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Vertex Reports First Quarter 2013 Financial Results and Reviews Recent Progress in Development Programs for Cystic Fibrosis and



  Vertex Reports First Quarter 2013 Financial Results and Reviews Recent
  Progress in Development Programs for Cystic Fibrosis and Hepatitis C

  -First quarter 2013 total revenues of $328 million, including net product
   revenues of $206 million for INCIVEK in hepatitis C and $62 million for
                         KALYDECO in cystic fibrosis-

    -Cystic fibrosis: Enrollment ongoing in Phase 3 program for VX-809 in
combination with ivacaftor for people with two copies of the F508del mutation-

     -Hepatitis C: multiple all-oral combination studies ongoing with the
             nucleotide analogue HCV polymerase inhibitor VX-135-

Business Wire

CAMBRIDGE, Mass. -- April 30, 2013

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated
financial results for the quarter ended March 31, 2013. The company also today
increased its KALYDECO net revenue guidance for 2013 and reiterated other
components of financial guidance, as provided on January 29, 2013.

Vertex reported total first quarter 2013 revenues of $328 million, including
net product revenues of $206 million from INCIVEK^® (telaprevir) and $62
million from KALYDECO^TM (ivacaftor). The GAAP net loss attributable to Vertex
was $(308.0) million, or $(1.43) per share, for the first quarter of 2013. The
company’s first quarter 2013 GAAP net loss includes certain charges of $313.8
million, comprised primarily of a one-time charge, net of a tax benefit, of
$285.3 million related to an impairment of an intangible asset. Non-GAAP net
income attributable to Vertex for the first quarter of 2013 was $5.7 million,
or $0.03 per diluted share. The company reported $1.24 billion in cash, cash
equivalents and marketable securities as of March 31, 2013.

“During the first quarter of the year, we have made significant advances
across our business as  we continue to execute on our strategy of developing
new medicines focused on serious diseases in specialty markets," said Jeffrey
Leiden, M.D., Ph.D., Chair, President and Chief Executive Officer of Vertex.
"With the recent initiation of a Phase 3 program in cystic fibrosis, multiple
ongoing all-oral studies in hepatitis C, label-expansion studies for
ivacaftor, and significant advances in our early stage pipeline, we are well
positioned for continued progress over the rest of this year and beyond."

Development Program Updates

Cystic Fibrosis

Vertex's strategy in cystic fibrosis (CF) 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.

Continued Progression of Label-Expansion Studies for Ivacaftor Monotherapy

  * Three Phase 3 label-expansion studies are ongoing for ivacaftor
    monotherapy, including a study in people with CF ages 6 and older who have
    at least one copy of the R117H mutation, a study in people with CF ages 6
    and older who have at least one non-G551D CFTR gating mutation and a study
    in children with CF ages 2 to 5 who have a gating mutation. Enrollment of
    the study of gating mutations is complete, with the first data expected in
    the second half of 2013. Enrollment is ongoing in the study of children
    ages 2 to 5 and in the study in people with the R117H mutation. Enrollment
    is also ongoing in a Phase 2 proof-of-concept study evaluating ivacaftor
    in people with CF who have clinical evidence of residual CFTR function.
    Vertex believes that ivacaftor monotherapy may be able to treat between
    10% and 15% of the estimated 70,000 CF patients worldwide, pending results
    of clinical studies.

Initiation of Phase 3 Studies for VX-809 in Combination with Ivacaftor

  * Vertex recently initiated a Phase 3 program for VX-809 in combination with
    ivacaftor that consists of two 24-week Phase 3 studies in people ages 12
    and older with two copies of the most common mutation in the cystic
    fibrosis transmembrane conductance regulator (CFTR) gene, known as
    F508del. Enrollment in the Phase 3 program is underway. Worldwide, nearly
    half of people with CF have two copies of the F508del mutation. Additional
    details on the Phase 3 program were provided in a press release issued
    February 26, 2013.

