRNA-1647 against primary CMV infection in a population
that includes women of childbearing age, expected to start in 2021.
Moderna owns worldwide commercial rights for mRNA-1647.
- Zika virus (mRNA-1893): The 10 µg and 30 µg cohorts in
the Phase 1 study of mRNA-1893 have been fully enrolled. This
development candidate is being developed in collaboration with the
U.S. Biomedical Advanced Research and Development Authority (BARDA)
within the Office of the Assistant Secretary for Preparedness and
Response at the U.S. Department of Health and Human Services.
Moderna owns worldwide commercial rights to mRNA-1893.
Serious respiratory infections
- Human metapneumovirus (hMPV) and parainfluenza type 3 (PIV3)
vaccine (mRNA-1653): The first participant in the Phase 1b age
de-escalation study of hMPV/PIV3 vaccine (mRNA-1653) has been
dosed. Moderna previously announced positive data from the second
pre-planned interim analysis of the Phase 1 study of mRNA-1653.
Moderna owns worldwide commercial rights to mRNA-1653.
- Respiratory syncytial virus (RSV) vaccine (mRNA-1172 or V172):
The Phase 1 study of mRNA-1172 led by Merck is ongoing. Moderna has
licensed worldwide commercial rights to mRNA-1172 to Merck.
Global public health programs
- The Company’s global public health portfolio is focused on
epidemic and pandemic diseases for which funding has been sought
from government and non-profit organizations. Given current funding
and priorities, the influenza H10N8 vaccine (mRNA-1440) and
chikungunya vaccine (mRNA-1388) are being deprioritized at
this time, contingent upon future funding. Discussions on funding
the Company’s influenza H7N9 vaccine program through
approval are ongoing.
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 vaccine (PCV) (mRNA-4157): The
randomized Phase 2 study investigating a 1 mg dose of mRNA-4157 in
combination with Merck’s pembrolizumab (KEYTRUDA®), compared to
pembrolizumab alone, for the adjuvant treatment of high-risk
resected melanoma is ongoing. The Phase 1 study is ongoing. Moderna
shares worldwide commercial rightsto mRNA-4157 with Merck.
- KRAS vaccine (mRNA-5671 or V941): The Phase 1
open-label, multi-center study to evaluate the safety and
tolerability of mRNA-5671 both as a monotherapy and in combination
with pembrolizumab, led by Merck, is ongoing. Moderna
sharesworldwide commercial rights to mRNA-5671 with Merck.
Intratumoral Immuno-Oncology: These programs aim to drive
anti-cancer T cell responses by injecting mRNA therapies directly
into tumors.
- OX40L (mRNA-2416): The first patient has been dosed in
the Phase 1 dose escalation cohort of mRNA-2416 in combination with
durvalumab (IMFINZI®). The Company has removed the top dose of 8 mg
in this cohort based on limitations due to the size of ovarian
lesions. Moderna owns worldwide commercial rights to
mRNA-2416.
- OX40L/IL-23/IL-36γ (Triplet) (mRNA-2752): The Phase 1 trial
evaluating mRNA-2752 as a single agent and in combination with
durvalumab in patients with advanced solid tumor malignancies and
lymphoma is ongoing. mRNA-2752 is an investigational mRNA
immuno-oncology therapy that encodes a novel combination of three
immunomodulators. Moderna owns worldwide commercial rights to
mRNA-2752.
- IL-12 (MEDI1191): The Phase 1 open-label, multi-center study of
intratumoral injections of MEDI1191 alone and in combination with
durvalumab in patients with advanced solid tumors, led by
AstraZeneca, is ongoing. MEDI1191 is an mRNA encoding for IL-12, a
potent immunomodulatory cytokine. Moderna shares worldwide
commercial rights to MEDI1191 with AstraZeneca.
Localized Regenerative Therapeutics: Localized production
of proteins has the potential to be used as a regenerative medicine
for damaged tissues.
- VEGF-A (AZD8601): The Phase 2a study of AZD8601 for
VEGF-A for ischemic heart disease in patients undergoing coronary
artery bypass grafting (CABG) surgery with moderately impaired
systolic function, led by AstraZeneca, is ongoing. Moderna has
licensed worldwide commercial rights to AZD8601 to
AstraZeneca.
Systemic Secreted & Cell Surface Therapeutics:
In this modality, mRNA is delivered systemically to create proteins
that are either secreted or expressed on the cell surface.
