Your guide to the pharmaceutical world Volume 6 Issue 6MultipleSclerosisExposéWorking towards a cureGene therapyHard cellLogisticsFrom point to pointPharmaBUSINESS INTERNATIONALThe total packageFrom development to dispensingThe total packageFrom development to dispensingMultipleSclerosisExposéWorking towards a cureGene therapyHard cellLogisticsFrom point to pointwww.pbiforum.net01.qxp_Layout 1 04/12/2018 15:04 Page 127 & 28 February 2019 . NEC, BirminghamREGISTER ONLINEwww.easyfairs.com/PIUK VISIT THE EVENT FOR FREE REGISTER ONLINE TODAY2IÀFLDOPHGLDSDUWQHU,QSDUWQHUVKLSZLWK It has been great to see such an array of different suppliers all under one roof. The show has been very informative, clearly laid RXWDQGZHZLOOGHÀQLWHO\FRPHDJDLQ$VKOH\/LJKWIRRW3ULQW $UWZRUN7HFKQRORJLVW0XOOHU8. ,UHODQG,WLVRXUÀUVWWLPHDWWKHVKRZDQGZHDUHreally impressed. We came to the show looking for suppliers and have had a number of leads.5HEHFFD:DONHU%UDQG0DQDJHU)ORUHWWHMeet the whole supply chain, network and learn all under one roof02.qxp_Layout 1 04/12/2018 15:04 Page 1@pbiforumwww.pbiforum.netcompany/pharma-business-internationalContentsVolume 6 Issue 6News4Stay up to date with the latest industry updates fromthe global pharmaceutical technology industry.M&A Round-up8 Pharma Business International looks back over thelast year, charting the biggest deals in the mergersand acquisition market.Multiple Sclerosis Expose12 Currently, there’s no cure for multiple sclerosis, butthe research space for the condition is full ofinnovation and breakthroughs as Pharma BusinessInternational explores. Gene Therapy16 Although there’s still a significant risk attached withgene and cell therapies, the sector is emerging as oneof the most vital and innovative across thepharmaceutical spectrum. Logistics20 Transport is a go-to location for cutting costs, buthow much further can the industry be stretched? Novel Medicines24 The novel drugs industry is one that relies oninnovation, but has continued to expand year on year. Manufacturing28 Keeping up with changes in manufacturing can bedifficult, which is why so many outsource suchconcerns to others.Events32We give a rundown of pharma events happeningaround the world.4121620Group 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 / MARTIN_STUARD03.qxp_Layout 1 04/12/2018 15:04 Page 1Enesi & Sementis team up onneedle-free peanut allergy & zikavaccinesEnesi Pharma has entered into an R&D agreement with Australianbiotech Sementis aimed to needle-free solid dose vaccines for peanutallergy as well as Zika infection.The collaboration is focused on the development and evaluation ofsolid dose versions of Sementis’ lead peanut hypoallergy vaccine and itssingle vectored chikungunya/Zika vaccine candidates for administrationvia Enesi’s ImplaVax technology.ImplaVax is a proprietary needle-free solid dose implant and devicetechnology for subcutaneous vaccination. An ImplaVax-enabled vaccinecould provide a simple and effective means to address peanut allergy on a large scale, particularly for younger sufferers who may have an aversion toneedles and traditional injections.Sementis’ single vectored vaccine for chikungunya/Zika virus infection is equally promising with extensive proof-of-concept studies in micedemonstrating the effectiveness of the vaccine in protecting against Zika and chikungunya from a single vaccination shot.4Pharma Business International www.pbiforum.netLatest newsFunding boost tostreamline UK medicinemanufacturing The UK government is investing millions of pounds to streamline themanufacture of life-enhancing medicines and treatments.Three companies in Northumberland, Oxford and London will adoptnew digital technologies – such as artificial intelligence – to supportthe manufacture of next-generation medicines and treatments.The £4.3 million funding aims to create more efficient and innovativemethods for manufacturing medicines.Life Sciences Minister Lord Henley said: “New technology can helpus live longer, healthier lives and the new projects announced todaywill improve the speed and accuracy with which medicines get to thepeople that need them.