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AndrewQuickSr VPClinicalDevelopment
October6,2016
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Disclaimer– ForwardLookingStatements
Thispresentationmayincludeforward-lookingstatements.Youcanidentifythesestatementsbythefactthattheyusewordssuchas“anticipate”,“estimate”,“expect”,“project”,“intend”,“plan”,“believe”,“target”,“may”,“assume”orsimilarexpressions.
Theseforwardlookingstatementsspeakonlyasatthedateofthispresentationandarebasedonmanagement’sexpectationsandbeliefsconcerningfutureevents.Forward-lookingstatementsarenecessarilysubjecttorisks,uncertaintiesandotherfactors,manyofwhichareoutsidethecontrolofAvita Medicalthatcouldcauseactualresultstodiffermateriallyfromsuchstatements.
Avita Medicalmakesnoundertakingtosubsequentlyupdateorrevisetheforward-lookingstatementsmadeinthisreleasetoreflecteventsorcircumstancesafterthedateofthisrelease.
Thispresentationisintendedtoprovidebackgroundinformationonlyanddoesnotconstituteorformpartofanofferofsecuritiesorasolicitationorinvitationtobuyorapplyforsecurities,normayitoranypartofitformthebasisof,orbereliedoninanyconnectionwithanycontractorcommitmentwhatsoever.
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AGlobalPioneerinRegenerativeCellTherapy
AvitaMedical– CompanyBackground
o Platformautologouscellharvestingtechnologyaddressingunmetneedinclinicalindicationsencompassingarangeofskininjuriesanddefects
o PubliclytradedinAustralia(ASX:AVH)andUSADRs(OTCQX:AVMXY)o ProductsclearedformarketinginEU,Australia,Chinao U.S.HHSBARDAcontract(US$61.9m)forsupportofPMA&Pre-EUAactivity,USburncenter
familiarityandacceptance,andtoestablishanationalstrategicstockpileofReCell®formasscasualtypreparedness
o USFDAPivotalTrialforReCellinburnsfullyrecruitedandparticipantsarebeingfollowedo OperationalbuildcenteredontheLAoffice,asthehubofClinical,Quality,Regulatory,SupplyChain
Financial,andUSSales&Marketingactivities
AcuteWoundsRepairwithlessdonorskin
ChronicWoundsRestarthealing
AestheticsRestorepigmentation
Burn/Trauma Centers
Hospitals
Private Clinics
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SkinRegenerationPlatformo AutologousCellHarvestingDevicesusedtotreatwounds
andskindefects• ProprietaryEnzymeformulation,• Processingunitincludingsterileenzymesoak-,bufferrinse-
andfiltering- chambersandasteriletrayformechanicaldisaggregationofskinsample
• Validatedsetofapplicatorsdesignedtooverlaywoundareawithsuspensionofhealthycells
o AllowsrapidcreationofRegenerativeEpithelialSuspension™(RES™)• Disaggregated(activated/“free-edge”),autologousskin
cells(keratinocytes,fibroblasts,melanocytes)• Signalingfactors(cytokines,chaperoneslikehsp90,growth
factors)• Catalyzeregenerativehealinguponapplicationto
wound
Woodetal.2012.Burns 38:44.Singer&Clark.1999.NEJM.341(10):738.
Fast,Easy,SafeandEfficaciousOn-SiteSkinRegenerationSystem
VitalStatistics1. 1hourtolearndeviceuse2. 30minstocreateRES™3. Treatmentareais80x
donorarea
ReCell is an investigational device in the United States and limited by US federal law to investigational use.