- Antibody against the chikungunya virus (mRNA-1944):
Moderna recently announced positive interim data from the first
analysis of safety and activity in the Phase 1 study evaluating
escalating doses of mRNA-1944 administered via intravenous infusion
in healthy adults. Dosing of the cohort at 0.6 mg/kg with steroid
premedication has begun. Dosing of an additional cohort with two
doses of 0.3 mg/kg (without steroid premedication) given one week
apart is expected. Moderna owns worldwide commercial rights to
mRNA-1944.
- IL-2 (mRNA-6231): mRNA-6231 is an mRNA encoding for a
long-acting IL-2 mutein. This new autoimmune development candidate
is designed to preferentially activate and expand the regulatory T
cell population. The Company plans to conduct a Phase 1 study of
mRNA-6231 in healthy adult volunteers. mRNA-6231 uses the same LNP
formulation as mRNA-1944. The Phase 1 study of mRNA-6231 will be
the first clinical demonstration of subcutaneous administration of
this delivery technology. Moderna owns worldwide commercial rights
to mRNA-6231.
- PD-L1 (mRNA-6981): mRNA-6981 is an mRNA encoding for
PD-L1. This new autoimmune development candidate is designed to
augment cell surface expression of PD-L1 on myeloid cells to
provide co-inhibitory signals to self-reactive lymphocytes. As an
initial step to addressing a range of autoimmune indications, the
Company intends to pursue proof-of-concept in a Phase 1 study of
mRNA-6981 in type 1 autoimmune hepatitis (AIH), a condition that
involves liver inflammation and can lead to cirrhosis and liver
failure. mRNA-6981 uses the same LNP formulation as mRNA-1944.
Moderna owns worldwide commercial rights to mRNA-6981.
- Relaxin (AZD7970): Partnered with AstraZeneca, AZD7970
is in preclinical development for the treatment of heart failure.
Under the terms of the collaboration, AstraZeneca would sponsor the
Phase 1 trial to assess safety, tolerability and duration of
systemic exposure to the Relaxin protein. Moderna shares worldwide
commercial rights to AZD7970 with AstraZeneca.
- Fabry disease (mRNA-3630): Individuals with Fabry
disease have a deficiency in the α-GAL enzyme resulting in a
reduced or complete inability to metabolize glycosphingolipids in
lysosomes. mRNA-3630 aims to instruct cells to produce α-GLA both
locally in multiple affected tissues, and to secrete it into
circulation from organs such as the liver for delivery to distal
tissues. mRNA-3630 is in preclinical development. Moderna owns
worldwide commercial rights to mRNA-3630.
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 Phase 1/2
open-label, dose escalation study is actively recruiting patients
for the first cohort at U.S. sites following a protocol amendment
expanding the eligibility criteria to patients 8 years and older
for the first cohort. This study is evaluating mRNA-3704 for the
treatment of MMA due to methylmalonyl-CoA mutase (MUT) deficiency.
The Company is planning to initiate several sites outside the U.S.
and recently received Medicines and Healthcare products Regulatory
Agency (MHRA) approval in the U.K. The objectives of this study are
to evaluate safety and tolerability, assess the pharmacodynamic
response and characterize the pharmacokinetic profile of mRNA-3704.
This is Moderna’s first rare disease program to advance into
clinical testing. The mRNA-3704 program uses the same LNP
formulation as mRNA-1944. Moderna owns worldwide commercial rights
to mRNA-3704.
- Propionic acidemia (PA) (mRNA-3927): Study start-up in
the U.S. is ongoing for the open-label, multi-center Phase 1/2
study of multiple ascending doses of mRNA-3927 in primarily
pediatric patients with PA. The objectives of this study are to
evaluate the safety and tolerability of mRNA-3927 administered via
IV infusion, assess the pharmacodynamic response as assessed by
changes in plasma biomarkers and characterize the pharmacokinetic
profile of mRNA-3927. The mRNA-3927 program uses the same LNP
formulation as mRNA-1944. Moderna owns worldwide commercial rights
to mRNA-3927.
- MMA and PA Natural History Study (MaP): This is a global,
multi-center, non-interventional study for patients with confirmed
diagnosis of MMA due to MUT deficiency or PA and is designed to
identify and correlate clinical and biomarker endpoints for these
disorders.
- Phenylketonuria (PKU) (mRNA-3283): Individuals with PKU
have a deficiency in phenylalanine hydroxylase (PAH) resulting in a
reduced or complete inability to metabolize the essential amino
acid phenylalanine into tyrosine. mRNA-3283 encodes human PAH to
restore the deficient or defective intracellular enzyme activity in
patients with PKU. mRNA-3283 is in preclinical development. Moderna
owns worldwide commercial rights to mRNA-3283.
