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Moderna Announces Presentation of Interim Data from Phase 1 Study of mRNA Personalized Cancer Vaccine at 2019 ASCO Annual Meeting


Data show the potential of using neoantigens identified from an
individual's tumor to elicit an immune response to cancer mutations

Tolerability and immunogenicity data support the randomized Phase 2
study of mRNA-4157 in combination with pembrolizumab

Conference call to be held on Monday, June 3 at 8:00 a.m. ET

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
interim data from an ongoing Phase 1 clinical study in patients with
both resected (adjuvant) and unresected (advanced) solid tumors. The
data showed that the Company's mRNA personalized cancer vaccine (PCV)
mRNA-4157, given alone or in combination with Merck's pembrolizumab
(KEYTRUDA®), was well-tolerated at all doses tested and
elicited neoantigen-specific T-cell responses. There were no
vaccine-related serious adverse events (SAEs) reported for the PCV when
administered to patients as a monotherapy or in combination with

Presented today at the 2019 American Society of Clinical Oncology (ASCO)
Annual Meeting, the study demonstrates the immunogenicity of Moderna's
mRNA platform for developing PCVs. In addition, clinical activity was
observed in some patients receiving mRNA-4157 in combination with
pembrolizumab. These safety, tolerability and immunogenicity data and
the initial clinical activity observed support Moderna's randomized
Phase 2 study investigating pembrolizumab in combination with a 1 mg
dose of mRNA-4157, compared to pembrolizumab alone, for the treatment of
high-risk adjuvant melanoma.

"We are encouraged by these interim data from our personalized cancer
vaccine program, which involves designing and manufacturing a unique
vaccine for each patient based on their specific tumor," said Tal Zaks,
M.D., Ph.D., chief medical officer at Moderna. "This study demonstrates
the ability of Moderna's mRNA personalized cancer vaccine to elicit
T-cells that are specific to the cancer mutations. We also observed
early signs of clinical activity of our personalized cancer vaccine in
combination with pembrolizumab, including in two patients previously
treated with checkpoint inhibitors. We look forward to building on these
learnings about tolerability and immunogenicity by assessing activity in
a randomized Phase 2 study for the treatment of adjuvant melanoma."

"For decades, the cancer community has been working on the concept of
developing medicines that can be personalized down to the individual
patient level," said Howard A. "Skip" Burris III, M.D., president,
clinical operations & chief medical officer at Sarah Cannon Research
Institute, and a principal investigator of the mRNA-4157 Phase 1 study.
"We know that cancer mutations are rarely shared between patients, so
it's encouraging to see individualized, personalized cancer vaccines
like mRNA-4157 eliciting immune responses. We're pleased to be a part of
a study that aims to advance the science of immunotherapy through mRNA
vaccines, and deliver a novel approach that is customized for each

About the Data

2523: A phase 1 multicenter study to assess the safety, tolerability and
immunogenicity of mRNA-4157 alone in patients with resected solid tumors
and in combination with pembrolizumab in patients with unresectable
solid tumors.

Presented by: Howard A. Burris, M.D., FACP, FASCO, Sarah Cannon Research
(Poster Session, Saturday, June 1, 8:00 a.m. - 11:00 a.m.
CT followed by a Poster Discussion at 1:15 p.m. - 2:45 p.m. CT)

The ASCO poster is now available on the "Events
and Presentations"
section of our website.

In this dose-escalation study, 13 patients with resected solid tumors
(melanoma, colon and lung cancers) received mRNA-4157 as adjuvant
monotherapy after resection of their primary tumor. An additional 20
patients with metastatic, unresected solid tumors (melanoma, bladder,
lung, colon, prostate, head and neck and endometrial cancers) received
at least one dose of mRNA-4157 in combination with pembrolizumab.


