Miracle Mineral Solution (MMS): Product as consumed produces a potent bleach

[Posted 07/30/2010]

AUDIENCE: Consumers, Emergency Medicine

ISSUE: FDA warned consumers not to consume or use Miracle Mineral Solution, an oral liquid solution also known as "Miracle Mineral Supplement" or "MMS." The product, when used as directed, produces an industrial bleach that can cause serious harm to health. The product instructs consumers to mix the 28 percent sodium chlorite solution with an acid such as citrus juice. This mixture produces chlorine dioxide, a potent bleach used for stripping textiles and industrial water treatment. High oral doses of this bleach, such as those recommended in the labeling, can cause nausea, vomiting, diarrhea, and symptoms of severe dehydration.

BACKGROUND: MMS is distributed on Internet sites and online auctions by multiple independent distributors. MMS claims to treat multiple unrelated diseases, including HIV, hepatitis, the H1N1 flu virus, common colds, acne, cancer, and other conditions. The FDA is not aware of any research that MMS is effective in treating any of these conditions. MMS also poses a significant health risk to consumers who may choose to use this product for self-treatment instead of seeking FDA-approved treatments for these conditions.

RECOMMENDATIONS: Consumers who have MMS should stop using it immediately and throw it away. The FDA advises consumers who have experienced any negative side effects from MMS to consult a health care professional as soon as possible.

Healthcare professionals and patients are encouraged to report adverse events or side effects related to the use of this product to the FDA's MedWatch Safety Information and Adverse Event Reporting Program:

Online: www.fda.gov/MedWatch/report.htm
Phone: 1-800-332-1088
Mail: return the postage-paid FDA form 3500, which may be downloaded from the MedWatch "Download Forms" page, to address on the pre-addressed form
Fax: 1-800-FDA-0178

[07/30/2010 - News Release - FDA]

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Improving the Inhibitory Control Task to Detect Minimal Hepatic Encephalopathy

Gastroenterology
Volume 139, Issue 2 , Pages 510-518.e2, August 2010

Piero Amodio, Lorenzo Ridola, Sami Schiff, Sara Montagnese, Chiara Pasquale, Silvia Nardelli, Ilaria Pentassuglio, Maria Trezza, Chiara Marzano, Cristiana Flaiban, Paolo Angeli, Giorgio Cona, Patrizia Bisiacchi, Angelo Gatta, Oliviero Riggio

Received 31 December 2009; accepted 29 April 2010. published online 13 May 2010.

Abstract

Background & Aims
Quantification of the number of noninhibited responses (lures) in the inhibitory control task (ICT) has been proposed for the diagnosis of minimal hepatic encephalopathy (MHE). We assessed the efficacy of ICT compared with recommended diagnostic standards.

Methods
We studied patients with cirrhosis and healthy individuals (controls) who underwent the ICT at 2 centers (center A: n = 51 patients and 41 controls, center B: n = 24 patients and 14 controls). Subjects were evaluated for MHE by psychometric hepatic encephalopathy score (PHES). Patients from center B also were assessed for MHE by critical flicker frequency and spectral electroencephalogram analyses.

Results
Patients with cirrhosis had higher ICT lures (23.2 ± 12.8 vs 12.9 ± 5.8, respectively, P < .01) and lower ICT target accuracy (0.88 ± 0.17 vs 0.96 ± 0.03, respectively, P < .01) compared with controls. However, lures were comparable (25.2 ± 12.5 vs 21.4 ± 13.9, respectively, P = .32) among patients with/without altered PHES (center A). There was a reverse, U-shaped relationship between ICT lure and target accuracy; a variable adjusting lures was devised based on target accuracy (weighted lures at center B). This variable differed between patients with and without MHE. The variable weighted lures was then validated from data collected at center A by receiver operator characteristic curve analysis; it discriminated between patients with and without PHES alterations (area under the curve = 0.71 ± 0.07). However, target accuracy alone was as effective as a stand-alone variable (area under the curve = 0.81 ± 0.06).

Conclusions
The ICT is not useful for the diagnosis of MHE, unless adjusted by target accuracy. Testing inhibition (lures) does not seem to be superior to testing attention (target accuracy) for the detection of MHE.

Keywords: Electroencephalogram, EEG, Attention, Inhibition

Abbreviations used in this paper: CFF, critical flicker frequency, EEG, electroencephalogram, ICT, inhibitory control task, HE, hepatic encephalopathy, LTT, line tracing test, MHE, minimal hepatic encephalopathy, PHES, psychometric hepatic encephalopathy score, ROC, receiving operative curves, TMT, trail making test, WL, weighted lures.

Conflicts of interest The authors disclose no conflicts.

Funding Supported by grants of the University of Padua (to P.A.) and of the University of Rome (to O.R.) and by a “Young Investigator Award” given (to L.R.) by the Italian Society of Gastroenterology (SIGE).

PII: S0016-5085(10)00677-3

doi:10.1053/j.gastro.2010.04.057

© 2010 AGA Institute. Published by Elsevier Inc. All rights reserved.

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Hepatic ISG Expression Is Associated With Genetic Variation in Interleukin 28B and the Outcome of IFN Therapy for Chronic Hepatitis C

Gastroenterology
Volume 139, Issue 2 , Pages 499-509, August 2010
 
Masao Honda, Akito Sakai, Tatsuya Yamashita, Yasunari Nakamoto, Eishiro Mizukoshi, Yoshio Sakai, Taro Yamashita, Mikiko Nakamura, Takayoshi Shirasaki, Katsuhisa Horimoto, Yasuhito Tanaka, Katsushi Tokunaga, Masashi Mizokami, Shuichi Kaneko, Hokuriku Liver Study Group

Received 9 October 2009; accepted 14 April 2010. published online 30 April 2010.

Abstract

Background & Aims
Multiple viral and host factors are related to the treatment response to pegylated-interferon and ribavirin combination therapy; however, the clinical relevance and relationship of these factors have not yet been fully evaluated.

Methods
We studied 168 patients with chronic hepatitis C who received pegylated-interferon and ribavirin combination therapy. Gene expression profiles in the livers of 91 patients were analyzed using an Affymetrix genechip (Affymetrix, Santa Clara, CA). The expression of interferon-stimulated genes (ISGs) was evaluated in all samples by real-time polymerase chain reaction. Genetic variation in interleukin 28B (IL28B; rs8099917) was determined in 91 patients.

Results
Gene expression profiling of the liver differentiated patients into 2 groups: patients with up-regulated ISGs and patients with down-regulated ISGs. A high proportion of patients with no response to treatment was found in the up-regulated ISGs group (P = .002). Multivariate logistic regression analysis showed that ISGs (<3.5) (odds ratio [OR], 16.2; P < .001), fibrosis stage (F1-F2) (OR, 4.18; P = .003), and ISDR mutation (≥2) (OR, 5.09; P = .003) were strongly associated with the viral response. The IL28B polymorphism of 91 patients showed that 66% were major homozygotes (TT), 30% were heterozygotes (TG), and 4% were minor homozygotes (GG). Interestingly, hepatic ISGs were associated with the IL28B polymorphism (OR, 18.1; P < .001), and its expression was significantly higher in patients with the minor genotype (TG or GG) than in those with the major genotype (TT).

Conclusions
The expression of hepatic ISGs is strongly associated with treatment response and genetic variation of IL28B. The differential role of host and viral factors as predicting factors may also be present.

Keywords: Pegylated Interferon, Ribavirin, Gene Expression, Single Nucleotide Polymorphism

Abbreviations used in this paper: aa, amino acid, AST, aspartate aminotransferase, cDNA, complementary DNA, CH-C, chronic hepatitis C, Down-ISGs, down-regulated ISGs, EVR, early virologic response, GWAS, genome-wide association studies, HCV, hepatitis C virus, IFN, interferon, IFI44, interferon-induced protein 44, IFIT1, interferon-induced protein with tetratricopeptide repeats 1, IL, interleukin, IL28B, interleukin 28B, ISDR, interferon sensitivity determining region, ISGs, interferon stimulated genes, Mx1, myxovirus (influenza virus) resistance 1 interferon-inducible protein p78 (mouse), NR, no response, Peg, pegylated, RBV, ribavirin, ROC, receiver operating characteristic, RTD, real-time detection, PCR, polymerase chain reaction, RTD-PCR, real-time detection-polymerase chain reaction, SNP, single nucleotide polymorphism, SVR, sustained viral response, TR, transient response, Up-ISGs, up-regulated ISGs

Participating investigators are listed in Appendix 1.

Conflicts of interest The authors disclose no conflicts.

Funding This work was supported in part by a grant-in-aid from the Ministry of Health, Labour and Welfare of Japan.

PII: S0016-5085(10)00657-8

doi:10.1053/j.gastro.2010.04.049

© 2010 AGA Institute. Published by Elsevier Inc. All rights reserved.

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Chronic Hepatitis C Virus Infection Is Associated with Early Atherosclerosis

SUMMARY: People with chronic hepatitis C are more likely than those never infected to have diabetes and visceral fat accumulation, but have lower levels of LDL "bad" cholesterol, according to a study described in the June 28, 2010 advance online issue of Gut. After HCV clearance, LDL levels rose to match those of never-infected individuals, but glucose levels and abdominal fat remained similar. After adjusting for other risk factors, chronic hepatitis C patients had greater carotid intima-media thickness, an indicator of the early stages of atherosclerosis.

By Liz Highleyman

Previous research has found that people with chronic hepatitis C virus (HCV) infection have an elevated risk for cardiovascular disease. While HCV has been linked to insulin resistance or diabetes -- known risk factors for heart disease -- it is also typically characterized by favorable blood lipid levels.

