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Showing posts with label News. Show all posts
Showing posts with label News. Show all posts

Saturday, March 19, 2016

Canadian Clinical Trials Asset Map was launched by researchers



The Canadian Clinical Trials Asset Map was launched by researchers, industry leaders and policy-makers including, left to right, Dr. Shurjeel Choudhri, Bayer Inc., Belinda Vandersluis, director of the Canadian Clinical Trials Coordinating Centre, and Senator Kelvin Ogilvie.

Canada is aiming to regain its research edge through a new national database highlighting the country’s resources for clinical trials in the hope it will attract international companies doing research.
The Canadian Clinical Trials Asset Map (CCTAM) website went live June 4 with over 800 database records, and it is expected to grow. The map provides information on conditions and diseases being studied, and identifies gaps. For example, a search for cardiology in the system, would allow you to see all the clinical sites involved in research in that domain on a Google map. The map also documents an investigator’s specialty, trial phase and the patient population. Registration is free, although user accounts have to be pre-approved by an administrator.
The designers of the asset map say that there is oversight to ensure the information about the research sites is accurate and up to date. The website’s software finds older records and asks the owner of of the records to provide more recent data, Dr. Shurjeel Choudhri, senior vice president and head, Medical and Scientific Affairs, Bayer Inc., toldCMAJ.
“Our goal is to have something that is sustainable. It has mechanisms built within it to maintain and keep itself updated so it will be a living document that will be available to Canadians for a long time to come,” said Choudhri.
The asset map was funded and led by multiple partners, including the Canadian Institutes of Health Research, Rx&D and the advocacy organization, HealthCareCAN.
Conservative Senator, Kelvin Ogilvie, a renowned biotechnologist and researcher observed that “the number of Canadian clinical trials has been declining for over a decade.”
Ogilvie hopes the map will “at the very least stem the decline.” However, Canada faces many barriers to becoming a research leader. Not the least of these is the unwillingness of research centres to set common requirements for clinical trials. This inhibits our ability to compete with other countries in a timely fashion.
The asset map is the first tangible outcome of the 2011 Canadian Clinical Trial Summit where research-industry leaders developed a plan for improving the ability to conduct research in Canada. The summit concluded that Canada’s system is expensive and that having numerous ethical boards hinders the timeliness of trial start-ups. The online database may not speed up ethical review boards for trials, but at least all the different components involved in running a clinical trial in Canada will be available in one place.
Belinda Vandersluis, the director of the Canadian Clinical Trials Coordinating Centre(CCTCC) said Canada has a long-time reputation for conducting practice-changing, high-quality clinical trials, with internationally renowned researchers. “We don’t want to lose the reputation, the researchers, or the opportunity to study innovative and important new drugs, devices and vaccines.”
The CCTCC was established in April 2014 with a mandate to improve the clinical-trial landscape. Its $1.5 million in funding came from the same partners as the asset map. Although it only has a three-year directive, the national database will continue to run.
Pharmaceutical companies were active partners in designing the national asset map. Choudhri offered advice on how Canada can compete with other countries. “We typically have only a few weeks to identify potential sites for the trial, determine the patient population needed for that trial. By having this tool you can do a quick search and identify the sites that are active in that particular area.”
Before the national map was created, there were about 25 smaller asset maps in Canada, said Choudhri, but the data was fragmented among federal, provincial and regional bodies and some of the information was only available on paper.
“All had some gaps and a lot of them were out of date. Those were the inspiration for thinking we needed something better at a national level.”
The most up-to-date online maps were in Ontario and British Columbia. The BC Clinical Research Infrastructure Network partnered with the asset map project in offering its data. Director of Operations Heather Harris said the collaboration among provinces highlights Canada’s strengths as a whole rather than having separate agendas. “I think that it’s important to speak with that national voice.”
The asset map isn’t only for global marketing, said CCTAM Project Manager Elena Aminkova; it’s also for policy-makers. Users will soon be able to search by federal electoral boundaries as well.

Synergy Pharmaceuticals Initiates Second Phase 3 Clinical Trial of Plecanatide in Patients with IBS with Constipation

NEW YORK, June 23, 2015 — Synergy Pharmaceuticals Inc. (NASDAQ:SGYP) announced today the initiation of the second of two pivotal phase 3 clinical trialsevaluating the efficacy and safety of two different plecanatide treatment doses (3.0 mg and 6.0 mg), taken as a tablet once-a-day, in patients with irritable bowel syndrome with constipation (IBS-C).
The phase 3 IBS-C program includes two randomized, 12-week, double-blind, placebo-controlled pivotal trials conducted in the United States and each trial is expected to enroll approximately 1050 adult patients with IBS-C. Patients with IBS-C are defined by Rome III Criteria as having a history of constipation and abdominal pain for at least 6 months, including hard or lumpy stools for 25% or more of defecations, loose or watery stools for 25% or less of defecations, and abdominal pain or discomfort for 3 days or more per month for the last 3 months. The primary efficacy endpoint for both trials is the percentage of patients who are Overall Responders during the 12 week treatment period. An Overall Responder, as defined by the FDA, is a patient who is a weekly responder (i.e. meets both the abdominal pain intensity reduction and stool frequency increase criteria in the same week) for at least 6 of the 12 treatment weeks.
Synergy initiated the first phase 3 IBS-C trial in December 2014. The phase 3 IBS-C program was designed to support regulatory submission in the US.

About Plecanatide

Plecanatide is Synergy’s lead uroguanylin analogue in late-stage clinical development to treat patients with chronic idiopathic constipation (CIC) and IBS-C. Uroguanylin is a naturally occurring gastrointestinal (GI) peptide produced by humans in the small intestine and plays a key role in regulating normal GI activity. Orally administered plecanatide is designed to mimic uroguanylin’s natural activity and regulate the movement of fluid required for normal digestion. Synergy recently announced positive top-line data results from the first of two pivotal phase 3 trials evaluating the efficacy and safety of plecanatide treatment in patients with CIC. The company plans to release results from the second phase 3 CIC trial in the third quarter of this year.

