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US-based medical technology firm Electronic Caregiver has partnered with Realtime Senior Living to help patients easily locate quality senior living options and care services in real-time.

With Realtime Senior Living’s advanced applications, case managers and care coordinators will be able to track patients and their families to senior living facilities equipped with Electronic Caregiver’s safety health monitoring devices.

Through the partnership, Realtime’s current search app will enable case managers to quickly track available care services in the area that meet the personalised needs of a patient.

Electronic Caregiver chief clinical officer Tim Washburn said: “With this partnership, we are really looking to reimagine the hospital discharge process for families and providers alike.

“We have patients who stay in hospitals extra days because they aren’t able to finalise placement in a senior care facility, so there’s a huge cost to the hospitals; there’s a huge cost to the patients and their families.

“Hospitals are places where people are when they’re sick, so if you’re going to be in a hospital, your very first goal is to get out of the hospital. Anything we can do to speed that up so it’s a good transition, that’s in our wheelhouse, and that’s what this partnership should help us to do.”

The products and service offerings by Electronic Caregiver include Premier and Pro Health.

Electronic Caregiver’s Premier is a wrist pendant and features a Medical Concern Button that connects users with Emergency Medical Dispatch Operators at any time.

ProHealth offers vitals monitoring and emergency response. Both Premier and ProHealth allow medication reminders and round the clock physician-on-demand service, pocketMD.

Realtime Senior Living founder and CEO Steve Gilbert said: “We’ve been looking for opportunities to enhance the experience within the communities and homes that we partner with.”

In early 2020, both the companies aim to launch the app featuring Electronic Caregiver providers.

Researchers at the University of Rhode Island, Butler Hospital and BayCare Health System have collaborated to launch retinal scanning tests for early detection of Alzheimer’s disease.

Morton Plant Mease Health Care Foundation and St Anthony’s Hospital Foundation provided funds for this five year, $5m Atlas of Retinal Imaging in Alzheimer’s Study (ARIAS).

Researchers claim that the simple retinal screening test could be more cost-effective compared to the current positron emission tomography scans, which can detect amyloid plaque in the brain, associated with Alzheimer’s before symptoms appear.

Snyder said: “When our study is completed, we want to make the technology available so that optometrists and ophthalmologists could screen for the retinal biomarkers we believe are associated with Alzheimer’s disease and watch them over time.

“If clinicians see changes, they could refer their patients to specialists early on. We believe this could significantly lower the cost of testing. We may then identify more people in the very earliest stage of the disease, and our drug therapies are likely to be more effective at that point and before decades of slow disease progression.”

The ARIAS study will focus on preclinical stage disease detection, considering the retina as a complex biological system, enabling the researchers to observe the retina in several different manners at the same time.

Through lasers, pictures of the retinas of participants can provide a microscopic look at the anatomy, changes in pigment chemicals and the movement of red blood cells in the retina.

Researchers will enrol 330 participants between the ages of 55 and 80, ranging from very healthy and low-risk adults to persons with concerns regarding their memory, as well as patients with mild Alzheimer’s disease.

Each participant will be examined at four different periods for over three years.

Each study visit consists of an eye exam, a medical history discussion, tests on how well individuals remember new information, retinal imaging and measures of mood, walking and balancing and sleep habits.

Medtech company Beta Bionics has received FDA Breakthrough Device designation for its automated bionic pancreas – the iLet Bionic Pancreas System.

The iLet Bionic Pancreas System is a pocket-sized, wearable investigational medical device that is designed to autonomously control blood-sugar levels. The on-body wear is similar to that of an insulin pump. The investigational system is designed such that users enter only their body weight for the iLet to initialise therapy. Immediately thereafter, the iLet begins controlling blood-sugar levels automatically, without requiring the user to count carbohydrates, set insulin delivery rates, or deliver bolus insulin for meals or corrections.

