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Vertex Reports First Quarter 2011 Financial Results and Reviews Milestones for Key Development Programs
-Cystic Fibrosis: Phase 3 program for VX-770 supports applications for approval in U.S. and E.U., with NDA and MAA planned for second half of 2011-
-Financial: Vertex enters second quarter with more than
"Our progress in recent months with both INCIVEK for people with
hepatitis C and VX-770 for people with cystic fibrosis marks a
significant step toward Vertex becoming a company capable of
discovering, developing and launching innovative new medicines for
serious diseases," said
"We are pleased with the outcome of our recent
"We have many important milestones still ahead in 2011, and we believe our financial position will continue to support our business as we prepare for the launch of INCIVEK and advance toward becoming a cash flow- and earnings-positive company in 2012," concluded Mr. Emmens.
Recent Clinical Development Progress
Vertex today reviewed recent progress in its clinical development programs and provided the following updates:
Preparing for Launch of INCIVEKTM (telaprevir)
- Vertex today announced that it intends to use the name INCIVEK (in-SEE-veck) as the trade name for telaprevir. If approved, telaprevir will be marketed by Vertex as INCIVEK in the U.S.
Vertex recently completed its
FDA Antiviral Drugs Advisory Committeemeeting for INCIVEK (telaprevir). At the conclusion of the meeting, the committee voted unanimously (18-0) to recommend FDAapproval of INCIVEK (telaprevir) for people with genotype 1 chronic hepatitis C who were not treated previously and those who were treated previously but not cured with currently available medicines. Vertex expects the FDAto provide its formal decision on the New Drug Application for INCIVEK (telaprevir) by May 23.
Janssen-Cilag International NV, is awaiting a formal decision from the European Medicines Agency(EMA) on its Marketing Authorisation Application (MAA) for telaprevir in the EU. The EMA accepted telaprevir for accelerated assessment, which is granted to new medicines of major public health interest. Vertex believes that Tibotec, a division of Janssen-Cilag, may receive a response on the MAA in the second half of 2011.
Vertex's entire commercial function is in place and prepared for the
planned launch of INCIVEK (telaprevir). Approximately 200 field-based
employees have been hired to date to support the future use of INCIVEK
the United Statesfollowing the planned launch, including a sales team of 115 therapeutic specialists and others who will support the future sale of INCIVEK (telaprevir).
Phase 3b Study of Twice-daily Dosing of INCIVEK (telaprevir)
- Patient enrollment is ongoing in a Phase 3b clinical trial to evaluate twice-daily dosing of INCIVEK (telaprevir; 1,125 mg; BID) compared to three-times-daily dosing of INCIVEK (telaprevir; 750 mg; q8h) in combination with Pegasys® (pegylated-interferon alfa-2a) and Copegus® (ribavirin) for people with chronic genotype 1 hepatitis C. The study, known as OPTIMIZE, does not include a control arm of pegylated-interferon and ribavirin alone. Sustained viral response (SVR or viral cure) data from OPTIMIZE are expected as early as the second half of 2012, which could support the submission of a supplemental NDA for twice-daily (BID) dosing of INCIVEK (telaprevir) by the end of 2012.
Phase 2 Combination Study of INCIVEK (telaprevir) and VX-222
- Vertex is conducting a Phase 2 clinical trial evaluating multiple 12- and 24-week response-guided regimens of INCIVEK (telaprevir) dosed in combination with Vertex's lead investigational polymerase inhibitor, VX-222, for the treatment of hepatitis C. The study currently includes three treatment arms. Two of the treatment arms are fully enrolled and are evaluating four-drug combinations of INCIVEK (telaprevir; 1,125 mg; BID), VX-222 (400 mg or 100 mg; BID), pegylated-interferon and ribavirin. Vertex expects to complete enrollment in the second quarter of 2011 in a three-drug treatment arm that will evaluate an all-oral, interferon-free regimen of INCIVEK (telaprevir; 1,125 mg), VX-222 (400 mg) and ribavirin dosed twice daily. A final arm may be added to the trial per protocol based on data from other arms of the study. In April, Vertex announced interim results from this study and expects to present additional data from the study in the second half of 2011.