About Synergy Pharmaceuticals Inc.

Synergy Pharmaceuticals (NASDAQ: SGYP) is a biopharmaceutical company focused on the development of novel therapies to treat GI diseases and disorders. Synergy’s proprietary platform of uroguanylin analogues includes two late-stage clinical assets, plecanatide and dolcanatide (SP-333). Dolcanatide has successfully completed a phase 2 study in patients with opioid-induced constipation and is presently being evaluated for the treatment of ulcerative colitis. For more information, please visit www.synergypharma.com.
Forward-Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward- looking words such as “anticipate,” “planned,” “believe,” “forecast,” “estimated,” “expected,” and “intend,” among others. These forward-looking statements are based on Synergy’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, substantial competition; our ability to continue as a going concern; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payer reimbursement; limited sales and marketing efforts and dependence upon third parties; and risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that future clinical trials discussed in this press release will be completed or successful or that any product will receive regulatory approval for any indication or prove to be commercially successful. Investors should read the risk factors set forth in Synergy’s Form 10-K for the year ended December 31, 2014 and other periodic reports filed with the Securities and Exchange Commission. While the list of factors presented here is considered representative, no such list should be considered to be a complete statement of all potential risks and uncertainties. Unlisted factors may present significant additional obstacles to the realization of forward-looking statements. Forward-looking statements included herein are made as of the date hereof, and Synergy does not undertake any obligation to update publicly such statements to reflect subsequent events or circumstances.


Source: Synergy Pharmaceuticals Inc.

Investing in hepatitis C drugs could save the economy billions, researchers suggest



by Catharine Paddock PhD
Medical News Today
Researchers who examined the effect of treating hepatitis C patients with the newer generation of oral drugs suggest while these medications may cost tens of thousands of dollars for a 12-week course, they could avert billions in lost productivity.
They conclude that the higher cure rate and reduced side effects of treating hepatitis C patients with an all-oral combination of ledipasvir and sofosbuvir (LDV/SOF) led to substantially less absenteeism and better work productivity that could save economies of the US and five European countries more than $3.2 billion a year.The study featured at Digestive Disease Week 2015, an international gathering being held May 17-19 in Washington DC of clinicians and researchers from gastroenterology, hepatology, endoscopy and gastrointestinal surgery.
Lead researcher Dr. Zobair Younossi, chairman of the department of medicine at Inova Fairfax Medical Campus, VA, says we have known for a long time about the devastating effect that chronic hepatitis C can have on patients’ health:
“But given the significant side effects previously associated with treating the disease, notably fatigue and neuropsychiatric side effects, we were interested in looking at the impact of new treatments on patients’ ability to work, and in a broader sense, how this affects employers and overall economies.”
For their study, Dr. Younossi and colleagues used data on over 1,900 chronic hepatitis C patients treated with LDV/SOF in a clinical trial. The drug combination – which interferes with enzymes that help the hepatitis C virus multiply – showed a cure rate of 94-99% with minimal side effects, they note.They contrast this with older, more traditional treatments, such as interferon and ribavirin that can have various side- effects, such as fatigue, flu-like symptoms, depression and lowered blood cell counts.The patients in their sample – including participants from the US and Europe – had filled in surveys that asked them questions specifically about work productivity, level of activity and health problems arising during the clinical trial.
CURING HEPATITIS C COULD SAVE US $2.67 BILLION A YEAR
The researchers gathered data not only on absenteeism but “presenteeism” where participants show up to work but are less productive.
They estimate that the reduced absenteeism and increased productivity resulting from curing hepatitis C with LDV/SOF could result in annual savings of $2.67 billion for the US and $556 million for five European countries: France, Germany, Italy, Spain and the UK.
Dr. Younossi says these are just preliminary results, and while they are encouraging, they need to be confirmed with further studies looking at the “real-world” effects on work productivity and associated economic gains outside the clinical trial setting.”Chronic hepatitis C is more than just a problem for the patient,” notes Dr. Younossi, “it has a ripple effect that impacts society at large.” He concludes:
“While previous reports have found the cost of these drugs as certainly significant, the long-term benefits of curing patients with hepatitis C makes this a worthwhile investment.”
The study was funded through a grant provided by Gilead Sciences, Inc, who market ledipasvir and sofosbuvir under the brand name Harvoni.The prices of Harvoni and other new hepatitis C drugs have been the subject of great controversy lately – with reports that the treatments are straining the budgets of both public and private payers.The debate recently took on a new twist when Sen. Bernie Sanders (I-Vt.) asked the US Department of Veterans Affairs to “invoke emergency powers to make expensive hepatitis C drugs available at affordable prices to treat tens of thousands of veterans now being denied the most effective care.”Meanwhile, Medical News Today recently learned that trials of another new course of antiviral therapy – based on a combination of daclatasvir, asunaprevir and beclabuvir – resulted in nearly all the patients with chronic hepatitis C infection becoming virus-free, including those with liver disease.

Hepatitis C treatments are ‘history in the making’ at a high cost !!