In the present study, Aya Mostafa from AinShams University in Cairo and colleagues looked at the effect of this paradoxical risk profile on metabolism and atherosclerosis (hardening of the arteries and build-up of plaque) in the setting of HCV infection and clearance.

This cross-sectional analysis included more than 1200 participants in Egypt aged 35 years or older. Within this study population, 329 had chronic hepatitis C, 173 had cleared HCV infection, and 795 were never infected with HCV; a subset of 192, 115, and 187 participants, respectively, from the 3 groups underwent ultrasound imaging.

The investigators evaluated presence of diabetes, fasting blood glucose, lipid levels, and body fat deposition using ultrasound. Carotid intima-media thickness (IMT), or width of the inner lining of the carotid arteries supplying the brain, was used as a measure of atherosclerosis.

Results

• Diabetes was more common among participants with chronic hepatitis C and cleared HCV infection (both with a prevalence of 10.1%) than among those who never had HCV (6.6%; P = 0.04 for chronic, 0.08 for cleared).
• The amount of mesenteric or visceral fat was greater in people with chronic hepatitis C (36.4 mm) and cleared infection (37.8 mm) relative to those never infected (32.7 mm; P = 0.004 for chronic, < 0.0001 for cleared).
• Low-density lipoprotein (LDL) cholesterol levels were lower in the chronic hepatitis C group (2.69 mmol/L; P < 0.001), but similar in those with cleared infection (3.56 mmol/L; P = 0.4) and those never infected (3.45 mmol/L).
• Carotid IMT did not differ significantly according to HCV infection status, at 0.73, 0.71, and 0.71 mm, respectively.
• After adjustmenting for traditional cardiovascular risk factors, however, IMT was greater in peoplewith chronic infection (0.76 mm) compared with never infected individuals (0.70 mm; P = 0.02).

Based on these findings, the researchers proposed, "Hepatic function normalization with HCV clearance may account for reversal of favorable lipids observed with HCV infection." However, they added, glucose levels and visceral fat accumulation "appear less amenable to HCV resolution."

"These different cardiovascular risk patterns may determine equivalent atherosclerosis risk by infection status," they suggested. "However, once these factors were accounted for, those with chronic infection had raised IMT, suggesting a direct effect of infection."

Department of Community Medicine, Faculty of Medicine, AinShams University, Cairo, Egypt; Department of Tropical Medicine, Faculty of Medicine, Cairo University, Cairo, Egypt; Viral Hepatitis Reference Laboratory, National Hepatology and Tropical Medicine Research Institute, Cairo, Egypt; Institut Pasteur, Paris, France; Department of Metabolic Medicine, Imperial College NHS Healthcare Trust, London, UK; International Centre for Circulatory Health, National Heart & Lung Institute, Imperial College NHS Healthcare Trust, London, UK; Faculty of Medicine, Minia University, Minia, Egypt.

7/30/10

Reference

A Mostafa, MK Mohamed, M Saeed, and others. Hepatitis C infection and clearance: impact on atherosclerosis and cardiometabolic risk factors. Gut (Abstract). June 28, 2010 (Epub ahead of print).

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Organ donor shortage makes survival a long shot

 By Sandra V. Rodriguez • srodriguez@citizen-times.com • July 30, 2010

ASHEVILLE — Julie Wallace always said yes to organ donation.

But in recent years, the little heart on her driver's license has held deeper meaning because she came so close to losing her 12-year-old son, Jacob. Both of his kidneys failed nine years ago, when Jacob was 3.

“It was a nightmare,” she said. “I literally just prayed every night that he would make it. It's a rough thing when you're waiting for a transplant for a loved one because you know that someone else's loved one has to pass away…”

That someone was a woman who suffered a fatal aneurysm while exercising. She left behind children and a husband, who consented to donate her organs. The woman gave at least seven people a second chance at life.

There are more than 3,300 North Carolinians included in the transplant waiting. Unfortunately for many, the number of donors isn't keeping up.

Kirk Truesdale was diagnosed with hepatitis C five years ago, but doctors didn't catch it in time to save his liver. Truesdale has been on the transplant waiting list for nearly three years. At one time, he was moved to hospice care because of how sick he became. His wife, Shirley, even began making funeral arrangements.

“A lot of people don't understand the things that you go through when you're waiting for a transplant,” Shirley said. “You're dealing with a very sick person that if you let yourself think about a lot, you'll feel like the sand is running out in the glass.”

Truesdale knows his sickness is harder on his wife and 12-year-old daughter, Keeley, than it is on him. The ammonia buildup in the one-time ironworker's body often leads to his legs or belly swelling up with fluid. This causes what Shirley describes as Alzheimer's-like behavior in Truesdale, who can get combative or ramble endlessly or wander off during these spells.

“It's been hell,” he said. “There are times when you want to just say to God, ‘Just take me.'”

The state has made strides to increase donor registration in recent years by making it easier to register in person at the Department of Motor Vehicles or online or at DonateLifeNC.org, and passing the Heart Prevails law in 2007. It turned the heart on the driver's license from a symbol of intent to donate to legal consent for organ and eye

“In the past, I think people realized it didn't mean a whole lot because they knew the family could override it,” Melissa Parker, organ procurement specialist with LifeShare of the Carolinas' office in Asheville. The nonprofit provides tissues and organs for transplantation for people in southwestern North Carolina. “But now that heart means something.”

Parker has worked with donors for the last 15 years, and she's never seen the waiting list numbers go down. When she first started doing this job, there were about 30,000 people on the waiting list, and now it's nearly 108,000. The list increases by one every 12 minutes, according to Carolina Donor Services.

The situation is especially critical for kidney transplants for minorities who make up “disproportionately” high number of people on the waiting list, said Debbie Gibbs, with LifeShare in Charlotte.

It's largely because of high blood pressure and diabetes, which are more common in the African-American, Hispanic and Native American communities.

In North Carolina, 60 percent of the kidney transplant waiting list is made up of African-Americans as opposed to 35 percent nationwide. The state's donor rate for African-Americans was 26 percent in 2009.

Generally, Parker said race does not play a role in who gets an organ transplant. For any transplant, the most important factor is always blood type. But it would be best, in cases of kidney or pancreas transplants, that a person received the organ from someone who has similar tissue, she said.

“We never think that someone in our family or one of our close friends is going to need a transplant,” Wallace said. “He (Jacob) was born a completely healthy child, and some freak illness caused him to be very ill. He would have died, absolutely, at age 5 had we not gotten his transplant.”

Even now, eight years after the “nightmare” ended, the fear and worry are never far. Wallace still gets anxious when Jacob shows signs of fever.

That's something new parents Larry and Meghan Robertson understand intimately. The couple's 7-month-old baby, Ty, was born without a right kidney, and the left one is only one-third its normal size. The doctors had very little hope that he would make it. It was devastating news for the couple, who had spun so many dreams in the last nine months.

“It's not what any mother looks for when she says, ‘I'm pregnant,'” Meghan said. “I didn't think about my baby being in NICU (neonatal intensive care unit) and going through all this.”

While families everywhere were waiting for Santa, Meghan spent last Christmas Eve in a Winston-Salem hospital praying that her baby would make it through the night.

But what a difference a few months makes. With the help of a dialysis machine, 7-month-old Ty has shown strength of will that makes his parents hopeful that he will hold on long enough to get on the national organ transplant waiting list.

It's hard to tell how long the wait will be for Ty once he gets on the list. He needs to weigh 22 pounds before that happens. But the Robertsons' family and friends are hoping to speed the process along by getting tested to see if there is a match.

“I want to see him grow up,” Meghan said. “I want to see him go to school. Go to college. Get married and have my grandbabies. I want to see all that, and I hope that he is more than able to do it.”

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4 vets positive for hepatitis after St. Louis clinic visit

ST. LOUIS — Four veterans treated at the St. Louis VA Medical Center's dental clinic have tested positive for hepatitis, but further testing is needed to determine if inadequately sterilized dental equipment is to blame.

The Veterans Administration provided test results on Friday to The Associated Press.

The VA last month sent letters to 1,812 veterans treated at the clinic from Feb. 1, 2009, through March 11, 2010, urging them to take blood tests because the cleaning errors could have exposed them to hepatitis B, hepatitis C or HIV.

The VA says the risk of infection is remote.

The VA says 1,022 veterans have been tested and notified of results. Of those, two tested positive for hepatitis B, two tested positive for hepatitis C, and none tested positive for HIV.

Copyright 2010 The Associated Press.

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Also See:

• Military men and women suffer abuse at the hands of their own doctors
• VA hospital may have infected 1,800 veterans with HIV

ANA598 Demonstrates SVR12 in 100% of First Group of HCV Patients Randomized to Stop All Treatment at Week 24

Benefit of ANA598 Post Therapy with IFN/RBV Persists in 6 of 6 Patients

Company to Review Data During Q2 Financial Results Call at 8:30 AM EDT Today

SAN DIEGO, July 29 /PRNewswire-FirstCall/ -- Anadys Pharmaceuticals, Inc. (Nasdaq: ANDS) today announced that six of six patients (100%) in the ANA598 200 mg twice daily (bid) arm who were randomized to stop all treatment at Week 24 in an ongoing Phase II trial maintained undetectable levels of virus 12 weeks after stopping treatment, referred to as Sustained Virological Response 12, or SVR12.

The Company also reported that all available patients from the ANA598 200 mg arm who were previously reported to have undetectable levels of virus at Week 24 and continued on pegylated interferon and ribavirin (current standard of care, or SOC) also maintained undetectable levels of virus at Week 36. In addition, all patients from the ANA598 400 mg arm who were previously reported to have undetectable levels of virus at Week 12 and continued on SOC maintained undetectable levels of virus at Week 24. ANA598, Anadys' direct-acting antiviral or DAA, is being developed to treat hepatitis C and is in an ongoing Phase II trial in combination with pegylated interferon and ribavirin.

