Your guide to the pharmaceutical world Volume 6 Issue 4Clinical trialsGetting better resultsOncologySeek and destroyRegulationRegulating a post-BrexitBritainPharmaBUSINESS INTERNATIONALThe total packageFrom development to dispensingThe total packageFrom development to dispensingClinical trialsGetting better resultsOncologySeek and destroyRegulationRegulating a post-BrexitBritainwww.pbiforum.netORGANISED BY25-27 SEPT 2018 NEC, BIRMINGHAM PROCESSING & PACKAGING MACHINERYMATERIALS I ROBOTS I INDUSTRIAL VISIONppmashow.co.ukTHE UK’S LEADING PRODUCTION LINE EVENTSee the latest processing and packaging machinery in actionFREETO ATTENDREGISTER NOW!NEW FOR 2018Join an inɓuential line-up of keynote speakers at our must attend seminarsNetwork with industry peers @pbiforumwww.pbiforum.netcompany/pharma-business-internationalContentsVolume 6 Issue 4News4Stay up to date with the latest industry updates fromthe global pharmaceutical technology industry.Biosimilars8 With the launch of its latest action plan, it looks asthough the Food and Drug Administration arethrowing their weight behind the fledgling biosimilarsmarket. Pharma Business International instigates. M&A Round-up12 It’s the season for deal completion with some of thebiggest pharma companies closing multi-billion-dollardeals. Oncology16 Immunotherapy is harnessing the cancer killingpotential coded within our own DNA to help treatpatients. Here biotechnology and oncology cometogether for some of the most exciting breakthroughs,developments and studies taking place in the field ofmodern medicine. Ethics20 The latest scandal to rock the Chinese pharmaceuticalindustry has ruptured public faith in government andsparked a global discussion on ethics in the supplychain. Regulation24 The repercussions of Brexit and the absence of theEuropean Medicines Agency means Britain’spharmaceutical regulation might get morecomplicated. Clinical Trials28 The launch of a new pharmaceutical or medicalproduct is a long road paved with exhaustive, oftenstrenuous research and development. As one of thekey stages, clinical trials are a proving ground wherenew therapies, treatments and medicines can bevalidated or discovered to be a dud. In any outcome,they provide researchers, clinicians and scientists withvaluable insight that will shape future researchendeavours and the fate of new product launches. Events32We give a rundown of pharma events happeningaround the world.481620Group 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 /Image Point Fr FDA approves first oral targetedtherapy for AMLTibsova has become the first oral, targetedtherapy for adult patients for relapsed or refractoryacute myeloid leukaemia (R/R AML) with an IDH1mutation approved in the US.David Schenkein, CEO of Agios, the makers ofthe treatment, said: “The FDA approval of Tibsova –our first wholly owned drug and the secondapproved medicine from our research platform inless than a year – is an incredibly exciting milestonefor our company and, importantly, for theapproximately 6-10% of AML patients with an IDH1mutation who have been waiting for new treatmentoptions that work radically different thanconventional chemotherapy.”The approval was based on the clinical data froman open-label, single-arm, multicentre dose-escalation and expansion trial of adult patients withR/R AML and an IDH1 mutation.Tibsova was approved concurrently with theAbbott RealTime IDH1 companion diagnostic testfor selection of patients with R/R AML for treatmentwith Tibsova.4Pharma Business International www.pbiforum.netLatest newsJ&J ordered to pay $4.7bn intalc caseJohnson and Johnson, the US consumer goods and pharmaceuticalgiant, has been ordered to pay $4.7 billion in damages to twenty-twowomen alleged to have developed ovarian cancer after using its talcproducts.This comes as the company wrangles with around 9,000 legal casesinvolving its baby powder product. Of the aforementioned women, sixhave died from ovarian cancer.The company said it plans to appeal.Pfizer organises companyinto three businessesPfizer is reorganising the company into three businesses aspart of growth plans.The new businesses include the science-based InnovatedMedicines, which will now include biosimilars and a newhospital business unit for anti-infectives and sterile injectables.Secondly, Pfizer will establish an off-patent branded andgeneric Established Medicines business operation withsubstantial autonomy. Thirdly, there’s a Consumer Healthcarebusiness.Pfizer said the changes will take effect at the beginning ofthe company’s 2019 fiscal year.© Shutterstock / senee sriyota© Shutterstock / pio3© Shutterstock / Medtech THAI STUDIO LAB 249 Pharma still heavilyinvesting in UK R&D despiteBrexitThe pharmaceutical industry continues to significantly invest in UKresearch and development despite the future uncertainty of Brexit,according to data from the Association of the British PharmaceuticalIndustry (ABPI).The data shows industry spent £370.9 million on partnerships relatingto research and development activities in the UK during 2017 – a 9.7%increase on 2016.Spending on R&D activities accounts for three quarters of the totalspending disclosed on the database for working in partnership withleading UK health experts and organisations to improve patient care.