Vertex Pharmaceuticals (VRTX) Reports Data on Investigational Hepatitis C Therapies Suggesting Potential For Novel Treatment Approaches

[Guest guest]

http://www.hcvadvocate.org/news/newsRev/2004/NewsRev-44.html

Vertex Pharmaceuticals (VRTX) Reports Data on Investigational Hepatitis C

Therapies Suggesting Potential For Novel Treatment Approaches

Source: PRNewswire

BERLIN, -- New data for proprietary investigational antiviral therapies for

hepatitis C virus (HCV) infection being developed by Vertex Pharmaceuticals

Incorporated were presented today at the Annual Meeting for the European

Association for the Study of the Liver (EASL) in Berlin, Germany. The

clinical and preclinical data presented highlight the potential of these

therapies to address significant unmet medical needs in the treatment of

HCV.

Vertex's extensive drug development portfolio includes two different

approaches for advancing the future standard-of-care in HCV. Merimepodib,

Vertex's lead oral therapy targeting HCV, is being developed in combination

with the current therapeutic standard, pegylated interferon alpha (peg-IFN)

and ribavirin, with the goal of enhancing antiviral efficacy and increasing

the proportion of patients who achieve a sustained response to treatment.

Vertex is also developing VX-950, an investigational HCV protease inhibitor

and one of the most advanced of a new class of direct antiviral agents for

the treatment of chronic HCV infection. Vertex owns worldwide development

and commercialization rights for both merimepodib and VX-950.

In a poster session at EASL today, clinical investigators presented results

for patients enrolled in the extension phase of a pilot Phase IIa study

designed to evaluate the safety and tolerability of merimepodib in

combination with peg-IFN and ribavirin in a treatment-refractory population.

Additionally, in oral presentations at EASL, Vertex scientists reported

preclinical data highlighting the promising antiviral profile of VX-950,

including in vitro data assessing the antiviral activity of VX-950 in

combination with interferon alpha.

" The results presented at EASL provide strong support for initiating late-

stage clinical development of merimepodib, as well as the first clinical

studies of VX-950, " stated Alam, M.D., Senior Vice President for Drug

Evaluation and Approval at Vertex. " These are key milestones for Vertex and

its interest in providing new therapies in HCV, and are anticipated in 2004.

Vertex's goal is to commercialize innovative therapies that will provide

additional options for patients and enhance HCV clinical care. "

Merimepodib Results and Clinical Plans

In 2003, Vertex completed the treatment arms of a placebo-controlled triple

combination Phase II study of merimepodib (MMPD) in combination with peg-IFN

and ribavirin. This trial was designed to evaluate the safety of the triple

combination in 31 patients with genotype 1 infection who did not respond to

a previous course of interferon alpha in combination with ribavirin. The

study provided for six months of treatment, with an optional six-month

extension phase for patients who responded to therapy. Data presented by

clinical investigators in 2003 demonstrated that the triple combination of

MMPD + peg-IFN + ribavirin was well-tolerated and did not appear to

exacerbate the incidence of hematological toxicities associated with peg-IFN

and ribavirin treatment. Study results also indicated that the addition of

merimepodib enhanced the antiviral activity of the peg-IFN + ribavirin

combination at 24 weeks.

In a poster presented at EASL today, clinical investigators presented data

on 11 patients (three subjects receiving placebo + peg-IFN + ribavirin and

eight subjects receiving MMPD + peg-IFN + ribavirin) who were eligible to

continue into the extension phase of the study. Ten patients completed 48

weeks of treatment. At the end of 48 weeks of treatment, the safety profile

of MMPD was similar to the core 24-week study, and the majority of adverse

events reported were consistent with the known side effect profile of

peg-IFN and ribavirin. One of three patients in the placebo group and three

of seven patients receiving MMPD who completed the extension phase achieved

a sustained viral response at week 72 (24 weeks post-treatment). Based on

the Phase IIa results, Vertex is planning to initiate larger studies to

evaluate the ability of merimepodib to increase sustained viral response

rates (SVR) in HCV- infected patients. These larger studies may involve, as

before, an initial treatment period with MMPD in combination with peg-IFN

and ribavirin, but followed by continued treatment with peg-IFN and

ribavirin alone, a clinical approach suggested by the clinical and

preclinical data to optimize the SVR.

" Merimepodib demonstrated a good tolerability profile and showed important

signs of antiviral activity in this study, " stated Dr. Marcellin,

Professor of Medicine at University of Paris VII, and the lead investigator

for the study. " Genotype 1 HCV is associated with the lowest response to

therapy, and patients who are non-responsive to prior combination therapy

have very limited treatment options available. These results support larger

well- controlled studies in treatment-refractory patients to evaluate the

ability of merimepodib to effect an increase in sustained viral response in

combination with peg-IFN and ribavirin. "

Vertex anticipates initiating in 2004 a Phase IIb clinical study of

merimepodib in patients who are non-responsive to prior treatment with

peg-IFN + ribavirin. The primary goal of this placebo-controlled study will

be to evaluate the antiviral activity of a triple combination regimen and to

perform an assessment of sustained virologic response (SVR) rates. SVR is

defined as an HCV-undetectable value at the end of a 24-week post-treatment

follow-up period (week 72).

