Category Archives: Cardiology

Urine exRNA may be source of biomarkers for muscular dystrophy

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Massachusetts General Hospital (MGH) researchers have found that extracellular RNA (exRNA) in urine may be a source of biomarkers for the two most common forms of muscular dystrophy, noninvasively providing information about whether therapeutic drugs are having the desired effects on a molecular level.

The report published in online journal Nature Communications, is the first to show that urine exRNA can be used to monitor systemic diseases that do not directly affect the urinary tract.

“Our findings could facilitate drug development by offering a convenient, painless and relatively low-cost assay that may reduce and perhaps eventually eliminate the need for multiple invasive muscle biopsies to track disease activity and therapeutic response,” says Thurman Wheeler, MD, MGH Department of Neurology, senior author of the report. “Urine exRNA monitoring could determine whether a drug is reaching its target long before clinical effects on muscle function could be detected and may enable early identification of whether dosage adjustments may be required, something that would be impossible with invasive muscle biopsies.”

There are several types of muscular dystrophy, all of which lead to progressive muscle weakness and loss of muscle mass. Duchenne muscular dystrophy (DMD) is the most common form, with symptoms that usually begin in children under the age of 5. Myotonic muscular dystrophy (DM) is the most common adult-onset form and has two subtypes – DM1 and DM2. Each form is caused by a different gene mutation. In DMD, the mutation affects the gene for dystrophin, a protein essential to the strength of muscle fibers. DM-associated mutations – in the DMPK gene for DM1 and in the CNBP gene for DM2 – involve excessive repeats of nucleotides, leading to abnormal processing of RNA molecules.

The mutation associated with DM1 affects RNA splicing, the process that removes non-coding segments from an RNA molecule. A single gene can normally give rise to several different proteins, with the differences being determined by alternative RNA splicing patterns. The DM1 mutation interferes with appropriate splicing of RNAs encoding several other proteins, and analysis of RNA splice variants in muscle biopsies has been used to determine disease severity in patients. In animal models, splice variants in muscle tissue have been used to indicate whether potential therapies are reaching their molecular target. A less invasive way of assessing disease severity and therapeutic response could expand the number of patients who could receive therapeutic drugs or participate in clinical trials. For example, a recent clinical trial for a DM1 drug was restricted to adult patients partially because of the need for repeat muscle biopsies.

Carried through bodily fluids like blood and urine in membrane bubbles called vesicles, exRNA encompasses messenger, non-coding, and microRNA molecules and can reflect mutations, deletions and other molecular variants. A few muscular-dystrophy-associated RNA or protein biomarkers have been identified in the blood of patients. Even though it seemed unlikely that exRNA from the skeletal and cardiac muscle tissues affected by DM1 could pass through the kidney’s filtration system into the urine, urine is such an easily accessible fluid that Wheeler’s team analyzed vesicles from both blood and urine for exRNAs that could reflect results of the DM1 mutation.

Their experiments comparing urine exRNAs from DM1 patients, patients with two other forms of muscular dystrophy and unaffected control volunteers identified 10 transcripts that are alternatively spliced in a pattern unique to DM1 patients, most of which had been previously found in patient muscle biopsies. A composite biomarker incorporating these 10 transcripts was 100 percent accurate in distinguishing DM1 patients from unaffected controls in a different group of participants. Samples taken from untreated DM1 patients over several months indicated consistency of splicing patterns within an individual, suggesting that repeat sampling could accurately reflect disease state and treatment response.

Along with developing a more precise assay for rapid measurement of alternative RNA splicing in urine and other bodily fluids, the team showed that splicing patterns in total RNA from the cells in the urine were different from and less useful as biomarkers than those from exRNAs and found that exRNAs in blood could not distinguish between DM1 patients and controls. They also found that the splicing patterns of some urine exRNA transcripts reflected the severity of DM1 symptoms, and that a small group of asymptomatic patients with the DM1 mutation had urine exRNA splicing patterns midway between those of symptomatic patients and unaffected controls.