“It is an encouraging testament to the pharmaceutical industry’scommitment to the UK as a hub of science and innovation that, in thewake of Brexit uncertainty, it continues to invest significantly in researchand development as shown in this latest disclosure data,” said ABPI ChiefExecutive Mike Thompson.Pharma Business International 5www.pbiforum.netLatest newsDrug aimed at Parkinson’s couldtreat heart failureResults from early animal studies suggests that a drug currently in clinical trialsfor treating symptoms of Parkinson’s may someday be used for treating heartfailure.Researchers from Johns Hopkins Medicines found that ITI-214 shows promisingeffects on dog and rabbit hearts, as well as on isolated rabbit heart cells, mostnotably an increase in the strength of the heart muscle’s contractions.Currently, dozens of drugs are available to treat or manage heart failuresymptoms, but drugs that improve the strength of the heart muscle’s contractionscarry the risk of dangerous complications such as developing an irregularheartbeat.However, in their study – published in Circulation– the researchers demonstratethat the new compound works differently than current drugs, suggesting its usemay be a safer way to increase heart contraction strength.It has already passed phase 1 safety trials in healthy individuals.First new drugapproved for malariain over 60 yearsThe US FDA has granted Priority Review to GSK and Medicines forMalaria Venture (MMV) for Krintafel for the radical cure of Plasmodiumvivax (P. vivax) malaria in patients aged 16 years and older.David Reddy, CEO of MMV, said: “The US FDA’s approval ofKrintafel is a major milestone and a significant contribution towardsglobal efforts to eradicate malaria. The world has waited decades for anew medicine to counter P. vivax malaria relapse.”He added: “Moreover, as the first ever single-dose for thisindication, Krintafel will help improve patient compliance.”The approval was based on efficacy and safety data from acomprehensive global clinical development P. vivax radical cureprogramme designed in agreement with the FDA.© Shutterstock / Manjurul Haque© Shutterstock / Gorodenkoff© Shutterstock / BluskystudioAxovant licences gene therapy programme aimed at musculardystrophyAxovant Sciences has licensed exclusive global rights to aninvestigational Silence-and-Replace gene therapy program from BenitecBiopharma for the treatment of oculopharyngeal muscular dystrophy(OPMD).The company has also entered into a research collaboration for thedevelopment of five additional gene therapy products in neurologicaldisorders.The Silence-and-Replace gene therapy technology is designed todeliver a combination of DNA-directed RNA interference (silence) alongwith a functional copy of the gene (replace) in a single vector construct.This approach is applicable to various genetic diseases, includingautosomal dominant disorders caused by nucleotide repeat expansion.The lead program – AXO-AAV-OPMD – is in preclinical developmentand Axovant plans to initiate a placebo-controlled clinical study in 2019.6Pharma Business International www.pbiforum.netLatest newsPromising ALS therapyimpresses in clinical trialsNew research led by Washington University School of Medicineindicates an investigational therapy for an inherited form of amyotrophiclateral sclerosis (ALS) extends survival and reverses signs ofneuromuscular damage in mice and rats.The findings, published in The Journal of Clinical Investigation, haveled to a phase one/two clinical trial to investigate whether the drug couldbenefit people with ALS whose disease is caused by mutations in a genecalled SOD1.In collaboration with Ionis Pharmaceuticals, Timothy Miller, the DavidClayson Professor of Neurology at Washington University, and colleaguestested DNA-based compounds that block the body from making SOD1protein.They tested two such compounds – known as antisenseoligonucleotides, or oligos for short – in mice and rats.“The phase one/two trial is really still a safety trial,” Miller said. “But we’re on the cusp of testingthe hypothesis that people with ALS caused by mutations in SOD1 can benefit fromthis treatment.”FDA approves first drug with indicationfor smallpoxThe US FDA has approved SIGA Technologies’ TPOXX (tecovirimat),the first drug with an indication for treatment of smallpox.Though the World Health Organisation declared smallpox eradicated in1980, there have been longstanding concerns that it could be used as abioweapon.“To address the risk of bioterrorism, Congress has taken steps toenable the development and approval of countermeasures to thwartpathogens that could be employed as weapons. Today’s approvalprovides an important milestone in these efforts,” said FDACommissioner Scott Gottlieb.“This new treatment affords us an additional option should smallpoxever be used as a bioweapon. This is the first product to be awarded aMaterial Threat Medical Countermeasure priority review voucher.”The safety of TPOXX was evaluated in 359 healthy human volunteers without a smallpox infection. The FDA granted this application Fast Track and PriorityReview designations. TPOXX also received Orphan Drug designation.