BillyBob McPherson lived on Ottawa’s streets as a young teenager before “running away with the carnival.” The 55-year-old doesn’t know exactly when during his colourful life he contracted hepatitis C — he thinks it might have been in Texas in the 1980s where he had surgery and blood transfusions while working as a carny.
But without treatment, he believes, the disease would have ended his life.Today, he is disease free, a living testament to the wonders of new drugs developed to cure the liver disease with few or no side effects. But he is also an example of the painful realities of the new  treatments.McPherson was cured because he took part in a clinical trial of the drug holkira pak, which was approved by Health Canada late last month. For 12 weeks he took nine pills a day, suffered no side-effects, and almost immediately began to feel better, he said.“These guys saved my life,” he says of the pharmaceutical company AbbVie, which developed the treatment and has held clinical trials in Ottawa, throughout Canada, and around the world.But without the benefit of the clinical trial, McPherson, who lives on a disability pension, would not have been able to afford the cure.
It is a reality Dr. Curtis Cooper, director of The Ottawa Hospital and Regional Hepatitis Program, who oversaw the AbbVie trials, deals with daily.Holkira pak, which had a 97-per-cent cure rate in clinical trials, including for patients with cirrhosis, is not yet on the market, and its price is not public. But other new drug regimes that treat and cure hepatitis C (genotype 1) without using interferon (which causes difficult side effects and is less effective), are priced well out of the range of most patients.For example, Gilead’s sofosbuvir drug, known as Sovaldi, costs $55,000 in Canada for a 12-week course of treatment, according to the company. Treatment costs to patients can be considerably higher, though, since often costly drugs are combined. One local patient with genotype 2 hepatitis C said he was told this week a combination of drugs, including sofosbuvir, taken over 16 weeks would cost $100,000.
The man is waiting to hear whether his insurance, or the drug company would cover part or all of the cost. If not, he said, he will pay for it himself.“It is literally a life-and-death situation for me. At the end of the day, it is more important to have the drug than to be worrying about the price.”Health Canada has approved three new drugs, sofosbuvir, simeprevir and harvoni since late 2013. Holkira pak was approved by Health Canada on Dec. 22, about the same time it received approval from the U.S. Federal Drug Administration.Dr. Jordan Feld, a hepatologist at the Francis Family Liver Clinic at Toronto Western Hospital, called the development of new therapies for hepatitis C “history in the making.“Hepatitis C is a devastating disease that causes more years of life lost than any infectious disease in the country. With the introduction of life-saving therapies that offer high cure rates, we can finally prevent complications of the disease and it actually raises the possibility that we even eliminate the disease from Canada altogether.”
But neither the Ontario drug plan nor many private plans cover the cost of the drugs, although simeprevir is covered under the province’s Exceptional Access Program, for some patients who meet defined criteria. AbbVie and other companies also provide drugs to some patients on a compassionate basis.Still, Cooper said he sees patients almost on a daily basis who can’t afford the cure.“It is very hard to explain to a patient that we have these great new treatment but I can’t put you on them because they are not paid for.”
Of 5,000 patients at the Ottawa Hospital clinic with hepatitis C, 100 of them are taking one of the new treatments. Some of those patients are covered by insurance that pays, or partly pays, for the treatment, said Cooper. Others receive treatment at no cost because of their circumstances and condition. Some patients, like McPherson, have benefitted from clinical trials of the drugs. A few have paid for the drugs out of their pockets.Government funding for the drugs might come too late to save the lives of some patients who could be saved now, said Cooper.
“If you have a patient who already has cirrhosis, it is really tough for them to understand they could die in the next year without getting this therapy that is Health Canada approved.”
Cooper believes Ontario, which he says has been reviewing some of the drugs for months, should immediately begin paying for treatment of patients with advanced liver disease.Hepatitis C patients have high rates of poverty, substance abuse and mental health issues — a demographic with little power. The virus, which affects 170 million people around the world, can scar the liver and lead to cirrhosis and liver cancer. Deaths are on the rise and baby boomers are considered most at risk. According to one report, two-thirds of the people with the virus are in that age group, and a large proportion is unaware of it.Cooper noted there are limited health budgets to pay for drugs, but he said the cost of paying health providers to care for hepatitis C patients year after year is also high, as is the cost of a liver transplant — between $200,00 or $300,000 — for those lucky enough to get one.McPherson knows he was lucky to get into the AbbVie drug trial. He was diagnosed with hepatitis C in the past few years but believes he had it for some time. His illness was at Stage 3, but McPherson says he was well on his way to Stage 4 and needing a liver transplant.Before being diagnosed, McPherson, who had long since quit drinking, said he felt like he had a “permanent hangover. I knew something was wrong.”That feeling went away soon after he began taking the drug regime in nine pills a day. Within two weeks of starting the trial, he said, his viral load was at zero.
“It is really remarkable.”

Redhill Biopharma successfully meets primary endpoint in Phase III study of RHB-105 for H. pylori infection