"The SVR12 data reported today for ANA598 are highly encouraging," said Steve Worland, Ph.D., President and CEO of Anadys. "These data illustrate the potential for HCV patients to be successfully treated with shortened courses of treatment, reflecting the continuing benefit of ANA598 post-therapy. We believe these data, coupled with the excellent barrier to resistance demonstrated in this trial as well as the favorable safety and tolerability, confirm ANA598's position as one of the most attractive agents in Phase II HCV development today."

The six patients who stopped all treatment at Week 24 were part of an investigation of response-guided treatment duration for ANA598 in which patients who had achieved undetectable levels of virus (<15 IU/mL) at Weeks 4 and 12 were randomized 1:1 to stop all treatment at Week 24 or Week 48. In addition to the six patients who stopped treatment at Week 24, six patients in the 200 mg bid arm are continuing to receive SOC alone through Week 48 for comparison purposes. Additionally, 14 patients from the ANA598 400 mg bid arm and 4 patients from the control arm (receiving placebo plus SOC) met the stopping criteria and have been randomized to stop all treatment at Week 24 or 48. The initial post-treatment results from these latter arms are expected later this year for those patients who stopped therapy at Week 24.

Conference Call Webcast and Slides

Anadys will hold a conference call and webcast today, Thursday, July 29, 2010 at 8:30 a.m. Eastern Daylight Time to discuss the post-treatment results from the ongoing Phase II combination study and Anadys' second quarter 2010 financial results A live webcast of the call, including accompanying slides, will be available online at www.anadyspharma.com. A telephone replay with slides will also be available approximately one hour after completion of the call. To access the telephone replay, dial 888-286-8010 (domestic) or 617-801-6888 (international), passcode 28631163. The webcast and telephone replay will be available through August 12, 2010.

Phase II Combination Study

In the ongoing Phase II study, approximately 90 treatment-naive genotype 1 HCV patients have received ANA598 or placebo in combination with Pegasys® (peginterferon alfa-2a) and Copegus® (ribavirin, USP) for 12 weeks at dose levels of 200 mg bid or 400 mg bid, each with a loading dose of 800 mg bid on day one. After week 12, patients are to continue receiving SOC. Patients who achieved undetectable levels of virus at weeks 4 and 12 were randomized to stop all treatment at week 24 or 48. The primary endpoint of the study is the proportion of patients who achieve undetectable levels of virus at week 12 (defined as complete Early Virological Response, or cEVR). Additional endpoints include safety and tolerability as well as the proportion of patients with undetectable levels of virus at week 4 (defined as Rapid Virological Response, or RVR). Patients will be followed for 24 weeks after stopping therapy to determine the rate of Sustained Virological Response, or SVR. Approximately 90 patients have been enrolled in this study – with approximately 30 patients receiving ANA598 plus SOC at each dose level and 30 patients receiving placebo plus SOC. The study is being managed by the Duke Clinical Research Institute (DCRI) and is being conducted at a number of clinical sites in the United States.

About ANA598

ANA598, a direct-acting antiviral or DAA, is a non-nucleoside inhibitor of the HCV RNA polymerase and is wholly owned by Anadys. In an ongoing Phase II study in which HCV patients received ANA598 at 200 mg bid or 400 mg bid in combination with interferon and ribavirin for twelve weeks, both dose levels showed comparable cEVR rates of 73-75% and a favorable safety profile. In a previous Phase I study, ANA598 demonstrated potent antiviral activity, including median end-of-treatment declines in viral load ranging from 2.4 to 2.9 log10 in a three day monotherapy study in treatment-naïve genotype 1 patients. ANA598 has also demonstrated a very favorable resistance profile.

Anadys has completed two long-term chronic toxicology studies of ANA598 (26 weeks duration in rats and 39 weeks duration in monkeys). The No Observed Adverse Effect Level, or NOAEL, is 1000 mg/kg, the highest dose tested, in both the rat and monkey. The completed toxicology studies support the ongoing Phase II clinical study as well as future clinical studies of longer duration.

Anadys has presented in vitro data supporting the use of ANA598 in combination with interferon-alpha as well as with other anti-HCV agents currently in development that act through diverse mechanisms. In particular, data has shown that ANA598 is synergistic in vitro with interferon-alpha as well as representative HCV protease inhibitors, polymerase inhibitors, NS5A inhibitors and cyclophilin inhibitors. In vitro combination treatment at clinically relevant concentrations of ANA598 with interferon-alpha as well as DAAs from multiple classes results in clearance of HCV RNA from cells rather than selection of resistant isolates. Furthermore, ANA598 retains full activity in vitro against mutations conferring resistance to protease inhibitors, nucleoside polymerase inhibitors and non-nucleoside polymerase inhibitors that act at binding sites distinct from that of ANA598, while protease and nucleoside polymerase inhibitors retain full activity in vitro against mutations conferring resistance to ANA598.

ANA598 has received Fast Track Status from the FDA for the treatment of chronic hepatitis C.

Safe Harbor Statement

Statements in this press release that are not strictly historical in nature constitute "forward-looking statements." Such statements include, but are not limited to, references to (i) ANA598's initial SVR12 profile based on the results from the six patients in this first group; (ii) the potential for HCV patients to be successfully treated with shortened courses of treatment, reflecting the continuing benefit of ANA598 post-therapy; (iii) the belief that ANA598 is one of the most attractive agents in Phase II HCV development today; (iv) the expected timing for post-treatment results from the other dose groups; and (v) the ability for patients to achieve an SVR in the Phase II combination study. Such forward-looking statements involve known and unknown risks, uncertainties and other factors, which may cause Anadys' actual results to be materially different from historical results or from any results expressed or implied by such forward-looking statements. For example, the results of preclinical and early clinical studies may not be predictive of future results, and Anadys cannot provide any assurances that ANA598 will not have unforeseen safety issues or will continue to have favorable results as the Phase II trial progresses. In addition, Anadys' results may be affected by competition from other biotechnology and pharmaceutical companies, its effectiveness at managing its financial resources, its ability to enter into transactions around its product candidates, its ability to successfully develop and market products, difficulties or delays in its preclinical studies or clinical trials, difficulties or delays in manufacturing its clinical trials materials, the scope and validity of patent protection for its products, regulatory developments and its ability to obtain additional funding to support its operations. Risk factors that may cause actual results to differ are more fully discussed in Anadys' SEC filings, including Anadys' Form 10-K for the year ended December 31, 2009, Form 10-Q for the quarter ended March 31, 2010 and Form 8-K filed on May 26, 2010. All forward-looking statements are qualified in their entirety by this cautionary statement. Anadys is providing this information as of this date and does not undertake any obligation to update any forward-looking statements contained in this document as a result of new information, future events or otherwise.

Pegasys® and Copegus® are registered trademarks of Hoffman-La Roche Inc.

SOURCE Anadys Pharmaceuticals, Inc.

RELATED LINKS
http://www.anadyspharma.com/

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On the street, you can see the harm caused by drug laws

By David Bratzer, Citizen Special July 29, 2010

Like many other police officers, I have witnessed the tragedy of the HIV epidemic first hand. It is one thing to read the statistics demonstrating the connection between illicit drug use and HIV; it is another matter entirely to patrol the streets, day in and day out, repeatedly arresting men and women infected with the HIV virus.

Our country has one of the finest health-care systems in the world, but our laws surrounding drug use result in unnecessary disease and death.

In this context, the recent announcement of the Vienna Declaration has bolstered my conviction that drug prohibition is a national policy failure.

The document, inspired by an international team of leading health scientists and academic physicians, is the official declaration of this month's International AIDS Conference in Vienna. It presents an important scientific fact that I see reflected in my work every day: "The criminalization of illicit drug users is fuelling the HIV epidemic and has resulted in overwhelmingly negative health and social consequences."

The declaration calls for a "full policy reorientation." This should not be misconstrued as an endorsement of drug use. It is simply a recognition that drug law enforcement is not an effective deterrent, citing studies showing "there is no evidence that increasing the ferocity of law enforcement meaningfully reduces the prevalence of drug use."

It is also a recognition that drug law enforcement is contributing directly to the HIV epidemic. In most parts of the world, approximately one in three HIV infections can be traced back to intravenous drug use. Toronto, Ottawa, Surrey, Winnipeg and other Canadian cities are not immune.

Drug prohibition increases the rate of HIV infections. When illegal drugs are sold through the black market, the only concern is making money. There is no financial incentive for traffickers to provide drug education, counselling or harm reduction services such as sterile needles.

In addition, in parts of Canada it is common for an injection drug user to be arrested for a minor drug charge and end up with a court-imposed condition to abstain from possessing drug paraphernalia. Addicts are then forced to choose whether to carry sterile needles and risk a new criminal charge, or to share a needle with another addict who may already have a blood-borne disease.

The Vienna Declaration is particularly important within Canada. Bill S-10 is before Parliament. It is the federal government's third attempt in as many years to create mandatory minimum sentences for certain drug offences.

The wording of this legislation virtually guarantees that street-level drug addicts will find themselves going to jail for lengthy prison terms. This will do nothing but channel limited tax dollars away from health and education and into costly incarceration policies which will turn petty drug users into hardened criminals.

HIV prevention efforts will be hampered if this bill passes. The HIV infection rates in federal prisons are similar to some African countries, according to the statistics provided by the Correctional Service of Canada. So, for many of these addicts, part of their sentence will include a substantial risk of contracting HIV or Hepatitis C.

Of course, the Vienna Declaration is not the first time a major initiative has been announced to coincide with the biannual International AIDS Conference. Ten years ago, the Durban Declaration stated a basic scientific truth: that HIV is the cause of AIDS. More than 5,000 scientists and medical doctors signed the document in an effort to confront AIDS denialism.