© Shutterstock / fotovapl© Shutterstock / Marcelo Ricardo Daros © Shutterstock / vchalTakeda advancing Ambys pipeline forserious liver diseasesTakeda Pharmaceutical has entered into partnership with biotech Ambys Medicine to advancethe latter’s platform and pipeline.Ambys is pioneering the application of novel modalities, including cell and gene therapy andgain-of-function drug therapy, to meet the urgent need for treatments that restore liver functionand prevent the progression to liver failure across multiple liver diseases that are untreatable orpoorly treated today.Takeda said this is aligned with its strategy in gastroenterology to focus on the considerableunmet need in liver through significant investment in novel chemistry and cell/gene therapyplatforms.To that end, Takeda has committed $100 million, including participation in the Series Afinancing. In return, it receives an option to ex-US commercialisation rights for the first fourproducts that reach an investigational new drug application created under the partnership.If Takeda elects to exercise an option for a product, it will share in 50% of the developmentcosts for any optioned program and will make development and regulatory milestone payments. Pharma Business International 7www.pbiforum.netLatest newsUK launch £10m researchcompetition to combatantimicrobial resistanceThe UK Government is launching a £10 million researchcompetition to fund innovations aimed at antibacterial resistance(AMR) in humans.AMR is on the rise and poses a significant threat to healthacross the world. Without a better understanding of how to tackleand prevent AMR, treatable infections could become life-threatening.The competition follows the announcement of £30 million tofund research and development projects as part of the GlobalAMR Innovation Fund (GAMRIF) in May.The £10 million will be made available in research grantsfunded through a Small Business Research Initiative (SBRI).It is being run by Innovate UK on behalf of the Department ofHealth and Social Care, with the aim of supporting theimplementation of the UK Five Year Antimicrobial ResistanceStrategy.© Shutterstock / i viewfinderResearchers developing method ofcreating side effect free medicinesResearchers at the University of Virginia School of Medicine are paving theway for side-effect free medicines after developing a new technique for preciselytargeting moleckes within cells.Researcher J. Julius Zhu and his colleagues have developed a way tomanipulate molecules from compartment to compartment within individual cells.Until now, drugs have targeted molecules in a very general way. If a moleculewas thought to be harmful, researchers might try to develop a drug to block itentirely.But Zhu’s new work, published in Neuron, highlights the downside of thatshotgun approach. Now, rather than trying to block a molecule regardless of itsmany functions, doctors can target a specific molecule doing a specific thing in aspecific location. That adds a new level of precision to the concept of precisionmedicine – medicine tailored exactly to a patient’s needs.Zhu thinks the technique will be useful for many different diseases, butespecially for cancers and neurological conditions such as autism andAlzheimer’s.© Dan Addison, University of Virginia School of Medicine© Shutterstock / i Magic MineJ. Julius ZhuBIOSIMILARSBiosimilars are a boon to medicine makers,helping to curtail any monopoly a companymight have over a product, which is prudentgiven the way in which big pharma is stillviewed by the public. As we publish this, theopioid crisis in the United States grows evermore intense, and President Trump is bringingmajor pharmaceutical companies to task overTwitter for rising drug prices. From a pragmatic point of view, biosimilarsmake sound business sense and the proof isin the purchasing, with major players such asNovartis and Pfizer operating in this growingmarket. According to Infoholic Research, theglobal biosimilar drugs market will reach$99.28 billion by 2024 and now the US Foodand Drug Administration (FDA) is throwing itsfull weight behind it. FDA Commissioner Scott Gottlieb has beennothing short of vocal in his crusade to lowerdrug prices. This, he says, will beaccomplished by introducing morecompetition into the biopharmaceuticalmarket. Part of his work in this arena hasseen him boost the biosimilar industry,helping to elevate it from this emerging fieldinto a critical part of the globalpharmaceutical market. It was only in 2015,after all, that regulators in the US approvedthe first biosimilar. The approval was grantedBoomingbiosimilars 10 Á8Pharma Business International www.pbiforum.netWith the launch of its latest action plan, it looks asthough the Food and Drug Administration is throwing itsweight behind the fledgling biosimilars market. PharmaBusiness International investigates. © Shutterstock /Frank Anusewicz© Shutterstock /Albert H. TeichNext >