Phase 3 Study of INCIVEK (telaprevir) in
- In February, Vertex reported interim results from a Phase 2 clinical trial of INCIVEK (telaprevir) dosed in combination with pegylated-interferon and ribavirin in people who are infected with genotype 1 chronic hepatitis C and HIV. Based on these results, Vertex plans to initiate in the second half of 2011 a Phase 3 study of INCIVEK (telaprevir) dosed in combination with pegylated-interferon and ribavirin in people co-infected with HCV and HIV. The Phase 3 trial will be designed to generate data that, if positive, could support the submission of a supplemental NDA for this population.
Phase 2 Study of INCIVEK (telaprevir) to Evaluate 3-month Treatment Regimens
- In the third quarter of 2011, Vertex plans to initiate a clinical trial to evaluate a 12-week treatment regimen of INCIVEK (telaprevir) dosed in combination with pegylated-interferon and ribavirin for people who have a specific genetic marker, known as CC, near the IL28B gene. In April, Vertex announced data from retrospective analyses that evaluated the relationship between variations at the IL28B gene and a patient's response to treatment with INCIVEK (telaprevir), pegylated-interferon and ribavirin. These data support the initiation of the study to evaluate 12-week treatment regimens for certain patients.
Phase 2 Post-Transplant Study of INCIVEK (telaprevir)
- Earlier in 2011, Vertex completed a drug-drug interaction study of INCIVEK (telaprevir) dosed with immunosuppressive agents that are commonly used following a liver transplant. In the fourth quarter of 2011, Vertex plans to initiate a Phase 2 study in the U.S. of INCIVEK dosed in combination with pegylated-interferon and ribavirin in people with recurrent hepatitis C following a liver transplant.
VX-770 NDA and MAA Submissions Planned for Second Half of 2011
The ongoing Phase 3 program for VX-770, Vertex's cystic fibrosis
transmembrane conductance regulator protein (CFTR) potentiator, is
nearing completion. The Phase 3 STRIVE trial in people with CF aged 12
and older with at least one copy of the G551D mutation is complete, as
is the Phase 2 DISCOVER trial, which was primarily a safety study that
enrolled people aged 12 and older with two copies of the F508del
mutation. Vertex reported 48-week top-line data from STRIVE and
16-week top-line data from DISCOVER in
February 2011. In March 2011, Vertex also reported 24-week top-line data from the Phase 3 ENVISION trial in children with CF aged six to 11 with at least one copy of the G551D mutation. 48-week data from ENVISION are expected in mid-2011. Vertex is on track to submit global regulatory applications for approval in the United States, Canadaand Europe, including an NDA and MAA in the second half of 2011.
Vertex expects to present clinical data from the Phase 3 registration
program for VX-770 at the 34th
European Cystic Fibrosis Conference, which will take place in Hamburg, Germanyfrom June 8 to 11, 2011. The presentations are expected to include results from the Phase 3 STRIVE and Phase 2 DISCOVER trials as well as data related to in vitro studies of VX-770.
Phase 2 Trial Combining Two CFTR Modulators for the Treatment of People with the Most Common Mutation of Cystic Fibrosis
- Vertex is conducting an exploratory Phase 2a clinical trial to evaluate combination regimens of its lead CFTR modulators - VX-770 and VX-809, a CFTR corrector - in people with the most common mutation of CF, known as F508del. Part One of the trial is ongoing and is evaluating VX-809 (200 mg), or placebo, dosed alone for 14 days and in combination with VX-770 (150 mg or 250 mg), or placebo, for 7 days. Vertex expects to obtain interim data from Part One of the trial in the second quarter of 2011.
April 2011, Vertex announced a new collaboration with Cystic Fibrosis Foundation Therapeutics, Inc.(CFFT) to support development activities for VX-661, Vertex's second corrector to enter clinical development, and the accelerated discovery and development of next-generation correctors. CFFT is the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation(CFF). Vertex intends to begin a Phase 2 study of VX-661 by the end of 2011 and expects the study to enroll people with CF who have the F508del mutation.
