Jan 11 2010
Vertex
Pharmaceuticals Incorporated (NASDAQ: VRTX) today provided an update
on key 2010 business priorities in conjunction with the 28th Annual J.P.
Morgan Healthcare Conference in San Francisco. The company also
discussed recent progress in its lead development programs in hepatitis
C virus (HCV) infection and cystic fibrosis (CF) and outlined
proof-of-concept clinical trials planned in other serious diseases for
2010.
“Supporting our vision to become a fully-capable biopharmaceutical
company, Vertex is also planning multiple proof-of-concept clinical
trials in other diseases, such as rheumatoid arthritis and epilepsy, and
remains committed to maintaining investment into research to enable
future product opportunities”
"2010 will be a defining year for Vertex as we seek to evolve into a
fully-capable biopharmaceutical company," said Matthew Emmens, Chairman,
President and Chief Executive Officer of Vertex. “With ongoing Phase 3
programs in hepatitis C virus infection and cystic fibrosis, a broad
pipeline of other emerging product candidate opportunities and a strong
capital structure, we believe Vertex today has a unique opportunity to
build a successful company focused on bringing multiple important
therapies to patients. In 2010, we look forward to a series of defining
events from late-stage and proof-of-concept clinical trials that may
support further growth in the years ahead.”
Mr. Emmens will deliver a live webcast presentation from the J.P. Morgan
Healthcare Conference on Tuesday, January 12 at 2:30 p.m. PT (5:30 p.m.
ET) where he will discuss recent clinical progress and provide an
overview of Vertex's 2010 priorities. The webcast will be available on
Vertex's website, www.vrtx.com.
"Phase 3 data for telaprevir, our lead drug candidate for the treatment
of hepatitis C virus infection, will begin to emerge in the spring of
2010 to support the planned submission of a New Drug Application in the
second half of this year. Our more than decade-long commitment to
improving patient care in HCV is unwavering, and the Phase 3 program for
telaprevir will remain our primary focus over the coming year.
Importantly, we also recognize the need for continued innovation in the
treatment of this disease, and we are preparing to initiate the first
clinical trial combining telaprevir with the investigational HCV
polymerase inhibitor VX-222 this quarter,” stated Mr. Emmens.
"Beyond HCV, Vertex is conducting mid-stage and late-stage development
of two novel compounds aimed at addressing, for the first time, the
underlying mechanism of the orphan disease of cystic fibrosis. The
VX-770 Phase 3 registration program is advancing rapidly, and we expect
to obtain Phase 3 data for VX-770 early in 2011. Additionally, we also
expect to obtain clinical data from a Phase 2 trial of VX-809 in the
coming weeks that could potentially support the evaluation of VX-770 and
VX-809 as part of a combination regimen in patients with the most common
mutation of this disease.
“Supporting our vision to become a fully-capable biopharmaceutical
company, Vertex is also planning multiple proof-of-concept clinical
trials in other diseases, such as rheumatoid arthritis and epilepsy, and
remains committed to maintaining investment into research to enable
future product opportunities,” concluded Mr. Emmens.
Phase 3 Registration Program for Telaprevir Nears Completion
Sustained viral response (SVR) data expected from Phase 3 ADVANCE
trial in second quarter 2010 and from Phase 3 ILLUMINATE & REALIZE
trials in third quarter 2010
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The ADVANCE, ILLUMINATE and REALIZE trials are evaluating
telaprevir-based regimens as part of a global Phase 3 registration
program in more than 2,200 genotype 1 treatment-naïve and
treatment-failure patients with HCV infection.
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Vertex today announced that all patients in the ADVANCE and ILLUMINATE
trials, which are evaluating telaprevir in treatment-naïve patients,
have completed dosing of all study drugs, including
pegylated-interferon (peg-IFN) and ribavirin (RBV), and are now in the
post-treatment follow-up period to determine the number of patients
who achieve SVR (defined as undetectable HCV RNA 24 weeks after the
end of treatment). Vertex expects SVR data to become available from
ADVANCE in the second quarter of 2010 and from ILLUMINATE in the third
quarter of 2010.
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Vertex today also announced that all patients in the REALIZE trial,
which is being conducted by Vertex’s collaborator Tibotec and is
evaluating telaprevir in patients who did not achieve SVR with a prior
pegylated interferon-based treatment, are expected to complete dosing
of all study drugs, including pegylated-interferon and ribavirin, by
the end of January. Vertex expects SVR data to become available from
REALIZE in the third quarter of 2010.
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Vertex plans to submit a New Drug Application (NDA) for telaprevir in
the second half of 2010 for both treatment-naïve and treatment-failure
patients.
Twice-daily dosing of telaprevir
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In November 2009, Vertex announced SVR results from Study C208, an
exploratory Phase 2, open-label clinical study of telaprevir. The
study was conducted by Tibotec in Europe and evaluated a twice-daily
(1125mg q12h) dosing schedule of telaprevir in combination with
peg-IFN-alfa-2a (PEGASYS(R)) or peg-IFN-alfa-2b (PEGINTRON(R))
and RBV, as compared to the three-times-daily (750mg q8h) telaprevir
dosing schedule used in telaprevir clinical trials to date. The C208
trial enrolled 161 treatment-naïve patients with genotype 1 HCV
infection. The C208 data included the first SVR data for
telaprevir-based regimens in treatment-naïve patients as part of a
response-guided therapy trial design, similar to that being used in
the ADVANCE and ILLUMINATE Phase 3 trials of telaprevir.
