(Interim analysis of this 30 patient Phase II trial today designed to assess the safety, tolerability and efficacy of multiple 12- and 24-week response-guided treatment regimens with Vertex's polymerase inhibitor VX-222 (400 mg or 100 mg), and Telaprevir plus Peg/ Riba in genotype1 treatment naive subjects. Results showed that 50 percent of people (15/30) in the study who received VX-222 (400 mg) in combination with INCIVEK, pegylated-interferon and ribavirin were eligible to stop all treatment at week 12, and 93 percent (14/15) had SVR12. Patients from the VX-222 (400 mg) treatment arm who were not eligible to stop all treatment at week 12 received an additional 12 weeks of pegylated-interferon and ribavirin alone for 24 total weeks of treatment. The hepatitis C virus was undetectable in 100 percent (13/13) of these patients at the end of 24 weeks. In this study, VX-222, Telaprevir and ribavirin were given twice daily (BID). Interim safety results from the four-drug treatment arms showed that mild gastrointestinal symptoms and mild fatigue were the most frequently reported adverse events. Side effects consistent with the known safety profile of INCIVEK combination treatment also were observed. Leaves one to wonder what sort of results we'd see with Telaprevir in combination with the recently purchased nucleoside analogs from Aliso Pharmaceuticals)
BusinessWire · Jul. 26, 2011 | Last Updated: Jul. 26, 2011 7:30 AM ET
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced interim results from ZENITH, an ongoing Phase 2 study designed to assess the safety, tolerability and efficacy of multiple 12- and 24-week response-guided treatment regimens with VX-222 (400 mg or 100 mg), its lead polymerase inhibitor in development, in combination with INCIVEK™ (telaprevir) tablets, pegylated-interferon and ribavirin in people with genotype 1 chronic hepatitis C who were new to treatment. This is an interim analysis from patients in the four-drug treatment arms and was conducted after these patients completed their assigned treatment. Results showed that 50 percent of people (15/30) in the study who received VX-222 (400 mg) in combination with INCIVEK, pegylated-interferon and ribavirin were eligible to stop all treatment at week 12, and 93 percent (14/15) of these patients had undetectable hepatitis C virus 12 weeks after treatment ended (sustained viral response 12, or SVR12). Patients from the VX-222 (400 mg) treatment arm who were not eligible to stop all treatment at week 12 received an additional 12 weeks of pegylated-interferon and ribavirin alone for 24 total weeks of treatment. The hepatitis C virus was undetectable in 100 percent (13/13) of these patients at the end of 24 weeks. In this study, VX-222, INCIVEK and ribavirin were given twice daily (BID). Interim safety results from the four-drug treatment arms showed that mild gastrointestinal symptoms and mild fatigue were the most frequently reported adverse events. Side effects consistent with the known safety profile of INCIVEK combination treatment also were observed.
“The results from this study are the first to show the potential for a combination of multiple direct-acting antiviral medicines to help people with hepatitis C clear the virus with as few as 12 and no more than 24 weeks of treatment,” said Robert Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical Officer for Vertex. “We look forward to additional data from this study, including data from the ongoing all-oral treatment arms, which will guide our future development plans with the goal of further improving treatment.”
ZENITH is an ongoing Phase 2 study that initially enrolled 106 people with genotype 1 chronic hepatitis C and began with four treatment arms designed to evaluate multiple response-guided treatment regimens with VX-222, Vertex’s lead polymerase inhibitor in development, in combination with INCIVEK, Pegasys® (pegylated-interferon alfa-2a) and Copegus® (ribavirin), three medicines approved to treat hepatitis C. In this study, VX-222, INCIVEK and ribavirin were given twice daily. The primary endpoint of the study is safety and tolerability. The secondary endpoint is on-treatment antiviral activity and the proportion of people in each treatment arm who achieve a sustained viral response. Additional results from this study will be submitted for presentation at an upcoming medical meeting.
Arms A (n=18) and B (n=29) were designed to evaluate all-oral, two-drug combination regimens of VX-222 (400 mg or 100 mg) and INCIVEK (1,125 mg). Data presented in March at The International Liver Congress™ 2011, the 46th annual meeting of the European Association for the Study of the Liver (EASL), in Berlin, Germany, showed significant initial antiviral activity in people who were treated with VX-222 (400 mg) and INCIVEK. However, these treatment arms were discontinued due to a pre-defined stopping rule related to viral breakthrough. Arms C (n=29) and D (n=30) are ongoing and are designed to evaluate four-drug combination regimens of VX-222 (400 mg or 100 mg), INCIVEK (1,125 mg), pegylated-interferon and ribavirin.
Data announced today are from the four-drug treatment arms (Arms C and D). In these two arms, patients were assigned to take all four medicines for the first 12 weeks of treatment. People who had undetectable hepatitis C virus levels in the blood (HCV RNA) at weeks two and eight of treatment were eligible to stop all treatment at week 12. In this study, the amount of hepatitis C virus in the blood was measured by the Roche COBAS® Taqman HCV test (<10 IU/mL undetectable). People who did not meet these criteria were assigned to receive 24 total weeks of treatment: 12 weeks of the four-drug combination regimen followed by 12 weeks of pegylated-interferon and ribavirin alone. This interim analysis includes SVR12 results for the people who were eligible for and completed 12 total weeks of treatment and end-of-treatment results for the people who were assigned to and completed 24 total weeks of treatment.
ZENITH: Interim Analysis
SVR12 in people who
were eligible for and
completed 12 total
weeks of treatment
Undetectable hepatitis C
virus in people who were
assigned to and
completed 24 weeks of
treatment
Undetectable
hepatitis C virus at
week 24 for all
patients (intent-to-
treat analysis)
VX-222 (400 mg), INCIVEK,
pegylated-interferon and ribavirin*
(n=30)
93%
(14/15)+
100%
(13/13)
90%
(27/30)
VX-222 (100 mg), INCIVEK,
pegylated-interferon and ribavirin**
(n=29)
82%
(9/11) ++
93%
(13/14)^
83%
(24/29)
SVR12: undetectable hepatitis C virus 12 weeks after treatment ended.
*50 percent (15/30) had undetectable hepatitis C virus at weeks 2 and 8 and were eligible to stop all treatment at week 12. Two people in the VX-222 (400 mg) treatment arm discontinued treatment before week 12 and did not achieve SVR12.
**38 percent (11/29) had undetectable hepatitis C virus at weeks 2 and 8 and were eligible to stop all treatment at week 12. Four people in the VX-222 (100 mg) treatment arm discontinued treatment before week 12 and two of them achieved SVR12.
+One person in the 12-week VX-222 (400 mg) treatment arm relapsed.
++Two people in the 12-week VX-222 (100 mg) treatment arm relapsed.
^24-week end-of-treatment data are not available for one patient.
Two additional treatment arms (E and F) were added to the study to evaluate a three-drug, all-oral, interferon-free regimen of VX-222 (400 mg), INCIVEK and ribavirin. Enrollment in these treatment arms is expected to be complete by the end of the third quarter of 2011. Arm E will evaluate people with genotype 1b chronic hepatitis C and Arm F will evaluate people with genotype 1a chronic hepatitis C. Vertex expects to report data from the all-oral treatment arms in the first half of 2012.
Interim Safety and Tolerability Results from the Four-drug Treatment Arms
The most frequent adverse events observed in the four-drug treatment arms were fatigue, nausea, diarrhea, anemia, pruritis (itchiness) and rash. The majority of events were mild or moderate. Mild diarrhea occurred more frequently in the VX-222 (400 mg) treatment arm. The majority of people in the study did not use medication to control diarrhea. There were no treatment discontinuations due to diarrhea. Six patients discontinued treatment due to adverse events; three each from the 400 mg and 100 mg treatment arms. Two people from each arm discontinued treatment before week 12 and one person in each arm discontinued treatment between weeks 12 and 24 while they were receiving pegylated-interferon and ribavirin alone.