The iLet is designed to function as three medical devices in one. It can be configured as an insulin-only bionic pancreas, a glucagon-only bionic pancreas, or a bihormonal bionic pancreas using insulin and glucagon. The insulin-only and bihormonal configurations may be helpful in diabetes. The glucagon-only configuration may be helpful in rare conditions that often lead to chronic, low blood-sugar conditions, such as congenital hyperinsulinism (CHI). Beta Bionics is committed to obtaining regulatory approval and commercialising all three iLet configurations.

The Breakthrough Designation for the iLet Bionic Pancreas System contemplates configurations with most insulin analogues approved for pumping as well as dasiglucagon, Zealand Pharma’s stable pumpable glucagon analogue, which has a unique stability profile in a ready-to-use aqueous solution.

Ed Damiano, president and CEO of Beta Bionics, said: “We believe the iLet Bionics Pancreas System represents a true breakthrough therapy for the management of glycemia, particularly in type 1 diabetes. We are particularly excited by the possibility that the iLet may be able to provide safer and more effective therapy in far more people than current therapies due to its simplicity of use.”

Dive Brief:

• FDA awarded premarket approval to Terumo’s MicroVention subsidiary for a flow diversion system to treat wide-neck intracranial aneurysms of the carotid artery. 
• The Flow-Redirection Endoluminal Device, or FRED, expands to a pre-determined diameter after implant to divert blood flow from entering the neck of the aneurysm, an abnormal bulge in an artery in the brain caused by weakness in the vessel wall.
• The approval was supported by a clinical study that demonstrated a primary effectiveness success rate of 56.7% in an evaluation of 135 patients at 12 months. Nine primary safety endpoint events included patients who experienced strokes including one death, given the population, the FDA said the risk-benefit ratio is favorable.
  

  Dive Insight:

  A subsidiary of Japanese medical device maker Terumo since 2006, MicroVention has been on a roll with a string of FDA approvals over the past two years.

  The California-based unit consolidated its operations in 2017 in a new 205,000-square-foot center in Aliso Viejo built to support its anticipated growth. The company specializes in catheter-based, minimally invasive treatments for cerebral aneurysms, ischemic stroke and carotid artery disease.

  The Brain Aneurysm Foundation estimates six million people in the United States have an unruptured brain aneurysm. MicroVention is among a number of companies, which include Stryker, Johnson & Johnson and Medtronic, focused on treatments for the condition.

  MicroVention’s latest premarket approval is its third in less than two years. Last January, the company was awarded a PMA for its WEB embolization system to treat intracranial aneurysms. That device, implanted in the aneurysm sac to create a scaffold and disrupt blood flow, was acquired through Terumo’s 2016 acquisition of Sequent Medical.

  The company in 2018 picked up a PMA for a pair of LVIS stents that support coil embolization for intracranial aneurysm treatment. Its SOFIA catheter also received FDA clearance in 2018 for a new indication for an aspiration technique for revasularization in patients who have suffered acute ischemic stroke.

  Tokyo-based parent Terumo’s products are used in cardiothoracic surgery, interventional procedures and transfusion medicine. The company also makes a range of syringes and hypodermic needles for hospital and physician office use.

AstraZeneca has started 2020 strongly, with the FDA granting a fast review for its big-selling Farxiga in heart failure and a key drug approval in China.

AZ said the FDA has accepted a filing for Farxiga (dapagliflozin) to reduce risk of cardiovascular death or worsening of heart failure in adults with reduced ejection fraction, with and without type 2 diabetes.

This allows for a faster six-month review of the trial data in the new indication for Farxiga instead of the standard 10-month period.

The filing is based on results from the phase 3 DAPA-HF trial, announced last year, which showed Farxiga on top of standard of care reduced the incidence of the composite outcome of cardiovascular death or worsening of heart failure versus placebo.

Farxiga was first established as a drug controlling blood sugar in patients with type 2 diabetes, but AZ has been working on extending its remit and building sales.

Originally brought into the company after AZ bought out Bristol-Myers Squibb’s share of a diabetes alliance, Farxiga is one of AZ’s most important drugs with revenues north of $1.1m in Q3 alone.

The FDA already approved it in October to reduce chances of hospitalisation for heart failure in adults with type 2 diabetes and other cardiovascular risks.

