[September 14, 2017] |
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Moderna Announces Pipeline and Corporate Update
Moderna Therapeutics, a clinical stage biotechnology company that is
pioneering messenger RNA (mRNA) therapeutics and vaccines to create a
new generation of transformative medicines for patients, provided an
update today, noting continued progress of clinical programs and the
introduction of several new development candidates (DCs) across its
broad, diverse development pipeline. Moderna announced the update in
conjunction with an Investor R&D Day hosted by its management team this
morning in New York.
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Moderna Development Pipeline as of September 14, 2017 (Graphic: Business Wire)
A Phase 1 study of mRNA AZD-8601, the first-ever mRNA therapeutic to be
evaluated in a clinical study, has been successfully completed. mRNA
AZD-8601 is anticipated to move into Phase 2 development. mRNA AZD-8601
is being developed by Moderna's partner AstraZeneca to express a local
and transient surge of vascular endothelial growth factor-A (VEGF-A) as
a potential treatment for cardiovascular diseases. The Phase 1
randomized, double-blind, placebo-controlled, single ascending dose
study, which assessed the safety, tolerability and pharmacokinetics (PK)
of mRNA-AZD-8601 after single dose administration in male patients with
Type 2 diabetes mellitus was successfully completed. The study met its
primary endpoint of safety and tolerability and also demonstrated proof
of mechanism as measured by expression of VEGF-A protein in the skin
(protein PK).
AstraZeneca has submitted a Clinical Trial Application (CTA) in Europe
to initiate a Phase 2a study of mRNA AZD-8601 in heart failure patients
undergoing coronary artery bypass grafting (CABG) surgery.
Moderna initiated a Phase 1 study of mRNA-2416, an intratumoral (iTu)
immuno-oncology (I-O) therapeutic that encodes for the membrane
expression of the co-stimulatory protein OX40 Ligand, or OX40L, to
potentially enhance T-cell attack against tumors. This is Moderna's
first I-O therapeutic to enter clinical study.
Moderna also unveiled its first clinical development candidate (DC) that
utilizes a novel modality, liver expression of therapeutic proteins.
Utilizing this modality, the company will advance toward the clinic an
mRNA DC, mRNA-3704, for methylmalonic acidemia (MMA), a serious
and often life-threatening rare liver disease for which there are no
approved therapies.
In total, Moderna announced the addition of four new DCs to its
development pipeline today. The company also announced that it has
replaced its preclinical stage Zika vaccine DC, mRNA-1706, with
mRNA-1893, a Zika vaccine with an enhanced mRNA construct anticipated to
augment immunogenicity. Moderna's development pipeline now comprises 16
mRNA therapeutics and vaccines spanning infectious diseases, cancer
(I-O), cardiovascular diseases and rare liver diseases. Approximately
460 subjects have been dosed to date across clinical studies for seven
mRNA vaccines and therapeutics.
"2017 is a major inflection point for Moderna, as we've made significant
progress advancing mRNA therapeutics for unmet needs across several
disease areas," said Stéphane Bancel, Chief Executive Officer of
Moderna. "In the cardiovascular space, our partner AstraZeneca
successfully completed a Phase 1 study for mRNA-AZD-8601, a VEGF-A
therapeutic, and planning is underway for a Phase 2a study. In addition,
we have initiated first-in-human dosing in a Phase 1 study of mRNA-2416,
our first immuno-oncology candidate, encoding OX40 ligand. We also have
progressed the expression of therapeutic proteins in the liver as a
development stage modality. As such, we are now able to advance
chronically dosed mRNA therapeutics to development for rare liver
diseases. I'm very thankful to the Moderna team for their commitment,
dedication and continued progress to deliver on our mission."
Mr. Bancel added, "This past May we experienced the extremely sad,
untimely loss of Henri Termeer, a Moderna board member, and a dear
friend and mentor to so many of us at Moderna and across our industry.
Henri was tirelessly dedicated to helping patients, particularly those
impacted by rare diseases. We hope to honor his remarkable legacy by
delivering on the promise of mRNA science for patients."