The main critic of the Durban Declaration was Thabo Mbeki, who was president of South Africa at the time. He believed, mistakenly, that there was a causal link between poverty and AIDS. In fact it is HIV that causes AIDS. His denials, rooted in ignorance and willful blindness, have cost many lives in South Africa.

Given this history, it will be interesting to see who opposes the Vienna Declaration. Police lobby groups have traditionally been the most vocal critics of drug policy reform. In this instance, however, they should choose their responses carefully. At stake is the very credibility of these organizations. They risk being remembered in the same light as President Mbeki, for it is clear that the new AIDS denialism is the failure to acknowledge the realities of HIV transmission.

The Vienna Declaration will play a significant role in HIV policy, and I am proud to have signed the document online at viennadeclaration.com.

I can only hope that my colleagues in law enforcement will be inspired to do the same.

David Bratzer is a member of the board of directors for Law Enforcement Against Prohibition and a police officer in British Columbia. The opinions expressed in this column do not represent the views of his employer.

© Copyright (c) The Ottawa Citizen

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RNA Offers a Safer Way to Reprogram Cells

MIT researchers used RNA to induce these fibroblast cells to express four genes necessary to reprogram cells to an immature state. (Credit: Yanik Laboratory, MIT)

ScienceDaily (July 29, 2010) — In recent years, scientists have shown that they can reprogram human skin cells to an immature state that allows the cells to become any type of cell. This ability, known as pluripotency, holds the promise of treating diseases such as diabetes and Parkinson's disease by transforming the patients' own cells into replacements for the nonfunctioning tissue.

However, the techniques now used to transform cells pose some serious safety hazards. To deliver the genes necessary to reprogram cells to a pluripotent state, scientists use viruses carrying DNA, which then becomes integrated into the cell's own DNA. But this so-called DNA-based reprogramming carries the risk of disrupting the cell's genome and leading it to become cancerous.


Now, for the first time, MIT researchers have shown that they can deliver those same reprogramming genes using RNA, the genetic material that normally ferries instructions from DNA to the cell's protein-making machinery. This method could prove much safer than DNA-based reprogramming, say the researchers, Associate Professor of Electrical and Biological Engineering Mehmet Fatih Yanik and electrical engineering graduate student Matthew Angel.

Yanik and Angel describe the method, also the subject of Angel's master's thesis, in the July 23 issue of the journal PLoS ONE.

However, the researchers say they cannot yet claim to have reprogrammed the cells into a pluripotent state. To prove that, they would need to grow the cells in the lab for a longer period of time and study their ability to develop into other cell types -- a process now underway in their lab. Their key achievement is demonstrating that the genes necessary for reprogramming can be delivered with RNA.

"Before this, nobody had a way to transfect cells multiple times with protein-encoding RNA," says Yanik. (Transfection is the process of introducing DNA or RNA into a cell without using viruses to deliver them.)

In 2006, researchers at Kyoto University showed they could reprogram mouse skin cells into a pluripotent, embryonic-like state with just four genes. More recently, other scientists have achieved the same result in human cells by delivering the proteins encoded by those genes directly into mature cells, but that process is more expensive, inefficient and time-consuming than reprogramming with DNA.

Yanik and Angel decided to pursue a new alternative by transfecting cells with messenger RNA (mRNA), a short-lived molecule that carries genetic instructions copied from DNA.

However, they found that RNA transfection poses a significant challenge: When added to mature human skin cells, mRNA provokes an immune response meant to defend against viruses made of RNA. Repeated exposure to long strands of RNA leads cells to undergo cell suicide, sacrificing themselves to help prevent the rest of the body from being infected.

Yanik and Angel knew that some RNA viruses, including hepatitis C, can successfully suppress that defensive response. After reviewing studies of hepatitis C's evasive mechanisms, they did experiments showing they could shut off the response by delivering short interfering RNA (siRNA) that blocks production of several proteins key to the response.

Once the defense mechanism is shut off, mRNA carrying the genes for cell reprogramming can be safely delivered. The researchers showed that they could induce cells to produce the reprogramming proteins for more than a week, by delivering siRNA and mRNA every other day.

Source
 
Also See: RNA offers a safer way to reprogram cells

Greater Risk Of Diabetes Found In Hepatitis C Patients

By Steven Marsh • Jul 28th, 2010 • Category: Blood Sugar, Health News, Health Resources News

Patients who have been diagnosed with hepatitis C may be more susceptible to developing type 2 diabetes, according to a study published in the journal Gastroenterology.

A blood-born disease, hepatitis C spreads through unprotected sexual intercourse or the use of injection drugs and causes liver damage. If people don’t receive treatments for this disease, they can suffer from liver cancer or failure of the organ, resulting in death.

During the study, a team of investigators monitored a total of 29 patients with hepatitis C who showed signs of insulin resistance, a symptom of diabetes.

The results of the trial showed that 15 of the participants experienced insulin complications in their muscle tissue compared to the liver. The inability to properly absorb the sugar created high levels of the nutrient in the blood, which could lead to developing diabetes.

"At this stage, it is helpful for people with hepatitis C to understand insulin resistance and what it can mean for them," said Don Chisholm, co-author of the study. He added that "if they have relatives with type 2 diabetes, they will be genetically prone to developing it themselves and so would be advised to manage their diets very carefully and take plenty of exercise – to slow onset."

As of 2007, an estimated 17,000 new cases of hepatitis C are diagnosed in the U.S. each year, according to the Centers for Disease Control and Prevention.

Source

Bid To Aid Transplant Cancer Patients

Article Date: 29 Jul 2010 - 1:00 PDT

Organ transplant patients who develop cancer may be helped by a treatment that uses blood cells to attack their tumour.

University of Edinburgh researchers have generated a bank of white blood cells from healthy blood donors to treat patients with a blood cancer called post transplant lymphoproliferative disease (PTLD).

The study found that patients treated with these blood cells - called 'killer' T cells - remained free from the cancer for up to nine years following treatment.

PTLD is associated with Epstein-Barr virus (EBV), a herpes virus that is carried by more than 90 per cent of the population and better known for causing glandular fever.

In most individuals the virus does not cause any illness but it can cause tumours in transplant patients.

This is because their immune systems are heavily suppressed to prevent rejection of the transplanted organ.

Up to 10 per cent of transplant patients may develop the cancer in the first few years following transplant and around 50 per cent of those will die even with standard treatment.

T cells are a type of white blood cell that patrol the body identifying and killing virus infected cells.

A team at the University of Edinburgh's Centre for Infectious Diseases grew T cells in the laboratory and gave them to PTLD patients for one month.

The T cells were programmed to find and kill the virus-infected tumour cells to reduce or eradicate the tumour.

A total of 33 PTLD patients who had not responded to standard treatments were treated in a Cancer Research UK-funded trial.

Around half the treated patients showed a good response after six months.

This latest study shows that 90 per cent of those who responded initially have remained cancer free for between four and nine years.

The long term survival rate was significantly better in the six month responder group compared to the six month non-responder group.

"Our results are very encouraging and show that not only are our cells effective in the short term but that they can also induce a long term remission of PTLD in patients with more unmanageable disease."

said Dr Tanzina Haque, Lead researcher, now of Royal Free Hospital, UCL Medical School.

The Edinburgh researchers in conjunction with the Scottish National Blood Transfusion Service have obtained translational funding from the Wellcome Trust to make a new bank of 'killer' T cells that will become available for use on a not-for-profit basis in the next few years.

The research is published in the journal Transplantation.

Source:
University of Edinburgh

Source

Hepatitis C retreatment offers hope if initial one fails

by paige varner pvarner@post-dispatch.com 314-340-8018

Posted: Thursday, July 29, 2010 12:00 am
 
For the 40 percent to 50 percent of patients who aren't cured of hepatitis C after their initial treatment, Dr. Bruce R. Bacon of St. Louis University and his team of researchers have increased the chances of a cure upon re-treatment.
 
About 11 percent of the 515 people retreated in Bacon's trials were cured using the Food and Drug Administration-approved medication combination, Infergen with an antiviral pill.
 
Of those with milder strains of hepatitis C, about 35 percent in the trials were cured.
 
In the first round of treatment, patients are given weekly shots of an alpha interferon — in larger amounts than that which the body normally makes to fight pathogens — along with the antiviral pill ribavirin.

This combination cures 50 percent to 60 percent of cases, but others can start another round, this time injecting the interferon medicine Infergen daily, along with taking the ribavirin, for up to 48 weeks.

Bacon, who holds the endowed chair in gastroenterology at SLU School of Medicine, and his team published their findings last summer after researching from 2004 to 2006.

For those with the virus who feel the 11 percent cure rate in the second round isn't high enough, Bacon said they will still have hepatitis C if they don't at least try re-treatment.

Another concern about the medicine combination is the side effects, which are similar to the flulike symptoms the virus already causes, such as fatigue, anemia, troubled breathing, irritability.

"But more than the side effects, you don't want to have hepatitis C," Bacon said.

Interferon and ribavirin are the only treatments for hepatitis C, which inflames the liver and after many years can lead to cirrhosis, or liver scarring, in 20 percent to 30 percent of patients.

Those with the virus can prevent cirrhosis development by not drinking excessive amounts of alcohol.

Some won't know they have the virus. Bacon said the flulike symptoms can set in gradually, making them feel sick for decades before they are diagnosed.

Contracting hepatitis C requires a blood-to-blood transmission, and Bacon said most cases have a history of one of three things:

• A blood transfusion before 1992.

• Sharing needles or illicit drugs — "even teenagers at a party who did something stupid," Bacon said

• Working in the health care field. Paramedics, physicians and nurses come into contact with blood daily. Just one needle stick could cause hepatitis C.

