Vertex Pharmaceuticals Incorporated : Vertex Announces Key 2012 Business Objectives as Company Prepares for Planned Global Launch of KALYDECO in Cystic Fibrosis
01/08/2012| 08:20pm US/Eastern
-More than 25,000 people have started treatment for hepatitis C with
INCIVEK®, positioning Vertex for continued growth,
earnings and cashflow in 2012-
-Preparations for approval and launch of KALYDECOTM
ongoing; additional studies of KALYDECO planned for mid-2012-
-Nine new medicines in development for serious diseases; multiple
proof-of-concept and later-stage studies planned for 2012-
Vertex Pharmaceuticals Incorporated
(NASDAQ: VRTX) today announced its 2012 business objectives in
conjunction with the 30th Annual J.P. Morgan Healthcare Conference in
San Francisco. Matthew Emmens, Chairman, President and Chief Executive
Officer of Vertex, and Jeffrey Leiden, M.D., Ph.D., who will become
Vertex's CEO on February 1, 2012, will discuss these objectives as part
of a live presentation, which will be available on Vertex's website, www.vrtx.com,
on Monday, January 9 at 11:00 a.m. PT (2:00 p.m. ET).
"In 2011, our team executed a highly successful launch for INCIVEK in
hepatitis C, with more than 25,000 people starting treatment since its
approval in mid-2011," said Mr. Emmens. "With the strength of the launch
for INCIVEK, the submission of our global approval applications for
KALYDECO in cystic fibrosis and the advancement of our pipeline
programs, we are positioned for significant growth, earnings and
cashflow in 2012."
Dr. Leiden commented, "Entering 2012, we are focused on becoming a
sustainable business with strong revenues from INCIVEK and the planned
global launch of KALYDECO for cystic fibrosis. Importantly, we are
pursuing opportunities to further improve treatment with our all-oral
regimens in development for hepatitis C and efforts to study our cystic
fibrosis medicines in a larger group of people with this devastating
disease. As these and other pipeline programs advance, we will manage
our business with financial discipline and focused investment to ensure
the greatest benefit for patients waiting for new treatments and for our
Preparing for Global Launch of KALYDECOTM in
Cystic Fibrosis and Expanding the KALYDECO Development Program
In December, Vertex announced that the U.S. Food and Drug Administration
(FDA) accepted the New Drug Application for KALYDECO (ivacaftor) and
granted the company's request for six-month Priority Review. A target
review date of April 18, 2012 is set under the Prescription Drug User
Fee Act for the FDA's approval decision. Vertex's marketing
authorization application for KALYDECO has also been validated by the
European Medicines Agency, which accepted Vertex's request for
accelerated assessment in Europe.
As Vertex prepares for the potential launch of KALYDECO for people with
the G551D mutation, the company is also planning to begin additional
studies of KALYDECO in children with CF as young as two years of age and
in people with CF who have certain mutations that were not evaluated in
the previous Phase 3 studies. Pending final feedback from regulatory
agencies, the company plans to begin three clinical studies of KALYDECO
Pediatric study: A study of KALYDECO in children ages 2
through 5 with gating mutations, including G551D, is expected to
evaluate the safety, tolerability and effect on sweat chloride and
other measures of clinical activity using a pediatric formulation of
Study in people with the R117H mutation: Vertex plans to
begin the first clinical study of KALYDECO in people who have at least
one copy of the R117H mutation in the CF gene. The R117H mutation
causes abnormal function of the CFTR protein at the cell surface.
Approximately 3 percent of people with CF in the U.S. have the R117H
Study in other gating mutations: Vertex also plans to
begin the first clinical study of KALYDECO in other gating mutations
where CFTR proteins are present at the cell surface but do not
function properly. G551D is the most common gating mutation, present
in approximately 4 percent of people with CF in the U.S., and was the
focus of previous Phase 3 KALYDECO studies. The remaining gating
mutations to be evaluated in this study account for an additional
approximately 1 percent of people with CF in the U.S.
