Phase 2 Personalized Cancer Vaccine (mRNA-4157)
study initiated, with first patient consented
Phase 1 CMV vaccine (mRNA-1647) study fully
enrolled
Four new Phase 1 studies have begun, two in
immuno-oncology and two in infectious diseases
Vertex Pharmaceuticals extended the companies’
CF research collaboration
Ended quarter with $1.44 billion in cash, cash
equivalents and investments
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 second quarter
of 2019 and provided business updates.
New updates announced today include:
Infectious Diseases
- Enrollment complete for Phase 1 CMV vaccine (mRNA-1647)
study
- Second planned interim analysis of Phase 1 hMPV and PIV3
vaccine (mRNA-1653) study showed antibody titers remained above
baseline at all dose levels at seven months
- First subject dosed in Phase 1 RSV vaccine (mRNA-1172 or Merck
V172) study
- First subject dosed in Phase 1 Zika vaccine (mRNA-1893)
study
Immuno-Oncology
- First patient consented in Phase 2 PCV (mRNA-4157) study in
patients with resected melanoma
- First patient dosed in Phase 1 KRAS cancer vaccine (mRNA-5671
or Merck V941) study
- First patient dosed in Phase 1 study of mRNA encoding IL12
(MEDI1191) injected intratumorally
Rare Diseases
- Six of eight subjects dosed in the third cohort (0.6 mg/kg) of
the Phase 1 antibody against chikungunya virus (mRNA-1944) study;
the lipid nanoparticle (LNP) formulation used for mRNA-1944 is also
utilized in Moderna’s MMA program
- Three clinical sites actively recruiting for Phase 1/2 MMA
(mRNA-3704) study
Research
- Vertex Pharmaceuticals extended the companies’ research
collaboration in cystic fibrosis (CF)
“Since our last quarterly update, our personalized cancer
vaccine program with Merck has advanced into Phase 2, the
cytomegalovirus vaccine Phase 1 study has completed enrollment and
four new Phase 1 trials in immuno-oncology and infectious diseases
have begun,” said Stéphane Bancel, Moderna’s chief executive
officer. “We are pleased by the advancement of our programs and
look forward to sharing new clinical data in the near term.
Additionally, we are excited that Vertex extended our research
collaboration based on the teams’ progress to date. Our balance
sheet remains strong, and we continue to deploy our capital toward
the advancement and expansion of our development pipeline and the
creation of potential new modalities.”
Moderna currently has 21 mRNA development candidates in its
portfolio with 13 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
https://www.modernatx.com/pipeline. Moderna and collaborators have
published more than 35 peer-reviewed papers, including 23 over the
last 12 months.
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-1172 or
V172): The first subject has been dosed in the Phase 1 study of
mRNA-1172 led by Merck. mRNA-1172 has shown enhanced potency in
preclinical studies compared with the companies’ first RSV
candidate (mRNA-1777).
- Cytomegalovirus (CMV) vaccine (mRNA-1647): The Phase 1
study of mRNA-1647 has been fully enrolled, at a top dose of 300µg.
This randomized, observer-blind and placebo-controlled study is
designed to evaluate the safety and immunogenicity of mRNA-1647, a
vaccine encoding the gB and pentamer complexes of CMV. CMV is a
common human pathogen and a leading cause of birth defects.
- Human metapneumovirus (hMPV) and parainfluenza type 3 (PIV3)
vaccine (mRNA-1653): Topline data from a second planned interim
analysis of an ongoing Phase 1 randomized, observer-blind,
placebo-controlled and dose-ranging study in healthy adults showed
that hMPV and PIV3 serum neutralizing antibody titers remained
above baseline through seven months and the vaccine was generally
well-tolerated. Topline data from the study’s first interim
analysis, announced in February 2019, showed that mRNA-1653 boosted
antibody titers one month after vaccination at all dose levels and
was generally well-tolerated. Data from the study will be presented
at IDWeek on October 2 - 6, 2019 in Washington, D.C. In a recent
type C meeting with the U.S. Food and Drug Administration (FDA),
the Company discussed a potential path forward to evaluate
protection against both hMPV and PIV3 in a single Phase 3 study.
Consistent with this development path, Moderna is planning to
initiate a Phase 1b study of mRNA-1653 in seropositive toddler
subjects as the next step.
- Zika virus vaccine (mRNA-1893): The first subject has
been dosed in the Phase 1 study of mRNA-1893. 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.
- Publications of note: In May, Moderna published its
clinical data in Vaccine from two Phase 1 studies showing that mRNA
vaccines against H10N8 and H7N9 influenza viruses were
well-tolerated and elicited robust immune responses. In July,
Moderna published preclinical data in the Journal of Infectious
Diseases demonstrating the ability of mRNA vaccines to protect
against congenital Zika virus infection in mice.
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): The first patient has been consented for the
randomized Phase 2 study investigating Merck’s pembrolizumab
(KEYTRUDA®) in combination with a 1 mg dose of mRNA-4157, compared
to pembrolizumab alone, for the adjuvant treatment of high-risk
resectable melanoma. Additionally, the protocol for the ongoing
Phase 1 trial has been amended to include a cohort of 17 patients
who are refractory to PD-1 inhibitors.
