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Vertex Outlines Corporate Strategy And Defines Key 2013 Business Priorities

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today outlined the company’s strategy and defined key 2013 business priorities to support continued growth and the creation of long-term shareholder value. Vertex’s Chairman, President and Chief Executive Officer, Jeffrey Leiden, M.D., Ph.D., will discuss the company’s strategy and 2013 priorities as part of a live presentation at the 31 st Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 7 at 10:00 a.m. PT (1:00 p.m. ET). The presentation will be webcast on Vertex’s website, www.vrtx.com.

“Since the approval of our first medicine less than two years ago, Vertex has undergone a rapid evolution that has positioned the company to move forward with a clear focus on using innovative science to develop transformative medicines for serious diseases in specialty markets,” said Dr. Leiden.

“In cystic fibrosis and hepatitis C, KALYDECO and INCIVEK are just the beginning of what we hope to provide to people with these diseases. In CF, multiple ongoing and planned studies of KALYDECO monotherapy and other combinations of our CF medicines in development aim to help many more people with this disease. In hepatitis C, we are focused on developing multiple all-oral regimens that could further improve treatment. In addition to our development programs, we continue to invest in research for future medicines, with a focus on serious diseases where we have significant scientific expertise and commercial capabilities.”

Entering 2013, Vertex’s key strategies and business priorities include:

1. Focusing investment on key development programs for:
  • Cystic Fibrosis (CF): Vertex’s CF strategy is to provide benefit to as many CF patients as possible, and to maximize the benefit for these patients, with our approved and investigational medicines. KALYDECO TM (ivacaftor) is currently approved for people with CF ages 6 and older who have at least one copy of the G551D mutation in the CFTR gene (approximately 2,000 people with CF worldwide). In 2013, the company is conducting multiple Phase 3 label-expansion and other proof-of-concept studies of ivacaftor monotherapy in people with certain mutations not studied in prior Phase 3 studies. Vertex also expects to initiate a pivotal Phase 3 development program for a combination regimen of VX-809 and ivacaftor in people with CF who have two copies of the F508del mutation in the first quarter of 2013.
  • Hepatitis C: Vertex’s strategy in hepatitis C is to develop new all-oral treatment regimens of 12 weeks or less in duration with a goal of providing a high viral cure rate and improved tolerability. In 2013, Vertex plans to conduct multiple Phase 2 studies of 12-week all-oral treatment regimens that include the company’s nucleotide analogue VX-135. These studies are expected to provide safety and viral cure rate data in the second half of 2013 to support the start of pivotal development of one or more all-oral regimens in 2014.
  • Autoimmune Diseases: Vertex’s strategy in autoimmune diseases is to maximize the value of VX-509 across multiple autoimmune diseases globally. The company will evaluate collaborative opportunities that provide funding and capabilities to broaden and accelerate global development of VX-509.

2. Investing in innovative research programs to support development of additional specialty medicines for serious diseases
  • Vertex’s research efforts are concentrated on additional advancements in CF and other genetic diseases and additional serious diseases in specialty markets.

3. Maximizing revenues and cash flow from the appropriate use of INCIVEK ® (telaprevir) and KALYDECO in the U.S. and Canada and from the growth of KALYDECO in Europe and other countries

4. Maintaining financial strength to support future growth and shareholder returns

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