Category Archives: R&D

Amazon sells exclusive diabetes and hypertension devices

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Amazon has launched an exclusive range of medical devices to help people manage their diabetes and hypertension at home.

The Choice devices, produced by the healthcare consultancy, Arcadia Group – which is not affiliated with the British fashion brand of the same name –include blood glucose monitors, blood pressure monitors and test strips.

Choice estimates that diabetes and hypertension affect up to 130 million Americans and hopes that its devices will help those afflicted to benefit from daily monitoring and progress tracking over time.

Conventional models and devices enabled with Bluetooth connectivity features will be available to buy, with pricing said to offer better value over devices that can be bought in pharmacies, according to the company.

Bob Guest, CEO of Arcadia Group, said: “The Choice brand is all about accessible wellness. Consumers no longer need to drive to a store to stand in-line and purchase their medical devices and supplies.

“Now, in the privacy of their home, consumers can review, compare and purchase the products of their choice. No insurance is required. Therefore, customers have the freedom of choice. They will no longer be told by their insurance company what brand they can buy. Choice is freedom.”

Guest went on to describe the products as “best in class and very affordable”. He said the company intends to incorporate voice-driven measurement interpretation as well as individualize wellness recommendations via Amazon’s Echo home speaker devices.

“This is all possible with Alexa, and will provide patients with a wellness experience not available until now,” he said.

Amazon is expanding its health-related products to capture more of the market. In June this year, the online retailer announced it is buying PillPack for $1 billion.

The move is expected to disrupt the pharmacy market by delivering pre-sorted medicines and refills to customers’ doors.

PillPack describes itself as “a full-service pharmacy that delivers a better, simpler experience for people managing multiple medications”. The firm liaises with doctors and also provides customer support.

SOURCE: www.pharmaphorum.com/news

FDA gives go-ahead for CRISPR-based sickle cell disease trial

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Development of a stem cell therapy for sickle cell disease from Vertex and development partner CRISPR therapeutics can go ahead, after the FDA lifted a hold on a review.

The companies had applied to begin an early stage trial of CTX001, a gene therapy derived from a patient’s own stem cells, for beta-thalassemia and sickle cell disease.

Although it’s a long way from the market, the drug could be one of the first to use the revolutionary CRISPR/Cas9 gene editing technology to correct a genetic defect causing a disease.

After an application in April for a phase 1/2 trial in the UNS for adults with sickle cell disease, the FDA had further questions that needed to be resolved.

Without revealing further details, the companies said the trial had been put on hold until they could provide the information the FDA required.

The FDA has now lifted the clinical hold and allowed development to go ahead, although the companies gave no further information about the information required by the regulator.

CRISPR and Vertex have obtained approval for clinical trial applications for several countries outside the US for beta-thalassemia and SCD.

They said they are on track to begin a phase 1/2 study in SCD by the end of 2018 and are enrolling patients transfusion dependent beta-thalassemia in a phase 1/2 trial in Europe.

CTX001 uses the CRISPR gene editing technique to make a patient’s haematopoietic stem cells produce high levels of foetal haemoglobin (HbF) in red blood cells.

HbF is a form of the oxygen carrying molecule haemoglobin naturally present at birth, which is replaced by the adult form of haemoglobin.

The elevation of HbF by CTX001 could alleviate transfusion requirements for beta-thalassemia patients and painful and debilitating sickle crises for sickle cell patients.

CRISPR and Vertex began a strategic research collaboration in 2015 to discover and develop gene editing treatments using the CRISPR/Cas9 technology to correct defects in genes known to cause or contribute to certain diseases.

Vertex has exclusive rights to license up to six new CRISPR/Cas9-based treatments that emerge from the collaboration, and CTX001 represents the first treatment to emerge from the joint research program.

For CTX001, CRISPR and Vertex will equally share all research and development costs and profits worldwide.

