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

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

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (NAS: VRTX) today announced that five abstracts from its cystic fibrosis (CF) research and development program will be presented at the 27th 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:


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 FEV1 >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.

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, the EU Summary of Product Characteristics for KALYDECO at, the KALYDECO Canadian Product Monograph at and the Australian Consumer Medical Information and Product Information at

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.


Vertex Pharmaceuticals Incorporated
Michael Partridge, 617-341-6108
Kelly Lewis, 617-961-7530
Zach Barber, 617-341-6470

KEYWORDS:   United States  North America  Massachusetts  Utah


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