Vertex Pharmaceuticals (VRTX) | Stock Discussion Forums

SiliconInvestor

sponsor

Sponsor

Biotech / Medical | Vertex Pharmaceuticals (VRTX)

Previous 10 | Next 10

From: tnsaf	3/29/2011 11:31:40 AM
	of 1169

Phase 3 Study of VX-770 in Children Ages 6 to 11 With a Specific Type of Cystic Fibrosis Showed Profound Improvements in Lung Function (FEV1) and Other Measures of Disease Through 24 Weeks -Mean relative improvement in lung function of 17.4% and mean absolute improvement of 12.5% from baseline compared to placebo in children treated with VX-770- - No discontinuations due to adverse events through 24 weeks - -Vertex on track to submit regulatory applications for the approval of VX-770 in the U.S. and Europe in the second half of 2011 - CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive results from a 24-week analysis of the ongoing Phase 3 ENVISION study of VX-770, an oral medicine in development that targets the defective protein that causes cystic fibrosis (CF). ENVISION (n=52) was designed to evaluate VX-770 among children ages 6 to 11 with the G551D mutation in the CFTR gene. Approximately 4 percent of people with CF in the United States have at least one copy of the G551D mutation. The study met its primary endpoint of mean absolute change from baseline in percent predicted FEV1 (forced expiratory volume in one second, or lung function) through week 24. A difference in mean absolute improvement from baseline in lung function of 12.5 percent and a difference in mean relative improvement from baseline in lung function of 17.4 percent compared to placebo (p<0.0001) were observed through week 24. Significant improvements in other measures of disease, including weight gain and a reduction in sweat chloride, were also observed through week 24 among those who received VX-770. At the time of this analysis, the most commonly reported adverse events were respiratory in nature and comparable across treatment groups. The study is ongoing, and additional assessments of key endpoints will be conducted at the end of the 48-week study. Vertex is on track to submit regulatory applications for approval in the United States and Europe in the second half of 2011. The submissions will be based on data from the Phase 3 STRIVE and ENVISION studies as well as the Phase 2 DISCOVER study. Vertex plans to submit data from all studies in the VX-770 Phase 3 registration program for presentation at upcoming medical meetings. "In this study, children with CF treated with VX-770 showed the same profound improvements in lung function seen in the recently-announced STRIVE study among an older group of people with the G551D mutation," said Robert Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical Officer for Vertex. "We are committed to making VX-770 available as soon as possible and are moving ahead quickly with our U.S. and European applications for regulatory approval that we plan to submit in the second half of this year." "The data announced today are highly encouraging because children with CF tend to be healthier than adults and significant improvements in lung function may be harder to demonstrate in a clinical study," said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. "Data from Phase 3 studies of VX-770 in people with the G551D mutation provide strong support for the concept that addressing the defective protein that leads to CF may significantly improve outcomes for patients and give hope for the future of other CF treatments that repair the basic defect of the disease." Vertex's medicines in development for CF were discovered as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) to discover and develop novel CF Transmembrane Conductance Regulator (CFTR) modulators. CFFT is the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. Vertex retains worldwide rights to develop and commercialize these potential medicines. Summary of Key Data from ENVISION In the ENVISION study, 52 children were enrolled and received VX-770 or placebo as a single 150 mg tablet every 12 hours. At the time of this analysis, the last patients had completed their week 40 visit. All children who remain on this study will be followed through 48 weeks. All children who complete 48 weeks of treatment in ENVISION, including those in the placebo group, and who meet eligibility criteria, may choose to receive VX-770 for up to an additional 96 weeks, or until VX-770 is approved, as part of an extension study called PERSIST. ENVISION was designed to evaluate VX-770 in children between the ages of 6 and 11 with at least one copy of the G551D CFTR mutation. The primary endpoint of the study was mean absolute change from baseline in percent predicted FEV1 through week 24. Lung function was assessed using a standard test that measures the amount of air a person can exhale in one second (forced expiratory volume in one second, or FEV1). Preliminary Efficacy Results Lung Function: Absolute and relative changes in lung function are being reported in today's announcement. Phase 3 results and product labeling for currently available CF medicines generally describe relative improvements in lung