BELS Brief Clips: January 2020

Following is a sampling of UK health and life science news from January 2020 curated by BELS and disseminated to members of our global community

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New target identified for repairing the heart after heart attack    An immune cell is shown for the first time to be involved in creating the scar that repairs the heart after damage. Billions of cardiac muscle cells are lost during a heart attack. The human heart cannot replenish these lost cells, so the default mechanism of repair is to form a cardiac scar. While this scar works well initially to avoid ventricular rupture, the scar is permanent, so it will eventually lead to heart failure and the heart will not be able to pump as efficiently as before the damage caused by heart attack. Zebrafish, a freshwater fish native to South Asia, is known to be able to fully regenerate its heart after damage due to the formation of a temporary scar as new cardiac muscle cells are formed. Professor Paul Riley and his team at the University of Oxford have been striving to understand and compare the composition of the cardiac scar in different animals as part of ongoing efforts to investigate whether it can be modulated to become a more transient scar like that of the zebrafish, and therefore potentially avoid heart failure in heart attack patients. (Oxford University  January 30, 2020) 

Boom Year for UK Biotech, Despite Brexit Concerns   Although biotech financing in the UK took a hit last year in large part thanks to uncertainty around Brexit, 2019 was still the British industry’s third-best year on record. £1.3 billion was raised by UK-based biotech companies. £679 million was raised in VC, with £64 million raised in IPOs and £596 million in all other public financings. The sector has seen five consecutive years of raising over £1 billion of investment. In VC financing, three UK biotechs managed to raise above £50 million, down from eight such deals in 2018. The UK remains the leading cluster in Europe for amounts raised through venture capital, accounting for 26% of the continent’s total.  (PharmaBoardRoom  January 29, 2020)

Information hubs and the beginning of a data-driven future   Will 2020 be the year the UK harnesses the potential of its health data, as seven new national ‘data hubs’ start to break down data silos and embrace real world evidence?  The NHS may be lacking in resources, but when it comes to data, it is the richest organisation in the world. Yet all too often the potential this affords us is thwarted by siloed working and fragmented sources. Liz Dobson, chief executive officer of the IBD Registry, which has been collecting longitudinal in-clinic real-world data since 2015 in order to improve understanding of IBD, especially in the care and treatment of patients, says: “We’ve got a wealth of rich data in the NHS, but they are held in different datasets, and these can’t easily talk to each other.  (PharmaPhorum  January 28, 2020)

Scientists Say Genetically Engineered Moths Could Save Crops   Scientists have conducted the first successful field release experiment of a genetically modified moth that could fundamentally change how crops are protected from pests. Scientists at Oxitec (Oxford) have created a new type of diamondback moth – a species often referred to as the “cabbage moth” due to their gluttonous and uninvited consumption of a number of crops such as broccoli and cabbage, that has been genetically modified to help control population numbers in any given field.  (Courthouse News  January 28, 2020)

Breast cancer approval in tow, AstraZeneca, Daiichi armed antibody scores in key gastric cancer study AstraZeneca kicked off Monday with a flurry of good news. Apart from unveiling positive results on its stroke trial testing its clot-fighter Brilinta and welcoming its experimental IL-23 inhibitor brazikumab back from Allergan—the British drugmaker also disclosed some upbeat gastric cancer data on its HER2-positive oncology therapy it is collaborating on with Daiichi Sankyo. Buoyed by the performance of its oncology drugs, last March AZ chief executive Pascal Soriot bet big to partner with Daiichi on the cancer drug, with $1.35 billion upfront in a deal worth up to roughly $7 billion. Roughly 8 months later, as 2019 drew to a close, the FDA swiftly approved the drug — trastuzumab deruxtecan — for use in breast cancer, months ahead of the expected decision date.  (Endpoint News  January 28, 2019)

Drug improves symptoms of autism by targeting brain’s chemical messengers   Bumetanide – a prescription drug for oedema (the build-up of fluid in the body) – improves some of the symptoms in young children with autism spectrum disorders and has no significant side effects, confirms a new study from researchers in China and at the University of Cambridge . The study demonstrates for the first time that the drug improves the symptoms by decreasing the ratio of the GABA to glutamate in the brain. GABA and glutamate are both neurotransmitters – chemical messengers that help nerve cells in the brain communicate.  (University of Cambridge  January 27, 2020)

