Positive interim results announced from Phase 1
CMV vaccine (mRNA-1647) study
Positive interim results announced from Phase 1
mRNA encoding chikungunya antibody (mRNA-1944) study
First participant dosed in Phase 1b age
de-escalation study of hMPV+PIV3 vaccine (mRNA-1653)
Cash, cash equivalents and investments at the
end of the quarter were $1.34 billion, and are expected to be
approximately $1.20 billion at the end of 2019
Net cash used in operating activities and
purchases of property and equipment is expected to total
approximately $500 million in 2019, and between $490 million and
$510 million in 2020
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 provided business updates and reported financial
results for the third quarter of 2019.
New updates and recent progress include:
Infectious Diseases
- Positive interim results announced from Phase 1 CMV vaccine
(mRNA-1647) study; Moderna is advancing mRNA-1647 to a
dose-confirmation Phase 2 study in the near term while planning for
a pivotal Phase 3 study
- First participant dosed in Phase 1b age de-escalation study of
hMPV+PIV3 vaccine (mRNA-1653)
- FDA granted Fast Track designation for Zika vaccine
(mRNA-1893)
Immuno-Oncology
- First patient dosed in Phase 2 PCV (mRNA-4157) study in
patients with resected melanoma
- OX40L (mRNA-2416) Phase 2 expansion cohort in ovarian cancer to
focus only on the combination with durvalumab (IMFINZI®)
Rare Diseases
- Positive interim results announced from Phase 1 chikungunya
antibody (mRNA-1944) study; dosing of two additional cohorts
planned to begin in the near term
- Phase 1/2 MMA (mRNA-3704) study is actively recruiting patients
at U.S. sites following a protocol amendment expanding the
eligibility criteria to patients 8 years and older
- FDA granted Fast Track designation for PA program (mRNA-3927);
study start-up is ongoing for the open-label, multi-center Phase
1/2 study of multiple ascending doses of mRNA-3927 in pediatric
patients with PA in the U.S.
Research
- Moderna announced a multi-year mRNA immunotherapy research
collaboration with Harvard University to explore fundamental
immunological processes and identify potential therapeutic
opportunities
“The positive Phase 1 cytomegalovirus vaccine results announced
this quarter represent an important step toward the prevention of
congenital CMV infections. These data resulted from investment in
our mRNA technology platform, which has now generated six positive
infectious disease vaccine clinical readouts. Additionally, the
recent positive Phase 1 chikungunya antibody results help de-risk a
delivery technology shared by our rare disease programs, further
validating our approach into new areas where mRNA medicines have
the potential to treat a wide range of diseases,” said Stéphane
Bancel, Moderna’s chief executive officer. “We ended the quarter
with a strong cash balance of $1.34 billion and up to $187 million
in additional untapped grant funding, giving us up to $1.5 billion
to invest in the Company moving forward. We expect investment
levels in 2020 to be approximately $500 million, similar to 2019.
We intend to expand our group of biopharmaceutical partners and
bring in additional non-dilutive grant and government funding as we
advance our development pipeline and create new modalities.”
Moderna currently has 21 mRNA development candidates in its
portfolio with 13 in clinical studies. Across Moderna’s pipeline,
more than 1,400 participants have been enrolled in clinical
studies. The Company’s updated pipeline can be found at
www.modernatx.com/pipeline. Moderna and collaborators have
published more than 40 peer-reviewed papers, including 23 over the
last 12 months.
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 September 30, 2019, Moderna had up to $187 million in additional
funding available from grants (including amounts not yet
committed)1.
Summary of Program 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.
- Cytomegalovirus (CMV) vaccine (mRNA-1647): At the
Company’s annual R&D Day, Moderna announced positive data from
the three-month interim analysis of safety and immunogenicity of
the Phase 1 study of mRNA-1647. Vaccination immunized seronegative
participants to levels consistent with or above seropositive titers
and boosted baseline titers in seropositive participants. The
vaccine was generally well-tolerated; the most common solicited
local adverse event was injection site pain, and the most common
systemic adverse events were headaches and chills. The next readout
from the study will be from a seven-month interim analysis. Moderna
also recently announced that it is advancing mRNA-1647 to a
dose-confirmation Phase 2 study in the near term. Preparation has
also begun for a pivotal Phase 3 study designed to evaluate the
efficacy of mRNA-1647 against primary CMV infection. The Phase 2
study will test the intended Phase 3 formulation, which contains
the same proprietary lipid nanoparticle (LNP) used in this Phase 1
study. mRNA-1647 is wholly owned by Moderna.