Novartis yesterday unveiled data showing its crizanlizumab reduced occurrence of the painful and potentially fatal vaso-occlusive crises that occur when blood cells become stacked in patients with SCD, blocking arteries and cutting the oxygen supply to vital organs.

SOURCE: www.pharmaphorum.com/news/fda

Nanoparticles useful in treating venomous snakebites

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In the future, venomous snakebites could be treated using nanoparticles to bind the venom toxins and prevent them from spreading around the body.

Researchers have identified a new way of treating snake bites, using nanoparticles to bind to venom toxins, preventing the spread of the venom through the body.

Venomous snakebites cause over 100,000 deaths annually, and leave over 400,000 individuals with permanent trauma each year. Snakebites affect 2.5 million people annually.

The standard treatment for snakebites is the intravenous administration of IgG immune molecules that recognize venoms. However, such antivenom therapies must be administered quickly–and by trained healthcare workers– to be effective and are highly specific to particular venoms. There is an ongoing need for a snakebite treatment which can be used in a rural setting and works against the bites of diverse venomous snakes.

In the new work, Dr Kenneth Shea, of the University of California, Irvine, and colleagues engineered nanoparticles that bind to and sequester an array of phospholipases A2 (PLA2)and three-finger toxin (3FTX) molecules found in Elapidae snake venoms. The Elapidae family is a large family of venomous snakes that includes cobras, kraits, tiger snakes, sea snakes, coral snakes and mambas, among other species. The researchers tested the ability of the nanoparticles to block Naja nigricollis (black-necked spitting cobra) venom in mice that received varying doses of the nanoparticles, injected into the skin. Envenomings by this snake in sub-Saharan Africa inflict serious cutaneous necrosis that may leave permanent tissue damage in the victims.

In experiments on isolated cells, the nanoparticles were found to sequester a wide range of Elapidae PLA and 3FTX venoms. Moreover, with collaborator Dr José María Gutiérrez from the Instituto Clodomiro Picado (Universidad de Costa Rica), experiments with mice demonstrated that injections of the nanoparticles at the site of venom injection significantly mitigated the typical necrotic effects–including blistering and ulcers– of the spitting cobra venom. The nanoparticles administered to mice that had not received venom did not have an effect on skin and did not induce systemic toxicity.

“The stable, low-cost nanoparticles have the potential to be administered subcutaneously immediately after the bite at the site of envenoming by this spitting cobra to halt or reduce the extent of local damage and mitigate the systemic distribution of toxins post-envenoming,” the researchers say.

The researchers reported their findings in PLOS Neglected Tropical Diseases.

SOURCE: www.europeanpharmaceuticalreview.com/news/79924

Novo Nordisk to launch connected insulin pens in early 2019

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Danish healthcare firm Novo Nordisk has unveiled plans to introduce new durable, connected insulin pens in early 2019.

The new NovoPen 6 and NovoPen Echo Plus connected insulin pens will replace the NovoPen 5 and NovoPen Echo insulin pens.

Novo Nordisk will commence the launch of new connected insulin pens in the first quarter of 2019. They are expected to be available in more than 50 countries.

The company expects to deliver connectivity for its disposable, pre-filled injection pens later in 2019.

Novo Nordisk commercial strategy and corporate affairs executive vice president said: “Our non-exclusive partnership strategy allows us to integrate with the various digital platforms that people are already using to help manage their diabetes.

“We firmly believe that this will help more people realise the full benefit of our innovative medicines and begin to ease the mental burden of diabetes treatment for those individuals.”

In parallel, the company has also entered into new partnership agreements with major diabetes technology firms such as Dexcom, Glooko and Roche to enable future integration of its connected pens with various digital health solutions.

As part of the deal with Roche, the partnership will incorporate insulin dosage information from Novo Nordisk’s connected pen technology into Roche’s open ecosystem, enabling to communicate with its digital diabetes management solutions such as mySugr.

The firm will also incorporate data from connected pen devices with its partners’ diabetes management solutions such as continuous glucose monitoring (CGM) systems and blood glucose meters (BGM).