About INCIVEK and VX-222
INCIVEK (in-SEE-veck) is an oral medicine that acts directly on the hepatitis C virus protease, an enzyme essential for viral replication. In May 2011, INCIVEK was approved by the U.S. Food and Drug Administration (FDA) for a broad group of people with genotype 1 chronic hepatitis C with compensated liver disease (some level of damage to the liver but the liver still functions), including cirrhosis (scarring of the liver). INCIVEK is approved for people who are new to treatment, and for people who were treated previously but who did not achieve a SVR, or viral cure (relapsers, partial responders and null responders).
VX-222 is an oral medicine in development that is a non-nucleoside inhibitor of the HCV NS5B polymerase. VX-222 is currently being evaluated in combination with INCIVEK, pegylated-interferon and ribavirin in a Phase 2 study. Vertex has worldwide commercial rights for VX-222.
Vertex developed telaprevir in collaboration with Tibotec Vicro-Virology BVBA, one of the Janssen Pharmaceutical companies of Johnson & Johnson, and Mitsubishi Tanabe Pharma. Vertex is commercializing telaprevir in North America, where it is known as INCIVEK. Through its affiliate, Janssen, Tibotec has rights to commercialize telaprevir in Europe, South America, Australia, the Middle East and certain other countries. In July 2011, Janssen announced that the Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion for INCIVO (telaprevir). Mitsubishi Tanabe Pharma has rights to commercialize telaprevir in Japan and certain Far East countries.
INCIVEK™ is a trademark of Vertex Pharmaceuticals Incorporated.
PEGASYS®, COPEGUS® and Roche COBAS® Taqman HCV test are registered trademarks of Hoffmann-La Roche.
Indication
INCIVEKTM (telaprevir) tablets is a prescription medicine used with the medicines peginterferon alfa and ribavirin to treat chronic (lasting a long time) hepatitis C genotype 1 infection in adults with stable liver problems, who have not been treated before or who have failed previous treatment.
It is not known if INCIVEK is safe and effective in children under 18 years of age.
IMPORTANT SAFETY INFORMATION
INCIVEK should always be taken in combination with peginterferon alfa and ribavirin. Ribavirin may cause birth defects or death of an unborn baby. Therefore, you should not take INCIVEK combination treatment if you are pregnant or may become pregnant, or if you are a man with a sexual partner who is pregnant.
INCIVEK and other medicines can affect each other and can also cause side effects that can be serious or life threatening. There are certain medicines you cannot take with INCIVEK combination treatment. Tell your healthcare provider about all the medicines you take, including prescription and non-prescription medicines, vitamins and herbal supplements.
INCIVEK can cause serious side effects including rash and anemia. The most common side effects of INCIVEK include itching, nausea, diarrhea, vomiting, anal or rectal problems, taste changes and tiredness. There are other possible side effects of INCIVEK, and side effects associated with peginterferon alfa and ribavirin also apply to INCIVEK combination treatment. Tell your healthcare provider about any side effect that bothers you or doesn’t go away.
Please see full Prescribing Information for INCIVEK, including the Medication Guide, available at www.INCIVEK.com.
About Hepatitis C
Hepatitis C is a serious liver disease caused by the hepatitis C virus, which is spread through direct contact with the blood of infected people and ultimately affects the liver.1 Chronic hepatitis C can lead to serious and life-threatening liver problems, including liver damage, cirrhosis, liver failure or liver cancer.1 Though many people with hepatitis C may not experience symptoms, others may have symptoms such as fatigue, fever, jaundice and abdominal pain.1
Unlike HIV and hepatitis B virus, chronic hepatitis C can be cured.2 However, approximately 60 percent of people do not achieve SVR,3,4,5 or viral cure,6 after treatment with 48 weeks of pegylated-interferon and ribavirin alone. If treatment is not successful and a person does not achieve a viral cure, they remain at an increased risk for progressive liver disease.7,8
More than 170 million people worldwide are chronically infected with hepatitis C.6 In the United States, nearly 4 million people have chronic hepatitis C and 75 percent of them are unaware of their infection.9 Hepatitis C is four times more prevalent in the United States compared to HIV.9 The majority of people with hepatitis C in the United States were born between 1946 and 1964, accounting for two of every three people with chronic hepatitis C.10 Chronic hepatitis C is the leading cause of liver cancer and liver transplantations in the United States and is reported to contribute to 4,600 to 12,000 deaths annually. 11,12 By 2029, total annual medical costs in the United States for people with hepatitis C are expected to more than double, from $30 billion in 2009 to approximately $85 billion.11
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 potential for a combination of multiple direct-acting antiviral medicines to treat genotype 1 hepatitis C in as few as 12 weeks and no more than 24 weeks; (ii) additional results from the study being submitted for presentation at an upcoming medical meeting; (iii) additional data from this study, including data from the ongoing all-oral treatment arms, guiding our future development plans with the goal of further improving treatment; (iv) the design of Arm E and Arm F of the study and the expectation that enrollment in these arms will be completed by the end of the third quarter of 2011; and (v) the expectation that Vertex will report data from the all-oral treatment arms in the first half of 2012. 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 interim data presented in this press release may not be predictive of the final outcomes from this clinical trial; the outcomes from additional arms in this clinical trial and/or from any future clinical trials of telaprevir/VX-222 may not be favorable; future scientific, clinical, competitive or other market factors may adversely affect the potential for telaprevir/VX-222-based therapy and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through Vertex's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
About Vertex
Vertex creates new possibilities in medicine. Our team discovers, develops and commercializes innovative therapies so people with serious diseases can lead better lives.
Vertex scientists and our collaborators are working on new medicines to cure or significantly advance the treatment of hepatitis C, cystic fibrosis, epilepsy and other life-threatening diseases.
Founded more than 20 years ago in Cambridge, MA, we now have ongoing worldwide research programs and sites in the U.S., U.K. and Canada.
(VRTX - GEN)
1 Centers for Disease Control and Prevention. Hepatitis C Fact Sheet: CDC Viral Hepatitis. Available at: http://www.cdc.gov/hepatitis/HCV/PDFs/HepCGeneralFactSheet.pdf. Accessed March 21, 2011.
2 Pearlman BL and Traub N. Sustained Virologic Response to Antiviral Therapy for Chronic Hepatitis C Virus Infection: A Cure and So Much More. Clin Infect Dis. 2011 Apr;52(7):889-900.
3 Manns MP, McHutchison JG, Gordon SC, et al. Peginterferon alfa-2b plus ribavirin compared with interferon alfa-2b plus ribavirin for initial treatment of chronic hepatitis C: a randomised trial. Lancet. 2001;358:958-965.
4 Fried MW, Shiffman ML, Reddy KR, et al. Peginterferon alfa-2a plus ribavirin for chronic hepatitis C virus infection. N Engl J Med. 2002;347:975-982.
5 McHutchison JG, Lawitz EJ, Shiffman ML, et al; IDEAL Study Team. Peginterferon alfa-2b or alfa-2a with ribavirin for treatment of hepatitis C infection. N Engl J Med. 2009;361:580-593.
6 Ghany MG, Strader DB, Thomas DL, Seeff, LB. Diagnosis, management and treatment of hepatitis C; An update. Hepatology. 2009;49 (4):1-40.
7 Morgan TR, Ghany MG, Kim HY, Snow KK, Lindsay K, Lok AS. Outcome of sustained virological responders and non-responders in the Hepatitis C Antiviral Long-Term Treatment Against Cirrhosis (HALT-C) trial. Hepatology. 2008;50(Suppl 4):357A (Abstract 115).
8 Veldt BJ, Heathcote J, Wedmeyer H. Sustained virologic response and clinical outcomes in patients with chronic hepatitis C and advanced fibrosis. Annals of Internal Medicine. 2007; 147: 677-684.
9 Institute of Medicine of the National Academies. Hepatitis and liver cancer: a national strategy for prevention and control of hepatitis B and C. Colvin HM and Mitchell AE, ed. Available at: http://www.iom.edu/Reports/2010/Hepatitis-and-Liver-Cancer-A-National-Strategy-for-Prevention-and-Control-of-Hepatitis-B-and-C.aspx. Updated January 11, 2010. Accessed March 21, 2011.
