Moderna Announces Recent Progress in Its Immuno-Oncology and Rare Disease Programs and Highlights Corporate Objectives
January 08 2019 - 8:30AM
Business Wire
Immuno-Oncology
- IND amendment submitted to the FDA for
a Phase 2 cohort of OX40L (mRNA-2416) to treat advanced ovarian
carcinoma
- First patient dosed in the Phase 1
study of OX40L + IL23 + IL36γ (Triplet) (mRNA-2752) for advanced or
metastatic solid tumor malignancies or lymphoma
- Planning for a Phase 2 study of
personalized cancer vaccine (mRNA-4157) initiated together with
strategic collaborator Merck
Rare Diseases
- IND submitted to the FDA for a Phase
1/2 study of methylmalonic acidemia (mRNA-3704)
Moderna, Inc. (Nasdaq: MRNA), a clinical stage biotechnology
company pioneering messenger RNA (mRNA) therapeutics and vaccines
to create a new generation of transformative medicines for
patients, today announced recent updates to several of its
immuno-oncology and rare disease programs and outlined its
2019-2020 corporate objectives. Moderna has 21 mRNA development
candidates in its pipeline, with 11 programs now in clinical
development.
“This year we are focused on making significant advances to our
pipeline as we work to bring multiple programs into Phase 2
clinical trials, move programs within our rare disease portfolio
toward the clinic and leverage our mRNA platform to create both new
development candidates and potential new modalities where we
believe there is an opportunity to develop therapies for a broad
range of diseases,” said Stéphane Bancel, Moderna’s Chief Executive
Officer. “I am pleased with the continued progress of our pipeline,
our ability to now manufacture mRNA for clinical development at our
new site in Norwood and the relentless work of our team. I believe
we are better positioned than we have ever been to deliver on the
promise of our science to bring forward mRNA medicines that have
the potential to improve the lives of patients.”
Mr. Bancel will present a company overview and strategy update
today at 10:30am PST at the 37th Annual J.P. Morgan Healthcare
Conference in San Francisco. A live webcast of today’s presentation
can be found at investors.modernatx.com.
Detailed program updates:
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
previously reported clinical observations in two patients with
advanced ovarian carcinoma in its Phase 1 study, Moderna has
submitted an Investigational New Drug (IND) amendment to the U.S.
Food and Drug Administration (FDA) and to the study’s clinical
research sites to commence a Phase 2 cohort of mRNA-2416 as a
monotherapy in advanced ovarian carcinoma within its current Phase
1 study. Thus far, 28 patients have been dosed in the ongoing Phase
1 trial for mRNA-2416, an open-label, multicenter study of repeated
intratumoral injections of mRNA-2416 in patients with advanced
relapsed/refractory solid tumor malignancies and lymphomas.
Initial data from the Phase 1 study were presented in a poster
session at the Annual Meeting of the Society for Immunotherapy
of Cancer in November 2018.
- OX40L + IL23 + IL36γ (Triplet)
(mRNA-2752): Moderna has dosed the first patient in the
Phase 1 study of mRNA-2752, an intratumoral injection comprising
three mRNAs encoding for OX40L + IL23 + IL36γ for the treatment of
advanced or metastatic solid tumor malignancies or lymphoma. The
open label, multi-center study is evaluating the safety and
tolerability of mRNA-2752 as a single agent and in combination
either with AstraZeneca’s durvalumab or tremelimumab, and will
assess anti-tumor activity, protein expression in tumors and
pharmacokinetics, and exploratory endpoints that include assessment
of immunological response.
Cancer Vaccines: These programs focus on stimulating a
patient’s immune system to tumor-related antigens to enable the
immune system to elicit a more effective antitumor response.
- Personalized Cancer Vaccine (PCV)
(mRNA-4157): Moderna and Merck are planning a randomized Phase
2 study comparing PCV and KEYTRUDA® against KEYTRUDA alone. To
date, interim Phase 1 PCV study data from 24 patients showed no
dose limiting toxicities up to 0.39 mg (the third of four dose
levels). Interim Phase 1 immunogenicity data have also been
collected in certain patients dosed with mRNA-4157 as a
monotherapy, and potential antigen-specific T cell responses have
been detected. The Phase 1 study continues in the dose-escalation
phase of the protocol.
