< PreviousALS EXPOSÉ10Pharma Business International www.pbiforum.netThere is currently no effective means oftreating the disease and a cure remainselusive. Although ALS isn’t technicallyclassed as a terminal illness, its diagnosisis, in most cases, tantamount to a deathsentence. Not content with acceptingthat awful state of affairs, scientists andresearchers are hard at work trying todevise ways of managing, treating and,eventually, curing the disease. In fact,there have been a number ofbreakthroughs over the year. Below we’llexplore some of the more excitingdevelopments. One of the most promisingdevelopments of late was one funded byproceeds from the Ice Bucket Challenge.A study spearheaded by a University ofCalgary researcher is investigating apotential drug treatment for the disease.A phase II clinical trial across ninehospital centres in Canada will recruitpatients to confirm whetherantipsychotic medication pimozide issafe and effective in stabilizing theprogression of ALS over a six-monthperiod. The trial follows promisingresults from a collaboration betweenUniversity of Calgary and researchers atthe University of Montreal. Using pimozide, science researchers inMontreal were able to stabilize mobility inanimal models — worms, fish and mice— born with the equivalent of the humanform of ALS. Pimozide works especiallywell in preventing paralysis in fish with agenetic form of ALS by preservingneuromuscular function, a key findingwith relevance to ALS in humans, whichnormally involves a progressive paralysisof the skeletal muscles. However,researchers are quick to point out that it’stoo soon to draw firm conclusions aboutthe safety and effectiveness of the drug,but these early signs are definitelyencouraging. As well as effectors from the academiaspace, the pharmaceutical industry isalso behind some of the recentdevelopments. For example, Pfizer iscollaborating with Sangamo Therapeuticson the development of a zinc fingerprotein gene therapy aimed at ALS aswell as fellow neurodegenerative disorderfrontotemporal lobar degeneration(FTLD).The biotech innovation space will alsolikely prove critical in tackling thedisease. To that end, Biogen has recentlyacquired Karyopharm Therapeutics’investigational oral SINE compound KPT-350 and other assets targeting ALS andother neurological conditions. Almost 500,000 people worldwide arebelieved to live with ALS, indicating thepressing need for a way of managing,treating and curing the disease.Breakthroughs from industry andacademia will continue to yield promisingresults in the years to come, workingtowards a silver bullet for sufferers andtheir families. © Shutterstock / Kateryna Kon08-11_Layout 1 13/02/2018 09:06 Page 3© Shutterstock / Twocoms08-11_Layout 1 13/02/2018 09:06 Page 4M&A ROUND-UP12Pharma Business International www.pbiforum.netOur December issue took a briefwhistle-stop tour of 2017’s global M&Amarket, chronicling the big deals thatkicked off the year, before cautionsaturated the market and deals began tocool. However, a few big deals crept inbefore the year’s end, and we’d beremiss without covering them.One of the last major deals of 2017saw UK-headquartered Mallinckrodt snapup biopharma outfit SucampoPharmaceuticals for $1.2 billion. At thetime, CEO and President Mark Trudeausaid the acquisition was the “latestmilestone towards our vision ofbecoming an innovation driven specialitypharmaceutical growth company”. In terms of total cost, the above dealwas topped offering one last huzzah toround off 2017. We’re talking, of course,about Roche’s $1.7 billion acquisition ofIgynta, which gave the company accessto Igynta’s lead molecyde entrectinib –an orally bioavailable, CNS-activetyrosine kinase inhibitor being developedfor tumours that harbour ROS1 or NTRKfusions. Roche said the deal will allow itto broaden its oncology portfolio globally. Moving into 2018 and the deals keepon coming. It’s worth noting that the firstfew weeks of the year didn’t replicate thefrenzy seen twelve months earlier. This isdue in no small part to an increasinglycautious outlook. One of the earliestdeals of note saw Takeda Pharmaceuticalswoop for biopharma TiGenix for €520million in a bid to expand itsgastroenterology pipeline. The deal,described by Takeda as a “naturalextension” of its existing partnershipagreement with TiGenix, aims to bringnew treatment option to patients withgastrointestinal disorders. 