10 Pyenson B, Fitch K, Iwasaki K. Consequences of hepatitis C virus (HCV): Costs of a baby boomer epidemic of liver disease. Available at: http://www.natap.org/2009/HCV/051809_01.htm. Updated May 2009. Accessed March 21, 2011. This report was commissioned by Vertex Pharmaceuticals, Inc.
11 Volk MI, Tocco R, Saini S, Lok, ASF. Public health impact of antiviral therapy for hepatitis C in the United States. Hepatology. 2009;50(6):1750-1755.
12 Davis GL, Alter MJ, El-Serag H, Poynard T, Jennings LW. Aging of hepatitis C virus (HCV)-infected persons in the United States: A multiple cohort model of HCV prevalence and disease progression. Gastroenterology. 2010;138:513-521.
Contacts
Vertex Pharmaceuticals Incorporated
Media:
Dawn Kalmar, 617-444-6992
or
Amy Pasqua, 617-444-6992
or
Zachry Barber, 617-444-6992
mediainfo@vrtx.com
or
Investors:
Michael Partridge, 617-444-6108
or
Lora Pike, 617-444-6755
or
Matthew Osborne, 617-444-6057
or
Patient Inquiries: 1-855-837-3894
Showing posts with label Hepatitis C therapy. Show all posts
Showing posts with label Hepatitis C therapy. Show all posts
Tuesday, July 26, 2011
Sunday, June 26, 2011
From NATAP.org: HCV Treaters Shortage Editorial - Distributive justice and the arrival of direct-acting antivirals: Who should be first in line?
Full credit to NATAP.org for this "Hepatology" published synopsis and editorial from Donald Jensen and Andrew Aronsohn on 'distributive justice' with the DAA's. Very compelling from an ethical standpoint, worth the read.
HCV Treaters Shortage Editorial - Distributive justice and the arrival of direct-acting antivirals: Who should be first in line?
"The availability of DAA therapy will forever change the landscape of HCV, in that we will be able to cure patients of disease who we were unable to cure in the past. Unfortunately, this medical breakthrough will be coupled with resource scarcity. Historically, some of the greatest scientific discoveries in medicine have failed to provide a distribution system that appropriately emphasized not only justice and equality but also medical need. We hope to learn from the past by proposing a preconceived plan that would both educate patients and allocate therapy to the neediest patients first, thereby fulfilling the moral framework of distributive justice."
"On average, a health care provider can reasonably initiate therapy on only three patients each week before exceeding their work capacity.....current staffing will be unable to meet the demands of all patients with HCV who are expected to request treatment......We propose a needs-based allocation system....patients who have previously deferred therapy and new patients will be prioritized on the basis of need, with patients with cirrhosis at one end of the spectrum and asymptomatic patients with F0-F2 fibrosis at the other end of the spectrum. This system satisfies the principle of justice while placing appropriate emphasis on medical need."
Hepatology
Volume 53, Issue 6, pages 1789Ð1791, June 2011
Andrew Aronsohn M.D.,à, Donald Jensen M.D.
Center for Liver Disease, Section of Gastroenterology, Hepatology and Nutrition, 5841 South Maryland Avenue, MC 7120, Chicago, IL 60637.
In recent years, each edition of HEPATOLOGY has included a viewpoint article written by a member of the Editorial team. This month Dr Michael Charlton has offered his turn at the microphone to Dr. Donald Jensen and Dr Andrew Aronsohn from the University of Chicago, in order that they can address a pressing issue that will emerge in tandem with the likely approval by the U.S. Food and Drug Administration of boceprevir and/or telaprevir.
More than 120 million people are infected with hepatitis C worldwide.1 Hepatitis C virus (HCV) is a leading cause of liver-related mortality and is the most common indication for liver transplantation in the United States.2 Since the introduction of pegylated interferon and ribavirin nearly 10 years ago, response rates have been relatively stagnant, with less than half of treated patients achieving a sustained virological response.2 Data from the first direct-acting antiviral (DAA) agent, BILN 2061, was initially presented at the American Association for the Study of Liver Diseases annual meeting in 2002, which sparked enthusiasm over improving therapeutic efficacy.3 Nearly a decade later, we find ourselves on the brink of a new era of HCV therapy. Telaprevir and boceprevir will likely receive U.S. Food and Drug Administration approval by mid-2011(they both recd FDA approval a few weeks ago), and based on phase 2 and 3 data, will significantly improve rates of sustained virological response in patients infected with HCV genotype 1 when compared to current standard-of-care therapy.4-7 This improved efficacy has been well-publicized for years, and anticipation of DAA availability has already become part of the HCV treatment algorithm. Greater understanding of the natural history of HCV and identification of risk factors for progression to advanced liver disease has allowed many physicians to recommend deferral of standard-of-care therapy in favor of waiting for DAA availability for patients who are at low risk to progress to significant liver disease in the near future. This was demonstrated in a large VA-based study of 4084 patients evaluated for HCV therapy with interferon and ribavirin.8 Of the eligible patients who declined therapy, 50.3% stated they had deferred treatment in anticipation of more effective medications.8 Treatment-naive patients who have deferred standard-of-care therapy, in addition to patients who have failed previous regimens of HCV treatment, will likely create a surge of requests to initiate therapy in mid-2011.
The influx of patients requesting HCV therapy will present a significant problem. HCV therapy is becoming increasingly complex, and the addition of DAAs will only add to the time needed to effectively educate and appropriately monitor patients while they are receiving treatment. This may be partially offset by response-guided therapy that shortens treatment duration. We recently performed a time analysis study at our institution to gain understanding of time allotment needed for each new DAA-treated patient and to estimate the maximum patient capacity of a health care provider. On average, a health care provider can reasonably initiate therapy on only three patients each week before exceeding their work capacity. Because we anticipate at least 500 requests for evaluation for HCV therapy within the first few weeks of DAA availability, current staffing will be unable to meet the demands of all patients with HCV who are expected to request treatment.
Resource scarcity will be a prominent issue once DAA therapy becomes available. Historically, enthusiasm over a scientific breakthrough has caused the medical community to lose sight of the importance of prediction and resolution of problems of scarcity.9 In 1922, Frederick Banting and Charles Best discovered how to produce and use insulin to treat diabetes mellitus. Word of this life-saving medication traveled quickly, creating a deluge of requests for insulin that could not possibly be fulfilled due to limitations of production capacity.10 Unprepared to handle this dilemma, Dr. Banting created a somewhat arbitrary and subjective plan where Banting himself decided whom to treat first, which resulted in friends and politically well-connected individuals unfairly receiving priority over others.9 Twenty years later, penicillin was discovered, and efforts were made to avoid the injustices seen with insulin allocation. From 1942 to 1944, the Committee on Chemotherapeutic and Other Agents was responsible for rationing penicillin to civilians.11 Although efforts were made to prioritize clinical need over political and social worth, penicillin allocation became subject to intense scrutiny due to a double standard in rationing for civilian and military personnel. Penicillin was carefully allocated to civilians based on severity of disease; however, it was liberally given to military personnel to treat gonorrhea, a less serious illness, because it was highly effective and allowed soldiers to quickly return to their duties.9 In 1960, Belding Scribner developed a shunt that allowed patients with chronic kidney disease to safely undergo dialysis.12 Given the paucity of dialysis machines compared to the number of patients with chronic kidney disease, physicians were once again forced to allocate scarce, life-saving technology. A nine-member committee consisting of two physicians and seven lay persons was formed in 1961 to determine who would receive dialysis services. Because most patients with chronic kidney disease had similar medical needs for dialysis, the committee made decisions in part based on a patient's Òsocial worth.Ó9 Not surprisingly, this criterion was widely criticized and eventually led to approval of legislation in 1972 to allow federal funding for hemodialysis to all Americans with chronic kidney disease.