Phase 2 Data for VX-661 in Combination with Ivacaftor

  * Vertex recently announced data from a Phase 2 study of VX-661 in
    combination with ivacaftor in people with two copies of the F508del
    mutation. In the study, treatment with a combination of VX-661 and
    ivacaftor for 28 days in the two highest dose groups resulted in mean
    relative increases in lung function (percent predicted FEV[1]) of 9.0%
    (p=0.01) and 7.5% (p=0.02) versus placebo. In contrast, patients who
    received placebo showed a 0.03% mean relative change in lung function at
    Day 28 (within-group). In the study, VX-661 was generally well-tolerated,
    both as monotherapy and in combination with ivacaftor, and most adverse
    events were pulmonary in nature, were mild to moderate in severity and
    similar between the treatment groups and those who received placebo.
    Additional details on these data were provided in a press release issued
    April 18, 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.

Multiple Ongoing Studies of VX-135 as Part of All-Oral Treatment Regimens

  * Vertex is currently evaluating multiple all-oral regimens that include
    VX-135, Vertex’s nucleotide analogue hepatitis C virus (HCV) polymerase
    inhibitor. Ongoing and planned studies include:

       * Genotype 1

            * Two Phase 2 studies of VX-135 in combination with ribavirin are
              currently ongoing in people with genotype 1 HCV infection.
              Vertex today announced that one of these studies is fully
              enrolled.
            * A drug-drug interaction study of VX-135 in combination with
              simeprevir is ongoing in healthy volunteers. Simeprevir (TMC435)
              is a once-daily investigational hepatitis C protease inhibitor
              being jointly developed by Janssen R&D Ireland and Medivir AB.

       * Genotypes 1, 2 or 3 and People with Cirrhosis

            * Vertex plans to conduct two Phase 2 studies of VX-135 and
              Bristol-Myers Squibb's NS5A replication complex inhibitor
              daclatasvir. An initial study in people with genotype 1 HCV
              infection is planned for the second quarter of 2013. Vertex
              plans to begin a subsequent study in people infected with
              genotype 1, 2 or 3 HCV, including those with cirrhosis, in the
              second half of 2013, pending data from the initial study.

       * Vertex expects to obtain the first data from all-oral studies of
         VX-135 in the second half of 2013, including data from the initial
         study of VX-135 with daclatasvir and from the studies of VX-135 with
         ribavirin.

Data for ALS-2200 (VX-135) in Genotypes 2, 3 and 4 and in People with
Cirrhosis Presented at EASL

  * At the 48th Annual Meeting of the European Association for the Study of
    the Liver (EASL), Vertex announced new data from a 7-day viral kinetic
    study of ALS-2200 in people with genotypes 2, 3 and 4 HCV and those with
    cirrhosis. The data showed significant reductions in HCV RNA after seven
    days of dosing with ALS-2200 (200 mg) once daily and were consistent with
    previously reported data in people with genotype 1 chronic HCV infection.
    ALS-2200 was well-tolerated in this study, there were no serious adverse
    events and no patients discontinued due to adverse events. Additional
    details on these data were provided in a press release issued April 23,
    2013.

Data from CONCISE Study of Telaprevir Presented at EASL

  * Also at EASL, Vertex announced new data from an interim analysis of the
    CONCISE study, which showed that treatment with telaprevir combination
    therapy for a total of 12 or 24 weeks resulted in high viral cure rates in
    people with genotype 1 HCV with the IL28B CC genotype who had a rapid
    viral response and completed at least 12 weeks of treatment. The safety
    profile of telaprevir combination therapy observed in the CONCISE study
    through the time of the interim analysis was similar to that seen in
    previously reported clinical trials. Additional details on these data were
    provided in a press release issued April 24, 2013.

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.