2018’s first billion dollar deal followedsuite, and arrived only one week into theyear, with Celgene acquiring ImpactBiomedicines. As well as an upfrontpayment of $1.1 billion, the deal alsoincludes up to $1.4 billion in contingentpayments based on regulatorymilestones. Celgene confirmed it will alsomake payments based on sales – with amaximum of $4.5 billion. All in all, thedeal could be worth as much as $7billion. As if that wasn’t enough, Celgenerevealed only weeks later that it wasacquiring CART and TRC therapeuticsspecialist Juno Therapeutics for $9billion. Celgene CEO Mark J Alles said atthe time: “The acquisition of Juno buildson our shared vision to discover andWhat’s newin M&APharma Business International checks in with theglobal M&A market, highlighting some of the biggerdeals to have taken place over the last month. 12-13_Layout 1 13/02/2018 09:08 Page 1Pharma Business International 13www.pbiforum.netM&A ROUND-UPdevelop transformative medicines for patientswith incurable blood cancers.”Despite Celgene’s impressive deals inJanuary, the month was dominated by Sanofi.Announced towards the end of the month,the company reported it was acquiring all theoutstanding shares of haemophilia-focusedbiopharma outfit Bioverativ in a deal worth$11.6 billion. Not even a week later, thecompany confirmed another acquisition – thistime taking over nanobodies specialist Ablynxfor €3.9 billion. Sanofi said the acquisitioncontinues its “commitment to breakthroughinnovation, focused on technologiesaddressing multiple disease targets withsingle multi-specific molecules”. That wraps up the major billion dollar dealsfor January, but there were a few smallerdeals of note that might have slipped underyour radar. Astellas Pharma completed its$225 million acquisition of Mitobridge whichhas now become a wholly-owned subsidiary.One of the final deals of the month sawSeattle genetics snap up CascadianTherapeutics in a $614 million deal thatenhances the company’s oncology pipeline. February has a lot to live up to in order tosustain the deals observed during the firstfive weeks of the year. Look out for our nextissue where we’ll highlight the biggest dealsthroughout February and March. © Shutterstock / Atstock Productions© Shutterstock / Marian Weyo12-13_Layout 1 13/02/2018 09:08 Page 2COLLABORATION14Pharma Business International www.pbiforum.netFosteringinnovation Collaborations bring together an immense level of skill and expertise of a number of companies, or through links withacademia, which can have a huge impact on the sector and the patients its serves. 14-17_Layout 1 13/02/2018 09:09 Page 1Pharma Business International 15www.pbiforum.netCOLLABORATIONCollaborations are commonplace in thepharmaceutical sector, with majorcompanies partnering with one anotheron research or commercialisationprojects, or forging links with smaller,innovative start-ups to develop newtechnologies or drug products. It’s alsocommon for companies to forge vitallinks with academic organisations anduniversities, where ground-breakingresearch is often taking place. The ability to merge the specialistareas of two or more individualcompanies ensure they can punch wellabove their weight on a global scale. Inmany cases, collaboration allows R&D tobe commercialised under the auspices ofa major manufacturer. Collaboration is usually undertakenwith a clear view for profitability in sight,especially where two manufacturers arepooling potentially valuable knowledgethat needs to be protected. This is nowalways the case, however, and someorganisations - noticeably in the medicalresearch field - instead pool theirknowledge and resources to create arepository of information available toothers. Such collaboration is of keyimportance to not only the industry as awhole, but also the future ofpharmaceutical research. In an era wherepathogens are becoming increasinglyresistant to standard antibiotics, researchinto new treatments is only going tobecome increasingly difficult and thusincreasingly expensive. Collaborationsare sometimes the only way for individualorganisations to punch above theirweight. Often, collaborations