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Press release from Business Wire

Vertex Announces Presentation of Data at European Cystic Fibrosis Society Conference

<p class='bwalignc'> <i>- Presentations include data from two Phase 2 studies in people with cystic fibrosis who have two copies of the F508del mutation -</i> </p>

Wednesday, June 05, 2013

Vertex Announces Presentation of Data at European Cystic Fibrosis Society Conference

10:21 EDT Wednesday, June 05, 2013

CAMBRIDGE, Mass. (Business Wire) -- 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 ECFS Conference 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 FEV1 (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 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 2,000 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

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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