A group of drugs being evaluated for the treatment of DMD manipulate splicing of the dystrophin gene in order to remove a specific exon – a protein-coding segment of RNA – producing a shortened but partially functional version of the dystrophin protein. The MGH team showed that urine exRNAs from six untreated DMD patients accurately reflected the specific gene mutation in each patient. In two DMD patients being treated with eteplirsen – an FDA-approved DMD drug that induces skipping of the target exon – analysis of urine exRNA was able to confirm the drug was reaching its molecular target, the first such confirmation not provided by muscle biopsy.

“Our demonstration of disease-specific splice variants in urine exRNA suggests the value of biofluids as a means of identifying novel splice variants that may help correlate gene variants with symptoms for several diseases for which noninvasive biomarkers are unavailable,” says Wheeler, an assistant professor of Neurology at Harvard Medical School. “These findings support studies of exRNA from urine, blood or cerebrospinal fluid as biomarker replacements for tissue biopsies in other conditions with altered RNA splicing – including other types of muscular dystrophy, spinal muscular atrophy and amyotrophic lateral sclerosis.”

SOURCE: www.news-medical.net/news

AZ, Amgen’s first-in-class asthma drug gets breakthrough status

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AstraZeneca and partner Amgen have picked up a breakthrough designation from the FDA for tezepelumab, a drug that AZ claims could be a “best in disease” therapy for severe asthma.

Tezepelumab is a thymic stromal lymphopoietin (TSLP) targeting antibody that would slot into AZ’s key respiratory portfolio alongside Fasenra(benralizumab), the company’s interleukin-5 inhibitor antibody which was approved for severe asthma in Europe in January but just failed a test in chronic obstructive pulmonary disease (COPD).

The new candidate is in a phase III programme called PATHFINDER – due to report results from 2020 – and according to AZ chief executive Pascal Soriot has shown remarkable activity in mid-stage testing, reducing several key asthma biomarkers including blood eosinophils, fractional exhaled nitric oxide (FENO) and immunoglobulin levels as well as cutting asthma attacks.

Drugs like Fasenra and GlaxoSmithKline’s rival IL-5 inhibitor Nucala(mepolizumab) have emerged as an important treatment option for people with severe asthma characterised by high levels of eosinophils. However, the FDA’s BTD for tezepelumab is for patients “without an eosinophilic phenotype, who are receiving inhaled corticosteroids/long-acting beta2-agonists with or without oral corticosteroids and additional asthma controllers,” says AZ.

Because it acts further upstream in the inflammatory cascade responsible for asthma, tezepelumab could be suitable for a broader range of patients than Fasenra and Nucala, and also potentially Sanofi and Regeneron’s new candidate Dupixent (dupilumab), an IL-4 and IL-13 inhibitor that is already approved for atopic dermatitis. Dupixent was filed for asthma in the US in March and is due for an FDA verdict by 20 October, but some analysts have said they also expect Dupixent to be used mainly in patients with eosinophilic asthma.

Tezepelumab new status comes on the back of the phase IIb PATHWAY study which showed a significant reduction in the annual asthma exacerbation rate compared with placebo in a broad population of severe asthma patients irrespective of patients’ characteristics at enrolment. Around 10% of all asthma patients are thought to have severe symptoms making them eligible for antibody therapies.

“Tezepelumab is exciting because it has the potential to treat a broad population of severe asthma patients, including those ineligible for currently-approved biologic therapies,” said Sean Bohen, AZ’s chief medical officer.  The BTD “will help us bring tezepelumab to patients as quickly as possible,” he added.

Biologic drugs for asthma are predicted to make several billions of dollars in sales at peak, and there are already signs of string growth for some new products. Nucala made £245m ($317m) in the first six months of the year, with later entrant Fasenra bringing in $86m. Analysts have suggested tezepelumab could be a blockbuster in its own right.