AstraZeneca makes case for use of blood thinner Brilinta in stroke patients AstraZeneca ’s extravagant projections for its clot fighter Brilinta may have fizzled in the face of underwhelming trial data — but a new pivotal study is set to expand its use substantially. When taken in conjunction with aspirin, AZ said the drug induced a statistically significant reduction in the risk of the primary composite endpoint of stroke and death, compared to aspirin alone, in 11,000 patients that have suffered minor acute ischemic stroke or a high-risk transient ischemic attack.   (Endpoints  January 27, 2020)

GSK hands TB vaccine to Gates Foundation’s nonprofit biotech GlaxoSmithKline has licensed a tuberculosis vaccine to the Bill & Melinda Gates Medical Research Institute. The deal sets the stage for a push to build on recent phase 2b data and make the vaccine available in low-income countries where TB is prevalent. The vaccine, M72/AS01E, is made up of an immunogenic fusion protein based on two TB antigens. GSK combined that protein with the adjuvant found in its shingles prophylactic Shingrix to create a subunit vaccine. Last year, a phase 2b trial linked the vaccine to 50% protection against progression to active pulmonary tuberculosis for three years in adults infected with the mycobacterium.  (FierceBiotech. January 27, 2020)

UK Newborn Trial to Assess PCR Test for Antibiotic-Induced Hearing Loss   The NIHR Manchester Biomedical Research Centre has launched a clinical trial for a point-of-care PCR-based pharmacogenetic test to predict hearing loss from antibiotic treatment in newborns. The test uses a cheek swab and delivers a result in less than 30 minutes. It is designed to detect a genetic variant that is present in 1 in 500 individuals. Individuals carrying the mutation suffer from severe hearing loss or deafness if they are treated with the antibiotic gentamicin. The six-month trial will involve 1,000 newborns from two intensive care units, at St Mary’s Hospital Manchester UniversityNHS Foundation Trust and at Liverpool Women’s Hospital , Liverpool Women’s NHS Foundation Trust . The goal is to test all newborns within 10 minutes of admission.  (GenomeWeb January 27, 2020)

Smith & Nephew picks up Tusker Medical and its in-office ear tube treatment   Just weeks after it received FDA approval for its in-office system for placing eardrum tubes and treating ear infections, Tusker Medical has been picked up by Smith & Nephew (London) for an undisclosed sum. The device maker described the acquisition as part of its strategy to invest in technologies that address unmet clinical needs. Tusker’s Tula system, previously granted an FDA breakthrough device designation, is aimed at the 700,000 children who undergo surgery annually for recurrent ear infections or persistent fluid in the middle ear. Typically, tympanostomy tubes are placed within the ear under general anesthesia in an operating room. The automated Tula device, meanwhile, is designed to be used within a physician’s office, with a local anesthetic, as the child sits up.  (FierceBiotech Janaury 24, 2020)

Oxford launches North American start-up scheme that will create 4,000 UK jobs   An innovative university research commercialisation initiative led by Saïd Business School at the University of Oxford is set to create nearly 4,000 jobs across the UK in artificial intelligence, healthy ageing, alternative energy, and quantum technologies. Funding from Research England , part of UK Research and Innovation, will help Creative Destruction Lab (Oxford) support 225 high-potential early-stage companies from all over the UK, with aims to raise £225m in capital, and generate £900m in equity value. The scheme will help transform some of the UK’s pioneering research ideas into viable products, improving the UK’s entrepreneurial and finance ecosystem, and build on international best practice. Start-ups involved will benefit from input from technology, finance and science mentors – including Canadian and Silicon Valley entrepreneurs and investors. These include Google’s former Chief Financial Officer, Patrick Pichette; Elizabeth Caley, Chief of Staff at the Chan Zuckerberg Initiative; and Shivon Zilis, Project Director at Elon Musk’s Neuralink.  (UKRI  January 24, 2020)

Summit Therapeutics loses R&D president after BARDA funding boost   What a difference a day makes in biotech: Yesterday, antibiotics player Summit Therapeutics (Oxford) was riding high after an $8.8 million cash infusion from the US Biomedical Advanced Research and Development Authority (BARDA). The boost comes as it is gearing up for a new clinical trial of its lead asset, an antibiotic for Clostridium difficile infection, as one of only a handful of players in this much needed, but much neglected (and troubled) area. But off the back of this, the company gave a very brief update today saying David Roblin has “resigned as Chief Operating Officer, Chief Medical Officer and President of R&D” as of the end of the month. No reason or successor has been named.  (FierceBiotech January 24, 2020)