CLINICAL AND DEVELOPMENT PROGRAM UPDATES
mRNA is a fundamental component of human biology, giving cells the
instructions they need to make proteins that carry out every function of
the body. Moderna is using mRNA as a drug to direct cells in the body to
produce proteins to fight or prevent disease. Moderna combines elements
of its mRNA platform into distinct approaches, called modalities, to
address diseases. A modality is a technological solution set that
Moderna can deploy to create a family of medicines for different
diseases.
Moderna's pipeline now comprises 16 mRNA DCs across five modalities:
prophylactic vaccines, therapeutic vaccines, intratumoral
immuno-oncology therapeutics, localized therapeutics and liver
therapeutics. Moderna also has an mRNA lung therapeutics modality that
is currently in the research stage, in which Vertex (News - Alert) and Moderna are
researching potential mRNA therapeutics for cystic fibrosis.
"We are very pleased with the progress our team has made advancing our
proprietary, next generation formulations, including N2GL," said Stephen
Hoge, President of Moderna. "Based on our preclinical data we believe
these represent a dramatic step change in performance, which we will
submit for publication in the coming months. Combining formulations like
N2GL with other platform improvements has allowed us to progress mRNA
medicines for devastating rare metabolic diseases into development, the
first of which is methylmalonic acidemia, or MMA."
mRNA Prophylactic Vaccines Modality
Prophylactic Vaccines - Clinical Development Updates
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Enrollment completed for Phase 1 studies of mRNA-1440 (influenza A
virus subtype H10N8 vaccine) and mRNA-1851 (influenza A virus subtype
H7N9 vaccine): The ongoing Phase 1 studies for mRNA-1440 (H10N8
mRNA vaccine) and mRNA-1851 (H7N9 mRNA vaccine) have completed
enrollment, with 201 and 156 healthy volunteers enrolled,
respectively. Both studies remain active, with subjects continuing to
be followed for safety monitoring.
Moderna published
interim data from the mRNA-1440 Phase 1 study in April 2017 in the
journal Molecular Therapy, and plans to publish complete
findings from both the mRNA-1440 and mRNA-1851 Phase 1 studies in 2018.
As
previously described,
these first two programs were strategically selected with the goal of
quickly assessing both the safety and efficacy of Moderna's mRNA
vaccine platform in humans. H10N8 and H7N9 both represent influenza
strains with pandemic potential. Currently, there is no approved
vaccine for either strain of the virus.
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Phase 1 study of mRNA MRK-1777 continues to progress: A Phase 1
randomized, placebo-controlled, dose-ranging study of mRNA MRK-1777,
an mRNA prophylactic vaccine program for an undisclosed target,
continues to enroll healthy volunteers. mRNA MRK-1777 is the first
named DC under the existing collaboration
between Merck and Moderna to discover and develop mRNA-based vaccines
against viral diseases. Moderna is conducting the Phase 1 study of
mRNA MRK-1777.
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Phase 1 study of Chikungunya vaccine, mRNA-1388, initiated: In
August, Moderna began enrolling healthy volunteers in the US for a
Phase 1 study of mRNA-1388, an mRNA Chikungunya prophylactic vaccine.
The randomized, placebo-controlled, dose-ranging study will evaluate
the safety and immunogenicity of mRNA-1388 in healthy adults.
Chikungunya
typically causes mild fever and transient joint pain. In approximately
15 percent of infected patients, it can cause long-term, severe
arthritis. Chikungunya historically has been limited to warmer
climates in Asia and Africa, but recent cases have been identified in
the Americas and Europe. Currently, there is no approved vaccine for
Chikungunya.
Clinical development of mRNA-1388 is supported
by an award from the Defense Advanced Research Projects Agency (DARPA),
an agency of the U.S. Department of Defense.
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Phase 1/2 study of Zika mRNA vaccine, mRNA-1325, continues to
progress: Moderna continues to enroll healthy subjects in a Phase
1/2 study of mRNA-1325, a Zika mRNA vaccine. The randomized,
placebo-controlled, dose-ranging study is underway in the US.