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RES™Mechanism– supplyingskincells
o Autologoussamplesderivedfromhealthyareasoftheskincontainacompletemixofallskincells (non-cultured)andfactorstocatalyzethehealingprocess
o Cellsinsuspensionarenolongercontact-inhibitedbyneighbouringcells(unlikeintacttissue)andundergophenotypicchangestopromoteclosure(free-edgeeffect)
o ApplicationofRESovercomestheusuallimitedavailabilityofhealthy,signallingcells
Activated,Autologous,AvailableandComplete
Trim&Quick.2015JWoundTech27:20-24.Singer&Clark.1999.NEJM.341(10):738.F
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RES™Mechanism– “Activated”
KeratinocytesfromRES™inawoundbedmodelafterAutologousCellHarvesting*
1day 3 days 5days 7 days
• LargenumbersofviableskincellsinRES™adheretothewoundbedalmostinstantly• Proliferativeandmigratorymorphologiescanbeseenasearlyasday1• Rapidisolationandthenimmediateapplicationtotheidealincubator,thehumanbody,supportsnormalcellprocesses
*RepresentativespecimensroutinelyisolatedusingReCell® deviceatHuddersfieldSkinIntegrityInstitute
RES™IntactskinInvolucrin isasignallingproteinpresentinnormal,intactskin.Whenskincellsareinahealing(proliferative)state,involucrin expressiondecreases.
P-ERK isasignallingproteinthatisactivatedduringskincellproliferation,itplaysaroleinthecoordinationofrepair.p-ERKincreases duringhealing
β-Actin- Loadingcontrol
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RES™Mechanism– signalingfortissuerepair
o Dangerassociatedmolecularpatterns(DAMPs)– keytoregulationoftissuerepair• HSP90α andHMGB1arenaturallypresentin
RESasaresultofthecellharvestingprocess
o Secretedfactors- alsoimportantintissuerepair• VEGF,EGF,RANTES,IL-1α,TNF-α – allconfirmed
presentinRES™
HMGB1 30kDa
β-actin 42kDa
HSP90α 90kDa
#1 #2 #5#4#3
Time 0 Time 0 Time 0Time 0 Time 0WoundWound Wound Wound Wound
0
1
2
3
4
Day1 Day3 Day5 Day7
OD450n
m
IL-1α
0
0.1
0.2
0.3
Day1 Day3 Day5 Day7OD450n
m
RANTES
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ClinicalEvidenceDemonstratesBetterOutcomesinMultipleIndications
o Earlyproductapprovalsbasedoncaseseries,60+presentationsandpublicationstodate
o Morerecently,pursuingrobust,randomizedcontrolledtrials
ClinicalIndication RCT Readout
Burns ReCelladjuncttowidelyexpandedautografts,fortreatmentofmixed-depth(incl.full-thickness)burninjuries(US,CTP001-6)
FullClinicalDataPackageCompleteQ12017
ChronicWounds ReGenerCellfortreatmentofhard-to-healvenouslegulcers(UK,CTP003)
presented,2016EWMA
ChronicWounds ReGenerCellfortreatmentofchronicwounds(China)
published2015,BritishJSurg
Aesthetics/Repigmentation
ReNovaCellforrepigmentationofsegmentalvitiligo/piedbaldism (Netherlands)
published2015,JAmer AcadDermatol;newpublicationinprep
Aesthetics/Repigmentation
ReNovaCellforrepigmentationofhypopigmentedscar(Germany)
Published2016,Burns(JournalofInt’lSocietyforBurnInjuries)
ClinicalEvidenceBaseF
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ReCell®andMeshedAutograft– healingwithlessdonorskin
o Treatmentoflargesurface/deepburnswithlimiteddonorsiteusage• Addressesunmetneedinburncare• Designedforclinicaleffectivenesswithminimaldonorsiterequirement
• Patientwith64%burn,hospitallengthofstay0.58daysper%TBSA(vs1.9±0.7daysforseveritymatchedhistoricalcontrol)
TreatmentRES™ + Meshed Autograft
1 Month post treatment
Canreducelengthofstayinlargeburnsbyover50%
HolmesJH.2016BiennialMeetingoftheInternationalSocietyforBurnInjuries,Miami,FL
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Treatment: excision, and RES™
Post-operation, Day 9Deep Partial Thickness Burn
Post-Operation, 4 months
Courtesy of Ms Isabel Jones, Chelsea and Westminster Hospital
AchievingHealingand NormalAppearance
o CaseReport:48-year-oldman,flameburninjuryfromanexplodingboiler.TreatedatChelseaandWestminsterHospital
o Sub-optimaltouseskingraftsonfacialwounds
o ApplicationofRES™triggeredwoundhealingo Reintroductionofmelanocytesclearlygivessuperiorcosmeticoutcome
RES™onaburninjury– excellentoutcome
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AutograftSparingandEmergencyPreparedness
o Greatbenefitisassociatedwithuseoflessdonorskintoachievedefinitiveclosureandbetterlong-termoutcomesinburncare
