-Collaboration to explore use of mRNA Therapeutics to treat the
underlying cause of CF by enabling cells to produce functional CFTR
proteins in the lungs-
-Moderna to receive $40 million upfront, made up of a $20 million
cash payment and a $20 million convertible note investment, with
potential for up to additional $275 million in milestones plus royalty
payments-
BOSTON & CAMBRIDGE, Mass.--(BUSINESS WIRE)--
Vertex
Pharmaceuticals Incorporated (Nasdaq: VRTX) and Moderna
Therapeutics today announced that the two companies have entered
into an exclusive research collaboration and licensing agreement aimed
at the discovery and development of messenger Ribonucleic Acid (mRNA)
Therapeutics™ for the treatment of cystic fibrosis (CF). The three-year
collaboration will focus on the use of mRNA therapies to treat the
underlying cause of CF by enabling cells in the lungs to produce
functional copies of the cystic fibrosis transmembrane conductance
regulator (CFTR) protein, which is known to be defective in people with
CF. Through the collaboration, the companies will explore the potential
utilization of pulmonary mRNA delivery.
"We are excited to begin this collaboration with Moderna to further
broaden our pipeline and support our goal of treating the underlying
cause of CF for all people with this disease. The collaboration with
Moderna underscores our commitment to invest in and pioneer multiple
innovative approaches to expand and enhance the future treatment of CF,"
said David Altshuler, M.D., Ph.D., Vertex's Executive Vice President,
Global Research and Chief Scientific Officer. "Moderna's messenger RNA
Therapeutics represent a promising new approach that could provide
functional CFTR proteins to the lungs of people with CF, which would
complement our existing approach of using CFTR modulators to treat CF."
"Vertex's deep scientific expertise and commitment to creating the best
treatment regimens for cystic fibrosis patients make them an ideal
partner to advance mRNA Therapeutics for this disease," said Stéphane
Bancel, Chief Executive Officer of Moderna. "This collaboration is
illustrative of our consistent strategy to partner with world class
therapeutic experts in order to maximize the potential of leveraging
mRNA as a novel therapeutic approach across a spectrum of serious
diseases. We're particularly excited to explore the potential of
pulmonary delivery as a new modality to bring transformative mRNA
medicines to patients and address unmet needs."
Under the terms of the collaboration, Vertex and Moderna will conduct
exclusive research, development and commercialization activities to
advance mRNA Therapeutics that aim to enable cells of people with CF to
produce functional CFTR proteins in the lungs. Moderna will lead
discovery efforts, leveraging its leading mRNA platform technology and
mRNA delivery expertise along with Vertex's scientific experience in CF
biology and the functional understanding of CFTR, as well as the
company's proprietary assay platform that utilizes human bronchial
epithelial (HBE) cells of multiple different CF gene mutations from
people with CF. Vertex will lead all preclinical, development and
commercialization activities associated with the advancement of mRNA
Therapeutics that result from this collaboration and will fund all
expenses related to the collaboration.
Vertex will pay Moderna$20 million in cash as part of its upfront
commitment to the collaboration. Vertex will also make a $20 million
investment in Moderna in the form of a convertible note that will
convert to equity. The investment will provide Vertex with an ownership
stake in Moderna. Vertex will also pay Moderna future development and
regulatory milestones of up to $275 million, including $220 million in
approval and reimbursement milestones, as well as tiered royalty
payments on future sales.
About Moderna's mRNA Therapeutics™
mRNA is responsible for carrying genetic instructions transcribed from
DNA, which cells then translate to produce proteins that, when defective
or missing, can underlie certain diseases, including CF. Moderna's
pioneering mRNA Therapeutics™ are designed to trigger the cellular
machinery to utilize their natural processes to produce specific
functional proteins. Through this collaboration, mRNA Therapeutics may
be developed to enable the delivery of correct genetic instructions into
cells in the lungs, which may trigger the cells to produce functional
CTFR protein. This mRNA-based approach could be applicable to any person
with CF regardless of a person's specific CFTR mutations. Moderna
currently has two Phase 1 clinical studies underway of mRNA vaccines for
the prevention of infectious diseases. The company has over 90 discovery
programs advancing across its ecosystem of therapeutically focused
ventures and external partners that span rare diseases, infectious
diseases, cancer and cardiovascular disease, among others.