RedHill Biopharma Ltd., an Israeli biopharmaceutical company focused on late clinical-stage, proprietary, orally-administered, small molecule drugs for inflammatory and gastrointestinal diseases, including gastrointestinal cancers, today announced positive top-line results from its Phase III study with RHB-105 for the treatment of Helicobacter pylori (H. pylori) bacterial infection. 
Top-line results from the study demonstrated 89.4% efficacy in eradicating H. pylori infection with RHB-105.
The ERADICATE Hp first Phase III study successfully met its primary endpoint of superiority over historical standard of care efficacy levels of 70%, with high statistical significance (p <0.001). No serious adverse events, new or unexpected safety issues were noted in the study.The top-line results from the RHB-105 Phase III study, demonstrating achievement of primary endpoint, were provided to RedHill by an independent third party following an independent analysis and remain subject to completion of the independent review and analysis of the underlying data, including all safety, secondary and other outcome measures, and completion of the clinical study report (CSR), expected in the third quarter of 2015.
Prof. David Graham, M.D., M.A.C.G., of the Baylor College of Medicine, a key opinion leader in the field of gastric cancer and H. pylori infection and Principal Investigator of the ERADICATE Hp study, said: “The outstanding results of the RHB-105 Phase III study, which demonstrated a 89.4% cure rate of H. pylori, are consistent with the hypothesis that this may represent a promise for a new and improved treatment for H. pylori infection, and could significantly contribute to the prevention of gastric cancer, MALT lymphoma and other gastrointestinal diseases and conditions. Given the current high levels of antibiotic resistance and treatment failures with current standard of care therapies, RHB-105 could become, if approved, a best-in-class treatment, improving and potentially saving patients’ lives.”
Ira Kalfus, M.D., RedHill’s Medical Director, added: “On the basis of the clear success of the 
ERADICATE Hp study, and the Fast-Track designation of RHB-105, we look forward to meeting with FDA to discuss the clinical and regulatory path towards marketing approval in the U.S. No new or unexpected safety issues were identified. Efficacy and safety data from this study will be submitted for presentation at an upcoming medical meeting.” Gilead Raday, RedHill’s Senior VP Corporate and Product Development, said: “We are enthusiastic about the strong Phase III top-line results of RHB-105 and its potential benefit to patients. Coupled with the QIDP designation, patent protection and expanded indication, RHB-105 should be well-positioned, if approved, for commercial success as a first-line therapy for the treatment of H. pylori infection. We would like to thank the patients, investigators and service providers who participated in this study”.
The randomized, placebo-controlled, ERADICATE Hp Phase III study was designed to evaluate the safety and efficacy of RHB-105 as a first-line treatment for confirmed H. pylori infection. A total of 118 dyspepsia patients with confirmed H. pylori infection were enrolled and treated in the ERADICATE Hp study, which was conducted in 13 clinical sites in the U.S. Subjects were randomized in a 2:1 ratio to receive either RHB-105 or a placebo, for a period of 14 days, and assessed for the eradication of H. pylori infection 28 to 35 days after completion of treatment and for the protocol-defined primary endpoint of superiority over historical standard of care efficacy levels of 70%.
RHB-105 is a new and proprietary fixed-dose oral combination therapy of two antibiotics and a proton pump inhibitor (PPI) in an all-in-one oral capsule with a planned indication for the treatment of H. pylori infection – a major cause of chronic gastritis, peptic ulcer disease, gastric cancer and mucosa associated lymphoid tissue (MALT) lymphoma. Gastric cancer is the second most common cause of cancer deaths worldwide with approximately 1 million deaths per year, of which 95% are caused by H. pylori and may be preventable. Several countries have already started, or are planning, population-based H. pylori screening and treatment programs designed to eliminate gastric cancer.
With RHB-105, RedHill is pursuing an indication of first-line treatment of H. pylori infection, regardless of ulcer status, a significantly broader indication than current standard treatments for 
H. pylori, which are typically indicated only in patients with active or recent history of duodenal ulcer disease. If approved, RHB-105 may be the first H. pylori eradication therapy to target this broader indication, which would significantly expand the potential patient population for this drug candidate. The ERADICATE Hp Phase III study is planned to be followed by a second Phase III study and additional studies may be required, subject to FDA feedback.
RHB-105 was designated by the FDA as a Qualified Infectious Disease Product (QIDP) under the Generating Antibiotic Incentives Now (GAIN) Act, which is intended to incentivize the development of new antibiotic drugs for the treatment of serious or life-threatening infections. The designation allows RedHill to benefit from Fast-Track development status for RHB-105, providing for an expedited development pathway, as well as Priority Review status, potentially leading to a shorter review time by the FDA of a New Drug Application (NDA), if filed. If approved, RHB-105 will also receive an additional five years of U.S. market exclusivity in addition to the standard exclusivity period, for a total of 8 years of market exclusivity.
The 2015 global and U.S. market potential for H. pylori eradication therapies, at current branded prices, was recently estimated at approximately $4.83 billion and $1.45 billion, respectively, and could potentially grow with increasing awareness of the health risks associated with 
H. pylori infection and the benefits of its eradication.

Lyxumia (lixisenatide) demonstrated cardiovascular safety in people with type 2 diabetes and high CV risk