function. Baseline lung function (FEV1) in ENVISION was 84.7 percent predicted for children in the VX-770 treatment group and 83.7 percent predicted among those in the placebo group. Results of the ENVISION study showed that the difference in mean absolute improvement from baseline in lung function through 24 weeks in children treated with VX-770 was 12.5 percent (p<0.0001). The difference in mean relative improvement from baseline was 17.4 percent compared to placebo. Additional secondary endpoints were measured to observe the effect of VX-770 through week 24. These secondary endpoints included: Weight: Many people with CF have a hard time gaining and maintaining weight due to factors such as reduced pulmonary function, nutrition, chronic infection and inflammation. In the ENVISION study, those who received VX-770 experienced an average weight gain of approximately 8.1 lbs (3.7 kilograms) through 24 weeks compared to baseline while those in the placebo group gained approximately 3.9 lbs (1.8 kilograms) compared to baseline. Sweat Chloride: Elevated sweat chloride levels are a diagnostic hallmark of CF and a marker of CFTR protein dysfunction, which is the underlying molecular mechanism responsible for CF. The amount of chloride in the sweat is measured using a standard test. People with CF typically have sweat chloride levels in excess of 60 mmol/L, while normal values are less than 40 mmol/L. Reduction in sweat chloride is considered to be a marker of improved CFTR function. In ENVISION, the baseline sweat chloride level for both treatment groups was approximately 104 mmol/L. Significant decreases in measurements of sweat chloride were observed among those treated with VX-770 compared to those in the placebo group. Through week 24, mean sweat chloride level for individuals treated with VX-770 was below 60 mmol/L. Significant decreases in sweat chloride were not observed among those in the placebo group. Patient Reported Outcomes: The Cystic Fibrosis Questionnaire — Revised (CFQ-R) is a validated patient reported outcome tool that was used in this study to measure the impact of VX-770 on overall health, daily life, perceived well-being and symptoms. One aspect of the CFQ-R, referred to as the respiratory domain, assesses patient reported symptoms such as coughing, congestion, wheezing and other respiratory symptoms. In the ENVISION study, children responded to the CFQ-R and reported a trend toward improvement in respiratory symptoms, which was a key secondary endpoint of the study. Safety: At the time of this analysis, the most commonly reported adverse events were respiratory in nature and comparable between treatment groups. The most commonly reported adverse events through 24 weeks included cough, headache, CF pulmonary exacerbation, throat pain, and vomiting. Pulmonary exacerbations were uncommon in ENVISION regardless of treatment arm. There were no discontinuations due to adverse events in either treatment group of the study through 24 weeks. The ENVISION safety evaluation is ongoing through 48 weeks. About the Cystic Fibrosis Transmembrane Conductance Regulator Protein (CFTR) CF is caused by a genetic defect that results in defective or missing CF transmembrane conductance regulator (CFTR) proteins, which result in poor ion flow across cell membranes, including in the lungs, and the accumulation of abnormally thick, sticky mucus that leads to chronic lung infections and progressive lung damage. In people with the G551D mutation in the CFTR gene, CFTR proteins are present at the cell surface but do not function properly. VX-770, known as a CFTR potentiator, aims to increase the function of defective CFTR proteins by increasing the ability to transport ions across the cell membrane of CFTR at the cell surface. In people with the F508del mutation, CFTR proteins do not reach the cell surface in normal amounts. VX-809, known as a CFTR corrector, aims to increase CFTR function by increasing the movement of CFTR to the cell surface. Combination Study of VX-770 and VX-809 Vertex is conducting a Phase 2a clinical trial to evaluate multiple combination regimens of VX-770 and VX-809 in people with two copies of the F508del mutation. The first part of the study 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 seven days. Vertex expects to obtain data from this part of the trial in the first half of 2011. About Cystic Fibrosis CF is a life-threatening genetic disease affecting approximately 30,000 people in the United States and 70,000 people worldwide. Today, the median predicted age of survival for a person with CF is approximately 36 years. According to the 2008 Cystic Fibrosis Foundation Patient Registry Annual Data Report, approximately 4 percent of the total CF patient population in the U.S. have at least one copy of the G551D mutation, 48 percent of the total CF patient population in the United States have two copies of the F508del mutation and an additional 39 percent of the total CF patient population have one copy of the F508del mutation. People interested in further information about clinical trials of VX-809 or VX-770 should visit www.clinicaltrials.gov or cff.org

