Once diagnosed with cancer, what a patient needs is morale support from friends and family to cope with their overwhelming grief, fear and confusion.

ILLUSTRATION: CHOOPONG EAMORAPHAN

“But what they need most is self morale. It’s very unfortunate that many of us died because they gave up too early,” said cancer patient Somsak (not his real name).

After learning that he had advanced liver cancer, Somsak said he broke down, cried and cried.

Somsak knew he had the hepatitis B virus 10 years ago. After that, he noticed he began to experience chronic fatigue and unexplained weight loss. his dark orange-coloured urine also worried him.

In addition, he suffered acute abdominal pain. An ultrasound screening detected an abdominal mass in the upper right area of his body. A CT scan and biopsy later confirmed the diagnosis of liver cancer.

“I was shocked when my doctor told me that the cancer was in advanced stage. And that I might only be able to live six more months.”

Never saying die, Somsak decided to join “the Advanced Liver Cancer Patient Assistance Programme” following his doctor’s suggestion. The programme gives patients cancer oral medicine support.

“I’m lucky because I have responded relatively well to the medication.” he said.

After two years of medication, Somsak continues to fight bravely against the deadly disease despite some adverse side effects from the medication.

“It is as good as it gets,” he said. “Apart from medication, I believe that positive thinking and good emotional health is equally important to fight cancer. we need to be strong and brave if we want to live longer.”

The liver cancer that Somsak has is called hepatocellular carcinoma (HCC), explained Dr Thiravud Khuhaprema, chairman of the National Cancer Institute Foundation. this form of liver cancer is closely linked to chronic hepatitis B and C. it is the most common type of liver cancer that begins in the cells of the liver itself.

“These patients are often found to have a history of viral hepatitis B or C infection. HCC is one of the main cancers that causes death in Thailand and chronic hepatitis B is found in 80% of these cases,” said Dr Thiravud. “The infection can cause continual damage to the liver and develop into cirrhosis and cancer.”

He said the frequency of liver cancer is highest in developing countries and quite rare in developed countries. Each year, it is estimated that there are about 350,000 new cases of liver cancer. And two-thirds of the cases are found in countries in Asia. Mongolia is ranked first and Thailand fifth.

In Thailand, liver cancer is the most common cancer in males. it is the third most frequent cancer in females, but it’s the number one cause of death among female patients. The incidence rate and the mortality rate of liver cancer in Thailand are steadily rising.

Assoc Prof Narin Voravud, an expert in medical oncology from Chulalongkorn University Hospital pointed out that the relative high rates of HCC in Thailand is reflected in the high frequency of hepatitis B infection.

“One person in every 12 people suffers from hepatitis B infection in Thailand,” he said. Meanwhile, the number of people with viral hepatitis C infection has been on the increase.

Heavy drinking is also another important cause of liver cancer in Thailand, he noted. The chance for liver cancer is even higher if the drinkers have snacks that contain alfatoxin, a dangerous mould in foodstuffs commonly found in peanuts, wheat and corns that are stored in a wet or humid environment. It’s odourless, colourless and tasteless.

“Most Thai people drink alcohol paired with peanuts. These combined factors may trigger the development of HCC,” said Dr Narin. 

HCC liver cancer often does not show any symptoms in its early stage. like Somsak, patients with advanced stage may experience fatigue, loss of appetite, weight loss, yellowing of the skin and the eyes, abdominal pain (particularly in the upper right area where the liver is present).

Primary liver cancer that affects just a small part of the liver is often treated with surgery to remove the cancerous tumour and the surrounding liver tissues to preserve the important areas of the liver for normal body function. it is possible for patients to suffer from the recurrence of the disease, however.

“Only 10-20% of HCC can be completely removed using surgery. And the recurrent rate is about 50-60%,” Dr Narin said.

Another treatment option involves inserting particles into the liver artery to block the flow of blood to the tumour. Liver transplants to replace the diseased liver can cure the disease but a living donor graft and matching of organ donors is not easy to find.

It is unfortunate that patients with HCC often suffer from liver cancer, viral hepatitis B infection and cirrhosis of the liver at the same time, he said.

It is not uncommon that patients with liver cancer die shortly after the diagnosis. if the cancer cannot be completely removed, the decline is rapid. Patients may usually continue to live only three to six months.

“Treating patients with liver cancer can be very challenging as we have to deal with three conditions simultaneously,” Dr Narin said.

Most chemotherapy drugs are not effective to liver cancer. And chemotherapy has not been shown to help patients live longer, he said. The liver often cannot tolerate radiation which harms healthy liver tissues.