Sanofi has announced the presentation of full results of the Phase IIIb ELIXA study, which was designed to assess the cardiovascular (CV) safety of Lyxumia® (lixisenatide) in adults with type 2 diabetes and high CV risk. As previously reported, lixisenatide met the pre-specified criterion of non-inferiority versus placebo for the composite primary endpoint of CV death, non-fatal myocardial infarction, non-fatal stroke and hospitalization for unstable angina but did not demonstrate superiority. The full results will be included in the U.S. New Drug Application for lixisenatide, which is on track to be resubmitted to the U.S. Food and Drug Administration in Q3 2015.
Additional safety findings include no signal for increased risk of heart failure, pancreatitis, pancreatic cancer or severe symptomatic hypoglycemia. Lixisenatide was generally safe and well tolerated; nausea and vomiting, which are known side effects of the GLP-1 RA class, were observed more frequently with lixisenatide.
“The importance of determining the CV safety of diabetes medicines, as set out in the FDA guidance issued in 2008, is widely recognized. People around the world are being treated with GLP-1 receptor agonists, and the CV effects were unknown,” said Dr. Marc Pfeffer, Professor of Medicine at Harvard Medical School, Senior Physician in the Division of Cardiovascular Medicine at Brigham and Women’s Hospital and Chair of the ELIXA Steering Committee. “ELIXA goes beyond the FDA guidance to deliver data related to heart failure and other insights that are not currently available for any other GLP-1 receptor agonist. Our data provide the medical community, patients and caregivers with information that will better inform them about how lixisenatide can be safely used to better control their glucose.”
“As the first completed long-term CV safety study of a GLP-1 receptor agonist, the successful ELIXA trial will be shared with health authorities around the world and provides important outcomes data that can be considered by healthcare professionals,” said Pierre Chancel, Senior Vice President, Head of Global Diabetes at Sanofi. “Sanofi is committed to developing and delivering safe and effective treatment options for people with diabetes. This study supports that important work.”
Full study results were presented during a symposium at the American Diabetes Association 75th Scientific Sessions in Boston.
Results of Analysis
Lixisenatide met the pre-specified criterion of non-inferiority versus placebo for the composite primary endpoint of MACE+: CV death, non-fatal myocardial infarction, non-fatal stroke and hospitalization for unstable angina (Hazard Ratio [95% CI]: 1.017 [0.886 to 1.168]). Since the upper bound of the 95% CI was greater than 1.0, superiority over placebo in reducing the composite primary endpoint was not met.
The CV safety of lixisenatide was also confirmed by further analyses (e.g. MACE Hazard Ratio [95% CI]: 1.02 [0.887 to 1.172]). No signal for increased risk of heart failure (HF) was observed (Hazard Ratio [95% CI]: 0.96 [0.75 to 1.23]).
Measures of non-CV safety showed pancreatitis (0.2% with lixisenatide and 0.3% with placebo), pancreatic cancer (<0.1% with lixisenatide and 0.3% with placebo), severe symptomatic hypoglycemia (0.3 events per 100 patient-years with lixisenatide; 0.6 per 100 patient-years with placebo), malignancy (2.9% with lixisenatide and 2.6% with placebo), drug-related allergic reactions (0.2% with both lixisenatide and placebo).
About ELIXA
ELIXA (Evaluation of Cardiovascular Outcomes in Patients With Type 2 Diabetes After Acute Coronary Syndrome During Treatment With Lixisenatide) is the first event-driven cardiovascular outcomes study to provide data for a glucagon-like peptide-1 receptor agonist (GLP-1 RA). ELIXA was a randomized, double-blind, parallel group trial designed to evaluate cardiovascular risk, comparing lixisenatide to placebo in a high-risk population of adults with type 2 diabetes. More than 6,000 adults with type 2 diabetes and high CV risk (i.e., patients who have recently experienced a spontaneous acute coronary syndrome event) participated in the trial. The composite primary endpoint, which was evaluated for non-inferiority and superiority, comprised cardiovascular (CV) death, non-fatal myocardial infarction, non-fatal stroke, or hospitalization for unstable angina. The global ELIXA study started in June 2010 and was completed in 2015.
About Lixisenatide
Lixisenatide is a once-daily prandial glucagon-like peptide-1 receptor agonist (GLP-1 RA) for the treatment of adult patients with type 2 diabetes mellitus. GLP-1 is a naturally-occurring peptide hormone that is released within minutes after eating a meal. It is known to suppress glucagon secretion from pancreatic alpha cells and stimulate glucose-dependent insulin secretion by pancreatic beta cells.
Lixisenatide was in-licensed from Zealand Pharma A/S (NASDAQ OMX Copenhagen: ZEAL), www.zealandpharma.com, and was approved in Europe in 2013 for the treatment of adults with type 2 diabetes mellitus to achieve glycemic control in combination with oral glucose-lowering medicinal products and/or basal insulin when these, together with diet and exercise, do not provide adequate glycemic control. Lixisenatide is currently approved in over 50 countries worldwide for the treatment of adults with type 2 diabetes, with commercial launches in most EU countries, Japan, Brazil, Mexico and other markets. Lyxumia is the proprietary name approved by the European Medicines Agency and other health authorities for the GLP-1 RA lixisenatide. Lixisenatide is an investigational product in the U.S. It will be resubmitted to the Food & Drug Administration (FDA) in the third quarter of 2015. The proprietary name in the U.S. is under consideration.

Clinical trial using dorsaVi sensors demonstrates significant reduction in back pain


New data from a cluster randomised controlled trial has demonstrated that back pain patients monitored and treated using wearable movement sensors from dorsaVi Ltd (ASX:DVL) had a significant and sustained improvement in pain and functional ability.Across all primary outcome measures, participants treated with the dorsaVi sensors with biofeedback showed a 35 % to 47 % improvement at 12 months, which were all above the threshold for clinically important difference (>30 % of baseline scores).
The study results were published online in the leading medical journal BMC Musculoskeletal Disorders.It reports on the 12 month, multicentre, cluster randomised, placebo controlled clinical trial sponsored by dorsaVi and the Victorian Government. The trial involved 112 patients – 58 in the interventional group (which received ViMove) and 54 in the control group. The results, as published in the paper, are highly positive and validate that intervention by ViMove resulted in “significant and sustained improvements in pain and activity limitation that persisted after treatment finished.”
This is the first clinical trial of its kind to investigate the effect that technology can have on the rehabilitation of low back pain (LBP). The clinical trial investigated whether changing patterns of lumbo-pelvic movement and/or posture using motion sensor biofeedback, provided by ViMove, in people with LBP would lead to reduced pain and activity limitation when compared with guidelines- based medical treatment or physiotherapy.
LBP is highly prevalent and globally is the leading cause of disability, ahead of ischaemic heart disease, chronic obstructive pulmonary disease, and other musculoskeletal disorders, including osteoarthritis. In the UK, LBP is very common, affecting 80% of the population at some point in their lives1 and the cause of 8.3 million missed work days last year2. In the past, compared with placebo or no treatment, most non-surgical treatments for non-specific LBP showed only small to moderate effects with one treatment showing little superiority over the other. In addition, short term treatment effects typically reduce over the subsequent year.
dorsaVi CEO, Dr Andrew Ronchi, said: “Medical adoption by health practitioners is based on having protocols supported by clinical evidence and published in peer reviewed journals. We are delighted with the results of the trial and that it has now been published in a peer reviewed journal.”
dorsaVi’s ViMove is a wearable sensor system that turns human movement into actionable, easily interpreted data. The sensors can track a patient’s movement at 200 frames per second. The sensors provide real-time feedback about high risk positions for patients with back pain and also encourages positive movement patterns. This data informs decisions by the treating healthcare professional to modify their patients’ daily movements, thus reducing their risk of another episode and reducing recovery time. ViMove is approved for use in Australia and Europe and cleared by the FDA under 510(k) for the United States.
About the trial
The 12 month study enrolled 112 patients at eight sites including the Austin and Epworth Hospitals. All patients reported back pain, with a vast majority (85%) classified as chronic back pain patients. All patients wore ViMove sensors and had 6-8 office visits over 10 weeks. Follow-up appointments were held at 2, 3, 6 and 12 months with their clinicians. Clinicians in the Intervention arm got access to the ViMove sensor data to guide care and their patients received real time feedback in weekly monitoring sessions. Clinicians and patients in the control group were blind to the data and were given no feedback by the ViMove device.