Acute cases of the virus can clear on their own. Infergen with ribavirin is FDA-approved in chronic cases, or for people who have had the virus for at least six months.

Infergen cannot be taken by anyone under 18 years old or with compensated liver disease due to cirrhosis complications.

Source

Middle school students co-author research on enzyme for activating promising disease-fighters

These computerized images show the innermost structure of a key bacterial enzyme that helps activate certain antibiotics and anti-cancer agents.

Public release date: 28-Jul-2010

Contact: Michael Bernstein
m_bernstein@acs.org
202-872-6042
American Chemical Society

Grown-ups aren't the only ones making exciting scientific discoveries these days. Two middle school students from Wisconsin joined a team of scientists who are reporting the first glimpse of the innermost structure of a key bacterial enzyme. It helps activate certain antibiotics and anti-cancer agents so that those substances do their job. Their study appears in ACS' weekly journal Biochemistry. The student co-authors of the study are from Edgewood Campus Middle School in Madison and participated in Project CRYSTAL, a special program that provides middle school students with hands-on laboratory experience.

In the report, study leader Hazel Holden and colleagues note intense scientific interest in a chemical process called methylation, which increases the activity of DNA, proteins, and other substances in the body by transferring methyl (CH3) groups to them. Special enzymes called methyltransferases make methylation possible, and these proteins are very important in a myriad of key biological processes.

Holden and colleagues studied a bacterial methyltransferase involved in the production of tetronitrose, a component of the promising anti-cancer agent, tetrocarcin, and the antibiotic kijanimicin. The methyltransferase seems to play a key role in activating these disease-fighters. The scientists identified the 3D structure of this methyltransferase, a key step in determining how it works and how it might be modified for potential use in medicine.

###

ARTICLE FOR IMMEDIATE RELEASE "Molecular Architecture of a C-3'-Methyltransferase Involved in the Biosynthesis of D-Tetronitrose"

DOWNLOAD FULL TEXT ARTICLE http://pubs.acs.org/stoken/presspac/presspac/full/10.1021/bi100782b

CONTACT:

Hazel Holden, Ph.D.
Department of Biochemistry
University of Wisconsin
Madison, Wisc. 53706
Phone: 608-262-4988
Fax: 608-262-1319
Email: Hazel_Holden@biochem.wisc.edu

Source

Vertex has begun telaprevir FDA application

UPDATE 1-Vertex has begun telaprevir FDA application

Wed Jul 28, 2010 4:41pm EDT

* Rolling telaprevir FDA application underway

* Adjusted Q2 loss of 71 cents per share

LOS ANGELES July 28 (Reuters) - Vertex Pharmaceuticals Inc (VRTX.O) on Wednesday projected a wider-than-expected 2010 net loss and said its U.S. regulatory application for experimental hepatitis C drug telaprevir is underway.

Vertex said it now expects a full-year net loss of about $750 million, compared with a previous estimate for a net loss of about $700 million to reflect the timing of non-cash expenses and revenue related to its 2009 financial transactions as well as potential milestone payments.

On an adjusted basis, the company said it still expects a loss of around $600 million for the year.

For the second quarter, Vertex posted a net loss of $200 million, or $1.00 per share, compared with a net loss of $171.3 million, or 99 cents per share, a year earlier.

Excluding one-time items, Vertex said it lost 71 cents a share for the quarter.

The company said it is on track to complete the telaprevir FDA application in the second half of this year.

Source
 
Also See: Vertex Pharmaceuticals Reports Second Quarter 2010 Financial Results and Highlights Recent Business and Clinical Progress

Genetic Risk Factors for Hepatopulmonary Syndrome in Patients With Advanced Liver Disease

Gastroenterology

Volume 139, Issue 1 , Pages 130-139.e24, July 2010

Kari E. Roberts, Steven M. Kawut, Michael J. Krowka, Robert S. Brown Jr, James F. Trotter, Vijay Shah, Inga Peter, Hocine Tighiouart, Nandita Mitra, Elizabeth Handorf, James A. Knowles, Steven Zacks, Michael B. Fallon

Received 2 December 2009; accepted 4 March 2010. published online 25 March 2010.

Abstract

Background & Aims
Hepatopulmonary syndrome (HPS) affects 10%–30% of patients with cirrhosis and portal hypertension and significantly increases mortality. Studies in experimental models indicate that pulmonary angiogenesis contributes to the development of HPS, but pathogenesis in humans is poorly understood. We investigated genetic risk factors for HPS in patients with advanced liver disease.

Methods
We performed a multicenter case-control study of patients with cirrhosis being evaluated for liver transplantation. Cases had an alveolar-arterial oxygen gradient ≥ 15 mm Hg (or ≥20 mm Hg if age > 64 years) and contrast echocardiography with late appearance of microbubbles after venous injection of agitated saline (intrapulmonary vasodilatation); controls did not meet both criteria for case status. The study sample included 59 cases and 126 controls. We genotyped 1086 common single nucleotide polymorphisms (SNPs) in 94 candidate genes.

Results
Forty-two SNPs in 21 genes were significantly associated with HPS after adjustments for race and smoking. Eight genes had at least 2 SNPs associated with disease: CAV3, ENG, NOX4, ESR2, VWF, RUNX1, COL18A1, and TIE1. For example, rs237872 in CAV3 showed an odds ratio of 2.75 (95% confidence interval: 1.65–4.60, P = .0001) and rs4837192 in ENG showed an odds ratio of 0.35 (95% confidence interval: 0.14–0.89, P = .027). Furthermore, variation in CAV3 and RUNX1 was associated with HPS in gene-based analyses.

Conclusions
Polymorphisms in genes involved in the regulation of angiogenesis are associated with the risk of HPS. Further investigation of these biologic pathways might elucidate the mechanisms that mediate the development of HPS in certain patients with severe liver disease.

Keywords: Genetic Polymorphism, Portal Hypertension

Abbreviations used in this paper: 95% CI, 95% confidence interval, AIM, Ancestry Informative Marker, CART, classification and regression trees, CAV3, Caveolin 3, COL18A1, collagen, type XVIII, α-1, D, linkage disequilibrium coefficient, EGF, epidermal growth, ENG, endoglin, ESR2, Estrogen receptor 2, HIF1A, Hypoxia-inducible factor 1, α subunit, HPS, hepatopulmonary syndrome, HWE, Hardy–Weinberg equilibrium, MELD, Model for End-stage Liver Disease, NOX4, NADPH Oxidase 4, OR, odds ratio, PaO2, partial pressure of oxygen in arterial blood, PC, principal component regression analysis, RUNX1, Runt-related transcription factor 1, SAT2, Spermidine/spermine N1-acetyltransferase family member, SHBG, Steroid hormone binding globulin, SNP, single nucleotide polymorphism, TIE1, Tyrosine kinase with Ig and EGF factor homology domains 1, VWF, von Willebrand factor

A listing of additional members of the Pulmonary Vascular Complications of Liver Disease Study Group can be found in Appendix 1.

Conflicts of interest The authors disclose no conflicts.

Funding Supported by NIH grants DK064103, DK065958, RR00645, RR00585, RR00046, RR00032, HL67771, HL089812 and, in part, under a grant with the Pennsylvania Department of Health, which specifically disclaims responsibility for any analysis, interpretations, or conclusions.

PII: S0016-5085(10)00463-4
doi:10.1053/j.gastro.2010.03.044
© 2010 AGA Institute. Published by Elsevier Inc. All rights reserved.

Source

Vertex Pharmaceuticals Reports Second Quarter 2010 Financial Results and Highlights Recent Business and Clinical Progress

-Hepatitis C: Rolling submission of New Drug Application underway; data from second pivotal Phase 3 trial - REALIZE - expected in the third quarter of 2010-

CAMBRIDGE, Mass., Jul 28, 2010 (BUSINESS WIRE) -- ---Cystic Fibrosis: Phase 3 registration program for VX-770 fully enrolled; data expected in first half of 2011-

---Financial: Vertex ends second quarter with cash position of approximately $980 million-

Vertex Pharmaceuticals Incorporated /quotes/comstock/15*!vrtx/quotes/nls/vrtx (VRTX 33.18, -0.01, -0.02%) today provided an update on recent progress in its development programs in hepatitis C virus (HCV) infection, cystic fibrosis (CF) and other diseases and reported consolidated financial results for the quarter ended June 30, 2010.

"We remain on track to complete the New Drug Application submission process for telaprevir later this year and to complete the buildout of our commercial function in advance of the potential launch of telaprevir," said Matthew Emmens, Chairman, President and Chief Executive Officer of Vertex Pharmaceuticals. "In the first half of this year, we established a seasoned commercial leadership team with broad experience in the area of infectious diseases, and we continue to bolster the internal infrastructure needed to support a field-based sales force for telaprevir and other potential future medicines.

"Our Phase 3 registration program in cystic fibrosis is now fully enrolled, positioning us for the planned submission of a New Drug Application for VX-770 in the second half of 2011. There is an urgent need for new and more effective therapies in cystic fibrosis, and we are committed to working toward improving the lives of people affected by this disease.

"As our development programs and commercial efforts advance, we remain focused on the management of our capital structure. We ended the second quarter of 2010 with a cash position of approximately $980 million, positioning us for continued investment in key activities to help support a successful potential launch for telaprevir," concluded Mr. Emmens.

Recent Clinical Development Progress

Telaprevir Phase 3 Program in Hepatitis C

-- Vertex expects efficacy and safety data from the supplemental Phase 3 ILLUMINATE trial to become available in August 2010, followed by efficacy and safety data from the pivotal Phase 3 REALIZE trial in September 2010.