- KRAS vaccine (mRNA-5671): Merck
will lead an open-label, multi-center, dose-escalation and
dose-expansion Phase 1 study to evaluate the safety and
tolerability of mRNA-5671 administered as an intramuscular
injection both as a monotherapy and in combination with KEYTRUDA.
KRAS is a frequently mutated oncogene in epithelial cancers,
primarily in non-small cell lung, colorectal and pancreatic
cancers. The IND for a KRAS vaccine was originally submitted by
Moderna and included an mRNA for the membrane protein STimulator of
INterferon Gene (STING) to help promote antitumor activity. That
IND was transferred to Merck which now will move the program
forward under the same IND with KRAS as the sole mRNA. Merck may
choose to include STING mRNA in later clinical development of the
KRAS vaccine.
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): An IND application has been submitted to the FDA
for mRNA-3704, Moderna’s development candidate for MMA. If
approved, this will be Moderna’s first rare disease program to
advance into clinical trials. The Company plans to conduct an
open-label, multi-center, dose escalation Phase 1/2 study of
multiple ascending doses of mRNA-3704 in pediatric patients with
isolated MMA due to MUT enzyme deficiency. The objectives of the
study are to evaluate safety and tolerability. mRNA-3704 has
received Rare Pediatric Disease Designation by the FDA and Orphan
Drug Designation by both the FDA and the European Medicines
Agency.
- Propionic Acidemia (PA)
(mRNA-3927): mRNA-3927 was granted Orphan Drug Designation by
the FDA in December 2018 and Rare Pediatric Disease Designation by
the FDA in January 2019. PA is a rare, life-threatening, inherited
metabolic disorder due to a defect in the mitochondrial enzyme
propionyl-CoA carboxylase, or PCC. It primarily affects the
pediatric population and there is no approved therapy. Moderna is
continuing to advance mRNA-3927 in pre-clinical studies. Moderna
also continues to enroll patients in a global natural history study
of MMA and PA (MaP Study) designed to identify and correlate
clinical and biomarker endpoints for these disorders. 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.
More than 760 subjects have been dosed with a therapeutic or
vaccine candidate developed with Moderna’s mRNA technology.
Information about each program in Moderna’s pipeline, including
those discussed in this press release, can be found on our investor
relations page of our website www.modernatx.com.
Corporate Objectives:
Moderna shared its corporate objectives for 2019 - 2020,
which include:
1. Generate human proof-of-concept data for
multiple medicines2. Execute on current development pipeline3.
Create new development candidates in existing modalities4. Invent
new modalities
Corporate Updates:
Continued growth across organization: Moderna ended 2018
with approximately 735 full time employees. The Company ended 2017
with 535 employees.
Continued strong cash position: We expect our cash, cash
equivalents, and investments in marketable securities as of
December 31, 2018 to be approximately $1.7 billion (unaudited), as
compared to $902 million (audited) as of December 31, 2017. The
year over year increase includes approximately $564 million
(unaudited) in net proceeds from our initial public offering
completed in December 2018, approximately $661 million in net
proceeds from our preferred stock issuances in 2018, and a $13
million (unaudited) premium associated with the 2018 amended and
restated personalized cancer vaccines agreement with Merck &
Co.
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. Moderna’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, and
Merck & Co., 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). 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.
Special Note Regarding 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: Moderna’s 2019-2020 corporate objectives;
Moderna’s plans to move multiple programs into Phase 2 clinical
trials and programs within its rare disease portfolio toward the
clinic; Moderna’s plans to create new development candidates;
Moderna’s plans to develop therapies in new modalities to treat a
broad range of diseases; the potential of intratumoral
immuno-oncology to drive anti-cancer T cell responses with mRNA
medicines; the commencement of a Phase 2 cohort for mRNA-2416; the
potential for Systemic Intracellular Therapeutic programs to
deliver mRNA into cells within target organs as a therapeutic
approach; the advancement of mRNA-3704 into clinical trials; and
Moderna’s expectations regarding its cash, cash equivalents, and
investments in marketable securities as of December 31, 2018. 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 category 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 category 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 category of medicines;
and those described in Moderna’s Prospectus filed with the U.S.
Securities and Exchange Commission (SEC) on December 7, 2018
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 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20190108005178/en/
Media:Jason GlashowHead of Corporate
Communications617-674-5648jason.glashow@modernatx.comInvestors:Lorence
KimChief Financial Officer617-209-5849lorence.kim@modernatx.com
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