SOURCE: www.pmlive.com/pharma_news

Can pharma halt the world’s obesity crisis?

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Major research published in the Lancet this week comes as no surprise, but the findings are still sobering: across the world there are too many people who are not doing enough exercise, putting themselves at risk of diseases such as obesity and type 2 diabetes.

The research published in Lancet Global Health showed that more than a quarter (1.4 billion) adults are at risk from not doing enough physical activity – these diseases are hugely costly to society and to individuals affected.

The levels of insufficient physical activity varied widely across income groups – 16% in low-income countries, compared with 37% in high-income countries.

And in 55 (33%) of 168 countries, more than a third of the population was insufficiently active according to the figures collated in 2016.

In four countries more than half of adults were insufficiently active – Kuwait (67%), American Samoa (53%), Saudi Arabia (53%) and Iraq (52%).

But the regions with the highest increase in insufficient activity over time were high-income Western countries (from 31% in 2001 to 37% in 2016), and Latin America and the Caribbean (33% to 39%).

Countries from these regions driving this trend include Germany, New Zealand, the USA, Argentina, and Brazil.

Authors also identified several socioeconomic forces at work behind the problem – including urbanisation, sedentary occupations, and motorised transport in the richer countries where lack of exercise is most prevalent.

This research will be of interest to the pharma companies that are attempting to tackle diabetes and obesity related diseases, not just with medications but by working with governments to try and influence policy to reduce incidence of the disease.

Leaders in the field such as Novo Nordisk and AstraZeneca are actively campaigning to try and encourage governments to think about how they can encourage people to become more active, and reducing the levels of obesity in society.

With networks of experts in diabetes in countries across the world big pharma companies have realised that there is a huge opportunity to reach out to health systems using corporate social responsibility programmes that aim to tackle the issues outlined in the Lancet research.

For instance Novo has created an initiative entitled “Cities Changing Diabetes” that specifically aims to tackle the problem of “urban diabetes”.

The project involves collecting qualitative and quantitative evidence that could lead to better understanding of the problem and the contributing factors.

It has built up a network of partners across the world, including city leads, city administrations, academia, diabetes associations, health insurances, community centres and business corporations.

So far it has built relations with 16 cities across the world, representing 100 million citizens, including Beirut, Copenhagen, Leicester and Shanghai.

The project is driven by the recognition that the problem with diabetes is only going to get worse unless immediate action is taken.

According to modelling from Novo Nordisk, in order to hold the rise in prevalence at 10%, the world must set itself a target of reducing obesity by 25% by 2045.

Novo organised a Cities Changing Diabetes Summit last year, where it made the call for joint working across sectors and disciplines in order to unite them behind the cause.

Novo has launched an Urban Diabetes Toolbox that gives policy makers tools on how to tackle the problem, including diabetes vulnerability assessment tools, and tips about how to promote healthy living.

AstraZeneca has also been active in this regard, taking part in the multi-year Action in Diabetes initiative and participating in the Global Diabetes Policy Forum in Rome last October.

Now in its third year, the event brought together more than 100 leading global experts in type 2 diabetes care to discuss best practice in policy-making.

Inspired and funded by AstraZeneca, the initiative operates in partnership with the Internatioinal Diabetes Federation, the World Heart Federation, and Primary Care Diabetes Europe, among other organisations.

AstraZeneca’s work aims to demonstrate the interconnectivity between metabolic, cardiovascular, and renal diseases and foster policies that deal with these diseases in an holistic manner.

Eli Lilly is also known for its work in diabetes, and has launched its non-communicable disease partnership with a similar aim.

It has three aims  – piloting new approaches to strengthen diabetes care, advocating to governments for better disease management, and increasing appropriate use of and compliance with medicines to improve outcomes.

The scale of the problem is daunting, but pharma’s focus on raising awareness about the issue, and bringing different stakeholders together towards the common goal of reducing obesity is an example of how industry can help to tackle one of the major social problems of our times.