o WeareworkingtoestablishReCell®asthego-to autograft-sparingtechnique,basedonclinicalandeconomicbenefito Autograftsparingreducestheburdenonthealready-injuredpatientfaced
withtheharvestingoftheirhealthyskino Autograftsparingpotentiallyreducesthenumberofproceduresandoverall
lengthofhospitalstayo Theneedforskinisakeybottleneckinmassresponse
o ReCell®isversatile,portableandself-contained
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TheBARDAContract
BARDAaddsresourcesandvalidationtoAvita
o AtotalofUSD$61.9mcommittedunderthefive-yearcontractawardedSept29
o BARDAislockedintopay$27.9mtocompletetheFDA-PMAprocess,ensureAvita ismarket-ready,andbuyaninitialinventoryofmorethan5,000devicestobestockpiled
o BARDAalsohasoptionstospend$34mmoreonlargerprocurement, andvariousstrandsofpost-marketentrysupport
o AvitaisalsoengagedwithotherbranchesoftheUSFederalgovernment:thedeviceisportable,flexibleandself-contained,andhasgreatpotentialformilitaryuse
o AvitaisnowusingBARDAfundstostrengthenitsoperationsandtodevelopclinicalandeconomicdatafortheburnscommunityinadvanceofaUSlaunch
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ProjectedU.S.ReCell® BurnsApproval:Q32017
InitiateCTP001-6
CompleteU.S.enrollment
PotentialFDAApproval*
ClinicalStudyReport,PMA
Submission
6months
1Q2015 1Q2016 1Q2018
LastSubjectLastFollow-up
1Q2017
FDAAdvisoryPanel
CompleteInitialPMAReview
*Estimated,timingsmayimprovebasedonFDA’s
ExpeditedAccessPathwaydesignation
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CaseStudy1:67yearoldfemalewithperipheralarterialdisease,controlledtypeIIdiabetesVLU(10cm2)onrightlateralmalleolusopenfor46weeks beforetreatmentwithReGenerCell.
CaseStudy2:70yearoldmalewithperipheralarterialdisease,controlledtypeIIdiabetes.RightmedialVLU(13cm2)openfor212weeksbeforetreatmentwithReGenerCell.
Baseline Week1 Week6 Week10 Week14
“Changed within a month, could see the change, getting smaller and not so deep. Pain was reduced after thecells were applied, no pain at all after week 4”
“[It’s] just a miracle. Got my life back, can go out and socialise. Three years ago I couldn’t walk 10 yards”
ReGenerCell™- ClosingWoundswhereotherroutesFailed
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ReGenerCell– venouslegulcerpilotRCThighlights
• Statistically significant improvements shown in wound size, pain and health-related quality of life
• Positive trends both in healing time, incidence of closure and all aspects of theQuality of Life questionnaire were observed. This was of particular note in largeulcers (over 10 cm2) which comprise the majority of VLUs
• Treatment using ReGenerCell™ definitively places the wounds on a healingtrajectory
*p=0.022
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Admission&Debridement
Courtesy of Dr Robert Manton, Charing Cross Hospital London
Treatment Potential only now being Realized
o CaseStudy:an85-year-oldwoman,suffered8cmx5cmgashonleftleg.TreatedatKingsCollegeHospital,Londono RES™appliedinanoutpatientclinic
o Patientreportedtobeverysatisfiedwiththeoutcome,whichallowedhertomaintainherindependentlifestyleo Pre-tibial Lacerationsareafrequentproblemforwomenagedover60,andarechallengingtotreat
ReNovaCellappliedafterone
weekofconventionalcare
ThreeweeksafterReNovaCell
treatment,25%reduction
Afterfiveweeks,75%reduction
After11weeks,100%healing
ReGenerCell™forpre-tibial laceration
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ReNovaCell:SimpleSolutionforSkinRepigmentationo Repigmentation ofhypo-pigmentedskinduetooldage,injury,skintreatments,vitiligo
• Mostsignificantunmetmedicalneedinaestheticdermatologyo CurrentInadequateTreatmentOptionsforRepigmentation
• Non-surgicaloptions“lotions&potions”andlighttherapysometimesefficacious• Lab-basedmelanocytetransferissolesurgicalchoicebutexpensive,timeconsuming
o ReNovaCell istheonlysimplified,cost-effectivesolutionforskinrepigmentation
Baseline 6-monthsfollow-up
RES™-treated
NegativeControl
From published RCT*: patient with segmental vitiligo (duration > 5yrs)
*KomenL,Vrijman C,Tjin EPM,Krebbers G,deRie MA,Luiten RM,vanderVeenJPW,Wolkerstorfer A.Autologouscellsuspensiontransplantationusingacellharvestingdeviceinsegmentalvitiligoandpiebaldism patients:arandomizedcontrolledpilotstudy.JAmAcad Dermatol 2015;73(1):170-172.