In 2011, nearly a century after the discovery of insulin, we continue to struggle with issues of resource scarcity. Equitable distribution of antiretroviral medication to patients with human immunodeficiency virus worldwide and organ allocation for transplantation illustrate that despite major advances in medical technology, our underlying moral dilemmas remain unchanged. History has taught us that failure to preemptively develop a moral framework to deal with problems of scarcity can lead to injustices to patients in need. Distributive justice is a moral principle that emphasizes Òfair, equitable, and appropriate distributionÓ of scarce resources.13 Equality for all patients should be paramount, yet this should be carefully balanced with appropriate emphasis on medical need. With the arrival of DAA therapy, we will be unable to initially treat all patients requesting therapy, and we will once again be forced to allocate a scarce resource. Critical analysis of this problem has yielded two potential solutions for this allocation dilemma: a first-come, first-served approach and a needs-based approach.
A conventional first-come, first-served approach, as the name implies, offers therapy, when appropriate, to patients in the order in which they are seen in the clinic after DAA launch. If (and when) the health care team becomes saturated, a waiting list will form, and patients on that list will be offered therapy when the health care team has more capacity. This approach has some advantages. It is the simplest plan and requires no planning prior to DAA availability. In fact, this approach will likely be a default system used by any center that has not enacted a preconceived DAA allocation system. First-come, first-served strictly adheres to the principle of justice, because all patients have an equal claim to therapy, and specific prioritizations are not made. However, it is inadequate because it ignores the importance of medical need where the workforce available to treat is limited in relation to the need.
We propose a needs-based allocation system. Much like the Model for End-Stage Liver Disease system, priority would be given to the sickest patients first in an effort to optimize outcomes for all patients with HCV. Prior to the launch of DAA therapy, treatment-eligible patients with HCV could be provided with an educational symposium outlining the natural progression of HCV and describing an allocation system, which would stress the importance of expediting treatment for the sickest patients and the safety of waiting for therapy in patients with early stages of disease. Then, patients who have previously deferred therapy and new patients will be prioritized on the basis of need, with patients with cirrhosis at one end of the spectrum and asymptomatic patients with F0-F2 fibrosis at the other end of the spectrum. This system satisfies the principle of justice while placing appropriate emphasis on medical need.
There will be inherent difficulties in this prioritization system. First, many patients with early stage disease have been eagerly awaiting the launch of DAA therapy and may be disappointed to find out that they may have to wait even longer to initiate therapy. Although it is anticipated that educational sessions will help these patients understand the reasoning behind their increased wait time, some patients will still choose not to wait and will seek more expedited treatment elsewhere. In addition, the choice to treat patients with more advanced liver disease first will likely result in poorer initial outcomes and more complications related to therapy. Finally, there may be medical legal implications to treating patients who are outside of approved indications for therapy. However, the benefit of a morally sound allocation system should outweigh these legal risks, and by ensuring that health care providers have adequate time with each patient, adverse outcomes can be greatly diminished.
The availability of DAA therapy will forever change the landscape of HCV, in that we will be able to cure patients of disease who we were unable to cure in the past. Unfortunately, this medical breakthrough will be coupled with resource scarcity. Historically, some of the greatest scientific discoveries in medicine have failed to provide a distribution system that appropriately emphasized not only justice and equality but also medical need. We hope to learn from the past by proposing a preconceived plan that would both educate patients and allocate therapy to the neediest patients first, thereby fulfilling the moral framework of distributive justice.
References
# 1Shepard CW, Finelli L, Alter MJ. Global epidemiology of hepatitis C virus infection. Lancet Infect Dis 2005; 5: 558-567.
# 2Strader DB, Wright T, Thomas DL, Seeff LB; American Association for the Study of Liver Diseases. Diagnosis, management, and treatment of hepatitis C. HEPATOLOGY 2004; 39: 1147-1171.
# 3Hinrichsen H. First report on the antiviral efficacy of BILN 2061, a novel oral serine protease inhibitor, in patients with chronic hepatitis C genotype 1 [Abstract]. HEPATOLOGY 2002; 36:Abstract 866.
# 4Poordad F, McCone J, Bacon BR, Bruno S, Manns MP, Sulkowski MS, et al. Boceprevir (BOC) combined with peginterferon alfa-2b/ribavirin (P/R) for treatment-na•ve patients with hepatitis C virus (HCV) genotype (G) 1: SPRINT-2 final results [Abstract]. HEPATOLOGY 2010; 52( 4 Suppl.): 402A-403A.
# 5Kwo PY, Lawitz EJ, McCone J, Schiff ER, Vierling JM, Pound D, Davis MN, et al. Efficacy of boceprevir, an NS3 protease inhibitor, in combination with peginterferon alfa-2b and ribavirin in treatment-naive patients with genotype 1 hepatitis C infection (SPRINT-1): an open-label, randomised, multicentre phase 2 trial. Lancet; 376: 705-716.
# 6McHutchison JG, Everson GT, Gordon SC, Jacobson IM, Sulkowski M, Kauffman R, McNair L, et al. Telaprevir with peginterferon and ribavirin for chronic HCV genotype 1 infection. N Engl J Med 2009; 360: 1827-1838.
# 7Sherman KE, Flamm SL, Afdhal NH, Nelson DR, Sulkowski MS, Everson GT, et al. Telaprevir in combination with peginterferon alfa2a and ribavirin for 24 or 48 weeks in treatment-na•ve genotype 1 HCV patients who achieved an extended rapid viral response: Final results of phase 3 ILLUMINATE study [Abstract]. HEPATOLOGY 2010; 52( 4 Suppl.): 401A-402A.
# 8Bini EJ, Brau N, Currie S, Shen H, Anand BS, Hu KQ, Jeffers L, et al. Prospective multicenter study of eligibility for antiviral therapy among 4,084 U.S. veterans with chronic hepatitis C virus infection. Am J Gastroenterol 2005;100:1772-1779.
# 9McGough LJ, Reynolds SJ, Quinn TC, Zenilman JM. Which patients first? Setting priorities for antiretroviral therapy where resources are limited. Am J Public Health 2005; 95: 1173-1180.
# 10Shampo MA, Kyle RA. Frederick bantingÐNobel laureate for discovery of insulin. Mayo Clin Proc 2005; 80: 576.
# 11Adams DP. Wartime bureaucracy and penicillin allocation: the Committee on Chemotherapeutic and Other Agents, 1942Ð44. J Hist Med Allied Sci 1989; 44: 196-217.
# 12Blagg CR. The early history of dialysis for chronic renal failure in the United States: a view from Seattle. Am J Kidney Dis 2007; 49: 482-496.
# 13Beauchamp TL, Childress JF. Principles of Biomedical Ethics. 5th ed. New York, NY: Oxford University Press; 2001.
HCV Treaters Shortage Editorial - Distributive justice and the arrival of direct-acting antivirals: Who should be first in line?
"The availability of DAA therapy will forever change the landscape of HCV, in that we will be able to cure patients of disease who we were unable to cure in the past. Unfortunately, this medical breakthrough will be coupled with resource scarcity. Historically, some of the greatest scientific discoveries in medicine have failed to provide a distribution system that appropriately emphasized not only justice and equality but also medical need. We hope to learn from the past by proposing a preconceived plan that would both educate patients and allocate therapy to the neediest patients first, thereby fulfilling the moral framework of distributive justice."
"On average, a health care provider can reasonably initiate therapy on only three patients each week before exceeding their work capacity.....current staffing will be unable to meet the demands of all patients with HCV who are expected to request treatment......We propose a needs-based allocation system....patients who have previously deferred therapy and new patients will be prioritized on the basis of need, with patients with cirrhosis at one end of the spectrum and asymptomatic patients with F0-F2 fibrosis at the other end of the spectrum. This system satisfies the principle of justice while placing appropriate emphasis on medical need."
Hepatology
Volume 53, Issue 6, pages 1789Ð1791, June 2011
Andrew Aronsohn M.D.,à, Donald Jensen M.D.
Center for Liver Disease, Section of Gastroenterology, Hepatology and Nutrition, 5841 South Maryland Avenue, MC 7120, Chicago, IL 60637.