Phase 2b Trial Planned for Second Half of 2011
- In March, Vertex announced results from a Phase 2a trial of VX-765 in treatment-resistant epilepsy. Based on these results, Vertex plans to initiate a larger and longer-duration Phase 2b study of VX-765 in people with treatment-resistant epilepsy as early as the fourth quarter of 2011.
Ongoing Phase 2 Study of JAK3 Inhibitor VX-509
Vertex recently completed enrollment in an ongoing Phase 2
proof-of-concept clinical trial of the JAK3 inhibitor VX-509 in people
with moderate to severe rheumatoid arthritis. The trial is evaluating
12 weeks of treatment with VX-509 dosed twice daily compared to
placebo. In the third quarter of 2011, Vertex expects to obtain
clinical data, including measurements of safety, tolerability and
clinical efficacy, as measured by
American College of Rheumatology (ACR) and Disease Activity Score(DAS) response criteria.
First Quarter Results
For the quarter ended
The non-GAAP loss for the quarter ended
Total revenues for the quarter ended
Research and development (R&D) expenses for the quarter ended
Sales, general and administrative (SG&A) expenses for the quarter ended
Net interest expense for the quarter ended
This section contains forward-looking guidance about the financial
Vertex is today reiterating its guidance for 2011 total operating
expenses, excluding costs of revenues and stock-based compensation
Non-GAAP Financial Measures
In this press release, Vertex's financial results and financial guidance
are provided both in accordance with accounting principles generally
2011 First Quarter Results
Three Months Ended
|Costs and expenses:|
|Research and development expenses (R&D)||158,612||143,012|
|Sales, general & administrative expenses (SG&A)||71,523||35,552|
|Total costs and expenses||233,561||182,711|
|Loss from operations||(159,899)||(160,282)|
|Net interest expense (Note 1)||(10,599)||(3,500)|
Change in fair value of derivative instruments (Note 1)
|Basic and diluted net loss per common share||$(0.87)||$ (0.83)|
|Basic and diluted weighted-average number of common shares outstanding||202,329||198,935|
Non-GAAP Loss and Loss per
Three Months Ended
|GAAP Net Loss||$(176,096)||$(165,271)|
|Pro Forma Adjustments:|
|Milestone revenues related to September 2009 financial transactions (Note 1)||$(50,000)||$---|
|Stock-based compensation expense included in R&D||$18,549||$14,320|
|Stock-based compensation expense included in SG&A||9,330||5,013|
Total stock-based compensation expense
Expenses related to September 2009 financial transactions (Note 1)
|Basic and diluted non-GAAP loss per common share||$(0.91)||$ (0.70)|
Note 1: 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, and
the liabilities related to milestone transactions reflected in the
Condensed Consolidated Balance Sheets Data, relate to two financial
transactions that the company entered into in September 2009 relating to
future milestone payments under the company's collaboration agreement
Note 2: 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
Note 3: On
Condensed Consolidated Balance Sheets Data
|Cash, cash equivalents and marketable securities||$823,452||$1,031,411|
|Inventories (Note 3)||17,816||---|
|Other current assets||31,706||25,628|
|Property and equipment, net||70,877||72,333|
|Intangible assets (Note 2)||518,700||518,700|
|Goodwill (Note 2)||26,102||26,102|
|Other non-current assets||15,723||17,182|
|Liabilities and Stockholders' Equity|
|Accrued restructuring expense||28,814||29,595|
|Deferred tax liability (Note 2)||160,278||160,278|
|Convertible notes (due 2015)||400,000||400,000|
|Liabilities related to milestone transactions (Note 1)||176,993||214,790|
|Total liabilities and stockholders' equity||$1,538,487||$1,725,446|
|Common shares outstanding||205,458||203,523|
Vertex creates new possibilities in medicine. Our team aims to discover, develop and commercialize 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, epilepsy and other life-threatening diseases.
Founded more than 20 years ago in
INCIVEKTM is a trademark of
PEGASYS® and COPEGUS® are registered trademarks of Hoffmann-La Roche.
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 statements
regarding (i) the expectation that the
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