are of means ofproviding a pharma company with anextension of its portfolio or services. Forexample, a company developing aninnovative new treatment might wish topartner with a manufacturer developingan equally innovative delivery system.That’s just what Japanese© Portal Instruments16 Á14-17_Layout 1 13/02/2018 09:09 Page 2COLLABORATION16Pharma Business International www.pbiforum.netpharmaceutical company Takeda hasdone. Takeda is collaborating withMassachusetts-based Portal Instruments’where it will leverage the company’sneedle-free drug delivery device as ameans of delivering its owninvestigational or approved biologicalmedicines. The Portal device was developed at theMassachusetts Institute of Technology(MIT) in the laboratory of Professor IanHunter. It has the potential forapplications across a range of biologicmedicines that currently requireadministration through an injection. Thedevice delivers the biologic through apressurised liquid instead of a needle,and has been clinically shown to be lesspainful and preferred by patientscompared to a standard needle-basedinjection.The first Takeda development programto potentially utilise this device will be forinvestigational use with Entyvio – amonoclonal antibody for adults withmoderately to severely active ulcerativecolitis (UC) or Crohn’s disease (CD),which is currently administered throughintravenous infusion.A Phase III clinical trial program iscurrently evaluating the efficacy andsafety of a subcutaneous formulation ofvedolizumab in adults with moderately toseverely active UC or CD.This is one of hundreds of examples ofcollaboration in action across thepharmaceutical sector, where innovationis fostered and new developments arebeing created and ushered into markethelping push forward our collectiveunderstanding and to help patients. © Shutterstock / docent14-17_Layout 1 13/02/2018 09:09 Page 3© Shutterstock / ASDF_MEDIA14-17_Layout 1 13/02/2018 09:09 Page 4TRACEABILITY18Pharma Business International www.pbiforum.net© Shutterstock / Alexander Yakimov18-21_Layout 1 13/02/2018 09:09 Page 1Pharma Business International 19www.pbiforum.netTRACEABILITYEffectively a tamper-proof digital ledger,blockchain establishes a trusted environmentfor transactions allowing all participants fromacross the supply chain to gain authorisedaccess to information. Its most ardentsupporters have declared that using blockchainwill usher in a safer, securer and betterconnected supply chain, but others havepointed out that, benefits aside, blockchain isanother tool which must be correctly used inorder to see any kind of result. The food industry has become one of thebiggest sectors to throw its weight behind thetechnology, with the pharmaceutical sectormore hesitant in comparison. But there aresome encouraging early signs that pharma islooking towards blockchain and the traceabilitygains it can bring, with the technology tippedas one of 2018’s biggest sector trends. There’s already a number of exciting projectsunderway to upscale, explore and applyblockchain to the pharmaceutical supply chain.Among those is the Institute of Electrical andElectronics Engineers’ on-going project ThePharma Blockchain initiative. The project isexploring how emerging technologies can beapplied in ways that will optimise processacross the pharmaceutical network. Althoughemphasis is, of course, on improving industryefficiencies, the project’s end goal is onpatient care and safety. GSK is one of the first big pharmacompanies to dip its toe into the technologyhaving announced in January its intentions tocollaborate with Viant – a blockchain platformbacked by open-source distributedcomputing platform Ethereum. Together thetwo organisations with collaborate withseveral others – including Microsoft and TheWorld Wildlife Fund – to boost thedeployment of blockchain-based supplychain systems. But what exactly does thismean for the industry? Well, blockchain couldhelp managed complicated supply chains,such as biopharma and biotech, and provideLinkingthe chain Blockchain has been dominating headlines over the last fewmonths with nary a conversation about traceability passingwithout mention of this disruptive technology. But what canblockchain do for pharma?20 Á18-21_Layout 1 13/02/2018 09:10 Page 2Next >