L E ADI NG RE GE NE RAT I VE ME DI CI NE May 2014 pr e se nte d at:
Cautionary Statement Concerning Forward-Looking Statements This presentation is intended to present a summary of ACT’s (“ACT”, or “Advanced Cell Technology Inc.”, or “the Company”) salient business characteristics. The information herein contains “forward-looking statements” as defined under the federal securities laws. Actual results could vary materially. Factors that could cause actual results to vary materially are described in our filings with the Securities and Exchange Commission. You should pay particular attention to the “risk factors” contained in documents we file from time to time with the Securities and Exchange Commission. The risks identified therein, as well as others not identified by the Company, could cause the Company’s actual results to differ materially from those expressed in any forward-looking statements. Ropes Gray 2
Robust Clinic al and Pr e c linic al Pr ogr ams Autoimmune/ Ophthalmology Drug Delivery Inflammation Engineered Platelets & MK Transplantable Lupus • Factor XIII • Wound Healing RPE Cells • Anti-cancer Multiple Sclerosis Transplantable Engineered RPE Photoreceptor Progenitors Alzheimer’s Disease Transplantable Ganglion Nerve Progenitors Engineered Photoreceptors Inflammatory Bowel Disease DSEK Corneal Transplant Tissue Sepsis Neuroprotective/Neuroregulatory Wound Healing Biologics 3
Robust Clinic al and Pr e c linic al Pr ogr ams Pre-clinical Development Phase 1 Phase 2 Transplantable Dry Age-Related Macular Dry AMD (US) Photoreceptor Progenitors Degeneration (US) Projected 2H-2014/1H-2015 Transplantable Stargardt’s Macular SMD (US & UK) Ganglion Nerve Progenitors Dystrophy (US & UK) Projected 2H-2014 Ophthalmology Myopic Macular DSEK Corneal Transplant Tissue Degeneration (US) Neuroprotective/Neuroregulatory Biologics Engineered RPE Adva nc e d Ce ll T e c hno lo g y’ s Re g e ne ra tive Ophtha lmolog y Prog ra ms TM fo c us o n pre se rving a nd re pa iring ne uro se nso ry re tina a nd c o rne a func tio n 4
Struc ture of the Re tina Re tina 5
L ife Suppor t to Photor e c e ptor s Provide s nutrie nts a nd g ro wth fa c to rs RPE L a ye r ha s • pho to re c e pto rs se e no b lo o d multiple c r itic al r ole s Re c yc le s Vita min A in the • ma inta ins pho to re c e pto r e xc ita b ility he alth and De toxifie s pho to re c e pto r la ye r func tion o f pho to re c e pto rs a nd Ma inta ins Bruc h’ s Me mb ra ne the re tina a s a who le . • na tura l a ntia ng io g e nic b a rrie r • immune privile g e o f re tina Absorbs stra y lig ht / pro te c ts fro m UV 6
L ife Suppor t to Photor e c e ptor s F a ilure o f RPE c e lls re sults in ma ny de g e ne ra tive dise a se s Sta rg a rdt’ s dise a se (SMD) Myo pic Ma c ula r Dystro phy (MMD) Ag e -re la te d ma c ula r de g e ne ra tio n (AMD) 7
pide mic Age - Re late d Mac ular De ge ne r ation will Soon T ake on Aspe c ts of an E T he pr o je c te d numbe r o f pe o ple wo r ldwide with age - r e late d mac ular de ge ne r atio n in 2020 is 196 millio n, inc r e asing to 288 millio n in 2040 - Wo ng e t al. L anc e t January 2014 40% % Pre va le nc e (U.S.) 30% E xpone ntial r ise in L a te AMD pr e vale nc e and inc ide nc e r ate s with age . Inte rme diate AMD 20% Pr e vale nc e r ate s of late AMD quadr uple pe r 10% de c ade 40-49 50-59 60-69 70-79 80+ Ag e Data fro m http:/ / www.ne i.nih.g o v/ e ye data/ and U.S . Ce nsus Bure au Pub lic atio n “65+ in the Unite d State s”, P23-209 8
T he r ape utic T he sis Ce lls c a n b e inje c te d into the sub re tina l Dissoc iate d RPE spa c e a nd will re b uild func tio na l RPE mo no la ye r whe re na tive RPE is c o mpro mise d o r lo st. • RPE la ye r is surg ic a lly a c c e ssib le - - le a st inva sive me a ns to lo c a lly de live r c e lls • Adult RPE tra nslo c a tio n studie s inc lude d disso c ia te d c e ll fo rma ts • hE SC-de rive d RPE suppo rte d b y b o th I n Vitro a nd I n Vivo Studie s a t ACT a nd OHSU • Simple Ha ndling Pro duc t Co ld Cha in • Optimize d fo r la rg e sc a le a nd c e ntra lize d ma nufa c turing is E a sily Sc a le d fo r • Sma ll Do se Size tra nsla te s into ACT ’ s a b ility to ma nufa c ture Glo b a l Sa le s 50,000-100,000 pe r ye a r in e xisting fa c ilitie s. 9