Children
born to mothers infected with Zika can develop microcephaly, a severe
disease characterized by abnormally small heads and severe neurologic
disabilities. Zika infection is also strongly associated with
Guillain-Barré Syndrome (GBS), an autoimmune disease that attacks the
peripheral nervous system, leading to rapidly progressive and
potentially life-threatening muscle weakness. GBS can lead to death
caused by respiratory arrest if a patient is not ventilated.
Currently, there are no treatment options or approved vaccines for the
Zika virus or congenital Zika syndrome.
Moderna received a
funding award of up to $125 million from 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), to accelerate
development of its Zika mRNA vaccine.
Prophylactic Vaccines - Preclinical Development Updates
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Additional mRNA prophylactic vaccine DCs continue to advance: Moderna
continued to make progress advancing three additional mRNA
prophylactic vaccine DCs toward the clinic, all of which utilize one
of the company's proprietary, novel formulations, V1GL:
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mRNA-1893, a Zika mRNA vaccine, replaced mRNA-1706, a Zika
mRNA vaccine previously announced by Moderna. mRNA-1706 contained
the same active pharmaceutical ingredient (API) as Moderna's
mRNA-1325 Zika mRNA vaccine, but utilized Moderna's V1GL
formulation. Based on experience with other mRNA-encoded antigens,
the leader sequence of the Zika antigen in mRNA-1325, which
encodes a signal peptide that instructs the cell to secrete the
protein, was from an Immunoglobulin E, or IgE, protein (a type of
antibody).
In a paper published
in Cell on Moderna's Zika mRNA vaccine, researchers found
that using a leader sequence from the Japanese encephalitis virus
(JEV) led to improved immunogenicity and superior protection
versus IgE in viral challenge models.
Following further
investigation, Moderna has decided to replace mRNA-1706 with a new
DC, mRNA-1893, that uses the JEV signal peptide. mRNA-1893 also
utilizes Moderna's V1GL formulation. While GLP toxicology studies
had been successfully completed for mRNA-1706, given mRNA-1893 has
a unique API, Moderna will now proceed with conducting GLP
toxicology studies on this new, enhanced Zika mRNA vaccine.
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mRNA-1647, a cytomegalovirus (CMV) mRNA vaccine, which
consists of six mRNAs, including five proteins (gH, gL, UL128,
UL130 and UL131A) designed to express the Pentamer complex, and
another CMV antigen, the herpesvirus glycoprotein (gB) protein.
GLP toxicology studies have been successfully completed for
mRNA-1647.
CMV is the most common cause of newborn
disability, leading to deafness, microcephaly (small, not fully
developed heads and severe disabilities), vision loss and mental
deficiencies, among other serious complications. It is also the
most frequent viral disease in transplant recipients, often
leading to transplant failure. Currently, there is no approved
vaccine for CMV.
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mRNA-1653, a combination human metapneumovirus (HMPV) and
parainfluenza virus (PIV3) vaccine. GLP toxicology studies
have been successfully completed for mRNA-1653. HMPV
and PIV3 typically cause mild respiratory illness, but can become
severe in young children, the elderly and other immunocompromised
adults. HMPV and PIV3 are the second and third most common causes,
respectively, of lower respiratory hospitalizations in children,
behind RSV. Currently, there is no approved vaccine for either
HMPV or PIV3.
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Second Merck-partnered infectious disease mRNA vaccine DC named,
mRNA MRK-V213: Merck named its second DC, mRNA MRK-V213, under its
eisting collaboration
with Moderna to discover and develop mRNA-based vaccines against viral
diseases. mRNA MRK-V213 is for an undisclosed infectious disease
indication.