VX-950 Results and Clinical Plans

In oral presentations at EASL on Thursday and Friday, Vertex researchers

presented a variety of preclinical results highlighting the emerging profile

of VX-950. Vertex researchers demonstrated that treatment of HCV replicon

cells with VX-950 decreased viral replication by 10,000-fold to undetectable

levels; when the drug was subsequently removed, no rebound of viral

replication was observed, suggesting that the HCV replicon had been

eradicated from the treated cells. A second line of experiments showed that

combination of interferon alpha with VX-950 in the HCV replicon system

allowed scientists to reduce the level of VX-950 and still obtain the same

degree of antiviral potency obtained with a 5-fold higher concentration of

VX-950 alone.

Data presented last year at the AASLD and International HCV meetings showed

that the dominant mutation(s) selected in the laboratory against VX-950

remained sensitive to BILN 2061, a protease inhibitor developed by another

company which has shown antiviral activity in HCV patients, and BILN 2061

resistant mutants remained sensitive to VX-950. In the studies presented at

EASL, researchers analyzed minor mutations that were cross-resistant to

VX-950 and BILN 2061 in the laboratory and found that enzymatic activity of

the cross-resistant HCV protease was reduced approximately 4 to 7-fold, a

condition which could impair the ability of the virus to grow.

" Vertex's leadership position in the discovery of novel approaches to HCV

treatment has been enhanced by the development of new technologies and

approaches for evaluating the clinical potential of experimental compounds, "

commented Dr. Alam. " The results we have seen with VX-950 confirm our

selection of HCV protease as an excellent target for the development of

powerful antiviral therapies and that VX-950 has the potential of becoming a

highly valuable therapeutic agent for the treatment of HCV patients. "

Vertex anticipates that it will initiate a Phase I clinical trial of VX- 950

in healthy volunteers in 2004. Positive results from this first Phase I

study will pave the way for the first evaluation of VX-950's antiviral

activity in HCV-infected patients.

Clinical Need and Market Opportunity in HCV Infection

HCV infection is a serious disease that causes inflammation of the liver,

which may lead to fibrosis and cirrhosis, liver cancer, and ultimately,

liver failure. Chronic hepatitis C infection afflicts approximately 2.7

million people in the U.S., many of whom are unaware of their infected

status. Current treatments provide a sustained viral response for only 40 to

50 percent of patients chronically infected with genotype 1 HCV, the most

difficult viral strain to treat and the most common form in the U.S.

Patients who are non-responsive to current HCV therapy have limited

treatment options, and clinical experience suggests that only a very low

proportion of such patients achieve a sustained viral response with

subsequent treatment regimens. HCV may go undetected for up to 20 years

following initial infection. Worldwide, the disease strikes as many as 185

million people. Each year, 8,000 to 10,000 people in the U.S. die from

complications of HCV.

About Vertex

Vertex Pharmaceuticals Incorporated is a global biotechnology company

committed to the discovery and development of breakthrough small molecule

drugs for serious diseases. The Company's strategy is to commercialize its

products both independently and in collaboration with major pharmaceutical

partners. Vertex's product pipeline is principally focused on viral

diseases, inflammation, autoimmune diseases and cancer. Vertex co-promotes

the new HIV protease inhibitor, Lexiva, with GlaxoKline.

This press release may contain forward-looking statements, including

statements that (i) Vertex anticipates commercializing new therapies,

including merimepodib and VX-950, for the treatment of hepatitis C and that

merimepodib and VX-950 each have the potential to address significant unmet

needs in HCV treatment; (ii) merimepodib and VX-950 each hold promise as

part of combination therapy for HCV patients who have limited treatment

options and represents an attractive commercial opportunity for Vertex;

(iii) further clinical study of merimepodib will be initiated in 2004; and

(iv) clinical study of VX-950 will be initiated in 2004. While management

makes its best efforts to be accurate in making forward-looking statements,

such statements are subject to risks and uncertainties that could cause

Vertex's actual results to vary materially. These risks and uncertainties

include, among other things, the risks that clinical trials for merimepodib

or VX-950 may not be initiated or, if initiated, may not proceed as planned

due to technical, scientific, or patient enrollment issues, that results

from planned clinical trials with merimepodib will not reflect the positive

results from earlier trials, that positive nonclinical study results for

either merimepodib or VX- 950 will not be duplicated in future nonclinical

or clinical studies and other risks listed under Risk Factors in Vertex's

form 10-K filed with the Securities and Exchange Commission on March 15,

2004. Lexiva is a registered trademark of the GlaxoKline group of

companies.

Vertex's press releases are available at http://www.vrtx.com/.

Vertex Pharmaceuticals Incorporated will host a conference call on Monday,

April 26, 2004 at 5:00 pm (EDT) to discuss its first quarter 2004 financial

results, at which time it will review and update its HCV product development

programs. The conference call also will be webcast, and the webcast will be

available to all interested parties through Vertex's website,

http://www.vrtx.com/. To access the webcast, go to the investor center and

select " conference calls. " To ensure a timely connection to the webcast, it

is recommended that users register at least 15 minutes prior to the

scheduled webcast.