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Vertex has submitted the Study C208 clinical data to the U.S. FDA, and
Vertex and Tibotec have initiated discussions with regulatory
authorities in the U.S. and E.U. regarding future development plans
for telaprevir as part of an every-12-hour (q12h) dosing regimen.
Vertex collaborator completes dosing of telaprevir in Phase 3
trials in Japan
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Vertex today announced that its collaborator, Mitsubishi Tanabe Pharma
Corporation, has completed the dosing portion for telaprevir in three
ongoing Phase 3 trials of telaprevir-based combination therapy in
approximately 300 treatment-naïve and treatment-failure HCV patients
in Japan.
New capabilities to support potential launch of telaprevir
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Vertex continues to build its commercial infrastructure in preparation
for the potential launch of telaprevir and has begun to develop and
implement internal systems and processes to support the company’s
commercial operation and the potential future sale of telaprevir.
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The company expects to establish key elements of its customer-facing
operation in 2010, including the hiring of the sales management team
to be charged with the implementation of a fully-functioning
commercial sales force in 2011.
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To inform launch preparedness activities, Vertex continues to conduct
extensive HCV market research and is in discussions with key managed
markets organizations and specialty pharmacies to generate additional
insights on the HCV patient population, HCV patient care and flow,
market communication strategies, and reimbursement processes for the
HCV market.
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Vertex has assembled a global supply chain network to support the
potential launch of telaprevir and is currently manufacturing
telaprevir at a commercial (metric ton) scale. The company has
successfully completed all registration campaigns as well as
validation campaigns of the active pharmaceutical ingredient (API) and
is prepared to complete drug product validation in advance of the
potential launch of telaprevir.
Potential Future Combination Regimens for HCV with Telaprevir and the
HCV Polymerase Inhibitor VX-222
Initiation of first clinical trial of telaprevir combined with
VX-222 planned for first quarter 2010
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Vertex recently completed a multiple-dose Phase 1b viral kinetic study
of the investigational oral HCV polymerase inhibitor VX-222. Interim
results from the trial are consistent with the findings of a
previously-conducted three-day viral kinetic study and support future
clinical evaluation of VX-222, including the initiation of the first
clinical trial of VX-222 in combination with telaprevir. Additional
results from this Phase 1b study of VX-222 are planned for
presentation at a medical meeting in 2010.
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Upon completion of ongoing discussions with regulatory authorities,
Vertex plans to initiate a combination trial of telaprevir and VX-222
in the first quarter of 2010. This trial is expected to evaluate SVR
rates using multiple regimens of telaprevir/VX-222-based therapy in
HCV patients.
Addressing the Underlying Defect of Cystic Fibrosis
VX-770 Phase 3 registration program progressing; STRIVE trial
completes planned enrollment
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Vertex is currently conducting the ENDEAVOR Phase 3 registration
program of VX-770, an investigational Cystic Fibrosis Transmembrane
Conductance Regulator (CFTR) potentiator compound for the treatment of
CF. The program consists of three ongoing clinical trials, known as
STRIVE, ENVISION and DISCOVER, and is designed to evaluate the utility
of VX-770 across different age groups and genotypes, including
children as young as six years of age.
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Vertex today announced that the STRIVE trial, which is evaluating
VX-770 in patients aged 12 years and older who carry the G551D
mutation on at least one allele, has completed planned patient
enrollment. To date, more than 120 patients have enrolled in the
STRIVE trial and additional patients currently are completing the
screening process. Vertex expects that all patients in STRIVE will
have received their first dose of VX-770 or placebo by the end of
February 2010.
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The ENVISION trial, which is evaluating VX-770 in patients aged six to
11 who carry the G551D mutation on at least one allele, is ongoing.
Vertex expects to complete enrollment for ENVISION in the first half
of 2010.
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Vertex today also announced that the DISCOVER trial, which is
evaluating VX-770 in patients 12 years and older who are homozygous
for the F508del mutation, has enrolled more than 60 patients and
additional patients currently are completing the screening process.
Vertex expects to enroll approximately 120 patients in DISCOVER and
that all patients will have received their first dose of VX-770 or
placebo by the end of February 2010.
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The primary endpoint for patients with the G551D mutation (STRIVE and
ENVISION trials) is change in forced expiratory volume in one second
(FEV1), which will be measured at 24 weeks, with
additional FEV1 measurements at 48 weeks as a secondary
endpoint to assess durability of any observed response. Patients in
the STRIVE and ENVISION trials will receive either VX-770 or placebo
for 48 weeks to gain additional safety data in G551D patients. For
patients with the F508del mutations (DISCOVER trial), the primary
endpoints are safety and change in FEV1, which will be
measured at 16 weeks. Additional secondary endpoints, including sweat
chloride, will be measured in each trial to evaluate the potential
effect of VX-770 on improving the function of the defective CFTR
protein.