“Advances in technology can help us address the challenges thatan ageing society presents and we are backing the technologies of tomorrow in ourmodern Industrial Strategy, with the biggest increase in public research anddevelopment investment in UK history.”Antibiotic-resistance bacteria to claim 33,000 lives a year by 205033,000 people will die every year by 2050 due to infections with antibiotic-resistant bacteria, according to a studyfrom the European Centre for Disease Prevention and Control (ECDC).The study estimates that the burden of these infections is comparable to that ofinfluenza, tuberculosis and HIV/AIDS combined.It also explains that 75% of the burden of disease is due to healthcare-associated infections and that reducing this through adequate infectionprevention and control measures, as well as antibiotic stewardship, could be anachievable goal in healthcare settings.Finally, the study shows that 39% of the burden is caused by infectionswith bacteria resistant to last-line antibiotics such as carbapenems andcolistin. This is an increase from 2007 and is worrying because these antibioticsare the last treatment options available.The study was developed by experts at ECDC and the Burden of AMR CollaborativeGroup and published in The Lancet Infectious Diseases.© Shutterstock / funnyangel© Shutterstock / Planar© Enesi Pharma04-07.qxp_Layout 1 04/12/2018 15:06 Page 1£5m to fund research intovaccines for diseaseepidemicsThe National Institute for Health Research (NIHR) has awarded £5million to explore how infectious diseases spread and develop inepidemics in low and middle-incomes, so that new vaccines can betested in clinical trials or used more effectively in future outbreaks.The best way to test the efficacy of new vaccines is during a diseaseoutbreak, but outbreaks occur sporadically and unpredictably. This new funding will support research into how epidemiologicalmodels, tools and technologies can assist with clinical trials anddeployment of new vaccines in outbreak situations in low- and middle-income countries.It is funded by UK aid funding from the Department of Health andSocial Care and delivered through the NIHR Policy Research Programme.The funding has been awarded to five research projects: three of whichwill be led by researchers at the London School of Hygiene & TropicalMedicine and two by the University of Oxford.Pharma Business International 5www.pbiforum.netLatest newsUK research organisationscombine to advance human-based drug evaluationThe UK’s Medicines Discovery Catapult and the Medical ResearchCouncil (MRC) Centre for Drug Safety Science (CDSS) are collaboratingto advance human-based drug evaluation.The two organisations aim to pool their collective academic andindustry experience to develop next-generation 3D human cell modelsand create robust cellular programmes.The innovations arising from this collaboration will be available to drug discovery SMEs in the UK. The purpose of the collaboration is to identify andestablish projects focused on the development of human 3D cell models and robust cellular platforms.Next-gen 3D cell models will provide even better predictive and patient relevant data by mimicking human tissue architecture, microenvironment, andsignalling.The first project to be undertaken under the new alliance will focus on the development of a next generation cardiac safety model. The human cell modelwill enable companies to measure a broader range of patient relevant toxicities in a single assay.LEO Pharma & PellePharmastrike $760m rare skin diseasescollaborationDanish dermatology specialists LEO Pharma and Californian raredisease pioneers PellePharm are working together to advanceinnovative therapies for rare skin diseases – such as Gorlin Syndrome.LEO Pharma has initially committed $70 million to fund the globalPhase 3 trial for patidegib topical gel 2% for the prevention andtreatment of Gorlin Syndrome. The company has also secured anoption to acquire all shares in PellePharm.PellePharm and its stockholders could receive up to an additional$690 million including merger consideration, and regulatory andcommercial milestone payments. In addition, PellePharm stockholdersare eligible to receive a double-digit royalty after achieving certaincommercial milestones.The agreement establishes a joint development committee withPellePharm maintaining responsibility for global development and LEOPharma supporting in an advising role. Both companies will jointly drivecommercialization planning, and Anders Kronborg, Chief FinancialOfficer of LEO Pharma, will join PellePharm’s board of directors.