Vertex Contacts: Partridge, Director, Corporate Communications (617)

444-6108 Lora Pike, Manager, Investor Relations (617) 444-6755

Vertex Pharmaceuticals Incorporated

CONTACT: Partridge, Director, Corporate Communications,

+1-617-444-6108, or Lora Pike, Manager, Investor Relations, +1-617-444-6755,

both of Vertex Pharmaceuticals Incorporated

Web site: http://www.vrtx.com/

Company News On-Call: http://www.prnewswire.com/comp

/938395.html

[Guest guest]

http://www.hcvadvocate.org/news/newsRev/2004/NewsRev-44.html

Vertex Pharmaceuticals (VRTX) Reports Data on Investigational Hepatitis C

Therapies Suggesting Potential For Novel Treatment Approaches

Source: PRNewswire

BERLIN, -- New data for proprietary investigational antiviral therapies for

hepatitis C virus (HCV) infection being developed by Vertex Pharmaceuticals

Incorporated were presented today at the Annual Meeting for the European

Association for the Study of the Liver (EASL) in Berlin, Germany. The

clinical and preclinical data presented highlight the potential of these

therapies to address significant unmet medical needs in the treatment of

HCV.

Vertex's extensive drug development portfolio includes two different

approaches for advancing the future standard-of-care in HCV. Merimepodib,

Vertex's lead oral therapy targeting HCV, is being developed in combination

with the current therapeutic standard, pegylated interferon alpha (peg-IFN)

and ribavirin, with the goal of enhancing antiviral efficacy and increasing

the proportion of patients who achieve a sustained response to treatment.

Vertex is also developing VX-950, an investigational HCV protease inhibitor

and one of the most advanced of a new class of direct antiviral agents for

the treatment of chronic HCV infection. Vertex owns worldwide development

and commercialization rights for both merimepodib and VX-950.

In a poster session at EASL today, clinical investigators presented results

for patients enrolled in the extension phase of a pilot Phase IIa study

designed to evaluate the safety and tolerability of merimepodib in

combination with peg-IFN and ribavirin in a treatment-refractory population.

Additionally, in oral presentations at EASL, Vertex scientists reported

preclinical data highlighting the promising antiviral profile of VX-950,

including in vitro data assessing the antiviral activity of VX-950 in

combination with interferon alpha.

" The results presented at EASL provide strong support for initiating late-

stage clinical development of merimepodib, as well as the first clinical

studies of VX-950, " stated Alam, M.D., Senior Vice President for Drug

Evaluation and Approval at Vertex. " These are key milestones for Vertex and

its interest in providing new therapies in HCV, and are anticipated in 2004.

Vertex's goal is to commercialize innovative therapies that will provide

additional options for patients and enhance HCV clinical care. "

Merimepodib Results and Clinical Plans

In 2003, Vertex completed the treatment arms of a placebo-controlled triple

combination Phase II study of merimepodib (MMPD) in combination with peg-IFN

and ribavirin. This trial was designed to evaluate the safety of the triple

combination in 31 patients with genotype 1 infection who did not respond to

a previous course of interferon alpha in combination with ribavirin. The

study provided for six months of treatment, with an optional six-month

extension phase for patients who responded to therapy. Data presented by

clinical investigators in 2003 demonstrated that the triple combination of

MMPD + peg-IFN + ribavirin was well-tolerated and did not appear to

exacerbate the incidence of hematological toxicities associated with peg-IFN

and ribavirin treatment. Study results also indicated that the addition of

merimepodib enhanced the antiviral activity of the peg-IFN + ribavirin

combination at 24 weeks.

In a poster presented at EASL today, clinical investigators presented data

on 11 patients (three subjects receiving placebo + peg-IFN + ribavirin and

eight subjects receiving MMPD + peg-IFN + ribavirin) who were eligible to

continue into the extension phase of the study. Ten patients completed 48

weeks of treatment. At the end of 48 weeks of treatment, the safety profile

of MMPD was similar to the core 24-week study, and the majority of adverse

events reported were consistent with the known side effect profile of

peg-IFN and ribavirin. One of three patients in the placebo group and three

of seven patients receiving MMPD who completed the extension phase achieved

a sustained viral response at week 72 (24 weeks post-treatment). Based on

the Phase IIa results, Vertex is planning to initiate larger studies to

evaluate the ability of merimepodib to increase sustained viral response

rates (SVR) in HCV- infected patients. These larger studies may involve, as

before, an initial treatment period with MMPD in combination with peg-IFN

and ribavirin, but followed by continued treatment with peg-IFN and

ribavirin alone, a clinical approach suggested by the clinical and

preclinical data to optimize the SVR.

" Merimepodib demonstrated a good tolerability profile and showed important

signs of antiviral activity in this study, " stated Dr. Marcellin,

Professor of Medicine at University of Paris VII, and the lead investigator

for the study. " Genotype 1 HCV is associated with the lowest response to

therapy, and patients who are non-responsive to prior combination therapy

have very limited treatment options available. These results support larger

well- controlled studies in treatment-refractory patients to evaluate the

ability of merimepodib to effect an increase in sustained viral response in

combination with peg-IFN and ribavirin. "

Vertex anticipates initiating in 2004 a Phase IIb clinical study of

merimepodib in patients who are non-responsive to prior treatment with

peg-IFN + ribavirin. The primary goal of this placebo-controlled study will

be to evaluate the antiviral activity of a triple combination regimen and to

perform an assessment of sustained virologic response (SVR) rates. SVR is

defined as an HCV-undetectable value at the end of a 24-week post-treatment

follow-up period (week 72).