Share	Recommend \| Keep \| Reply \| Mark as Last Read

From: tnsaf	4/1/2011 12:05:02 AM
	of 1169

Results From Phase 3 REALIZE Study Showed Telaprevir-Based Therapy Significantly Improved SVR (Viral Cure) Rates in People Whose Prior Treatment For Hepatitis C Was Unsuccessful - All major subgroups achieved significantly higher viral cure rates with telaprevir-based therapy compared to pegylated-interferon and ribavirin: 86% vs. 24% in prior relapsers, 57% vs. 15% in prior partial responders and 31% vs. 5% in prior null responders - - No clinical benefit was observed in delaying telaprevir therapy by four weeks (lead-in) compared to starting telaprevir, pegylated-interferon and ribavirin simultaneously - - Safety and tolerability results were consistent with prior Phase 3 studies of telaprevir - BERLIN--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced final results from its pivotal Phase 3 REALIZE study that evaluated people with genotype 1 chronic hepatitis C whose prior treatment with pegylated-interferon and ribavirin was unsuccessful either because they relapsed, had a partial response or had a null response. Data from the study showed that people in each of these subgroups who were treated with telaprevir-based combination therapy achieved superior rates of sustained viral response (SVR, or viral cure) compared to those treated with pegylated-interferon and ribavirin alone. REALIZE also evaluated whether viral cure rates could be further improved by delaying the start of telaprevir by four weeks, during which time patients received four weeks of pegylated-interferon and ribavirin alone (lead-in), compared to a simultaneous start. The data showed no clinical benefit to a lead-in for people treated with telaprevir-based combination therapy. Safety and tolerability results were consistent with results from the prior Phase 3 studies of telaprevir. These data were presented today at The International Liver Congress™ 2011, 46th annual meeting of the European Association for the Study of the Liver (EASL) in Berlin, Germany. REALIZE was conducted by Vertex's collaborator, Tibotec BVBA. "Patients with chronic hepatitis C who undergo re-treatment with currently available medicines rarely achieve a viral cure and remain at an increased risk for advancing to more serious liver disease," said Stefan Zeuzem, M.D., Professor of Medicine and Chief of the Department of Medicine at the JW Goethe University Hospital, Frankfurt, Germany and principal investigator for REALIZE. "These data are important because they showed that viral cure rates were three to six times higher for patients treated with a telaprevir-based regimen compared to re-treatment with currently available medicines." Among those in the simultaneous start arm of REALIZE, 83 percent (121/145) of prior relapsers, 59 percent (29/49) of prior partial responders and 29 percent (21/72) of null responders achieved viral cures compared to 24 percent (16/68), 15 percent (4/27) and 5 percent (2/37), respectively, who received pegylated-interferon and ribavirin. The viral cure rates among those in the lead-in arm were 88 percent (124/141) among prior relapsers, 54 percent (26/48) among prior partial responders and 33 percent (25/75) among prior null responders. In a combined endpoint analysis of the two telaprevir-based treatment arms, 86 percent (245/286) of prior relapsers, 57 percent (55/97) of prior partial responders and 31 percent (46/147) of prior null responders achieved viral cures. "The REALIZE results are encouraging, especially considering people in this study had been unsuccessfully treated in the past and many already had scarring of the liver," said Robert Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical Officer for Vertex. "Rates of viral cure among those treated with telaprevir-based regimens were similar between the simultaneous and delayed start arms of the study, supporting the conclusion that there was no clinical benefit to a lead-in strategy with telaprevir." In this study, 48 percent (316/662) of patients overall had advanced liver fibrosis or cirrhosis (scarring of the liver) and 89 percent (586/662) of patients overall had high amounts of hepatitis C virus (high viral load; HCV RNA = 800,000 IU/mL) upon study entry. Summary of REALIZE Results REALIZE is the only Phase 3 hepatitis C study to date of a direct-acting antiviral medicine in development that was designed to evaluate people whose prior treatment was unsuccessful, including those who had a null response. In this study, patients were randomized 2:2:1 to two telaprevir-based treatment arms (simultaneous or lead-in) or a control arm of 48 weeks of pegylated-interferon and ribavirin alone. Patients in the telaprevir treatment arms received a total of 12 weeks of telaprevir-based combination therapy. In the lead-in arm, patients received four weeks of pegylated-interferon and ribavirin followed by telaprevir in combination with pegylated-interferon and ribavirin for 12 weeks followed by 32 weeks of pegylated-interferon and ribavirin alone. For those in the simultaneous start arm, the telaprevir-based combination was followed by an additional 36 weeks of pegylated-interferon and ribavirin alone. The primary endpoint of the REALIZE study