© Shutterstock / Andrey_Popov© Shutterstock/Sergey Nivens© Shutterstock / Numstocker04-07.qxp_Layout 1 04/12/2018 15:06 Page 26Pharma Business International www.pbiforum.netLatest news$200m Mount Sinai program toaccelerate data driven precisionmedicineThe Icahn School of Medicine at Mount Sinai is launching a new $200million programme aimed at accelerating data driven precision medicine.Dr Adam Margolin has been named Professor and Chair of the Departmentof Genetics and Genomic Sciences and Senior Associate Dean of PrecisionMedicine. He will also lead Mount Sinai’s Icahn Institute, which has beenrenamed the Icahn Institute for Data Science and Genomic Technology.He will lead the new enterprise-wide program that focuses on harnessinghuge molecular datasets to predict new therapies for complex diseases byusing advanced biotechnologies to rapidly tailor novel therapies to eachpatient faster than has ever been possible.He said: “We are creating a program built for the way science will be donein the future. The traditional academic model, organized around siloed individual labs, cannot achieve the speed, agility,and integration we need to advance biomedical research of today.”Over the next 10 years, Mount Sinai plans to recruit 30 new faculty members.Cloud-based platform creates AI-readylife sciences dataA new cloud-based data platform has been launched to help life sciences businessesovercome the challenges of modern R&D by enriching and harmonising proprietary andexternal data and delivering it in an AI-ready environment.With nearly a fifth of pharmaceutical spending going to R&D and the average cost ofbringing a new drug to market estimated at $4 billion, life sciences companies urgentlyneed more efficient ways to analyse data.Drawing on its longstanding experience in the life sciences, information analyticsbusiness Elsevier has created Entellect.This new platform can save time and costs by de-siloing, contextualising andconnecting drug, target, and disease data to deliver normalized, discoverable and model-ready information.Entellect allows researchers to produce far more accurate predictive models across arange of pre- and post-market activities, including drug efficacy studies, risk-benefitanalyses and pharmacovigilance activities.Cannabis now availableon prescription in UKPatients with exceptional clinical need in England, Wales andScotland can now be prescribed cannabis-based medicines for thefirst time.UK specialist clinicians can now freely prescribe cannabis-basedmedicinal products for patients with exceptional clinical need byrelying on their own expertise rather than having to apply for aspecial license.Professor Marie Fallon of The University of Edinburgh said: “TheUK’s rescheduling of cannabis-based medicine has come about froma groundswell of patient need but is rooted in scientific evidence andexperience.“There is a significant real-world experience and clinical evidence pointing to the fact that cannabis-based medicines can be safe andeffective treatments for some patients with unmet clinical needs, for instance in the areas of cancer pain and chemotherapy inducednausea & vomiting.”© Shutterstock / MaximP© Shutterstock / Apple’s Eyes Studio© Shutterstock/Lifestyle discover04-07.qxp_Layout 1 04/12/2018 15:06 Page 3Eye onapprovalsEUROPEKeytruda recommended for use on Cancer DrugsFund Keytruda has been recommended by NICE for use on the UK’sCancer Drugs Fund for patients with a type of advanced skincancer. The recommendation applies to adults who havemelanoma that has spread to the lymph nodes, an important partof the body’s natural defence system. Keytruda would be offeredto patients who have already had surgery to remove the cancer.Called an adjuvant immunotherapy, it aims to remove any cancerleft behind.Kymriah recommended for young blood cancerpatients Young people with relapsed or refractory B-cell acutelymphoblastic leukaemia (ALL) will now have access to Novartis’sKymriah (tisagenlecleucel) through the UK’s Cancer Drugs Fundfollowing a recommendation from NICE. Tisagenlecleucel will beoffered to people under the age of 25 who have not responded tocurrent treatment or who have relapsed after stem cell transplant.The therapy, which involves taking a person’s own immune cellsand modifying them to fight their cancer cells, has the potentialto be a cure.Lilly’s migraine treatment approvalEli Lilly has gained European marketing authorisation forEmgality (galcanezumab) for the prophylaxis of migraine in adultswho have at least four migraine days per month. Galcanezumabis a humanised monoclonal antibody that binds to the calcitoningene-related peptide (CGRP), which plays a