- 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.
mRNA-1653 is wholly owned by Moderna.
- Respiratory syncytial virus (RSV) vaccine (mRNA-1172 or V172):
The Phase 1 study of mRNA-1172 led by Merck is ongoing.
- Zika virus vaccine (mRNA-1893): In August, Moderna
announced that the U.S. Food and Drug Administration (FDA) granted
Fast Track designation for mRNA-1893. The Phase 1 study of
mRNA-1893 is ongoing.
- Presentations of note: Moderna presented data from its
prophylactic vaccines at IDWeek, World Vaccine Congress Europe and
the International Society of Vaccines Annual Congress, including
interim Phase 1 hMPV+PIV3 vaccine (mRNA-1653) data, preclinical
Zika vaccine (mRNA-1893) data and interim Phase 1 CMV vaccine
(mRNA-1647) data.
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 first
patients have been dosed in 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. The Phase 1
study is ongoing.
- PCV (NCI-4650): The National Cancer Institute (NCI)
Phase 1 study of NCI-4650 as a monotherapy for patients with
advanced metastatic cancers has completed enrollment with five
participants. No responses to monotherapy in this heavily
pretreated group were observed. NCI-4650 uses Moderna’s mRNA
technology but uses a different neoantigen selection process and
study design than Moderna’s Phase 1 mRNA-4157 study. Interim
results from NCI-4650 were reported at the 2019 American Society of
Clinical Oncology (ASCO) Annual Meeting in June and have been
submitted for publication.
- 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.
Intratumoral Immuno-Oncology: These programs aim to drive
anti-cancer T cell responses by injecting mRNA therapies directly
into tumors.
- OX40L (mRNA-2416): Based on available data, the Company
has decided to focus its development of mRNA-2416 for the treatment
of patients with ovarian cancer in combination with durvalumab
(IMFINZI®), a PD-L1 inhibitor. The safety cohort of the combination
arm (mRNA-2416 and durvalumab) of this Phase 1/2 study is ongoing
and will be followed by a Phase 2 expansion cohort in patients with
advanced ovarian carcinoma. The Company will not move forward with
the mRNA-2416 monotherapy ovarian cancer arm of this study.
- OX40L + IL23 + IL36γ (Triplet) (mRNA-2752): The Phase 1
trial evaluating mRNA-2752 as a single agent and in combination
with durvalumab in patients with accessible solid tumors and
lymphomas is ongoing. mRNA-2752 is an investigational mRNA
immuno-oncology therapy that encodes a novel combination of three
immunomodulators.
- IL12 (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
IL12, a potent immunomodulatory cytokine.
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 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. mRNA-1944 successfully encoded for functional
antibody (CHKV-24) in humans at all dose levels tested (0.1, 0.3
and 0.6 mg/kg). Antibody level predicted to protect against
chikungunya infection was achieved within hours and was projected
to be maintained for at least 16 weeks at the middle and high
doses. No significant adverse events were observed at the low and
middle doses; infusion-related adverse events were observed at the
high dose, which resolved spontaneously without treatment. These
results mark the first systemic mRNA therapeutic to show production
of therapeutic levels of a secreted protein in humans,
demonstrating predictable translation from preclinical species.
mRNA-1944 uses the same proprietary LNP delivery technology as the
systemic intracellular therapeutics targeting MMA and PA. The
safety and pharmacology of mRNA-1944 will continue to be explored
in two additional cohorts: one cohort with steroid premedication at
the 0.6 mg/kg dose and a second cohort with two doses of 0.3 mg/kg
(without steroid premedication) given one week apart.
