BELS Brief Clips: December 2019

Following is a sampling of UK health and life science news from December 2019 curated by BELS and disseminated to members of our global community

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UK looks to shake up trial, medicines regulation amid Brexit   As the UK prepares to leave the EU, newly elected Prime Minister Boris Johnson has sketched out plans that aim to speed up trials. Details are still thin, but the so-called Medicines and Medical Devices Bill will be brought forward by the Conservative government, which won a large majority in the general election just before Christmas. There is a series of ideas in the bill that includes relaxing certain rules around studies and speeding up the approval of new medicines. This will create a “streamlined, internationally competitive” process for approving new drugs and devices, the government said, and would focus on “removing unnecessary bureaucracy” for what it deems low-risk clinical trials.  (FierceBiotech  December 31, 2019)

Sensyne Health pairs with Roche for AI trial work   Clinical trial artificial intelligence tech company Sensyne Health (Oxford) has signed a new research project deal with Swiss major Roche. Details are a little thin on the ground (with no financials being shared), but the company said the collaboration will for starters focus on “identifying patient populations in one disease area and assessing and collating anonymised patient data with anonymised electronic patient record information to support clinical trial planning.”  (FierceBiotech  December 29, 2019)

GSK puts faith in AI to make more successful drugs more quickly GlaxoSmithKline is ramping up its use of artificial intelligence and recruiting 80 AI specialists by the end of 2020 as it turns to cutting-edge computing to develop medicines of the future. However, the UK’s largest drugmaker by revenue is struggling to hire enough AI researchers and engineers from areas such as Silicon Valley and is looking to former employees in academia, the US Navy and the music industry to fill positions in the new team. They will be spread across London, Heidelberg, San Francisco, Philadelphia and Boston.  (The Guardian  December 25, 2019)

Scientists discover molecular ‘lock’ that prevents genetic mistakes when cells divide   Research from Queen Mary University of London has provided new insight into how dividing cells control the separation of their DNA. The study has shown that a protein called Astrin, important for the timely and even separation of chromosomes during cell division, is recruited to correct attachments and works to secure them further. (QMUL  December 24, 2019)

Cannabis-based medicine for epilepsy available on NHS from January   People with severe epilepsy will be able to access a cannabis-based medicine on the NHS from early next year after it was fast-tracked for use. NHS England said doctors would be able to prescribe GW Pharmaceuticals’ (Cambridge UK) Epidyolex from 6 January. It will be for children from age two, as well as adults, but some campaigners warn it is “too little too late”. Clinical trials have shown the oral solution, which contains cannabidiol (CBD), could reduce the number of seizures by up to 40% in some children. The medicine will be used to treat two rare, but severe, forms of childhood epilepsy–Lennox Gastaut syndrome and Dravet syndrome–which can cause multiple seizures a day.  (BBC  December 21, 2019)

Daiichi, AstraZeneca breast cancer drug joins bumper crop of early FDA approvals   The FDA approved Daiichi Sankyo and AstraZeneca ‘s drug trastuzumab deruxtecan for heavily pretreated breast cancer, a decision that came more than three months ahead of the agency’s deadline. The drug, which will be sold as Enhertu, combines the active ingredient in Roche’s top-selling Herceptin with a toxic payload designed to destroy tumor cells. It’s now cleared for use in women who test positive for a breast cancer protein called HER2, just as Herceptin is. AstraZeneca made a substantial bet to gain access to the drug, paying Daiichi Sankyo $1.35 billion upfront to split marketing rights for Enhertu outside Japan. The deal was unusually financed through the sale of $3.5 billion worth of the UK pharma’s shares. (BioPharma Dive  December 20, 2019)

Prostate Cancer Modeling Study Supports Precision Screening Approach   A new mathematical analysis suggests prostate cancer screening programs tailored to an individual’s polygenic risk of the disease may catch disease and prevent deaths, while decreasing the overdiagnosis compared to age-based screening. A University College London study shows that targeted screening can reduce unnecessary diagnoses while helping to prevent people dying from the disease by enabling earlier detection.  (GenomeWeb  December 20, 2019)

Large Pan-Cancer CRISPR Screens by Broad, Sanger Institutes Show High Concordance   Researchers from the Broad Institute and the Wellcome Sanger Institute have found that results from two pan-cancer CRISPR-Cas9 screens recently published in  Nature Communications by the institutes were highly concordant across multiple metrics despite significant differences in experimental protocols and reagents. The researchers said both common and specific cancer dependencies were jointly identified across the two studies. Further, robust biomarkers of gene dependency found in one dataset were recovered in the other.  (GenomeWeb  December 20, 2019)

