Translation of Regenerative Medicine. Prof Trevor M Jones CBE

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1 Translation of Regenerative Medicine Prof Trevor M Jones CBE

2 ReNeuronre UK s only clinical stage stem cell therapy Company Established King s College London (IOP) Based in Guildford, Surrey Focused on novel stem cell therapies for poorly met medical needs Developed pathway to clinical studies with stem cells in UK 2

3 Core Therapeutic Programs Program (cell type) First indication R&D Pre - Clinical Phase I Phase Ib/II Phase III Registration ReN001 (CTX cells) ReN009 (CTX cells) ReN003 (hrpc cells) Stroke Peripheral Arterial Disease Retinitis Pigmentosa 3

4 CTX0E03 Conditionally immortalized CTX - a unique standardised neural stem cell line ReNeuron s c-mycer technology Add 4-OHT Cell proliferation c-mycer No proliferation * Add 4-OHT Remove 4-OHT Banking of candidate lines Cell proliferation Banking * 4OHT = 4-hydroxy tamoxifen, a metabolite of tamoxifen 4

5 Translation challenges manufacturing Purity Identity of cell line Master Cell Banking Potency - biological characteristics Stability of cell line in culture and in frozen state Formulation of cell product Product delivery/compatibility Process development (automation, etc) cgmp standards We ve got a few problems going from lab scale up to full-scale commercial 5

6 cgmp manufacture Manufacture and control of Drug Substance Master Cell Bank (MCB) Working Cell Bank (WCB) Drug Substance (DS) lot Manufacture and control of Drug Product Drug substance expansion QP release on day of administration Isolation Primary Cell Bank EUCTD HTA licence MCB WCB DS DP GMP/contract manufacture 6

7 Clonality/insertional mutagenesis CTX is a clonal cell line with single integration site mapped to SPATA13, locus chromosome13; 13q12.12 SPATA13 has no known oncogenic function The insertion site does not fall in the vicinity of any endogenous retroviruses that could spark oncogenic activation There are no neighbouring genes that raise concerns about oncogenic activation Quantitative PCR of SPATA13 and neighbouring genes shows no mutagenesis-related gene activation or disruption c-mycer 7

8 arbitrary unit Control 1 week 4 week c-mycer TAM transcription (% of control) % positive nuclei c-mycer downregulation in vitro A 100 B Control 1 week 4 weeks 0 Control 1 week 4 weeks C D E c-myc Er Merge F cmyc Tubulin G Densitometry analysis cmyc ER alpha ER Tubulin Stevanato et al., BMC Neuroscience, 2009 Control 1 week 4 weeks 8

9 Rotameter Footfault BAT Asymetry Contralateral/Ipsilateral Contralateral/Ipsilateral Contralateral/Ipsilateral % Ipsi faults % Faults % Faults % Faults Removal Difference Removal Time (sec) Removal Time (sec) Removal Time (sec) CTX0E03 improves stable sensory and motor deficits in rats Combined Striatal Striatal + Cortical Pre Time (Weeks) 4&5 6&7 8&9 10&11 12& Pre Time (Weeks) 4&5 6&7 8&9 10&11 12& Pre Time (Weeks) 4&5 6&7 8&9 10&11 12& Pre 4 12 Time (Weeks) 0 Pre 4 12 Time (Weeks) 0 Pre 4 12 Time (Weeks) Time (Weeks) Time (Weeks) Time (Weeks) 9

10 ReN001 Phase I clinical study STROKE 10 Minimally invasive procedure, performed under local anaesthetic Inject close to site of damage in brain Cells migrate preferentially to lesion damage, survive and demonstrate site-specific differentiation. Measure of function reestablished (quality of life issue)

11 11 The ReNeuron Cell Implantation Cannula

12 Does implantation site influence recovery? Intra-lesion Intra-parenchymal Intra-cerebroventricular 12

13 Translation challenges regulation 13 Source: Department of Health National agencies (currently) involved in stem cell regulation British Standards Institute Department of Health European Medicines Agency Gene Therapy Advisory Committee Health & Safety Executive Home Office Animal Licensing Human Fertilisation & Embryology Authority Human Tissue Authority Medicines and Healthcare products Regulatory Agency Medical Research Council National Research Ethics Service UK Stem Cell Bank

14 ReN001 - PISCES Clinical Trial PISCES clinical trial Pilot Investigation of Stem Cells in Stroke World s first fully regulated clinical trial using neural stem cells in a major brain condition ReNeuron has pioneered UK regulatory pathway first company to gain approval for stem cell clinical trial in UK 14

15 PISCES clinical trial stable disability post stroke Single centre open label study at Glasgow Southern General Hospital (Prof Keith Muir) Phase I single administration dose escalation safety study 12 moderate to severely disabled ischemic stroke patients with stable baseline and 24 month follow-up post treatment Stereotaxic cell implantation Evaluation of efficacy measures for future trials including functional MRI, sensorimotor function 5 patients now treated 15

16 Critical Limb Ischaemia Model Conducted at Bristol Heart Institute (BHI), University of Bristol Diabetic and Non-diabetic animals / Immunocompetent and immunosuppressed Artery Ligated between both points and cauterized Site of cell injection X X X X 16 Right leg Control Side Left leg Ischemic Side

17 Consistent Efficacy in CLI Models: 7 studies undertaken Endpoint Reduced loss of digits Increased Doppler blood flow Increase tissue oxygenation Increased capillaries Increased small arterioles Reproduced in more than one study Effective in diabetic animals Clear dose-response curve Success Yes Yes Yes Yes Yes Yes Yes Yes 17

18 ReN003 for Retinitis Pigmentosa (RP) Blindness-causing disease of the retina - approx 20,000 patients in UK; 100,000 in the US 18

19 ReN003 for Retinitis Pigmentosa Collaboration with Schepens Eye Research Institute/ Mass Eye & Ear Product: Human retinal precursor cells (hrpcs) Project-specific funding secured for first phase of pre-clinical work-up from major US healthcare company Established optimal growth conditions and preliminary evidence of proof of concept GMP process validated First GMP DS lot made Key in vivo studies underway 19

20 GTP/EUCTD-compliant hrpc isolations From Californian tissue bank (ABR), 3 fetal eye donations acquired and imported into our clean room facility, all compliant with GTP/EUCTD Seed banks to P2/P3 established under local quality assurance and in compliance with GB therapeutic tissues and cells regulations ( vials each) P1 P3 20

21 ReNeuron Group plc 10 Nugent Road Surrey Research Park Guildford Surrey GU2 7AF UK (t) +44 (0) (f) +44 (0) (e) 21 Ticker: RENE.L

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