Novo Nordisk will incorporate insulin dosing data with Dexcom CGM data in the coming years.

Roche diabetes care global head Marcel Gmuender said: “We believe in the tremendous benefits integrated digital diabetes management solutions can bring to people with diabetes, caregivers and healthcare systems as part of an open ecosystem and are excited to partner with Novo Nordisk to further drive innovation in this area.”

With marketing activities in more than 170 countries, Novo Nordisk is engaged in providing treatment solutions for obesity, haemophilia, growth disorders and other serious chronic diseases.

SOURCE: www.compelo.com/medical-devices/news

Patients call for end to CF drug price row at key meeting

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Representatives of NHS England and cystic fibrosis drug company Vertex are to meet on Thursday to try and resolve a two-year impasse over access to life-changing medications.

Charities and patients called for a resolution to the ongoing pricing row, which began two years ago when NICE said Vertex’s combination therapy Orkambi (lumacaftor+ivacaftor) is too expensive for the NHS.

Orkambi is the first medicine to treat the underlying cause of cystic fibrosis in people with two copies of the F508del mutation, aged six or over, and Vertex has other drugs in its pipeline that will mean a wider group of patients will get treated.

NHS England and Vertex have been locked in an increasingly bitter argument over pricing, with the manufacturer accusing the NHS of undervaluing cystic fibrosis patients.

Vertex is trying to get the NHS to fund all its approved CF drugs, and any future medications in its pipeline in a long-term deal.

In July Vertex said it received an offer worth about £500 million over five years, and more than £1 billion over 10 years for Orkambi.

This was rejected and Vertex has made veiled threats that it will consider spending its R&D budget elsewhere because of the row.

It has also refused to engage with NICE until the cost-effectiveness body changes its assessment methods.

David Ramsden, chief executive of the Cystic Fibrosis Trust has written to Vertex’s chief executive Jeffrey Leiden, and NHS England’s chief executive Simon Stevens calling for the matter to be resolved.

Ramsden urged Leiden to do “everything in [his] power” to reach an agreement, while calling on Stevens to find a way to “value and reward” the innovative drugs from Vertex.

The UK has the second largest population of patients with CF in the world, with around 10,000 people affected.

Christina Walker, from the patient group UKneedsorkambi, said the drug should be made available as soon as possible so that patients such as her son Luis can receive it.

Walker said: “It’s been a devastating summer for the campaign group while this impasse has persisted. We’ve watched our loved ones’ health decline with exacerbations, made many hospital visits and have mourned CF angels who’ve lost their final battle.

“Whether or not this situation continues unchecked is in the hands of the people from Vertex and the NHS around the table on Thursday.

“They must both compromise heavily – more than they want to – because lives are at stake and what’s the alternative? Too many people have died already. These transformational treatments can reduce the considerable suffering of this cruel condition, and patients must have them now. Time is up, and any further delay will be unforgivable.”

SOURCE: www.pharmaphorum.com/news

Shire’s von Willebrand disease therapy Veyvondi approved in EU

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The European Commission has approved Shire’s Veyvondi for treatment of the bleeding disorder von Willebrand disease (VWD).

VWD is the most common inherited bleeding disorder, affecting up to 1% of the global population, and is caused by deficiency or dysfunction in the protein known as von Willebrand factor (VWF).

The commission granted a marketing authorisation for Veyvondi (vonicog alfa, recombinant von Willebrand factor) for bleeding events and treatment or prevention of surgical bleeding in adults with VWD when desmopression treatment alone is ineffective or not indicated.

Veyvondi is the first recombinant treatment for VWD that addresses primary deficiency or dysfunction of VWF while also allowing the body to restore and maintain adequate Factor VIII plasma levels.

Approval is based on outcomes from three clinical trials of a total of 80 patients with VWD exposed to Veyvondi.

These include a phase 1 multicentre, controlled, randomised, single-blind, dose-escalation study of the safety, tolerability and pharmacokinectics  in subjects 18 to 60 years of age with severe VWD.