Spurned in Britain, Woodford Heads to China for His Next Act   Neil Woodford, the famed UK stock picker who was ousted from eponymous funds, has flown to China to test investor appetite for a potential comeback.  (Bloomberg  December 18, 2019)

Healthy Sleep Patterns May Counteract Genetic Susceptibility of Heart Disease, Stroke   Good sleep habits may in part counteract genetic susceptibility to developing cardiovascular disease, a new analysis has found. Heart disease kills 647,000 people each year in the US, according to the CDC, and both genetic and behavioral factors contribute to its development. Additionally, recent studies using data collected by the UK Biobank have suggested that unhealthy sleep behaviors like insomnia or snoring may be risk factors for cardiovascular disease. Individuals with poor sleep habits and a high genetic susceptibility score were two-and-a-half times as likely to develop heart disease, but healthier sleep habits appear to partially counter this effect, as individuals with high genetic susceptibility scores but good sleep habits had a lower rate of heart disease.  (GenomeWeb  December 18, 2019)

Abcodia and AstraZeneca China fight for earlier cancer detection Abcodia (Cambridge UK) has formed a strategic partnership with AstraZeneca China focusing on the early detection of ovarian cancer. With Abcodia’s ROCA Test as the central technology the parties will collaborate to establish ovarian cancer early detection networks across China. The intent of the collaboration fits strategically with AstraZeneca’s mission to improve cancer outcomes in support of the Chinese government’s ‘Healthy China 2030’ initiative, which includes a focus on early screening, early diagnosis and early treatment, reducing cancer morbidity and mortality and improving the quality of life of patients. The announcement follows on from Abcodia’s commitment to locate its China subsidiary in Wuxi, on the newly formed I·Campus, which has been created by Wuxi National High-Tech Industrial Development Zone and AstraZeneca China.  (Business Weekly  December 18, 2019)

Researchers identify possible link between cannabis use and structural changes to heart   Analysing MRI images from the UK Biobank population study, researchers led by a team at Queen Mary University of London identified an association between regular cannabis use and an enlarged left ventricle–the heart’s main pumping chamber–together with early signs of impairment of heart function.  (QMUL  December 18, 2019)

UK antitrust watchdog clears Roche/Spark deal — but what is the FTC thinking?   Roche has gained the blessing from the UK antitrust watchdog to proceed with its $4.3 billion acquisition of Spark, just in time for the latest deadline the pharma giant has set for Spark’s investors to tender their shares following 10 delays. The UK Competition and Markets Authority confirmed speculation and rumors — pretty much assumed true at this point — that they had been looking into the competitive landscape for hemophilia A treatments. After concluding that there will still be an adequate choice of alternatives even after the makers of Hemlibra and a potential gene therapy are blended into one, regulators gave the deal an unconditional clearance.  (Endpoints  December 16, 2019)  

GSK poised to leap back into the oncology market with more positive belantamab mafodotin results — but BCMA rivals are swarming in behind GSK took an important step toward a key new approval, posting their second positive round of pivotal data for the BCMA-targeting multiple myeloma conjugate belantamab mafodotin (GSK2857916). And with upbeat DREAMM-2 data, they’ve filed for an approval with hopes of getting a priority review for their “breakthrough” drug candidate. But after the stellar rounds of new data in the BCMA field at ASH a few days ago, don’t expect a tremendous level of excitement to greet them over the outcome.  (Endpoints  December 16, 2019)

NICE u-turn backs NHS use of Veltassa NICE has given its stamp of approval for NHS use of Vifor Pharma’s hyperkalaemia drug Veltassa (patiromer). The drug, which was initially turned down by the cost regulator in 2018, will be offered as an option for adults in emergency care or for people with chronic hyperkalaemia and may benefit 13,600 people. Hyperkalaemia is characterised by elevated potassium levels in the blood. Risk of developing the condition increases significantly for patients with chronic kidney disease and for those who take life-saving medications for heart failure, such as renin-angiotensin-aldosterone system (RAAS) inhibitors, which can increase potassium in the blood.  (PharmaTimes  December 16, 2019)

