New development candidate for combination
respiratory COVID-19 booster + seasonal flu booster vaccine
New pediatric combination development candidate
for RSV + hMPV vaccine
Interim Phase 1 data from RSV vaccine candidate
in older adults significantly boosted neutralizing antibody titers
above baseline
New infectious disease therapeutic vaccine
candidate, to complement Epstein-Barr virus prophylactic
vaccine
Phase 2 randomized, placebo-controlled study of
personalized cancer vaccine fully enrolled; primary endpoint is
recurrence free survival at 12 months
Company continues to scale with 37 programs in
development, including 22 in ongoing clinical studies
Moderna, Inc. (Nasdaq: MRNA), a biotechnology company pioneering
messenger RNA (mRNA) therapeutics and vaccines, today announced
significant advances across its portfolio of mRNA pipeline programs
being presented at the Company’s fifth annual R&D Day
today.
“I am proud of the progress that the Moderna team has made in
advancing our best-in-class mRNA pipeline while addressing the
global COVID-19 pandemic. We believe our mRNA platform can solve
the world’s greatest health challenges, from diseases impacting
millions, to ultra-rare diseases impacting dozens, to medicines
personalized down to the individual level,” said Stéphane Bancel,
Chief Executive Officer of Moderna. “Today we are announcing the
first step in our novel respiratory vaccine program with the
development of a single dose vaccine that combines a booster
against COVID-19 and a booster against flu. We are making progress
on enrolling patients in our rare disease programs, and we are
fully enrolled in our personalized cancer vaccine trial. We believe
this is just the beginning of a new age of information-based
medicines.”
Updates and recent progress include:
COVID-19 Vaccine Development
- Moderna completed the rolling submission process for a
Biologics License Application (BLA) for its COVID-19 vaccine
(mRNA-1273) in the U.S.
- Robust antibody responses have been observed in a Phase 2 study
of a third dose/booster of mRNA-1273
New Development Candidates
- Combination vaccine candidate (mRNA-1073) that combines
Moderna’s COVID-19 vaccine and flu vaccine candidate
- Pediatric combination vaccine candidate (mRNA-1365) that
combines Moderna’s RSV vaccine candidate with its hMPV vaccine
candidate
- EBV therapeutic vaccine candidate (mRNA-1195)
- Ultra-rare disease, Crigler-Najjar Syndrome Type 1 (mRNA-3351);
Moderna to provide investigational mRNA CN-1 therapy to the
nonprofit Institute for Life Changing Medicines (ILCM) free of
charge
Infectious Diseases
- Phase 1 study of quadrivalent seasonal flu vaccine candidate
(mRNA-1010) fully enrolled
- Positive interim data from Phase 1 study of RSV vaccine
candidate (mRNA-1345) from older adult cohort (ages 65-79
years)
- Preparing for a global Phase 2/3 study with approximately
34,000 participants of RSV vaccine candidate (mRNA-1345); expected
to begin by the end of 2021
Oncology
- Phase 2 randomized, placebo-controlled study of personalized
cancer vaccine (PCV) (mRNA-4157) in combination with Merck’s
pembrolizumab (KEYTRUDA®), compared to pembrolizumab alone, for the
adjuvant treatment of high-risk resected melanoma is fully
enrolled
Cardiovascular
- Phase 2a study of AZD8601 VEGF-A, being developed for patients
with ischemic heart disease undergoing coronary artery bypass
grafting surgery with moderately impaired systolic function, led by
AstraZeneca, has completed recruitment after enrollment of the low
dose cohort
Rare Diseases
- Enrollment of the first cohort in Propionic Acidemia
(mRNA-3927) Phase 1/2 Paramount study is complete
- Investigational New Drug application (IND) open and Orphan Drug
Designation granted by U.S. FDA for GSD1a program (mRNA-3745)
Corporate Update
- Moderna Genomics (MGX) will leverage Moderna’s current mRNA and
lipid nanoparticle (LNP) platform and will pursue novel technology
within nucleic acids
Moderna continues to scale, now with 37 programs in development
across 34 development candidates1, including 22 in ongoing clinical
studies. The Company’s updated pipeline can be found at
www.modernatx.com/pipeline. Moderna and collaborators have
published more than 80 peer-reviewed manuscripts.
Select Program Highlights
Core Modalities
Prophylactic Vaccines: Moderna is developing vaccines
against viral diseases where there is unmet medical need –
including vaccines against acute respiratory infections, vaccines
against persistent infections, as well as vaccines against threats
to global public health.