VX-950 Results and Clinical Plans

In oral presentations at EASL on Thursday and Friday, Vertex researchers

presented a variety of preclinical results highlighting the emerging profile

of VX-950. Vertex researchers demonstrated that treatment of HCV replicon

cells with VX-950 decreased viral replication by 10,000-fold to undetectable

levels; when the drug was subsequently removed, no rebound of viral

replication was observed, suggesting that the HCV replicon had been

eradicated from the treated cells. A second line of experiments showed that

combination of interferon alpha with VX-950 in the HCV replicon system

allowed scientists to reduce the level of VX-950 and still obtain the same

degree of antiviral potency obtained with a 5-fold higher concentration of

VX-950 alone.

Data presented last year at the AASLD and International HCV meetings showed

that the dominant mutation(s) selected in the laboratory against VX-950

remained sensitive to BILN 2061, a protease inhibitor developed by another

company which has shown antiviral activity in HCV patients, and BILN 2061

resistant mutants remained sensitive to VX-950. In the studies presented at

EASL, researchers analyzed minor mutations that were cross-resistant to

VX-950 and BILN 2061 in the laboratory and found that enzymatic activity of

the cross-resistant HCV protease was reduced approximately 4 to 7-fold, a

condition which could impair the ability of the virus to grow.

" Vertex's leadership position in the discovery of novel approaches to HCV

treatment has been enhanced by the development of new technologies and

approaches for evaluating the clinical potential of experimental compounds, "

commented Dr. Alam. " The results we have seen with VX-950 confirm our

selection of HCV protease as an excellent target for the development of

powerful antiviral therapies and that VX-950 has the potential of becoming a

highly valuable therapeutic agent for the treatment of HCV patients. "

Vertex anticipates that it will initiate a Phase I clinical trial of VX- 950

in healthy volunteers in 2004. Positive results from this first Phase I

study will pave the way for the first evaluation of VX-950's antiviral

activity in HCV-infected patients.

Clinical Need and Market Opportunity in HCV Infection

HCV infection is a serious disease that causes inflammation of the liver,

which may lead to fibrosis and cirrhosis, liver cancer, and ultimately,

liver failure. Chronic hepatitis C infection afflicts approximately 2.7

million people in the U.S., many of whom are unaware of their infected

status. Current treatments provide a sustained viral response for only 40 to

50 percent of patients chronically infected with genotype 1 HCV, the most

difficult viral strain to treat and the most common form in the U.S.

Patients who are non-responsive to current HCV therapy have limited

treatment options, and clinical experience suggests that only a very low

proportion of such patients achieve a sustained viral response with

subsequent treatment regimens. HCV may go undetected for up to 20 years

following initial infection. Worldwide, the disease strikes as many as 185

million people. Each year, 8,000 to 10,000 people in the U.S. die from

complications of HCV.

About Vertex

Vertex Pharmaceuticals Incorporated is a global biotechnology company

committed to the discovery and development of breakthrough small molecule

drugs for serious diseases. The Company's strategy is to commercialize its

products both independently and in collaboration with major pharmaceutical

partners. Vertex's product pipeline is principally focused on viral

diseases, inflammation, autoimmune diseases and cancer. Vertex co-promotes

the new HIV protease inhibitor, Lexiva, with GlaxoKline.

This press release may contain forward-looking statements, including

statements that (i) Vertex anticipates commercializing new therapies,

including merimepodib and VX-950, for the treatment of hepatitis C and that

merimepodib and VX-950 each have the potential to address significant unmet

needs in HCV treatment; (ii) merimepodib and VX-950 each hold promise as

part of combination therapy for HCV patients who have limited treatment

options and represents an attractive commercial opportunity for Vertex;

(iii) further clinical study of merimepodib will be initiated in 2004; and

(iv) clinical study of VX-950 will be initiated in 2004. While management

makes its best efforts to be accurate in making forward-looking statements,

such statements are subject to risks and uncertainties that could cause

Vertex's actual results to vary materially. These risks and uncertainties

include, among other things, the risks that clinical trials for merimepodib

or VX-950 may not be initiated or, if initiated, may not proceed as planned

due to technical, scientific, or patient enrollment issues, that results

from planned clinical trials with merimepodib will not reflect the positive

results from earlier trials, that positive nonclinical study results for

either merimepodib or VX- 950 will not be duplicated in future nonclinical

or clinical studies and other risks listed under Risk Factors in Vertex's

form 10-K filed with the Securities and Exchange Commission on March 15,

2004. Lexiva is a registered trademark of the GlaxoKline group of

companies.

Vertex's press releases are available at http://www.vrtx.com/.

Vertex Pharmaceuticals Incorporated will host a conference call on Monday,

April 26, 2004 at 5:00 pm (EDT) to discuss its first quarter 2004 financial

results, at which time it will review and update its HCV product development

programs. The conference call also will be webcast, and the webcast will be

available to all interested parties through Vertex's website,

http://www.vrtx.com/. To access the webcast, go to the investor center and

select " conference calls. " To ensure a timely connection to the webcast, it

is recommended that users register at least 15 minutes prior to the

scheduled webcast.