“Skeleton Key” T-Cell Receptor Works across Multiple Cancer Types   Anticancer T-cell therapy has a lot in common with lock picking, a painstaking business that often ends in frustration. Every cancer, like every lock, presents a unique challenge. But what if there were such a thing as an anticancer T cell that worked as though it carried a skeleton key? Imagine it: a T cell equipped with a T-cell receptor (TCR) that bypasses obstructions and engages a shared mechanism, that is, a mechanism common to the locks presented by different kinds of cancer. A skeleton key-like TCR hasn’t been found on ordinary T cells. However, one has been discovered on the keychain of another kind of T cell, the mucosal-associated invariant T cell. Unlike ordinary anticancer T cells, which fumble over molecular locks involving the human leukocyte antigen system, MAIT cells have a TCR that works with another cell surface display system, the monomorphic MHC class I-related protein, MR1. The new discovery, which comes from scientists based at Cardiff University (Wales), raises the possibility of developing pan-cancer, pan-population cancer immunotherapies.  (GEN  January 23, 2020)

Gut bacteria linked to personality    Sociable people have a higher abundance of certain types of gut bacteria and also more diverse bacteria, an Oxford University study has found.  Dr Katerina Johnson of Oxford University’s Department of Experimental Psychology has been researching the science of that ‘gut feeling’ – the relationship between the bacteria living in the gut (the gut microbiome) and behavioural traits. In a large human study she found that both gut microbiome composition and diversity were related to differences in personality, including sociability and neuroticism. (Oxford University  January 23, 2020)

Mosquito-borne diseases could be prevented by skin cream   A skin cream used to treat warts and skin cancer could help protect people against viral diseases such as Zika and dengue, according to new research. Scientists at the University of Leeds studied four types of virus transmitted by mosquitoes and found that applying a cream within an hour of a bite caused a rapid activation of the skin’s immune response that fights any potential viral threat.   (University of Leeds  January 22, 2020)

Autolus Announces Pricing of Public Offering Autolus Therapeutics (London), a clinical-stage company developing next-generation programmed T cell therapies, announced the pricing of an underwritten public offering in the US of 7,250,000 American Depositary Shares (“ADSs”) representing 7,250,000 ordinary shares at a public offering price of $11.00 per ADS, for total gross proceeds of approximately $80.0 million. All ADSs sold in the offering were offered by Autolus.  (Press release January 22, 2020)

AstraZeneca aims to go carbon free by 2025—and it’ll spend $1B to do it   For a pharma company selling respiratory drugs, climate change would probably mean more patients and increased sales. But AstraZeneca has committed to contributing its fair share to stop that trend. Just as the topic of climate change takes center stage at the World Economic Forum in Davos, Switzerland, AZ unveiled its Ambition Zero Carbon program, which aims to achieve zero carbon emissions from its global operations by 2025. To do that, it will invest up to $1 billion, part of which will go toward developing next-generation respiratory inhalers that have almost no negative impact on global warming, the company said.  (FiercePharma  January 22, 2020)

Vomiting bumblebees show that sweeter is not necessarily better   Animal pollinators support the production of three-quarters of the world’s food crops, and many flowers produce nectar to reward the pollinators. A new University of Cambridge study using bumblebees has found that the sweetest nectar is not necessarily the best: too much sugar slows down the bees. The results will inform breeding efforts to make crops more attractive to pollinators, boosting yields to feed our growing global population.  (University of Cambridge  January 22, 2020)

Magnetised molecules used to monitor breast cancer   A new type of scan that involves magnetising molecules allows doctors to see in real-time which regions of a breast tumour are active, according to research at the University of Cambridge . This is the first time researchers have demonstrated that this scanning technique, called carbon-13 hyperpolarised imaging, can be used to monitor breast cancer.  (University of Cambridge  January 22, 2020)

PredictImmune Inks Multinational Commercialization Pact for IBD Test PredictImmune (Cambridge UK) has signed an agreement for promotion and distribution of its PredictSure IBD test in the UK, Ireland, the Middle East, and the Balkans with Cambridge Clinical Laboratories (Cambridge UK). PredictSure IBD is a PCR-based test designed to analyze the expression of 17 genes in whole blood in order to differentiate mild and aggressive forms of Crohn’s disease and ulcerative colitis and to guide treatment options.  (GenomeWeb  January 21, 2020)