In recent years, each edition of HEPATOLOGY has included a viewpoint article written by a member of the Editorial team. This month Dr Michael Charlton has offered his turn at the microphone to Dr. Donald Jensen and Dr Andrew Aronsohn from the University of Chicago, in order that they can address a pressing issue that will emerge in tandem with the likely approval by the U.S. Food and Drug Administration of boceprevir and/or telaprevir.
More than 120 million people are infected with hepatitis C worldwide.1 Hepatitis C virus (HCV) is a leading cause of liver-related mortality and is the most common indication for liver transplantation in the United States.2 Since the introduction of pegylated interferon and ribavirin nearly 10 years ago, response rates have been relatively stagnant, with less than half of treated patients achieving a sustained virological response.2 Data from the first direct-acting antiviral (DAA) agent, BILN 2061, was initially presented at the American Association for the Study of Liver Diseases annual meeting in 2002, which sparked enthusiasm over improving therapeutic efficacy.3 Nearly a decade later, we find ourselves on the brink of a new era of HCV therapy. Telaprevir and boceprevir will likely receive U.S. Food and Drug Administration approval by mid-2011(they both recd FDA approval a few weeks ago), and based on phase 2 and 3 data, will significantly improve rates of sustained virological response in patients infected with HCV genotype 1 when compared to current standard-of-care therapy.4-7 This improved efficacy has been well-publicized for years, and anticipation of DAA availability has already become part of the HCV treatment algorithm. Greater understanding of the natural history of HCV and identification of risk factors for progression to advanced liver disease has allowed many physicians to recommend deferral of standard-of-care therapy in favor of waiting for DAA availability for patients who are at low risk to progress to significant liver disease in the near future. This was demonstrated in a large VA-based study of 4084 patients evaluated for HCV therapy with interferon and ribavirin.8 Of the eligible patients who declined therapy, 50.3% stated they had deferred treatment in anticipation of more effective medications.8 Treatment-naive patients who have deferred standard-of-care therapy, in addition to patients who have failed previous regimens of HCV treatment, will likely create a surge of requests to initiate therapy in mid-2011.
The influx of patients requesting HCV therapy will present a significant problem. HCV therapy is becoming increasingly complex, and the addition of DAAs will only add to the time needed to effectively educate and appropriately monitor patients while they are receiving treatment. This may be partially offset by response-guided therapy that shortens treatment duration. We recently performed a time analysis study at our institution to gain understanding of time allotment needed for each new DAA-treated patient and to estimate the maximum patient capacity of a health care provider. On average, a health care provider can reasonably initiate therapy on only three patients each week before exceeding their work capacity. Because we anticipate at least 500 requests for evaluation for HCV therapy within the first few weeks of DAA availability, current staffing will be unable to meet the demands of all patients with HCV who are expected to request treatment.
Resource scarcity will be a prominent issue once DAA therapy becomes available. Historically, enthusiasm over a scientific breakthrough has caused the medical community to lose sight of the importance of prediction and resolution of problems of scarcity.9 In 1922, Frederick Banting and Charles Best discovered how to produce and use insulin to treat diabetes mellitus. Word of this life-saving medication traveled quickly, creating a deluge of requests for insulin that could not possibly be fulfilled due to limitations of production capacity.10 Unprepared to handle this dilemma, Dr. Banting created a somewhat arbitrary and subjective plan where Banting himself decided whom to treat first, which resulted in friends and politically well-connected individuals unfairly receiving priority over others.9 Twenty years later, penicillin was discovered, and efforts were made to avoid the injustices seen with insulin allocation. From 1942 to 1944, the Committee on Chemotherapeutic and Other Agents was responsible for rationing penicillin to civilians.11 Although efforts were made to prioritize clinical need over political and social worth, penicillin allocation became subject to intense scrutiny due to a double standard in rationing for civilian and military personnel. Penicillin was carefully allocated to civilians based on severity of disease; however, it was liberally given to military personnel to treat gonorrhea, a less serious illness, because it was highly effective and allowed soldiers to quickly return to their duties.9 In 1960, Belding Scribner developed a shunt that allowed patients with chronic kidney disease to safely undergo dialysis.12 Given the paucity of dialysis machines compared to the number of patients with chronic kidney disease, physicians were once again forced to allocate scarce, life-saving technology. A nine-member committee consisting of two physicians and seven lay persons was formed in 1961 to determine who would receive dialysis services. Because most patients with chronic kidney disease had similar medical needs for dialysis, the committee made decisions in part based on a patient's Òsocial worth.Ó9 Not surprisingly, this criterion was widely criticized and eventually led to approval of legislation in 1972 to allow federal funding for hemodialysis to all Americans with chronic kidney disease.
In 2011, nearly a century after the discovery of insulin, we continue to struggle with issues of resource scarcity. Equitable distribution of antiretroviral medication to patients with human immunodeficiency virus worldwide and organ allocation for transplantation illustrate that despite major advances in medical technology, our underlying moral dilemmas remain unchanged. History has taught us that failure to preemptively develop a moral framework to deal with problems of scarcity can lead to injustices to patients in need. Distributive justice is a moral principle that emphasizes Òfair, equitable, and appropriate distributionÓ of scarce resources.13 Equality for all patients should be paramount, yet this should be carefully balanced with appropriate emphasis on medical need. With the arrival of DAA therapy, we will be unable to initially treat all patients requesting therapy, and we will once again be forced to allocate a scarce resource. Critical analysis of this problem has yielded two potential solutions for this allocation dilemma: a first-come, first-served approach and a needs-based approach.
A conventional first-come, first-served approach, as the name implies, offers therapy, when appropriate, to patients in the order in which they are seen in the clinic after DAA launch. If (and when) the health care team becomes saturated, a waiting list will form, and patients on that list will be offered therapy when the health care team has more capacity. This approach has some advantages. It is the simplest plan and requires no planning prior to DAA availability. In fact, this approach will likely be a default system used by any center that has not enacted a preconceived DAA allocation system. First-come, first-served strictly adheres to the principle of justice, because all patients have an equal claim to therapy, and specific prioritizations are not made. However, it is inadequate because it ignores the importance of medical need where the workforce available to treat is limited in relation to the need.
We propose a needs-based allocation system. Much like the Model for End-Stage Liver Disease system, priority would be given to the sickest patients first in an effort to optimize outcomes for all patients with HCV. Prior to the launch of DAA therapy, treatment-eligible patients with HCV could be provided with an educational symposium outlining the natural progression of HCV and describing an allocation system, which would stress the importance of expediting treatment for the sickest patients and the safety of waiting for therapy in patients with early stages of disease. Then, patients who have previously deferred therapy and new patients will be prioritized on the basis of need, with patients with cirrhosis at one end of the spectrum and asymptomatic patients with F0-F2 fibrosis at the other end of the spectrum. This system satisfies the principle of justice while placing appropriate emphasis on medical need.
There will be inherent difficulties in this prioritization system. First, many patients with early stage disease have been eagerly awaiting the launch of DAA therapy and may be disappointed to find out that they may have to wait even longer to initiate therapy. Although it is anticipated that educational sessions will help these patients understand the reasoning behind their increased wait time, some patients will still choose not to wait and will seek more expedited treatment elsewhere. In addition, the choice to treat patients with more advanced liver disease first will likely result in poorer initial outcomes and more complications related to therapy. Finally, there may be medical legal implications to treating patients who are outside of approved indications for therapy. However, the benefit of a morally sound allocation system should outweigh these legal risks, and by ensuring that health care providers have adequate time with each patient, adverse outcomes can be greatly diminished.
The availability of DAA therapy will forever change the landscape of HCV, in that we will be able to cure patients of disease who we were unable to cure in the past. Unfortunately, this medical breakthrough will be coupled with resource scarcity. Historically, some of the greatest scientific discoveries in medicine have failed to provide a distribution system that appropriately emphasized not only justice and equality but also medical need. We hope to learn from the past by proposing a preconceived plan that would both educate patients and allocate therapy to the neediest patients first, thereby fulfilling the moral framework of distributive justice.