“The good news is that the patients who cannot be treated with surgery can now resort to new liver cancer drugs,” he said. this new medication tackles the cancerous cells and tumour blood vessels directly.

A liver tumour needs new blood vessels for it to grow. since this medication blocks the growth of new blood vessels, the growth of the tumour is then stopped. The possible side effects include diarrhoea, skin rash and high blood pressure.

In clinical trials overseas, the drug succeeded in extending the lives of patients who cannot be treated with surgery and injection by 44 % one year when compared to 33% of those who received a placebo, said Dr Narin.

In Thailand, a clinical trial involving 140 Thai patients with advanced liver cancer showed that the patients continued to live up to 14 months.

“An improvement of survival probability of four months in Thai patients means that the drug is effective with Thai patients,” said Dr Narin.

The bad news is that this new medication is extremely expensive. it costs about 250,000 baht a month for the drugs.

“This is why the patients with advanced liver cancer may consider joining the Advanced Liver Cancer Patient Assistance Programme,” said Dr Thiravud.

The programme which requires participants to pay for the medicine for the first three months before they are accepted, is a collaboration between the National Cancer Institute Foundation and the country’s leading pharmaceutical company. since its inception in 2009, more than 170 patients have participated in the programme.

“But prevention is better than cure,” stressed Dr Thiravut. to keep liver cancer at bay, people should be vaccinated for hepatitis B. Meanwhile, patients with viral hepatitis B should also be periodically monitored for HCC.”

For more on the Advanced Liver Cancer Patient Assistance Programme, call the National Cancer Institute Foundation at 02 354 7025 ext.1221.

Latest stories in this category:

February 23, 2011 07:00 AM Eastern Daylight Time 

- Relative mean improvement in lung function of approximately 17% from baseline compared to placebo achieved by people treated with VX-770; mean absolute improvement from baseline of approximately 10.5% compared to placebo; both measures through 24 and 48 weeks -

- Significant improvements in all key secondary endpoints for VX-770; patients were 55% less likely to experience a pulmonary exacerbation, had significant reductions in sweat chloride and, on average, gained nearly 7 pounds -

- Discontinuations due to adverse events were less frequent among people treated with VX-770 -

- Data support Vertex plan to submit U.S. and European regulatory applications for approval in the second half of 2011 -

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive results from the Phase 3 STRIVE study of VX-770, an oral medicine in development that targets the defective protein that causes cystic fibrosis (CF). STRIVE was designed to evaluate people with a mutation in the CF gene known as G551D. in this study, profound improvements in lung function (forced expiratory volume in one second, or FEV1) were observed through week 24, and sustained through week 48, among those who received VX-770 (n=83) compared to those treated with placebo (n=78). Significant improvements in all key secondary endpoints were also observed through week 48 among those who received VX-770.

“Based on the results of DISCOVER, we continue to believe the combination of a potentiator and corrector may be the best approach to treating people with two copies of the F508del mutation”

Data from the study showed a mean absolute improvement in lung function from baseline compared to placebo through week 24 of 10.6 percent among those treated with VX-770 (p<0.0001). Mean absolute improvement in lung function among those treated with VX-770 was 10.5 percent through week 48 (p<0.0001).

the primary endpoint of the study was mean absolute change from baseline compared to placebo in percent predicted FEV1 (lung function) through week 24. Data from the study showed a mean absolute improvement in lung function from baseline compared to placebo through week 24 of 10.6 percent among those treated with VX-770. Mean absolute improvement in lung function among those treated with VX-770 was 10.5 percent through week 48.

Highly statistically significant improvements in key secondary endpoints in this study were also reported through week 48. Compared to those treated with placebo, people who received VX-770 were 55 percent less likely to experience a pulmonary exacerbation (periods of worsening in signs and symptoms of the disease requiring treatment with antibiotics) and, on average, gained nearly seven pounds (3.1 kilograms) through 48 weeks. there was a significant reduction in the amount of salt in the sweat (sweat chloride) among people treated with VX-770 in this study. Increased sweat chloride is a diagnostic hallmark of CF. Sweat chloride is a marker of CFTR protein dysfunction, which is the underlying molecular mechanism responsible for CF. People who received VX-770 also reported having fewer respiratory symptoms.

Adverse events that were 5 percent greater among those treated with VX-770 compared to placebo were headache, upper respiratory tract infections, nasal congestion, rash, dizziness and bacteria in the sputum. the most commonly reported serious adverse events included pulmonary exacerbation (13 percent in the VX-770 group compared to 33 percent in the placebo group) and hemoptysis (or bloody cough; 1 percent in the VX-770 group and 5 percent in the placebo group). Discontinuations through 48 weeks due to adverse events were less frequent in the VX-770 treatment group compared to placebo (1 percent compared to 5 percent).