Lokelma approved in China for hyperkalaemia

In a separate announcement AZ said its Lokelma (sodium zirconium cyclosilicate) has been approved by regulators in China for adults with hyperkalaemia – elevated levels of potassium in the blood.

Approval by China’s National Medical Products Administration (NMPA) was based on positive results from the clinical trials of Lokelma and a pharmacodynamic study in China.

Results showed that patients receiving Lokelma experienced a significant, rapid and sustained reduction of potassium in the blood.

Lokelma was only recently launched in the US and sales have yet to take off, but AZ has high hopes for the drug after acquiring its original developer ZS Pharma for $2.7 billion in 2015.

The news follows the Queen’s decision to award R&D guru Mene Pangalos with a knighthood in the New Year Honours List, an indication of the company’s growing reputation as a scientific powerhouse.

Pangalos has overall responsibility for AstraZeneca’s research and development (R&D) activities in BioPharmaceuticals.

Since joining in 2010, he has pioneered programmes to promote novel open innovation partnerships with non-governmental and peer organisations, and more than 200 academic institutions to help drive the UK’s position as a world leader in life sciences R&D.

Created by a luminary sleep researcher and doctor, the intelligent device features novel cooling technology to calm “a racing mind”

LAS VEGAS and PITTSBURGH, Jan. 4, 2020 /PRNewswire/ — Ebb Therapeutics is on a mission to make an impact in both the professional healthcare and consumer sleep markets. At CES 2020(Booth # 43824/Sands Expo, January 7-10, Las Vegas, NV), the sleep tech company will debut a wearable sleep device with proprietary technology that targets the root cause of sleeplessness: a racing, or overactive, mind.

THE RESEARCH
Founder and Chief Medical Officer Dr. Eric Nofzinger spent 35 years studying the brain mechanisms of healthy sleep. Studies suggest that reduction in metabolic activity in the frontal cortex (the thinking part of the brain) is key to the restorative properties of sleep. Meanwhile, higher-than-normal activity when the body is at rest leads to hyperarousal, which is often associated with clinical complaints of not being able to turn off one’s mind when trying to sleep. While Director of the Sleep Neuroimaging Research Program at the University of PittsburghSchool of Medicine, Dr. Nofzinger discovered that when the forehead is cooled, brain activity decreases and the mind can relax. This finding inspired Ebb.

“Sleeplessness is an epidemic. More than ever, people are struggling with being able to ‘turn off’ their minds in order to get to sleep,” said Dr. Nofzinger. “Lack of sleep can have a dire effect on one’s ability to function mentally, emotionally and physically, so it is critical that we keep innovating in this space in order to offer safe, drug-free solutions that work.”

THE PRODUCT AND TECHNOLOGY
Ebb is the first and only sleep solution that uses precise cooling, called PrecisionCool™ Technology, to reduce metabolic activity in the frontal cortex. A scientifically-engineered, fluid-filled headband softly wraps around the head and cools the forehead using an intelligent cooling algorithm in order to maintain an optimal temperature range over the course of the night. As brain activity declines, the mind calms and the user is able to fall into a deeper, more restorative sleep.

The device hits the consumer market after several years of research and fundraising. At the end of 2016, the company closed a $38 million Series B round led by KKR & Co. LP. (Other investors included Versant Ventures, Arboretum Ventures, and Partner Ventures.) These investments allowed the company to conduct the type of studies and clinical tests needed to perfect the technology, and build a first-class team, which includes former executives from Respironics, a leader in sleep and respiratory markets that was acquired by Philips in 2008 for $5 billion.

EBB THERAPEUTICS AT CES
“At CES, we are introducing technology that is truly first-of-its-kind. As consumers, finding a solution for any issue concerning health can be both emotional and overwhelming,” said Ebb Therapeutics CEO Eduard Roosli. “We are proud to be able to provide a product that is backed by decades of science, has been clinically tested for efficacy, and is completely safe.”

At the show, the startup will also tease another innovation, set to hit the market in late 2020: a second-generation device with similar cooling technology that is battery-powered and portable, for consumers with busy, on-the-go lifestyles.