SOURCE: www.pharmaphorum.com/views-and-analysis

Low dose Aspirin daily – benefits and risks measured in two studies

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Low dose Aspirin has been advocated to reduce the risk of a second heart attack or a stroke or other heart problems in persons who have had one episode.

A major new study published in the Lancet, has found that taking Aspirin to prevent the first heart attack fails to work. In fact the risks of taking low dose Aspirin to prevent the first heart attack outweigh the benefits.

There have been numerous studies on the prophylactic and protective effect of this low cost medication. While certain studies have shown that it can prevent first strokes or heart attacks in persons who are at a moderate risk of heart attacks and strokes, some studies have not shown significant benefit.

Aspirin has also been tested in people with cardiac disease risk factors such as diabetes, high blood pressure, smoking, high cholesterol etc. In diabetes for example Aspirin intake on a regular basis may offer some protection against heart attacks or strokes but the risk of serious bleeding as a side effect of the drug remains high. Aspirin was also purported to have anti-cancer or cancer preventing properties. This has also been negated in studies. In the studies the researchers have used 100 milligrams per day.

Study leader Dr. Jane Armitage of the University of Oxford in England said, “There’s been a lot of uncertainty among doctors around the world about prescribing aspirin. If you’re healthy, it’s probably not worth taking it.” The study was presented this Sunday (26th August 2018) at the European Society of Cardiology meeting in Munich.

In this latest study, the team of researchers from Boston used aspirin or placebo pills on 12,546 participants. These participants had a moderate risk of suffering a heart attack or stroke and other health problems. The participants were followed up for five years and it was seen that 4 percent persons in each group had a heart problem. These participants took medications for lowering blood pressure and cholesterol explained Dr. J. Michael Gaziano of Brigham and Women’s Hospital. Side effects such as internal bleeding or stomach bleeding, was mild but seen twice as much with Aspirin than the placebo pills. Aspirin users also complained more of reflux, abdominal pain, indigestion etc. This new study was sponsored by Bayer.

Low dose aspirin/fish oil supplements in diabetics

In yet another study, researchers from Oxford, randomly assigned 15,480 adults with Type 1 or 2 diabetes with aspirin or 1 gram of fish oil, both the aspirin and fish oil or placebo pills. The pills were administered each day.

The participants were followed for seven and a half years. Heart problems were lower among aspirin users. The risk of bleeding however was raised with aspirin. Fish oil supplements did not help though said study leader Dr. Louise Bowman of the University of Oxford. This study was published by the New England Journal of Medicine.

The British Heart Foundation sponsored the study. Bayer and Mylan provided aspirin and the fish oil supplements, respectively.

SOURCE: www.news-medical.net/news

AZ’s potassium drug Lokelma finally approved in US

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AstraZeneca badly needs new drugs on the market as several former blockbusters have been hit by generic competition – and finally its high potassium treatment Lokelma has been approved by US regulators.

The drug, a highly selective potassium-removing agent, has been approved at the third time of asking by the FDA, which had been concerned about issues at its manufacturing plant in Texas.

European regulators approved the drug formerly known as ZS-9 in March after their concerns over the issues were resolved, and after two previous rejections the US regulator is also satisfied with the technical arrangements at the facility.

AZ gained rights to the drug after buying ZS Pharma in 2015 for $2.7 billion and is designed to treat hyperkalaemia, where high potassium levels threaten kidney and heart function.

Lokelma (sodium zirconium cyclosilicate) will compete with Vifor Pharma group member Relypsa’s rival Veltassa (patiromer), which has been on the market for a few years in the US and Europe.

The Anglo-Swedish pharma has predicted sales in excess of $1 billion annually for ZS-9, although some analysts say this is a conservative estimate.