- Presentation of note: In October, Moderna presented data
from the Phase 1 study of its chikungunya antibody (mRNA-1944) at
the Annual Meeting of the Oligonucleotide Therapeutics
Society.
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.
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.
- Propionic acidemia (PA) (mRNA-3927): This quarter,
Moderna announced an open Investigational New Drug (IND) and FDA
Fast Track designation for mRNA-3927. Study start-up is ongoing for
the open-label, multi-center Phase 1/2 study of multiple ascending
doses of mRNA-3927 in pediatric patients with PA in the U.S. 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.
- MMA and PA Natural History Study (MaP): As of October
2019, a total of 87 patients have been enrolled in the study (37
MMA, 50 PA). 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.
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.
Research Update
- In September, Moderna announced a multi-year research
collaboration with Harvard University with the goal of identifying
and developing novel therapeutic approaches that could improve the
lives of patients with immunological diseases. Additional funding
from Moderna to Harvard Medical School (HMS) has established an
initiative at HMS called the Alliance for RNA Therapies for the
Modulation of the Immune System (ARTiMIS), which will enable basic
science research in the field of immunology using Moderna’s mRNA
and nanoparticle delivery technology.
Corporate Updates
- Moderna appointed Tracey Franklin as Chief Human Resources
Officer.
- Moderna was named a top biopharmaceutical employer by Science
for the fifth consecutive year.
- The Company will host its Manufacturing and Digital Day on
March 4, 2020 at its Norwood, MA facility.
Financial Guidance
- The Company expects to end 2019 with approximately $1.20
billion in cash, cash equivalents and investments.
- For 2019, the Company expects net cash used in operating
activities and purchases of property and equipment to total
approximately $500 million.
- In 2020, the Company expects net cash used in operating
activities and purchases of property and equipment to be similar to
2019, between $490 million and $510 million.
Third Quarter 2019 Financial Results
- Cash Position: Cash, cash equivalents and investments as
of September 30, 2019 and December 31, 2018 were $1.34 billion and
$1.69 billion, respectively.
- Net Cash Used in Operating Activities: Net cash used in
operating activities was $363.2 million for the nine months ended
September 30, 2019 compared to $239.8 million for the same period
in 2018. Net cash used in operating activities includes $22.0
million and $25.0 million for the nine months ended September 30,
2019 and 2018, respectively, of in-licensing payments to
Cellscript, LLC and its affiliate, mRNA RiboTherapeutics, Inc., to
sublicense certain patent rights. After the first quarter of 2019,
we have no further in-licensing payment obligations to Cellscript
and its affiliate.
- Cash Used for Purchases of Property and Equipment: Cash
used for purchases of property and equipment was $24.9 million for
the nine months ended September 30, 2019 compared to $92.1 million
for the same period in 2018.
- Revenue: Total revenue was $17.0 million for the three
months ended September 30, 2019 compared to $41.8 million for the
same period in 2018. Total revenue was $46.2 million for the nine
months ended September 30, 2019 compared to $99.6 million for the
same period in 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 decreases in 2019 were due to
decreases in collaboration revenue across all our strategic
alliances, particularly AstraZeneca and Merck, largely driven by
our adoption of ASC 606. Total revenue under the previous revenue
recognition standard would have been $24.7 million and $80.2
million for the three months and nine months ended September 30,
2019, respectively.
- Research and Development Expenses: Research and
development expenses were $119.7 million for the three months ended
September 30, 2019 compared to $109.1 million for the same period
in 2018. Research and development expenses were $378.8 million for
the nine months ended September 30, 2019 compared to $303.7 million
for the same period in 2018. The increases were primarily
attributable to an increase in personnel related costs, including
stock-based compensation, with additional increases for the nine
months ended September 30, 2019 being driven by higher clinical
trial and manufacturing costs, an increase in lab supplies and
materials, and an increase in consulting and outside services.
- General and Administrative Expenses: General and
administrative expenses were $28.2 million for the three months
ended September 30, 2019 compared to $18.5 million for the same
period in 2018. General and administrative expenses were $84.0
million for the nine months ended September 30, 2019 compared to
$56.2 million for the same period in 2018. These increases were
mainly due to the additional costs of operating as a publicly
traded company, including an increase in personnel related costs
and stock-based compensation, consulting and outside services,
legal and insurance related costs.