Glaucoma Polygenic Risk Score Emerges From Multi-Trait Analysis in UK Biobank Participants   An international team of researchers has developed a polygenic risk score for glaucoma that could help identify people who are at risk of developing the condition at an earlier age and may benefit from earlier screening and treatment. Once vision is gone, it cannot be restored, meaning that early detection is important to prevent disease progression. For their study, the researchers characterized the optic nerves from 67,040 UK Biobank participants, as optic nerve degeneration is a sign of glaucoma, and used those results and intraocular pressure data, another sign of glaucoma, from 103,914 UK Biobank participants to conduct two GWAS. Through these, they identified 49 novel genetic loci associated with glaucoma.  (GenomeWeb  January 21, 2020)

UK biotech PhoreMost to work with Otsuka on gene therapy projects   Building on an R&D tie-up with an Indian stem cell institution last year, PhoreMost  (Cambridge UK) has begun a collaboration with Japan’s Otsuka working on several gene therapy projects. PhoreMost said it will use its next-generation phenotypic screening platform Siteseeker to identify novel targets for Otsuka’s therapeutics discovery programmes. Novel targets identified will be further validated and characterised by Otsuka as part of its internal development pipeline, with an initial focus on gene therapy applications of identified targets. Siteseeker looks at different protein shapes to find functionally active peptides that can be targeted by new therapies. The technology looks at the entire proteome – all of the proteins expressed – in a live cell environment looking for druggable targets for a chosen disease.  (PharmaPhorum  January 21, 2020)

GSK bets on tech to help boost lagging R&D as it looks to start 23andMe trial   As the dust settles on another busy and ruinously expensive four days at the annual JP Morgan Healthcare Conference in San Francisco, GlaxoSmithKline ’s CEO Emma Walmsley took some time out on the sidelines to talk R&D. Since taking over from Sir Andrew Witty a few years back and proceeding to shake up the pharma’s R&D focus and leadership, what’s become clear is the new-look GSK is making its bed with tech companies and adhering to a tech outlook. One of the new appointments has been Hal Barron, head of R&D at GSK and alumnus of Roche and Calico, who has remained based in San Francisco and not in GSK’s UK headquarters, with the plan being that some of that embedded tech culture in the region can take root within the Big Pharma.  (FierceBiotech  January 21, 2020)

Multi-Disease Diagnostic Platform Project Receives €22.5M in EU Funding   An international team of investigators has received a five-year, €22.5 million ($24.9 million) grant for a project to develop a test that may be able to diagnose multiple conditions, including pneumonia, tuberculosis, sepsis, and others with one blood sample and in less than two hours, the Imperial College of London announced. The project is being funded by the EU’s Horizon 2020 Research and Innovation Actions and is being spearheaded by Michael Levin, a professor of infectious diseases at Imperial College. The project’s goal is to bring the test to hospitals across Europe. Rather than performing multiple blood tests and scans to diagnose a condition, the project called Diagnosis and Management of Febrile Illness Using RNA Personalised Molecular Signature Diagnosis, or DIAMONDS, seeks to develop a platform that will allow a diagnosis to be done with just one blood sample. Other diseases being targeted for the test include inflammatory and immune diseases.  (GenomeWeb  January 20, 2020)

Future prospects for an Innovative Medicines Fund for England   On the 12 December 2019, the UK general public voted for a further five years of Conservative government. Whilst a big part of the Conservative campaign was ‘to get Brexit done’, other promises affecting the industry were made too. One of these was to extend the Cancer Drugs Fund (CDF) into an ‘Innovative Medicines Fund’. The CDF was an election promise made by David Cameron back in 2010. Boris Johnson took a leaf out of the same play book and has promised a new Innovative Medicines Fund. The promise is that “doctors can use the most advanced, life-saving treatments for conditions such as cancer or autoimmune diseases, or for children with other rare diseases,” as set out in the Conservative Manifesto. The Innovative Medicines Fund could add £160 million to the CDF’s current pot of £340 million. (PharmaPhorum  January 17, 2020)

JPM: Glaxo’s cancer unit, in commercial ‘building mode,’ gets choosy with M&A   It was just about a year ago when GlaxoSmithKline sealed its deal for Tesaro, bringing on already-marketed PARP inhibitor Zejula. But don’t expect the company to spring for more commercial-stage oncology products anytime soon. While “we are not excluding the opportunity” for products on the market, it “has to be a good fit for the situation”—and GSK is being particular about the situation it’s currently in”, said Axel Hoos, the company’s SVP of Oncology R&D.  (FiercePharma  January 17, 2020)