The US Food and Drug Administration’s (FDA) momentum of new drug approvals dropped marginally in 2019 — while 62 novel drugs were approved in 2018, 54 were approved in 2019. FDA’s Center for Drug Evaluation and Research (CDER) approved 48 drugs while another six were approved by the Center for Biologics Evaluation and Research (CBER).

Novartis dominated new drug approvals as CDER approved five of its new drugs across five different therapeutic areas. But then, it also made headlines when the FDA raised data accuracy issues over the Swiss drugmaker’s gene therapy — Zolgensma — which sent the company into a crisis management mode.

The gene therapy drug came into the fold of Novartis in April 2018 through the US$ 8.7 billion acquisition of Illinois-based AveXis Inc.

Zolgensma — the world’s most expensive drug — was approved as a one-time treatment for spinal muscular atrophy (SMA) in May 2019.

However, in March 2019, Novartis learnt of manipulation in the raw data derived from a mice assay used at the time to test Zolgensma samples, but only alerted the FDA in June — about a month after the drug gained US approval. 

Post that, Novartis came under fire from US lawmakers who said the company should have informed the regulators about the data irregularities before the drug’s approval in May, instead of waiting to conclude an internal investigation. 

Novartis’ other approvals were brolucizumab (for treatment of macular degeneration), siponimod (to treat multiple sclerosis), alpelisib (breast cancer), crizanlizumab (sickle cell disease) and triclabendazole (fascioliasis, a tropical disease).

Vertex’s Trikafta leads in estimated sales potential 

At PharmaCompass, we compiled the sales forecasts for the new drugs approved by the FDA in 2019. Vertex’s cystic fibrosis treatment — Trikafta — with expected sales of US$ 3.935 billion by 2024, leads our list.

Trikafta is a combination of ivacaftor, tezacaftor and elexacaftor. Vertex already has three cystic fibrosis drugs on the market — Kalydeco (a standalone ivacaftor treatment), Symdeko (a combination of ivacaftor and tezacaftor) and Orkambi (which contains ivacaftor and lumacaftor).

Trikafta’s stellar clinical data made the FDA approve the drug within three months of Vertex’s application filing and five months before FDA’s action date.

Abbvie’s Humira, the top-selling drug in the world, is beginning to face generic competition in certain parts of the world and two new drug approvals in 2019 — Skyrizi (risankizumab for plaque psoriasis) and Rinvoq (upadacitinib for rheumatoid arthritis) — are critical to AbbVie’s plans to grow beyond Humira. The two drugs are expected to contribute over US$ 5.5 billion by 2024 to AbbVie’s top-line. 

In April, AstraZeneca struck a deal worth US$ 6.9 billion with Japanese drug majorDaiichi Sankyo, to jointly develop and commercialize the antibody drug conjugate cancer therapy —trastuzumab deruxtecan. The breast cancer treatment won FDA approval in December and analysts forecast sales of US$ 2.5 billion by 2024 with some believing that the drug could achieve US$ 7 billion in peak sales.

The rise of new drugs from Asia

While Daiichi won the FDA nod for its cancer treatment — Turalio (pexidartinib) — new drugs from established Japanese drug makers like Shionogi, Kyowa Kirin and Eisai also bagged the American agency’s approval.

Shionogi’s novel antibiotic — Fetroja (cefiderocol) — got approved for complicated urinary tract infections while Kyowa won the nod for its Parkinson’s treatment — Nourianz (istradefylline). Eisai’s insomnia drug — Dayvigo (lemborexant) — was one of the last drugs approved in the year and is expected to generate sales of US$ 329 million.

In 2019, the FDA also approved new drugs from China and Korea.  Zanubrutinib (branded as Brukinsa) developed by Chinese biotech BeiGene won accelerated approval for adult patients with mantle cell lymphoma (MCL) — a typically aggressive and rare form of blood cancer.

This was followed, almost immediately, by news that the agency approved a new treatment for partial-onset seizures in adults. The drug — cenobamate — will be sold as Xcopri by SK Life Science, the US subsidiary of the massive Korean conglomerate SK Group. Xcopri will provide another therapeutic option to patients with epilepsy. This approval marked the first time a Korean company independently took a compound from the stage of discovery to FDA approval.