Vaccines against acute respiratory infections
COVID-19 vaccine development
- Moderna COVID-19 Vaccine (mRNA-1273): The World Health
Organization (WHO) and health agencies in more than 50 countries
have granted emergency use authorization or emergency use listing
for the use of the Moderna COVID-19 vaccine in adults. In addition,
Moderna has received authorization for use of its COVID-19 vaccine
in adolescents ages 12 and up in the European Union and other
jurisdictions. On August 25, 2021, Moderna completed the rolling
submission process for a Biologics License Application (BLA) for
the vaccine in the U.S. Moderna is working with additional health
agencies on the authorization and/or approval of its vaccine in
additional jurisdictions2. Moderna retains worldwide rights to
develop and commercialize the Moderna COVID-19 Vaccine.
- Final Analysis of Phase 3 COVE Study: In the final
analysis of the Phase 3 COVE study, the Moderna COVID-19 Vaccine
showed 93% efficacy (95% CI), with the efficacy remaining durable
through six months after administration of the second dose. In this
analysis, the Moderna COVID-19 vaccine showed 98.2% efficacy
against severe COVID-19 disease and 100% efficacy against death
caused by COVID-19. Sub-group analyses were consistent across
different populations and the safety profile based on extended
safety follow-up was consistent with the Phase 3 COVE study primary
results.
- Addressing Variants of Concern: Moderna has four
development candidates against SARS-CoV-2 variants of concern,
including three which have been administered in a Phase 2/3
clinical trial. Initiation of a clinical cohort with the fourth
candidate is planned in the coming weeks. The Company’s strategy is
to develop booster vaccines against current variants of concern and
against potential future variants of concern.
- mRNA-1273.351: Variant-specific candidate against the Beta
variant
- mRNA-1273.617: Variant-specific candidate against the Delta
variant
- mRNA-1273.211: Multivalent candidate combining the
Beta-specific variant and mRNA-1273
- mRNA-1273.213: Multivalent candidate combining the
Beta-specific and Delta-specific candidates
- Booster (Third) Dose: Moderna has submitted for a
booster (third) dose of mRNA-1273 at the 50 µg dose level for the
following: Emergency Use Authorization (EUA) with the U.S. FDA,
Conditional Marketing Approval (CMA) with the European Medicines
Agency (EMA) and to additional regulatory agencies. In the amended
Phase 2 study, a booster dose of mRNA-1273 at the 50 µg dose level
boosted neutralizing titers significantly above the Phase 3
benchmark. After a third dose, a similar level of neutralizing
titers was achieved across age groups, notably in older adults
(ages 65 and above). The safety profile following dose 3 was
similar to that observed previously for dose 2 of mRNA-1273. An
additional analysis showed that a booster dose of mRNA-1273 at the
50 µg dose level induced robust antibody responses and
significantly increased geometric mean titers (GMT) for all
variants of concern including importantly, Delta (B.1.617.2) by
42.3-fold.
- Additional Clinical Studies of mRNA-1273
- Phase 2/3 “TeenCOVE” study of mRNA-1273 in adolescents:
The Phase 2/3 study of mRNA-1273 in adolescents ages 12-17 years
showed that no cases of COVID-19 were observed after two doses of
vaccine using the primary case definition, consistent with a
vaccine efficacy of 100%. Generally well tolerated, the majority of
adverse events were mild or moderate in severity. The most common
solicited local adverse event was injection site pain. The most
common solicited systemic adverse events after the second dose of
mRNA-1273 were headache, fatigue, myalgia and chills. The
Conditional Marketing Authorization (CMA) for Spikevax3 in the
European Union (EU) has been expanded to include adolescents 12
years of age and older. In addition, the Japanese Ministry of
Health, Labor and Welfare, Health Canada, as well as other
regulatory agencies around the world have also authorized Moderna's
COVID-19 vaccine for ages 12 to 17. Moderna has filed for an EUA
for adolescents with the U.S. FDA.
- Phase 2 “KidCOVE” study of mRNA-1273 in young children:
The Phase 2 study of mRNA-1273 in pediatric population ages 6
months to 11 years is ongoing. The Company selected the 50 µg dose
for expanded enrollment in the 6 to <12 years old cohort which
is now fully enrolled (N=4,000). Dose selection studies are still
underway for 2 years to <6 years old and 6 months to <2 years
old age groups.
- Phase 3 “COVE Transplant” study of mRNA-1273: The Phase
3 study of mRNA-1273 in adults with a kidney or liver transplant is
ongoing, including the evaluation of the safety and immunogenicity
of a third vaccine dose to these immunocompromised
participants.
- Next-generation vaccine against COVID-19
(mRNA-1283): The Phase 1 study of mRNA-1283 is fully enrolled.
mRNA-1283 is a next-generation vaccine candidate against COVID-19
that encodes for the portions of the SARS-CoV-2 spike protein
critical for neutralization, specifically the Receptor Binding
Domain (RBD) and N-terminal Domain (NTD). It is being developed as
a potential refrigerator stable mRNA vaccine that will facilitate
easier distribution and administration by healthcare
providers.
