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Vertex Announces Key Business Objectives To Support Planned Launch of Telaprevir in Hepatitis C and Continued Progress in Other Serious Diseases
-Hepatitis C: Submission of New Drug Application complete for telaprevir-
-Cystic Fibrosis: First Phase 3 data for VX-770 expected in first quarter 2011-
-Additional ongoing trials in HCV, CF, epilepsy and rheumatoid arthritis-
-Vertex enters 2011 with cash and cash equivalents position of more than
"2011 will be a landmark year for Vertex as we prepare for the expected launch of telaprevir in hepatitis C and advance other new therapies in development," said Mr. Emmens.
"Our commercial team is in place and prepared for the planned launch of telaprevir this year. We believe that telaprevir will dramatically change the treatment of hepatitis C and establish Vertex as a company capable of discovering, developing and launching transformative medicines to treat serious diseases.
"We are in a unique position, as just behind telaprevir is VX-770, a medicine in development that aims to treat the underlying cause of cystic fibrosis. We will soon obtain data from the Phase 3 registration program of VX-770 that may support the planned submission of a New Drug Application to the
"We also expect to receive important data from multiple ongoing Phase 2 trials this year, including those evaluating new combination regimens for hepatitis C and cystic fibrosis, which may provide further development opportunities," Mr. Emmens concluded.
Hepatitis C: Preparing for Launch of Telaprevir
Submission of New Drug Application Completed in
November 22, 2010, Vertex completed the submission of its New Drug Application (NDA) for telaprevir to the United States Food and Drug Administration ( FDA). A response from the FDA regarding the company's request for Priority Review of the telaprevir NDA is expected this month. The FDA's goal for completion of its review for NDA submissions granted Priority Review status is six months from the NDA submission date.
December 2010, Vertex's collaborator, Janssen-Cilag International NV, submitted a Marketing Authorization Application (MAA) for telaprevir to the European Medicines Agency (EMA). The EMA accepted telaprevir for accelerated assessment, which is granted to new medicines of major public health interest.
- To support the planned launch of telaprevir, Vertex has hired more than 200 new employees into its expanding commercial function. The sales and commercial leadership team is in place, and more than 100 field-based employees have been hired to date and are prepared to support the future use of telaprevir across
the United States following the planned launch.
- Patient enrollment is ongoing in a Phase
3b clinical trial to evaluate twice-daily dosing of telaprevir (1,125 mg; BID) compared to three-times-daily dosing of telaprevir (750 mg; q8h) in combination with pegylated-interferon and ribavirin for people with genotype 1 hepatitis C. The study, known as OPTIMIZE, is the first Phase 3 study to evaluate twice-daily dosing of a protease inhibitor for the treatment of hepatitis C. The study 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 2012, which could potentially support the submission of a supplemental NDA for twice-daily (BID) dosing of telaprevir by the end of 2012.
Cystic Fibrosis: Phase 3
VX-770 NDA Submission Planned for Second Half of 2011
- Three trials of the novel cystic fibrosis transmembrane conductance regulator protein (CFTR) potentiator VX-770 are fully enrolled and ongoing as part of a global Phase 3 registration program focused on patients with the G551D mutation. The G551D mutation is present in approximately four percent of people with CF.
- The first Phase 3 data for VX-770 are expected in the first quarter of 2011 and will come from the Phase 3 STRIVE trial in people aged 12 and older with at least one copy of the G551D mutation. Data from 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, are also expected in the first quarter of 2011.
- Data from the Phase 3 ENVISION trial in people aged six to 11 with at least one copy of the G551D mutation are expected in mid-2011.
- If positive, the results from the Phase 3 program for VX-770 could support the submission of an NDA for VX-770 in the second half of 2011.