Prophylactic Vaccines Publications
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First human proof-of-concept data published in Molecular Therapy:
In April 2017, Moderna published
in the journal Molecular Therapy positive interim data from a
100 µg intramuscular (IM) cohort from its ongoing Phase 1 study of
mRNA-1440, an influenza A virus subtype H10N8 mRNA prophylactic
vaccine. 31 subjects were enrolled in the cohort, 23 of whom received
mRNA-1440 and 8 of whom received placebo. mRNA-1440 demonstrated
strong efficacy based Hemagglutination Inhibition Assay (HAI) and
Microneutralization Assay (MN) titers, two measures of immunogenicity
in response to vaccination, and was safe and well-tolerated. 100% (n =
23) and 87% (n = 20) achieved titers consistent with protection at day
43 as measured by HAI titers and MN titers, respectively, compared to
0% in the placebo arm.
These were the first-ever published
data demonstrating an mRNA vaccine's ability to elicit robust
prophylactic immunity in humans, as well as the first human
proof-of-concept data from Moderna's mRNA platform.
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First Zika mRNA vaccine preclinical data published in Cell: In
February, the first pre-clinical data for Moderna's Zika mRNA vaccine
were published
in Cell. The data demonstrated
that an mRNA vaccine encoding for Zika prM-E protected against Zika
virus in three different mouse strains after two doses: prime plus
boost. Extraordinarily high titers of neutralizing antibodies were
produced, achieving sterilizing immunity. In addition, a fusion loop
mutant vaccine reduced production of potentially disease-enhancing
anti-Dengue antibodies. The research was led by Moderna scientists and
researchers at the Washington University School of Medicine.
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Preclinical Data Showing Zika mRNA Vaccine Prevents In Utero
Transmission Published in Cell: In July, data were published
in Cell demonstrating that Moderna's Zika mRNA vaccine
prevented Zika virus transmission from pregnant mice to their fetuses.
The findings also demonstrated
that Moderna's Zika mRNA vaccine protected the placenta and fetus from
Zika virus-induced injury. In the study, Moderna's Zika mRNA vaccine
was evaluated in addition to a live-attenuated vaccine candidate
developed by the University of Texas Medical Branch (UTMB). The
research was conducted by scientists from the National
Institute of Allergy and Infectious Diseases (NIAID), part of the
National Institutes of Health (NIH), Washington
University School of Medicine and UTMB.
mRNA Therapeutic Vaccines Modality
Moderna's mRNA therapeutic vaccines utilize one of the company's
proprietary formulations, V1GL.
Therapeutic Vaccines - Clinical Development Updates
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IND for mRNA-based personalized cancer vaccine (PCV), mRNA-4157,
filed and deemed safe to proceed to clinic: Moderna filed an IND
with the FDA for mRNA-4157, its mRNA-based PCV, and has received a
safe to proceed notification from the FDA.
Moderna, under
its existing collaboration
with Merck to develop personalized cancer vaccines (PCVs), will
evaluate mRNA-4157 in combination with Merck's anti-PD-1 therapy,
KEYTRUDA® (pembrolizumab). Under the IND, Moderna will
identify neoepitopes present in a patient's tumor and then create an
mRNA-based PCV encoding for approximately 20 neoepitopes. When
injected into the patient, the mRNA-based PCV will direct the
patient's cells to express the selected neoepitopes. In turn, this may
help the patient's immune system better recognize cancer cells as
foreign and eradicate them. mRNA-4157 also has the potential to
enhance clinical outcomes associated with checkpoint inhibitor
therapies. Leveraging its rapid cycle time, small-batch manufacturing
technique and digital infrastructure, Moderna plans to manufacture and
supply each individually tailored and manufactured PCV to patients
within weeks.
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National Cancer Institute (NCI) to sponsor and conduct study of
mRNA-based PCV: In collaboration with Moderna, the Surgery Branch
of the NCI's Center for Cancer Research will sponsor and conduct a
Phase 1/2 study to investigate the safety and immunogenicity of
mRNA-based PCVs for patients with advanced-stage, metastatic cancers
under the direction of Steven A. Rosenberg, MD, Ph.D., Chief of
Surgery, NCI. NCI will supply Moderna with amino acid sequences of
confirmed and predicted neoepitopes for up to 12 patients. Based on
these sequences, Moderna will manufacture an mRNA-based personalized
cancer vaccine (NCI-4650) for each patient.
