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Vertex Reports First Quarter 2012 Financial Results and Provides Update on Launch of KALYDECO™
-Approximately 600 people with cystic fibrosis have started treatment
with KALYDECO since approval on
-Multiple ongoing clinical trials to generate data beginning in second quarter, including study of KALYDECO combined with VX-809 in cystic fibrosis and first data for Alios nucleotides in hepatitis C-
Vertex reported total first quarter 2012 revenues of approximately
"Since January, approximately 600 people with cystic fibrosis have
started treatment with KALYDECO in the U.S., underscoring the importance
of this new medicine to the CF community and further demonstrating our
ability to develop and launch transformative new medicines," said
"The success of our first two approved medicines provides for continued investment in our pipeline, where multiple ongoing clinical studies will begin to generate important new data beginning this quarter. We're also preparing to initiate Phase 2b studies of all-oral, short-duration treatment regimens for hepatitis C and of our JAK3 inhibitor VX-509 for rheumatoid arthritis, as well as pivotal studies of KALYDECO in people with types of cystic fibrosis not studied in earlier trials," concluded Dr. Leiden.
KALYDECO Launch: Vertex today announced that
approximately 600 people have started treatment with KALYDECO
FDAapproval on January 31. KALYDECO is approved in the U.S. for people with cystic fibrosis ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
European Marketing Authorization Application: In December, the
European Medicines Agency(EMA) validated Vertex's marketing authorization application (MAA) for KALYDECO. The acceptance of the MAA marked the start of the regulatory review process by the Committee for Medicinal Products for Human Use(CHMP). Vertex expects to obtain approval of KALYDECO in Europein the third quarter of 2012.
- Studies of KALYDECO in other CFTR Mutations: In 2012, Vertex plans to begin three additional pivotal studies of KALYDECO that will enroll people with CF who have certain CFTR mutations that were not evaluated in the previous Phase 3 studies, as well as children with CF as young as two years of age. The first two of these studies are expected to begin in the middle of the year and will include a study of people with CF who have at least one copy of the R117H CFTR mutation, which is present in approximately three percent of people with CF in the U.S., and a study in people with CF who have other CFTR 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 four percent of people with CF. The remaining gating mutations to be evaluated in the second study account for an additional approximately one percent of people with CF in the U.S. Pending final feedback from regulatory agencies, a third study is planned for later this year in children with CF as young as two years of age who have gating mutations.
- Combination Study in Most Common Type of CF: Vertex is also conducting a Phase 2 study of the CFTR corrector VX-809 dosed in combination with KALYDECO in people with the most common CFTR mutation, known as F508del. Final data from this study are expected in mid-2012. The study enrolled people with two copies of the F508del mutation (homozygotes) and also included the first evaluation of KALYDECO combined with VX-809 in people with one copy of the F508del mutation and one copy of certain non-gating mutations (heterozygotes). A Phase 2 study of VX-661, a second CFTR corrector, dosed in combination with KALYDECO is also ongoing, with data expected in the second half of 2012.
Global Availability of Telaprevir: Telaprevir (INCIVEK,
INCIVO, TELAVIC) is now available in countries in
North America, Europe, South Americaand the Far East. Vertex's collaborator, Janssen, is marketing telaprevir in more than 15 countries in Europeand other regions as INCIVO. Vertex's collaborator Mitsubishi Tanabe Pharma markets this medicine in Japanas TELAVIC®.
- Viral Kinetic Studies of Two Alios Nucleotides: Seven-day viral kinetic studies of the nucleotide analogues ALS-2200 and ALS-2158 are ongoing in people with hepatitis C. The first data from these studies are expected in the second quarter of 2012. If successful, Vertex plans to begin Phase 2 studies in the second half of 2012 to evaluate combination regimens of ALS-2200 or ALS-2158 with INCIVEK or VX-222 with or without ribavirin, as well as other potential interferon-free combination regimens.
Phase 2b Study of 12-week All-Oral Regimen of INCIVEK, VX-222 and
Ribavirin: In the second quarter of 2012, Vertex plans to begin an
approximately 100-person Phase 2b study of an all-oral treatment
regimen of INCIVEK, VX-222 and ribavirin in people with genotype 1
hepatitis C. The study will evaluate treatment regimens as short as 12
weeks. If successful, data from this study will be used to design a
Phase 3 program with the goal of submitting a New Drug Application
(NDA) to the
U.S. Food and Drug Administration(FDA) for Vertex's first interferon-free regimen for genotype 1 (1a and 1b) patients as early as the end of 2014, pending regulatory discussions.