“Treating the underlying cause of cystic fibrosis with VX-770 led to clinical improvements that were far beyond our expectations, providing support for an entirely new approach to the treatment of this disease,” said Peter Mueller, Ph.D., Executive Vice President, Global Research and Development, and Chief Scientific Officer for Vertex. “All primary and key secondary outcome measures in this study supported VX-770 over placebo. Patients’ lung function improved, they gained weight, experienced fewer respiratory symptoms and felt substantially better. Due to the significance of these data and the great need for new, more effective medicines, we will work with regulatory agencies to determine the fastest way to get VX-770 approved for people with this specific type of CF.”

“The results from STRIVE are highly encouraging for the CF community and provide scientific evidence supporting our long-standing belief that targeting the underlying defect of CF may have a profound effect on the disease,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “We have much more to do to eliminate this disease, but these data are extremely exciting, especially for people with the G551D mutation and their families. They also offer significant hope that a similar approach to treatment may help others living with CF.”

all patients who completed 48 weeks of treatment in STRIVE (n=144), including those in the placebo group, were eligible to receive VX-770 as part of an extension study called PERSIST. all patients (n=77) who completed dosing in the VX-770 arm and all but one patient (n=67) in the placebo arm chose to enroll in the extension study and receive VX-770 for up to an additional 96 weeks or until VX-770 is approved.

STRIVE is one of three studies in the VX-770 registration program. Vertex plans to submit data from STRIVE for presentation at an upcoming medical meeting. the registration program for VX-770 also includes two other studies, the Phase 2 DISCOVER study and Phase 3 ENVISION study. Data from DISCOVER were also reported today. Data from ENVISION are expected in mid-2011. Vertex plans to submit regulatory applications for approval in the United States and Europe in the second half of 2011.

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 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 STRIVE

in the STRIVE study, 161 people were enrolled and received at least one dose of either VX-770 as a single 150 mg tablet or placebo twice daily. the study was designed to evaluate VX-770 in people with at least one copy of the G551D CFTR mutation. the primary endpoint of the study was mean absolute change from baseline in predicted FEV1 (lung function) 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. the primary endpoint of STRIVE was mean absolute improvement from baseline. Phase 3 results and product labeling for currently available CF medicines generally describe relative improvements in lung function.

Baseline lung function in STRIVE was 63.5 percent predicted for patients in the VX-770 treatment group and 63.7 percent predicted among those in the placebo control group. Results of the STRIVE study showed that people treated with VX-770 achieved a mean absolute improvement from baseline compared to placebo of 10.6 percent through 24 weeks (p<0.0001). Mean absolute improvement in lung function achieved by people who received VX-770 was sustained through 48 weeks (10.5 percent; p<0.0001).

in addition, people treated with VX-770 experienced a 16.7 percent relative mean improvement in lung function from baseline compared to placebo (p<0.0001) through week 24, which was sustained through week 48 (16.9 percent; p<0.0001).

Additional secondary endpoints were measured to observe the effect of VX-770 through week 48. These secondary endpoints included:

Pulmonary Exacerbations: People treated with VX-770 in STRIVE were 55 percent less likely to experience a pulmonary exacerbation compared to those treated with placebo through week 48. through 48 weeks, 67 percent of people treated with VX-770 were exacerbation free compared to 41 percent of people treated with placebo.

Weight: Many people with CF have a hard time gaining and maintaining weight due to factors such as nutrition, chronic infection and inflammation. in the STRIVE study, those who received VX-770 experienced an average weight gain of approximately 6.8 lbs (3.1 kilograms) at 48 weeks compared to baseline. those in the placebo group gained approximately 0.9 lbs (0.4 kilograms).

Sweat Chloride: Elevated sweat chloride levels are a diagnostic hallmark that occur in all people with CF and result directly from defective CFTR activity in epithelial cells in the sweat duct. the amount of chloride in the sweat is measured using a standard test. People with CF typically have elevated 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 STRIVE, the baseline sweat chloride level for both treatment groups was approximately 100 mmol/L. Statistically significant decreases in measurements of sweat chloride were observed among those treated with VX-770 but not those treated with placebo. through week 48, mean sweat chloride levels for patients treated with VX-770 were below 60 mmol/L.

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, addresses patient reported symptoms including things such as coughing, congestion, wheezing and other respiratory symptoms. in this study, statistically significant and clinically meaningful improvements in respiratory symptoms (a secondary endpoint of the study) were reported among patients who received VX-770.

