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VX-787 Showed Significant Antiviral Activity and Reduced the Severity and Duration of Influenza Symptoms in Phase 2 Challenge

  VX-787 Showed Significant Antiviral Activity and Reduced the Severity and
  Duration of Influenza Symptoms in Phase 2 Challenge Study

- Treatment with highest dosing regimen of VX-787 reduced viral shedding by 94
percent versus placebo; Duration of flu symptoms were reduced by nearly half -

  - VX-787 is an investigational new class of medicine designed to directly
                 inhibit replication of the influenza virus -

Business Wire

CAMBRIDGE, Mass. -- March 4, 2013

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that
treatment with VX-787 in a Phase 2 influenza challenge study resulted in
statistically significant improvements in viral and clinical measurements of
infection. VX-787 is an investigational new class of medicine for the
treatment of influenza and is designed to directly inhibit replication of the
virus. The study met its primary endpoint, and showed a statistically
significant decrease in the amount of virus in nasal secretions (viral
shedding) over the seven-day study period. In addition, at the highest dosing
regimen evaluated in the study, there was a statistically significant
reduction in the severity and duration of influenza-like symptoms. People in
this dose group experienced influenza-like symptoms for a median of 1.9 days,
compared to 3.7 days in the placebo group. In addition, 93 percent of people
in this dose group showed no clinical symptoms of influenza after three days
of treatment, compared to 41 percent of people in the placebo group. In this
study, VX-787 was generally well-tolerated, with no adverse events leading to
discontinuation. Based on these data, Vertex will explore collaborative
opportunities to support further development of VX-787.

“There is an urgent need for new medicines targeting influenza that work more
quickly, address resistant and pandemic strains, and are effective when taken
more than two days after symptoms appear,” said Chris Wright, M.D., Ph.D.,
Vertex's Senior Vice President, Global Medicines Development and Medical
Affairs. “Further development of VX-787 may offer an opportunity to address
these needs. The data from this proof-of-concept study validate VX-787’s new
mechanism of action, and underscore its potential to significantly reduce the
severity and duration of influenza.”

About the Phase 2a Study

This double-blind, randomized, placebo-controlled Phase 2a study of VX-787
enrolled and dosed 104 healthy people (72 in the VX-787 arms; 32 in the
placebo arm) ages 18 to 45 who volunteered to be experimentally exposed to an
attenuated form of live H3N2 influenza A virus. H3N2 is a common type of
influenza virus, and was the most common type observed in the 2012/2013
influenza season in the United States. The study evaluated four dosing
regimens of VX-787 given once-daily (QD) for five days beginning 24 hours
after infection with flu virus. This was followed by an additional two days of
observation. The study was conducted in a controlled quarantine facility with
participants under close observation.

The primary objective of this study was to determine the effect of VX-787 on
levels of the influenza virus by measuring the total area under the curve of
viral titers (quantity of the virus shed in nasal secretions) over time. Viral
shedding is a validated marker for assessing antiviral agents in
proof-of-concept clinical studies in influenza. Higher total area under the
curve (AUC) indicates greater shedding of the influenza virus from the upper
respiratory tract, whereas lower total AUC indicates inhibition of, or
decreased, viral shedding.

Secondary objectives of the study included safety and tolerability, as well as
duration and severity of clinical symptoms. In the study, only people who
developed influenza infection after exposure, as measured by detectable virus
in nasal secretions or a pre-defined antibody response, were included in the
efficacy analysis, whereas all study participants were included in the safety
analysis. Of the 72 patients enrolled across the VX-787 treatment arms, 52
developed influenza infection. Of 32 patients enrolled in the placebo arm, 22
developed influenza infection.

Efficacy Data

Primary Endpoint: Across the VX-787 doses studied, a statistically significant
dose response in reduction in viral shedding AUC was observed (p=0.036). In
addition, over the seven days of observation, the 14 people in the highest
dose group showed a total median AUC of 0.4 log[10] virus compared to the 22
people who received placebo, who showed a total median AUC of 5.9 log[10]
virus, a difference that was statistically significant. These median AUC
values reflect a 94 percent reduction in the amount of virus shed during the
study for people treated with the highest dosing regimen of VX-787 compared to
placebo.

Secondary Endpoints: In addition to reductions in total viral AUC, treatment
with VX-787 in the highest dose group also resulted in statistically
significant improvements in multiple clinical measures of influenza. People in
this group experienced influenza-like symptoms for a median duration of 1.9
days, compared to 3.7 days for those who received placebo. In addition, there
was a statistically significant decrease in the severity of influenza-like
symptoms, as measured by AUC and peak severity of symptoms reported by
participants in the study. Participants graded seven influenza-like symptoms
on a scale of 0 to 3; this was summed to obtain a symptom severity score
(maximum symptom severity score of 21.)