was SVR in each of the two telaprevir treatment arms compared to the control arm and for the three groups of people included in the study. The total treatment time for all patients in REALIZE was 48 weeks. REALIZE SVR Results % (n) Prior Relapsers Prior Partial Responders Prior Null Responders (n=354)* (n=124)* (n=184)* TVR-based 83% 59% 29% Simultaneous Start Arm+ (121/145) (29/49) (21/72) TVR-based 88% 54% 33% Lead-In Arm++ (124/141) (26/48) (25/75) Control Arm+++ 24% 15% 5% (16/68) (4/27) (2/37) The SVR rates observed were statistically significant when compared with the control arm (p <0.001). +Simultaneous start: 12 weeks of telaprevir (750 mg, q8h), Pegasys® (PEG, pegylated-interferon alfa-2a) & Copegus® (RBV, ribavirin), followed by 36 weeks of PEG & RBV alone. ++Lead-in: 4 weeks of PEG & RBV alone followed by 12 weeks of telaprevir (750 mg, q8h), PEG & RBV, followed by 32 weeks of PEG & RBV alone. There was no clinical benefit with the use of a four-week lead in with no significant improvement in SVR rates and no significant reduction in virologic failure and relapse rates in the lead-in start arm compared to the simultaneous start arm. +++Control: 12 weeks of placebo, PEG & RBV, followed by 36 weeks of Peg & RBV alone. Prior Relapser: Defined as a person whose hepatitis C virus was undetectable at the completion of at least 42 weeks of a prior course of therapy but whose virus became detectable during the follow-up period. Prior Partial Responder: Defined as a person who achieved at least a 2 log in10 reduction in HCV RNA at week 12, but whose hepatitis C virus never became undetectable by week 24 of a prior course of therapy. Prior Null Responder: Defined as a person who achieved a less than 2 log10 reduction in HCV RNA at week 12 of a prior course of therapy. Safety and Tolerability Information for the Phase 3 Studies of Telaprevir* The safety and tolerability results of the telaprevir-based combination regimens were consistent across the Phase 3 studies. The most common adverse events were fatigue, pruritus, nausea, headache, rash, anemia, flu-like symptoms, insomnia and diarrhea with the majority being mild to moderate. Rash and anemia occurred more frequently in the telaprevir-based treatment arms compared to the control group. Rash was primarily characterized as eczema-like, manageable and resolved upon stopping telaprevir. More than 90 percent of rash was mild to moderate and primarily managed with the use of topical corticosteroids and/or antihistamines. Anemia was primarily managed by reducing the dose of ribavirin. To optimize each patient's opportunity to achieve viral cure in the Phase 3 studies, sequential discontinuation of the medicines was recommended as a strategy to manage certain adverse events. This strategy allowed patients to continue on pegylated-interferon and ribavirin after stopping telaprevir. Discontinuation of all medicines due to either rash or anemia during the telaprevir/placebo treatment phase was 1 percent to 3 percent in the telaprevir treatment arms. About the Study REALIZE was a pivotal Phase 3, randomized, double-blind, placebo-controlled, global study. The majority of clinical trial sites were in Europe. The study was designed to evaluate the efficacy, safety and tolerability of telaprevir-based combination regimens in people infected with genotype 1 chronic hepatitis C who did not achieve a viral cure after at least one course of prior treatment with interferon-based therapy. Status of Telaprevir Regulatory Applications The regulatory applications for the approval of telaprevir have been granted Priority Review by the U.S. Food and Drug Administration (FDA) and Health Canada and accelerated assessment by the European Medicines Agency for the treatment of people with genotype 1 chronic hepatitis C. The FDA has scheduled its Antiviral Drugs Advisory Committee to discuss the New Drug Application for telaprevir on April 28, 2011. A target response date of May 23, 2011 is set under the Prescription Drug User Fee Act (PDUFA). The applications include data from three registration studies, ADVANCE, ILLUMINATE and REALIZE, which evaluated telaprevir in combination with pegylated-interferon and ribavirin in people with hepatitis C who were new to treatment as well as those who did not achieve a viral cure after treatment with currently available medicines. For complete information on the telaprevir clinical trials or a fact sheet on the trial designs visit: www.vrtx.com/press.cfm. About the Telaprevir Development Program Telaprevir is an investigational, oral inhibitor that acts directly on the HCV protease, an enzyme essential for viral replication. To date, more than 2,500 people with hepatitis C have received telaprevir-based therapy as part of Phase 2 studies and the Phase 3 ADVANCE, ILLUMINATE and REALIZE studies. Together, these studies enrolled people with genotype 1 chronic hepatitis C who had not been treated for their disease previously as well as people who had been treated before but did not achieve a viral cure. Vertex is developing telaprevir in collaboration with Tibotec BVBA and Mitsubishi Tanabe Pharma. Vertex has rights to commercialize telaprevir in North America. Through its affiliate, Janssen, Tibotec has rights