DISCOVER Data

Vertex also announced today the results of the Phase 2 DISCOVER study, which was primarily designed to provide additional safety data for VX-770 and is part of the registration program. DISCOVER enrolled 140 people who had two copies of the F508del mutation, which prevents the CFTR protein from moving to its proper location at the cell surface. the majority of people with CF have at least one copy of the F508del mutation.

the primary endpoints of DISCOVER were safety and absolute change from baseline in lung function through 16 weeks. Adverse events were similar between the treatment groups. Adverse events that occurred more frequently (?5 percent) in the VX-770 treatment group compared to placebo were cough, nausea, rash and contact dermatitis. none of these events were serious or led to discontinuation of VX-770. Data from the DISCOVER study will be submitted for presentation at an upcoming medical meeting.

Mean baseline lung function (FEV1) was 79.7 percent predicted for people who received VX-770 compared to 74.8 percent predicted for patients in the placebo group. Results of the DISCOVER study showed that people treated with VX-770 achieved a mean absolute improvement from baseline compared to placebo of 1.6 percent through 16 weeks (p=0.25). the improvement was not statistically significant and was not considered clinically meaningful. Data from the study also showed a mean relative improvement in lung function from baseline compared to placebo of 2 percent through week 16. A mean reduction in sweat chloride of 2.9 mmol/L compared to placebo through 16 weeks was observed among those treated with VX-770. this improvement was statistically significant but small (p<0.04).

“Based on the results of DISCOVER, we continue to believe the combination of a potentiator and corrector may be the best approach to treating people with two copies of the F508del mutation,” said Robert Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical Officer for Vertex. “Data are anticipated later this year from the first study to evaluate the combination of VX-770 and VX-809 in this group of people with cystic fibrosis.”

ENVISION Study

in addition to the STRIVE and DISCOVER studies, a third study known as ENVISION is evaluating VX-770 in children 6 to 11 years old with CF who have at least one copy of the G551D mutation. Data from the ENVISION study are anticipated in mid-2011.

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 part one of the trial in the first half of 2011.

About the Cystic Fibrosis Transmembrane Conductance Regulator Protein (CFTR)

CF is caused by defective or missing CFTR proteins, which result in poor ion flow across cell membranes, including in the lung, and the accumulation of abnormally thick, sticky mucus that leads to chronic lung infections and progressive lung damage. in people with the G551D mutation, CFTR proteins are present on the cell surface but do not function normally. VX-770, known as a potentiator, aims to increase the function of defective CFTR proteins by increasing the gating activity, or ability to transport ions across the cell membrane, of CFTR once it reaches 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 amount of CFTR at the cell surface.

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 37 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 U.S. 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 clinicaltrials.gov or cff.org/clinicaltrials.

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. Vertex has received approximately $75 million from CFFT to support CF research and development efforts.

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.

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.

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) Vertex’s plan to submit U.S. and European regulatory applications for approval in the second half of 2011; (ii) the support provided by this study for an entirely new approach to treating cystic fibrosis; (iii) the plan to work with regulatory agencies to determine the fastest way to get VX-770 approved; (iv) the hope that a similar approach to treatment, targeting CFTR protein dysfunction caused by mutations other than the G551D mutation, may help others living with CF; (v) the planned presentation of data from STRIVE and DISCOVER at upcoming medical meetings; (vi) the expectation that data from ENVISION will be available in mid-2011; and (vii) our belief that a combination of a potentiator and corrector may be the best approach to treating people with two copies of the F508del mutation and the expectation that data from the ongoing Phase 2a combination study of VX-770 and VX-809 will be available in the first half of 2011. 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 Vertex could experience unforeseen delays, that future outcomes from ENVISION and from the various extension studies of VX-770 may not be favorable or may be less favorable than observed to date in STRIVE and other studies, that unexpected side effects may appear as VX-770 or VX-809 are more broadly dosed, that regulatory authorities may require more extensive data for VX-770 regulatory filings than currently expected; that future clinical, competitive and other factors may adversely affect the potential for VX-770; that the company may not be able to successfully develop VX-770 or combination therapies involving VX-770 and VX-809, 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 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, Wednesday, February 23, 2011 at 8:30 a.m. ET to review recent developments in cystic fibrosis. this call and webcast will be broadcast via the Internet at vrtx.com. It is suggested that webcast participants go to the web site at least 10 minutes in advance of the call to ensure that they can access the slides. the link to the webcast is available on the Events and Presentations button on the home page. to listen to the call on the telephone, dial 866-501-1537 (U.S. and Canada) or 720-545-0001 (International). Vertex is also providing a podcast MP3 file available for download on the Vertex website at vrtx.com. the conference ID number is 46977282. the call will be available for replay via telephone commencing February 23, 2011 at 12:00 p.m. ET running through 5:00 p.m. ET on March 1, 2011. the replay phone number for the U.S. and Canada is 800-642-1687. the international replay number is 706-645-9291. the conference ID number is 46977282. following the live webcast, an archived version will be available on Vertex’s website until 5:00 p.m. ET on March 9, 2011.