UK accounts for over 12% of global cell and gene therapy clinical trials   The UK and the NHS are providing the right environment and infrastructure to allow innovative cell and gene therapies to reach patients. International companies recognise the appeal of the UK cell and gene therapy ecosystem and are sponsoring the majority of the UK commercial clinical trials which account for 77% of the total 127 ongoing trials, as reported in the UK Cell & Gene Therapy Catapult (Stevenage) 2019 UK Advanced Therapy Medicinal Products clinical trials database , a 45% increase on 2018, and accounting for 12% of the total global cell and gene therapy clinical studies.  (CT Catapult  January 15, 2020)

New heart disease drug to be made available to NHS patients through ground-breaking collaboration   Up to 30,000 lives could be saved over the next decade thanks to a proposed pioneering government collaboration with pharmaceutical company Novartis to tackle heart disease – a leading cause of death in the UK. In early January, Novartis completed the acquisition of The Medicines Company which developed iIclisiran, a bi-annual injection, that is expected to be filed for approval as a preventative add-on treatment to statins for patients who have already been diagnosed with cardiovascular disease later this year. It will also be put through the NICE approval programme at the earliest opportunity possible and NHS England will agree a population-level commercial arrangement with the company to make it widely available to patients as soon as 2021. The agreement will make a significant contribution towards meeting the NHS long term plan commitment to preventing 150,000 cardiovascular deaths over 10 years. UK leaders believe that this as a really exciting announcement that changes the way we bring new medicines to patients earlier and also will propel the NHS and the UK as a world leader in this sort of clinical research. Recent Phase III trials have shown inclisiran can halve bad cholesterol in just two weeks. Inclisiran will be studied in UK patients as part of a large-scale NHS clinical trial expected to start later this year. It will also be put through NICE’s approval programme at the earliest opportunity possible and NHS England will agree a population-level commercial arrangement with the company to make it widely available to patients as soon as 2021 through a population-level agreement – pioneering a game-changing approach to reducing the risk of heart disease. The collaboration between Novartis, NHS England, the National Institute for Health Research and the Nuffield Department of Population Health at Oxford University represents an innovative approach to tackling major public health issues and positions the UK as a world-leading destination to develop revolutionary medicines. (Oxford University  January 14, 2020)

Mereo BioPharma Announces Additional Positive Data from Phase 2b ASTEROID Study of Setrusumab in Adults with Osteogenesis Imperfecta and Provides Update on Regulatory Progress Mereo BioPharma (London) announced additional endpoint data from its Phase 2b dose-ranging ASTEROID clinical study of setrusumab (BPS-804), an anti-sclerostin antibody, in adults with Type I, III or IV osteogenesis imperfecta (OI). These additional prespecified endpoint data build upon its 12-month topline data from the ASTEROID study announced in November 2019, which demonstrated a dose-dependent, statistically significant bone building effect of setrusumab at multiple anatomical sites in adult OI patients (irrespective of OI subtype). OI is a rare disorder characterized by fragile bones and reduced bone mass with no approved treatments.  (Press release  January 14, 2020)

Astellas to work with Adaptimmune to develop ‘off the shelf’ T-cell therapies   Astellas is to work with Adaptimmune Therapeutics (Oxford) to develop allogeneic ‘off-the-shelf’ T-cell therapies for cancer. The Japanese pharma said its wholly-owned subsidiary Universal Cells will work with Adaptimmune on a co-development and co-marketing agreement. The deal could be worth up to $897.5 million for Adaptimmune, including an upfront payment of $50 million with other payments when certain goals are met. News of the deal emerged as Adaptimmune’s share price skyrocketed after it announced at the JP Morgan conference that its SPEAR T-cell platform delivered responses in four solid tumour types – liver, gastro-oesophageal junction, head and neck, as well as melanoma.  (PharmaPhorum   January 14, 2020)

Rutgers Licenses Base Editing Tech to Horizon Discovery Horizon Discovery (Cambridge UK) has exercised its option to exclusively license a novel base editing technology from Rutgers University for use in therapeutic, diagnostic, and service applications. The base editing platform — which modifies genes by creating single point mutations in DNA without making double-stranded breaks — has potential applications in the development of new cell therapies.  (GenomeWeb  January 14, 2020)

