Your guide to the pharmaceutical world Volume 6 Issue 5DiabetesExposéFinding a new solutionDistributionPrescription for digitalPrecisionTreatmentTailored treatmentPharmaBUSINESS INTERNATIONALThe total packageFrom development to dispensingThe total packageFrom development to dispensingDiabetesExposéFinding a new solutionDistributionPrescription for digitalPrecisionTreatmentTailored treatmentwww.pbiforum.net01.qxp_Layout 1 04/10/2018 12:15 Page 1EUROPE 20195-7 FEBRUARY | VIENNAJoin us at the Crossroads of HealthcareTo find out more and book your ticket, visit www.diaglobal.org/DIAeurope19 now.DIA Europe is more than a traditional meeting. Our members say, “There is something special about DIA Europe”: It’s a chance to learn, engage, and grow as a healthcare professional. DIA Europe has become a landmark, must-attend event for many.We welcome participants from across the entire drug development spectrum, from discovery to marketed use, to facilitate open collaboration by incorporating representatives from the full life sciences landscape. DIA ensures that industry, payers, patients, and regulators all have an equal voice.We look forward to welcoming YOU to DIA Europe 2019 in Vienna!Kristel Van der ElstCEO, Global Foresight GroupHealth Systems: Doomed to Fail or About Be Saved by a Copernican Shift?Dave deBronkart‘ePatient’ DavePatient Experience, Empowerment, Engagement: A Business Leader’s ViewHans Lehrach'LUHFWRUDQG6FLHQWL²F0HPEHUDWWKH0D[3ODQFN,QVWLWXWHIRU0ROHFXODU*HQHWLFVWill Virtual Patients Allow for Virtual Trials?KEYNOTE SPEAKERS02.qxp_Layout 1 04/10/2018 12:15 Page 1@pbiforumwww.pbiforum.netcompany/pharma-business-internationalContentsVolume 6 Issue 5News4Stay up to date with the latest industry updates fromthe global pharmaceutical technology industry.M&A Round-up8 There’s been a few major deals over the billion-dollar-deal threshold of late, but this issue’s M&A round-upis focussed on some of the smaller deals ascompanies consolidate their pipelines and look tounmet needs.R&D12 Artificial intelligence has emerged as one of the mostdisruptive tools in the pharmaceutical R&D space,with established companies and academicorganisations utilising AI in drug developmentalongside start-up companies arriving on the scene tomeet unmet needs and address some of the biggesthealthcare and medical issues facing the world today. Distribution16 Blockchain technology is transforming thepharmaceutical and life science supply chains, butthere are barriers and challenges that need to beovercome before widespread adoption can occur. Precision Medicine20 At the opposite end of the scale of the ‘one size fitsall’ model of medicine is precision medicine which ishelping to transform the way in which patients aretreated. Finance24 There are several channels open to pharmaceuticalcompanies, universities and organisations ro raisecapital to fund critical research and developments. Diabetes Exposé28 With the number of people diagnosed with diabetesrising, the need for new treatments and therapies iscritical. Events32We give a rundown of pharma events happeningaround the world.12162032Group Editor:Steve Fisher (s.fisher@blmgroup.co.uk) Editor:Michael Fisher (m.fisher@blmgroup.co.uk) Journalist:Dominic Cuthbert(d.cuthbert@blmgroup.co.uk)Sales Director:Angie Cooper (a.cooper@blmgroup.co.uk)Sales Manager:Sam Trott (s.trott@blmgroup.co.uk)Sales:Vicky Hunt (v.hunt@blmgroup.co.uk)Tel: +44 (0) 1472 310302 Accounts & Subscriptions:Angela Sharman(a.sharman@blmgroup.co.uk)Design & Production:Gary Jorgensen,Mark Casson,(studio@blmgroup.co.uk)Editorial:Tel: +44 (0) 1472 310305, Fax: +44 (0) 1472 310317Part of BLMGroup:Armstrong House, Armstrong Street,Grimsby, N E Lincs DN31 2QE EnglandAll rights reserved. No part of this publication may be reproduced,transmitted, photocopied, recorded or otherwise without expresspermission of the copyright holder, for which application should beaddressed first to the publisher. While every reasonable care istaken, neither the publisher nor its participating agents acceptliability for loss or damage to prints, colour transparencies,negatives or other material of whatever nature submitted to thispublication. The views expressed in this publication are notnecessarily the views of those held by the publisher.This magazine is now fully recyclable. By recycling magazines, youcan help reduce waste and add to the 5.5 million tonnes of paperalready recycled by the UK paper industry each year. Before yourecycle your magazine, please ensure you remove all plasticwrapping, free gifts and samples. COVER PHOTO: © SHUTTERSTOCK / RATMANER 03.qxp_Layout 1 04/10/2018 12:16 Page 1Anne White named new headof Lilly OncologyEli Lilly has promoted Anne White to President of its oncologybusiness, replacing Sue Mahony.White is currently Vice President of portfolio management, Chorus andNext Generation Research & Development. She has more than 25 yearsof experience in the pharmaceutical industry, serving in leadership rolesin oncology, neuroscience, infectious disease drug development andprocess transformation.White also worked in biotech, having founded and led a start-upcompany in oncology drug development with partnerships with theMoffitt Cancer Centre and the National Cancer Institute.