Vertex Contacts: Partridge, Director, Corporate Communications (617)

444-6108 Lora Pike, Manager, Investor Relations (617) 444-6755

Vertex Pharmaceuticals Incorporated

CONTACT: Partridge, Director, Corporate Communications,

+1-617-444-6108, or Lora Pike, Manager, Investor Relations, +1-617-444-6755,

both of Vertex Pharmaceuticals Incorporated

Web site: http://www.vrtx.com/

Company News On-Call: http://www.prnewswire.com/comp

/938395.html

[Guest guest]

http://www.hcvadvocate.org/news/newsRev/2004/NewsRev-44.html

Vertex Pharmaceuticals (VRTX) Reports Data on Investigational Hepatitis C

Therapies Suggesting Potential For Novel Treatment Approaches

Source: PRNewswire

BERLIN, -- New data for proprietary investigational antiviral therapies for

hepatitis C virus (HCV) infection being developed by Vertex Pharmaceuticals

Incorporated were presented today at the Annual Meeting for the European

Association for the Study of the Liver (EASL) in Berlin, Germany. The

clinical and preclinical data presented highlight the potential of these

therapies to address significant unmet medical needs in the treatment of

HCV.

Vertex's extensive drug development portfolio includes two different

approaches for advancing the future standard-of-care in HCV. Merimepodib,

Vertex's lead oral therapy targeting HCV, is being developed in combination

with the current therapeutic standard, pegylated interferon alpha (peg-IFN)

and ribavirin, with the goal of enhancing antiviral efficacy and increasing

the proportion of patients who achieve a sustained response to treatment.

Vertex is also developing VX-950, an investigational HCV protease inhibitor

and one of the most advanced of a new class of direct antiviral agents for

the treatment of chronic HCV infection. Vertex owns worldwide development

and commercialization rights for both merimepodib and VX-950.

In a poster session at EASL today, clinical investigators presented results

for patients enrolled in the extension phase of a pilot Phase IIa study

designed to evaluate the safety and tolerability of merimepodib in

combination with peg-IFN and ribavirin in a treatment-refractory population.

Additionally, in oral presentations at EASL, Vertex scientists reported

preclinical data highlighting the promising antiviral profile of VX-950,

including in vitro data assessing the antiviral activity of VX-950 in

combination with interferon alpha.

" The results presented at EASL provide strong support for initiating late-

stage clinical development of merimepodib, as well as the first clinical

studies of VX-950, " stated Alam, M.D., Senior Vice President for Drug

Evaluation and Approval at Vertex. " These are key milestones for Vertex and

its interest in providing new therapies in HCV, and are anticipated in 2004.

Vertex's goal is to commercialize innovative therapies that will provide

additional options for patients and enhance HCV clinical care. "

Merimepodib Results and Clinical Plans

In 2003, Vertex completed the treatment arms of a placebo-controlled triple

combination Phase II study of merimepodib (MMPD) in combination with peg-IFN

and ribavirin. This trial was designed to evaluate the safety of the triple

combination in 31 patients with genotype 1 infection who did not respond to

a previous course of interferon alpha in combination with ribavirin. The

study provided for six months of treatment, with an optional six-month

extension phase for patients who responded to therapy. Data presented by

clinical investigators in 2003 demonstrated that the triple combination of

MMPD + peg-IFN + ribavirin was well-tolerated and did not appear to

exacerbate the incidence of hematological toxicities associated with peg-IFN

and ribavirin treatment. Study results also indicated that the addition of

merimepodib enhanced the antiviral activity of the peg-IFN + ribavirin

combination at 24 weeks.

In a poster presented at EASL today, clinical investigators presented data

on 11 patients (three subjects receiving placebo + peg-IFN + ribavirin and

eight subjects receiving MMPD + peg-IFN + ribavirin) who were eligible to

continue into the extension phase of the study. Ten patients completed 48

weeks of treatment. At the end of 48 weeks of treatment, the safety profile

of MMPD was similar to the core 24-week study, and the majority of adverse

events reported were consistent with the known side effect profile of

peg-IFN and ribavirin. One of three patients in the placebo group and three

of seven patients receiving MMPD who completed the extension phase achieved

a sustained viral response at week 72 (24 weeks post-treatment). Based on

the Phase IIa results, Vertex is planning to initiate larger studies to

evaluate the ability of merimepodib to increase sustained viral response

rates (SVR) in HCV- infected patients. These larger studies may involve, as

before, an initial treatment period with MMPD in combination with peg-IFN

and ribavirin, but followed by continued treatment with peg-IFN and

ribavirin alone, a clinical approach suggested by the clinical and

preclinical data to optimize the SVR.

" Merimepodib demonstrated a good tolerability profile and showed important

signs of antiviral activity in this study, " stated Dr. Marcellin,

Professor of Medicine at University of Paris VII, and the lead investigator

for the study. " Genotype 1 HCV is associated with the lowest response to

therapy, and patients who are non-responsive to prior combination therapy

have very limited treatment options available. These results support larger

well- controlled studies in treatment-refractory patients to evaluate the

ability of merimepodib to effect an increase in sustained viral response in

combination with peg-IFN and ribavirin. "

Vertex anticipates initiating in 2004 a Phase IIb clinical study of

merimepodib in patients who are non-responsive to prior treatment with

peg-IFN + ribavirin. The primary goal of this placebo-controlled study will

be to evaluate the antiviral activity of a triple combination regimen and to

perform an assessment of sustained virologic response (SVR) rates. SVR is

defined as an HCV-undetectable value at the end of a 24-week post-treatment

follow-up period (week 72).