The risk of hyperkalaemia increases significantly for patients with chronic kidney disease (CKD) and for those who take common medications for heart failure (HF), such as renin-angiotensin-aldosterone system (RAAS) inhibitors, which can increase potassium in the blood.

To help prevent the recurrence of hyperkalaemia, RAAS-inhibitor therapy is often modified or discontinued, which can compromise cardio-renal outcomes and increase the risk of death.

Sean Bohen, chief medical officer at AstraZeneca, said: “The consequences of hyperkalaemia can be very serious and it’s reassuring for treating physicians that Lokelma has demonstrated lowering of potassium levels in patients with chronic kidney disease, heart failure, diabetes and those taking RAAS inhibitors.”

AZ badly needs the new sales – sales of its Crestor (rosuvastatin), a former blockbuster were down 38% in Q1, to $338 million, and overall revenues fell 4% to just under $5.2 billion.

The company is selling off its old and unwanted drugs to prop up revenues and reduce costs – but this can only be seen as a short-term measure before new revenues come on stream.

CEO Pascal Soriot also faces a shareholder revolt, after more than 37% of shareholders voted against or abstained at the firm’s annual meeting when asked to approve a £9.4m pay package for Soriot, down from £14.3 million last year.

Soriot has set a sales target of above $40 billion by 2023, despite the struggles getting new drugs to the market.

SOURCE: www.pharmaphorum.com/news

New indication for evolocumab in established atherosclerotic cardiovascular disease

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Amgen has announced announced that the European Commission (EC) has approved a new indication in the Repatha® (evolocumab) label for adults with established atherosclerotic cardiovascular disease (myocardial infarction, stroke or peripheral arterial disease) to reduce cardiovascular risk by lowering low-density lipoprotein cholesterol (LDL-C) levels.

With the expanded label now in place, Amgen is working with payers in Europe to remove prescribing barriers and expand access in order to reach patients with established cardiovascular disease who are at risk of another event.

“With its proven ability to prevent heart attacks and strokes, Repatha offers hope for one of the greatest health challenges we face today. However, the majority of patients in Europe who could benefit from treatment with a PCSK9 inhibitor remain unserved and at risk of a cardiovascular event,” said Anthony C. Hooper, executive vice president of Global Commercial Operations at Amgen. “To help ensure eligible patients around the world can access and benefit from Repatha, Amgen is willing to work in partnership with payers to help manage affordability concerns from increased patient access. Furthermore, we are committed to excellence in LDL-C management and collaborating with healthcare providers to deliver comprehensive solutions for patients.”

Of all the modifiable risk factors for heart attack and stroke, lowering high LDL-C is one of the most important and impactful.1,2 Yet, even among patients currently taking a lipid-lowering therapy, many patients still have high LDL-C levels and remain at risk for cardiovascular events. Repatha is a groundbreaking medicine proven to significantly lower “bad cholesterol” or LDL-C for high-risk patients who suffer from a combination of high LDL-C and cardiovascular disease, and who continue to struggle with lowering their LDL-C levels despite statin therapy.

“We know that patients with a previous history of cardiovascular events are at an increased risk of subsequent events, especially in the first year,”3-5 said Sean E. Harper, MD, executive vice president of Research and Development at Amgen. “With far too many patients at risk of recurrent cardiovascular events, we are pleased that the European Commission has approved Repatha to prevent heart attacks and strokes in adults with established atherosclerotic cardiovascular disease. The science clearly indicates that ‘lower LDL-C is better’ and this approval underscores the role for Repatha among high-risk patients for whom statins alone are not enough.”

