-ENaC inhibition aims to restore or improve
hydration of cell surfaces in the lungs to improve lung
function-
-Parion to receive $80 million up-front payment
with potential for additional development and regulatory milestones
and royalty payments-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Parion
Sciences today announced that the companies will collaborate to
develop investigational epithelial sodium channel (ENaC) inhibitors
for the potential treatment of cystic fibrosis (CF) and other
pulmonary diseases. Under the agreement, Vertex gains worldwide
development and commercial rights to Parion’s investigational ENaC
inhibitors, including P-1037 and P-1055, for CF and other pulmonary
diseases. P-1037 is currently being evaluated in an exploratory
Phase 2a study in people with CF, regardless of genotype. Vertex
and Parion plan to begin an additional Phase 2a study that adds
P-1037 to treatment with the investigational combination of
lumacaftor and ivacaftor for people with CF who have two copies of
the F508del mutation. Parion will receive an $80 million up-front
payment from Vertex with the potential to receive additional
development and regulatory milestone payments and tiered royalties
related to P-1037 and P-1055 in CF and other pulmonary
diseases.
“This collaboration with Parion complements our ongoing work in
CF and supports our two key goals in this disease – to increase the
number of people eligible for new CF medicines and to enhance the
benefit of treatment,” said Jeffrey Chodakewitz, M.D., Executive
Vice President and Chief Medical Officer at Vertex. “The goal of
these planned studies of P-1037 is to determine whether ENaC
inhibition can improve lung function in people with CF, including
those with mutations unlikely to respond to treatment with the
investigational combination of lumacaftor and ivacaftor. Beyond CF,
this agreement helps to diversify our pipeline by providing
opportunities to evaluate P-1037 as part of Phase 2a studies in
multiple other diseases that impact the lungs.”
“ENaC inhibition represents a promising opportunity to
potentially enhance the benefit of existing treatments for people
with CF, and we have worked diligently to bring P-1037 from our
research labs and into Phase 2 development,” said Paul Boucher,
President and Chief Executive Officer of Parion. “Vertex is the
leader in developing new medicines that treat the underlying cause
of CF. We are pleased to enter into this collaboration to unify the
scientific expertise of both companies to advance P-1037 in CF and
other pulmonary diseases.”
Cystic fibrosis is a rare genetic disease that is caused by
defective or missing cystic fibrosis transmembrane conductance
regulatory (CFTR) proteins resulting from mutations in
the CFTR gene. The defective or missing CFTR proteins
result in poor flow of salt and water into and out of the cell in a
number of organs, including the lungs. The defective CFTR protein
that causes CF is also believed to increase the function of ENaC,
which may contribute to dehydration of the cell surface of lungs in
people with CF. In CF, the poor flow of salt and water in cells
prevents cilia on the surface of the cell from beating properly,
which leads to a buildup of abnormally thick, sticky mucus that can
cause chronic lung infections and progressive lung damage,
eventually leading to death.
About the Collaboration
Under the terms of the collaboration, Vertex obtained worldwide
development and commercial rights to Parion’s lead investigational
ENaC inhibitors, including P-1037 and P-1055, for the potential
treatment of CF and all other pulmonary diseases. Parion received
an $80 million up-front payment and has the potential to receive up
to an additional $490 million in development and regulatory
milestone payments for development of ENaC inhibitors in CF,
including $360 million related to global filing and approval
milestones. Parion has the potential to receive up to $370 million
in additional development and regulatory milestones for P-1037
and P-1055 in non-CF pulmonary indications. Parion may also receive
an additional $230 million in development and regulatory
milestones should Vertex elect to develop an additional ENaC
inhibitor from Parion’s research program. Parion will receive
tiered royalties on potential sales of P-1037 and P-1055 in CF and
other pulmonary diseases that range from the low double digits to
mid-teens as a percentage of sales. Vertex will lead future
development activities for P-1037 and P-1055 in CF and other
pulmonary diseases.
