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Results from Phase 3 Study of Ivacaftor Monotherapy Showed Statistically Significant Improvements in Lung Function in People



  Results from Phase 3 Study of Ivacaftor Monotherapy Showed Statistically
  Significant Improvements in Lung Function in People with non-G551D Gating
  Mutations

   -Mean absolute treatment difference in lung function (percent predicted
  FEV[1]) between treatment with ivacaftor and placebo was 10.7% (p<0.0001);
   mean relative treatment difference between treatment with ivacaftor and
                        placebo was 14.2% (p<0.0001)-

-Statistically significant improvements in weight gain and in patient-reported
 quality of life as measured by the respiratory domain of the Cystic Fibrosis
                        Questionnaire Revised (CFQ-R)-

 -Supplemental New Drug Application (sNDA) in the United States and Marketing
Authorization Application (MAA) variation in Europe planned for second half of
                          2013 for gating mutations-

Business Wire

CAMBRIDGE, Mass. -- July 29, 2013

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the first
clinical data from one of the company’s ongoing Phase 3 label-expansion
studies for ivacaftor monotherapy in people with cystic fibrosis (CF). In the
Phase 3 study evaluating ivacaftor monotherapy in people ages six and older
with at least one non-G551D gating mutation, the mean absolute treatment
difference in percent predicted FEV[1] between treatment with ivacaftor and
placebo was 10.7% (p<0.0001) and the mean relative treatment difference in
percent predicted FEV[1] was 14.2% (p<0.0001) through the 8-week treatment
period. KALYDECO (ivacaftor) is currently approved for people with cystic
fibrosis (CF) ages 6 and older who have at least one copy of the G551D
mutation in the cystic fibrosis transmembrane conductance regulator (CFTR)
gene. Worldwide, approximately 2,000 people with CF ages six and older have at
least one copy of the G551D mutation, and approximately 400 people with CF
ages six and older have at least one non-G551D gating mutation. The study in
gating mutations is one of three ongoing Phase 3 label-expansion studies for
ivacaftor designed to evaluate whether additional people with CF may benefit
from treatment with ivacaftor.

Based on these data, Vertex plans to submit a supplemental New Drug
Application (sNDA) in the United States and a Marketing Authorization
Application (MAA) variation in Europe in the second half of 2013 for the use
of ivacaftor monotherapy in people with CF ages 6 and older who have at least
one non-G551D CFTR gating mutation.

“Our goal in CF is to help as many people as possible with our medicines, and
these data are an important step toward that goal,” said Robert Kauffman,
M.D., Ph.D., Senior Vice President and Chief Medical Officer at Vertex. “The
data announced today in people with CF who have a gating mutation showed a
meaningful improvement in lung function and support our plans to seek approval
of ivacaftor for these patients later this year.”

About the Study

The Phase 3 crossover study of ivacaftor enrolled 39 people with CF ages 6 and
older who have at least one non-G551D gating mutation. Patients received
either ivacaftor, or placebo, for eight weeks, followed by a 4-week washout
period. Following the washout period, patients who received placebo in the
first eight weeks received ivacaftor for weeks 12 to 20, and patients who
received ivacaftor for the first eight weeks received placebo for weeks 12 to
20. The primary analysis was conducted at week 20 of the study, and the
primary endpoint of the study was absolute change from baseline in percent
predicted FEV[1]. Following the end of the crossover periods, all patients
receive ivacaftor from week 20 to week 36 as part of an open-label dosing
period.

In this study, the mean absolute treatment difference in percent predicted
FEV[1] between treatment with ivacaftor and placebo was 10.7% (p<0.0001) and
the mean relative treatment difference in percent predicted FEV[1] was 14.2%
(p<0.0001) through the 8-week treatment period. The study met its primary
endpoint. The mean absolute and relative percent predicted FEV[1] improvements
during ivacaftor treatment (within-group) were 7.5% (p<0.0001) and 10.8%
(p<0.0001), respectively. Additionally, treatment with ivacaftor in this study
resulted in statistically significant improvements in weight gain and in
improvements in patient-reported quality of life as measured by the
respiratory domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R). Full
data from this study will be submitted for presentation at a medical meeting
in the second half of 2013.

The safety and tolerability results observed in this study were consistent
with those observed in prior Phase 3 studies of ivacaftor monotherapy in
people with CF who have the G551D mutation. The most commonly observed adverse
events, regardless of treatment assignment, included pulmonary exacerbation,
cough, headache and abdominal pain, each occurring more frequently while
patients received placebo than when patients received ivacaftor.