Enrollment Complete in Phase 2b Study of VX-509 in Rheumatoid Arthritis

  * Vertex today announced that enrollment is complete in a 24-week Phase 2b
    study of VX-509, a selective JAK3 inhibitor, in people with moderate to
    severe rheumatoid arthritis (RA) receiving methotrexate. The primary
    endpoints of this study will be measured after 12 weeks of treatment, and
    data from this analysis are expected in the second half of 2013.

First Quarter 2013 Financial Results

Total Revenues: Total revenues for the first quarter of 2013 were $328.4
million, compared with $438.7 million in total revenues for the first quarter
of 2012. The components of total revenues for the first quarter of 2013 and
2012 were:

                                   Three Months Ended March 31,
                                   2013               2012
Product revenues                   (in millions)
INCIVEK revenues, net              $  205.6           $  356.9
KALYDECO revenues, net             61.8               18.4
Total product revenues, net        267.4              375.4
Royalty revenues
Royalty revenues from INCIVO       39.0               32.9
Other royalty revenues             4.6                6.0
Total royalty revenues             43.6               39.0
Collaborative revenues             17.4               24.4
Total revenues                     $  328.4           $  438.7
                                                          

A table of the components of total revenues for the first quarter of 2013 and
each quarter in 2012 is provided following the Condensed Consolidated
Statements of Operations Data.

  * Net Product Revenues from INCIVEK

    Vertex's first quarter 2013 net product revenues from INCIVEK were $205.6
    million, compared to $356.9 million for the first quarter of 2012. The
    reduced revenues from INCIVEK were due to fewer HCV patients initiating
    treatment in the first quarter of 2013 compared to the first quarter of
    2012.

  * Net Product Revenues from KALYDECO

    Vertex's first quarter 2013 net product revenues from KALYDECO were $61.8
    million, compared to $18.4 million for the first quarter of 2012. The
    increased revenues, compared to the first quarter of 2012, resulted
    primarily from the rapid uptake of KALYDECO in the vast majority of
    eligible patients in the U.S. following FDA approval in January 2012.

  * Royalty Revenues from INCIVO^®

    Vertex recognized $39.0 million in INCIVO royalty revenues for the first
    quarter of 2013 from our collaborator Janssen, compared to $32.9 million
    in INCIVO royalty revenues for the first quarter of 2012. The increase in
    INCIVO royalties was due to expanded availability of INCIVO in
    international markets.

Cost of Product Revenues: Cost of product revenues was $31.0 million for the
first quarter of 2013, compared to cost of product revenues of $25.9 million
for the first quarter of 2012. The increase in cost of product revenues was
due to a $9.3 million commercial milestone related to net sales of KALYDECO
under our agreement with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT).

Research and Development (R&D) Expenses: R&D expenses were $218.1 million for
the first quarter of 2013, including $23.3 million of Vertex stock-based
compensation expense and Alios expenses related to the accounting for the
collaboration with Vertex, compared to $196.4 million for the first quarter of
2012, including $21.1 million of Vertex stock-based compensation expense and
Alios expenses related to the accounting for the collaboration with Vertex.
The increase in Vertex's R&D investment is principally due to progression and
expansion of clinical development programs in cystic fibrosis, hepatitis C and
rheumatoid arthritis, including initiation of a pivotal program for a
combination of VX-809 and ivacaftor and advancement of all-oral studies for
VX-135.

Sales, General and Administrative (SG&A) Expenses: SG&A expenses were $92.9
million for the first quarter of 2013, including $13.1 million of Vertex
stock-based compensation expense and Alios expenses related to the accounting
for the collaboration with Vertex, compared to $111.1 million for the first
quarter of 2012, including $11.6 million of Vertex stock-based compensation
expense and Alios expenses related to the accounting for the collaboration
with Vertex. This decrease in SG&A expenses resulted primarily from reduced
HCV marketing and commercial expenses.