Opportunities to Further Advance Future Treatment of Hepatitis C and Cystic Fibrosis
Interim Data from Phase 2 Study of Telaprevir and VX-222 Expected in First Quarter of 2011
- Vertex is conducting a Phase 2 clinical trial evaluating multiple 12-week, response-guided regimens of telaprevir dosed in combination with its lead investigational HCV polymerase inhibitor, VX-222. The study currently includes three treatment arms. Two of the treatment arms are fully enrolled and are evaluating four-drug combinations of telaprevir (1,125 mg; BID), VX-222 (400 mg or 100 mg; BID), Pegasys® (pegylated-interferon alfa-2a) and Copegus® (ribavirin). Approximately two-thirds of the people in the four-drug treatment arms have received eight weeks or more of treatment. More than one-third of patients have received 10 weeks or more of treatment, with some people having completed all therapy. Interim data from both of the four-drug treatment arms are expected in the first quarter of 2011.
November 2010, Vertex announced the planned addition of a three-drug treatment arm to evaluate the potential of an all-oral, interferon-free regimen of telaprevir (1,125 mg), VX-222 (400 mg) and ribavirin dosed twice daily. Enrollment in this new treatment arm is anticipated to begin in the first quarter of 2011.
Additional Trials of Telaprevir to Advance Leadership Position in Hepatitis C
- Vertex and Tibotec also plan to conduct several additional clinical trials of telaprevir in 2011 that aim to expand the future patient population for telaprevir-based regimens. These trials include:
- Phase 3 HCV/Human Immunodeficiency Virus Co-Infection Trial: Vertex recently completed enrollment in a Phase 2 clinical trial of telaprevir-based regimens in people who are infected with genotype 1 hepatitis C virus and the human immunodeficiency virus (HIV), also known as HCV-HIV co-infection. If positive, results from this trial could support the planned initiation of a Phase 3 study of telaprevir-based regimens in people co-infected with HCV and HIV in 2011. 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 Short-Duration Treatment Study: Also in 2011, Vertex and Tibotec plan to initiate a clinical trial to evaluate the role of telaprevir as part of hepatitis C treatment regimens involving less than six total months of therapy. One part of the trial may evaluate a telaprevir-based treatment regimen as short as 12 total weeks in duration for certain subsets of patients.
- Phase 2 Post-Transplant Study: Vertex recently completed a drug-drug interaction study of telaprevir with immunosuppressive agents commonly used following a liver transplant. Based on results from this study, Vertex and Tibotec plan to initiate in 2011 a Phase 2 study of telaprevir-based regimens in people with recurrent hepatitis C following a liver transplant.
Combination of Two CFTR Modulators for the Treatment of People with the Most Common Mutation of Cystic Fibrosis
- Vertex is conducting a Phase 2a clinical trial to evaluate multiple combination regimens of its lead CFTR Modulators - VX-770, a CFTR potentiator, and VX-809, a CFTR corrector - in people with the most common mutation of CF, known as F508del. Enrollment is ongoing in Part One of the trial, which is designed to evaluate 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 first half of 2011.
Additional Phase 2 Trials and Continued Productivity in Research for Other Serious Diseases
Data from Phase 2 Trials in Epilepsy and Rheumatoid Arthritis Expected in 2011
- Vertex recently completed a Phase 2 trial of the novel caspase-1 inhibitor VX-765 in people with epilepsy. The double-blind, randomized, placebo-controlled trial was designed to evaluate the safety, tolerability and clinical activity of VX-765. The primary endpoints of the trial were safety and tolerability. Analyses of the data are ongoing and Vertex expects to announce top-line data from the trial in the first quarter of 2011.
- In the first quarter of 2011, Vertex expects to complete 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. 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 scores (ACR) and Disease Activity Scores (DAS).
- 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 2011.
Vertex anticipates a GAAP net loss for 2010, including certain charges, of approximately
Vertex will report full-year 2010 financial results on
Non-GAAP Financial Measures
In this press release, Vertex's financial results are provided both in accordance with accounting principles generally accepted in
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 in the second through fifth paragraphs of the press release, and statements regarding (i) Vertex's planned launch of telaprevir in 2011; (ii) the expectation that the first Phase 3 data for VX-770 will be available in the first quarter of 2011; (iii) the expectation that Vertex will obtain a response from the
Vertex creates new possibilities in medicine. Our team aims to discover and develop 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.
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