Massachusetts General Hospital launches phase II trial of BCG vaccine to reverse type 1 diabetes



FDA approval of trial testing generic vaccine announced at ADA Scientific Sessions
A phase II clinical trial testing the ability of the generic vaccine bacillus Calmette-Guérin (BCG) to reverse advanced type 1 diabetes has received approval from the U.S. Food and Drug Administration (FDA). The approval of this trial, which will shortly begin enrolling qualified patients, was announced today at the 75th Scientific Sessions of the American Diabetes Association (ADA) by Denise Faustman, MD, PhD, director of the Massachusetts General Hospital (MGH) Immunobiology Laboratory and principal investigator of the study.
The five-year trial will investigate whether repeat BCG vaccination can clinically improve type 1 diabetes in adults between 18 and 60 years of age who have small but still detectable levels of insulin secretion from the pancreas. Faustman’s research team was the first group to document reversal of advanced type 1 diabetes in mice and subsequently completed a successful phase I human clinical trial of BCG vaccination. She announced the FDA approval to launch the phase II trial during her ADA presentation, “Low Levels of C-Peptide Have Clinical Significance for Established Type 1 Diabetes.”
“We have learned a lot since the early studies in mice – not just about how BCG works but also about its potential therapeutic benefits, similar to what are being seen in trials against other autoimmune diseases,” says Faustman. “We are so grateful to all of the donors, large and small, who have made this trial possible – especially the Iacocca Foundation, which has believed in us and has been a supporter since our early days. Our goal is to complete enrollment and also to raise the remaining funds needed for the trial by the end of this year.”
A generic drug with over 90 years of clinical use and safety data, BCG is currently approved by the FDA for vaccination against tuberculosis and for the treatment of bladder cancer. The vaccine is known to elevate levels of the immune modulator tumor necrosis factor (TNF), which Faustman’s team previously showed can temporarily eliminate in both humans and mice the abnormal white blood cells responsible for autoimmune type 1 diabetes. Increased TNF levels also stimulated production of protective regulatory T cells.
In the phase I clinical trial, which was published in the August 8, 2012, issue of PLOS Medicine, two injections of BCG spaced four weeks apart led to temporary elimination of diabetes-causing T cells and provided evidence of a small, transient return of insulin secretion. The phase II clinical study will include more frequent dosing over a longer time period to determine the potential of repeat BCG vaccination to ameliorate the autoimmune state and improve clinical parameters such as HbA1c, a marker of average blood sugar control.
In the new trial, which will be double blinded and conducted at MGH, 150 adults with long-term type 1 diabetes will be randomized to receive two injections four weeks apart of either BCG or placebo and then a single injection annually for the next four years. Patients will be closely monitored over the five-year trial period. The primary outcome measure will be improved results on the HbA1c blood test, which have been shown to prevent complications.
“In the phase I clinical trial we demonstrated a statistically significant response to BCG, but our goal in phase II is to create a lasting therapeutic response,” says Faustman, an associate professor of Medicine at Harvard Medical School. “We will be working again with people who have had type 1 diabetes for many years. This is not a prevention trial; instead, we are trying to create a regimen that will treat even advanced disease. In addition to our phase I trial, we took guidance from the BCG clinical trials that are underway globally for other autoimmune diseases such as multiple sclerosis.”
Lee Iacocca, founder of the Iacocca Foundation, says, “My family and I have been fortunate to be part of this research for many years. It is incredibly exciting to be talking about curing people, not mice. I made a promise to my late wife to find a cure for type 1 diabetes. Now my family and I look forward to the continued progress and are proud to support this effort to get closer to that goal.” The Iacocca Foundation provided major funding for the phase I trial and has taken a leadership role in funding the phase II trial.
More than $19 million has been raised out of a total of $25 million needed to conduct the phase II study over the next five years. Additional information about the clinical trial, including information for potential participants and financial supporters, is available athttp://www.faustmanlab.org/ or by emailing DiabetesTrial@partners.org. Details on the trial and enrollment also are available at: https://clinicaltrials.gov/ct2/show/NCT02081326.

