VRTX

Vertex Pharmaceuticals Incorporated (VRTX)

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Vertex Pharmaceuticals Incorporated (VRTX)

Phase II Results for VX-661 Conference

April 18, 2013 4:30 pm ET

Executives

Michael Partridge - Senior Director of Strategic Communications

Jeffrey M. Leiden - Chairman, Chief Executive Officer and President

Peter R. Mueller - Chief Scientific Officer, Executive Vice President of Global Research & Development and Member of The Scientific Advisory Board

Ian F. Smith - Chief Financial Officer and Executive Vice President

Eric Olson

Analysts

Geoffrey C. Porges - Sanford C. Bernstein & Co., LLC., Research Division

Geoffrey C. Meacham - JP Morgan Chase & Co, Research Division

Omar Saad - ISI Group Inc., Research Division

Rachel L. McMinn - BofA Merrill Lynch, Research Division

Michael J. Yee - RBC Capital Markets, LLC, Research Division

Liisa A. Bayko - JMP Securities LLC, Research Division

Yaron Werber - Citigroup Inc, Research Division

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Matthew Roden - UBS Investment Bank, Research Division

Brian Corey Abrahams - Wells Fargo Securities, LLC, Research Division

Ying Huang - Barclays Capital, Research Division

Alethia Young - Deutsche Bank AG, Research Division

Y. Katherine Xu - William Blair & Company L.L.C., Research Division

Howard Liang - Leerink Swann LLC, Research Division

Koon Ching

Philip Nadeau - Cowen and Company, LLC, Research Division

Presentation

Operator

Good day, ladies and gentlemen, and welcome to the Vertex Phase II results for VX-661 conference call [Operator Instructions] As a reminder, this conference is being recorded. I would now like to turn the call over to Michael Partridge. You may begin.

Michael Partridge

Thank you, everyone, for dialing in today. This afternoon, we are pleased to announce Phase II results for VX-661 dosed alone and in combination with ivacaftor for 28 days in patients who are homozygous for the F508del mutation.

With me on the call today are Jeff Leiden, our Chairman and CEO, and Peter Mueller, Chief Scientific Officer. After prepared remarks, we will be joined by Eric Olson, cystic fibrosis disease area research lead, and Ian Smith, CFO, and we will take your questions. We anticipate concluding the call before 5:30 p.m. and we will be available in our offices following the call.

Before we begin, I'll remind you that the information discussed on this conference call includes forward-looking statements, which are subject to the risks and uncertainties discussed in detail in our reports, including our 10-K and 10-Q reports, which have been filed with the Securities and Exchange Commission, and are also discussed in our press release today. These statements, including without limitation, those regarding KALYDECO and the ongoing discovery and development and potential commercialization of Vertex's cystic fibrosis drug candidates, are based on management's current assumptions. All are subject to risks and uncertainties that could cause actual outcomes and events to differ materially.

During the call, our speakers will be referring to data and graphs contained on the webcast slides. For those of you dialing in, it may be helpful for you to go to the events section of our website and access the webcast slides now, if you haven't done so already. I'll now turn it over to Jeff.

Jeffrey M. Leiden

Thanks, Michael. Good afternoon, everyone. As most of you know, Vertex has had a long-standing and broad strategy to develop breakthrough drugs that address the underlying genetic defects that cause cystic fibrosis. Our overall goal is to develop a portfolio of medicines that will enable us to offer effective treatment to the vast majority of patients with this serious and debilitating genetic disease.

Today, I'll briefly review that strategy and then summarize our significant progress in discovering and developing our CF medicines, in order to provide context for the Phase II clinical results for VX-661 that Peter will discuss today. Our CF strategy has 3 parts: First, we aim to use KALYDECO monotherapy to treat up to 10% to 15% of the 70,000 patients with CF worldwide.

Last year, we launched KALYDECO, known generically as ivacaftor, for CF patients with a G551D mutation. We believe there are approximately 2,000 patients aged 6 and older globally with this type of CF. Today, the vast majority of G551D patients in the U.S. are receiving KALYDECO, and we continue to work to ensure access for G551D patients in Europe and Canada.

During the last several months, we've achieved reimbursement in a number of key markets outside the U.S., and we believe this will drive further access to KALYDECO for more patients this year. Over the next several years our goal is to expand the number of CF patients that we treat with ivacaftor monotherapy. Towards this end we are currently conducting a number of clinical studies of ivacaftor monotherapy, including first Phase III study in patients with other gating mutations, these are similar mutations to the G551D. This study is now fully enrolled, and we expect data in the second half of the year.

Second, a Phase III study in people with the R117H mutation, R117H is a specific type of residual function mutation this study continues to enroll. Third, a study of children ages 2 through 5 who have a G551D or a gating mutation. All of these studies are studies that, if positive, could help to expand the label for KALYDECO. We are also conducting a clinical study in patients with residual CFTR function. This study includes several different types of less common mutations, as well as R117H, and the goal here is the be able to define more broadly, the types of patients who may benefit from ivacaftor alone.

The first results from these trials will be available in the second half of this year, and we continue to have productive discussions with the USFDA about how the breakthrough designation of ivacaftor may help us accelerate its development to provide access to patients beyond G551D. If these studies are successful, we may have the opportunity to use ivacaftor monotherapy to treat up to 10% to 15% of the estimated 70,000 CF patients worldwide.

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