Also in the dossier was a phase 3 multicentre, open-label study to assess the pharmacokinetics, safety and efficacy of the Veyvondi and recombinant factor VIII and Veyvondi alone in the treatment of bleeding episodes in adult subjects with severe VWD.

There was also a phase 3, prospective, open-label, uncontrolled, non-randomised, international multicentre study to assess the haemostatic efficacy and safety of rVWF with or without recombinant factor VIII in 15 adult subjects with severe VWD undergoing major, minor, or oral elective surgical procedures.

Andreas Busch, head of R&D and chief scientific officer at Shire, said: “The approval in Europe for Veyvondi marks a key milestone in our efforts to tackle unmet medical needs for those living with von Willebrand disease.

“We are excited to take the next steps in ensuring that Veyvondi is widely available across Europe to address the individual needs of those affected by the condition and in need of factor replacement.”

SOURCE: www.pharmaphorum.com/news

New C.diff drug to be tested on patients for first time

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A new drug aimed at treating potentially deadly Clostridium difficile (C. diff) infections is set to be tested on patients for the first time.

Glasgow-based life sciences firm MGB Biopharma (MGB) said it was preparing to launch a Phase II clinical trial of its anti-bacterial agent MGB-BP-3.

The trial is expected to involve 30 patients based in North America.

All have been diagnosed with C.diff-associated disease (CDAD).

C.diff infections can cause diarrhoea and fever.

They have been a major problem in hospitals around the world, with thousands of deaths in the US alone linked to the bug each year.

The bacteria are able to take over the gut when a course of antibiotics kills off the bugs that normally live there.

MGB’s announcement came after it raised £1.3m from investors for trials of the new drug, which was invented at the University of Strathclyde.

The funding round was led by Edinburgh-based Archangels, with co-funding from a range of sources, including the Scottish Investment Bank, Barwell and Melrose-based Tri Capital.

The cash supplements a £2.7m grant awarded earlier this year by Innovate UK.

MGB said its trial would “evaluate safety and tolerability, efficacy and in particular look for improvement in global (or sustained) cure rates”.

Chief executive Dr Miroslav Ravic said: “We are already witnessing renewed interest in our new anti-bacterial agent and its trial in key medical centres in North America where CDAD is particularly prevalent.

“This offers opportunities both to progress the study rapidly and to attract increased attention to the results for this important trial.”

The company said it was aiming to start the trials in areas of the US and Canada with a high incidence of CDAD early next year.

SOURCE: www.bbc.co.uk/news/

From molecule to medicine, the importance of persistence

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Here, Dr Sheuli Porkess, deputy chief scientific officer, Association of the British Pharmaceutical Industry (ABPI), outlines how the pharmaceutical industry takes a substance from molecule to medicine and how the process requires persistence.

A report last week from the Office of Health Economics (OHE) shows the amazing impact medicines have had on the NHS and more widely. The antipsychotic chlorpromazine, first used in the NHS in 1954, paved the way for deinstitutionalisation and community-based care for people with mental illness. In 1948, there were almost 400,000 cases of measles in England and Wales, and 327 people died. By 2015 the number of cases of measles in England and Wales had fallen below 1,200.

These medicines, and others, had a variety of benefits including better clinical outcomes, saving lives, improving quality of life, greater health service efficiency and wider societal impacts. But making medicines is a complicated and costly business. It costs billions of pounds and can take decades. Successes can change the world; failures are an inevitable part of the discovery and development process. But when medicines get through the development process, they can clearly change millions of lives.

There are broadly three stages to creating a new medicine: research, development and approval. Here’s how it works:

Drug discovery and development

The process usually starts with chemical compounds or biological molecules. With advances in technology over the last few years, we can screen compounds that have the potential to become treatments faster than ever before. AstraZeneca — a British pharmaceutical company — launched a new screening robot in 2016 called ‘NiCoLA-B’ which is able to test 300,000 compounds a day. Its job is to find those chemicals that show the slightest potential of being useful as a medicine.