References
# 1Shepard CW, Finelli L, Alter MJ. Global epidemiology of hepatitis C virus infection. Lancet Infect Dis 2005; 5: 558-567.
# 2Strader DB, Wright T, Thomas DL, Seeff LB; American Association for the Study of Liver Diseases. Diagnosis, management, and treatment of hepatitis C. HEPATOLOGY 2004; 39: 1147-1171.
# 3Hinrichsen H. First report on the antiviral efficacy of BILN 2061, a novel oral serine protease inhibitor, in patients with chronic hepatitis C genotype 1 [Abstract]. HEPATOLOGY 2002; 36:Abstract 866.
# 4Poordad F, McCone J, Bacon BR, Bruno S, Manns MP, Sulkowski MS, et al. Boceprevir (BOC) combined with peginterferon alfa-2b/ribavirin (P/R) for treatment-na•ve patients with hepatitis C virus (HCV) genotype (G) 1: SPRINT-2 final results [Abstract]. HEPATOLOGY 2010; 52( 4 Suppl.): 402A-403A.
# 5Kwo PY, Lawitz EJ, McCone J, Schiff ER, Vierling JM, Pound D, Davis MN, et al. Efficacy of boceprevir, an NS3 protease inhibitor, in combination with peginterferon alfa-2b and ribavirin in treatment-naive patients with genotype 1 hepatitis C infection (SPRINT-1): an open-label, randomised, multicentre phase 2 trial. Lancet; 376: 705-716.
# 6McHutchison JG, Everson GT, Gordon SC, Jacobson IM, Sulkowski M, Kauffman R, McNair L, et al. Telaprevir with peginterferon and ribavirin for chronic HCV genotype 1 infection. N Engl J Med 2009; 360: 1827-1838.
# 7Sherman KE, Flamm SL, Afdhal NH, Nelson DR, Sulkowski MS, Everson GT, et al. Telaprevir in combination with peginterferon alfa2a and ribavirin for 24 or 48 weeks in treatment-na•ve genotype 1 HCV patients who achieved an extended rapid viral response: Final results of phase 3 ILLUMINATE study [Abstract]. HEPATOLOGY 2010; 52( 4 Suppl.): 401A-402A.
# 8Bini EJ, Brau N, Currie S, Shen H, Anand BS, Hu KQ, Jeffers L, et al. Prospective multicenter study of eligibility for antiviral therapy among 4,084 U.S. veterans with chronic hepatitis C virus infection. Am J Gastroenterol 2005;100:1772-1779.
# 9McGough LJ, Reynolds SJ, Quinn TC, Zenilman JM. Which patients first? Setting priorities for antiretroviral therapy where resources are limited. Am J Public Health 2005; 95: 1173-1180.
# 10Shampo MA, Kyle RA. Frederick bantingÐNobel laureate for discovery of insulin. Mayo Clin Proc 2005; 80: 576.
# 11Adams DP. Wartime bureaucracy and penicillin allocation: the Committee on Chemotherapeutic and Other Agents, 1942Ð44. J Hist Med Allied Sci 1989; 44: 196-217.
# 12Blagg CR. The early history of dialysis for chronic renal failure in the United States: a view from Seattle. Am J Kidney Dis 2007; 49: 482-496.
# 13Beauchamp TL, Childress JF. Principles of Biomedical Ethics. 5th ed. New York, NY: Oxford University Press; 2001.
Sunday, June 19, 2011
NATAP presents "New Advances in Hep C Therapy: The World Is Changing" conference in NYC, Saturday June 25, 2011
S a t u r d a y, June 25th, 2011, 10am-2:30pm
NYU Medical Center: Schwartz Lecture Hall F
550 First Avenue, N.Y. NY 10016 (Between 31st and 32nd st)
New Advances in Hep C Therapy:
The World is Changing!
Speakers: Ira Jacobson MD: Vincent Astor distinguished Professor of Medicine
Cheif, Division of Gastroenterology & Hepatolog Medical Director Center for the study of Hepatitis C.
Andrew Talal MD: Associate Professor of Medicine & Associate Medical Director of the Center for the Study of Hepatitis C.
Nurse Mary Olson: Director, Hepatitis Clinical Trails: From Weill Cornell Medical College in NYC
• How to talk to your Care Provider
• Care Management & side effects
• New HCV Drugs
• HCV/HIV Coinfection treatment issues
• When to begin therapy?
• Future new HCV drugs in development
• Taking new HCV therapy properly
• What is “Response-Guided Therapy”?
• Drug Resistance
F r e e
F o r u m !
Seating is limited.
To reserve your seat:
Call: 1 (888)-266-2827
Fax: (212) 219- 8473
or Email- register@natap.org
Free Breakfast & Raffle!
Nursing contact hours available (must attend entire session), CASAC, and
certificates of attendance.
Breakfast & Registration Begin promptly at 8:30am
This Nursing education activity was approved by NCNA an accredited
approver by the American Nurses credentialing center’s commission on
accreditation. This educational activity has been awarded 3 . 6 6 contact hours
NYU Medical Center: Schwartz Lecture Hall F
550 First Avenue, N.Y. NY 10016 (Between 31st and 32nd st)
New Advances in Hep C Therapy:
The World is Changing!
Speakers: Ira Jacobson MD: Vincent Astor distinguished Professor of Medicine
Cheif, Division of Gastroenterology & Hepatolog Medical Director Center for the study of Hepatitis C.
Andrew Talal MD: Associate Professor of Medicine & Associate Medical Director of the Center for the Study of Hepatitis C.
Nurse Mary Olson: Director, Hepatitis Clinical Trails: From Weill Cornell Medical College in NYC
• How to talk to your Care Provider
• Care Management & side effects
• New HCV Drugs
• HCV/HIV Coinfection treatment issues
• When to begin therapy?
• Future new HCV drugs in development
• Taking new HCV therapy properly
• What is “Response-Guided Therapy”?
• Drug Resistance
F r e e
F o r u m !
Seating is limited.
To reserve your seat:
Call: 1 (888)-266-2827
Fax: (212) 219- 8473
or Email- register@natap.org
Free Breakfast & Raffle!
Nursing contact hours available (must attend entire session), CASAC, and
certificates of attendance.
Breakfast & Registration Begin promptly at 8:30am
This Nursing education activity was approved by NCNA an accredited
approver by the American Nurses credentialing center’s commission on
accreditation. This educational activity has been awarded 3 . 6 6 contact hours
Tuesday, March 29, 2011
C-Pharma - new company focused on development of Hepatitis C products
Cyplasin Biomedical Ltd Announces Name Change And Creation of a Subsidiary C-Pharma Inc.
EDMONTON, March 29 /CNW/ - Cyplasin Biomedical is a product-oriented, specialty pharmaceutical company focused on developing products for multi-billion dollar markets. The Company is pleased to announce that in order to better reflect multiple new product development opportunities and to continue its current product development programs for Hepatitis C; will effective immediately, change its name to Compass Biotechnologies Inc., and will continue to be traded on the OTC Link exchange under a new trading symbol "COBI".
Compass Biotechnologies Inc., plans on developing several new technologies and revenue generating products and through its wholly owned subsidiary C-Pharma, continue to finance and develop the previously announced Hepatitis C products.
Compass CEO Garth Likes commented, "The creation of C-Pharma to focus solely on the current and future Hepatitis franchise creates a dedicated focus AND VALUE for our shareholders. Compass Biotechnologies will in parallel be able to take advantage of several new and complementary technologies and products that will further add increased value. The new product opportunities within Compass will be announced in the near future as the various components of the project are finalized."
Shareholders of the previously named company "Cyplasin" will be able to physically exchange their share certificates for "Compass" certificates by contacting the Company's transfer agent Pacific Stock Transfer. Detailed exchange instructions will be posted on our website www.c-pharma.net in the next few days.