Vertex’s press releases are available at vrtx.com.

Patients on drug had significant weight gain, reduced sweat chloride and were less likely to experience a pulmonary exacerbation

Bethesda, MD /PRNewswire-USNewswire/ – The Cystic Fibrosis Foundation and Vertex Pharmaceuticals announced today that VX-770, an oral medicine in development that targets the defective protein that causes cystic fibrosis, showed promising results in a Phase 3 clinical trial.

The trial was designed to evaluate patients age 12 and up who carry at least one copy of a CF mutation called G551D. The study included 161 patients who received at least one dose of VX-770 or placebo.

Patients who took the drug, compared to those on placebo, showed a marked improvement in lung function at 24 weeks, which was sustained for the duration of the 48-week trial.

Patients also showed improvement across all key secondary endpoints in the study, including reduced likelihood of experiencing a pulmonary exacerbation, decreased respiratory symptoms and improved weight gain. each of these areas is critically important to the health of people with CF.

In addition, average sweat chloride levels of patients on VX-770 dropped toward normal levels, while those on placebo did not change—indicating the drug is impacting the underlying defect in CF. Excessive sweat chloride (salt) is a key clinical indicator of CF.

VX-770 is being developed by Vertex, and was discovered in collaboration with the CF Foundation, which provided substantial support to Vertex throughout the development process, including an approximately $75 million investment.

About four percent of people with CF carry the G551D mutation. more studies are needed to determine whether other CF mutations may benefit from VX-770.

“These results are highly encouraging. They provide scientific evidence that support our long-standing belief that targeting the underlying defect of CF may have a profound effect on the disease,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “We have much more to do to end the suffering caused by this disease, but these data are extremely exciting, especially for people with the G551D mutation and their families. The results also offer significant hope that a similar approach to treatment may help the majority of patients living with CF.”

The Phase 3 data support Vertex’s plan to submit a new Drug Application for VX-770 to the U.S. Food and Drug Administration (FDA) in the second half of 2011. Generally, the FDA takes between 6 and 12 months to review and rule on a drug application.

“The Cystic Fibrosis Foundation has played an instrumental role in our more than 10-year effort to discover and develop potential new CF medicines such as VX-770,” said Matthew W. Emmens, chairman, president and CEO of Vertex. “The data announced today reflect a significant investment of time, dollars and scientific expertise from both Vertex and the CF Foundation, and we look forward to working closely with the Foundation as we seek to bring VX-770 to people with CF.”

Said Preston W. Campbell III, M.D., executive vice president for medical affairs of the Cystic Fibrosis Foundation: “As a physician who has treated CF patients for many years, the VX-770 results are more than just numbers — they represent hope.” he added, “It’s not surprising that patients felt better on the drug because of the magnitude of lung function improvement and weight gain. these are important clinical outcomes, and the fact they were maintained through 48 weeks is very encouraging.”

Developing a therapy for individuals with the most common CF mutation, Delta F508, remains a top priority of the CF Foundation. A Phase 2 trial for those with Delta F508 is underway to test VX-770 in combination with a drug known as VX-809. Like VX-770, VX-809 resulted from discovery efforts between Vertex and the CF Foundation. Laboratory studies suggest a combination of the two drugs may be more beneficial than either one alone. Results of this clinical trial are expected later this year.

In addition, the CF Foundation is expanding its medical research program by $100 million over the next five years to focus on developing therapies, known as CFTR modulators, which address the underlying defect.

Vertex’s VX-770 Phase 3 program involves three different clinical trials, including the trial for people age 12 and up with the G551D mutation described above. In addition, a VX-770 trial of children ages 6 to 11 with the G551D mutation is ongoing and results are expected in mid-2011. The company also announced results for a trial evaluating VX-770 in patients with two copies of the Delta F508 mutation. Safety was the study’s primary focus, and adverse events were similar between the VX-770 and placebo groups in the study. Patients did not show a clinically meaningful improvement in lung function, although sweat chloride improved by a small amount.

Results from all three studies will be part of the drug application Vertex will submit to the FDA.

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 cff.org.

SOURCE Cystic Fibrosis Foundation

Copyright [2011] PR Newswire. All Rights Reserved.