Parion recently initiated an exploratory Phase 2a study of
inhaled P-1037 in approximately 120 people with CF. The study is
enrolling people with a confirmed diagnosis of CF and any CFTR
mutation, including those who have mutations not expected to
respond to ivacaftor alone. The study is evaluating the use of
P-1037, with and without hypertonic saline, compared to placebo.
Patients in the study will continue to receive standard CF
medicines.
Preclinical evaluation in human bronchial epithelial cells from
people with CF who have two copies of the F508del mutation showed
that the addition of investigational P-1037 to the investigational
combination of lumacaftor and ivacaftor resulted in an additional
increase in both airway surface liquid and cilia beat frequency
compared to baseline and to the use of P-1037 or
lumacaftor/ivacaftor alone. Improvements in airway surface liquid
height and cilia beat frequency are direct measures of increased
hydration of the cell surface. This in vitro observation suggests
that the addition of P-1037 to the investigational combination of
lumacaftor and ivacaftor could enable enhanced function of the
cell’s cilia to clear mucus from the cell surface, potentially
resulting in improved lung function. Based on these preclinical
results, Vertex is preparing to conduct a Phase 2a study to
evaluate whether the addition of P-1037 to the combination of
lumacaftor and ivacaftor in people with CF who have two copies of
the F508del mutation provides additional benefit as compared to the
combination of lumacaftor and ivacaftor alone. This Phase 2a study
is expected to begin in early 2016.
Beyond CF, Vertex and Parion plan to conduct additional Phase 2a
studies of P-1037 across multiple other pulmonary diseases where
the disease results in defective hydration of the cell surfaces in
the lung. These diseases include Chronic Obstructive Pulmonary
Disease (COPD), Non-CF Bronchiectasis (NCFB) and Primary Ciliary
Dyskinesia (PCD). Parion will conduct Phase 2a development in these
diseases and retains an option to participate in co-development and
co-commercialization activities related to one of these non-CF
pulmonary diseases.
Vertex continues to expect 2015 non-GAAP Research and
Development and Sales, General and Administrative expenses to be in
the range of $1.05 to $1.10 billion.
About Cystic Fibrosis
CF is a rare, life-threatening genetic disease affecting
approximately 75,000 people in North
America, Europe and Australia. 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 proteins at the cell surface.
The defective or missing CFTR protein 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. The defective CFTR protein that causes CF is also believed
to increase the function of ENaC, which may contribute to
dehydration of the cell surface of lungs in people with CF. 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.
About Parion Sciences
Parion Sciences is a development stage biopharmaceutical company
dedicated to research, development and commercialization of
treatments to improve and extend the lives of patients with innate
mucosal surface defense deficiencies of the lung or eye. Parion has
a diverse pipeline of pre- clinical and clinical candidates for the
treatment of these diseases via distinctive mechanisms of action
and approaches. Parion is at the forefront of ENaC development and
leverages its scientific expertise in epithelial biology to expand
the company’s platforms and novel chemical compounds into new
potential indications to treat mucosal defects. Parion has received
grant funding from the National Institutes of Health and continues
to have a long-standing and valued relationship with Cystic
Fibrosis Foundation Therapeutics, Inc. For more information, please
see our website at www.Parion.com.
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
five 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.
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. Chodakewitz’s statements in the
second paragraph of the press release, Mr. Boucher’s statements in
the third paragraph of the press release, information regarding
Vertex’s 2015 non-GAAP operating expenses and the information
provided regarding the development timeframe of P-1037. 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 the company's expectations regarding its
2015 non-GAAP operating expenses may be incorrect (including
because one or more of the company's assumptions underlying its
expense expectations may not be realized) or that data may not
support further development of the compounds 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,
617-341-6108orKelly Lewis, 617-961-7530orEric Rojas,
617-961-7205orMedia:Zach Barber,
617-341-6992mediainfo@vrtx.comorParion
SciencesPaul Boucher, 919-313-1195President &
CEO
Vertex Pharmaceuticals (NASDAQ:VRTX)
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