-- The ILLUMINATE trial of telaprevir-based regimens is designed to evaluate the comparability of the sustained viral response (SVR), or viral cure, rates between the 24-week and 48-week treatment arms in treatment-naive people with genotype 1 hepatitis C who achieved undetectable virus levels at weeks 4 and 12 of treatment (eRVR) and who remained in the trial through week 20. Patients who met these criteria were randomized at week 20 to receive either 24 or 48 total weeks of therapy.

-- The REALIZE trial is being conducted by Vertex's collaborator Tibotec and is evaluating telaprevir in people with genotype 1 hepatitis C who did not achieve SVR, or a viral cure, with a prior pegylated interferon-based treatment, including difficult-to-treat null responder patients and patients who had a partial response or relapse in prior therapy. This is the only current Phase 3 trial of an investigational therapy for hepatitis C to enroll a difficult-to-treat patient population that includes patients who had a null response to a prior course of pegylated-interferon and ribavirin therapy.

-- Data from ILLUMINATE are expected to supplement the data obtained in the two pivotal Phase 3 trials of telaprevir - ADVANCE and REALIZE -- as part of the planned New Drug Application (NDA) submission for telaprevir.

Rolling NDA Submission Underway

-- Vertex recently submitted the Non-clinical and the Chemistry, Manufacturing and Controls (CM) sections of its NDA to the U.S. FDA as part of a rolling NDA submission for telaprevir. The company remains on track to complete the NDA submission for telaprevir in the second half of 2010.

Phase 3b Trial to Evaluate Twice-Daily (BID) Compared to Three-Times-Daily Dosing of Telaprevir

-- In the fourth quarter of 2010, Vertex and its collaborator Tibotec expect to initiate a Phase 3b clinical trial to evaluate twice-daily dosing of telaprevir (1,125 mg BID) compared to three-times-daily dosing of telaprevir (750 mg; q8h). This trial is expected to enroll approximately 700 treatment-naive people with genotype 1 hepatitis C in two telaprevir-based treatment arms and will be conducted in the U.S., E.U. and certain other countries. Based on advice from regulatory authorities in the U.S. and E.U., the trial will not include a control arm of pegylated-interferon and ribavirin.

Telaprevir/VX-222 Combination Trial

-- Vertex is currently conducting the first clinical trial to evaluate telaprevir dosed in combination with Vertex's lead HCV polymerase inhibitor, VX-222. This Phase 2 proof-of-concept trial is designed to evaluate SVR, or viral cure, rates using multiple 12-week response-guided regimens of telaprevir/VX-222-based combination therapy, including two-drug regimens that contain only telaprevir and VX-222. Vertex expects to obtain on-treatment clinical data from this trial in the second half of 2010.

Phase 3 Registration Program for VX-770

-- Three trials of the novel Cystic Fibrosis Transmembrane Conductance Regulator protein (CFTR) potentiator VX-770 are now fully enrolled as part of a global Phase 3 registration program focused on patients with the G551D mutation. These trials include the Phase 3 STRIVE trial in patients aged 12 years and older with the G551D mutation, the Phase 3 ENVISION trial in patients aged six to 11 years with the G551D mutation, and the Phase 2 DISCOVER trial in patients aged 12 and older homozygous for the F508del mutation.

-- Data from the Phase 3 registration program of VX-770 are expected in the first half of 2011.

Planned Combination Trial of VX-770 and VX-809

-- Vertex expects to initiate a Phase 2a clinical trial that will evaluate combination regimens of VX-809 and VX-770 later this year. The trial will enroll patients who are homozygous for the F508del mutation.

Proof of Concept Trials of VX-509 in Rheumatoid Arthritis and VX-765 in Epilepsy

-- Vertex is currently conducting Phase 2 proof-of-concept clinical trials of the novel caspase-1 inhibitor VX-765 in epilepsy and of the novel Janus kinase 3 (JAK3) inhibitor VX-509 in rheumatoid arthritis (RA).

-- Enrollment is complete in the trial of VX-765, and interim data are expected in the second half of 2010. Interim data from the trial of VX-509 are expected in 2011.

Second Quarter Results

For the quarter ended June 30, 2010, the Company's GAAP net loss was $200.0 million, or $1.00 per share, including certain charges totaling $57.5 million, compared to a GAAP net loss for the quarter ended June 30, 2009 of $171.3 million, or $0.99 per share, including certain charges totaling $42.0 million.

The non-GAAP loss, before certain charges, for the quarter ended June 30, 2010 was $142.5 million, or $0.71 per share, compared to $129.3 million, or $0.75 per share, for the quarter ended June 30, 2009. The increase in the Company's 2010 non-GAAP loss was principally attributable to an increase in total operating expenses as the company prepares for the potential launch of telaprevir and conducts a Phase 3 registration program for VX-770.

Total revenues for the quarter ended June 30, 2010 were $31.6 million, compared to $19.1 million for the second quarter of 2009. The increase is primarily attributable to higher collaborative revenues.

Research and development (R&D) expenses for the quarter ended June 30, 2010 were $155.1 million, compared to $139.3 million for the second quarter of 2009. The increase primarily reflects greater commercial supply investment for telaprevir.

Sales, general and administrative (SG&A) expenses for the quarter ended June 30, 2010 were $40.9 million, compared to $32.5 million for the second quarter of 2009. This increase reflects building of capabilities, including an increase in the number of employees and our commercial investments, to support advancement of telaprevir toward potential launch.

At June 30, 2010, Vertex had $979.1 million in cash, cash equivalents and marketable securities.

Full Year 2010 Financial Guidance

This section contains forward-looking guidance about the financial outlook for Vertex Pharmaceuticals.

The company is today reiterating its guidance for 2010 non-GAAP loss of approximately $600 million, as provided on February 4, 2010. The company is revising its guidance for 2010 GAAP net loss from approximately $700 million to approximately $750 million to reflect the timing of certain non-cash expenses and revenues related to the company's 2009 financial transactions and the milestone payments the company is eligible to earn from Janssen.

Non-GAAP Financial Measures

In this press release, Vertex's financial results are provided both in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, Vertex provides its second quarter 2010 and 2009 loss, and guidance for its projected 2010 loss, excluding stock-based compensation and executive transition expenses, restructuring expense, acquisition-related expenses, loss on exchange of convertible subordinated notes, and revenue and expenses related to certain September 2009 financial transactions. These results are provided as a complement to results provided in accordance with GAAP because management believes these non-GAAP financial measures help indicate underlying trends in the Company's business, are important in comparing current results with prior period results and provide additional information regarding its financial position. Management also uses these non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally, and to manage the Company's business and to evaluate its performance. A reconciliation of the other non-GAAP financial results to GAAP financial results is included in the attached financial statements.

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 companies. Vertex's product pipeline is focused on viral diseases, cystic fibrosis, inflammation, autoimmune diseases, epilepsy, cancer, and pain.

Special Note Regarding Forward-looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including statements regarding (i) the expectation that the company will complete the NDA submission for telaprevir later in the year, (ii) the expectation that safety and efficacy data will be available from REALIZE in September 2010 and from ILLUMINATE in August 2010, (iii) the company remaining on track to complete the buildout of its commercial function in advance of the potential launch of telaprevir, (iv) the company being positioned to submit a New Drug Application for VX-770 in the second half of 2011, (v) the company's cash position positioning it for continued investment in key activities to help support a successful potential launch for telaprevir, (vi) the expectation that the ILLUMINATE data will supplement the data obtained from ADVANCE and REALIZE, (vii) the expectation that a Phase 3b clinical trial to evaluate twice-daily dosing of telaprevir will be initiated in the fourth quarter of 2010 and a Phase 2a clinical trial to evaluate combination regimens of VX-809 and VX-770 will be initiated later this year, (viii) the expected clinical trial designs for the Phase 3b clinical trial of telaprevir and the VX-809/VX-770 clinical trial, (ix) the anticipated timing of receiving clinical data from the Phase 3 registration program of VX-770 and from clinical trials of telaprevir/VX-222, VX-509 and VX-765 and (x) the company's guidance regarding 2010 GAAP and non-GAAP net loss. 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 the outcomes for each of its planned clinical trials and studies, and in particular its planned clinical trials of telaprevir, may not be favorable, that regulatory authorities may require supplemental clinical trials in order to support registration of telaprevir and/or VX-770, that planned or potential clinical trials may be delayed or may not be conducted, that the company may not be able to successfully develop telaprevir, VX-770, VX-509, VX-765 or combination therapies involving telaprevir and VX-222 or VX-770 and VX-809, that the company's expectations regarding its 2010 GAAP and non-GAAP net loss may be incorrect, 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 www.vrtx.com. The company disclaims any obligation to update the information contained in this press release as new information becomes available.

Source

Intercept signs CRADA with NIDDK for study of obeticholic acid in nonalcoholic steatohepatitis

28. July 2010 09:35

Intercept Pharmaceuticals, Inc., a clinical stage biopharmaceutical company developing novel therapeutics for chronic fibrotic and metabolic diseases, today announced the signing of a cooperative research and development agreement (CRADA) with the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) of the National Institutes of Health to conduct a double blind, multi-center, study to evaluate the effects of obeticholic acid in patients with nonalcoholic steatohepatitis (NASH). Obeticholic acid is the generic name for INT-747, Intercept's first-in-class FXR agonist.

The planned study will enroll 280 patients at the eight U.S. centers constituting the NIDDK-sponsored NASH clinical research network (CRN), which will make it the largest study conducted in this disease. The objectives of the 72 week study will be to assess whether obeticholic acid improves histological disease activity and other disease markers, along with the safety of the drug in this patient population. The study is expected to begin in the fourth quarter of 2010 and the NIDDK will provide a majority of the funding needed under the CRADA.