Therapeutic Vaccines - Preclinical Development Updates
New KRAS cancer vaccine DC named, mRNA-5671: Moderna has named a
novel cancer vaccine DC that encodes for KRAS epitopes. KRAS is one of
the most frequently mutated oncogenes in human cancer (approximately 30%
of all cases). KRAS mutations are found principally in non-small cell
lung cancer (NSCLC), colorectal cancer and pancreatic cancer, and are
associated with worse outcomes. Hotspots of KRAS mutations are found in
different tumor types and can serve as tumor rejection epitopes.
Presentation of these epitopes to the immune system may elicit a robust
anti-tumor response.
For this vaccine, Moderna plans to include an mRNA that encodes for the
four most commonly found KRAS mutations, which will cover most of the
mutations that occur in NSCLC, colorectal cancer and pancreatic cancer.
Moderna has successfully completed GLP toxicology studies for mRNA-5671.
mRNA Intratumoral (iTu) Immuno-Oncology (I-O)
Modality
Moderna's intratumoral (iTu) immuno-oncology (I-O) therapeutics utilize
one of the company's proprietary formulations, N1GL.
iTu I-O Therapeutics - Clinical Development Updates
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Phase 1 study of mRNA-2416 (encoding for OX40 Ligand, or OX40L)
initiated: In August, Moderna began dosing for a Phase 1 study of
mRNA-2416, an iTu I-O therapeutic targeting OX40L. This marks the
first I-O program from Moderna's pipeline to move into clinical study.
The
Phase 1 open-label, multicenter, dose escalation study of mRNA-2416 is
designed to determine the safety and tolerability of escalating iTu
doses of mRNA-2416 in patients with relapsed/refractory solid tumor
malignancies or lymphoma, and define the maximum tolerated dose (MTD)
and recommended dose for expansion (RDE) and schedule for iTu
injections of mRNA-2416. The study will enroll patients up to 10 sites
in the US.
OX40L is a powerful co-stimulatory protein that
enhances the expansion, function and survival of T cells to mount an
attack against cancer cells. When mRNA-2416 is delivered directly into
a tumor, cells in the tumor express the OX40L protein as a
membrane-bound homotrimer on their surface, which, in turn, may lead
to a stronger T cell attack against the tumor. mRNA-2416 may elicit an
abscopal effect in metastatic cancer, in which localized injection
into one tumor would lead not only to shrinking of that tumor but also
shrinking of tumors elsewhere in the body.
iTu I-O Therapeutics - Preclinical Development Updates
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iTu triple combination I-O therapeutic, mRNA-2752, nominated: mRNA-2752
is a triple combination I-O therapeutic that leverages the
multi-target activation enabled by Moderna's mRNA technology to
enhance immune response against tumors. Specifically, mRNA-2752
comprises three mRNAs that express OX40L, Interleukin 23 (IL23) and
Interleukin 36 ? (IL36?). OX40L is a powerful co-stimulatory protein
that enhances the expansion, function and survival of T cells to mount
an attack against cancer cells. IL23 and IL36? are secreted proteins
that have established roles in mediating immune responses and have
been implicated in driving various inflammatory diseases.
The
three mRNAs are co-formulated and injected directly into a tumor. iTu
delivery may enable delivery of targets locally that are too toxic
systemically. mRNA-2752 may also elicit an abscopal effect in
metastatic cancer. In addition, based on pre-clinical models, Moderna
anticipates synergies combining mRNA-2752 with checkpoint inhibitors.
In
April, Moderna gave oral and poster presentations at the American
Association for Cancer Research (AACR) Annual Meeting in Washington,
D.C., demonstrating that the triple combination of OX40L, IL23 and
IL36? and had potent and durable anti-tumor activity in multiple
preclinical models.