VX-950 Results and Clinical Plans

In oral presentations at EASL on Thursday and Friday, Vertex researchers

presented a variety of preclinical results highlighting the emerging profile

of VX-950. Vertex researchers demonstrated that treatment of HCV replicon

cells with VX-950 decreased viral replication by 10,000-fold to undetectable

levels; when the drug was subsequently removed, no rebound of viral

replication was observed, suggesting that the HCV replicon had been

eradicated from the treated cells. A second line of experiments showed that

combination of interferon alpha with VX-950 in the HCV replicon system

allowed scientists to reduce the level of VX-950 and still obtain the same

degree of antiviral potency obtained with a 5-fold higher concentration of

VX-950 alone.

Data presented last year at the AASLD and International HCV meetings showed

that the dominant mutation(s) selected in the laboratory against VX-950

remained sensitive to BILN 2061, a protease inhibitor developed by another

company which has shown antiviral activity in HCV patients, and BILN 2061

resistant mutants remained sensitive to VX-950. In the studies presented at

EASL, researchers analyzed minor mutations that were cross-resistant to

VX-950 and BILN 2061 in the laboratory and found that enzymatic activity of

the cross-resistant HCV protease was reduced approximately 4 to 7-fold, a

condition which could impair the ability of the virus to grow.

" Vertex's leadership position in the discovery of novel approaches to HCV

treatment has been enhanced by the development of new technologies and

approaches for evaluating the clinical potential of experimental compounds, "

commented Dr. Alam. " The results we have seen with VX-950 confirm our

selection of HCV protease as an excellent target for the development of

powerful antiviral therapies and that VX-950 has the potential of becoming a

highly valuable therapeutic agent for the treatment of HCV patients. "

Vertex anticipates that it will initiate a Phase I clinical trial of VX- 950

in healthy volunteers in 2004. Positive results from this first Phase I

study will pave the way for the first evaluation of VX-950's antiviral

activity in HCV-infected patients.

Clinical Need and Market Opportunity in HCV Infection

HCV infection is a serious disease that causes inflammation of the liver,

which may lead to fibrosis and cirrhosis, liver cancer, and ultimately,

liver failure. Chronic hepatitis C infection afflicts approximately 2.7

million people in the U.S., many of whom are unaware of their infected

status. Current treatments provide a sustained viral response for only 40 to

50 percent of patients chronically infected with genotype 1 HCV, the most

difficult viral strain to treat and the most common form in the U.S.

Patients who are non-responsive to current HCV therapy have limited

treatment options, and clinical experience suggests that only a very low

proportion of such patients achieve a sustained viral response with

subsequent treatment regimens. HCV may go undetected for up to 20 years

following initial infection. Worldwide, the disease strikes as many as 185

million people. Each year, 8,000 to 10,000 people in the U.S. die from

complications of HCV.

About Vertex

Vertex Pharmaceuticals Incorporated is a global biotechnology company

committed to the discovery and development of breakthrough small molecule

drugs for serious diseases. The Company's strategy is to commercialize its

products both independently and in collaboration with major pharmaceutical

partners. Vertex's product pipeline is principally focused on viral

diseases, inflammation, autoimmune diseases and cancer. Vertex co-promotes

the new HIV protease inhibitor, Lexiva, with GlaxoKline.

This press release may contain forward-looking statements, including

statements that (i) Vertex anticipates commercializing new therapies,

including merimepodib and VX-950, for the treatment of hepatitis C and that

merimepodib and VX-950 each have the potential to address significant unmet

needs in HCV treatment; (ii) merimepodib and VX-950 each hold promise as

part of combination therapy for HCV patients who have limited treatment

options and represents an attractive commercial opportunity for Vertex;

(iii) further clinical study of merimepodib will be initiated in 2004; and

(iv) clinical study of VX-950 will be initiated in 2004. While management

makes its best efforts to be accurate in making forward-looking statements,

such statements are subject to risks and uncertainties that could cause

Vertex's actual results to vary materially. These risks and uncertainties

include, among other things, the risks that clinical trials for merimepodib

or VX-950 may not be initiated or, if initiated, may not proceed as planned

due to technical, scientific, or patient enrollment issues, that results

from planned clinical trials with merimepodib will not reflect the positive

results from earlier trials, that positive nonclinical study results for

either merimepodib or VX- 950 will not be duplicated in future nonclinical

or clinical studies and other risks listed under Risk Factors in Vertex's

form 10-K filed with the Securities and Exchange Commission on March 15,

2004. Lexiva is a registered trademark of the GlaxoKline group of

companies.

Vertex's press releases are available at http://www.vrtx.com/.

Vertex Pharmaceuticals Incorporated will host a conference call on Monday,

April 26, 2004 at 5:00 pm (EDT) to discuss its first quarter 2004 financial

results, at which time it will review and update its HCV product development

programs. The conference call also will be webcast, and the webcast will be

available to all interested parties through Vertex's website,

http://www.vrtx.com/. To access the webcast, go to the investor center and

select " conference calls. " To ensure a timely connection to the webcast, it

is recommended that users register at least 15 minutes prior to the

scheduled webcast.