- KRAS vaccine (mRNA-5671 or V941): The first patient was
dosed in 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. The study, led by Merck, will
enroll patients with KRAS mutant advanced or metastatic non-small
cell lung cancer, colorectal cancer or pancreatic adenocarcinoma,
and centrally confirmed Human Leukocyte Antigen (HLA) HLA-A*1101
and/or HLA-C*0802 allele expression. mRNA-5671 is designed to
generate and present the four most prevalent KRAS mutations as
neoantigens to the immune system. These four mutations comprise an
estimated 80-90 percent of KRAS mutations in the study
indications.
- Presentations of note: Moderna presented interim data
from its ongoing Phase 1 study of PCV candidate mRNA-4157,
demonstrating safety, tolerability and immunogenicity of mRNA-4157
alone and in combination with pembrolizumab, at the 2019 American
Society of Clinical Oncology (ASCO) Annual Meeting. Additionally,
the National Cancer Institute (NCI) also presented data at ASCO
from its Phase 1 study of PCV mRNA-4650 as a monotherapy for
patients with advanced metastatic cancers. The NCI program uses
Moderna’s mRNA technology but uses a different neoantigen selection
process and study design than Moderna’s Phase 1 mRNA-4157
study.
Intratumoral Immuno-Oncology: These programs aim to drive
anti-cancer T cell responses by injecting mRNA therapies directly
into tumors.
- OX40L (mRNA-2416): Dosing is ongoing at the highest
levels (8 mg) in the Phase 1/2 open-label, multi-center, dose
escalation and efficacy study of intratumoral injections of
mRNA-2416 in patients with advanced malignancies. A Phase 2
expansion cohort in patients with advanced ovarian carcinoma is
preparing to start enrollment; this will include the combination of
intratumoral mRNA-2416 with durvalumab (IMFINZI®).
- OX40L + IL23 + IL36γ (Triplet) (mRNA-2752): The first
patient was dosed in the combination arm of the Phase 1 trial of
mRNA-2752. This study is evaluating mRNA-2752 as a single agent and
in combination with durvalumab in patients with accessible solid
tumors and lymphomas. mRNA-2752 is an investigational mRNA
immuno-oncology therapy that encodes a novel combination of three
immunomodulators.
- IL12 (MEDI1191): The first patient was dosed with
MEDI1191 monotherapy in the Phase 1 open-label, multi-center study
of intratumoral injections of MEDI1191 alone and in combination
with a checkpoint inhibitor in patients with advanced solid tumors,
being led by AstraZeneca. 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): Six
of eight subjects have been dosed in the third cohort (0.6 mg/kg)
of the Phase 1 antibody against chikungunya virus (mRNA-1944)
study. This study is evaluating the safety and tolerability of
escalating doses of mRNA-1944 via intravenous infusion in healthy
adults. mRNA-1944 is the first monoclonal antibody encoded by mRNA
to be dosed in a human and the first development candidate from
Moderna’s systemic secreted therapeutics modality to start clinical
testing. The lipid nanoparticle (LNP) formulation used for
mRNA-1944 is also utilized in Moderna’s MMA program.
- Publication of note: In May, Moderna published
preclinical data in Science Immunology showing mRNA encoding an
antibody against the chikungunya virus (mRNA-1944) was
well-tolerated, resulted in linear dose-dependent protein
expression and provided complete protection in preclinical
species.
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): Three clinical
trial sites are open and actively recruiting patients for the Phase
1/2 open-label, dose escalation study evaluating mRNA-3704 for the
treatment of MMA. The objectives of the study are to evaluate
safety and tolerability, assess the pharmacodynamic response and
characterize the pharmacokinetic profile of mRNA-3704. Moderna
recently updated the study protocol to widen the age bracket of the
first cohort for this trial to allow for the enrollment of
pediatric patients (now includes patients 8 years and older, a
modification from adolescents aged 12-18.) This is Moderna’s first
rare disease program to advance into clinical testing.
- MMA and propionic acidemia (PA) Natural History Study
(MaP): As of July 15, 2019, a total of 71 patients have been
enrolled in the study (35 MMA, 36 PA). 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.
- Publication of note: In July, Moderna published
preclinical data in EBioMedicine, a Lancet publication, showing
mRNA therapy demonstrated long-term efficacy and safety, with
dose-dependent and reproducible biomarker responses, in mouse
models of MMA.
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
- Vertex CF research collaboration: In July 2016, Moderna
and Vertex announced an exclusive research collaboration and
licensing agreement aimed at the discovery and development of mRNA
therapeutics for the treatment of CF. Based on preclinical work to
date, Vertex has extended this collaboration through the first
quarter of 2020 with options to extend further based on future
progress. Pulmonary mRNA delivery represents a potential new route
of administration for Moderna.
Annual R&D Day
- Moderna will host its annual R&D Day in New York City on
September 12, 2019.