“Anne brings a wealth of experience, expertise and leadership to our executive team,” said Chairman and CEO, David A. Ricks. “She has led anddelivered against our Next Generation Development objectives, accelerating medicines to patients and playing a key role in our recent R&Dproductivity. Her experience leading significant change transformation within the company will be incredibly valuable as we look toward the future.”4Pharma Business International www.pbiforum.netLatest newsTolero working withAbbVie on potential AMLtreatmentTolero Pharmaceuticals aims to explore the potential combinationtherapy of its investigational agent alvocidib with AbbVie’s venetoclaxfor treating relapsed/refractory acute myeloid leukaemia (AML).Alvocidib is a small molecule inhibitor of cyclin-dependent kinase 9which controls the expression of a survival factor, MCL-1. Venetoclaxis a small molecule inhibitor of B-cell lymphoma-2 (BCL-2).Both are key proteins used by certain cancer cells to avoidapoptosis, and non-clinical studies have shown that cancer cells canresist inhibition of BCL-2 by using MCL-1 to avoid cell death.Alvocidib is currently in Phase II development for the treatment ofMCL-1-dependent AML.David J. Bearss, Tolero CEO, said: “Preclinical data suggest that themechanisms of action for venetoclax and alvocidib may synergistically drive apoptosis in cancercells. We hope to further investigate this hypothesis with our planned trial of thiscombination therapy in patients with relapsed/refractory AML.”£8m fund to boostmedicine manufacturingThe UK’s Industrial Strategy Challenge Fund is making up to £8million available to businesses to digitise and improve themanufacture of medicines.UK businesses can apply for a share of up to £8 million forprojects that significantly improve the efficiency, effectiveness andscope of medicines manufacture.The funding, part of the government’s modern £181 millionIndustrial Strategy, is provided by UK Research and Innovationand delivered through Innovate UK.© Shutterstock / Vasily Deyneka© Shutterstock / Medtech THAI STUDIO LAB 249Anne White04-07.qxp_Layout 1 04/10/2018 12:29 Page 1Zika vaccine adapted tofight brain cancerResearchers at The University of Texas Medical Branch at Galvestonhave successfully harnessed a Zika virus vaccine under development totarget and kill the brain cancer known as glioblastoma.The Zika virus can cause microcephaly, a condition where the foetalbrain doesn’t fully develop when a pregnant woman is infected with thevirus.In contrast, glioblastoma dangerously multiplies brain cells intocancerous tumours. The altered Zika vaccine effectively targeted anddestroyed the cancerous brain cells in mice but not healthy cells.“These findings represent major progress toward developing the Zikavaccine as a safe and effective virotherapeutic treatment for humanglioblastoma,” said UTMB’s Pei-Yong Shi, lead investigator and professorin the department of biochemistry and molecular biology.The researchers found that the Zika vaccine caused no neurologicalsymptoms or behavioural abnormalities while significantly reducingtumour growth and prolonging survival.The findings were published in MBio.Pharma Business International 5www.pbiforum.netLatest news8,000 new antibiotic combos surprisingly effective,biologists sayA team of UCLA biologists has discovered thousands of four- and five-drugcombinations of antibiotics that are more effective at killing harmful bacteriathan the prevailing views suggested.Reported in the npj Systems Biology and Applications, the findings couldbe a major step toward protecting public health at a time when pathogensand common infections are increasingly becoming resistant to antibiotics.“There is a tradition of using just one drug, maybe two,” said Pamela Yeh,one of the study’s senior authors and a UCLA assistant professor of ecologyand evolutionary biology. “We’re offering an alternative that looks verypromising. We shouldn’t limit ourselves to just single drugs or two-drugcombinations in our medical toolbox. We expect several of thesecombinations, or more, will work much better than existing antibiotics.”The researchers are creating open-access software based on their workthat they plan to make available to other scientists next year.New drug blocks pancreaticcancer growth in miceA newly developed drug has been found to prevent the mostcommon type of pancreatic cancer from growing and spreading inlaboratory mice in a study led by Cedars-Sinai.The study, published in Gastroenterology, also demonstrated inmice that the drug – called Metavert – can prevent patients fromdeveloping a resistance to currently used pancreatic cancerchemotherapies.