Amgen CEO says Aimovig is “one of most successful launches”

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As biosimilars start to eat into sales of its blockbuster biologic drugs, Amgen needs new products to deliver growth – and it says migraine therapy Aimovig is off to a great start.

Chief executive Bob Bradway told investors on the company’s third-quarter results call that Aimovig (erenumab) is “shaping up to be one of the industry’s most successful recent launches” with sales reaching $22m in the quarter, ahead of analyst expectations.

“People suffering from migraine and their physicians have been waiting for years for an effective new therapy and they’ve reacted very well to Aimovig,” he said. The company estimates that more than 100,000 patients have taken the Novartis-partnered CGRP inhibitor since its launch in May as a once-monthly, self-injected preventive treatment for migraines.

The strong take-up has come despite approvals for rival CGRP inhibitors from Teva and Lilly, and Amgen says its first-mover advantage is backed up by a patient-friendly delivery device, as well as a willingness by healthcare payers to cover the product thanks to a price that encourages access.

The company is also making much of the fact that Aimovig is the only drug that targets the CGRP receptor rather than the CGRP protein itself, although for now there’s no strong evidence to suggest that confers a clinical advantage.

There’s no doubt that the rapid roll-out comes from pent-up demand for the drug among people with migraine who still suffer headaches despite other therapies. However Amgen’s commercial head Murdo Gordon said he believes “we’re scratching the surface of helping chronic migraine sufferers [and] we think that we’ll see continued demand growth.”

Overall, Amgen posted a 2% rise in revenues to $5.78bn in the quarter, despite pressure on older drugs like while blood cell booster Neulasta (pegfilgrastim) which showed the first signs of weakness from biosimilar competition after Mylan/Biocon launched their Fulphila rival in July. TNF inhibitor Enbrel (etanercept) also fell 5% in the face of competition from newer drugs in indications such as psoriasis.

There was also disappointing news once again for cholesterol drug Repatha (evolocumab) which rose 35% to $120m but missed expectations. Last week, Amgen reduced its price by 60% to $5,850 in a bid to kickstart sales growth and overcome payer resistance to the drug.

“This should substantially lower their out-of-pocket costs and lower the abandonment rate which is as high as 75%,” said Gordon, who said the cut was necessary to compete and would build much-needed volumes at the expense of near-term sales.

The Repatha discount comes as the US administration is ramping up the pressure on the biopharma industry on drug pricing, most recently proposing a reference pricing model to bring process closer into line with other affluent nations.

Commenting on the overall environment and how it looks likely to develop in 2019, Bradway said that the US has benefitted enormously from a market which allows patients to access new therapies “more quickly and more comprehensively than in other countries.”

“We think what’s required is more innovation, not less, and the system that enables people to get access to that innovation swiftly,” he suggested.

SOURCE: www.pmlive.com/pharma_news

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

Roche and SQZ expand cell therapy deal to more than $1.3bn

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US biotech SQZ Biotechnologies has expanded a collaboration with Roche to develop cancer therapies based on antigen presenting cells, which may be able to target solid tumours and which are cheaper to make than the recently approved CAR-T drugs.

The deal announced late yesterday expands a collaboration that began in 2015 to a deal worth more than $1.375 billion if all development targets are met.

SQZ’s technology is based around antigen presenting cells (APCs), which occur naturally in the body and present antigen on their surface to stimulate a T-cell response against that particular antigen.

SQZ is able to genetically engineer APCs with tumour antigens, that when injected into the body can produce powerful responses from CD8 T-cells – also known as killer T-cells.

The hope is that these killer T-cells will produce a strong response against any tumour target of interest, across a wide range of cancers.

Roche already has a cancer immunotherapy on the market – its Tecentriq (atezolizumab) is already approved in certain kinds of bladder and lung cancer.

But unlike Novartis and Gilead it does not have cell therapy approved yet and is looking to develop cell therapies that could be used in solid tumours, instead of blood cancer like its rivals’ CAR-T therapies.