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GLP toxicology studies for mRNA IL12 iTu I-O, mRNA-2095, ongoing:
GLP toxicology studies continue for mRNA-2905, an iTu I-O mRNA
therapeutic that encodes for IL12, a powerful cytokine that activates
the immune system after being released from cells. When mRNA-2905, a
partnered program with AstraZeneca, is delivered directly into a
tumor, cells in the tumor express IL12 at a high concentration in the
local microenvironment, which, in turn, may lead to a stronger T cell
attack against the tumor. By expressing IL12 locally, systemic side
effects that previously have been seen from delivery of the IL12
protein into the blood may be more manageable. Moderna is also
investigating the potential of mRNA-2905 to elicit an abscopal effect
in metastatic cancer. Combining mRNA-2905 with a checkpoint inhibitor
may improve outcomes from cancer therapy.
mRNA-2905 is
being developed through a collaboration Moderna announced in
2016 with AstraZeneca to discover, co-develop and co-commercialize
immuno-oncology mRNA therapeutics.
mRNA Localized Therapeutics Modality
Localized Therapeutics - Clinical Development Update
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Phase 1 study of mRNA AZD-8601 completed enrollment; CTA filed for
Phase 2a Study: A Phase 1 study for mRNA AZD-8601, an mRNA
localized therapeutic being developed by Moderna's partner AstraZeneca
that encodes for vascular endothelial growth factor-A (VEGF-A), has
been successfully completed. The Phase 1 randomized, double-blind,
placebo-controlled, single ascending dose study assessed the safety,
tolerability and pharmacokinetics (PK) of mRNA AZD-8601 after single
dose administration in male patients with Type 2 diabetes mellitus.
The study met its endpoint of safety and tolerability and also
demonstrated proof of mechanism as measured by expression of VEGF-A
protein in the skin (protein PK).
When directed via local
tissue injection, VEGF-A mRNA may potentially lead to the creation of
more blood vessels and improved blood supply. mRNA AZD-8601 could one
day provide a unique regenerative treatment option for patients with
heart failure or after a heart attack, as well as for diabetic wound
healing and other ischemic vascular diseases.
AstraZeneca
has submitted a Clinical Trial Application (CTA) in Europe to initiate
a Phase 2a study of mRNA AZD-8601 in heart failure patients undergoing
coronary artery bypass grafting (CABG) surgery.
"The potential impact of harnessing mRNA as a therapeutic to address
human disease has long been understood, but the possibility of
translating this potential into reality had remained elusive given the
complexities of using mRNA as a drug," said Tal Zaks, Chief Medical
Officer at Moderna. "We've reached an important milestone with the
successful completion of the Phase 1 study of mRNA-AZD-8601. And the
Phase 2a study of mRNA-AZD-8601 in patients undergoing coronary artery
bypass grafting surgery will provide further insight into the potential
of VEGF-A mRNA to provide a unique regenerative treatment option for
patients with cardiovascular disease. Seeing the first mRNA therapeutic
program move to Phase 2 study will be an important step for the entire
mRNA field."
mRNA Liver Therapeutics Modality
Moderna's liver therapeutics utilize one of the company's proprietary
formulations, N2GL.
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New liver therapeutics modality advanced from research to
development stage: Moderna advanced liver therapeutics from a
research modality to a development modality through technology
advancements that are believed to enable intravenous (IV) chronic
dosing of mRNA therapeutics that will express protein in the liver.
This may enable Moderna to address many serious diseases that are
undruggable with existing drug development approaches. This is
Moderna's fifth modality to advance to the development stage.
Many
diseases, including many rare genetic diseases, are caused by defects
or deficits in proteins expressed by liver cells. By delivering mRNA
drugs via IV to the liver, and directing liver cells to express
functional proteins, Moderna can potentially stimulate production of
therapeutic proteins in ways that cannot be achieved with other
current technologies.
Liver Therapeutics - Preclinical Development Updates
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First liver therapeutic DC nominated, mRNA-3704 for methylmalonic
acidemia (MMA), a rare liver disease: MMA is a rare, autosomal
recessive organic acidemia/aciduria, most commonly (approximately 60%
of cases) caused by a deficiency of the enzyme methylmalonic CoA
mutase, or MUT, due to a defective or missing MUT gene.