Vertex Contacts: Partridge, Director, Corporate Communications (617)

444-6108 Lora Pike, Manager, Investor Relations (617) 444-6755

Vertex Pharmaceuticals Incorporated

CONTACT: Partridge, Director, Corporate Communications,

+1-617-444-6108, or Lora Pike, Manager, Investor Relations, +1-617-444-6755,

both of Vertex Pharmaceuticals Incorporated

Web site: http://www.vrtx.com/

Company News On-Call: http://www.prnewswire.com/comp

/938395.html

[Guest guest]

http://www.hcvadvocate.org/news/newsRev/2004/NewsRev-44.html

Vertex Pharmaceuticals (VRTX) Reports Data on Investigational Hepatitis C

Therapies Suggesting Potential For Novel Treatment Approaches

Source: PRNewswire

BERLIN, -- New data for proprietary investigational antiviral therapies for

hepatitis C virus (HCV) infection being developed by Vertex Pharmaceuticals

Incorporated were presented today at the Annual Meeting for the European

Association for the Study of the Liver (EASL) in Berlin, Germany. The

clinical and preclinical data presented highlight the potential of these

therapies to address significant unmet medical needs in the treatment of

HCV.

Vertex's extensive drug development portfolio includes two different

approaches for advancing the future standard-of-care in HCV. Merimepodib,

Vertex's lead oral therapy targeting HCV, is being developed in combination

with the current therapeutic standard, pegylated interferon alpha (peg-IFN)

and ribavirin, with the goal of enhancing antiviral efficacy and increasing

the proportion of patients who achieve a sustained response to treatment.

Vertex is also developing VX-950, an investigational HCV protease inhibitor

and one of the most advanced of a new class of direct antiviral agents for

the treatment of chronic HCV infection. Vertex owns worldwide development

and commercialization rights for both merimepodib and VX-950.

In a poster session at EASL today, clinical investigators presented results

for patients enrolled in the extension phase of a pilot Phase IIa study

designed to evaluate the safety and tolerability of merimepodib in

combination with peg-IFN and ribavirin in a treatment-refractory population.

Additionally, in oral presentations at EASL, Vertex scientists reported

preclinical data highlighting the promising antiviral profile of VX-950,

including in vitro data assessing the antiviral activity of VX-950 in

combination with interferon alpha.

" The results presented at EASL provide strong support for initiating late-

stage clinical development of merimepodib, as well as the first clinical

studies of VX-950, " stated Alam, M.D., Senior Vice President for Drug

Evaluation and Approval at Vertex. " These are key milestones for Vertex and

its interest in providing new therapies in HCV, and are anticipated in 2004.

Vertex's goal is to commercialize innovative therapies that will provide

additional options for patients and enhance HCV clinical care. "

Merimepodib Results and Clinical Plans

In 2003, Vertex completed the treatment arms of a placebo-controlled triple

combination Phase II study of merimepodib (MMPD) in combination with peg-IFN

and ribavirin. This trial was designed to evaluate the safety of the triple

combination in 31 patients with genotype 1 infection who did not respond to

a previous course of interferon alpha in combination with ribavirin. The

study provided for six months of treatment, with an optional six-month

extension phase for patients who responded to therapy. Data presented by

clinical investigators in 2003 demonstrated that the triple combination of

MMPD + peg-IFN + ribavirin was well-tolerated and did not appear to

exacerbate the incidence of hematological toxicities associated with peg-IFN

and ribavirin treatment. Study results also indicated that the addition of

merimepodib enhanced the antiviral activity of the peg-IFN + ribavirin

combination at 24 weeks.

In a poster presented at EASL today, clinical investigators presented data

on 11 patients (three subjects receiving placebo + peg-IFN + ribavirin and

eight subjects receiving MMPD + peg-IFN + ribavirin) who were eligible to

continue into the extension phase of the study. Ten patients completed 48

weeks of treatment. At the end of 48 weeks of treatment, the safety profile

of MMPD was similar to the core 24-week study, and the majority of adverse

events reported were consistent with the known side effect profile of

peg-IFN and ribavirin. One of three patients in the placebo group and three

of seven patients receiving MMPD who completed the extension phase achieved

a sustained viral response at week 72 (24 weeks post-treatment). Based on

the Phase IIa results, Vertex is planning to initiate larger studies to

evaluate the ability of merimepodib to increase sustained viral response

rates (SVR) in HCV- infected patients. These larger studies may involve, as

before, an initial treatment period with MMPD in combination with peg-IFN

and ribavirin, but followed by continued treatment with peg-IFN and

ribavirin alone, a clinical approach suggested by the clinical and

preclinical data to optimize the SVR.

" Merimepodib demonstrated a good tolerability profile and showed important

signs of antiviral activity in this study, " stated Dr. Marcellin,

Professor of Medicine at University of Paris VII, and the lead investigator

for the study. " Genotype 1 HCV is associated with the lowest response to

therapy, and patients who are non-responsive to prior combination therapy

have very limited treatment options available. These results support larger

well- controlled studies in treatment-refractory patients to evaluate the

ability of merimepodib to effect an increase in sustained viral response in

combination with peg-IFN and ribavirin. "

Vertex anticipates initiating in 2004 a Phase IIb clinical study of

merimepodib in patients who are non-responsive to prior treatment with

peg-IFN + ribavirin. The primary goal of this placebo-controlled study will

be to evaluate the antiviral activity of a triple combination regimen and to

perform an assessment of sustained virologic response (SVR) rates. SVR is

defined as an HCV-undetectable value at the end of a 24-week post-treatment

follow-up period (week 72).