GAAP Net Income (Loss) Attributable to Vertex: Vertex's first quarter 2013
GAAP net loss was $(308.0) million, or $(1.43) per share. The company’s first
quarter 2013 GAAP net loss includes certain charges of $313.8 million,
comprised primarily of a one-time charge of $412.9 million, which was
partially offset by a tax benefit of $127.6 million, related to an impairment
of an intangible hepatitis C asset (VX-222). Vertex's GAAP net income for the
first quarter of 2012 was $91.6 million, or $0.43 per diluted share, including
$27.0 million in certain charges.

Non-GAAP Net Income Attributable to Vertex: Vertex's first quarter 2013
non-GAAP net income was $5.7 million, or $0.03 per diluted share. Vertex's
non-GAAP net income for the first quarter of 2012 was $118.6 million, or $0.55
per diluted share. The decrease in the company's first quarter 2013 non-GAAP
net income, compared to the first quarter of 2012, is primarily attributable
to a decrease in total revenues, specifically decreased INCIVEK revenues due
to fewer HCV patients initiating treatment. Total non-GAAP operating expenses
for the first quarter of 2013 were consistent with the first quarter of 2012.

Cash Position: As of March 31, 2013, Vertex had $1.24 billion in cash, cash
equivalents and marketable securities compared to $1.32 billion in cash, cash
equivalents and marketable securities as of December 31, 2012.

Convertible Debt: As of March 31, 2013, Vertex had $400.0 million in
convertible debt due in October 2015. The conversion price of the debt is
$48.83 per share and is callable on or after October 1, 2013. Vertex holds a
provisional redemption option that allows the debt to be called prior to
October 1, 2013 if the closing price of Vertex shares is above $63.48 per
share for 20 of 30 consecutive trading days.

2013 Financial Guidance

This section contains forward-looking guidance about the financial outlook for
Vertex Pharmaceuticals.

Vertex today updated its financial guidance for full-year 2013 KALYDECO net
revenues. The company now expects full-year 2013 KALYDECO net revenues to be
in the range of $300 million to $340 million. The prior range, provided on
January 29, 2013, was for full-year 2013 KALYDECO net revenues to be in the
range of $280 million to $320 million.

The company today reiterated its financial guidance for total 2013 revenues to
be in the range of $1.10 billion to $1.25 billion. The company also reiterated
its guidance for total 2013 non-GAAP operating expenses, excluding cost of
revenues, stock-based compensation expense, intangible asset impairment
charges and Alios expenses related to the accounting for the collaboration
with Vertex, of $1.09 billion to $1.15 billion, including full-year 2013 R&D
expenses of $750 million to $790 million and full-year 2013 SG&A expenses
of $340 million to $360 million.

Non-GAAP Financial Measures

In this press release, Vertex's financial results and financial guidance are
provided in accordance with accounting principles generally accepted in the
United States (GAAP) and using certain non-GAAP financial measures. In
particular, Vertex provides its first quarter 2013 and 2012 non-GAAP net
income excluding stock-based compensation expense, restructuring expense,
intangible asset impairment charges, net of tax, and charges related to
changes in the fair value of expected future payments under Vertex's
collaboration with Alios. These results are provided as a complement to
results provided in accordance with GAAP because management believes these
non-GAAP financial measures help indicate underlying trends in the company's
business, are important in comparing current results with prior period results
and provide additional information regarding its financial position.
Management also uses these non-GAAP financial measures to establish budgets
and operational goals that are communicated internally and externally, and to
manage the company's business and to evaluate its performance. A
reconciliation of the GAAP financial results to non-GAAP financial results is
included in the attached financial statements.