MMA
is primarily a pediatric disease with onset in early infancy. The
majority of patients with MMA have no functional MUT enzyme, leading
to, on average, three life-threatening metabolic crises per year. As a
result, MMA is associated with significant mortality and morbidity,
and there are no approved therapies. Standard of care includes dietary
and palliative measures. Currently, liver and/or kidney transplant is
the only effective treatment.
mRNA-3704 is being developed
as a chronically dosed mRNA therapeutic that directs cells in the
liver to produce and express the MUT enzyme, restoring the metabolic
pathway to reduce toxic metabolite build-up.
YEAR-TO-DATE BUSINESS UPDATES AND HIGHLIGHTS
Board of Directors Updates
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Israel Ruiz appointed to Board of Directors: In February,
Moderna announced
that Israel Ruiz, Executive Vice President and Treasurer of the
Massachusetts Institute of Technology (MIT (News - Alert)), was appointed to its
Board of Directors. Mr. Ruiz also serves as Audit Committee Chair. As
MIT's Chief Financial Officer, a Trustee of the MIT Corporation and a
member of its Executive Committee, Mr. Ruiz is the chief steward of
over $17 billion of financial assets, $3.4 billion in operating
revenues and is responsible for administering MIT's $5 billion capital
plan through 2030. In addition, Mr. Ruiz currently serves as Audit
Committee Chair on the Board of Directors of Fortive (NYSE:
FTV), a diversified industrial growth company with 24,000 global
employees and more than $6 billion in annual revenue. In 2015, Fortive
was spun out from Danaher (NYSE:
DHR), a Fortune 150 company and global science and technology
innovator.
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Moncef Slaoui, Ph.D., appointed to Board of Directors: In July,
Moncef Slaoui was appointed
to Moderna's Board of Directors. While at GlaxoSmithKline PLC (GSK),
Dr. Slaoui led that company's global pharmaceutical R&D activities
from 2006 through to 2015, most recently serving as its Chairman of
Global Vaccines. Dr. Slaoui also served on GSK's Board of Directors
for 11 years. In conjunction with Dr. Slaoui's appointment to
Moderna's Board of Directors, Lee Babiss, Ph.D., stepped down from his
role as a Director.
Management Team Updates
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Melissa Moore, Ph.D., elected to National Academy of Sciences:
In May, Melissa J. Moore, Ph.D., Chief Scientific Officer of Moderna's
mRNA research platform, was elected
to the National Academy of Sciences (NAS). Established in 1863, the NAS
provides independent and objective advice to the US government and
other organizations on matters related to science and technology.
Election to membership in the NAS is widely considered one of the
highest honors a scientist can receive. The NAS membership totals
approximately 2,250 members and nearly 440 foreign associates, of whom
approximately 200 have received Nobel (News - Alert) prizes.
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Juan Andres named Senior Vice President of Late Stage Technical
Development and Manufacturing: In August, Moderna announced
that Juan Andres joined Moderna as Senior Vice President of Late Stage
Technical Development and Manufacturing. Mr. Andres previously served
as Global Head Technical Operations (Manufacturing and Supply) at
Novartis. Mr. Andres will be responsible for the scale-up of Moderna's
manufacturing, quality and operations efforts as it continues to
advance its growing pipeline of mRNA medicines and prepares to bring
its state-of-the-art Good Manufacturing Practice (GMP) mRNA clinical
manufacturing facility online in 2018. In addition, he will lead
efforts aimed at preparing Moderna for Phase 3 and commercial
capabilities and capacity. Steve Harbin, who previously served as
Senior Vice President of Manufacturing and Operations at Moderna, has
transitioned to new roles as Chief of Staff and Chief Sustainability
Officer.
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Saqib Islam, Chief Business Officer, and James Kasinger, General
Counsel and Secretary, depart Moderna: Mr. Islam and Mr. Kasinger
have decided to pursue other opportunities. Mr. Islam will be
departing the company in mid-September. Mr. Kasinger left Moderna
earlier this year. A search to identify successors is underway.
Partner Updates
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Moderna and Alexion terminate partnership: In July, Alexion announced
its plans to conclude its partnership contract with Moderna, one of
several actions undertaken by the company to reshape its R&D strategy.