VX-950 Results and Clinical Plans

In oral presentations at EASL on Thursday and Friday, Vertex researchers

presented a variety of preclinical results highlighting the emerging profile

of VX-950. Vertex researchers demonstrated that treatment of HCV replicon

cells with VX-950 decreased viral replication by 10,000-fold to undetectable

levels; when the drug was subsequently removed, no rebound of viral

replication was observed, suggesting that the HCV replicon had been

eradicated from the treated cells. A second line of experiments showed that

combination of interferon alpha with VX-950 in the HCV replicon system

allowed scientists to reduce the level of VX-950 and still obtain the same

degree of antiviral potency obtained with a 5-fold higher concentration of

VX-950 alone.

Data presented last year at the AASLD and International HCV meetings showed

that the dominant mutation(s) selected in the laboratory against VX-950

remained sensitive to BILN 2061, a protease inhibitor developed by another

company which has shown antiviral activity in HCV patients, and BILN 2061

resistant mutants remained sensitive to VX-950. In the studies presented at

EASL, researchers analyzed minor mutations that were cross-resistant to

VX-950 and BILN 2061 in the laboratory and found that enzymatic activity of

the cross-resistant HCV protease was reduced approximately 4 to 7-fold, a

condition which could impair the ability of the virus to grow.

" Vertex's leadership position in the discovery of novel approaches to HCV

treatment has been enhanced by the development of new technologies and

approaches for evaluating the clinical potential of experimental compounds, "

commented Dr. Alam. " The results we have seen with VX-950 confirm our

selection of HCV protease as an excellent target for the development of

powerful antiviral therapies and that VX-950 has the potential of becoming a

highly valuable therapeutic agent for the treatment of HCV patients. "

Vertex anticipates that it will initiate a Phase I clinical trial of VX- 950

in healthy volunteers in 2004. Positive results from this first Phase I

study will pave the way for the first evaluation of VX-950's antiviral

activity in HCV-infected patients.

Clinical Need and Market Opportunity in HCV Infection

HCV infection is a serious disease that causes inflammation of the liver,

which may lead to fibrosis and cirrhosis, liver cancer, and ultimately,

liver failure. Chronic hepatitis C infection afflicts approximately 2.7

million people in the U.S., many of whom are unaware of their infected

status. Current treatments provide a sustained viral response for only 40 to

50 percent of patients chronically infected with genotype 1 HCV, the most

difficult viral strain to treat and the most common form in the U.S.

Patients who are non-responsive to current HCV therapy have limited

treatment options, and clinical experience suggests that only a very low

proportion of such patients achieve a sustained viral response with

subsequent treatment regimens. HCV may go undetected for up to 20 years

following initial infection. Worldwide, the disease strikes as many as 185

million people. Each year, 8,000 to 10,000 people in the U.S. die from

complications of HCV.

About Vertex

Vertex Pharmaceuticals Incorporated is a global biotechnology company

committed to the discovery and development of breakthrough small molecule

drugs for serious diseases. The Company's strategy is to commercialize its

products both independently and in collaboration with major pharmaceutical

partners. Vertex's product pipeline is principally focused on viral

diseases, inflammation, autoimmune diseases and cancer. Vertex co-promotes

the new HIV protease inhibitor, Lexiva, with GlaxoKline.

This press release may contain forward-looking statements, including

statements that (i) Vertex anticipates commercializing new therapies,

including merimepodib and VX-950, for the treatment of hepatitis C and that

merimepodib and VX-950 each have the potential to address significant unmet

needs in HCV treatment; (ii) merimepodib and VX-950 each hold promise as

part of combination therapy for HCV patients who have limited treatment

options and represents an attractive commercial opportunity for Vertex;

(iii) further clinical study of merimepodib will be initiated in 2004; and

(iv) clinical study of VX-950 will be initiated in 2004. While management

makes its best efforts to be accurate in making forward-looking statements,

such statements are subject to risks and uncertainties that could cause

Vertex's actual results to vary materially. These risks and uncertainties

include, among other things, the risks that clinical trials for merimepodib

or VX-950 may not be initiated or, if initiated, may not proceed as planned

due to technical, scientific, or patient enrollment issues, that results

from planned clinical trials with merimepodib will not reflect the positive

results from earlier trials, that positive nonclinical study results for

either merimepodib or VX- 950 will not be duplicated in future nonclinical

or clinical studies and other risks listed under Risk Factors in Vertex's

form 10-K filed with the Securities and Exchange Commission on March 15,

2004. Lexiva is a registered trademark of the GlaxoKline group of

companies.

Vertex's press releases are available at http://www.vrtx.com/.

Vertex Pharmaceuticals Incorporated will host a conference call on Monday,

April 26, 2004 at 5:00 pm (EDT) to discuss its first quarter 2004 financial

results, at which time it will review and update its HCV product development

programs. The conference call also will be webcast, and the webcast will be

available to all interested parties through Vertex's website,

http://www.vrtx.com/. To access the webcast, go to the investor center and

select " conference calls. " To ensure a timely connection to the webcast, it

is recommended that users register at least 15 minutes prior to the

scheduled webcast.

Vertex Contacts: Partridge, Director, Corporate Communications (617)

444-6108 Lora Pike, Manager, Investor Relations (617) 444-6755

Vertex Pharmaceuticals Incorporated

CONTACT: Partridge, Director, Corporate Communications,

+1-617-444-6108, or Lora Pike, Manager, Investor Relations, +1-617-444-6755,

both of Vertex Pharmaceuticals Incorporated

Web site: http://www.vrtx.com/

Company News On-Call: http://www.prnewswire.com/comp

/938395.html