Under the collaboration, SQZ may receive up to $125 million in upfront payment and near-term milestones.

SQZ could earn up to $250 million in clinical, regulatory and sales milestones per product that emerges from the collaboration.

The Massachusetts-based biotech may receive development milestone payments of over $1 billion. Within the collaboration, SQZ and Roche could share commercial rights for certain approved products.

Howard Bernstein, chief scientific officer of SQZ, said: “This collaboration allows for a SQZ APC product engine that could potentially generate products with more potent immunologic responses through a simplified, more efficient manufacturing process.”

Chimeric antigen receptor T-cell (CAR-T) drugs are potentially curative in the patients that respond – but are very expensive to make as they involve harvesting a patient’s T-cells, genetically modifying them in a lab, and then re-injecting them to fight cancer.

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

Sequiris invest £40m into expansion of Liverpool plant creating 100 new jobs

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The Liverpool-based vaccine-maker Sequiris is investing £40 million into the expansion of its operations in the Speke area of Liverpool.

The multi-million pound investment is set to create 100 new jobs while allowing vaccines to be created more quickly in preparation for outbreaks of the flu. While vaccines are currently shipped to Italy for packaging, the expansion will now allow the process of packing the vaccines into syringes and capsules to be done in the UK.

The company, which is as of now the second biggest influenza vaccine company in the world, provides around half of the vaccines used in the UK. The project which is expected to be complete in 2019 will increase the safety of the government contracted vaccine makers products.

The investment will also increase the speed with which vaccines reach the market in the UK.

Dr Laura O’Brien, vice president of operations and site head at Liverpool, commented: “This investment means we can complete production of more pandemic vaccine much more rapidly, saving vital days in getting vaccines out to protect the British population as well as to other countries around the world.”

SOURCE: www.pharmafile.com/news/519043

Ahead of the curve

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Pharma often criticises the NHS for failing to adopt the latest therapies, arguing that NICE’s technology appraisals can drag on and that hospitals often fail to provide the recommended therapies anyway.

However in the case of CAR-T therapies, it seems that it has responded to the criticism – at least in part, anyway.

Chimeric antigen receptor T-cell (CAR-T) therapies from Novartis and Gilead were approved by the European regulators in August – and the NHS was quick to strike deals to make them available to patients in England.

At the end of August, the European Commission approved Kymriah in children and young adults aged up to 25 with refractory acute lymphoblastic leukaemia (ALL), in relapse post transplant or in second or later relapse.

Within days the NHS had announced a deal with Novartis giving paediatric patients access to its CAR-T, Kymriah (tisagenlecleucel).

And although Gilead’s CAR-T, approved for adults with relapsed or refractory large cell lymphoma, was initially rejected by NICE, the manufacturer has managed to strike a deal with the NHS to allow patients access with reimbursement from the Cancer Drugs Fund.

Novartis’ CAR-T has been rejected by NICE in its adult indication, but this could well change if the Swiss pharma strikes a similar deal.

Horizon scanning

What stands out about the CAR-T story is that NHS officials saw that these therapies were heading to market quickly, after regulators allowed them to be approved on the basis of smaller trials, something that was possible because the therapies can be very effective in the patients that respond.

NHS chief executive Simon Stevens publicly said that getting CAR-Ts to patients is a priority – even though they are costly to manufacture, and patients need intensive monitoring to ensure they do not develop the fearsome side-effects associated with the therapy.

At Kisaco Research’s Combined CAR-T conference in Berlin earlier this year, experts revealed how the NHS had already begun to develop specialist centres capable of providing the therapies.

And a mock assessment by cost-effectiveness experts prior to approval had already indicated that the therapies could be good use of NHS resources, despite their high costs.

The NHS has built on this and is working on further CAR-T centres at hospitals across the country.

Speaking after the latest deal with Gilead, Stevens said: “CAR T-cell therapy is one of the most promising new treatments in a generation for lymphoma and leukaemia, and NHS patients will now be among the first in the world to benefit.”