ABOUT Compass Biotechnology Inc and its wholly owned subsidiary C-Pharma Inc : http://www.c-pharma.net/
Compass Biotechnologies Inc., is a publically-traded specialty pharmaceutical company (OTCQB:COBI) with headquarters in Edmonton, Alberta.
C-PHARMA Inc has amassed a portfolio of key products for the treatment and prevention of HCV infection, which affects over 4 million people in the U.S. and several hundred million people worldwide.
C-Pharma's technology encompasses the use of recombinant DNA technology to manufacture recombinant cytokines and virus like particles (VLPs). VLPs can be engineered to incorporate various viral and non-viral antigens for use as vaccines against many different types of targets such as hepatitis C. C-Pharma is using the technology to develop a hepatitis C vaccine ( C- Vaxin) to prevent hepatitis C viral infection. The Company is also pursuing a revenue generation strategy by commercializing highly profitable therapeutic drugs (C-Virin & C-Pegferon) for use in the hepatitis C market.
EDMONTON, March 29 /CNW/ - Cyplasin Biomedical is a product-oriented, specialty pharmaceutical company focused on developing products for multi-billion dollar markets. The Company is pleased to announce that in order to better reflect multiple new product development opportunities and to continue its current product development programs for Hepatitis C; will effective immediately, change its name to Compass Biotechnologies Inc., and will continue to be traded on the OTC Link exchange under a new trading symbol "COBI".
Compass Biotechnologies Inc., plans on developing several new technologies and revenue generating products and through its wholly owned subsidiary C-Pharma, continue to finance and develop the previously announced Hepatitis C products.
Compass CEO Garth Likes commented, "The creation of C-Pharma to focus solely on the current and future Hepatitis franchise creates a dedicated focus AND VALUE for our shareholders. Compass Biotechnologies will in parallel be able to take advantage of several new and complementary technologies and products that will further add increased value. The new product opportunities within Compass will be announced in the near future as the various components of the project are finalized."
Shareholders of the previously named company "Cyplasin" will be able to physically exchange their share certificates for "Compass" certificates by contacting the Company's transfer agent Pacific Stock Transfer. Detailed exchange instructions will be posted on our website www.c-pharma.net in the next few days.
ABOUT Compass Biotechnology Inc and its wholly owned subsidiary C-Pharma Inc : http://www.c-pharma.net/
Compass Biotechnologies Inc., is a publically-traded specialty pharmaceutical company (OTCQB:COBI) with headquarters in Edmonton, Alberta.
C-PHARMA Inc has amassed a portfolio of key products for the treatment and prevention of HCV infection, which affects over 4 million people in the U.S. and several hundred million people worldwide.
C-Pharma's technology encompasses the use of recombinant DNA technology to manufacture recombinant cytokines and virus like particles (VLPs). VLPs can be engineered to incorporate various viral and non-viral antigens for use as vaccines against many different types of targets such as hepatitis C. C-Pharma is using the technology to develop a hepatitis C vaccine ( C- Vaxin) to prevent hepatitis C viral infection. The Company is also pursuing a revenue generation strategy by commercializing highly profitable therapeutic drugs (C-Virin & C-Pegferon) for use in the hepatitis C market.
Saturday, December 18, 2010
SciClone drops development of SCV-07 for previous HCV therapy relapsers....
"We are probably going to see more companies drop development programs for HCV therapies as the treatment landscape and probabilities of advancement for competitive compounds continues to increase. At some point, the sponsoring company has to make a decision based on simple cost/benefit ratios and potential for a compound to sustain itself in what will become a crowded market. SciClone will continue its development program for SCV-07 for oral mucositis in patients with head and neck cancer - Chris"
FOSTER CITY, CA -- (MARKET WIRE) -- 12/15/10 -- SciClone Pharmaceuticals, Inc. (NASDAQ: SCLN) today announced topline results from the Company's phase 2b clinical trial of SCV-07 for the treatment of hepatitis C (HCV). The study evaluated the safety and immunomodulatory effects of SCV-07 as a monotherapy and in combination with ribavirin in relapsed HCV patients. Study data demonstrated SCV-07 to be safe and well-tolerated at both administered doses. Topline results showed a clear biological signal from SCV-07 but did not meet the study's primary efficacy endpoint of a 2 log reduction in viral load from baseline level. A secondary measure of efficacy, defined as a reduction in viral load of greater than 0.5 log from baseline level, was seen in 38.5% of the low-dose patients (5/13) and in 44.4% of the high-dose patients (8/18). Additionally, while no patients in the low-dose group achieved greater than a 1 log reduction, 3 of the high-dose patients achieved greater than a 1 log reduction in viral load. This proof of concept study was designed to provide an estimate of SCV-07's treatment effect in relapsed HCV patients and guide further studies of SCV-07 in addressing this chronic infection.
"Although the data showed an interesting biological signal, due to the rapidly changing landscape of effective treatments which increase the complexity and risks of developing drugs in chronic HCV, we have decided not to continue development in this indication. On another front, we continue to be excited about the potential for SCV-07 in the prevention of oral mucositis in patients with head and neck cancer and the initiation of our phase 2b study, which should begin by early 2011," stated Friedhelm Blobel, Ph.D., President and Chief Executive Officer of SciClone. "Our primary focus remains on rapidly growing our commercially successful specialty pharmaceutical business in China and other key emerging markets to increase profitability and generate cash for our shareholders."
Study Design
The phase 2b multicenter, multi-dose, open-label study was designed to evaluate the safety and immunomodulatory effects of SCV-07 as a monotherapy or in combination with ribavirin in non-cirrhotic patients with genotype 1 chronic HCV who have relapsed after at least 44 weeks of treatment with pegylated interferon and ribavirin. The study, which also monitored biomarkers of immune activation and HCV viral load dynamics, included two treatment cohorts of 20 patients each who received SCV-07 at a dose of either 0.1 mg/kg or 1.0 mg/kg. The eight week treatment period included four weeks of SCV-07 monotherapy followed by four weeks of SCV-07 in combination with ribavirin. The trial also included three follow-up visits within seven weeks after the completion of treatment.
About SCV-07
SCV-07 (gamma-D-glutamyl-L-tryptophan) is a small molecule which appears to stimulate the immune system through inhibition of STAT3 signaling and the resulting effects on T-helper 1 cells. SCV-07 has been shown to be efficacious in animal models of immune-sensitive diseases, including prevention of oral mucositis, treatment of cancer, viral infections, and enhancement of response to vaccines.
Additionally, SciClone is currently planning to initiate a phase 2b study of SCV-07 for the prevention of oral mucositis by early 2011. As compared to the company's recently completed phase 2a trial, the phase 2b study design is expected to include higher doses of SCV-07 and be adequately powered to demonstrate statistical significance. Additionally, researchers expect to continue to investigate the role of specific genetic profiles on patient response to SCV-07, as well as the potential link between cytokine activity and SCV-07's sub-cellular mechanism of action.
SCV-07 is protected by composition of matter patents as well as multiple method of treatment patents. SciClone has exclusive worldwide rights to SCV-07 outside of Russia, where the molecule has recently been approved for stimulation of depressed immune systems.
About SciClone
SciClone Pharmaceuticals (NASDAQ: SCLN) is a revenue-generating, China-centric, specialty pharmaceutical company with a substantial international business and a product portfolio of novel therapies for cancer and infectious diseases. The Company is focused on continuing sales growth and executing a clinical development strategy with prudently managed costs. ZADAXIN® (thymalfasin) is approved in over 30 countries for the treatment of hepatitis B (HBV) and hepatitis C (HCV), certain cancers, and as a vaccine adjuvant. In addition to further studying thymalfasin's use as a vaccine enhancer, SciClone is planning to evaluate SCV-07 in a phase 2b trial to modify the course of oral mucositis in patients with head and neck cancer; and recently completed a phase 2b trial of SCV-07 for the treatment of HCV. The Company also has exclusive commercialization and distribution rights in China to a novel treatment for advanced liver cancer, DC Bead®, currently under review by regulatory agencies in that country. Additionally, SciClone owns exclusive commercialization and distribution rights to the anti-nausea drug ondansetron RapidFilm® in China, including Hong Kong and Macau, and Vietnam. The Company intends to seek regulatory approval for the product, commonly used to treat and prevent nausea and vomiting caused by chemotherapy, radiotherapy, and surgery, in these markets. For additional information, please visit www.sciclone.com.