Additional vaccines against acute respiratory infections
- Seasonal influenza vaccine (mRNA-1010): The Phase 1/2
study evaluating safety and reactogenicity of three different dose
levels of mRNA-1010 in adults ages 18-49 years and above 50 years
is fully enrolled (N=180). mRNA-1010 encodes for hemagglutinin (HA)
glycoproteins of 4 flu strains and targets lineages recommended by
the World Health Organization (WHO) for the prevention of
influenza, including seasonal influenza A H1N1, H3N2 and influenza
B Yamagata and Victoria.
- COVID-19 and flu combination vaccine (mRNA-1073):
mRNA-1073 encodes for the COVID-19 spike protein and the Flu HA
glycoproteins. Moderna owns worldwide commercial rights to
mRNA-1073.
- Respiratory syncytial virus (RSV) vaccine (mRNA-1345):
mRNA-1345 is a vaccine against RSV encoding for a prefusion F
glycoprotein, which elicits a superior neutralizing antibody
response compared to the postfusion conformation. RSV is the
leading cause of severe respiratory illness in young children and
older adults (65+). The Phase 1 study of mRNA-1345 to evaluate the
tolerability and reactogenicity of mRNA-1345 in younger adults,
women of child-bearing age, older adults and seropositive toddlers
is ongoing. All four cohorts of younger adults (ages 18-49 years)
and all four cohorts of older adults (ages 65-79 years) are fully
enrolled. Today, the Company is sharing Phase 1 interim data from
the older adult cohort. Neutralizing antibodies were confirmed to
be present at baseline in all participants, as expected. The data
showed that a single mRNA-1345 vaccination of 50 µg, 100 µg or 200
µg boosted neutralizing antibody titers against RSV-A by
approximately 14-fold and against RSV-B by approximately 10-fold.
Data were pooled across dose levels because there was not a
significant difference between doses. A single vaccination of 50
µg, 100 µg or 200 µg was well-tolerated in older adults through
month 1. Moderna is preparing for a Phase 2/3 study of RSV in older
adults (ages older than 60 years) and expects to begin this study
by the end of 2021. The Company expects this Phase 2/3 study will
be a global study conducted in locations influenced by the
epidemiology of RSV and expects to enroll approximately 34,000
participants, subject to agreement with regulatory authorities. The
FDA has granted Fast Track designation for mRNA-1345 in adults
older than 60 years of age. There is no approved vaccine to prevent
RSV. Moderna owns worldwide commercial rights to mRNA-1345.
- Human metapneumovirus (hMPV) and parainfluenza type 3 (PIV3)
vaccine (mRNA-1653): Moderna is enrolling seropositive
pediatric participants (12-36 months of age) in the Phase 1 study
of hMPV/PIV3 study (mRNA-1653). The first cohort in this study is
fully enrolled. Moderna owns worldwide commercial rights to
mRNA-1653.
- Pediatric RSV and hMPV combination vaccine (mRNA-1365):
mRNA-1365 encodes for the RSV prefusion F glycoprotein and the hMPV
F protein. Moderna owns worldwide commercial rights to
mRNA-1365.
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.
- Relaxin (mRNA-0184): mRNA-0184 encodes for the relaxin
fusion protein. The mRNA sequence of mRNA-0184 is engineered to
increase protein expression and prolong half-life. Moderna is
planning for a Phase 1 study in participants with chronic heart
failure. The Company expects that mRNA-0184 will be administered
after heart failure decompensation to bridge patients through the
vulnerable period. Moderna owns worldwide commercial rights to
mRNA-0184.
Exploratory Modalities
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, placebo-controlled 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 fully enrolled (n=150).
The primary endpoint of the Phase 2 study is recurrence-free
survival at 12 months. The Phase 1 in multiple cohorts is ongoing
and the expanded head and neck cohort is recruiting additional
patients. Moderna shares worldwide commercial rights to mRNA-4157
with Merck.
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 VEGF-A,
which is being developed for patients with ischemic heart disease
undergoing coronary artery bypass grafting surgery with moderately
impaired systolic function, led by AstraZeneca, has completed
recruitment after enrollment of the low dose cohort (n=11). Moderna
has licensed worldwide commercial rights to AZD8601 to
AstraZeneca.
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.
- Propionic acidemia (PA) (mRNA-3927): The Phase 1/2
Paramount study of mRNA-3927 is ongoing and enrollment of the first
cohort is complete. Moderna owns worldwide commercial rights to
mRNA-3927.