The research stage benefits hugely from collaborative partnerships between the pharmaceutical industry, charities and universities, all working together to find a potential medicine. This stage can take four to five years and takes about 22% of the total budget it takes to find a treatment. Each compound has a less than 0.01% chance of success.

Preclinical research

From a batch of about 10,000 compounds screened in the drug discovery phase, only about 10–20 go into the pre-clinical phase, where scientists determine how safe a medicine might be through testing in cells and animals as well as using computational models.

Clinical research

If any of those 10-20 compounds show real potential of being turned into something useful, they’re developed in to a medicine that will move into clinical trial stage. There are three steps: Phase I involves about 20 to 100 volunteers. If medicines are successful here, they will move onto Phase II where they are tested in people with the disease.

Phase III can include up to 5,000 patients. Going through the three phases can take six or seven years. Over half, or about 65%, of the money it takes to make a medicine is spent in the development stage.

Phase IV clinical trials are after the medicine has a licence and are there to help monitor the medicine’s safety and help clinicians better understand how the medicine works in everyday life, not just in clinical trials.

Approval

The final stage is when regulators review the medicine and it can get ‘market authorisation’ — which shows the medicine is safe and effective. By this point, the manufacturing of the medicine has been scaled up. Only one medicine of 5,000–10,000 compounds discovered will make it to this stage.

The approval processes last anywhere from six months to two years. The medicine is continually monitored once it starts being prescribed for patients.

Researching and developing medicines takes a lot of time and work along the way; there is no guarantee that any particular medicine will make it through the various stages of this highly regulated process. The process is fascinating and once medicines get through this system, their impact can be huge.

Of course, the pharmaceutical industry is pioneering new ways to find treatments. The future looks exciting and how we detect, diagnose and treat disease is set to change significantly.

Advances in medical technology and the miniaturisation of diagnostics, wearables and devices will have a huge impact on our lives and could help people with chronic diseases to remain out of hospital.

Advances in understanding how cells monitor and repair damaged DNA enables us to develop game-changing treatments for cancer. Progress in immuno-oncology sees patients own immune cells used to attack cancer cells, and stem cell therapy is treating rare sight conditions.

We see AI and synthetic biology used for treating malaria, HIV and hepatitis. Gene-editing technology is happening in labs right now, identifying new disease targets, accelerating the discovery of novel treatments.

Passionate pioneers, such as those who invented the groundbreaking treatments in the report, have always been at the heart of our industry and it’s exciting to imagine what their successors could achieve in the next 70 years.

SOURCE: www.epmmagazine.com/opinion

A majority of Americans support using biotechnology to grow human organs in animals for transplants

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Almost six-in-ten Americans (57%) consider it an appropriate use of technology to genetically engineer animals to grow organs or tissues that could be used for humans needing a transplant, while 41% say this would be going too far, according to a new survey by Pew Research Center.

The findings are part of a larger pattern that reveals Americans are more likely to support the bioengineering of animals if it benefits human health.

Demand for transplantable organs and tissues continues to grow in the United States. Last year saw the most organ transplants ever performed in the country. Organs were recovered from more than 10,000 donors – an increase of more than 25% over the past 10 years. Health experts attribute this increase, in part, to breakthroughs in medical technology that have made it possible to recover organs that previously would have been unsuitable for transplants. But, despite these advances, the U.S. Department of Health and Human Services reports that the gap between supply and demand remains wide.

Researchers are hoping to close that gap through the development of new medical technologies. One such approach is 3D organ printing– a process that uses “bio-ink” to print layers of cells that grow to form transplantable tissue.

Another method under development uses genetic engineering to grow human organs and tissues in animals. There was a breakthrough with this technique earlier this year, when scientists used gene editing to create hybrid embryos containing both human and sheep cells.