NightstaRx and The University of Alberta announce the start of the first Canadian gene therapy study to treat Choroideremia



NightstaRx Ltd (“Nightstar”), the biopharmaceutical company specialising in bringing therapies for retinal dystrophies to patients, announces that the University of Alberta, has begun enrolling and dosing subjects in a Phase II clinical trial of the Company’s gene therapy for the treatment of choroideremia (CHM). This gene therapy approach uses a viral vector known as adeno-associated virus (AAV) to deliver a wild-type copy of the Rab-escort protein 1 (REP-1) gene (AAV2-REP1) into cells of the eye.
The trial, which is sponsored by the University of Alberta, is an open label study involving a total of 6 male patients, who will each receive a single dose of AAV2-REP1 via a sub retinal injection.
Choroideremia is an inherited X-linked recessive disease which inevitably causes blindness. It is caused by mutations to the CHM gene which encodes Rab-escort protein 1 and affects approximately 1 in 50,000 people. The first symptom of the condition is usually an impairment of night vision which often occurs in early childhood. This is followed by progressive narrowing of the field of vision, as well as a decrease in the ability to see details, culminating in blindness, most commonly in late adulthood. No effective treatment currently exists.
David Fellows, CEO of Nightstar said:
“Gene therapy treats genetic diseases at the molecular level by correcting what is wrong with defective genes. We are broadening our pipeline of products in development and are leading the way in the development of an effective gene therapy treatment for choroideremia. This new study, sponsored by the University of Alberta, is another step forward in the development of AAV2-REP1. We have been granted Orphan Drug Designation for the product in the United States and Europe and the data to date has shown very promising results.”
Ian MacDonald, Professor of Ophthalmology and Visual Sciences, University of Alberta commented:
“We are very excited to be working with Nightstar and to have treated our first choroideremia patient. Choroideremia is a devastating condition for individuals and families, but we believe our new gene therapy will arrest any further deterioration of vision and will provide long lasting benefit. The next challenge will be making this therapy available to all individuals with the condition as soon as we possibly can.”
About Choroideremia
Choroideremia is an inherited disorder that leads to progressive loss of vision due to degeneration of the choroid and retina which is caused by a lack of Rab Escort Protein-1 (REP-1) and occurs almost exclusively in males. The first symptoms occur in childhood, with night blindness being the most common first symptom. As the disease progresses, there is loss of peripheral vision or ‘tunnel vision’, and later a loss of central vision. Progression of the disease continues throughout the individual’s life, although both the rate and the degree of visual loss can vary, even within the same family. There is currently no treatment or cure for this disease.


More than 95% of the global population has at least one health problem, study finds


The largest and most in-depth study of global health trends to date finds that more than 95% of the world’s population has health problems, with more than a third of us experiencing five or more conditions.

The results of the Global Burden of Disease Study 2013 (GBD 2013) were recently published inThe Lancet.Lead study author Prof. Theo Vos, of the Institute of Health Metrics and Evaluation at the University of Washington in Seattle, and colleagues set out to calculate up-to-date estimates of disease and injury incidence and prevalence among 188 countries between 1990 and 2013, using data from more than 35,000 sources.
The team also sought to estimate the number of disability-adjusted life years (DALYS) – that is, the number of healthy years lost due to illness – over the 23-year period.In total, the researchers were able to provide global estimates of incidence and prevalence for 301 acute and chronic diseases, and they were able to assess the effects of 2,337 health consequences that result from at least one of these disorders.
Prof. Vos and colleagues found that in 2013, only 1 in 20 people (4.3%) around the globe had no health problems, meaning more than 95% of us had one or more illnesses.
The researchers found that 2.3 billion people worldwide – more than a third of us – had at least five health conditions in 2013. Over the 23-year study period, the number of people with 10 or more health conditions rose by 52%.
The number of people with multiple illnesses increased with age. In 2013, 36% of children in developed countries aged 0-4 years had no illnesses, compared with only 0.03% of adults aged 80 and older.
The number of healthy years lost due to illness increased from 21% in 1990 to 31% in 2013, and the number of years lived with disability (YLD) rose from 537.6 million in 1990 to 764.8 million in 2013.
The researchers attribute the increase in YLD over the 23-year period to population growth and aging. They found that the main drivers for YLD were musculoskeletal disorders, mental illnesses, substance abuse disorders, neurological conditions and chronic respiratory disorders.

Disability rates not declining as fast as death rates

The leading causes of health loss did not change much between 1990 and 2013, according to the researchers.
They found that in 2013, musculoskeletal disorders, such as low back pain and arthritis, and mental and substance use disorders – particularly anxiety, depression and drug and alcohol use disorders – accounted for almost 50% of all health loss globally.
Low back pain and major depression were ranked as two of the top 10 disability contributors in 2013 among every country. These conditions caused more health loss than chronic obstructive pulmonary disease (COPD), diabetes and asthma combined, according to the results.
The team found the leading causes of disability and health loss varied by regions. Falls, for example, were found to be the second leading cause of disability in 11 of 13 countries in central Europe, while anxiety disorders were more prominent causes of disability in Caribbean regions.
Past war conflict was identified as the main contributor to health loss in Cambodia, Nicaragua, Rwanda, while ranking as the second leading cause of health loss in Vietnam.
Perhaps one of the most important findings was that rates of disability as a result of health problems are not declining as rapidly as death rates from such conditions. The team points to diabetes as an example; while diabetes rates rose by 43% between 1990 and 2013, death rates from the condition only increased by 9%.
“The fact that mortality is declining faster than non-fatal disease and injury prevalence is further evidence of the importance of paying attention to the rising health loss from these leading causes of disability, and not simply focusing on reducing mortality,” says Prof. Vos, adding:
“Large, preventable causes of health loss, particularly serious musculoskeletal disorders and mental and behavioral disorders, have not received the attention that they deserve. Addressing these issues will require a shift in health priorities around the world, not just to keep people alive into old age, but also to keep them healthy.”
The Bill and Melinda Gates Foundation funded the study.