Vertex's CF Research and Development Pipeline
Vertex's strategy in CF is to increase the number of people eligible for
treatment with its two currently approved CF medicines and to develop
new medicines that have the potential to treat all people with the
disease in the future. Vertex's two approved CF medicines, known as CFTR
modulators, are designed to treat the defective CFTR protein in people
with specific mutations in the CF gene. Beyond its two approved
medicines, Vertex is also advancing multiple other potential medicines
for the treatment of CF, including additional CFTR modulators as well as
other collaborative approaches, including sodium channel inhibition,
gene editing and, as announced today, mRNA-based therapies. The most
advanced approach is the development of an additional two-drug
combination of CFTR modulators, which is currently being evaluated in a
broad Phase 3 development program. The company plans to advance a
three-drug combination of CFTR modulators into Phase 2 development in
people with CF in the second half of 2016.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-shortening genetic disease affecting
approximately 75,000 people in North America, Europe and Australia.
Today, the median predicted age of survival for a person with CF is
between 34 and 47 years, but the median age of death remains in the
mid-20s.
CF is caused by a defective or missing CFTR protein resulting from
mutations in the CFTR gene. Children must inherit two
defective CFTR genes — one from each parent — to have CF. There are more
than 1,900 known mutations in the CFTR gene. Some of these mutations,
which can be determined by a genetic, or genotyping test, lead to CF by
creating non-working or too few CFTR protein at the cell surface. The
defective function or absence of CFTR proteins in people with CF results
in poor flow of salt and water into and out of the cell in a number of
organs, including the lungs. This leads to the buildup of abnormally
thick, sticky mucus that can cause chronic lung infections and
progressive lung damage.
About Vertex
Vertex is a global biotechnology company that aims to discover, develop
and commercialize innovative medicines so people with serious diseases
can lead better lives. In addition to our clinical development programs
focused on cystic fibrosis, Vertex has more than a dozen ongoing
research programs aimed at other serious and life-threatening diseases.
Founded in 1989 in Cambridge, Mass., Vertex today has research and
development sites and commercial offices in the United States, Europe,
Canada and Australia. For six years in a row, Science magazine has named
Vertex one of its Top Employers in the life sciences. For additional
information and the latest updates from the company, please visit www.vrtx.com.
About Moderna Therapeutics
Moderna is a clinical stage pioneer of messenger RNA (mRNA)
Therapeutics™, an entirely new in vivo drug technology that
produces human proteins, antibodies and entirely novel protein
constructs inside patient cells, which are in turn secreted or active
intracellularly. This breakthrough platform addresses currently
undruggable targets and offers a potentially superior alternative to
existing drug modalities for a wide range of diseases and conditions.
Moderna is developing and plans to commercialize its innovative mRNA
drugs through its own ventures and its strategic relationships with
established pharmaceutical and biotech companies. Its current ventures
are: Onkaido, focused on oncology, Valera, focused on infectious
diseases, Elpidera, focused on rare diseases, and Caperna, focused on
personalized cancer vaccines. Cambridge-based Moderna is privately held
and currently has strategic agreements with AstraZeneca, Alexion
Pharmaceuticals, Merck and Vertex Pharmaceuticals. To learn more, visit www.modernatx.com.
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as defined in the
Private Securities Litigation Reform Act of 1995, including, without
limitation, Dr. Altshuler's statements in the second paragraph of the
press release, Mr. Bancel's statements in the third paragraph of the
press release and the information provided regarding the future
development of mRNA therapies to treat the underlying cause of CF. While
Vertex believes the forward-looking statements contained in this press
release are accurate, these forward-looking statements represent the
company's beliefs only as of the date of this press release and there
are a number of factors that could cause actual events or results to
differ materially from those indicated by such forward-looking
statements. Those risks and uncertainties include, among other things,
that data may not support further development of the mRNA therapies
subject to the collaboration due to safety, efficacy or other reasons,
and other risks listed under Risk Factors in Vertex's annual report and
quarterly reports filed with the Securities and Exchange Commission and
available through the company's website at www.vrtx.com.
Vertex disclaims any obligation to update the information contained in
this press release as new information becomes available.
(VRTX-GEN)
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Vertex Pharmaceuticals Incorporated
Investors:
Michael
Partridge, 617-341-6108
or
Eric Rojas, 617-961-7205
or
Zach
Barber, 617-341-6992
or
Media:
US: 617-341-6992
Europe
& Australia: +44 20 3204 5275
mediainfo@vrtx.com
or
Moderna
Therapeutics
Investors:
Maren Winnick,
617-674-5297
maren.winnick@modernatx.com
or
Media:
Liz
Melone, 617-256-6622
liz.melone@modernatx.com
Source: Vertex Pharmaceuticals Incorporated
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