Xcopri is SK Life Science’s first commercial product and wraps a decade of quiet growth from SK Life Science, which has been developing its presence in the pharmaceutical industry. 

The company plans to hire a salesforce to support the drug’s launch, which is expected to generate over US$ 1.5 billion in sales. 

Sanofi’s controversial dengue vaccine, cast-off drug get approved 

The FDA very narrowly approved Sanofi’s controversial dengue vaccine — Dengvaxia. The vaccine, which took 20 years to develop, got embroiled in a controversy when in 2017, Sanofi disclosed that Dengvaxia could increase the risk of severe dengue in children who had never been exposed to the virus. 

In Philippines, 800,000 school-age children had been vaccinated against dengue, the world’s fastest growing infectious disease.

The year 2019 had begun with Bristol-Myers Squibb (BMS) and Celgene announcing their US$ 74 billion mega-merger. As the firms worked through the year to get the merger approved, the FDA signed off on Celgene’s myelofibrosis drug Inrebic (fedratinib).

Inrebic — the once-daily oral drug — is the first new treatment in nearly a decade approved to treat myelofibrosis. The story of Inrebic’s rise from the dead is a major success for Celgene, which picked up the drug as a part of a US$ 1.1 billion acquisition of Impact Biomedicines in January 2018. Before that, Inrebic had passed through multiple hands.

Fedratinib was a flop for Sanofi as patients began to develop a dangerous neurological condition tied to vitamin B deficiency called Wernicke’s encephalopathy. As a result, the FDA put a clinical hold on it in 2013 and Sanofi ultimately shelved the effort.

Executives at BMS have said the drug is among a group of potential blockbusters at Celgene.

Boehringer Ingelheim is to manufacture the first biologic drug approved in China using a new regulatory system, designed to strengthen the country’s domestic pharma infrastructure.

The German pharma successfully applied the Marketing Authorization Holder (MAH) system under China’s revised drug laws, allowing it to manufacture a biologic drug for partner BeiGene under multinational arrangements.

Chinese pharma BeiGene has developed tislelizumab, a PD-1 immune checkpoint inhibitor that is under development for a range of solid tumours.

In a statement Boehringer also noted that this is the first innovative biologic drug commissioned under the new model in China.

John Oyler, chairman and CEO of BeiGene, said the collaboration between BeiGene and Boehringer Ingelheim Biopharmaceuticals China demonstrates that the country’s MAH trial project can be successfully applied in order to advance the life sciences industry in China.

“This is an important milestone, not only to ensure the supply of medicines for patients in China, but also for the rapidly emerging Chinese biopharmaceutical research and development landscape,” he said.

In 2014, the Chinese government began reforming regulation to enable third parties to manufacture biopharmaceutical products for marketing authorisation holders.

Tislelizumab became the pilot project in 2015 and is now the first biologic approved under the new regulatory setup.

It is in pivotal phase 3 and phase 2 trials, including a pivotal trial in relapsed/refractory Hodgkin’s lymphoma.

It is also being tested in non-small cell lung cancer, hepatocellular carcinoma, and oesophageal squamous cell carcinoma, as well as two global phase 2 trials in patients with previously treated hepatocellular carcinoma or with R/R mature T- and NK-cell lymphomas, and an additional pivotal phase 2 trial in China in urothelial cancer.

Tislelizumab is also in development in combination with chemotherapy in first-line gastric cancer and first-line oesophageal cancer, and in China in first-line non-small cell lung cancer.

It is also being developed in combination with Beigene’s tyrosine kinase inhibitor, Brukinsa (zanubrutinib), which is a challenger to AbbVie/Johnson & Johnson’s blood cancer drug Imbruvica and has already been approved in the US for mantle cell lymphoma.

With the books being closed on 2019, it’s time to prognosticate about what the next year will hold in the biotech and pharmaceutical industry. And, to paraphrase a famous song from the 1980s, grab a set of shades, because the future looks bright.