Two additional Phase 3 label-expansion studies are ongoing for ivacaftor
monotherapy, including a study in people with CF ages 6 and older who have at
least one copy of the R117H mutation and a study in children with CF ages 2 to
5 who have a gating mutation, including the G551D mutation. A Phase 2
proof-of-concept study evaluating ivacaftor in people with CF who have
clinical evidence of residual CFTR function is also ongoing.

Indication and Important Safety Information for KALYDECO^TM (ivacaftor)

Ivacaftor (150mg tablets) is indicated for the treatment of cystic fibrosis
(CF) in patients age 6 years and older who have a G551D mutation in the CFTR
gene.

Ivacaftor is not for use in people with CF due to other mutations in
the CFTR gene. It is not effective in CF patients with two copies of the
F508del mutation (F508del/F508del) in the CFTR gene. The efficacy and safety
of ivacaftor in children younger than 6 years of age have not been evaluated.

High liver enzymes (transaminases, ALT and AST) have been reported in patients
receiving ivacaftor. It is recommended that ALT and AST be assessed prior to
initiating ivacaftor, every 3 months during the first year of treatment, and
annually thereafter. Patients who develop increased transaminase levels should
be closely monitored until the abnormalities resolve. Dosing should be
interrupted in patients with ALT or AST of greater than 5 times the upper
limit of normal. Following resolution of transaminase elevations, consider the
benefits and risks of resuming ivacaftor dosing. Moderate transaminase
elevations are common in subjects with CF. Overall, the incidence and clinical
features of transaminase elevations in clinical trials was similar between
subjects in the ivacaftor and placebo treatment groups. In the subset of
patients with a medical history of elevated transaminases, increased ALT or
AST have been reported more frequently in patients receiving ivacaftor
compared to placebo.

Use of ivacaftor with medicines that are strong CYP3A inducers such as the
antibiotics rifampin and rifabutin; seizure medications (phenobarbital,
carbamazepine, or phenytoin); and the herbal supplement St. John's Wort
substantially decreases exposure of ivacaftor, which may diminish
effectiveness. Therefore, co-administration is not recommended.

The dose of ivacaftor must be adjusted when concomitantly used with potent and
moderate CYP3A inhibitors. The dose of ivacaftor must be adjusted when used in
patients with moderate or severe hepatic disease.

Ivacaftor can cause serious adverse reactions including abdominal pain and
high liver enzymes in the blood. The most common side effects associated with
ivacaftor include headache; upper respiratory tract infection (the common
cold), including sore throat, nasal or sinus congestion, and runny nose;
stomach (abdominal) pain; diarrhea; rash; and dizziness. These are not all the
possible side effects of ivacaftor. A list of the adverse reactions can be
found in the full product labeling for each country where ivacaftor is
approved. Patients should tell their healthcare providers about any side
effect that bothers them or doesn't go away.

Please see full U.S. Prescribing Information for KALYDECO at www.KALYDECO.com,
the EU Summary of Product Characteristics for KALYDECO at http://goo.gl/N3Tz4,
and the KALYDECO Canadian Product Monograph at www.vrtx.ca.

About Vertex

Vertex creates new possibilities in medicine. Our team discovers, develops and
commercializes innovative therapies so people with serious diseases can lead
better lives.

Vertex scientists and our collaborators are working on new medicines to cure
or significantly advance the treatment of hepatitis C, cystic fibrosis,
rheumatoid arthritis and other life-threatening diseases.

Founded more than 20 years ago in Cambridge, Mass., we now have ongoing
worldwide research programs and sites in the U.S., U.K. and Canada. Today,
Vertex has more than 2,000 employees around the world, and for three years in
a row, Science magazine has named Vertex one of its Top Employers in the life
sciences.

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. Kauffman’s statements in the third paragraph of this press
release and statements regarding (i) Vertex’s plan to submit an sNDA in the
United States and an MAA variation in Europe in the second half of 2013 and
(ii) Vertex’s plan to submit full data from this study for presentation at a
medical meeting in the second half of 2013. While Vertex believes the
forward-looking statements contained in this press release are accurate, 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 Vertex could
experience unforeseen delays in submitting regulatory filings, 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)

Contact:

Vertex Pharmaceuticals Incorporated
Investors:
Michael Partridge, 617-341-6108
or
Kelly Lewis, 617-961-7530
or
Media:
Zach Barber, 617-341-6470
mediainfo@vrtx.com
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