- Glycogen storage disease type 1a (GSD1a) (mRNA-3745):
Individuals with GSD1a have a deficiency in glucose-6-phosphatase
resulting in pathological blood glucose imbalance. mRNA-3745 is an
IV-administered mRNA encoding human G6Pase enzyme, designed to
restore deficient or defective intracellular enzyme activity in
patients with GSD1a. mRNA-3745 is in preclinical development.
Moderna owns worldwide commercial rights to mRNA-3745.
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/.
Corporate Objectives
Moderna continues to execute on its corporate objectives for
2019-2020, which include:
- Generate human proof-of-concept data for
multiple medicines
- Execute on current development pipeline
- Create new development candidates in
existing modalities
- Invent new modalities
Corporate Updates
- Continued growth across organization: Moderna ended 2019
with approximately 820 full time employees. The Company ended 2018
with 735 employees.
- Continued strong cash position: The Company expects
cash, cash equivalents, and investments at December 31, 2019 to be
approximately $1.26 billion (unaudited), as compared to $1.69
billion as of December 31, 2018. Reiterating prior guidance, in
2020, the Company expects net cash used in operating activities and
purchases of property and equipment to be between $490 million and
$510 million.
- Strategic alliances and available grant funding: The
Company has established a wide range of strategic alliances with
leading biopharmaceutical companies, as well as
government-sponsored and private organizations focused on global
health initiatives. Strategic collaborators contribute their
therapeutic expertise, help to validate Moderna’s mRNA platform and
have provided a quarter of the Company’s total capital to date. As
of December 31, 2019, Moderna had up to $184 million in additional
funding available from grants (including amounts not yet
committed)1.
- NASDAQ Biotech Index: The Company was added to the
NASDAQ Biotech Index effective December 23, 2019.
- Shareholder Letter: Moderna CEO Stéphane Bancel
published a letter to shareholders on January 6, 2020.
Key 2020 Investor and Analyst Event Dates
- Manufacturing & Digital Day – March 4 at Moderna’s Norwood,
MA facility
- Vaccines Day – April 14 in New York City
- Science Day – June 4 in New York City
- R&D Day – September 17 in New York City
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 have a therapeutic or preventive
benefit with 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.
(NASDAQ: AZN) and Merck, Inc. (NASDAQ: MRK), as well as the Defense
Advanced Research Projects Agency (NASDAQ: 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 named a top biopharmaceutical employer by Science for the past
five years. To learn more, visit www.modernatx.com.
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 limited to, statements concerning:
the success of the Company’s new autoimmune and inflammatory
diseases therapeutic area; the timing and success of the Company’s
planned Phase 1 studies of mRNA-6231 and mRNA-6981; the timing of
expansion of its pipeline of innovative vaccines; the timing of the
initiation of the Phase 2 study for mRNA-1647; the adequacy of the
Company’s cash, cash equivalents and investments; the Company’s
ability to retain its senior management and employees; the
Company’s ability to maintain current and enter into new
collaborations with partners; the Company’s ability to obtain
funding to support the future clinical development of mRNA-1440 and
mRNA-1388; the Company’s ability to fund its influenza H7N9 vaccine
program through approval; the timing and expected outcome of
Moderna’s clinical studies of its other development candidates; the
Company’s ability to advance its preclinical-stage development
candidates into clinical development; the Company’s ability to
execute on its corporate objectives; and the Company’s ability to
receive additional grant funding. 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: the success of the Company’s new
autoimmune and inflammatory diseases therapeutic area; the clinical
success of subcutaneous administration of its delivery technology;
the ability to build upon the clinical validation of the systemic
delivery of mRNA provided by mRNA-1944; the Company’s belief that
recent positive Phase 1 data from its infectious disease vaccine
portfolio and chikungunya antibody program have de-risked its
prophylactic vaccines and systemic therapeutics modalities; the
timing and success of Moderna’s clinical studies of its development
candidates; the ability of the Company to advance a rare disease
program into clinical testing; 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 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.
_________________________
1Biomedical Advanced Research and Development Authority (BARDA),
Defense Advanced Research Projects Agency (DARPA) and The Bill and
Melinda Gates Foundation (BMGF). Additional funding is subject to
agreement on scope of additional projects.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20200112005052/en/
Media: Colleen Hussey Senior Manager, Corporate
Communications 203-470-5620 Colleen.Hussey@modernatx.com
Dan Budwick 1AB 973-271-6085 Dan@1abmedia.com
Investors: Lavina Talukdar Head of Investor Relations
617-209-5834 Lavina.Talukdar@modernatx.com
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