An!Update!to!the!Cord! Blood!Marketplace,! September!2014!

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GENReports: Market & Tech Analysis An!Update!to!the!Cord! Blood!Marketplace,! September!2014! > Enal Razvi, Ph.D. Managing Director Select Biosciences, Inc. enal@selectbio.us!

The,focus,of,this,GEN,Market,&,Tech,Analysis,Report,is,to,provide, an, update, on, the, cord, blood, marketplace, as, it, evolves, and, matures, We,present,here,the,following,industry,data both,qualitafve,and, quanftafve,in,nature:, Cord!Blood!Transplants!over!the!Past!Decade!and!their!EvoluGon! QualitaGve!Commentary!on!Cord!Blood!Usage! Pricing!Challenges! Impact!of!FDA!Licensure!Requirement!on!Cord!Blood!Banks:!!Economic!Impact!! Topic Introduction and Scope Taken,together,,these,data,frame,some,of,the,current,quanFtaFve, metrics, and, provide, a, snapshot, of, the, cord, blood, space,, as, of, September,2014,

Umbilical Cord Blood Transplants: Quantitative Trends Over the Past Decade! The!Ordinate!Represents!the!Number!of!Transplants! or!shipments!in!the!united!states!! Note!the!Decline!in!2013!in!CordSbased!Transplants!

Umbilical Cord Blood Transplants: Qualitative Trends! There,is,overall,declining,uFlizaFon,of,cord,blood,for,transplants,in, the,united,states,! There,is,increasing,use,in,Japan,,China,,also,France,and,the,UK,[as, the,uk,nhs,seeks,to,build,a,crifcal,mass,of,cord,blood,units,in, inventory,in,the,uk,public,banking,system],! There,are,some,potenFal,reasons,for,the,decline,in,uFlizaFon,in, the,us,! Clinical!Outcomes/Results!can!be!Challenging!and!CompeGGve! Approaches!might!be!more!OpGmal!for!PaGent!Recovery!and! ReconsGtuGon/EngraUment!! Cost!of!the!product!too!high product!here!refers!to!the!single/ double!cord!blood!units!used!as!the!inoculum!! Cost!of!the!procedure!too!high Ranging!between!$250,000S $300,000!

Hematopoietic Transplants by Cell Source Breakout,of,the,Type,of,Cell,Sources,UFlized:, Provides,Snapshots,of,the,EvoluFon,of,this,Space,in,Time,

Allogeneic!Stem!Cell!Sources!by!Recipient! Age:!Pediatric!vs.!Adult!PaGents! Click to add title CIBMTR!Reports!the!Penetrance!of!Cord!Blood!Much!Higher! in!pediatric!pagent!populagon!as!compared!to!adult!pagents!!!!!!!

Clinical!IndicaGons!for!HematopoieGc!Stem!Cell! Transplants!in!the!United!States! Click to add title

Banked Cord Blood Total Nucleated Cell (TNC) Evolution Over the Past Decade What Does the Transplant Physician Want? An average TNC of 1.8 x 10 9 What Does the Typical Transplant Physician Find? 2.8 1.6 2.1 Source:!Dr.!Brian!Freed,!! Professor!of!Medicine!and!Immunology! University!of!Colorado! ExecuGve!Director,!ClinImmune!Labs! HLA-A, B and DRB1 Match

There,has,been,much,discussion,of,the,cost,structure,underlying,the,cord,blood, transplantafon,marketplace,and,the,challenges,this,poses,for,the,sustainability,of, this,industry,, Range,of,costs,per,unit,of,cord,blood,banked:,US$300YUS$2,000,[COGS], Cost Metrics and Associated Challenges Average!cost!of! producgon!of!a!unit!of!cord!blood :!US$900! Average,price,of,cord,blood,units,[ASP], Single!Cord!Priced!at!US$39,400! Double!Cord!Priced!at!US$78,800! Source:,,Mike,Boo,,JD,PresentaFon,at,Cord,Blood,Conference,,San,Francisco,,2014, Indeed!the!costs!of!cord!blood!transplantaGon!are!increasing!due!to!the!FDA! requirement!that!cord!blood!banks!be!licensed!and!this!regulagon!is!driving!cost! increases! The!costs!of! producing!cord!blood!units!has!increased! 10S20%! On!the!other!hand,!the!transplant!centers!(the!end!user!community)!are!driving!down! the!costs!of!cord!blood,!especially!for!the!adults!where!double!cords!are!required this!is!pueng!downward!pressure!on!revenue!generagon!to!the!industry! Source:,,Joanne,Kurtzberg,,MD,,Duke,University, Industry s,business,model,needs,to,adjust,or,be,modified,

Key Takeaways: Quantitative Utilization Trends of Cord Blood and the Associated Challenge in this Space Annual,Turnover,(UFlizaFon),of,cord,blood,units,from,public, banks:,1y3%,of,units,inventoried this,is,not,a,sustainable,business, model, These,are,serious,challenges,in,this,space, Large!numbers!of!stored!cord!blood!units!have!subSopGmal!total!nucleated! cell!(tnc)!count!and!therefore!are!unsuitable!for!transplantagon/cellular! therapy! The!turnover!of!stored!cord!blood!is!exceedingly!low!and!this!is!a!driver!for! the!high!costs!of!the!stored!cord!blood!units,!this!is!coupled!with!the!fact! that!adult!pagents!require!double!cords!doubling!the!cost! Compliance!with!US!FDA!regulaGons/licensure!of!cord!blood!banks! increases!cost!per!unit!of!cord!blood!that!is!shipped! New!therapeuGc!applicaGons!will!be!needed!to!drive!forward!the!uGlizaGon! of!cord!blood!in!cellular!therapy! New!technologies!for!cell!expansion!need!to!be!developed! The!value!proposiGon!of!cord!blood!vs.!other!cellular!types!for! transplantagon!needs!to!be!made!via!clinical!uglity!and!pagent! management!which!ulgmately!translates!into!lower!cost!per!therapy!and! therefore!is! accepted!more!by!the!payors!