  • mRNA-4157 was well-tolerated at all dose levels studied with no
    dose-limiting toxicities or grade 3/4 adverse events (AEs) or SAEs
    reported when administered as a monotherapy or in combination with
    pembrolizumab. The most common grade 2 adverse events were fatigue,
    soreness at the injection site, colitis and myalgias.
  • A cohort of patients at the top dose level (1 mg) are undergoing
    apheresis and deeper characterization of immunogenicity responses.
    Data from one such patient was available at the data cutoff and showed
    neoantigen-specific CD8 T-cell responses were detected to 10 out of 18
    class I neoantigens after the 4th dose of the vaccine (compared to
    0/18 at baseline).
  • Clinical responses (one complete response + five partial responses) at
    doses ranging from 0.04-1.0 mg were observed in 6 out of 20 patients
    receiving at least one dose of mRNA-4157 in combination with
    pembrolizumab. The complete response occurred to pembrolizumab
    monotherapy before mRNA-4157 was administered. Of the five partial
    responses, two were seen in patients previously treated with a
    checkpoint inhibitor.
  • Of the 13 patients who received adjuvant mRNA-4157 monotherapy, all
    patients have completed a full course of vaccination per the study
    protocol. Eleven patients remained disease free up to 75 weeks on

Additionally, the National Cancer Institute (NCI) presented early data
today 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.

2643: A Phase 1/2 study to assess the immunogenicity and tolerability of
personalized mRNA vaccine mRNA-4650 encoding defined neoantigens
expressed by the autologous cancer.

Presented by: Gal Cafri, Ph.D., Postdoctoral Fellow, National Cancer
Institute Surgery Branch
(Poster Session, Saturday, June 1, 8:00
a.m. - 11:00 a.m. CT)

Conference Call

Moderna will host a conference call and webcast on Monday, June 3
at 8:00 a.m. ET to discuss these mRNA-4157 data. Participants are
invited to listen by dialing (866) 922-5184 (domestic) or (409) 937-8950
(international) and providing conference ID 3016438 or join the live
webcast by going to the "Events
and Presentations"
 area on the Investors page of the Company's
An archived webcast of the conference call can also be accessed through
the Company's website and a replay of the call will be available there
for four weeks after the call.

About Moderna's Immuno-Oncology Programs

Moderna's oncology programs are currently focused on two main areas:
cancer vaccines and intratumoral immuno-oncology (I/O) therapies.
Moderna is developing these potential mRNA treatments as monotherapies
and/or in combination with checkpoint inhibitors from our strategic
collaborators Merck and AstraZeneca. The company currently has five I/O
programs in development, including two programs advancing into Phase 2

An advantage of Moderna's mRNA platform is that it allows for
investigational medicines that combine in a single mRNA therapy several
different approaches to activate the immune system to attack cancer,
either with mRNA encoding for common tumor proteins found across cancer
types or multiple mRNAs encoding for various immunomodulatory proteins.

Moderna's investigational PCVs are designed to use neoantigens
identified from an individual's tumor to program the body's immune
system to elicit a more effective anti-tumor response. Upon sequencing
the tumor, Moderna's proprietary algorithms predict the neoantigens
(antigens encoded by tumor-specific mutated genes) most likely to
trigger the immune system to attack a particular cancer. Today, mRNA
encoding up to 34 unique neoantigens can be delivered in a single
vaccine. Moderna develops and manufactures these investigational PCVs at
its personalized vaccines unit within its Norwood, Mass. manufacturing

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 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
ranked in the top ten of Science's list of top biopharma industry
employers for the past four years. To learn more, visit
and follow on Twitter at @moderna_tx.

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: the
potential for Moderna's investigational PCVs to use neoantigens
identified from an individual's tumor to program the body's immune
system to elicit a more effective antitumor response; and the planned
Phase 2 study investigating pembrolizumab in combination with mRNA-4157,
compared to pembrolizumab alone, in high-risk adjuvant melanoma. 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 Phase 1 results for mRNA-4157 and mRNA-4650 will be
predictive of any future clinical studies; whether mRNA-4157 and
mRNA-4650 will be shown to be unsafe or intolerable during future
clinical studies; clinical development is lengthy and uncertain,
especially for a new class of medicines such as mRNA, and therefore our
clinical programs or development candidates may be delayed, terminated,
or may never advance; 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; 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
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.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a
subsidiary of Merck & Co., Inc.

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