Vertex Pharmaceuticals Incorporated
First Quarter Results
Condensed Consolidated Statements of Operations Data
(in thousands, except per share amounts)
(unaudited)
 
                                                Three Months Ended
                                                March 31,
                                                2013               2012
Revenues:
Product revenues, net                           $ 267,381          $ 375,375
Royalty revenues                                43,573             38,981
Collaborative revenues                          17,414             24,381     
Total revenues                                  328,368            438,737    
Costs and expenses:
Cost of product revenues                        30,955             25,918
Royalty expenses                                11,788             13,293
Research and development expenses (R&D)         218,095            196,371
Sales, general and administrative               92,879             111,146
expenses (SG&A)
Restructuring expense                           39                 360
Intangible asset impairment charge (Note        412,900            —          
1)
Total costs and expenses                        766,656            347,088    
Income (loss) from operations                   (438,288   )       91,649
Other income (expense), net                     (4,652     )       (3,741    )
Income (loss) before provision for              (442,940   )       87,908
(benefit from) income taxes
Provision for (benefit from) income taxes       (130,313   )       32         
(Note 1)
Net income (loss)                               (312,627   )       87,876
Net loss attributable to noncontrolling         4,611              3,714      
interest (Note 2)
Net income (loss) attributable to Vertex        $ (308,016 )       $ 91,590   
                                                                    
Net income (loss) per share attributable
to Vertex common shareholders:
Basic                                           $ (1.43    )       $ 0.44
Diluted                                         $ (1.43    )       $ 0.43
                                                                    
Shares used in per share calculations:
Basic                                           215,421            208,018
Diluted                                         215,421            219,264
                                                                              
                                                                              

Consolidated Revenues
(in millions)
(unaudited)
                   
                    Three Months Ended
                    March       December     September     June        March
                    31,         31,          30,           30,         31,
                    2013        2012         2012          2012        2012
Product
revenues
INCIVEK             $ 205.6     $  222.8     $  254.3      $ 327.7     $ 356.9
revenues, net
KALYDECO            61.8        58.5         49.2          45.5        18.4
revenues, net
Total product       267.4       281.3        303.5         373.3       375.4
revenues, net
Royalty
revenues
Royalty
revenues from       39.0        36.8         20.0          28.0        32.9
INCIVO
Other royalty       4.6         6.7          5.6           5.5         6.0
revenues
Total royalty       43.6        43.5         25.6          33.5        39.0
revenues
Collaborative       17.4        9.2          6.9           11.6        24.4
revenues
Total               $ 328.4     $  334.0     $  336.0      $ 418.3     $ 438.7
revenues
                                                                          
                                                                          

Reconciliation of GAAP to Non-GAAP Financial Information-First Quarter
(in thousands, except per share amounts)
(unaudited)
 
Three Months
Ended March                         Adjustments                                                      
31, 2013
                                                                     Intangible
                                                    Stock-based      Asset
                   GAAP             Alios           Compensation     Impairment     Restructuring     Non-GAAP
                                    Transaction     Expense          Charge,        Expense
                                                                     Net of
                                                                     Tax
Income (loss)
from               $ (438,288 )     $  5,289        $   31,152       $  412,900     $      39         $ 11,092
operations
Other income       (4,652     )     8               —                —              —                 (4,644   )
(expense), net
Income (loss)
before
provision for      (442,940   )     5,297           31,152           412,900        39                6,448
(benefit from)
income taxes
Provision for
(benefit from)     (130,313   )     3,426           —                127,586        —                 699       
income taxes
Net income         (312,627   )     1,871           31,152           285,314        39                5,749
(loss)
Net loss
(income)
attributable
to                 4,611            (4,611    )     —                —              —                 —         
noncontrolling
interest
(Alios)
Net income
(loss)             $ (308,016 )     $  (2,740 )     $   31,152       $  285,314     $      39         $ 5,749   
attributable
to Vertex
Net income
(loss) per
diluted share
attributable       $ (1.43    )                                                                       $ 0.03
to Vertex
common
shareholders
(Note 3)
                                                                                                         
                                                                                                         