Diurnal files Chronocort in the EU   Specialty pharma Diurnal (Cardiff) has filed an application in Europe to market Chronocort (modified release hydrocortisone) for patients with the rare condition congenital adrenal hyperplasia (CAH). The submission is based on analysis of data from a Phase III study, the largest ever interventional clinical trial in CAH, and an open-label safety extension study. The firm said it expects marketing approval for Chronocort in Europe in the first quarter of next year 2021.  (PharmaTimes  December 16, 2019)GSK’s ViiV seeks marketing licence for baby-friendly HIV pill GSK applied for a licence to market its HIV drug dolutegravir in a formulation designed to be easier for babies and children who are living with the virus to swallow. About 1.7 million children have HIV, most of them in sub-Saharan Africa, the United Nations agency UNAIDS says. If approved by regulators, the medicine will be the first new generation HIV medicine available in baby-friendly form.  (Reuters  December 13, 2019)

Horizon Discovery, Mammoth Biosciences Team Up to Develop CRISPR Tools Horizon Discovery (Cambridge UK) and Mammoth Biosciences will collaborate to develop new CRISPR tools, with the aim of generating new engineered cell lines for the biopharmaceutical industry. Under the terms of the agreement, Horizon and Mammoth will collaborate to identify and optimize novel proteins owned by Mammoth, and then license those proteins to Horizon. They will then be used to develop engineered cell lines. Horizon will have exclusive rights to utilize this novel bioproduction approach, and will also be allowed to sublicense it to other companies for purposes of modifying their own proprietary cell lines.  (GenomeWeb  December 13, 2019)

New Drug Approvals More Likely With Supporting Human Genetic Evidence   New drugs are more likely to be approved if they target a gene linked to disease, according to a new analysis from AbbVie researchers that confirms earlier findings. Only between 5 and 10% of new drug candidates that enter early-stage clinical trials are eventually approved. In 2015, researchers led by GSK ‘s Philippe Sanseau reported that drugs with supporting genetic evidence were twice as likely to be approved.  (GenomeWeb  December 13, 2019) 

Tory landslide election victory sets UK on path to Brexit   A thumping election win for the Conservative Party has positioned the UK to push ahead with plans to leave the EU. The new parliamentary arithmetic gives Boris Johnson the numbers to pass the withdrawal agreement and take the UK out of the EU, although what will happen after that remains a mystery. The Conservative Party gained more than 60 seats, largely at the expense of its chief rival Labour, leaving Johnson with a sizable majority. Johnson plans to use his majority to pass the withdrawal agreement, setting the UK up to leave the EU at the end of January. That gives the biopharma industry a level of certainty, a commodity that has been in short supply since the UK voted to leave the EU in 2016.  (FierceBiotech  December 13, 2019)

UK Study Details Liquid Biopsy’s Ability to Guide Metastatic Breast Cancer Treatment   Researchers from the Institute of Cancer Research (London) and the Royal Marsden NHS Foundation Trust have presented data demonstrating that blood-based genotyping assays can accurately detect specific biomarkers and potentially be used to guide targeted treatment of metastatic breast cancer. The researchers used Guardant Health’s liquid biopsy assay and Bio-Rad Technologies’ droplet digital PCR testing to identify errors — including mutations in the HER2, ESR1 and AKT1 genes — in circulating tumor DNA that had been shed into the patients’ bloodstream.   (GenomeWeb  December 13, 2019)

UK’s NHS to fund Shionogi’s Mulpleo after backing from NICE and SMC   Shionogi’s Mulpleo for severely low levels of blood platelets caused by liver disease is to be made available to NHS patients across the UK after authorities in England and Scotland said it is cost-effective. Mulpleo (lusutrombopag) is approved for treatment for severe thrombocytopenia in patients with chronic liver disease undergoing invasive procedures. It is the first licensed treatment to be made available for these patients through the NHS in England, Wales, and Scotland after backing from NICE and the Scottish Medicines Consortium .   (PharmaPhorum  December 12, 2019)

At ASH, Researchers Discuss Rapid AML Nanopore Sequencing Assay for Analyzing Actionable Alterations   Researchers at Ohio State University have developed a rapid single-molecule nanopore sequencing assay that can analyze blood samples from acute myeloid leukemia patients for clinically actionable and prognostic markers in a few hours. The team, led by OSU’s Esko Kautto and James Blachly, designed the test on Oxford Nanopore Technologies ‘ (Oxford) MinIon sequencer, aiming for same-day analysis of AML patients’ DNA so the detected genomic variants may be used to guide treatment or inform their prognosis.  (GenomeWeb  December 11, 2019)