NASH is a more serious form of nonalcoholic fatty liver disease (NAFLD) and occurs in patients who drink little or no alcohol. The disease is believed to be caused by abnormal metabolism of fats and, although it is often associated with obesity and insulin resistance, it also occurs in lean individuals. NASH is associated with fibrosis (scarring) in the liver that may lead to cirrhosis, liver cancer and death, and the disease also carries an additional risk of death due to heart disease. NASH is now the most common liver disease in the developed world, affecting at least 3 percent of the U.S. population, and there is no approved treatment for the disease.

Mark Pruzanski, MD, founder, President and CEO of Intercept, commented, "We are excited to collaborate with the NIDDK and CRN to test our drug in such a robust NASH study. Last year we presented data showing that obeticholic acid improved insulin sensitivity, lowered liver enzymes and induced weight loss in type 2 diabetic patients with NAFLD. These results and the novel mechanism of action of our drug are a promising basis for pursuing NASH."

Pat Robuck, PhD, MPH, the senior advisor for clinical trials in digestive and liver diseases, in the NIDDK's Division of Digestive Diseases and Nutrition, stated, "There is a huge unmet medical need in this patient population. The NIDDK, working together with our NASH CRN investigators, is committed to discovering effective and safe treatments for this serious disease. The preclinical and clinical data obtained so far with obeticholic acid suggest that it has beneficial effects on glucose metabolism and the liver, and the NASH CRN steering committee thinks it warrants rigorous clinical evaluation in NASH."

SOURCE Intercept Pharmaceuticals, Inc.

Source

Peginterferon α-2a and ribavirin treatment of patients with haemophilia and hepatitis C virus infection: a single-centre study of 367 cases

Liver International
Early View (Articles online in advance of print)
Published Online: 8 Jul 2010
© 2010 John Wiley & Sons A/S

Seyed-Moayed Alavian 1 , Seyed Vahid Tabatabaei 1 , Maryam Keshvari 2 , Bita Behnava 1 , Seyyed Mohammad Miri 1 , Pegah Karimi Elizee 2 and Kamran Bagheri Lankarani 3

1 Research Center for Gastroenterology and Liver Disease, Baqiyatallah University of Medical Sciences, Tehran, Iran
2 Iranian Blood Transfusion Organization Research Center (IBTO), Tehran, Iran
3 Shiraz University of Medical Sciences, Shiraz, Iran

Correspondence

Seyed-Moayed Alavian, Baqiyatallah Research Center for Gastroenterology and Liver Diseases, Ground floor of Baqiyatallah Hospital, Mollasadra Ave., Vanak Sq. P. O. Box 14155-3651, Tehran, Iran
Tel/Fax: +98 21 88 067 114
e-mail: editor@hepmon.com

KEYWORDS
congenital bleeding disorder • HCV • haemophilia • peginterferon α-2a • ribavirin

ABSTRACT

Background/aims: Chronic hepatitis C virus infection (HCV) is a major comorbidity in patients with haemophilia. Peginterferon alpha and ribavirin is current standard anti-HCV thrapy but there is little information about safety and efficacy of peginterferon α-2a and ribavirin combination therapy in these patients.

Material and methods: In an open-label single-treatment arm cohort study, 367 haemophilia patients seronegative for hepatitis B and human immunodeficiency virus markers and chronically infected with HCV (HCV RNA>50 IU/ml for at least 6 months) received 180 μg of Pegasys® and 800–1200 mg of ribavirin according to body weight. Genotypes 1 and 4, mixed and untypable infections were treated for 48 weeks, while genotypes 2 and 3 were treated for 24 weeks. The efficacy of therapy was expressed as sustained virological response (SVR).

Results: Two hundred and twenty-five subjects [61%, 95% confidence interval (CI) 56–66] achieved SVR, 66 patients relapsed and 30 subjects did not respond and nine patients developed breakthrough during treatment. In a multivariate logistic regression model, age<24 odds ratio (OR)=1.8 (95% CI 1.1–3.1), genotype non-1 OR=1.8 (95% CI 1.1–3.2), BMI<25 OR=2.1 (95% CI 1.3–3.3) and HCV RNA<600 000 IU/ml OR=1.7 (95% CI 1.1–3.2) were independent predictors of SVR. Eight patients discontinued the treatment because of persistent neutropaenia and 22 subjects were dropped out because of intractable side effects. Furthermore, two patients died during treatment and five were lost to follow-up after treatment cessation.

Conclusions: Peginterferon alpha-2a in combination with weight-based ribavirin has SVR rate of 51% for genotype 1 and 71% for genotype non-1 infections in haemophilia patients. Age<24, BMI<25, viral load<600 000 IU/ml and genotype non-1 are the major determinants of SVR achievement in these patients.

Received 9 February 2010
Accepted 24 May 2010

DIGITAL OBJECT IDENTIFIER (DOI)
10.1111/j.1478-3231.2010.02296.x About DOI
 
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NY Times HCV Article Misleads Public About Treatment & Need for Screening

from Jules: In an attempt to create controversy the NY Times writer Andrew Pollack in an article published in the NY Times on July 21 where he talks about the new HCV drugs in development prints some very inflammatory & incorrect information. While at the same time appearing to say it is good that new more effective HCV drugs are in development that will save people's lives, he quotes a Dr Koretz who is based in LA and apparently not a hepatitis doctor as saying 'there is no good evidence that HCV treatment makes a difference since many patients cured might never develop serious liver disease'. This expresses an incorrect & irresponsible misunderstanding of hepatitis C care & treatment, it ignores much of the published and well known facts and does a disservice to patients & the entire health system. Over 4 million people in the USA are estimated to have hepatitis C virus (HCV). Many have been infected for 30 years or longer. A recently published paper by a well respected hepatitis researcher and doctor Gary Davis based at Baylor University in Texas reported in a model how the many people infected with HCV over 20 years ago are now aging and in these people the disease has progressed to serious disease where there is much greater increased risk for liver cancer and dying from the disease. This is true. The disservice by the Times article is that it discourages individuals from getting tested to see if they have HCV, because if you don't know you have HCV you certainly will not see a clinician to receive further care. It is known that 75% of HCV-infected individuals in the USA do not know they have HCV. Many individuals have died of liver disease and liver cancer because they were not tested. Once diagnosed with HCV an individual can then receive advise from a good doctor regarding whether they should be treated or if they can defer treatment, they can be monitored over years to see if the disease is progressing or not, they can improve their lifestyle to take better care of their health & liver, and they can make informed decisions about treatment. We need a large national HCV screening/testing program throughout the USA, particularly in major urban cities, as HCV very disproportionately affects African-Americans & Latinos, the health disparities are enormous. At the cost of The abandoning ethical reporting the Times reporter chose to create harmful controversy, as reporters sometimes do. The costs of many people getting sick from HCV to the governments in the USA will be enormous as large numbers of patients age with HCV and develop serious liver disease, costs for hospitalizations will zoom. If tested and in care not all patients require treatment but they can at least make informed decisions. Jules Levin, NATAP

Aging of Hepatitis C Virus (HCV)-Infected Persons in the United ...
Stacked prevalence curves showing number of cases by year with cirrhosis according to gender and age at time of initial hepatitis C virus infection. ...
www.natap.org/2010/HCV/031110_02.htm

Hope Against Hepatitis C, New Drugs - 'this will be revolutionary' - (07/19/10)
New cases of liver cancer are already rising year by year. And hepatitis C is the leading cause of liver transplants, like the one recently received by the rock musician Gregg Allman.

Hopes for new treatments were buoyed in May by the first results from a late-stage clinical trial of one of the new drugs, telaprevir from Vertex. When added to the existing treatment - a combination of alpha interferon and ribavirin - telaprevir effectively cured 75 percent of patients, compared with 44 percent of those treated with the existing drugs alone. And for many patients, the course of treatment could be halved to 24 weeks.

Dr. Poordad, who is a consultant to some of the pharmaceutical companies, said that one-fifth of his patients were being "warehoused," meaning they were forgoing treatment now to wait for the new drugs.

"I think the companies have done a superb job of marketing this disease," said Dr. Ronald L. Koretz, emeritus professor of clinical medicine at the University of California, Los Angeles. Dr. Koretz said there was no good evidence that treatment made a difference since many patients cured by the drugs might never have developed serious problems anyway.

NVHR Responds to New York Times Article on Hepatitis C Testing

WASHINGTON, July 22 /PRNewswire-USNewswire/ -- In response to today's New York Times article, "Hope against Hepatitis C," Andrew Muir, M.D., M.H.S., Director, Gastroenterology/Hepatology Research, Duke Clinical Research Institute and Steering Committee Member of the National Viral Hepatitis Roundtable (NVHR) released the following statement:

"Today's New York Times article details potential promising new drug therapies that could significantly improve the way we treat individuals infected with hepatitis C. Regrettably, the article suggests that expanded screening for hepatitis C may not be warranted. This approach is wrong and contrary to the direction in which we should and must move our health care system, particularly through improved access to care under health care reform. More than 5 million Americans are estimated to be infected with viral hepatitis B or C - and most are unaware they are infected as there are often few symptoms. Our health care system misses most infected individuals, who only learn that they have hepatitis C once they have progressed to liver cancer, cirrhosis, or liver failure. At that juncture, treatment options are limited and success rates are lower.

"Precisely because we do not know which individuals with hepatitis C will advance to these terrible diseases, it is critical that our public health infrastructure be modernized to achieve early detection of new infections and also to screen for individuals within specific risk groups, such as baby boomers and disproportionately affected populations. Once individuals are aware of their status, they will be empowered with this information, not only to make treatment choices, but also lifestyle choices to decrease their likelihood of disease progression and not to spread this infectious disease to others. In our current health care system, there are far too few options for diagnosis, care, and treatment. Unless or until the health care system provides access to all persons in need of hepatitis C treatment, it is important for the pharmaceutical industry to provide comprehensive compassionate care programs for those who are un/under insured.

