EquityResearch3January2019COREBarclaysCapitalInc.and/oroneofitsaffiliatesdoesandseekstodobusinesswithcompaniescoveredinitsresearchreports.Asaresult,investorsshouldbeawarethatthefirmmayhaveaconflictofinterestthatcouldaffecttheobjectivityofthisreport.Investorsshouldconsiderthisreportasonlyasinglefactorinmakingtheirinvestmentdecision.ThisresearchreporthasbeenpreparedinwholeorinpartbyequityresearchanalystsbasedoutsidetheUSwhoarenotregistered/qualifiedasresearchanalystswithFINRA.PLEASESEEANALYSTCERTIFICATION(S)ANDIMPORTANTDISCLOSURESBEGINNINGONPAGE42.U.S.Small&MidCapBiotechnologyCompanyProfiles–AFocuson2019SentimentremainsquitenegativeinBiopharma,followingatough2018(NBI:-9%;DRG:+4%;S&P500:-6%),butlookingto2019,expectationsaremodestandthefundamentalbackdropissolid,whichsetsthegroupupwell.Inthisreport,weprovideone-pageprofilesforcompaniesundercoverageintheBarclaysGlobalTherapeuticsUniverseincludingUSBiopharma,USSMidCapBiotechandEUPharma,highlightingkeyevents,potentialvaluedrivers/catalystsandwherewedifferfromconsensus.Ourthoughtsonbroaderindustry-specifictrendsforthisyearcanbefoundinouraccompanyingreport,The2019GlobalBiopharmaOutlook,alsopublishedtoday.USSMidCapBiotech(Wang)-FavoriteNames:RGNX,SRPT,BLUE.ForRegenxbio,weseemultipleupsidepotentialsin2019includinghigherroyaltyfromNovartis’AVXS-101inSMA(higherpricing,robustlaunchandsuccessfulIPextension),positive4thcohortwetAMDdata,andprogressfromotherpartnerprograms.ForSarepta,whilecautiousforitsDMDgenetherapyprogram,webelieveSareptasetsahighbarwithlikelymeaningfulfirsttomarketadvantage;additionalupsidepotentialfrommultiplecatalystsincludingPPMO,LGMD,andgolodirsenapproval.ForBluebirdBio,despiteincreasingcompetitioninBCMA-targetedtherapiesinMM,wecontinuetoviewbb2121asthebestandlikelythefirsttomarketwithastrongpartner.WeseesignificantupsideifCelgeneremainscommittedtomovebb2121toearlierlinesinMMandadditionalupsidewithhigherpricingofLentiGlobininBT/SCD.LargecapBiopharma(Meacham)–FavoriteNames:ALXN,CELG,LLY,SGEN.ForAlexion,la...