Abu Dhabi Teams Up With BGI, Oxford Nanopore for Population Sequencing Project   The Genome Program plans to sequence 100,000 samples in its first phase, free of charge for United Arab Emirates citizens and will proceed in two stages: a research stage to study the genetic variation of the population and a strategy stage to improve health outcomes based on the research results. The program will be led by the Department of Health-Abu Dhabi and executed by Group 42, a technology firm based in the emirate, in collaboration with BGI and Oxford Nanopore Technologies (Oxford). In addition, Abu Dhabi Health Services Company will use the results to deliver healthcare services.  (GenomeWeb  December 10, 2019)

CMR changes CEO ahead of US robotic surgery launch CMR Surgical (Cambridge UK) has appointed Per Vegard Nerseth as its CEO. Nerseth joins CMR from ABB, a provider of industrial robots where he worked for almost 20 years. The leadership change comes at a time when CMR is transitioning from a development-stage startup into a challenger for the robotic surgery market. …Having developed its Versius robotic surgery system and raised $240 million to fund commercialization, the big tasks now revolve around its effort to bring down the cost of adopting robotic surgery and thereby enable more sites to move away from manual procedures  (MedTechDive  December 10, 2019)

DeepMatter will help AstraZeneca digitise discovery chemistry   Digital technologies are permeating throughout pharma’s internal processes, and AstraZeneca has teamed up with DeepMatter (Glasgow) to apply a digital approach to early-stage drug discovery. AZ has become the second group to sign up to use DeepMatter’s DigitalGlassware platform, intended to improve and accelerate the compound synthesis stage of new drug development, in particular lead identification and optimisation. Earlier this year DeepMatter signed its first revenue-generating partnership for the platform with Anglo-Indian medicinal chemistry service provider o2h discovery (Cambridge UK), but the deal with AZ is a step up in scale and a big boost for DeepMatter whose shares surged more than 80% on the AIM exchange after the deal was announced. (PharmaPhorum December 9, 2010)

Autolus Reveals Early Clinical Data of Next-Gen CAR-T Therapy Autolus (London) has shown its next-generation CAR T-cell therapy does not induce some of the severe side effects that have led to deaths in patients treated with CAR T-cell treatments currently on the market. …Autolus has designed a new generation of CAR T-cell therapy in which the T cells are modified to prevent excessive activation of the T cells, which can cause toxic side effects.  (Labiotech  December 9, 2019)

ASH: AstraZeneca’s Calquence posts sky-high survival numbers in previously untreated CLL   Over the last couple of weeks, regulators in the US, Canada and Australia have all green-lighted AstraZeneca ’s Calquence in newly diagnosed chronic lymphocytic leukemia (CLL) patients. And Saturday, the company revealed more of the data that convinced them to dole out those approvals. In a phase 3 trial, 93% of CLL patients taking a combination of Calquence and Roche’s Gazyva hadn’t seen their disease worsen two years after therapy, while just 47% of those taking chlorambucil and Gazyva could say the same. Meanwhile, 87% of people receiving solo Calquence met that benchmark.  (FiercePharma  December 8, 2019)

ViiV Healthcare submits HIV drug fostemsavir in the US ViiV Healthcare (Brentford), the global specialist HIV company majority owned by GSK, with Pfizer and Shionogi Limited as shareholders, has filed an NDA in the US for fostemsavir, an investigational, first-in-class attachment inhibitor for the treatment of HIV-1 infection. The drug is being developed for use in combination with other antiretroviral agents in heavily treatment-experienced adults with multidrug-resistant HIV-1 infection who are unable to form a suppressive regimen due to resistance, intolerance or safety considerations. (PharmaTimes December 6, 2019)

Scientists modify plants to fight off widespread crop disease   Scientists have tested a new method to protect crops from disease by making the plants manufacture antibiotics derived from bacteria. They hope it can replace conventional antibiotics used to treat plants, removing a driver of antibiotic resistance that could spread to human diseases. The team at Glasgow University genetically engineered plants to fight off bacterial infection on their own by producing a targeted protein antibiotic, or bacteriocin. This was effective against the common crop bacteria Pseudomonas syringae, which causes diseases including blight and spot.  (  December 5, 2019)