Three Months
Ended March                       Adjustments                                                      
31, 2012
                                                                   Intangible
                                                  Stock-based      Asset
                   GAAP           Alios           Compensation     Impairment     Restructuring     Non-GAAP
                                  Transaction     Expense          Charge,        Expense
                                                                   Net of
                                                                   Tax
Income (loss)
from               $ 91,649       $  5,086        $   27,627       $     —        $     360         $ 124,722
operations
Other income         (3,741 )        (62    )         —                  —              —             (3,803  )
(expense), net
Income (loss)
before
provision for        87,908          5,024            27,627             —              360           120,919
(benefit from)
income taxes
Provision for
(benefit from)       32              2,280            —                  —              —             2,312    
income taxes
Net income           87,876          2,744            27,627             —              360           118,607
(loss)
Net loss
(income)
attributable
to                   3,714           (3,714 )         —                  —              —             —        
noncontrolling
interest
(Alios)
Net income
(loss)             $ 91,590       $  (970   )     $   27,627       $     —        $     360         $ 118,607  
attributable
to Vertex
Net income
(loss) per
diluted share
attributable       $ 0.43                                                                           $ 0.55
to Vertex
common
shareholders
(Note 3)
                                                                                                               
                                                                                                               

                                                  Three Months Ended March 31,
                                                  2013             2012
GAAP operating costs and expenses                 $  766,656       $ 347,088
Adjustments:
Cost of product revenues                          (30,955    )     (25,918   )
Royalty expenses                                  (11,788    )     (13,293   )
Stock-based compensation expense                  (31,152    )     (27,627   )
Alios transaction                                 (5,289     )     (5,086    )
Intangible asset impairment charge                (412,900   )     —
Restructuring expense                             (39        )     (360      )
Non-GAAP operating costs and expenses             $  274,533       $ 274,804
                                                                    
GAAP research and development expenses            $  218,095       $ 196,371
Adjustments:
Stock-based compensation expense                  (19,273    )     (17,161   )
Alios transaction                                 (4,048     )     (3,960    )
Non-GAAP research and development expenses        $  194,774       $ 175,250
                                                                    
GAAP sales, general, and administrative           $  92,879        $ 111,146
expenses
Adjustments:
Stock-based compensation expense                  (11,879    )     (10,466   )
Alios transaction                                 (1,241     )     (1,126    )
Non-GAAP sales, general, and administrative       $  79,759        $ 99,554
expenses
                                                                              
                                                                              

Condensed Consolidated Balance Sheets Data
(in thousands)
(unaudited)
                                                            
                                        March 31, 2013       December 31, 2012
Assets
Cash, cash equivalents and              $  1,239,354         $    1,321,215
marketable securities
Restricted cash and cash                63,008               69,983
equivalents (Alios) (Note 2)
Accounts receivable, net                194,054              143,250
Inventories                             21,532               30,464
Other current assets                    47,835               24,673
Restricted cash                         31,934               31,934
Property and equipment, net             504,232              433,609
Intangible assets (Note 1)              250,600              663,500
Goodwill                                30,992               30,992
Other non-current assets                8,693                9,668
Total assets                            $  2,392,234         $    2,759,288
                                                              
Liabilities and Shareholders'
Equity
Other liabilities                       $  398,628           $    429,372
Accrued restructuring expense           22,459               23,328
Deferred tax liability (Note 1)         151,664              280,367
Deferred revenues                       125,830              123,808
Construction financing lease            316,821              268,031
obligation
Convertible notes (due 2015)            400,000              400,000
Noncontrolling interest (Alios)         230,717              235,202
(Note 2)
Shareholders' equity (Vertex)           746,115              999,180
Total liabilities and                   $  2,392,234         $    2,759,288
shareholders' equity
                                                              
Common shares outstanding               218,652              217,287
                                                              
                                                              

Note 1: As of March 31, 2013, the intangible assets and deferred tax liability
reflected in the Condensed Consolidated Balance Sheets Data relate to the
company's collaboration agreement with Alios BioPharma, Inc.