Dr Alasdair Rankin, director of research and patient experience at the blood cancer charity Bloodwise, said: “CAR-T therapies have shown huge promise in treating patients with lymphoma who have no other chance of cure. It’s admirable that the NHS and the pharmaceutical company have worked hard to make this pioneering treatment available so quickly, giving hope to current patients and their families.”

But as Bloodwise’s Rankin points out, getting reimbursement in place is just a first step – the experience in the US shows that despite all the prep work by hospitals, getting the therapies to patients is still a logistical challenge.

The manufacturing process involves taking a patient’s own T-cells, sending them to a lab where they are genetically modifies, before sending them back to the patient where they are re-injected to fight the cancer.

Financial results from Novartis and Gilead, and anecdotal evidence, suggests that only a few patients have received the therapies outside of clinical trials as hospitals struggled through a backlog of eligible patients.

Rankin said in a statement: “The next big challenge for the NHS will be to deliver this new and complex treatment on a scale that ensures access for the hundreds of patients with these lymphomas who could benefit from CAR-T therapy each year.

Off-the shelf

The challenges posed by CAR-T therapies are immense – but the good news is that scientists are working on ‘off-the-shelf’ drugs that are made from banks of cells and can be administered to many different patients.

These therapies promise to have similar efficacy to CAR-T, but at a fraction of the price and without the costly and time consuming electrophoresis process used to harvest the T-cells.

At the CAR-T Congress earlier this year, researchers at MD Anderson Cancer Center at the University of Texas estimated that ‘off-the-shelf’ therapies derived from natural killer (NK) cells could be made available at 1% of the cost of CAR-T cells.

CAR-NK cells would be derived from cord blood, allowing them to be stored in banks and used on several patients, vastly reducing manufacturing expenses.

There is also the possibility that future cell therapies could target solid tumours, something that is not possible with Kymriah or Yescarta.

These therapies are not able to target the antigens present on solid tumours, and the CAR-T cells are not tough enough to infiltrate tumours.

Kymriah and Yescarta are just the start – whether the NHS is agile enough to keep pace with cell therapies for cancer and other diseases as they are developed remains to be seen.

Bloodwise’s Ranking concluded: “It is likely that we are only beginning to see the benefits that CAR-T therapy can bring. Treatments will continue to improve and become more effective over the coming decade and will benefit patients with other types of cancer.”

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

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

Ultra-thin meta lens enables full colour imaging

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A meta lens developed by researchers at only one micron thick, has the ability to perform better than any compound lens currently available.

The first flat lens capable of correctly focusing a large range of colours of any polarisation to the same focal spot without the need for any additional elements has been developed.

Researchers at Colombia University School of Engineering and Applied Science developed the lens which is only a micron thick, but offers the performance of the best compound lens systems.

Associate Professor Nanfang Yu led the study and said: “The beauty of our flat lens is that by using meta-atoms of complex shapes, it not only provides the correct distribution of delay for a single colour of light but also for a continuous spectrum of light.

“And because they are so thin, they have the potential to drastically reduce the size and weight of any optical instrument or device used for imaging, such as cameras, microscopes, telescopes, and even our eyeglasses. Think of a pair of eyeglasses with a thickness thinner than a sheet of paper, smartphone cameras that do not bulge out, thin patches of imaging and sensing systems for driverless cars and drones, and miniaturised tools for medical imaging applications.”

The meta-lenses used standard 2D planar fabrication techniques similar to those used for fabricating computer chips. The researchers mention that mass manufacturing these meta-lenses should be simple as they are only one layer of nanostructure, and there is no need to go through the expensive and time-consuming grinding and polishing process.

“The production of our flat lenses can be massively parallelised, yielding large quantities of high performance and cheap lenses,” noted Sajan Shrestha, a doctoral student in Yu’s group who was co-lead author of the study. “We can therefore send our lens designs to semiconductor foundries for mass production and benefit from economies of scale inherent in the industry.”

