DISCLOSUREAPPENDIXATTHEBACKOFTHISREPORTCONTAINSIMPORTANTDISCLOSURES,ANALYSTCERTIFICATIONS,LEGALENTITYDISCLOSUREANDTHESTATUSOFNON-USANALYSTS.USDisclosure:CreditSuissedoesandseekstodobusinesswithcompaniescoveredinitsresearchreports.Asaresult,investorsshouldbeawarethattheFirmmayhaveaconflictofinterestthatcouldaffecttheobjectivityofthisreport.Investorsshouldconsiderthisreportasonlyasinglefactorinmakingtheirinvestmentdecision.10February2019Americas/UnitedStatesEquityResearchBiotechnologyU.S.BiotechnologyCOMMENTResearchAnalystsMartinAuster,M.D.2123256573martin.auster@credit-suisse.comTiagoFauth2123257569tiago.fauth@credit-suisse.comMarkConnolly2123257576mark.connolly@credit-suisse.comModelUpdates■ModelChanges.Weupdatedourmodelstoincorporatequarterly2019projections.WedecreasedourTPforXLRN(from$60/shto$58/sh)andRARX(From$34/shto$32/sh).WealsorevisedourestimatesforBMRN,SRPT,UTHR,RARE,ONCE,ANAB,OBSV,FTSVandETTX.CategoryEarningsFocusAreasQ1CatalystsCommercialUpdatesProgramStatusUpdates10February2019U.S.Biotechnology2Figure1:CatalystsAcrossOurCoverageUniverseTickerCatalystTimingALXNALXN1210-gMGPhase3Trialtoinitiateearly2019ALXNOralProceedings-NewSolirisCoMPatentinEUPostponed,TBDALXNSYNT001Phase1b/2ainWAIHA1H2019ALXNSoliris-NMOUSLaunchanticipatedH22019ALXNSYNT001Phase3WAIHATrialtoinitiate2H2019ALXNALXN1210-ALSPoCTrial2019ALXNALXN1210-PPMSPoCTrial2019ALXNALXN1210-NMOPhase3Trialtoinitiate2H2019ALXNWTX-101Ph3CompleteenrollmentbyYE2019ALXNALXN1210IV100mg/mlFormulationLaunch2020ALXN1210WeeklySCFilingQ12021ALXNWTX-101Ph3Data~2021ALXNALXN1810SC(1210+PH20)PotentialLaunch2023BMRNBMN290INDSubmission2H2018BMRNPegvaliaseCHMPOpinionQ12019BMRNValroxPh3EnrollmentCompletion-6E13Q32019BMRNValroxPh2-2yrUpdate4E13;3yrUpdate6E13Mid-2019BMRNValroxDecisiononPotentialAcceleratedFiling2H2019BMRNBMN307(PKUGeneTherapy)INDFiling2H2019BMRNBMN250Ph1/2Update2019BMRNValroxPh3EnrollmentCompletion-4E132019BMRNVosoritidePh3ResultsYE2019BMRNValroxPh3ResultsMid-2020SRPTExon51Amenable(ExonSkipping)SRPTSRP-5051(...
