-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-
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
IncorporatedInvestors:Michael Partridge,
617-341-6108orEric Rojas, 617-961-7205orZach Barber,
617-341-6992orMedia:US: 617-341-6992Europe & Australia:
+44 20 3204 5275mediainfo@vrtx.comorModerna TherapeuticsInvestors:Maren
Winnick, 617-674-5297maren.winnick@modernatx.comorMedia:Liz
Melone, 617-256-6622liz.melone@modernatx.com
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