Ce ll T he r apy for RPE , Ac hie vable by a Small Company Sma ll dosa g e size • le ss tha n 200K c e lls Re la tive ly Immune - privile g e d site • minima l immuno suppre ssio n a se of a dministra tion E • no se pa ra te de vic e a ppro va l Unique me a suring a nd obse rva tion e nvironme nt • me a sura b le a na to mic a l a nd func tio na l e ndpo ints 10
F irst T re a tme nts informe d a more a g g re ssive stra te g y to tre a t “be tte r vision” c ohort, c ould le a d to broa de r la be l a nd/ or e a rlie r a pprova l Initial T r ial De sign is Asc e nding Dosage Safe ty Study 12 Subje c ts – F our Cohor ts of 3 subje c ts e ac h Inc lusio n Crite ria Inc lusio n Crite ria Inc lusio n Crite ria Inc lusio n Crite ria BCVA: 20/ 800 o r wo rse BCVA: 20/ 400 o r wo rse BCVA: 20/ 400 o r wo rse BCVA: 20/ 400 o r wo rse Co ho rt 1 Co ho rt 2 Co ho rt 3 Co ho rt 4 50K Ce lls 100K Ce lls 150K Ce lls 200K Ce lls Co ho rt 2a Inc lusio n Crite ria 100K Ce lls BCVA: 20/ 100 o r wo rse Ja n 2013: F DA a ppro ve d a dditio na l 4 sub je c t “b e tte r visio n” c o ho rts in e a c h tria l. F o r Co ho rt 2a – c a n e nro ll sub je c ts with visio n a s g o o d a s 20/ 100 . 11
Curre nt Sta tus of P1 T ria ls e na ble s Pla nning for P2 IND 50% Pa tie nt 100% Pa tie nt Appr ove d E nr ollme nt E nr ollme nt 12/16 patie nts tre ate d U.S. – Dr y AMD 12/16 patie nts tre ate d U.S. – SMD U.K. – SMD 10/12 patie nts tre ate d E nro lling – 12 patie nts to tal U.S. – MMD 34 Patients Treated to Date E a rly Hypo the sis De ve lo pe d fo r PI I , Ve tte d with I nve stig a to rs, Dia lo g ing with MHRA a nd F DA 12
Clinic al T r ials be ing le d by Wor ld L e ade r s in Ophthalmology Ma ss E ye & E a r Infirmary E dinb urg h Ro ya l I nfirma ry Mo o rfie lds Jule s Ste in Wills E ye E ye (UCL A) Institute Ho spita l Ba sc o m Pa lme r E ye Institute Wo rld Re no wne d L e a de rship to he lp us na vig a te the Clinic a l Pa th a nd Ultima te ly Suppo rt Ma rke t L a unc h 13
De live ry of Ce lls Builds on Common Surg e ry Proc e dure : • 25 Ga ug e Pa rs Pla na Vitre c to my • Po ste rio r Vitre o us Se pa ra tio n • Sub re tina l hE SC-de rive d RPE c e lls inje c tio n • Ble b Co nfirma tio n • Da y Surg e ry/ Se da tio n o nly Vitre c to my is the 3 rd Mo st F re q ue ntly Pe rfo rme d Ophtha lmic Surg e ry; 1.1m g lo b a lly, 300k in US, pe r ye a r 14
E la pse d T ime Sinc e T ra nspla nt – a s Ma y 2014 1 ye a r 2 ye a rs 3 ye a rs US Dr y AMD Inte rim Da ta Ana lysis US SMD Inte rim Da ta Ana lysis UK SMD * US AMD & SMD Co ho rt 2a Cohor t sub je c ts ra ng e fro m 1-12 mo nths 2a po st-tra nsplant 15
Ove rvie w of Inte rim Da ta Ma nusc ript de ta iling inte rim da ta unde r re vie w a t impa c tful jo urna l Co -autho re d b y partic ipating surg e o ns and c o mpany o Cle a n sa fe ty pro file o E vide nc e o f e ng ra ftme nt a nd pe rsiste nc e o f tra nspla nte d RPE c e lls o Pe rsisting I mpro ve me nt o f Visua l Ac uity fo r ma ny pa tie nts A numb e r o f patie nts with c linic ally sig nific ant impro ve me nts in BCVA E xe mplar s o f AMD-207 impro ve d 20/ 400 to 20/ 40 Patie nts With • Impro ve me nt ha s pe rsiste d thro ug h fo llo w-up visits (> 1 ye a r) Impr o ve d SMD-001 impro ve d fro m HM to 20/ 500 Visual Ac uity • Impro ve me nt (with furthe r g a ins) ha s pe rsiste d thro ug h fo llo w-up visits (>2 ye a r) Base d on advic e fr om all 5 c linic al tr ial site s and par tic ipating sur ge ons, as we ll as input fr om r e gulator s, we ar e advanc ing to phase 2 studie s › Ac tive dia lo g with MHRA a nd F DA › Antic ipa te P2 c o mme nc e me nt in 2H2014 16
E xpanding RPE Pr ogr am to Addr e ss an Additional L e ading Cause of Blindne ss Myo pia c re a te s a hig he r risk o f pe rma ne nt visio n lo ss due to Myopic Ma c ula r De g e ne ra tion (MMD) • Se ve re ne a r-sig hte dne ss c a use s e lo ng a tio n o f the e ye b a ll -- whic h c a n c a use fissure s in RPE la ye r. As the cause of legal blindness, MMD is ranked: 7 th in the United Sates • 4 th in Hong Kong • • 2 nd in mainland China and Japan Jule s Ste in E ye (UCL A) a nd ACT to I nitia te Pha se I / I I Study 17
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