"We can't prevent or treat what we don't know, which is why screening is critical. Access to screening would capture more infected individuals who can respond favorably to early intervention, reduce transmission, avoid needless medical expenses, and ultimately save thousands of lives annually."

NVHR is a coalition of more than 150 public, private, and voluntary organizations dedicated to reducing the incidence of infection, morbidity, and mortality from chronic viral hepatitis that afflicts more than 5 million Americans. http://www.nvhr.org/

SOURCE National Viral Hepatitis Roundtable

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Opportunities for the hepatitis C therapeutics market in Australia

28 July 2010

Spirited efforts in Australia to raise awareness of hepatitis C have increased the number of diagnosed cases and expanded the base of patients available for treatment, thus enabling the introduction of novel treatments in this market sector, says a new multi-client report from Frost & Sullivan.

Physicians expect the rate of diagnosis to continue rising in Australia due to heightening of awareness among general practitioners regarding screening and treatment for hepatitis C. Screening of patients in Australia, especially among illicit drug users, has resulted in a spike in the number of diagnosed cases of hepatitis C virus (HCV). In 2009, an estimated 77% of patients-at-risk with hepatitis C were diagnosed, notes F&S, and this figure is expected to reach up to 87% by 2016.

New anti-virals set to come to market

Higher efficacy and safety are the most preferred drug attributes. Physicians expect new oral anti-virals (boceprevir and telaprivir) and interferon with improved safety (albuferon) to be launched by 2013 in Australia. According to physicians, prevalence had increased rapidly in the early 2000s, but the rate had declined over the years and the pace is likely to slacken in the future owing to a major disruption to the supply of heroin in Australia in 2001.

"The Australian government has unleashed Initiatives to address the Hepatitis C virus in the country, allocating $14.3 million (A$17 million) for hepatitis C education and prevention over the 2007-2011 period," notes an F&S analyst, adding: "State and territory governments also have substantial budgets for hepatitis C prevention and education."

The priority action areas identified as part of the National Hepatitis C Strategy are prevention, education; diagnosis, treatment, surveillance; and research. The government is avidly addressing health care workforce development as well as issues surrounding discrimination and stigma.

Only 3% of diagnosed patients pursuing treatment

Although there is a high diagnosis rate in Australia, only a paltry 3% of diagnosed patients pursue treatment owing to a lack of patient understanding about treatment options. According to physicians, one of the main reasons for such a large proportion of diagnosed patients left untreated is that they are predominantly from disadvantaged groups such as illicit drug users, aboriginal people, those serving prison sentences, and some migrant groups, which do not have access to conventional healthcare.

Often the reason is real or perceived stigma and discrimination. Aside from this, there is a great deal of angst about debilitating side effects, frequent dosing, and lackluster efficacy of the existing HCV treatments, especially for those afflicted with genotype 1. Moreover, there is a belief that a liver biopsy is required to receive treatment, which deters patients from undergoing treatment.

To circumvent the challenges clouding the market landscape, companies must offer treatment options that expedite efficiency, have fewer side effects and are cost efficient, states F&S. Patients should be made aware of their disease status and encouraged to remain compliant. "As more improvements are required to educate at-risk patients, public and private organizations - including pharmaceutical companies - must up the ante to spread awareness," notes the analyst, stressing that "greater information outreach will help patients obtain the right mode of treatment and, ultimately, stem the onslaught of the hepatitis C virus in Australia."

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Pharmasset Initiates Phase 1b Multiple Ascending Dose Clinical Trial of PSI-938 in Patients with Chronic Hepatitis C

-- PSI-938 single ascending dose study in healthy volunteers supports progression to 7 day monotherapy study in HCV infected patients
-- PSI-938 was generally safe and well tolerated with no dose-limiting toxicity

-- Further results from single ascending and multiple ascending dose trials are expected in the third quarter 2010

PRINCETON, N.J., July 28 /PRNewswire-FirstCall/ -- Pharmasset, Inc. (Nasdaq: VRUS) announced today that safety and pharmacokinetic data from the PSI-352938 ("PSI-938") single ascending dose study support progression to a multiple ascending dose trial with PSI-938, which has initiated dosing. PSI-938 is a guanine nucleotide analog polymerase inhibitor for the treatment of chronic hepatitis C virus (HCV) infection. This study is designed to assess the safety, tolerability and antiviral activity of PSI-938 monotherapy administered over 7 days in HCV-infected individuals.

"PSI-938 is the third differentiated nucleoside analog that Pharmasset has advanced into clinical development for HCV," stated Michelle Berrey, MD, MPH, Pharmasset's Chief Medical Officer. "As with RG7128 and PSI-7977, PSI-938 demonstrates a high barrier to resistance in vitro; but unlike the cytidine and uridine analogs, PSI-938 retains equivalent potency against the S282T mutant. As the HCV field moves toward IFN-sparing, all-oral DAA combinations, we continue to believe nucleosides could become the backbone of care given these differentiating attributes and have the potential to be combined with all DAA classes."

PSI-938 Phase 1 Program Overview

The Phase 1 program is investigating the safety, tolerability and pharmacokinetics of PSI-938 in healthy subjects following escalating single doses (Phase 1a), and in patients chronically infected with HCV genotype 1 following repeat dosing for 7 days (Phase 1b). The Phase 1b study will additionally investigate hepatitis C viral dynamics and monitor for the development of drug resistance.

Subjects in the Phase 1a single ascending dose study received single doses of PSI-938 ranging from 100 mg to 800 mg or a matching placebo. Preliminary data from the phase 1a single ascending dose study includes:

•No serious adverse events or discontinuations;
•No dose-related adverse events or dose-limiting toxicity;
•No grade III / IV lab abnormalities;
•No clinically significant changes in vital signs or ECGs; and
•PK which supports QD dosing.

A Phase 1b multiple ascending dose trial has now been initiated in treatment-naïve patients with chronic HCV genotype 1 infection. Subjects will be enrolled at multiple centers in the US and randomized to PSI-938 or placebo. Based upon the results from the first time in human study, the first dose of PSI-938 to be tested will be 100 mg administered once daily. The primary objectives of this study are to assess the safety, tolerability, pharmacokinetics and viral dynamics of PSI-938 after repeat dosing over 7 days.

Results from both studies are expected in the third quarter of 2010.
Purine and Pyrimidine Analogs

Pharmasset's purine nucleoside/tide analogs share many of the benefits of pyrimidine nucleoside/tide analogs, such as RG7128 and PSI-7977, in that they have demonstrated in vitro activity across multiple HCV genotypes, have a higher barrier to resistance than other classes of HCV small molecules in development, and, in spite of the prodrug technology, have no CYP 3A4 liability and thus a lower risk of drug interactions when combined with other direct acting antivirals targeting HCV. In addition, Pharmasset's purine analogs retain equivalent potency against wild type HCV and virus with the S282T mutation associated with in vitro resistance in other nucleoside/tide analogs in development. Furthermore, the purines are metabolized to the active triphosphate form through a different phosphorylation pathway than the pyrimidine analogs, thus decreasing the risk of competition between the two analogs for conversion to the active triphosphate. Given these characteristics, Pharmasset's purine and pyrimidine analogs have the potential to be combined as part of a future treatment regimen, which will be the focus of upcoming trials.

About Pharmasset

Pharmasset is a clinical-stage pharmaceutical company committed to discovering, developing, and commercializing novel drugs to treat viral infections. Pharmasset's primary focus is on the development of oral therapeutics for the treatment of hepatitis C virus (HCV) and, secondarily, on the development of Racivir(TM) for the treatment of human immunodeficiency virus (HIV). Our research and development efforts focus on nucleoside/tide analogs, a class of compounds which act as alternative substrates for the viral polymerase, thus inhibiting viral replication. We currently have four clinical-stage product candidates. RG7128, a cytosine analog for chronic HCV infection, is in two Phase 2b clinical studies in combination with Pegasys(R) plus Copegus(R) and is also in the INFORM studies, the first series of studies designed to assess the potential of combinations of small molecules without Pegasys(R) and Copegus(R) to treat chronic HCV. These clinical studies are being conducted through a strategic collaboration with Roche. Our other clinical stage HCV candidates include PSI-7977, an unpartnered uracil nucleotide analog that has recently completed a Phase 2a study, and PSI-938, an unpartnered guanine nucleotide analog in Phase 1. We also have in our pipeline an additional purine nucleotide analog, PSI-661, in advanced preclinical development. Racivir, for the treatment of HIV, has completed a Phase 2 clinical study.

Pegasys® and Copegus® are registered trademarks of Roche.

Contact

Richard E. T. Smith, Ph.D.
VP, Investor Relations and Corporate Communications
richard.smith@pharmasset.com
Office: +1 (609) 613-4181

Forward-Looking Statements


Pharmasset "Safe Harbor" Statement under the Private Securities Litigation Reform Act of 1995: Statements in this press release that are not historical facts are "forward-looking statements," including, without limitation, statements that involve risks, uncertainties, and other important factors, including, without limitation, the risk of cessation or delay of any of the ongoing or planned clinical trials and/or our development of our product candidates, the risk that the results of previously conducted studies involving our product candidates will not be repeated or observed in ongoing or future studies involving our product candidates, the risk that our collaboration with Roche will not continue or will not be successful, and the risk that any one or more of our product candidates will not be successfully developed and commercialized. For a discussion of risks, uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in our Annual Report on Form 10-K for the fiscal year ended September 30, 2009 and our Quarterly Reports on Form 10-Q for the periods ended December 31, 2009 and March 31, 2010 filed with the Securities and Exchange Commission and discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission.

SOURCE Pharmasset

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