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SubstantialOpportunityTreatingLarge,ComplexWounds
SelectedIndicationse.g.,excludesplasticandmaxillofacialsurgeries
Incidence/Prevalence (Patients)Percent
Applicable
PotentialMarketSize(assume1device
perpatient)
USpop.316M
(11.4%diabetes1)
UK,FR, DE,ITpop.271M
(8%diabetes,avg1)
Auspop.23M
(5.1%diabetes1)
Chinapop.1.4B
(9.3%diabetes1)
ChronicUlcers
DFU2 9.0M 5.5M 0.3M 31.6M 20– 40% 9 – 19MVLU3 3.2M 2.7M 0.2M 13.6M 60– 65% 12– 13M
Burnsannual admissions
40K4 42K5 8.6K6 3.4M7 90% 3.1M
Aestheticsannual procedures8 1.7M 585K 117K 157K 90% 2.3M
Vitiligo0.1%to2%ofpop.9 316K 271K 23K 1.4M 30% 0.6M
TOTAL* 14.3M 9.1M 0.7M 50.1M 35%-50% ~27-38M1InternationalDiabetesFederation(IDF)DiabetesAtlas,SixthEdition(2014)2[Lifetimeincidence:25%ofdiabetics]Singhetal."Preventingfootulcersinpatientswithdiabetes." JAMA 293,no.2(2005):217.3[Prevalence:1%ofpop.]Humphreysetal."Managementofmixedarterialandvenouslegulcers.“Br.J.Surg.94,no.9(2007):1104.4 AmericanBurnAssociation2013FactSheet(www.ameriburn.org)5Brusselaers etal."SevereburninjuryinEurope:asystematicreviewoftheincidence,etiology,morbidity,andmortality."Crit Care14(5)(2010):R188.6 Australianhospitalstatistics.AustralianInstituteofHealthandWelfare.(2012)7 PeckMD.Epidemiologyofburninjuriesglobally www.uptodate.com8 ISAPS2013InternationalSurveyonAesthetic/CosmeticProceduresPerformed(dermabrasion,resurfacing,facialrejuvenation)9 Alkhateeb A,FainPR,Thody A,BennettDC,SpritzRA."EpidemiologyofvitiligoandassociatedautoimmunediseasesinCaucasianprobands andtheirfamilies." PigmentCellResearch 16,no.3(2003):208-214.
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o USBurnstrialenrollmentcomplete• ExpeditedAccessPathway(EAP)designation• ContinuedAccessandCompassionateUseIDEPrograms
o RCTsinotherkeyareaseitherpublishedorinprepforpublication
o MechanismofActiondetailsfurtherexploredandinprepforpublication
o Currentlygeneratingadditionaldatainlargermarketopportunities(chronicwounds,aesthetics,etc.)
o Broadeningandbolsteringsalesandmarketingarmamentariumwithrobusthealtheconomicanalysestoshowpositivebudgetimpacttobothpayersandproviders
o Acceleratingbusinessdevelopmentactivitiesforindication-specificcommercialpartnering
Translatingclinicaldevelopmenttosuccessfulcommercialization
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