Vertex Announces Upcoming Presentations of Data at North American Cystic Fibrosis Conference

  Vertex Announces Upcoming Presentations of Data at North American Cystic
  Fibrosis Conference

Business Wire

CAMBRIDGE, Mass. -- September 3, 2013

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that five
abstracts from its cystic fibrosis (CF) research and development program will
be presented at the 27^th Annual North American Cystic Fibrosis Conference
(NACFC) in Salt Lake City, Utah, October 17-19, 2013. Data from a Phase 3
study of ivacaftor in non-G551D gating mutations will be presented as well as
long-term safety and efficacy data for KALYDECO™ (ivacaftor) from the PERSIST
open-label rollover study. In addition, Fred Van Goor, Ph.D., Head of Biology
for Vertex's CF program, and David Rodman, M.D., Vice President of Clinical
Development for Vertex's CF program, will participate in invited talks
regarding their work to discover and develop medicines that target the
underlying cause of CF.

The accepted abstracts are now available in the online edition of Pediatric
Pulmonology: Supplement: The 27th Annual North American Cystic Fibrosis
Conference, Salt Palace Convention Center, Salt Lake City, Utah, October
17-19, 2013: http://onlinelibrary.wiley.com/doi/10.1002/ppul.v48.S36/issuetoc.

Abstracts

All abstracts will be presented as part of Poster Session 1 on October 17,
11:50 a.m. - 1:50 p.m. MDT:

  *“Ivacaftor, a CFTR potentiator, in cystic fibrosis patients who have a
    non-G551D-CFTR gating mutation: Phase 3, Part 1 results.” Poster 241.
    Vertex also expects these data to be presented as part of Symposium III,
    “CFTR: Matching CFTR Mutations and Drugs,” on October 19, 10:30 a.m. -
    12:20 p.m. MDT.
  *“Effect of ivacaftor in patients with cystic fibrosis and the G551D-CFTR
    mutation who have baseline FEV[1] >90% of predicted.” Poster 257.
  *“Long-term safety and efficacy of ivacaftor in patients with cystic
    fibrosis who have the G551D-CFTR mutation: response through 144 weeks of
    treatment (96 weeks of PERSIST).” Poster 227.
  *“Effect of ivacaftor on circulating inflammatory indices in CF patients
    with the G551D-CFTR mutation.” Poster 259.
  *“Evaluation of the drug-drug interaction potential of ivacaftor on a
    combined oral contraceptive.” Poster 262.

Symposium Sessions

Two additional Vertex presentations will take place as part of invited talks
during Symposium Sessions II and III:

  *“Matching Novel Therapies to CF-Causing Mutations in Cell-based Systems.”
    Fred Van Goor, Ph.D., will deliver an invited talk during Symposium
    Session III, “CFTR: Matching CFTR Mutations & Drugs,” on October 19 at
    11:30 a.m. MDT.
  *“Lessons Learned From the Development of First Generation CFTR
    Modulators.” David Rodman, M.D., will deliver an invited talk during
    Symposium II, “NT/CFTR: Advances in the Therapeutic Pipeline for CFTR
    Repair (Combination),” on October 18 at 11:55 a.m. MDT.

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, by Health Canada in November 2012 and by the Therapeutic
Goods Administration in Australia in July 2013 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.

Indication and Important Safety Information for KALYDECO™ (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,
the KALYDECO Canadian Product Monograph at www.vrtx.ca and the Australian
Consumer Medical Information and Product Information at http://bit.ly/18wlMld.

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 with Cystic 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 2,200 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.

(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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