“This is an exciting step toward improving survival rates inpancreatic cancer patients,” said study lead author MouadEdderkaoui, assistant professor of Medicine and Biomedical Sciencesat the Samuel Oschin Comprehensive Cancer Institute at Cedars-Sinai. “If the results are confirmed in humans, we could have a drugwith the potential to significantly extend the lives of patients withpancreatic ductal adenocarcinoma (PDAC), which is very difficult totreat.”The investigators currently are developing a version of the drug totest in humans.© Shutterstock / Shidlovski© Reed Hutchinson/UCLAPamela Yeh with leadstudy author Elif Tekin© Shutterstock / sfam_photo04-07.qxp_Layout 1 04/10/2018 12:29 Page 26Pharma Business International www.pbiforum.netLatest newsGilead subsidiary to launchgenerics of chronic hep ctreatmentsGilead Sciences is planning to launch authorised generic versions of itschronic hepatitis C virus (HCV) treatments in the US through a newlycreated subsidiary, Asegua Therapeutics.The generics of Epclusa and Harvoni will launch at a list price of$24,000 for the most common course of therapy and will be available inJanuary 2019.Gilead said the generics are priced to more closely reflect thediscounts that health insurers and government payers receive today.Insurers will have the choice of offering either the authorised generics orthe branded medications for both Epclusa and Harvoni.“Launching these authorised generics is the best solution available to us today to quickly introduce a lower-pricedalternative to our HCV medications without significant disruption to the healthcare system and our business,”said John F. Milligan, President and CEO of Gilead Sciences.Universal flu vaccine finds pre-clinical successPre-clinical success for a universal flu vaccine has been demonstrated by researchers atthe University of Oxford’s Department of Zoology writing in Nature Communications.Influenza is thought to be a highly variable virus, but flu seasons tend to be dominatedby a limited number of antigenically and genetically distinct influenza viruses. Mathematicalmodels produced in Professor Sunetra Gupta’s group at the University of Oxford over thepast 20 years have sought to find an answer to this paradox.Finally, through a collaborative approach across multiple departments, the groupbelieves they have the answer.The results can be exploited to create a novel type of ‘universal’ or broadly protectiveinfluenza vaccine, which once administered would provide lifelong protection againstinfluenza.The team also hopes to apply the approach to other viruses such as HIV and HCV andbelieves that they can use it to produce a vaccine that protects against the common cold.Partnership to accelerateuptake of biomedicalinnovations into NHSThe Association of the British Pharmaceutical Industry (ABPI) and the AHSNhave launched a five-year partnership to accelerate the introduction anduptake of biomedical innovations into the NHS.Under the terms of the new partnership, a process will be created whereAcademic Health Science Networks (AHSNs) and ABPI member companiescan identify and collaborate on projects to accelerate the spread of proveninnovation in the NHS.The collaborative projects will address the new national priority areas of the 15 AHSNs – including digital and artificial intelligence;research; genomics and personalised medicine, and diagnostics and medicines optimisation.Mike Thompson, Chief Executive of the ABPI, said: “As a result of this new partnership, patients will be able to benefit from theinnovative medicines and technologies of the future faster and more consistently.”© Shutterstock / Image Point Fr© Shutterstock / Kateryna Kon© Shutterstock/Marbury04-07.qxp_Layout 1 04/10/2018 12:29 Page 3Pharma Business International 7www.pbiforum.netEye onapprovalsEUROPEHumira biosimilar gains marketing authorisation Mylan and Fujifilm Kyowa Kirin Biologics have gainedmarketing authorisation for Hulio – a biosimilar of AbbVie’sHumira – for all indications. This follows a positive opinion bythe committee for Medical Products for Human Use, whichconcluded the development programme demonstratedbiosimilarity. Marketing approval for Gilead’s CAR T therapy Gilead has secured marketing approval for Yescarta as atreatment for relapsed or refractory diffuse large B-celllymphoma and primary mediastinal large B-cell lymphoma.Yescarta is a chimeric antigen receptor T cell (CAR T) therapy,which harnesses a patient’s own immune system to fightcertain types of blood cancer. It was approved by the US FDAin 2017.Jazz Pharma’s chemotherapy gains marketingapprovalMarketing authorisation has also been granted to Vyxeos,Jazz Pharmaceutical’s treatment aimed at adults with certaintypes of high-risk acute myeloid leukaemia (AML). Vyxeos is anadvanced liposomal formulation that delivers a synergisticmolar ratio of daunorubicin and cytarabine.Novartis gains EU approval for Kymria Novartis has become the only company with an approvedCAR-T cell therapy for paediatricB-cell acute lymphoblasticleukaemia (ALL) after Kymriahwas approved in Europe.Developed in collaboration withthe University of Pennsylvania,Kymriah is a ground-breaking one-time treatment that uses apatient’s own