to commercialize telaprevir in Europe, South America, Australia, the Middle East and certain other countries. Mitsubishi Tanabe Pharma has rights to commercialize telaprevir in Japan and certain Far East countries. About Hepatitis C Hepatitis C is a serious liver disease caused by the hepatitis C virus, which is spread through direct contact with the blood of infected people and ultimately affects the liver.1 Chronic hepatitis C can lead to serious and life-threatening liver problems, including liver damage, cirrhosis, liver failure or liver cancer.1 Though many people with hepatitis C may not experience symptoms, others may have symptoms such as fatigue, fever, jaundice and abdominal pain.1 Approximately 60 percent of people who undergo treatment with an initial 48-week regimen with pegylated-interferon and ribavirin, the currently approved medicines for genotype 1 hepatitis C, do not achieve SVR,2,3,4 or viral cure.5 If treatment is not successful and a person does not achieve a viral cure, they remain at an increased risk for progressive liver disease.6,7 More than 170 million people worldwide are chronically infected with hepatitis C.5 In the United States, nearly 4 million people have chronic hepatitis C and 75 percent of them are unaware of their infection.8 The majority of people with hepatitis C in the United States were born between 1946 and 1964, accounting for two of every three people with chronic hepatitis C.9 Hepatitis C is the leading cause of liver transplantations in the United States and is reported to contribute to 4,600 to 12,000 deaths annually.10,11 By 2029, total annual medical costs in the United States for people with hepatitis C are expected to more than double, from $30 billion in 2009 to approximately $85 billion.9 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 date of the scheduled meeting of the FDA's Antiviral Advisory Committee and (ii) the FDA's target review date for the telaprevir NDA. While the company 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 Vertex could experience unforeseen delays in obtaining approval to market telaprevir; that there may be varying interpretations of the data from the telaprevir clinical trials; that future outcomes from clinical trials of telaprevir may not be favorable; that future scientific, clinical, competitive or other market factors may adversely affect the potential for telaprevir-based therapy and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through Vertex's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. About Vertex 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 Cambridge, MA, we now have ongoing worldwide research programs and sites in the U.S., U.K. and Canada. For more information and to view Vertex's press releases, please visit www.vrtx.com. (VRTX - GEN) 1 Centers for Disease Control and Prevention. Hepatitis C Fact Sheet: CDC Viral Hepatitis. Available at: cdc.gov Accessed March 21, 2011. 2 Manns MP, McHutchison JG, Gordon SC, et al. Peginterferon alfa-2b plus ribavirin compared with interferon alfa-2b plus ribavirin for initial treatment of chronic hepatitis C: a randomised trial. Lancet. 2001;358:958-965. 3 Fried MW, Shiffman ML, Reddy KR, et al. Peginterferon alfa-2a plus ribavirin for chronic hepatitis C virus infection. N Engl J Med. 2002;347:975-982. 4 McHutchison JG, Lawitz EJ, Shiffman ML, et al; IDEAL Study Team. Peginterferon alfa-2b or alfa-2a with ribavirin for treatment of hepatitis C infection. N Engl J Med. 2009;361:580-593. 5 Ghany MG, Strader DB, Thomas DL, Seeff, LB. Diagnosis, management and treatment of hepatitis C; An update. Hepatology. 2009;49 (4):1-40. 6 Morgan TR, Ghany MG, Kim HY, Snow KK, Lindsay K, Lok AS. Outcome of sustained virological responders and non-responders in the Hepatitis C Antiviral Long-Term Treatment Against Cirrhosis (HALT-C) trial. Hepatology. 2008;50(Suppl 4):357A (Abstract 115). 7 Veldt BJ, Heathcote J, Wedmeyer H. Sustained virologic response and clinical outcomes in patients with chronic hepatitis C and advanced fibrosis. Annals of Internal Medicine. 2007; 147: 677-684. 8 Institute of Medicine of the National Academies. Hepatitis and liver cancer: a national strategy for prevention and control of hepatitis B and C. Colvin HM and Mitchell AE, ed. Available at: iom.edu Updated January 11, 2010. Accessed March 21, 2011. 9 Pyenson B, Fitch K, Iwasaki K. Consequences of hepatitis C virus (HCV): Costs of a baby boomer epidemic of liver disease. Available at: natap.org Updated May 2009. Accessed March 21, 2011. This report was commissioned by Vertex Pharmaceuticals, Inc. 10 Volk MI, Tocco R, Saini S, Lok, ASF. Public health impact of antiviral therapy for hepatitis C in the United States. Hepatology. 2009;50(6):1750-1755. 11 Davis GL, Alter MJ, El-Serag H, Poynard T, Jennings LW. Aging of hepatitis C virus (HCV)-infected persons in the United States: A multiple cohort model of HCV prevalence and disease progression. Gastroenterology. 2010;138:513-521. Vertex Pharmaceuticals Incorporated Media: Dawn Kalmar Amy Pasqua Zachry Barber 617-444-6992 or mediainfo@vrtx.com or Investors: Michael Partridge, 617-444-6108 or Lora Pike, 617-444-6755 or Matthew Osborne, 617-444-6057 Source: Vertex Pharmaceuticals Incorporated