                                                     
        Influenza-Like Symptoms           Placebo (n=22)   1,200 mg / 600
                                                               mg* (n=14)
        Peak (mean; severity score)       3.4              1.4**
        Duration (median; days)           3.7              1.9**
        AUC (median; severity score       4.1              1.8**
        over 7 days)
        * First dose was 1,200 mg for one day, subsequent doses were 600 mg
        QD for four days
        ** Statistically significant; p<0.05
        

Safety Data

In this study, VX-787 was generally well-tolerated, and all participants
completed treatment. There were no serious adverse events or adverse events
that led to discontinuation of treatment. Overall, the most frequently
reported class of adverse events in the VX-787 and placebo arms were those
typically associated with influenza-like illness. In the development program
to date, VX-787 has been dosed in approximately 170 people. The highest single
dose given was 1,600 mg, and the longest duration of dosing was 800 mg QD for
10 days. In these studies, there were no serious adverse events, and no
adverse events that led to treatment discontinuation.

About VX-787

VX-787 is an investigational medicine being developed by Vertex that is
designed to directly inhibit replication of influenza A, including recent H1
(pandemic) and H5 (avian) influenza strains, based on in-vitro data. VX-787's
mechanism represents a new class of potential medicines for the treatment of
influenza, distinct from neuraminidase inhibitors, the current standard of
care for the treatment of influenza. VX-787 is intended to provide a rapid
onset of action and an expanded treatment window. VX-787, which was discovered
by Vertex scientists, underscores the Company’s expertise in antiviral drug
development, beginning with early research in HIV and more recently in
hepatitis C. Vertex has worldwide rights to develop and commercialize VX-787.

About Influenza

Each year in the U.S., more people die from influenza than die from
HIV/AIDS.^i Often called “the flu,” seasonal influenza is caused by influenza
viruses, which infect the respiratory tract.^ii The flu can result in seasonal
epidemics^iii and can produce severe disease and high mortality in certain
populations, such as the elderly.^iv Each year, on average 5 to 20 percent of
the U.S. population gets the flu^v resulting in more than 200,000 flu-related
hospitalizations and 36,000 deaths.^vi The overall national economic burden of
influenza-attributable illness for adults is $83.3 billion.^vi Direct medical
costs for influenza in adults totaled $8.7 billion including $4.5 billion for
adult hospitalizations resulting from influenza-attributable illness.^vi The
treatment of the flu consists of antiviral medications that have been shown in
clinical studies to shorten the disease and reduce the severity of symptoms if
taken within two days of infection.^vii There is a significant need for new
medicines targeting flu that provide a wider treatment window, greater
efficacy and faster onset of action.

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. Wright’s statements in the second paragraph of this press
release and the statement regarding Vertex exploring collaborative
opportunities to support further development of VX-787. While the company
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, the
risks listed under Risk Factors in Vertex's annual report and quarterly
reports filed with the Securities and Exchange Commission and available
through Vertex'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

______________________________
^i Centers for Disease Control and Prevention. HIV in the United States: At a
Glance. CDC Web site: http://www.cdc.gov/hiv/resources/factsheets/us.htm.
Accessed February 28, 2013.

^ii Centers for Disease Control and Prevention. Key Facts about Influenza (Flu)
& Flu Vaccine. CDC Web site: www.cdc.gov/flu/keyfacts.htm. Accessed February 28,
2013.

^iii Centers for Disease Control and Prevention. Influenza (Flu) Viruses. CDC
Web site: http://www.cdc.gov/flu/about/viruses/index.htm. Accessed February 28,
2013.

^iv Centers for Disease Control and Prevention. Key Facts about Influenza (Flu)
& Flu Vaccine. CDC Web site: www.cdc.gov/flu/keyfacts.htm. Accessed February 28,
2013.

^v Centers for Disease Control and Prevention. Season Influenza. CDC Web site:
http://www.cdc.gov/flu/about/qa/disease.htm. Accessed February 28, 2013.

^vi Centers for Disease Control and Prevention. Adult Immunization. CDC Web
site:
http://www.cdc.gov/workplacehealthpromotion/evaluation/topics/immunization.html.
Accessed February 28, 2013.

^vii Centers for Disease Control and Prevention. What you should know about flu
antiviral drugs. CDC Web site:
http://www.cdc.gov/flu/antivirals/whatyoushould.htm. Accessed February 28, 2013.


Contact:

Vertex Contacts:
Investors
Michael Partridge, 617-341-6108
Kelly Lewis, 617-961-7530
or
Media
Erin Emlock, 617-341-6992
mediainfo@vrtx.com