The ten product options that were part of the strategic agreement the
two companies formed in 2014 were terminated, and the rights to
development treatments for those rare diseases reverted to Moderna.
While Alexion's previously announced mRNA program in Crigler-Najjar
syndrome type 1 (CN1) remains an area of research interest for
Moderna, the company's initial rare liver disease program in MMA is
the current priority for development.
Operational Updates
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R&D organizational update: Moderna has decided to move from
its venture-based R&D model to a therapeutic area R&D model.
Research/Preclinical Development will report to Moderna's President
Stephen Hoge, MD, and Clinical Development will report to Tal Zaks,
MD, Ph.D., Moderna's Chief Medical Officer. Research and development
teams will be aligned around three core matrixed therapeutic areas -
infectious diseases, immuno-oncology and rare liver diseases. As
Moderna advances parallel opportunities for mRNA vaccines and
therapeutics into and through the clinic, a therapeutic area R&D model
will enable Moderna to more effectively share clinical learnings
across therapeutic areas. In addition, the continued evolution of
Moderna's platform coupled with the rapid cycle time in drug discovery
will allow tighter integration between therapeutic area discovery
teams and the platform research teams.
Moderna is not
eliminating any headcount as part of the organizational update.
Moderna's venture names and branding (Valera, Elpidera, Onkaido and
Caperna) are being dissolved, with the entire organization moving
forward under the corporate "Moderna" entity and brand.
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Continued progress on Norwood GMP manufacturing facility build-out:
To support and manage the breadth of simultaneous clinical studies
Moderna anticipates in the coming years, the company is building
a 200,000 square foot GMP mRNA clinical manufacturing facility in
Norwood, Mass. The build-out continues to progress according to plan,
and Moderna anticipates beginning operations at the Norwood facility
in mid-2018.
The fully integrated facility will enable the
manufacture, quality, control and supply of clinical grade mRNA
therapies and vaccines for GLP toxicology studies as well as Phase 1
and Phase 2 clinical studies. Moderna will carry out all manufacturing
activities at the site-from raw material production to APIs,
formulation, filling and finish.
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Continued growth across organization: Since January 2017,
Moderna added approximately 50 new team members and now has more than
550 team members.
FINANCIALS AS OF JUNE 30, 2017
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Maintained strong cash position: Moderna has maintained a
strong cash position in 2017. As of June 30, 2017, the company had
$1.098 billion in cash, as compared to $1.307 billion in cash as of
December 31, 2016. This affords Moderna several years of runway to
support its continued growth and pipeline acceleration.
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Continued significant investments in the business: Moderna's
gross investment in the business in the first half of 2017 totaled
$212 million in operating expense and capital expenditures. Net of
reimbursements and product milestones, $183 million of cash was used
for operating expense and capital expenditures.
About Moderna Therapeutics
Moderna is a clinical stage pioneer of messenger RNA (mRNA) therapeutics
and vaccines, an entirely new drug technology that directs the body's
cells to produce intracellular or secreted proteins. With its
breakthrough platform, Moderna is developing mRNA vaccines and
therapeutics as a new class of medicines for a wide range of diseases
and conditions, in many cases by addressing currently undruggable
targets. Moderna is developing its innovative mRNA medicines for
infectious diseases, cancer (immuno-oncology), rare liver diseases,
cardiovascular diseases and pulmonary diseases, through proprietary
development and collaborations with strategic partners.
Headquartered in Cambridge, Mass., privately held Moderna currently has
strategic agreements with AstraZeneca, Merck and Vertex Pharmaceuticals,
as well as the Defense Advanced Research Projects Agency (DARPA), an
agency of the U.S. Department of Defense; the Biomedical Advanced
Research and Development Authority (BARDA), a division of the Office of
the Assistant Secretary for Preparedness and Response (ASPR) within the
U.S. Department of Health and Human Services (HHS); and the Bill &
Melinda Gates Foundation. To learn more, visit www.modernatx.com.
View source version on businesswire.com: http://www.businesswire.com/news/home/20170914005352/en/
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