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Based on 48-week clinical data from the STRIVE and ENVISION trials,
and on 16-week clinical data from the DISCOVER trial, Vertex plans to
submit an NDA for VX-770 in patients with the G551D CFTR
mutation in the second half of 2011.
Phase 2a trial of VX-809 complete; Data expected in first quarter
2010
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Vertex recently completed a Phase 2a trial of VX-809, an
investigational CFTR corrector compound for the treatment of CF, in
patients homozygous for the F508del CFTR mutation.
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Vertex expects to obtain clinical data from the trial, including
measures of sweat chloride and nasal potential difference, in the
first quarter of 2010. These data could potentially support the
initiation of a combination trial of VX-770 and VX-809 in the second
half of 2010, with the goal of generating initial proof-of-concept
clinical data in late 2010.
Additional Proof-of-Concept Trials and Research Progress Support
Future Growth
Janus kinase 3 (JAK3) inhibitor VX-509 to enter Phase 2 trial in
rheumatoid arthritis
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Vertex plans to initiate a Phase 2 proof-of-concept clinical trial of
VX-509 in approximately 200 patients with moderate to severe
rheumatoid arthritis (RA) in the first quarter of 2010. The company
expects to obtain interim clinical data, including measurements of
safety, tolerability and clinical activity, in the second half of 2010.
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The double-blind, randomized, placebo-controlled trial will evaluate
the safety, tolerability and clinical activity of four doses of
VX-509. Patients will receive 12 weeks of treatment with VX-509 dosed
twice daily compared to placebo.
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The primary endpoints of the trial are to evaluate safety and to
measure the improvement in clinical signs and symptoms of RA in
patients after 12 weeks of treatment. Efficacy assessments will
include the American College of Rheumatology criteria (ACR20, ACR50
and ACR70) for defining clinical improvement in RA patients. ACR20,
ACR50 and ACR70 are standardized measures of the number of patients
who achieve at least a 20, 50 or 70 percent improvement, respectively,
in ACR-specified measures of RA activity. The trial will also utilize
disease activity score (DAS) and European League Against Rheumatism
(EULAR) response criteria as additional efficacy assessments.
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VX-509 may have broad potential for the treatment of multiple
immune-mediated inflammatory diseases. Vertex plans to pursue
collaborative opportunities for VX-509 with major pharmaceutical
companies.
Caspase-1 inhibitor VX-765 to enter Phase 2 trial in epilepsy
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Vertex plans to initiate a Phase 2 proof-of-concept clinical trial of
VX-765 in patients with epilepsy in the first quarter of 2010, which
could result in interim clinical data being available as early as the
second half of 2010.
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VX-765 has been shown to inhibit acute seizures in preclinical models
of chronic epilepsy and has shown activity in preclinical models of
chronic epilepsy that do not respond to standard anti-epileptic drugs.
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The Phase 2 trial announced today for VX-765 is expected to enroll
approximately 75 patients with treatment-resistant epilepsy. The
double-blind, randomized, placebo-controlled trial will evaluate the
safety, tolerability and clinical activity of VX-765. Patients will
receive six weeks of treatment with VX-765 following a six-week
baseline period to monitor seizure frequency. The primary endpoints of
the trial are safety and tolerability. The secondary endpoints will
evaluate the clinical efficacy of VX-765 based on the percent
reduction in seizure frequency and percent of patients with a 50
percent or greater reduction in seizure frequency, known as the
responder-rate.
Capital Structure Supports Investment into Key Business Priorities
"Vertex is committed to maintaining a strong balance sheet and capital
structure that will support our mission to discover, develop and
commercialize new medicines that provide high therapeutic benefit to
patients with major diseases and deliver return to our shareholders,"
said Ian Smith, Executive Vice President and Chief Financial Officer of
Vertex. “With a cash position of approximately $1.3 billion and
approximately $32 million of 2013 convertible debt, Vertex enters 2010
in a strong financial position to support the advancement of our key
development programs in HCV and CF and to generate important
proof-of-concept clinical data in other significant diseases.
“As our late-stage compounds advance, the company’s financial strategy
will evolve toward ensuring that our potential marketed products provide
financial support for continued investment in development programs and
in product creation from research,” continued Mr. Smith.
As of December 31, 2009, Vertex had approximately $1.3 billion in cash,
cash equivalents and marketable securities, approximately $32 million of
outstanding 2013 convertible notes and approximately 200 million shares
outstanding.
Vertex anticipates a GAAP net loss for 2009 of less than $650 million.
Vertex anticipates a 2009 non-GAAP loss of less than $515 million,
excluding stock-based compensation and executive transition expenses,
restructuring expense, acquisition-related expenses, loss on exchange of
convertible subordinated notes, and interest expense related to the
September 2009 financial transactions.
Vertex will report full-year 2009 financial results and financial
guidance for 2010 on February 4, 2010.
SOURCE Vertex Pharmaceuticals Incorporated