Share	Recommend \| Keep \| Reply \| Mark as Last Read

From: tnsaf	4/15/2011 11:31:24 PM
	of 1169

Vertex and Cystic Fibrosis Foundation Therapeutics to Collaborate on Discovery and Development of New Medicines to Treat the Underlying Cause of Cystic Fibrosis -Expanded collaboration supports development of a second corrector, VX-661, and accelerated discovery and development of next-generation correctors- -Phase 2 study of VX-661 planned for 2011 in people with CF who have the F508del mutation- CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) today announced they will collaborate on the continued discovery and development of new medicines known as correctors that aim to treat the underlying cause of cystic fibrosis (CF) in people with the most common form of the disease. The expanded collaboration will support development activities for VX-661, Vertex's second corrector to enter clinical development, and the accelerated discovery and development of next-generation correctors. Vertex plans to begin the first study of VX-661 by the end of 2011 in people with CF who have the F508del mutation. Vertex and CFFT, a nonprofit drug discovery and development affiliate of the CF Foundation, began their collaborative research and development efforts in 1998, and to date, three potential new medicines, known as CFTR modulators, have resulted from the collaboration — the potentiator VX-770 and the correctors VX-809 and VX-661. Correctors and potentiators are medicines in development that aim to treat the underlying cause of CF by improving the function of the defective protein known to cause the disease. Vertex retains worldwide rights to develop and commercialize these potential medicines. "The CF Foundation is widely recognized by doctors, nurses, scientists and those with CF as a driving force in the search for new CF medicines, and we are pleased to further expand our strong collaboration with them," said Matthew Emmens, Chairman, President and Chief Executive Officer of Vertex. "The collaboration announced today underscores our commitment to CF and accelerates our efforts to develop new medicines as quickly as possible for people with the most common type of this disease. By advancing VX-809 and VX-661 in parallel, we hope to generate data to inform future studies of corrector regimens while continuing to invest in additional research for CF." "With the recently announced positive Phase 3 results for VX-770, we believe that - together with Vertex - we are on the right path to fundamentally change the treatment of CF by targeting the cause of the disease," said Robert J. Beall, Ph.D., President and CEO of the CF Foundation and CFFT. "This new collaboration is a milestone in our long-standing relationship with Vertex and provides for additional opportunities to accelerate our discovery efforts and to potentially improve treatment for people with the most common type of CF." CF is caused by defective or missing CF transmembrane conductance regulator (CFTR) proteins, which result in poor ion flow across cell membranes, including in the lungs, causing the accumulation of abnormally thick, sticky mucus that leads to chronic lung infections and progressive lung damage. In people with the F508del mutation, which is the most common CFTR mutation, CFTR proteins do not reach the cell surface in normal amounts. VX-809 and VX-661, known as CFTR correctors, aim to increase CFTR function by increasing the movement of CFTR to the cell surface. In people with the G551D mutation in the CFTR gene, CFTR proteins are present at the cell surface but do not function properly. VX-770, known as a CFTR potentiator, aims to increase the function of defective CFTR proteins by increasing their ability to transport ions across the cell membrane of CFTR at the cell surface. About The Collaboration The collaboration announced today is focused on development activities for VX-661 and on the accelerated discovery and development of additional correctors for the treatment of people with the F508del mutation. As part of the collaboration, CFFT will provide Vertex with up to $75 million to support research and development activities. Vertex expects to receive payments from CFFT as reimbursement for research and development activities over a period of five years beginning in 2011. The collaboration will help support the development of VX-661. 