Investor Call and Webcast Information
Moderna will host a live conference call and webcast at 8:00
a.m. ET on Wednesday, August 7, 2019. To access the live conference
call, please dial 866-922-5184 (domestic) or 409-937-8950
(international), and refer to conference ID 4799277. 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.
Second Quarter 2019 Financial Results
- Cash Position: Cash, cash equivalents and investments as
of June 30, 2019 and December 31, 2018 were $1.44 billion and $1.69
billion, respectively.
- Net Cash Used in Operating Activities: Net cash used in
operating activities was $256.1 million for the six months ended
June 30, 2019 compared to $159.6 million for the six months ended
June 30, 2018. Net cash used in operating activities includes $22.0
million and $25.0 million for the six months ended June 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 $18.2 million for
the six months ended June 30, 2019 compared to $66.0 million for
the six months ended June 30, 2018.
- Revenue: Total revenue was $13.1 million for the three
months ended June 30, 2019 compared to $28.9 million for the three
months ended June 30, 2018. Total revenue was $29.1 million for the
six months ended June 30, 2019 compared to $57.9 million for the
six months ended June 30, 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
$16.9 million and $55.5 million for the three months and six months
ended June 30, 2019, respectively.
- Research and Development Expenses: Research and
development expenses were $128.5 million for the three months ended
June 30, 2019 compared to $104.5 million for the three months ended
June 30, 2018. The increase was primarily attributable to an
increase in personnel related costs including stock-based
compensation, an increase in lab supplies and materials, and an
increase in clinical trial and manufacturing costs. Research and
development expenses were $259.1 million for the six months ended
June 30, 2019 compared to $194.6 million for the six months ended
June 30, 2018. The increase was primarily attributable to an
increase in personnel related costs including stock-based
compensation, an increase in 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 $28.5 million for the three months
ended June 30, 2019 compared to $21.4 million for the three months
ended June 30, 2018. The increase was mainly due to an increase in
personnel related costs including stock-based compensation, and an
increase in consulting and outside services. General and
administrative expenses were $55.8 million for the six months ended
June 30, 2019 compared to $37.7 million for the six months ended
June 30, 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,
an increase in consulting and outside services, and an increase in
information technology, facility and insurance related costs.
- Net Loss: Net loss was $135.1 million for the three
months ended June 30, 2019 compared to $90.6 million for the three
months ended June 30, 2018. Net loss was $267.7 million for the six
months ended June 30, 2019 compared to $163.0 million for the six
months ended June 30, 2018.
2019 Expected Cash Position
- Moderna reiterated its expectation for cash, cash equivalents
and investments at December 31, 2019 to be in the range of $1.15
billion to $1.20 billion.
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. The Company’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: sharing new clinical data in the near term; the
advancement and expansion of Moderna’s development pipeline and the
creation of potential new modalities; the anticipated commencement
of Moderna’s clinical studies, including the Phase 1b study of
mRNA-1653 in seropositive toddler subjects and the Phase 2
expansion cohort of mRNA-2416 in patients with advanced ovarian
carcinoma; whether mRNA-5671 will generate and present KRAS
neoantigens from the four most prevalent KRAS mutations; and the
Company’s expected cash, cash equivalents, and investments at
December 31, 2019. 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: 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.
KEYTRUDA is a registered trademark of Merck Sharp & Dohme
Corp., a subsidiary of Merck & Co., Inc. IMFINZI is a
registered trademark of AstraZeneca AB.
MODERNA, INC.
CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS
(Unaudited, in
thousands)
Three Months Ended June
30,
Six Months Ended June
30,
2019
2018
2019
2018
Revenue:
Collaboration revenue
$
10,030
$
25,831
$
24,145
$
53,291
Grant revenue
3,053
3,020
4,963
4,599
Total revenue
13,083
28,851
29,108
57,890
Operating expenses:
Research and development
128,496
104,479
259,071
194,603
General and administrative
28,523
21,387
55,806
37,704
Total operating expenses
157,019
125,866
314,877
232,307
Loss from operations
(143,936
)
(97,015
)
(285,769
)
(174,417
)
Interest income
10,322
6,401
21,294
11,610
Other (expense) income, net
(1,764
)
171
(3,584
)
(12
)
Loss before income taxes
(135,378
)
(90,443
)
(268,059
)
(162,819
)
(Benefit from) provision for income
taxes
(324
)
158
(348
)
158
Net loss
$
(135,054
)
$
(90,601
)
$
(267,711
)
$
(162,977
)
MODERNA, INC.
CONDENSED CONSOLIDATED BALANCE
SHEETS AND STATEMENTS OF CASH FLOWS DATA
(Unaudited, in
thousands)
June 30,
December 31,
2019
2018
Cash, cash equivalents and investments
$
1,435,378
$
1,694,417
Total assets
1,685,279
1,962,149
Total liabilities
346,725
431,908
Total stockholders’ equity
1,338,554
1,530,241
Six Months Ended June
30,
2019
2018
Net cash used in operating activities
$
(256,147
)
$
(159,634
)
Cash used for purchases of property and
equipment
(18,181
)
(65,989
)
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190807005154/en/
Media: 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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