In the first quarter of 2013, the company determined that the value of VX-222
had become impaired and that the fair value of VX-222 was zero as of March 31,
2013. This resulted in a $412.9 million impairment charge. In connection with
this impairment charge, the company recorded a credit of $127.6 million in its
provision for income taxes.

Note 2: The company has consolidated the financial statements of its
collaborator Alios as of March 31, 2013, December 31, 2012, and for the three
months ended March 31, 2013 and 2012. The company's interest and obligations
with respect to Alios' assets and liabilities are limited to those accorded to
the company in its collaboration agreement with Alios. Restricted cash and
cash equivalents (Alios) reflects Alios' cash and cash equivalents, which
Vertex does not have any interest in and which will not be used to fund the
collaboration. Each reporting period Vertex estimates the fair value of the
contingent milestone payments and royalties payable by Vertex to Alios. Any
increase in the fair value of these contingent milestone and royalty payments
results in a decrease in net income attributable to Vertex (or an increase in
net loss attributable to Vertex) on a dollar-for-dollar basis.

Note 3: Shares used in non-GAAP net income per diluted share attributable to
Vertex common shareholders were 218,317,000 and 219,264,000 for the three
months ended March 31, 2013 and 2012, respectively.

Indication and Important Safety Information for KALYDECO^TM (ivacaftor)

Ivacaftor (150mg tablets) is indicated for the treatment of cystic fibrosis
(CF) in patients age 6 years and older who have a G551D mutation in the CFTR
gene.

Ivacaftor 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. The efficacy and safety
of ivacaftor in children younger than 6 years of age have not been evaluated.

High liver enzymes (transaminases, ALT and AST) have been reported in patients
receiving ivacaftor. It is recommended that ALT and AST be assessed prior to
initiating ivacaftor, every 3 months during the first year of treatment, and
annually thereafter. Patients who develop increased transaminase levels should
be closely monitored until the abnormalities resolve. Dosing should be
interrupted in patients with ALT or AST of greater than 5 times the upper
limit of normal. Following resolution of transaminase elevations, consider the
benefits and risks of resuming ivacaftor dosing. Moderate transaminase
elevations are common in subjects with CF. Overall, the incidence and clinical
features of transaminase elevations in clinical trials was similar between
subjects in the ivacaftor and placebo treatment groups. In the subset of
patients with a medical history of elevated transaminases, increased ALT or
AST have been reported more frequently in patients receiving ivacaftor
compared to placebo.

Use of ivacaftor with medicines that are strong CYP3A inducers such as the
antibiotics rifampin and rifabutin; seizure medications (phenobarbital,
carbamazepine, or phenytoin); and the herbal supplement St. John's Wort
substantially decreases exposure of ivacaftor, which may diminish
effectiveness. Therefore, co-administration is not recommended.

The dose of ivacaftor must be adjusted when concomitantly used with potent and
moderate CYP3A inhibitors. The dose of ivacaftor must be adjusted when used in
patients with moderate or severe hepatic disease.

Ivacaftor can cause serious adverse reactions including abdominal pain and
high liver enzymes in the blood. The most common side effects associated with
ivacaftor include headache; upper respiratory tract infection (the common
cold), including sore throat, nasal or sinus congestion, and runny nose;
stomach (abdominal) pain; diarrhea; rash; and dizziness. These are not all the
possible side effects of ivacaftor. A list of the adverse reactions can be
found in the full product labeling for each country where ivacaftor is
approved. Patients should tell their healthcare providers about any side
effect that bothers them or doesn't go away.

Please see full U.S. Prescribing Information for KALYDECO at www.KALYDECO.com,
the EU Summary of Product Characteristics for KALYDECO at http://goo.gl/N3Tz4,
and the KALYDECO Canadian Product Monograph at www.vrtx.ca.

Indication and Important Safety Information for INCIVEK (telaprevir)

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® (telaprevir) should always be used in combination with peginterferon
alfa and ribavirin. INCIVEK combination treatment may cause serious side
effects including skin rash and serious skin reactions, anemia (low red blood
cell count) that can be severe, and birth defects or death of an unborn baby.