BioSpace spoke to leaders from various corners of the industry who provided their insights into what the coming year is likely to hold for their particular sphere. Below are predictions for three spheres within the industry for the coming year.

Oncology

Immuno-oncology has been one of the research cornerstones in cancer research and that will continue. However, in 2020, traditional targeted therapies will continue to be of importance. Stephen Gately, chief executive officer of TD2 (Translational Drug Development), an oncology-focused contract research organization, said there are still roles for traditional targeted therapeutics for specific indications. Following Merck’s acquisition of ArQule, Inc. and its kinase inhibitor discovery and development programs for cancer treatment, predicted the industry might see a refocus on understanding cancer and how it can respond to different drugs. He said Merck would not have spent $2.7 billion for ArQule’s Phase II Bruton’s tyrosine kinase inhibitor ARQ 531 if there was not a potential upside for the therapy, particularly as a treatment for B-cell cancers. If trial protocols are well-established and there are stricter guidelines for patient selections, Gately said the use of certain inhibitors are vastly superior to immunotherapies in development.

When it comes to clinical trials, Gately predicted there could be a change in how those are organized. He said the goal for companies is to attempt to get their assets into trials as quickly as possible but noted there is a heated and competitive battle for trial sites. As companies attempt to elbow their way to the front of the line, Gately said it is possible that the industry will begin to see more opportunities for economic incentives used to convince the clinical trial sites to take up trials sooner. If that is established, Gately said it will become a game-changer.

Cell and Gene Therapies

This past year saw the approval of gene therapies that can address devastating diseases. And that is likely to continue in 2020. But, safety will still be paramount. Ena Cratsenburg, chief business officer of Ginkgo Bioworks, said over the course of the next year, we will see drug developers use synthetic biology to improve their ability to develop therapeutics with unprecedented levels of activity and control. “In 2020, synbio companies will solidify their positions as key enablers in bringing the most innovative medicines to consumers,” Cratsenburg said.

Mark Sawicki, chief commercial officer at Cryoport, developer of the first shippers certified for the delivery of human advanced therapies, said that as the cell and gene therapies continue to show their importance for treating new diseases, 2020 will usher in a record year for biologics license applications and marketing authorization applications. Sawicki suggested that over the course of the coming year, eight or nine companies will file such applications to gain regulatory approval of their products. In addition to the high number of filing companies, he suggested that there will be multiple new products launching with three or four generating revenues in 2020. By 2023, Sawicki predicts there will be at least 22 commercial launches in the space.

Jason Steiner, vice president of business development and strategy at gene editing company Synthego, said in 2020, the gap between the pace of innovation in the cell and gene therapy landscape and the infrastructure required for commercialization and deployment will continue to widen before it converges again. Steiner said the development of engineered cell therapies will continue to “move toward non-viral approaches” in order to boost engineering sophistication while decreasing the time and cost of traditional engineering. He added that manufacturing consideration of cell and gene therapies will continue to move farther upstream. They will be “more tightly integrated with early development to avoid commercialization bottlenecks that are being caused by a rapid increase in the pace of development and regulatory approval cycles.”

Real World Evidence

The importance of Real World Evidence (RWE) will continue to grow in 2020. Jane Reed, director of life sciences at U.K.-based Linguamatics, said the industry will continue to harness RWE in its drug development programs. RWE was not a topic of conversation in the industry 10 years ago, Reed said, but now there is significant value seen from what RWE brings to the table. RWE relates to the collection of information about a drug’s safety and efficacy outside the structure of a clinical trial. The data can be collected from a variety of sources, including electronic health records, wearable devices, lab tests and more. The pharma industry will need to not only continue to collect the data, but learn to use it in a way that improves the outcomes of the patients it serves. Reed said the industry has to show they are listening to the patient community and be flexible. From there, the industry will need to feed that patient-driven data into drug discovery efforts. To boost the collection of RWE, Reed said the use of targeted social media research will also increase the amount of data that companies have at their fingertips. As companies within the pharmaceutical industry continue to dig into the depths of disease states, particularly those of rare diseases, Reed said the use of RWE will prove to be increasingly important.