The approval by the EC recognises the positive findings from the Repatha cardiovascular outcomes study (FOURIER), expanding the label to include data on the additional reduction and prevention of heart attacks, strokes and coronary revascularisations on top of maximally tolerated statin therapy. FOURIER showed reductions in the risk of heart attack by 27%, the risk of stroke by 21% and the risk of coronary revascularisation procedures by 22% in patients treated with Repatha and statin therapy compared to patients treated with placebo and statin therapy over a mean duration of 26 months.6

References

  1. National Heart, Lung, and Blood Institute. How To Prevent and Control Coronary Heart Disease Risk Factors. Accessed April 10, 2018
  2. Kuklina, EV. Centers for Disease Control and Prevention. Vital signs: prevalence, treatment, and control of high levels of low-density lipoprotein cholesterol. United States, 1999–2002 and 2005–2008. MMWR. 2011;60(4):109–14
  3. Mohan KM, et al. Stroke. 2011;42:1489-94
  4. Bhatt DL, et al. JAMA.  2010;304:1350-7.
  5. Jernberg, T., et al. Eur Heart J. 2015. 36(19), 1163-117
  6. Sabatine MS, Giugliano RP, Keech AC, et al, for the FOURIER Steering Committee and Investigators. N Engl J Med. Evolocumab and Clinical Outcomes in Patients with Cardiovascular Disease. 2017;376:1713-22.

SOURCE: www.hospitalpharmacyeurope.com/editors-pick

Biosense Webster enrolls and treats first patient in QDOT AF study

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Johnson & Johnson Medical Devices Companies announced today that Biosense Webster, a worldwide leader in the diagnosis and treatment of heart arrhythmias, has enrolled and treated the first patient in its QDOT AF Study.

The study will evaluate the delivery of high power, short duration ablation with QDOT MICRO, a novel radiofrequency (RF) ablation catheter for the treatment of paroxysmal atrial fibrillation (AF). The first AF patient was treated at OLV Hospital in Aalst, Belgium, one of eight centers in Europe part of the study that will be enrolling up to 50 patients.

AF is fast becoming one of the world’s most significant health issues – affecting 14 million people across Europe, the Middle East and Africa, as well as placing a critical burden on healthcare systems with up to 2.5% of total healthcare expenditure associated with the disease.

QDOT MICRO is a next generation catheter designed to treat AF in a catheter ablation procedure. In what will be a world-first, it delivers 90 watts of RF power in a four-second temperature-controlled catheter ablation session. Its optimized temperature control and micro-electrode technology is designed to provide more efficient and consistent lesion creation with advanced diagnostics, while simplifying the technique and reducing total ablation time.

“The concept of high power short duration ablation is novel and a potentially groundbreaking advancement for the industry,” said Tom De Potter, MD, FEHRA, Associate Director, Cardiovascular Center Department of Cardiology, Electrophysiology Section at OLV Hospital. “The new modality could result in improvements in clinical outcomes and procedural efficiencies and I look forward to further investigation.” Dr De Potter performed the first QDOT MICRO procedure, and is one of the study clinical investigators.

QDOT MICRO, which is only available for investigational use in Europe, is a steerable multi-electrode catheter with a deflectable tip designed to facilitate electrophysiological mapping of the heart and to transmit RF current to the catheter tip electrode for ablation purposes. In addition to force-sensing technology, the catheter incorporates six thermocouple temperature sensors and three micro electrodes embedded in its tip.

“For over 20 years Biosense Webster has pioneered the development of atrial fibrillation treatment” explains Gabriele Fischetto (Vice President of Johnson & Johnson Cardiovascular Specialty Solutions in EMEA). “QDOT MICRO continues our commitment to deliver solutions that help clinicians heal more hearts and has the potential to increase the standard of treatment for paroxysmal atrial fibrillation”.

SOURCE: www.news-medical.net/news/20180511

3-D printing is transforming care for congenital heart disease

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3-D printing is an emerging technology that is impacting the way cardiologists treat patients with congenital heart disease.

3-D printing is an emerging technology that is impacting the way cardiologists treat patients with congenital heart disease (CHD).

In cardiovascular 3-D printing, the 3-D model is a replica of a patient’s anatomy. These models may be used for precise pre-surgical planning and simulation. This may potentially reduce time spent in the operating room and result in fewer complications.