role in migraineattacks, blocking its downstream function. The authorisationfollows a positive opinion from the Committee for MedicinalProducts for Human Use (CHMP) that was received in September2018.EU nod for Vertex’s cystic fibrosis combo Vertex Pharmaceuticals has secured European marketingauthorisation for Symkevi (tezacaftor/ivacaftor) incombination with ivacaftor for treating people with cysticfibrosis aged 12 and older with certain mutations in the cysticfibrosis transmembrane conductance regulator gene. In addition,the European Medicines Agency’s Committee for OrphanMedicinal Products recently recommended themaintenance of orphan designation fortezacaftor/ivacaftor.UNITED STATESSobi secures first US approval for HLH treatmentSobi has gained US approval for Gamifant (emapalumab-lzsg), the first and only treatment for haemophagocyticlymphohistiocytosis (HLH). Primary HLH is an ultra-raresyndrome of hyperinflammation with high morbidity andmortality and for which there was previously no approved drug.Gamifant represents a major advance in the treatment of thesepatients through a targeted mode of action.AcelRx secures approval for acute pain treatmentAcelRx Pharmaceuticals has secured the approval of USregulators for its acute pain treatment Dsuvia. Dsuvia isindicated for the management of acute pain in adults that issevere enough to require an opioid analgesic in certifiedmedically supervised healthcare settings – such as hospitals,surgical centres and emergency departments. The commerciallaunch of Dsuvia is slated for the first quarter of 2019.FDA approves first and only single-dose oral flumedicineGenentech has picked up FDA approval for Xofluza, itstreatment for acute, uncomplicated flu in people 12 years ofage and older. Xofluza is a first-in-class, single-dose oralmedicine with a novel proposed mechanism of action thatinhibits polymerase acidic endonuclease, anenzyme essential for viral replication. Ithas demonstrated efficacy against awide range of influenza viruses,including oseltamivir-resistant strainsand avian strains, in non-clinicalstudies.Epidiolex cannabinoid nowavailable on prescription in US Epidiolex oral solution, the first FDA-approved plant-derived cannabinoidmedicine, is now available onprescription in the US. The medicineis now available in the US fortreating seizures associatedwith Lennox-Gastautsyndrome (LGS) or Dravetsyndrome in patients twoyears of age or older. www.pbiforum.netPharma Business International 704-07.qxp_Layout 1 04/12/2018 15:06 Page 4M&A ROUND-UP8Pharma Business International www.pbiforum.net© Shutterstock /MichaelJayBerlin08-11.qxp_Layout 1 04/12/2018 15:07 Page 1Pharma Business International 9www.pbiforum.netM&A ROUND-UPAn increasingly cautious outlook saw aslowdown in the pharmaceutical mergersand acquisitions market going into 2018,which seemed to be floundering followingthe frenzied rate of deals we’d witnessedin the previous year. However, 2018’s firstbillion deal came after only a few weeksinto the year after Celgene swooped forImpact Biomedicines in a deal worth asmuch as $7 billion. That breaks down tothe initial upfront payment of $1.1 billion,contingent payments of $1.4 billionbased on regulatory milestones, andsales-based payments capped at $4.5million. Though companies were playing theircards closer to their chests, 2018 wasevidently off to a good start. Certaintywhere Celgene was concerned, who onlyweeks after announcing its intensions toacquire Impact Biomedicines, revealed itwas buying CART and TRC specialistJuno Therapeutics for $9 billion. It’s testament to the strength of themarket in the January that Celgene’simpressive double deal wasovershadowed by Sanofi. The Frenchpharma giant swooped for haemophilia-focussed biopharma outfit Bioverativ in adeal worth $11.6 billion which completedin March. Just under a week later, thecompany confirmed that it was buyingnanobodies specialist Ablynx for €3.9billion as it looked to home in ontechnologies addressing multiple diseasetargets with single multi-specificmolecules. The billion-dollar deals continued intoFebruary with Mallinckrodt completing itsacquisition of Sucampo Pharmaceuticals.The $1.2 billion deal saw Sucampobecame a wholly-owned subsidiary.Shortly after, Roche swooped for2018 under themicroscope Pharma Business International looks back over the last year, charting the biggestdeals in the mergers and acquisition market. 11 Á08-11.qxp_Layout 1 04/12/2018 15:07 Page 2Next >