- Initiation of Phase 2b Study: In May, Vertex plans to initiate a global Phase 2b study of the JAK3 inhibitor VX-509 in people with moderate to severe rheumatoid arthritis (RA) that will evaluate once and twice-daily dosing in combination with methotrexate. The six-month study is expected to enroll approximately 350 people.
- Ongoing Phase 2 Study of VX-787: Vertex recently initiated a Phase 2, randomized, double-blind, placebo-controlled study of VX-787 that is expected to enroll approximately 140 healthy volunteers who will be infected with live influenza virus as part of this study. The primary efficacy endpoint of the study is the reduction in viral shedding. Data from the study are expected in the second half of 2012. VX-787 is an investigational medicine that is designed to treat influenza A, including recent H1 (pandemic) and H5 (avian) influenza strains.
First Quarter 2012 Financial Results
"With the launch of KALYDECO earlier this year and the global
availability of INCIVEK and INCIVO, Vertex has begun to generate
significant revenues from approved medicines worldwide," said
Total Revenues: Total revenues for the first quarter of
Net Product Revenues from INCIVEK: Net product revenues from
INCIVEK for the first quarter of 2012 were
Net Product Revenues from KALYDECO: Net product revenues from
KALYDECO, which was approved on
January 31, 2012, were $18.4 millionin the first quarter of 2012.
Royalty Revenues: Vertex recognized
$39.0 millionin royalty revenues in the first quarter of 2012, including $32.9 millionin INCIVO royalty revenues from our collaborator Johnson and Johnson.
Collaborative Revenues: Vertex recognized
$24.4 millionin collaborative revenues in the first quarter of 2012.
Research and Development (R&D) Expenses: R&D expenses for the
first quarter of 2012 were
Sales, general and administrative (SG&A) expenses: SG&A
expenses for the first quarter of 2012 were
GAAP Net Income (Loss) Attributable to Vertex: Vertex's GAAP net
income for the first quarter of 2012 was
Non-GAAP Net Income (Loss) Attributable to Vertex: Vertex's
non-GAAP net income for the first quarter of 2012, was
2012 Financial Guidance
Vertex today reiterated its financial guidance, as provided on
Non-GAAP Financial Measures
In this press release, Vertex's financial results and financial guidance
are provided both in accordance with accounting principles generally
First Quarter Results
Consolidated Statements of Operations Data
(in thousands, except per share amounts)
Three Months Ended
Product revenues, net
|Costs and expenses:|
|Cost of product revenues||25,918||---|
Research and development expenses (R&D)
|Sales, general & administrative expenses (SG&A)||111,146||71,523|
|Total costs and expenses||347,088||233,561|
|Income (loss) from operations||91,649||(159,899)|
|Net interest expense (Note 2)||(3,741)||(10,599)|
|Change in fair value of derivative instruments (Note 2)||---||(5,598)|
|Income (loss) before provision for income taxes||87,908||(176,096)|
|Provision for income taxes||32||---|
|Net income (loss)||87,876||(176,096)|
|Net loss attributable to noncontrolling interest (Note 1)||(3,714)||---|
|Net income (loss) attributable to Vertex||
|Net income (loss) per share attributable to Vertex common shareholders:|
|Shares used in per share calculations:|
Reconciliation of GAAP to Non-GAAP Financial Information
(in thousands, except per share amounts)
Three Months Ended
|Operating costs and expenses||347,088||(5,086||)||(27,627||)||—||(360||)||314,015|
|Income from operations||91,649||5,086||27,627||—||360||124,722|
|Other income and expenses||(3,741||)||(62||)||—||—||—||(3,803||)|
|Income before provision for income taxes||87,908||5,024||27,627||—||360||120,919|
|Provision for income taxes||32||2,280||—||—||—||2,312|
|Net loss attributable to noncontrolling interest (Alios)||(3,714||)||3,714||—||—||—||—|
|Net income attributable to Vertex||$||91,590||$||(970||)||$||27,627||$||—||$||360||$||118,607|
|Net income per share attributable to Vertex common shareholders:|
|Shares used in per share calculations:|
Three Months Ended
|Operating costs and expenses||233,561||—||(27,879||)||—||(760||)||204,922|
|Loss from operations||(159,899||)||—||27,879||(50,000||)||760||(181,260||)|
|Other income and expenses||(16,197||)||—||—||13,532||—||(2,665||)|
|Net loss attributable to Vertex||$||(176,096||)||$||—||$||27,879||$||(36,468||)||$||760||$||(183,925||)|