3-D printing also has the potential to bring transformative change in the education and training of physicians. This technology may lead to an educational shift from an apprenticeship model to a simulator-based learning method that augments traditional mentored training. 3-D models in CHD can reduce the learning curve for cardiac trainees in three key areas–understanding complex 3-D anatomy, high-fidelity simulation experiences and exposure to rare cases.

Experienced practitioners can also benefit by using models for lifelong learning, maintenance of certification or for practice before challenging cases.

Additionally, 3-D models serve a communicative purpose as well. Models can be used between specialists to discuss pathology, surgical plans, anticipated outcomes and peri-operative care, which may reduce medical errors. Models can also be used to help the medical team provide patients and caregivers with a better understanding of the disease process, risks, benefits and alternatives.

“3-D printing is rapidly evolving in medicine, with technical improvements in printers and software fueling new and exciting applications in patient care, innovation and research,” said Dr Shafkat Anwar, a paediatric cardiologist at Washington University in St. Louis, School of Medicine. “The ultimate viability of medical 3-D printing will in large part depend on the impact it has on improving patient care,” he added.

Dr Anwar and colleagues said they predict that the next advances in 3-D printing will likely be driven by improvements in printer technology and print materials. Tissue mimicking materials, which would enable the creation of more life-like models that replicate a patient’s unique anatomy and physiology, are currently in development. As models become more realistic, they may be used to study pathophysiology–or the functional changes observed from a particular disease or syndrome–as well as predict long-term outcomes and choose optimal treatment plans or surgical repairs. Finally, while the technology is in its infancy, there is the potential to print living tissue.

While this technology has the potential to be game-changing, broad adoption is currently hampered by relatively high costs of modelling and printing.

The review paper has been published today in JACC: Basic to Translational Science.

SOURCE: www.europeanpharmaceuticalreview.com/news/75381

GlaxoSmithKline and Innoviva publish positive new IMPACT data

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GlaxoSmithKline, alongside Nasdaq-listed partner Innoviva, announced the publication of the landmark IMPACT study – one of the biggest ever conducted in patients with chronic obstructive pulmonary disease with a history of exacerbation – in the New England Journal of Medicine on Thursday.

The FTSE 100 pharmaceutical giant said that in the study, ‘Trelegy Ellipta’ (fluticasone furoate/umeclidinium/vilanterol) achieved superiority to members of two different classes of dual combination therapy, Relvar/Breo and Anoro, on the primary endpoint of reduction in the annual rate of on-treatment moderate/severe exacerbations and a range of other clinically important outcomes, including lung function and health-related quality of life.

It said results from additional secondary and other endpoints included a “statistically significant” 34% reduction in COPD hospitalisations for Trelegy compared to Anoro, and a reduction of 13% compared to Relvar/Breo, which was not statistically significant.

They also included a “significant” reduction in the risk of on-treatment all-cause mortality, observed for both inhaled corticosteroid containing arms compared to Anoro.

A 42.1% reduction in the risk of on-treatment all-cause mortality was observed for Trelegy compared to Anoro as well.

To fully understand the implications of the all-cause mortality observation, off-treatment data also needed to be considered, GSK explained.

Work was ongoing to investigate that further, and would be presented at future scientific meetings.

“Reducing exacerbations to keep patients out of hospital is a key goal of COPD management alongside improving lung function and quality of life,” said Dave Allen, GSK’s head of respiratory therapy area research and development.

“The IMPACT study shows how Trelegy Ellipta can help patients with a history of exacerbation achieve these goals.

“We believe its publication in NEJM is an important addition to the evidence base that informs the management of this progressive and debilitating disease.”

Dr Fernando Martinez, chief of the division of pulmonary and critical care medicine at the New York-Presbyterian Hospital/Weill Cornell Medical Center, said IMPACT “significantly advances” medicine’s understanding of COPD management by addressing a number of key evidence gaps.