Forward-looking statements
The information in this press release contains forward-looking statements, including our expectations and beliefs regarding the timing and results of our clinical trials. You are urged to consider statements that include the words "may," "will," "would," "could," "should," "might," "believes," "estimates," "projects," "potential," "expects," "plans," "anticipates," "intends," "continues," "forecast," "designed," "goal," or the negative of those words or other comparable words to be uncertain and forward-looking. These statements are subject to risks and uncertainties that are difficult to predict and actual outcomes may differ materially. These risks and uncertainties include our forward-looking statements regarding our commercial and development objectives because of uncertainties, including future sales, product pricing, the timing of clinical trial events such as patient enrollment, requirements of, and future actions of, the U.S. Food and Drug Administration, the fact that experimental data, and clinical results derived from studies with animals or a limited group of patients, as well as comparisons with other clinical trials, may not be predictive of the results of larger studies and, therefore, such experimental or clinical data are not necessarily predictive indicative of the efficacy or safety or the results of larger studies and clinical trials. Please also refer to the other risks and uncertainties described in SciClone's filings with the Securities and Exchange Commission. All forward-looking statements are based on information currently available to SciClone, and SciClone assumes no obligation to update any such forward-looking statements.
DC Bead is a registered trademark of Biocompatibles UK Limited.
RapidFilm is a registered trademark of Labtec Gesellschaft für technologische Forschung und Entwicklung mbH.
Source: SciClone Pharmaceuticals, Inc.
News Provided by Acquire Media
FOSTER CITY, CA -- (MARKET WIRE) -- 12/15/10 -- SciClone Pharmaceuticals, Inc. (NASDAQ: SCLN) today announced topline results from the Company's phase 2b clinical trial of SCV-07 for the treatment of hepatitis C (HCV). The study evaluated the safety and immunomodulatory effects of SCV-07 as a monotherapy and in combination with ribavirin in relapsed HCV patients. Study data demonstrated SCV-07 to be safe and well-tolerated at both administered doses. Topline results showed a clear biological signal from SCV-07 but did not meet the study's primary efficacy endpoint of a 2 log reduction in viral load from baseline level. A secondary measure of efficacy, defined as a reduction in viral load of greater than 0.5 log from baseline level, was seen in 38.5% of the low-dose patients (5/13) and in 44.4% of the high-dose patients (8/18). Additionally, while no patients in the low-dose group achieved greater than a 1 log reduction, 3 of the high-dose patients achieved greater than a 1 log reduction in viral load. This proof of concept study was designed to provide an estimate of SCV-07's treatment effect in relapsed HCV patients and guide further studies of SCV-07 in addressing this chronic infection.
"Although the data showed an interesting biological signal, due to the rapidly changing landscape of effective treatments which increase the complexity and risks of developing drugs in chronic HCV, we have decided not to continue development in this indication. On another front, we continue to be excited about the potential for SCV-07 in the prevention of oral mucositis in patients with head and neck cancer and the initiation of our phase 2b study, which should begin by early 2011," stated Friedhelm Blobel, Ph.D., President and Chief Executive Officer of SciClone. "Our primary focus remains on rapidly growing our commercially successful specialty pharmaceutical business in China and other key emerging markets to increase profitability and generate cash for our shareholders."
Study Design
The phase 2b multicenter, multi-dose, open-label study was designed to evaluate the safety and immunomodulatory effects of SCV-07 as a monotherapy or in combination with ribavirin in non-cirrhotic patients with genotype 1 chronic HCV who have relapsed after at least 44 weeks of treatment with pegylated interferon and ribavirin. The study, which also monitored biomarkers of immune activation and HCV viral load dynamics, included two treatment cohorts of 20 patients each who received SCV-07 at a dose of either 0.1 mg/kg or 1.0 mg/kg. The eight week treatment period included four weeks of SCV-07 monotherapy followed by four weeks of SCV-07 in combination with ribavirin. The trial also included three follow-up visits within seven weeks after the completion of treatment.
About SCV-07
SCV-07 (gamma-D-glutamyl-L-tryptophan) is a small molecule which appears to stimulate the immune system through inhibition of STAT3 signaling and the resulting effects on T-helper 1 cells. SCV-07 has been shown to be efficacious in animal models of immune-sensitive diseases, including prevention of oral mucositis, treatment of cancer, viral infections, and enhancement of response to vaccines.
Additionally, SciClone is currently planning to initiate a phase 2b study of SCV-07 for the prevention of oral mucositis by early 2011. As compared to the company's recently completed phase 2a trial, the phase 2b study design is expected to include higher doses of SCV-07 and be adequately powered to demonstrate statistical significance. Additionally, researchers expect to continue to investigate the role of specific genetic profiles on patient response to SCV-07, as well as the potential link between cytokine activity and SCV-07's sub-cellular mechanism of action.
SCV-07 is protected by composition of matter patents as well as multiple method of treatment patents. SciClone has exclusive worldwide rights to SCV-07 outside of Russia, where the molecule has recently been approved for stimulation of depressed immune systems.
About SciClone
SciClone Pharmaceuticals (NASDAQ: SCLN) is a revenue-generating, China-centric, specialty pharmaceutical company with a substantial international business and a product portfolio of novel therapies for cancer and infectious diseases. The Company is focused on continuing sales growth and executing a clinical development strategy with prudently managed costs. ZADAXIN® (thymalfasin) is approved in over 30 countries for the treatment of hepatitis B (HBV) and hepatitis C (HCV), certain cancers, and as a vaccine adjuvant. In addition to further studying thymalfasin's use as a vaccine enhancer, SciClone is planning to evaluate SCV-07 in a phase 2b trial to modify the course of oral mucositis in patients with head and neck cancer; and recently completed a phase 2b trial of SCV-07 for the treatment of HCV. The Company also has exclusive commercialization and distribution rights in China to a novel treatment for advanced liver cancer, DC Bead®, currently under review by regulatory agencies in that country. Additionally, SciClone owns exclusive commercialization and distribution rights to the anti-nausea drug ondansetron RapidFilm® in China, including Hong Kong and Macau, and Vietnam. The Company intends to seek regulatory approval for the product, commonly used to treat and prevent nausea and vomiting caused by chemotherapy, radiotherapy, and surgery, in these markets. For additional information, please visit www.sciclone.com.
Forward-looking statements
The information in this press release contains forward-looking statements, including our expectations and beliefs regarding the timing and results of our clinical trials. You are urged to consider statements that include the words "may," "will," "would," "could," "should," "might," "believes," "estimates," "projects," "potential," "expects," "plans," "anticipates," "intends," "continues," "forecast," "designed," "goal," or the negative of those words or other comparable words to be uncertain and forward-looking. These statements are subject to risks and uncertainties that are difficult to predict and actual outcomes may differ materially. These risks and uncertainties include our forward-looking statements regarding our commercial and development objectives because of uncertainties, including future sales, product pricing, the timing of clinical trial events such as patient enrollment, requirements of, and future actions of, the U.S. Food and Drug Administration, the fact that experimental data, and clinical results derived from studies with animals or a limited group of patients, as well as comparisons with other clinical trials, may not be predictive of the results of larger studies and, therefore, such experimental or clinical data are not necessarily predictive indicative of the efficacy or safety or the results of larger studies and clinical trials. Please also refer to the other risks and uncertainties described in SciClone's filings with the Securities and Exchange Commission. All forward-looking statements are based on information currently available to SciClone, and SciClone assumes no obligation to update any such forward-looking statements.
DC Bead is a registered trademark of Biocompatibles UK Limited.
RapidFilm is a registered trademark of Labtec Gesellschaft für technologische Forschung und Entwicklung mbH.
Source: SciClone Pharmaceuticals, Inc.
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