- Methylmalonic acidemia (MMA) (mRNA-3705): The Phase 1/2
Landmark study to evaluate the safety and pharmacology of mRNA-3705
in patients 1 year of age and older with methylmalonic acidemia
(MMA) is ongoing and the first participant has been dosed. Moderna
received rare pediatric designation for mRNA-3705. Moderna owns
worldwide commercial rights to mRNA-3705.
- Glycogen storage disease type 1a (GSD1a) (mRNA-3745):
The U.S. FDA has granted mRNA-3745 Orphan Drug Designation and
completed its review of the IND application allowing it to proceed
to clinic. 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 the deficient or defective
intracellular enzyme activity in patients with GSD1a. Moderna owns
worldwide commercial rights to mRNA-3745.
- Crigler-Najjar Syndrome Type 1 (CN-1)
(mRNA-3351): mRNA-3351 encodes for the human UGT1A1 and is
designed to restore the missing or dysfunctional proteins that
causes Crigler-Najjar Syndrome Type 1. mRNA-3351 has been granted
Rare Pediatric Disease designation by the U.S. FDA. Moderna will
provide investigational mRNA-3351 to the nonprofit Institute for
Life Changing Medicines (ILCM) free of charge. ILCM will be
responsible for the clinical development of mRNA-3351 and plans to
initiate clinical studies of mRNA-3351 in 2022.
Corporate Update
- Moderna Genomics (MGX) will leverage Moderna’s current mRNA and
lipid nanoparticle (LNP) platform but will also pursue novel
technology within nucleic acids. The MGX vision is to be a leader
in large, complex genomic editing. Eric Huang, Ph.D. will serve as
the Chief Scientific Officer of Moderna Genomics.
- Moderna announces a cash position as of August 31, 2021 of
approximately $15 billion.
Information about each development candidate in Moderna’s
pipeline can be found at investors.modernatx.com. The R&D Day
program can be accessed here beginning today at 8:00 am ET.
About Moderna
In 10 years since its inception, Moderna has transformed from a
science research-stage company advancing programs in the field of
messenger RNA (mRNA), to an enterprise with a diverse clinical
portfolio of vaccines and therapeutics across six modalities, a
broad intellectual property portfolio in areas including mRNA and
lipid nanoparticle formulation, and an integrated manufacturing
plant that allows for both clinical and commercial production at
scale and at unprecedented speed. Moderna maintains alliances with
a broad range of domestic and overseas government and commercial
collaborators, which has allowed for the pursuit of both
groundbreaking science and rapid scaling of manufacturing. Most
recently, Moderna’s capabilities have come together to allow the
authorized use of one of the earliest and most effective vaccines
against the COVID-19 pandemic.
Moderna’s mRNA platform builds on continuous advances in basic
and applied mRNA science, delivery technology and manufacturing,
and has allowed the development of therapeutics and vaccines for
infectious diseases, immuno-oncology, rare diseases, cardiovascular
diseases and auto-immune diseases. Moderna has been named a top
biopharmaceutical employer by Science for the past six 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 regarding: the Company’s development of
the Moderna COVID-19 Vaccine (mRNA-1273); its efforts to continue
developing vaccines against variant strains of SARS-CoV-2 and for
booster doses; the ability of the Moderna COVID-19 Vaccine and
booster doses to provide protection against COVID-19 over time and
to trigger an antibody response against variants of concern; the
safety profile associated with COVID-19 booster candidates; the
Company’s plans to submit for a Biologics License Application for
mRNA-1273 and other approvals; the enrollment, conduct and timing
of clinical trials for programs in the Company’s pipeline,
including its vaccine candidates against COVID-19, seasonal flu,
CMV, and RSV; plans to develop combination respiratory vaccines;
the ability to expand the Company’s portfolio of development
programs; the potential to combine different vaccines into a single
dose; the ability to use mRNA to enable combination therapeutics
personalized for individual tumors and patients; the potential for
mRNA medicines to address various diseases with unmet medical need;
and the scalability of the Company and its ability to bring
potential medicines to market. 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 those other 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.
1 Includes separate COVID-19 Vaccine (mRNA-1273) programs in
development for adults, pediatrics & adolescents and separate
RSV vaccine (mRNA-1345) programs in development for adults and
pediatrics
2 BARDA, part of the Office of the Assistant Secretary for
Preparedness and Response (ASPR) within the U.S. Department of
Health and Human Services (HHS), partially supported the research
and development of the Moderna COVID-19 Vaccine with federal
funding under Contract no. 75A50120C00034.
3Spikevax is the trade name authorized by the European Medicines
Agency (EMA) for the Moderna COVID-19 vaccine.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210909005497/en/
Moderna Media: Colleen Hussey Director, Corporate
Communications 617-335-1374 Colleen.Hussey@modernatx.com Investors:
Lavina Talukdar Senior Vice President & Head of Investor
Relations 617-209-5834 Lavina.Talukdar@modernatx.com
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