|Net loss per share attributable to Vertex common shareholders:|
|Shares used in per share calculations:|
Condensed Consolidated Balance Sheets Data
|Cash, cash equivalents and marketable securities||
|Restricted cash and cash equivalents (Alios) (Note 1)||58,017||51,878|
|Accounts receivable, net||232,228||183,135|
|Other current assets||35,407||14,889|
|Property and equipment, net||170,331||133,176|
|Intangible assets (Note 3)||663,500||663,500|
|Goodwill (Note 3)||30,992||30,992|
|Other non-current assets||10,816||11,268|
|Liabilities and Shareholders' Equity|
|Accrued restructuring expense||25,473||26,313|
|Deferred tax liability (Note 3)||241,426||243,707|
|Convertible notes (due 2015)||400,000||400,000|
|Noncontrolling interest (Alios) (Note 1)||175,079||178,669|
|Shareholders' equity (Vertex)||930,498||786,843|
|Total liabilities and shareholders' equity||
|Common shares outstanding||210,863||209,304|
Note 1: The company has consolidated the financial statements of
Note 2: In the first quarter of 2011, a portion of the
collaborative revenues, the change in fair value of derivative
instruments and a portion of the net interest expense reflected in the
Consolidated Statements of Operations Data relate to two financial
transactions that the company entered into in September 2009 relating to
milestone payments under the company's collaboration agreement with
Note 3: The intangible assets, the goodwill and the deferred tax
liability reflected in the Condensed Consolidated Balance Sheets Data
relate to the company's acquisition of
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 diseases.
Founded more than 20 years ago in
Vertex's press releases are available at www.vrtx.com.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO
KALYDECOTM is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients ages 6 years and older who have a certain mutation in their CFTR gene called the G551D mutation.
KALYDECO 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.
It is not known if KALYDECO is safe and effective in children under 6 years of age.
KALYDECO should not be used with certain medicines, including the antibiotics rifampin and rifabutin; seizure medications (phenobarbital, carbamazepine, or phenytoin); and the herbal supplement St. John's Wort.
KALYDECO can cause serious side effects. High liver enzymes in the blood have occurred in patients taking KALYDECO. Regular assessment is recommended.
The most common side effects associated with KALYDECO include headache; upper respiratory tract infection (common cold) including sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness.
These are not all the possible side effects of KALYDECO. Patients should tell their healthcare providers about any side effect that bothers them or doesn't go away.
Please see full Prescribing Information for KALYDECO at www.KALYDECO.com.
INDICATON AND IMPORTANT SAFETY INFORMATION FOR INCIVEK
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.
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as defined in the
Private Securities Litigation Reform Act of 1995, including Dr. Leiden's
statements in the third and fourth paragraphs of this press release, Mr.
Smith's statements in the paragraph following the caption "First Quarter
2012 Financial Results," the information provided under the caption
"2012 Financial Guidance" and statements regarding (i) multiple ongoing
clinical trials generating data beginning in the second quarter of 2012;
(ii) the expected timing of data from (A) studies of VX-809 and VX-661,
in each case dosed in combination with KALYDECO, (B) studies of ALS-2200
and ALS-2158 and (C) studies of VX-787; (iii) the plans to conduct
additional studies of KALYDECO, a Phase 2b study of VX-222, INCIVEK and
ribavirin, Phase 2 studies of ALS-2200 or ALS-2158 and a global Phase 2b
study of VX-509; and (iv) the expected timing of an approval of KALYDECO
Conference Call Information
Vertex will host a conference call and webcast today,
To listen to the live call on the telephone, dial 1-877-250-8889 (
The conference ID number for the live call and replay is 63676268.
The call will be available for replay via telephone commencing
Following the live webcast, an archived version will be available on
Vertex's website until
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