Skin rashes are common with INCIVEK combination treatment. Sometimes these
skin rashes and other skin reactions can become serious, require treatment in
a hospital, and may lead to death. Patients should call their healthcare
provider right away if they develop any skin changes during treatment with
INCIVEK. Their healthcare provider will decide if they need treatment or if
they need to stop INCIVEK or any of their other medicines. Patients should not
stop taking INCIVEK combination treatment without talking with their
healthcare provider first.

Patients' healthcare providers will do blood tests regularly to check for
anemia. If anemia is severe, the healthcare providers may tell them to stop
taking INCIVEK.

INCIVEK combined with peginterferon alfa and 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. Females who can become pregnant and
females whose male partner takes these medicines must have a negative
pregnancy test before starting treatment, every month during treatment, and
for 6 months after treatment ends. Patients must use two forms of effective
birth control during treatment and for 6 months after all treatment has ended.
These two forms of birth control should not contain hormones, as these 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.

The most common side effects of INCIVEK combination treatment include itching,
nausea, diarrhea, vomiting, anal or rectal problems (including hemorrhoids,
discomfort, burning or itching around or near the anus), 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 provider about
any side effect that bothers them or doesn't go away.

Please see full Prescribing Information including Boxed Warning, and the
Medication Guide for INCIVEK available at www.INCIVEK.com.

About Vertex

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 and other life-threatening diseases.

Founded more than 20 years ago in Cambridge, Mass., 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 for three years in
a row, Science magazine has named Vertex one of its Top Employers in the life
sciences.

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, without
limitation, Dr. Leiden's statements in the third paragraph of the press
release, the information provided in the section captioned "2013 Financial
Guidance" and statements regarding (i) Vertex’s strategies in cystic fibrosis,
HCV and autoimmune diseases; (ii) the potential timing of clinical data from
ongoing clinical trials; (iii) the percentage of patients that Vertex may be
able to treat with ivacaftor monotherapy; (iv) ongoing and planned studies
involving VX-135; and (v) potential collaborative opportunities that could
providing funding and capabilities to broaden and accelerate global
development of VX-509. While Vertex 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 company's expectations regarding its
2013 total revenues and/or operating expenses may be incorrect (including
because one or more of the company's assumptions underlying its revenue or
expense expectations may not be realized), that the outcomes of Vertex's
ongoing and planned clinical studies may not be favorable, that the initiation
of planned studies may be delayed or prevented, 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. Vertex disclaims any obligation to update the information
contained in this press release as new information becomes available.

Conference Call and Webcast
Vertex will host a conference call and webcast today, April 30, 2013 at 5:00
p.m. ET to review financial results and recent developments. The conference
call will be webcast live, and a link to the webcast may be accessed from the
‘Vertex Events' page of Vertex's website at www.vrtx.com.

To listen to the live call on the telephone, dial 1-866-501-1537 (United
States and Canada) or 1-720-545-0001 (International). To ensure a timely
connection, it is recommended that users register at least 15 minutes prior to
the scheduled webcast.

The conference ID number for the live call and replay is 30435497.

The call will be available for replay via telephone commencing April 30,
2013 at 8:00 p.m. ET running through 5:00 p.m. ET on May 7, 2013. The replay
phone number for the United States and Canada is 1-855-859-2056. The
international replay number is 1-404-537-3406.

Following the live webcast, an archived version will be available on Vertex's
website until 5:00 p.m. ET on May 7, 2013. Vertex is also providing a podcast
MP3 file available for download on the Vertex website at www.vrtx.com.

(VRTX-GEN)

Contact:

Vertex Contacts:
Investors:
Michael Partridge, 617-341-6108
or
Kelly Lewis, 617-961-7530
or
Media:
Zach Barber, 617-341-6470
mediainfo@vrtx.com
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