T cells to fightcancer. It is the only CAR-Ttherapy to receive regulatoryapproval in the EU for thesetwo distinct B-cellmalignancies. It was also thefirst CAR-T cell therapy everapproved by the US FDA.UNITED STATESApproval for Teva’s injectable migrainetherapyTeva has gained US approval for Ajovy, its injectablemigraine treatment. Ajovy is a humanised monoclonalantibody that binds to calcitonin gene-related peptide (CGRP)ligand and blocks its binding to the receptor. It is the first andonly anti-CGRP treatment for the prevention of migraine withquarterly (675 mg) and monthly (225 mg) dosing options. AZ gains approval for hairy cell leukaemiatreatmentAstraZeneca has gained approval in the US for Lumoxiti forthe treatment of certain adult patients with relapsed orrefractory hairy cell leukaemia (HCL). The approval is basedon data from the Phase III single-arm, open-label ‘1053’ trialof Lumoxiti monotherapy in 80 patients who have received atleast two prior therapies, including a purine nucleosideanalogue.Dompé gains its first ever FDA approvalItaly’s Dompé has gained FDA approved for cenegermin, itsbreakthrough therapy for neurotrophic keratitis, a rare andprogressive eye disease that can lead to corneal scarring andvision loss. cenegermin is the first-ever application of ahuman nerve growth factor as drug or treatment and is thefirst-ever topical biologic medication approved inophthalmology.FDA approves first oral medicine for Fabrydisease Amicus Therapeutics has gained an acceleratedapproval from the FDA for Galfold for treatingcertain adult patients with Fabry disease. Galafoldis a capsule taken once every other day, at thesame time of day. The FDA has approvedGalafold for 348 amenable GLA variants. To date,it is the first oral medicine for Fabry disease, andthe first new therapyapproved to treatFabry disease in theUnited States inmore than 15years.04-07.qxp_Layout 1 04/10/2018 12:29 Page 4M&A ROUND-UP8Pharma Business International www.pbiforum.net© Shutterstock/Melina.photos08-11.qxp_Layout 1 04/10/2018 12:19 Page 1www.pbiforum.netIn our last issue, we began withShire. It’s only fitting that ourOctober/November column doesthe same. The end of August sawthe Dublin-headquartered giantcomplete the $2.4 billion sale of its oncology businessto French pharmaceutical business Servier.The deal sees Servier take charge of the globalrights to Oncaspar as well as the ex-US andex-Taiwan rights of Onivyde on top ofoncology pipeline assets. The sale alsosees a management shake-up withDavid Lee – previously the head ofShire’s Global Genetic Diseases andOncology franchises – continuing as CEO of itsnew US commercial subsidiary, ServierPharmaceuticals. Although Shire dominates this month’s column, we havegot news of one other potential billion-dollar-plus dealthat’s taken place in the pharmaceutical M&A market overthe last few months. Alexion has swooped forSyntimmune, a Boston, MA-based biotech firm developingantibody therapeutics targeting the neonatal Fc receptor(FcRn), for up to $1.2 billion. Broken down, the deal sees Alexion paying an upfrontpayment of $400 million, with the potential for additionalpayments of up to $800 million dependent on milestones.It’s a good deal for Alexion who gains access to SYNT001– a humanised monoclonal antibody that inhabits theinteraction of FcRn with Immunoglobulin G (IgG) and IgGimmune complexes. SYNT001 is currently being evaluatedin Phase 1b/2a studies in patients with warm autoimmunehaemolytic anaemia and in patients with pemphigusvulgaris or pemphigus foliaceus.Alexion CEO Ludwig Hantson said at the time that thedeal: “represents a critical step in rebuilding Alexion’spipeline and further diversifying the company’s clinical-stage rare disease portfolio”.Although there’s been a slowdown in deals over thebillion-dollar-mark, the M&A market has been far fromquiet. Danish pharma firm Novo Nordisk has bolstered itsinsulin profile with the $800 million acquisition of Ziylo, aspin-out company from the UK’s University of Bristolpioneering the use of synthetic glucose binding moleculesfor therapeutic and diagnostic applications. The deal gives Novo Nordisk full rights to Ziylo’sglucose binding molecule platform to develop glucoseresponsive insulins. The company said that thedevelopment of these insulins is a “strategic area” in itsongoing effort to develop a next-gen safer, more effectiveinsulin therapy. Before the deal was sealed, certain research activitieswere spun out of Ziylo to form a new company calledCarbometrics. This spin-out has now entered into aseparate research collaboration with Novo Nordisk toassist with ongoing optimisation of glucose bindingmolecules for use in glucose responsive insulins.Looking at thelatest dealsThere’s been a few major deals over the billion-dollar-deal threshold of late, but this issue’s M&A round-up isfocussed on some of the smaller acquisitions as companies consolidate their pipelines and look to unmet needs. 10 ÁPharma Business International 9M&A ROUND-UP08-11.qxp_Layout 1 04/10/2018 12:19 Page 2Next >