Additional Studies in Hepatitis C, CF and Other Serious Diseases
Multiple additional studies of INCIVEK, KALYDECO and Vertex's pipeline
medicines in development are ongoing or planned for 2012, including:
Phase 2 ZENITH study: The ongoing Phase 2 ZENITH study is
designed to assess the safety, tolerability and efficacy of the
polymerase inhibitor VX-222 dosed in combination with INCIVEK and
ribavirin, with and without pegylated-interferon, in people with
genotype 1a and 1b chronic hepatitis C who were new to treatment. In
the first quarter, Vertex expects to announce data, including
sustained viral response rates at 4 weeks post-treatment (SVR4), from
the two all-oral, interferon-free study arms (Arms E and F) in which
patients received VX-222, INCIVEK and ribavirin.
All-oral, interferon-free studies of the nucleotide analogues
ALS-2200 and ALS-2158: Vertex and Alios are currently conducting
two Phase 1 studies of the pan-genotypic hepatitis C polymerase
inhibitors ALS-2200 and ALS-2158. The studies are evaluating safety
and tolerability in healthy volunteers as well as 7-day viral kinetics
in people with chronic genotype 1 hepatitis C. Data are expected in
the second quarter of 2012, which could enable the initiation of Phase
2 proof-of-concept studies to evaluate multiple all-oral,
interferon-free combination regimens in the second half of 2012. These
Phase 2 studies are expected to evaluate combination regimens of
ALS-2200 or ALS-2158 with INCIVEK or VX-222, potential dual nucleotide
regimens (adenosine and uracil) and other interferon-free combination
regimens that may also include ribavirin.
Phase 3 study in people co-infected with hepatitis C and HIV:
Enrollment is ongoing in a Phase 3 trial of INCIVEK combination
therapy in people co-infected with genotype 1 hepatitis C virus and
Phase 3 study of twice-daily dosing of INCIVEK: Enrollment is
complete in a Phase 3 clinical trial to evaluate twice-daily dosing of
INCIVEK (1,125 mg; BID) compared to three-times-daily dosing of
INCIVEK (750 mg; q8h) as part of INCIVEK combination therapy.
Phase 4 study of INCIVEK combination treatment in African
Americans: Vertex plans to begin in the first quarter of 2012 a
study of INCIVEK combination therapy in African Americans with
hepatitis C who were not cured with a prior treatment of
pegylated-interferon and ribavirin.
Phase 3b study of INCIVEK combination treatment for a total
duration of 12 weeks: Enrollment is ongoing in a Phase 3b trial to
evaluate the potential for INCIVEK combination therapy to be shortened
to 12 weeks in people with genotype 1 chronic hepatitis C who have the
'CC' variation near the IL28B gene.
Phase 2b and 3b studies in people with hepatitis C following a
liver transplant: Enrollment is expected to begin in the first
quarter for clinical studies of INCIVEK combination treatment in
people who have recurrent hepatitis C following a liver transplant.
Two CFTR correctors in development for people with the most common
CF mutation, F508del: Enrollment is ongoing in the second part of
a Phase 2 clinical trial of combination regimens of KALYDECO, a CFTR
potentiator, and VX-809, a CFTR corrector, in people with the most
common mutation in CF, known as F508del. In addition, Vertex plans to
begin Phase 2 development of VX-661, a second CFTR corrector, in the
first quarter of 2012. Data from the study with VX-809 is expected
mid-year, followed by data from the study with VX-661 later in 2012.
Rheumatoid Arthritis (RA)
350-patient Phase 2b study of VX-509: A six-month Phase
2b study of the JAK3 inhibitor VX-509 is planned to begin in the first
quarter of 2012 for the treatment of moderate to severe rheumatoid
arthritis. This study will evaluate once-daily (QD) and twice-daily
(BID) doses of VX-509 in combination with methotrexate, a commonly
prescribed disease-modifying antirheumatic drug (DMARD) for RA that is
frequently used in combination with other RA medicines.
Proof-of-concept study planned for mid-2012 with VX-787: A
Phase 1 study is ongoing for VX-787, an investigational medicine that
is designed to treat influenza A, including recent H1 (pandemic) and
H5 (avian) influenza strains. Following the completion of this Phase 1
study, Vertex plans to initiate a proof-of-concept study for VX-787 in
the second quarter of 2012.