McMaster researchers test fecal transplantation to treat ulcerative colitis



Two new studies led by researchers from the Farncombe Family Digestive Health Research Institute at McMaster University show that transplantation of fecal matter may be a useful tool in the fight against ulcerative colitis (UC).
Ulcerative colitis is a chronic, debilitating inflammatory bowel condition characterized by symptoms including bloody stools, diarrhea, abdominal pain, weight loss and malnutrition. It results from the development of abnormal immune responses to the normal bacteria in the digestive tract. It is difficult to treat and standard therapy doesn’t always work.
There is currently great interest in treating UC with fecal microbiota transplantation (FMT), which involves transplanting gut fecal bacteria from healthy people into patients with UC.
A study recently published in Inflammatory Bowel Diseases found that UC can be controlled by the type of bacteria that inhabits the gut. The study was led by Elena Verdu, an associate professor of medicine with the Michael G. DeGroote School of Medicine.
“Our animal research provides insight that selected bacterial groups, involved in gut health, are important for protecting the colon against injury and inflammation,” said Verdu.
Along the same theme, in research published on June 29, 2015 in Gastroenterology, professor of medicine Paul Moayyedi and his team explored the safety and efficacy of FMT by conducting a placebo-controlled, randomized trial. They found that “FMT induces remission in a significantly greater percentage of patients with active UC than placebo,” the authors wrote.
“Our study in patients with ulcerative colitis is the first randomized trial of fecal microbiota transplantation in adults with ulcerative colitis and shows that this therapy may work,” said Moayyedi. “The effect of fecal transplant seems to be dependent on the sort of bacteria that is in the donor stool, which fits with the observations of Dr. Verdu’s animal study.”
In Verdu’s study, mice were given gut bacteria from patients with severe UC and the effects were compared to those produced in mice that were given bacteria from a healthy person. The results identified a reduced amount of the bacterial families that are important for gut health in the feces of patients with severe colitis.
Second, they found that when mice were given these bacteria and then exposed to a toxin that causes gut injury, the resulting inflammation was higher in the mice with UC bacteria than in mice with bacteria from the healthy person, in whom the beneficial bacterial groups were abundant.
“The study also showed that the same protective effect could be achieved using the fecal material from the healthy person as with specific groups of bacteria that were isolated from the ‘healthy’ fecal matter,” said Verdu. “This suggests that specific combinations of beneficial bacteria extracted from healthy people could be tested in future clinical fecal transplantation studies, and could potentially replace fecal matter.”
Verdu said the implications of her study relate to the selection of healthy donors for fecal transplantation.
“In addition to screening for infections and disease, donors that harbour an abundance of the beneficial bacterial groups identified in our study could be selected to increase the chances of success of transplantation,” said Verdu.
Moayyedi and his team, including McMaster professors Michael Surette and Christine Lee, recruited 75 patients with a flare up of their UC and randomized them to fecal transplant therapy given as an enema derived from stool donated by an anonymous healthy donor once per week for six weeks, or a placebo consisting of a water enema. They found 24 per cent were in remission in the fecal transplant group compared to five per cent in the placebo group. There were two main healthy donors, donor A and donor B – one of which was the healthy donor from Verdu’s mouse study – and benefit seemed to be mostly related to those that received stool from donor B. The effect was also greater in those that had recently been diagnosed with UC.
“Many questions remain, but this provides interesting data suggesting that altering the gut microbial flora may be promising for treating ulcerative colitis,” the authors noted.
Moayyedi added that the data suggests more research is needed using the FMT approach.

FDA strengthens heart safety warnings on painkillers


People should think carefully about taking these drugs, both over-the-counter versions and prescription pills, the FDA says. It’s asking manufacturers to change the labels.”They used to say they might cause risk of heart attack or stroke. Now we are saying they do cause increased risk of heart attack and stroke,The warning covers drugs called nonsteroidal anti-inflammatory drugs or NSAIDS for short. They include ibuprofen, sold under brand names like Advil or Motrin; naproxen (Aleve), as well as prescription arthritis drugs known as COX-2 inhibitors, such as Celebrex. Tylenol, known generically as acetaminophen, is not an NSAID.Cough and cold remedies can also contain NSAIDs as an ingredient.”Because many prescription and OTC medicines contain NSAIDs, consumers should avoid taking multiple remedies with the same active ingredient,” the FDA said.
“FDA is strengthening an existing warning in prescription drug labels and over-the-counter (OTC) Drug Facts labels to indicate that nonsteroidal anti-inflammatory drugs (NSAIDs) can increase the chance of a heart attack or stroke, either of which can lead to death,”
“Those serious side effects can occur as early as the first few weeks of using an NSAID, and the risk might rise the longer people take NSAIDs. (Although aspirin is also an NSAID, this revised warning doesn’t apply to aspirin.)”
Last year, FDA said it was reviewing the safety of these drugs.
Some of the studies they looked at showed a clear pattern: people who took NSAIDS were more likely to have heart attacks or strokes.
“There is no period of use shown to be without risk,” said Dr. Judy Racoosin, deputy director of FDA’s Division of Anesthesia, Analgesia and Addiction Products.”In the coming months, the FDA will request that manufacturers update the existing cardiovascular risk information in Drug Facts labels for over-the-counter (OTC) non-aspirin NSAIDs. Consumers and health care professionals should remain alert for the development of heart- and stroke-related symptoms throughout the time a consumer takes any NSAID,” FDA said.This doesn’t mean people should just stop taking NSAIDS, FDA said.”Take the lowest effective dose for the shortest amount of time possible,” said FDA’s Dr. Karen Mahoney.The American Heart Association advises people to try acetaminophen (Tylenol) first.
“If you have heart disease or high blood pressure, consult a health care provider before using an NSAID,” FDA added.
“Balance the benefits of NSAIDs with the possible risks and weigh your options. If you take low-dose aspirin for protection against heart attack and stroke, you should know that some NSAIDs, including ibuprofen and naproxen, can interfere with that protective effect.”
And the agency gives advice on what symptoms to look for.
“Stop taking NSAIDs and seek medical help if you experience symptoms that might signal heart problems or stroke, such as chest pain, trouble breathing, sudden weakness in one part or side of the body, or sudden slurred speech.”
In 2013 Americans bought more than 275 million boxes of over-the-counter NSAIDs, racking up $1.7 billion in sales, according to retail tracker IRI.

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