Genomics England, Illumina join forces on whole genome sequencing Genomics England and Illumina announced a new agreement to sequence up to 300,000 whole genome equivalents over the next five years, with an option to increase to 500,000. The partnership comes off the back of the success of the 100,000 Genomes Project in December 2018, which established protocols for consent from patients with rare genetic diseases and cancer, tissue sample requirements, standardised DNA sequencing, data analysis and reporting. As a part of the collaboration samples from NHS patients in England with known rare diseases and cancer types will be eligible for whole genome sequencing, to support diagnosis, inform and improve treatment pathways, and ultimately improve outcomes, with the expectation that the number of eligible clinical indications will expand over time.  (PharmaTimes  January 14, 2020)

NICE backs Medtronic’s AF monitor in post-stroke patients   England’s NHS could prevent hundreds of strokes of unknown cause with a new implantable monitor that transmits data to doctors remotely, which is recommended for funding following draft guidance from NICE . The Reveal LINQ monitor developed by Medtronic identifies more people who have atrial fibrillation after a cryptogenic stroke (CS) – with no identified cause – or transient ischaemic attack (TIA). One-third of the size of an AAA battery, it is implanted under the skin in the chest usually under local anaesthetic, to track a heart’s activity over long periods of time, transmitting data back to the patient’s doctor via a mobile phone network.  (PharmaPhorum  January 14, 2020)

Sosei Heptares: Significant Progress by Orexia and Inexia With Orexin Agonist Program Triggers Next Tranche of Funding From Medicxi   Significant scientific progress at Sosei Heptares ’ (Cambridge UK) spin-off companies Orexia (London) and Inexia (London), targeting neurological diseases, has triggered the next tranche of funding from venture capital firm Medicxi (London) under its €40 million commitment. Sosei and Medicxi, which specializes in financing asset-centric companies, created Orexia and Inexia in 2019 to develop novel therapies based on positive modulators of the G protein-coupled receptors (GPCRs) Orexin OX1 and OX2 for neurological diseases, including narcolepsy.  (Press release  January 13, 2020)

ViiV submits fostemsavir MAA to EMA   GlaxoSmithKilne’s HIV company ViiV Healthcare (Brentford) has submitted a marketing authorisation application to the European Medicines Agency seeking approval of fostemsavir. The drug, an investigational, first-in-class attachment inhibitor, is seeking indication for the treatment of HIV-1 infection when used in combination with other antiretrovirals. The company said that the submission is for the treatment of adults with multidrug resistant HIV-1 infection for whom it is otherwise not possible to construct a suppressive antiviral regimen due to resistance, intolerance or safety considerations.  (PharmaTimes  January 13, 2020)

Elasmogen secures £2m to support next gen biologics Aberdeen University spin-out Elasmogen (Aberdeen) has secured follow-on equity investment from Deepbridge Capital (Chester) totaling £2 million, taking the company’s total investment in Elasmogen to £3.15 million. The funding will be used to accelerate the development of the company’s next generation biologics for “site-specific delivery and treatment of diseases refractory to existing drug therapies.” Elasmogen’s proprietary, humanised versions of drugs – also known as SoloMER biologics – are originally based on a protein found in nature called a VNAR. VNARs are small, antibody-like proteins and key components of a shark’s immune system.  (PharmaTimes  January 13, 2020)

Cardiff researchers identify new genetic link to schizophrenia Cardiff University (Wales) researchers have identified new mutations in a gene that provides novel insights into the biological causes of schizophrenia. The study looked specifically at previously identified high-risk genes that can predispose people to neuropsychiatric disorders such as autism and developmental delay. The researchers found that one in particular, SLC6A1, had significantly more new mutations than expected by chance. The mutations were very rare, found in around three in 3,000 individuals diagnosed with schizophrenia. The importance lies not in how common the mutations are but the area of biology highlighted, namely one of the main chemical transmitters of the central nervous system, known as GABA. This points towards alterations in how brain cells communicate as a potential cause of schizophrenia. Also, these mutations appear to substantially increase the risk of developing the condition, suggesting that treatments developed to target this system could potentially have large therapeutic effects.  (Cardiff University  January 13, 2020)

