Vertex Announces Presentation of Data at European Cystic Fibrosis Society Conference

  Vertex Announces Presentation of Data at European Cystic Fibrosis Society
  Conference

 - Presentations include data from two Phase 2 studies in people with cystic
            fibrosis who have two copies of the F508del mutation -

Business Wire

CAMBRIDGE, Mass. -- June 5, 2013

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that six
abstracts from its cystic fibrosis (CF) program will be presented at the 36th
European Cystic Fibrosis Society (ECFS) Conference in Lisbon, Portugal, June
12 to 15, 2013. Presentations include data from Cohorts 2 and 3 of a Phase 2
study of lumacaftor (VX-809) combined with ivacaftor in people with the most
common mutation in the cystic fibrosis transmembrane conductance regulator
(CFTR) gene, F508del, as well as data from a Phase 2 study of VX-661 combined
with ivacaftor in people with two copies of the F508del mutation.
Additionally, poster presentations will feature data on the use of KALYDECO™
(ivacaftor) in people with CF ages 6 and older who have the G551D mutation.

The accepted abstracts have been published today by the Journal of Cystic
Fibrosis and are available from the "Session Planner" on the ECFSConference
website at: http://www.ecfs.eu/lisbon2013

Vertex Oral Presentations

1.“VX-661, an investigational CFTR corrector, in combination with ivacaftor,
    a CFTR potentiator, in patients with CF and homozygous for the
    F508Del-CFTR mutation: interim analysis.” An oral presentation (Abstract
    WS7.3) is scheduled during Workshop 7 – Novel Therapies on June 13, 2013,
    5:00 – 6:30 p.m. BST from lead investigator Scott H. Donaldson, M.D.,
    Associate Professor of Medicine, University of North Carolina at Chapel
    Hill. The presentation will include previously announced results from this
    study as well as additional data on the pattern of FEV[1] (forced
    expiratory volume in one second) response.
2.“Lumacaftor, an investigational CFTR corrector, in combination with
    ivacaftor, a CFTR potentiator, in CF patients with the F508Del-CFTR
    mutation: Phase 2 interim analysis.” An oral presentation (Abstract WS7.4)
    is scheduled during Workshop 7 – Novel Therapies on June 13, 2013, 5:00 –
    6:30 p.m. BST from lead investigator Michael P. Boyle, M.D., F.C.C.P.,
    Associate Professor, Director of the Johns Hopkins Adult Cystic Fibrosis
    Center. The presentation will include previously announced results from
    Cohorts 2 and 3 of the study as well as additional pharmacokinetic data
    and an additional analysis of pooled lung function data during the
    combination dosing portions of these cohorts.

Following these presentations, the slides are expected to be available on the
ECFS Conference website at: http://www.ecfs.eu/lisbon2013

Vertex Poster Presentations

1.Poster 13: “Genotype, disease severity, and healthcare resource use by
    patients with CF in the UK National Health Service.”
2.Poster 53: “Lung function, weight, and sweat chloride responses in
    patients with cystic fibrosis and the G551D-CFTR mutation treated with
    ivacaftor: A secondary analysis.”
3.Poster 56: “Effect of withdrawal of ivacaftor therapy on CFTR channel
    activity and lung function in patients with cystic fibrosis.”
4.Poster 58: “Pulmonary exacerbations in CF patients with the G551D-CFTR
    mutation treated with ivacaftor.”

Symposium Presentation

1.“CF drug discovery and personalized medicine.” Fred Van Goor, Ph.D., Head
    of Biology for Vertex’s CF program, will deliver an invited talk during
    Symposium 1 – CFTR Modulators on June 13, 2013, from 8:30 – 10:00 a.m.
    BST.

About KALYDECO

KALYDECO™ (ivacaftor) is the first medicine to treat the underlying cause of
CF in people with the G551D mutation in the CFTR gene. Known as a CFTR
potentiator, KALYDECO is an oral medicine that aims to help the CFTR protein
function more normally once it reaches the cell surface, to help hydrate and
clear mucus from the airways. KALYDECO (150mg, q12h) was first approved by the
U.S. Food and Drug Administration in January 2012, by the European Medicines
Agency in July 2012 and by Health Canada in November 2012 for use in people
with CF ages 6 and older who have at least one copy of the G551D mutation in
the CFTR gene.

Vertex retains worldwide rights to develop and commercialize KALYDECO. A
Marketing Authorization application is under review by the Therapeutic Goods
Administration (TGA) of Australia.

Indication and Important Safety Information for KALYDECO™ (ivacaftor)

KALYDECO (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.

KALYDECO 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
KALYDECO 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 KALYDECO. It is recommended that ALT and AST be assessed prior to
initiating KALYDECO, 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 KALYDECO 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 KALYDECO 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 KALYDECO compared
to placebo.

Use of KALYDECO 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 KALYDECO, which may diminish
effectiveness. Therefore, co-administration is not recommended.

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

KALYDECO can cause serious adverse reactions including abdominal pain and high
liver enzymes in the blood. The most common side effects associated with
KALYDECO 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 KALYDECO. A list of the adverse reactions can be
found in the full product labeling for each country where KALYDECO 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 Cystic Fibrosis

Cystic fibrosis is a rare, life-threatening genetic disease affecting
approximately 70,000 people worldwide, including 30,000 people in the United
States, 35,000 in Europe, 4,000 in Canada and 3,000 in 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 1,800 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 absence of working 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.

Collaborative History withCystic Fibrosis Foundation Therapeutics,
Inc.(CFFT)

Vertex initiated its CF research program in 1998 as part of a collaboration
with CFFT, the nonprofit drug discovery and development affiliate of the
Cystic Fibrosis Foundation. This collaboration was expanded to support the
accelerated discovery and development of Vertex's CFTR modulators.

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, statements regarding the data that Vertex expects will be
presented at the 36th European Cystic Fibrosis Society (ECFS) Conference in
Lisbon, Portugal, June 12 to 15, 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, the 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 Media:
Zach Barber, +1-617-341-6992
or
Megan Goulart, +44 22 593 6066
mediainfo@vrtx.com
or
Investors:
Michael Partridge, +1-617-341-6108
Kelly Lewis, +1-617-961-7530
 
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