- Net Loss: Net loss was $123.2 million for the three
months ended September 30, 2019 compared to $80.3 million for the
same period in 2018. Net loss was $390.9 million for the nine
months ended September 30, 2019 compared to $243.3 million for the
same period in 2018.
Investor Call and Webcast Information
Moderna will host a live conference call and webcast at 8:00
a.m. ET on Wednesday, November 6, 2019. To access the live
conference call, please dial 866-922-5184 (domestic) or
409-937-8950 (international) and refer to conference ID 6287715. A
webcast of the call will also be available under “Events and
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 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.
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 named a top biopharmaceutical
employer by Science for the past five years. To learn more, visit
www.modernatx.com.
_________________________________________________________________________________________
1 Biomedical 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.
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: initiating clinical trial sites outside of the U.S. for
mRNA-3704; study start-up for mRNA-3927; anticipated commencement
of a dose-confirmation Phase 2 study and planning for a pivotal
Phase 3 study for mRNA-1647; the Company’s intent to expand its
group of biopharmaceutical partners and bring in additional
non-dilutive grant and government funding; the availability of
additional funding from grants (including amounts not yet
committed); the dosing of two additional cohorts in the near term
for mRNA-1944; projected protection against chikungunya infection
for at least sixteen weeks at the 0.3 and 0.6 mg/kg doses of
mRNA-1944; the Company’s expected cash, cash equivalents, and
investments at December 31, 2019; and the Company’s expected net
cash used in operating activities and purchases of property and
equipment in 2019 and 2020. 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 the interim Phase 1 results
for mRNA-1944 will be predictive of any future clinical studies for
mRNA-1944 or other development candidates with the same LNP
formulation, including mRNA-3704 and mRNA-3927; preclinical and
clinical development is lengthy and uncertain, especially for a new
class 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 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; despite having ongoing
interactions with the FDA or other regulatory agencies, the FDA or
such other regulatory agencies may not agree with our regulatory
approval strategies, components of our or filings, such as clinical
trial designs, conduct and methodologies, or the sufficiency of
data submitted; 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.
MODERNA, INC.
CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS
(Unaudited, in
thousands)
Three Months Ended September
30,
Nine Months Ended September
30,
2019
2018
2019
2018
Revenue:
Collaboration revenue
$
13,338
$
36,662
$
37,483
$
89,696
Grant revenue
3,708
5,095
8,671
9,951
Total revenue
17,046
41,757
46,154
99,647
Operating expenses:
Research and development
119,715
109,050
378,786
303,653
General and administrative
28,188
18,525
83,994
56,229
Total operating expenses
147,903
127,575
462,780
359,882
Loss from operations
(130,857
)
(85,818
)
(416,626
)
(260,235
)
Interest income
9,252
6,519
30,546
18,129
Other expense, net
(1,767
)
(1,032
)
(5,351
)
(1,044
)
Loss before income taxes
(123,372
)
(80,331
)
(391,431
)
(243,150
)
(Benefit from) provision for income
taxes
(178
)
—
(526
)
158
Net loss
$
(123,194
)
$
(80,331
)
$
(390,905
)
$
(243,308
)
MODERNA, INC.
CONDENSED CONSOLIDATED BALANCE
SHEETS AND STATEMENTS OF CASH FLOWS DATA
(Unaudited, in
thousands)
September 30,
December 31,
2019
2018
Cash, cash equivalents and investments
$
1,338,400
$
1,694,417
Total assets
1,590,899
1,962,149
Total liabilities
339,092
431,908
Total stockholders’ equity
1,251,807
1,530,241
Total liabilities and stockholders’
equity
1,590,899
1,962,149
Nine Months Ended September
30,
2019
2018
Net cash used in operating activities
$
363,195
$
239,845
Purchases of property and equipment
24,892
92,129
View source
version on businesswire.com: https://www.businesswire.com/news/home/20191106005332/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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