“Our design algorithm exhausts all degrees of freedom in sculpting an interface into a binary pattern, and, as a result, our flat lenses are able to reach performance approaching the theoretic limit that a single nanostructured interface can possibly achieve,” Adam Overvig, the study’s other co-lead author and also a doctoral student with Yu, said.

“In fact, we’ve demonstrated a few flat lenses with the best theoretically possible combined traits: for a given meta-lens diameter, we have achieved the tightest focal spot over the largest wavelength range.”

The study was published by Nature’s Light: Science and Applications.

SOURCE: www.europeanpharmaceuticalreview.com/news/79753/meta-lens-full-imaging

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

Study using DFMO shows positive results for children with high risk neuroblastoma

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A paper published September 27 in Scientific Reports shows the positive results of a phase II clinical trial using the oral medication DFMO to prevent relapse in children with High Risk Neuroblastoma (HRNB).

Neuroblastoma is a form of cancer that develops from immature nerve cells found in several areas of the body. It occurs most often in infants and young children, usually under the age of five. The disease remains a challenge in pediatric oncology and current treatments include therapies that have significant long-term side effects for patients.

HRNB accounts for 15 percent of all childhood cancer deaths, in part, due to the fact that nearly half of all patients who reach remission will relapse.

“These results are promising and have changed the outlook for our patients with high risk neuroblastoma,” said Giselle Sholler, MD, director of pediatric oncology research at Spectrum Health Helen DeVos Children’s Hospital and principal investigator of the study.

“By using DFMO for two years after finishing conventional therapy, we’ve seen an overall two-year survival rate for these children of 97 percent. This is a large increase in survival,” Sholler added. “Previously it was believed that children with refractory and relapsed neuroblastoma were considered incurable. This study shows more than 50 percent of patients remaining in remission up to four years.”

Beat Childhood Cancer’s trial studied the use of difluoromethylornithine (DFMO) as a single agent for enrolled patients at 20 children’s hospitals from June 2012 to February 2016. The children received two years of oral DFMO twice daily and were evaluated for outcomes of event free survival (EFS*) and overall survival (OS). The study used targeted oral therapy of an ODC inhibitor (DFMO), as a maintenance therapy to prevent relapse in HRNB patients after standard therapy. DFMO works by targeting specific cancer stem cell pathways and “turning off” the cells, thereby preventing the cancer from growing back.

There were two arms in this study, the first designed for patients who had completed standard therapy, and the second for children who were able to achieve remission after having previously relapsed. Both of these patient populations are at very high risk of relapsing after completing treatment and therefore can be very good candidates for using a maintenance therapy with the goal of preventing relapse.

With a median follow up of 3.5 years, the first arm of the study had 100 eligible patients. The results show that two-year EFS was 84 percent and two-year OS was 97 percent.

With a median follow up of 3.7 years, the study enrolled 39 previously relapsed patients and the results reported in the journal showed that two year EFS was 54 percent and two-year OS was 84 percent for these children who had previously relapsed.

“While these EFS and OS figures at two years are remarkable, the really exciting part of these results is that EFS and OS are stable out to four years,” said Patrick Lacey of Beat NB Cancer Foundation, one of the childhood cancer parent-led foundations that funded this clinical trial. “Not only did this oral drug lead to a prolonged and stable remission for the children in this study, but the drug was extremely safe and well tolerated in this patient population.”

“While many children have been able to attain remission with the current, albeit harsh, upfront therapies, these remissions are not historically durable,” Dr. Sholler added. “The current five-year survival curves have not changed significantly in the past two decades despite recent increases in two-year survival as a result of intensified therapies and new multimodal therapies.”

Principal Investigator at MUSC, Jaqueline Kraveka, MD, states survival for children with high-risk neuroblastoma remains a challenge. “These results are groundbreaking and very exciting for oncologists and their patient families. I am thrilled to have our confirmatory study open at so many sites across the USA and Canada, enabling children to receive this treatment close to home.”

SOURCE: www.news-medical.net/news/20181001