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. The collaboration will also help support the accelerated discovery and early development of next-generation correctors that aim to treat the underlying cause of CF in people with the F508del mutation. The majority of Vertex's CF research is conducted at Vertex's San Diego, CA research site, where each of Vertex's medicines in development for CF were discovered. Development activities for Vertex's CF medicines are being led by Vertex's research and development site in Cambridge, MA. Under the terms of the collaboration, CFFT is entitled to receive a royalty on future net sales of correctors developed as part of the research collaboration. As part of previous collaborations, CFFT is entitled to receive a royalty on future net sales of VX-770, VX-809 and VX-661. Vertex retains worldwide rights to VX-770, VX-809 and VX-661. Development Program for Vertex's CF Medicines Vertex recently reported positive results from two Phase 3 studies of VX-770 — the STRIVE trial in adolescents and adults with CF and the ENVISION trial in children ages 6 to 11. The Phase 3 program for VX-770 is focused on people with CF who have at least one copy of the G551D mutation. The results from the Phase 3 program will form the basis for Vertex's planned submission of applications for approval in the United States and Europe in the second half of 2011. Vertex is also conducting a Phase 2 clinical trial to evaluate different dose combinations of VX-809 and VX-770 in people with two copies of the F508del mutation. The first part of the study is designed to evaluate VX-809, or placebo, dosed alone for 14 days and in combination with VX-770, or placebo, for seven days. Vertex expects to obtain data from Part One of the trial in the first half of 2011. VX-661 is the third potential new medicine for CF to emerge from Vertex's CF research efforts. As a corrector, VX-661 aims to increase CFTR function by increasing the movement of CFTR to the cell surface. In in vitro studies, a combination of VX-661 and VX-770 resulted in greater CFTR activity, as compared to treatment with VX-661 alone. Vertex plans to initiate a Phase 2 study of VX-661 by the end of 2011. About Cystic Fibrosis CF is a life-threatening genetic disease affecting approximately 30,000 people in the United States and 70,000 people worldwide. Today, the median predicted age of survival for a person with CF is approximately 36 years. According to the 2008 Cystic Fibrosis Foundation Patient Registry Annual Data Report, approximately 4 percent of the total CF patient population in the United States have at least one copy of the G551D mutation, 48 percent of the total CF patient population in the United States have two copies of the F508del mutation and an additional 39 percent of the total CF patient population have one copy of the F508del mutation. People interested in further information about clinical trials of VX-809 or VX-770 should visit www.clinicaltrials.gov or cff.org Collaborative History with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) Vertex initiated its CF research program in 1998 as a part of a collaboration with CFFT, the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation. Vertex and CFFT expanded the agreement in 2000 and again in 2004, and in March 2006 entered into a collaboration for the accelerated development of VX-770. In addition to the development collaboration for VX-770, in January 2006 Vertex and CFFT entered into an expanded research collaboration to develop novel corrector compounds. As part of these collaborations, Vertex has received approximately $75 million from CFFT to date to support CF research and development efforts led by Vertex. About the Cystic Fibrosis Foundation The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, go to www.cff.org. Source: Vertex Pharmaceuticals Incorporated

Share	Recommend \| Keep \| Reply \| Mark as Last Read

Previous 10 | Next 10

Copyright/IP Policy

Copyright © 1995-2013 Knight Sac Media. All rights reserved.