转折点：临床试验结果如何影响生物制药估值（英）

WhitePaper InflectionPoint:HowClinicalTrialResultsImpactBiopharmaValuations Understandingthedriversofvaluecreationtonavigateanoptimaldevelopmentpath MARKUSGORES,VicePresident,EMEAThoughtLeadership,IQVIA WILLIAMHARRIES,SeniorConsultant,EMEAThoughtLeadership,IQVIA FRANCESCOCAPUZZI,SeniorConsultant,EMEAIntegratedResearch,IQVIA Tableofcontents Introduction2 Methodology:briefoverview3 Theasymmetryofmarketresponse3 Driversofvaluationimpact:therapyareas,trialdesigns,companysize6 Considerationsformanagementteams11 Closingthoughts13 Appendix:additionalmethodologydetails14 References16 Abouttheauthors18 Disclaimer Theanalyses,theirinterpretation,andrelatedinformationcontainedhereinaremadeandprovidedsubjecttotheassumptions,methodologies,caveats,andvariablesdescribedinthisreportandarebasedonthirdpartysourcesanddatareasonablybelievedtobereliable.Nowarrantyismadeastothecompletenessoraccuracyofsuchthirdpartysourcesordata.Noinvestmentadviceisgiven. Introduction Developingnoveltherapiestoaddressunmetpatientneedisthelifebloodofthebiopharmaceuticalindustry.Itisalsoacapital-intensiveandhigh-stakesendeavour,requiringanestimated$3.1billiontobringanewtherapytomarket,1withacompositesuccessrateof11%fromphase1throughregulatorysubmission.2 Clinicaltrialreadouts,therefore,representmajorinflectionpointsinthisjourney,asmomentsoftruth,thatresolveuncertaintyaroundanasset’sfutureprospects,includingitsultimatepotentialforrevenuegeneration.Consequently,companyvaluationsrespondtoclinicaltrialresults—positiveandnegative —asinvestorsre-calibratetheirexpectationsbasedonthenewinformationbecomingavailable.3-6 Inthiswhitepaper,wewillsystematicallyinvestigatehowclinicaltrialoutcomesimpactcompanyvaluationsandexploretheunderlyingdrivers,suchashowtrialresultscomparetoinvestors’priorexpectations,developmentphaseatreadout,therapyareaortrialdesign.Wefocusonemergingbiopharmacompanies(EBPs),inparticularthosewith<$1billionmarket capitalisation,becausetheirvaluationsarehighlyresponsivetotrialresults,asmostoftheirvalueisconcentratedintheirpipelinewhichoftencomprisesjustasingleasset.Unsurprisingly,wefoundthatEBPvaluationsaremuchmoresensitivetoclinicaltrialreadouts,byuptotwoordersofmagnitude,comparedtobigpharmacompanies. Furthermore,wewillelaborateonthepracticalimplicationsofunderstandingthosedriversofvalueinflection.Forexample,howsuchinsightmayinformstrategicdecisionsandhelpmanagementteamsnavigateacompany’soptimalpaththatbalancesvalueupsidevs.incrementalclinicalrisk,suchastheoptimaltimingforexploringpartnershipsorwhentopursueanexitviatheM&Aroute. iqvia.com|2 Methodology:briefoverview IQVIAperformedaneventstudyanalysisthatquantifiedthesharepricereactiontoclinicaloutcomesformorethan2,600trialsfrom2017to2023. Ouranalysisdefinestheeventdateastheprimaryendpointreporteddate,i.e.,theearliestdateofpublicreportofresultsthataddressestheprimaryendpointsofthetrial.Positiveandnegativeoutcomesattheprimaryendpointreporteddatewereallocatedbasedonthecompany-reportedclinicaldefinition. Arobuststatisticalmodelwasdevelopedtoanalysethechangeinsponsorcompanysharepriceattheprimaryendpointreporteddate.Thechangewascalculatedastheaverageattheclosepricesoftwodayspriorandonedaypriortotheprimaryendpointreporteddateversustheaverageattheclosepricesonthedayoftheeventandthedayafter. Sponsorcompaniesandtherapeuticareas(TAs)wereallocatedbasedonIQVIAofficialclassifications. Furthermethodologicaldetailsaredocumentedintheappendix. Theasymmetryofmarketresponse Amainfocusofouranalysiswasunderstandingthemarketresponsetopositiveandnegativeclinicalresultsfordifferenttrialphasesalongtheclinicaldevelopmentpath. Weobservedanintriguingasymmetryinthestatisticallysignificantimpactofpositivevs.negativeclinicaltrialreadoutsoncompanyvaluations,withnegativetrialresultsconsistentlycausingalargerrelativemarketreactionthanpositiveresults.Thispatternholdstrueforalltrialphases. Specifically,wefoundthattheimpactofnegativenewswas2.3,1.3and2.0timeshighervs.positivenewsforclinicaltrialphases1,2and3,respectively,implying amostfavourablerisk-rewardprofileforphase2readouts(seeFigure1). Thisconsistentasymmetryobservedacrossalltrialphasessuggeststhatinvestorsgiveinnovatorsthebenefitofthedoubt,onthebasisofrisk-adjustedexpectations.Consequently,anyvalueupliftfollowing Figure1:Theasymmetryofmarketresponse Trialresults ImpactofclinicaltrialresultsonEBPvaluations,bytrialphase (%changeinshareprice;allresultsarestatisticallysignificantatthe95%confidencelevel) Phase1Phase2Phase3 NEGATIVEPOSITIVE 12%11% 3% -7% -16% -22% Risk/reward profile* 2.3 1.3 2.0 Basedonreadoutsofover1,500EBP-sponsoredclinicaltrials *Ratioofabsolutenegativeimpact/positiveimpactSource:IQVIAEMEAThoughtLeadershipanalysis positiveclinicalnewsfromananticipatedeventislesspronouncedasitconfirmspriorassumptionsthatarealreadysomewhatreflectedinacompany’sshareprice. Conversely,negativetrialresultsdefypriorexpectationsandthusdestroysignificantvalue.Takenbysurprise,investorsfundamentallyrevisetheirexpectations,includingreducingpeaksalesandpossiblyloweringassumed