Bayer, Exscientia to explore AI in drug discovery   A new collaboration between Bayer and Exscientia (Oxford) is set to accelerate the discovery of small molecule drugs, focused on cardiovascular disease and oncology. The companies will use artificial intelligence to further the development of drug candidates for three of Bayer’s projects in the areas of cardiovascular disease and oncology. Under the terms of the deal, Exscientia will begin by working on three projects with targets agreed between both parties, with eligibility to receive up to £203 million, including “upfront and research payments, near term and clinical milestones.”  (PharmaTimes  January 10, 2020)

Emergex Vaccines Secures €10M to Advance Synthetic Vaccine Development Emergex Vaccines (Abingdon) has raised over €9.9M in a Series A funding round, which will allow it to progress the development of vaccines against diseases such as dengue, influenza, and Ebola to phase I clinical trials. Emergex specializes in the development of ‘set-point’ vaccines, which the company describes as vaccines that convert potentially lethal infections into mild infections that stimulate the body to become immune to the disease in question.  (Labiotech  January 9, 2020)

Whitehall needs more scientists to compete with China: chief adviser     Britain’s civil service is suffering from a serious lack of scientific talent that threatens its ability to compete with nations such as China. That is the stark view of the government’s own chief scientific adviser, Sir Patrick Vallance. In a rare interview, Vallance said a dearth of scientists and engineers in government posed major problems for the nation as it attempts to deal with the threats of climate change, an ageing population and tightened national security. Vallance is backing a major push inside the civil service to boost numbers of science and engineering graduates. “This is the one thing in my tenure that I want to get fixed,” he said.  (The Guardian  January 12, 2020)

JPM: Novartis teams up with NHS England in ‘world-first’ access deal for PCSK9 hopeful inclisiran   In a multibillion-dollar bet on the cholesterol drug candidate inclisiran, Novartis late last year picked up the Medicines Company in one of 2019’s largest biopharma M&A deals. Now, it’s unveiled a partnership with the British government to make the as-yet-unapproved med widely available for high-risk cardiovascular disease patients. At the JP Morgan Healthcare Conference, Novartis CEO Vas Narasimhan and British health officials said they’d agreed to provide the drug—after it’s cleared by regulators and reviewed by cost watchdog NICE—to high-risk patients. Negotiated and signed with MedCo before Novartis bought the company, the “population-level agreement” would be a world-first, officials said. As a novel approach, it could inspire similar deals in other markets. And it could pose a threat to rival PCSK9 drugs Repatha, from Amgen, and Praluent, from Regeneron and Sanofi—both of which have never lived up to their initial hype. Aside from the access deal, Novartis and England’s NHS plan to study the drug in a large-scale clinical trial in primary prevention patients with atherosclerotic cardiovascular disease. The deal “demonstrates how this medicine in particular can transform population health like few other drugs could in the past,” Narasimhan said.  (FiercePharma  January 13, 2020)

Horizon Discovery, Mammoth Biosciences Sign Second CRISPR Tools Development Agreement Horizon Discovery (Cambridge UK) has signed a collaboration and license agreement with Mammoth Biosciences to develop a new generation of genetically engineered CHO cells for the production of biotherapeutics such as therapeutic antibodies. Horizon’s bioproduction business unit currently offers a CHO cell line and customized services to the biopharmaceutical industry to facilitate the development and identification of best performing gene-edited cell lines. Under the terms of the agreement with Mammoth, Horizon will have access to Mammoth’s CRISPR platform, and the companies will collaborate to optimize certain CRISPR tools to rapidly develop proprietary CHO cell lines in which selected genes will be knocked out to improve performance parameters such as productivity or purification.  (GenomeWeb  January 13, 2020)

Redx Pharma nominates RXC007, a novel, orally active ROCK2 inhibitor as next drug development candidate in fibrosis Redx Pharma (Cheshire) announced the nomination of RXC007 as its next drug development candidate for the treatment of fibrosis. RXC007 is a novel and selective Rho Associated Coiled-Coil Containing Protein Kinase 2 (ROCK2) inhibitor, an enzyme which sits at a nodal point in cell signalling pathways believed to be central to fibrosis. Fibrosis is a key pathogenic factor in multiple diseases with high unmet medical need. ROCK2 is therefore an important emerging drug target and RXC007 will be developed as a best-in-class drug to target several fibrotic diseases. These include the orphan disease, idiopathic pulmonary fibrosis, a severe and life-threatening chronic lung condition with very poor prognosis and limited treatment options; non-alcoholic steatohepatitis (NASH), an inflammatory and fibrotic disease of the liver; and diabetic nephropathy, a serious diabetic kidney disease.  (Press release  January 9, 2020)