Enrollment is ongoing in a Phase 2 study of VX-765 in people with
Continued Productivity in Research
Vertex continues to focus its research efforts in the areas of
infectious diseases, including viral infections - such as influenza -
and bacterial infections, inflammatory diseases, cancer and neurological
disorders, including pain. Vertex expects additional development
candidates for the treatment of one or more of these diseases to emerge
from research in 2012.
The company will report full-year 2011 financial results and financial
guidance on February 2, 2012.
Vertex Pharmaceuticals will webcast its corporate presentation at the
30th Annual J.P. Morgan Healthcare Conference on January 9, 2012 at
11:00 a.m. PT (2:00 p.m. ET). A link to the live webcast will be
available via Vertex's website, www.vrtx.com,
in the Events & Presentations section. An archived webcast of the
presentation will be available on Vertex's website through January 23,
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, epilepsy and other life-threatening
Founded more than 20 years ago in Cambridge, MA, 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 Science
magazine named Vertex number one on its 2011 list of Top Employers in
the life sciences.
IMPORTANT SAFETY INFORMATION
INCIVEK? (telaprevir) is a prescription medicine used with the medicines
peginterferon alfa and ribavirin to treat chronic (lasting a long time)
hepatitis C genotype 1 infection in adults with stable liver problems,
who have not been treated before or who have failed previous treatment.
It is not known if INCIVEK is safe and effective in children under 18
years of age.
Important Safety Information
INCIVEK should always be taken in combination with peginterferon alfa
and ribavirin. Ribavirin may cause birth defects or death of an unborn
baby. Therefore, a patient should not take INCIVEK combination treatment
if she is pregnant or may become pregnant, or if he is a man with a
sexual partner who is pregnant. Patients must use two forms of effective
birth control during treatment and for the 6 months after treatment with
these medicines. Hormonal forms of birth control, including birth
control pills, vaginal rings, implants or injections, may not work
during treatment with INCIVEK.
INCIVEK and other medicines can affect each other and can also cause
side effects that can be serious or life threatening. There are certain
medicines patients cannot take with INCIVEK combination treatment.
Patients should tell their healthcare providers about all the medicines
they take, including prescription and non-prescription medicines,
vitamins and herbal supplements.
INCIVEK can cause serious side effects including skin reactions, rash
and anemia that can be severe. The most common side effects of INCIVEK
include itching, nausea, diarrhea, vomiting, anal or rectal problems,
taste changes and tiredness. There are other possible side effects of
INCIVEK, and side effects associated with peginterferon alfa and
ribavirin also apply to INCIVEK combination treatment. Patients should
tell their healthcare providers about any side effect that bothers them
or doesn't go away.
Please see full Prescribing Information for INCIVEK including the
Medication Guide, available at www.INCIVEK.com.
Safe Harbor Statement
This press release contains forward-looking statements as defined in the
Private Securities Litigation Reform Act of 1995, including the
statements made by Mr. Emmens and Dr. Leiden in the second and third
paragraphs of the press release, and statements regarding (i) Vertex
being positioned for continued growth, earnings and cashflow in 2012;
(ii) regulatory timelines for KALYDECO; (iii) Vertex's preparations for
the potential launch of KALYDECO; (iv) proof-of-concept studies and
later-stage studies planned for 2012; (v) planned and ongoing studies of
INCIVEK, KALYDECO and the company's drug candidates and the expected
timelines for initiating and announcing data from these studies and (vi)
the expectation that additional development candidates will emerge from
the company's research programs in 2012. While the company 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,
that the outcomes for each of Vertex's planned clinical trials and
studies may not be favorable, that regulatory authorities may require
supplemental clinical trials in order to support the registration of
KALYDECO, that planned or potential clinical trials may be delayed or
may not be conducted, that the company may not be able to successfully
develop its drug candidates, and other 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.
The company disclaims any obligation to update the information contained
in this press release as new information becomes available.
(VRTX - GEN)
Vertex Pharmaceuticals Incorporated
Lora Pike, 617-444-6755
(at J.P. Morgan Healthcare Conference:
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