Typhoid vaccine over 81% effective in tackling disease in Nepal   A large field study of typhoid conjugate vaccine (TCV) in Nepal has shown a single dose to be safe and effective in reducing typhoid in children aged 9 months to <16 years in an endemic setting. Although TCV has been shown to protect against the disease in studies involving healthy volunteers in the UK, no efficacy studies in endemic populations had been completed. Now, the Typhoid Vaccine Acceleration Consortium, which includes researchers from the University of Oxford , the University of Maryland School of Medicine, and PATH has completed a large field study in Nepal and published the interim analysis in the New England Journal of Medicine.  (University of Oxford  December 4, 2019)

Sangamo granted authorisation of first-in-human CAR-Treg trial   Sangamo Therapeutics has been granted authorisation from the UK’s Medicines Healthcare Products Regulatory Agency to conduct the first-in-human clinical trial of a novel type of cell therapy – Chimeric Antigen Receptor Regulatory T Cell. The drug, TX200, is being evaluated for the prevention of immune-mediated rejection following HLA-A2 mismatched kidney transplantation for end-stage renal disease (ESRD). The genomic medicine company says it expects to open the first clinical sites for the STEADFAST study in 2020, which makes it the “first company to test a CAR-Treg candidate in humans”.  (PharmaTimes  December 3, 2019)

Regenerative Medicine Advanced Therapy Designation Granted by FDA to ADP‑A2M4 for the Treatment of Synovial Sarcoma Adaptimmune Therapeutics (Oxford) received Regenerative Medicine Advanced Therapy designation from the FDA for its T-cell therapy ADP-A2M4 for the treatment of synovial sarcoma, which earlier this year was granted Orphan Drug Designation for the treatment of soft tissue sarcomas. The FDA designated ADP-42M4 as a regenerative medicine advanced therapy. The RMAT designation will enable expedited development and review, with the goal of commercialization in 2022 for patients with few other treatment options.  (Press release  December 3, 2019)

Lynparza shows benefits in prostate cancer   Lynparza (olaparib) continues to go from strength to strength, as the MSD and AstraZeneca PARP inhibitor has now been shown to slow or stop tumour growth in some men with advanced prostate cancer, shown by a new clinical trial. The pioneering precision medicine, already licensed for breast and ovarian cancer, was found to be effective in over 80% of men with prostate cancer whose tumours had mutations in the BRCA genes.  (PharmaTimes  December 3, 2019)

Human Protein Atlas to Use Horizon Discovery Cell Models Horizon Discovery Group (Cambridge UK) said that its CRISPR-edited knockout cell models have been selected for use by the Human Protein Atlas (HPA) project. Based in Sweden, the HPA was established in 2003 to map all the human proteins in cells, tissues, and organs using a variety of omics technologies. Horizon Discovery said that its cell models will be integrated into the HPA’s Cell Atlas program, which focuses on the subcellular localization of proteins in single cells. HPA researchers will initially use 500 of the UK company’s CRISPR-edited knockout cell models as part of large-scale protein expression and imaging studies.  (GenomeWeb  December 3, 2019)

NICE grants Lynparza expansion for advanced ovarian cancer   Lynparza (olaparib), the joint MSD and AstraZeneca cancer drug, has been granted an expansion by NICE for maintenance treatment of certain cancers. The decision means that the PARP inhibitor is now the first and only of its kind in the UK to be approved in all lines of maintenance treatment. The label extension now covers relapsed, platinum-sensitive high grade epithelial ovarian, fallopian tube or peritoneal cancer in adults whose disease has responded to platinum-based chemotherapy if they have a BRCA1 or BRCA2 mutation, meaning that the drug will be made available to patients within NHS England and Wales via routine commissioning if they have had three or more courses of platinum-based chemotherapy. It will also be made available to patients within NHS England via the Cancer Drugs Fund if they have had two courses of platinum-based chemotherapy. (PharmaTimes  December 2, 2019)

Owkin, NVIDIA team up to deliver AI to hospitals   Owkin announced a new partnership with NVIDIA and King’s College London to deliver US-based Owkin’s “Federated Learning” software in the healthcare and life sciences sector. The project will initially connect four of London’s premier teaching hospitals before expanding throughout the UK, and will offer AI services to accelerate research and improve clinical practice in a wide range of therapeutic areas, including cancer, heart failure and neurodegenerative disease.  (PharmaTimes  December 2, 2019)