Heart Failure GWAS Leads to Risk Loci, Causal Clues   A new study has unearthed a dozen variants with apparent ties to heart failure risk, including SNPs at 10 sites not linked to the condition in the past. Results from the genome-wide association meta-analysis are expected to “extend our knowledge of the pathways underlying [heart failure] and may inform new therapeutic strategies,” a researcher affiliated with the University College London and St Bartholomew’s Hospital Bart’s Heart Centre and his colleagues wrote. Researchers from the UK, Australia, the US, and elsewhere brought together data for almost 1 million individuals with or without heart failure, focusing in on 12 variants at 11 loci that coincided with the heart failure cases. They also attempted to tease out variants associated with underlying heart failure causes — from atrial fibrillation to coronary artery disease — which highlighted risk loci in and around genes from pathways contributing to cardiac development and other processes.  (GenomeWeb  January 9, 2020)

AstraZeneca and MiNA Partner to Tackle saRNAs for Metabolic Diseases   AstraZeneca has forged a relationships with RNA activation therapeutics pioneer  MiNA Therapeutics  (London) to tackle metabolic diseases with small activating RNA. MiNA said the collaboration will combine its “expertise in the discovery and development of saRNA therapeutics” with AstraZeneca’s experience in “identifying and bringing breakthrough treatments to patients with metabolic diseases.”  (Biospace  January 7, 2020)

Detailed genetic study provides most comprehensive map of risk to date of breast cancer risk   A major international study of the genetics of breast cancer has identified more than 350 DNA ‘errors’ that increase an individual’s risk of developing the disease. The scientists involved say these errors may influence as many as 190 genes.  (University of Cambridge  January 7, 2020)

Merck dives deeper into KRAS, teaming up with Taiho, Astex to develop small molecule drugs   As the race — led by Amgen — to subdue the notorious KRAS oncogene heats up in the early clinical space, Merck has decided it’s not enough to rely on their flagship PD-1 or the mRNA shared antigen vaccine they’re developing with Moderna. The pharma giant is betting on a slate of preclinical small molecule candidates from Japan’s Taiho and Astex (Cambridge UK) – the latter is a subsidiary of Otsuka Pharmaceutical – that target, among several others, KRAS. Altogether, Taiho and Astex will receive a combined $50 million upfront with $2.5 billion in biobucks to look forward to.   (Endpoints  January 6, 2020)

GSK divests travel vaccines to Bavarian Nordic as part of strategic refocus  GlaxoSmithKline has penned a deal worth up to €955m with Bavarian Nordic, to offload its travel vaccines Rabipur and Encepur. Rabipur is indicated for the prevention of rabies, while Encepur is a preventative treatment for tick-borne encephalitis. Under the terms of the deal, GSK has received an upfront payment of €308m, and is eligible to receive milestone payments of up to €955m.  (PM Live  January 3, 2020)

New DNA tests mean babies and children can be diagnosed with rare diseases in just days   The NHS is promising a “genomic revolution,” with rare child diseases diagnosed in days, with far more accuracy than has previously possible. Up to 700 babies and children a year will be offered a new form of DNA test, which can rapidly identify mutations in critically ill patients. The Health Secretary, Matt Hancock, said the new technology, known as “whole exome sequencing”, can reveal what is wrong with patients in days rather than weeks and was “game changing” for the NHS, with experts saying the tests could double the chance of an accurate diagnosis and ensure swift treatment.  (The Telegraph  January 3, 2020)

An instant 2nd opinion: Google’s DeepMind AI bests doctors at breast cancer screening   Google’s DeepMind (London) team showed that it can outperform trained radiologists in spotting cases of breast cancer, and that its artificial intelligence is capable of providing an independent, automated and immediate second opinion. By using programs trained on 2D and 3D mammography images from nearly 30,000 women in the US and the UK, DeepMind’s system was able to identify those that had their cancer confirmed within the following year via a tissue biopsy or subsequent X-rays. Among patients from the US, the AI cut the number of people incorrectly referred for further screening with a false-positive result by 5.7%—while also detecting 9.4% of potentially missed breast cancer cases. Published in Nature , the study said it surpassed the work of six independent physicians.  (FierceBiotech  January 2, 2020)