“By comparing various combinations of effective medications in the same device the study clarifies which type of patient gains greatest benefit from each class of medicine,” Dr Martinez explained.

“As many patients experience frequent exacerbations or ‘flare ups’, which can often result in hospitalisation, these data will be highly relevant to patients and clinicians as they consider the optimal treatment.”

GSK and Innoviva said the safety profile of single inhaler triple therapy was consistent with the safety profile of the individual components.

The most common adverse events across the treatment groups were viral upper respiratory tract infection, worsening of COPD, upper respiratory tract infection, pneumonia and headache.

Consistent with previous studies, the incidence of pneumonia as a serious adverse event was 4%, 4%, and 3% for FF/UMEC/VI, FF/VI and UMEC/VI, respectively.

“The role of inhaled corticosteroids in COPD have long been debated, and this landmark trial provides further evidence of their benefit in the population studied and compelling data towards clarifying the role of ICS containing regimens in the COPD treatment paradigm,” said Dr Ted Witek, Innoviva’s senior vice president and chief scientific officer.

“We congratulate our partners at GSK for this vital contribution to the field of respiratory medicine.”

Results from IMPACT were submitted to the regulatory authorities in the US and EU in November 2017 and February this year, respectively.

Further regulatory submissions in other countries were expected during 2018.

SOURCE: www.uk.webfg.com/news/news-and-announcements

Novo Nordisk and Red Cross to tackle NCDs in humanitarian crises

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Pharmaceutical company Novo Nordisk has announced it will work with The International Committee of the Red Cross (ICRC) and the Danish Red Cross (DRC) to tackle chronic care in humanitarian crises.

The partnership will tackle non-communicable diseases (NCDs)─a  growing issue that affect millions of people living in humanitarian crises around the world.

NCDs are long-term conditions that generally progress slowly, such as cardiovascular diseases, asthma and diabetes. Although NCDs affect many of the 65 million people currently fleeing conflict, this is not reflected in resources for humanitarian crises, which focus instead on more acute health threats such as infectious diseases and injuries.

There is a great unmet need for those living with NCDs in humanitarian crises, with the risk of exacerbating chronic conditions such as diabetes and hypertension two to three times higher than beforehand.

The partnership will focus on three main areas: supplying low-cost human insulin to Red Cross operations globally, Novo Nordisk supporting ICRC’s and DRC’s health programmes particularly efforts to improve NCD prevention, and projects to provide care to people with hypertension and diabetes in humanitarian crises.

Novo Nordisk will adapt its ordering and production procedures to better serve the needs of humanitarian organisations and share its knowledge on handling and distributing cold chain products. The company will make a financial contribution of DKK 21.5m.

The ICRC and DRC will be responsible for the implementation of demonstration projects on the ground.

ICRC president Peter Maurer said: “Non-communicable diseases are a silent killer and often overlooked during times of armed conflict. All people need access to appropriate health infrastructure and medicines to manage their health. If you look at Yemen, Syria, Iraq and beyond, thousands will remain with life-threatening illnesses if they are not able to receive essential medical supplies such as insulin to treat diabetes.”

The partnership will also collaborate with the Health in Humanitarian Crises Centre at the London School of Hygiene and Tropical Medicine.

DRC secretary general Anders Ladekarl said: “As partner organisations we will use our influence and scale to do more to address the needs of people living with NCDs in humanitarian crises, and advocate together with other humanitarian and health actors to promote this agenda. This partnership is a first step in realising our collective aspiration that all people with NCDs in humanitarian settings have access to care.”

Several other pharmaceutical companies have joined efforts to tackle NDCs in developing countries in recent years, with GlaxoSmithKline partnering with UK and South African medical research councils to carry out research on NCDs in Africa in 2014, and Novartis and the International Committee of the Red Cross supplying NCD drugs to refugee camps in Lebanon in 2016.

SOURCE: www.pharmaceutical-technology.com