When the survey – conducted April 23-May 6 – asked the 41% of respondents who opposed this application of genetic engineering to explain, in their own words, the main reason behind their view, the objections included concerns about the use of animals in this way for human benefit (21% of those asked) and the potential risks for human health (16% of those asked).

The responses included:

“In manufacturing organs, the existence of these animals would be miserable … I can’t ethically say that I would agree with such a practice.”

“Factory farming already as an industry unethically treats animals. I imagine organ growing wouldn’t treat the animals any differently.”

“When you mix human and non-human genetics I believe that will cause extreme problems down the road.”

“Even human-to-human organ transplants often reject, so I can only imagine the bad side effects that an animal-to-human transplant would cause. Keep things simple and the way nature intended.”

Majority supports bioengineering animals to grow human organs

SOURCE: www.pewresearch.org/fact-tank

JSM could lead to improved arthritis treatment

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An algorithm to monitor the joints of patients with arthritis, which could change the way that the severity of the condition is assessed.

An algorithm to monitor the joints of patients with arthritis, which could change the way that the severity of the condition is assessed, has been developed by a team of engineers, physicians and radiologists led by the University of Cambridge.

The technique, which detects tiny changes in arthritic joints, could enable greater understanding of how osteoarthritis develops and allow the effectiveness of new treatments to be assessed more accurately, without the need for invasive tissue sampling.

Osteoarthritis is the most common form of arthritis in the UK. It develops when the articular cartilage that coats the ends of bones and allows them to glide smoothly over each other at joints, is worn down, resulting in painful, immobile joints. Currently, there is no recognised cure and the only definitive treatment is surgery for artificial joint replacement.

Osteoarthritis is normally identified on an x-ray by a narrowing of the space between the bones of the joint due to a loss of cartilage. However, x-rays do not have enough sensitivity to detect subtle changes in the joint over time.

Joint space in hip, knee and ankle joints as analyzed by the JSM algorithm. – Tom Turmezei

 

“In addition to their lack of sensitivity, two-dimensional x-rays rely on humans to interpret them,” said lead author Dr Tom Turmezei from Cambridge’s Department of Engineering. “Our ability to detect structural changes to identify disease early, monitor progression and predict treatment response is frustratingly limited by this.”

The technique developed by Dr Turmezei and his colleagues uses images from a standard computerised tomography (CT) scan, which isn’t normally used to monitor joints but produces detailed images in three dimensions.

The semi-automated technique, called joint space mapping (JSM), analyses the CT images to identify changes in the space between the bones of the joint in question, a recognised surrogate marker for osteoarthritis. After developing the algorithm with tests on human hip joints from bodies that had been donated for medical research, they found that it exceeded the current ‘gold standard’ of joint imaging with x-rays in terms of sensitivity, showing that it was at least twice as good at detecting small structural changes. Colour-coded images produced using the JSM algorithm illustrate the parts of the joint where the space between bones is wider or narrower.

“Using this technique, we’ll hopefully be able to identify osteoarthritis earlier, and look at potential treatments before it becomes debilitating,” said Dr Turmezei, who is now a consultant at the Norfolk and Norwich University Hospital’s Department of Radiology. “It could be used to screen at-risk populations, such as those with known arthritis, previous joint injury, or elite athletes who are at risk of developing arthritis due to the continued strain placed on their joints.”

While CT scanning is regularly used in the clinic to diagnose and monitor a range of health conditions, CT of joints has not yet been approved for use in research trials. According to the researchers, the success of the JSM algorithm demonstrates that 3D imaging techniques have the potential to be more effective than 2D imaging. In addition, CT can now be used with very low doses of radiation, meaning that it can be safely used more frequently for the purposes of ongoing monitoring.

“We’ve shown that this technique could be a valuable tool for the analysis of arthritis, in both clinical and research settings,” said Dr Turmezei. “When combined with 3D statistical analysis, it could be also be used to speed up the development of new treatments.”

The results are published in the journal Scientific Reports.

SOURCE: www.europeanpharmaceuticalreview.com/news/76547