INVESTOR PRESENTATION
6 January 2021
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© ReNeuron Group plc 2021 All rights reserved | 2 |
RENEURON TEAM AND KEY CLINICAL ADVISERS
Olav Hellebø
Chief Executive Officer
Olav has held leadership roles internationally at big pharma companies, including Novartis and Schering Plough, and biotechs including Clavis Pharma ASA. Product launches include the TNF-blocker Cimzia whilst at specialty biopharma business UCB
Michael Hunt ACA
Chief Financial Officer
Michael qualified at Ernst & Young after which he joined Bunzl plc before focusing on healthcare at Biocompatibles International plc and then ReNeuron. He sits on the board of the US-based Alliance for Regenerative Medicine and other industry bodies
Dr. Rick Beckman
Chief Medical Officer
After a career as an ophthalmologist in academics, then private practice, Rick moved into leadership roles at large companies including Allergan, Alcon and BD. He then moved on to serve as CMO at ophthalmology- focused biotechs including Neurotech, Ophthotech, and Clearside.
Clinical Advisors
Dr Jason Comander
Associate Director of the Inherited Retinal Disorders Service at Massachusetts Eye and Ear Infirmary and Assistant Professor of Ophthalmology at Harvard Medical School
Prof. Robert MacLaren
Professor of Ophthalmology, University of Oxford, directs research into new treatments for blindness. Co-founded Nighstar Therapeutics, which was acquired by Biogen.
Dr Timothy Stout
Chair of the Ophthalmology Department and Director of the Cullen Eye Institute at Baylor College of Medicine.
Dr Jordi Monés
Macula and Vitreorretinal Specialist and Researcher. Director of the Institut de la Màcula and the Director, Principal Investigator and one of the founder governors of the Barcelona Macula Foundation: Research for Vision.
Dr. Tim Corn | Dr. Mike Owen | Sir Chris Evans Non- | Mark Evans |
Chairman | Non-executive Director | executive Director | Non-executive Director |
© ReNeuron Group plc 2021 All rights reserved
Dr Karl Csaky
T. Boone Pickens Director, Molecular Ophthalmology Laboratory and Clinical Center of Innovation for Macular Degeneration.
RENEURON: HIGHLIGHTS
Leading clinical stage cell therapy company with presence in the UK and US
Proprietary allogeneic retinal and neural stem cell therapy platforms
Lead programme an Orphan Drug treatment with Fast Track Designation targeting retinitis pigmentosa (RP) - positive early Phase 2a clinical data with study ongoing
Planning to commence pivotal RP clinical trial in H2 2022, with top-line data targeted for 2024, ahead of market approval application
Proprietary exosome programme - collaborations ongoing with pharma & biotech, with further collaborations anticipated
Well-funded, with multiple value inflection points expected in the next 12 months, including extended RP Phase 2a clinical data read-outs and exosome pre-clinicalproof-of-concept data
© ReNeuron Group plc 2021 All rights reserved | 4 |
PROPRIETARY PLATFORM TECHNOLOGIES
hRPC
Exosome Platform
iPSC Platform | CTX Cells | |
Human Retinal Progenitor Stem Cells with sub-
retinal delivery enabling engraftment
Cryopreserved formulation allows global ship-
and-store
Positive early Phase 2a data in ongoing retinitis
pigmentosa study
Partnered with Fosun Pharma for China
Peers
Example
High-yielding neural
stem cell derived exosomes
Proven ability to load exosomes with siRNA,
miRNA and proteins
Favourable distribution of exosomes across the
Blood Brain Barrier
Potential as drug load/delivery vehicle and as a therapeutic. 3 pharma collaborations ongoing
© ReNeuron Group plc 2021 All rights reserved
CTX-based induced
pluripotent
stem cell platform
Neural stem cells
engineered into other
forms of stem cells while
preserving the immortalisation
Potential to create
allogeneic CAR-T cell
therapies and pancreatic
islet cells
Immortalised neural
progenitor
stem cell line
Positive clinical data in
stroke disability. Potential in Huntington's disease, TBI and other indications
Out-licensing strategy
Partnered with Fosun
Pharma for China
5
INTERNAL RESEARCH AND DEVELOPMENT PIPELINE
Programme
Human Retinal Progenitor Cells
Exosome platform
iPSC platform
CTX cell line
Indication | Pre-clinical | Phase 1 | Phase 2 | Next Milestones | |
• Further data read-outs from | |||||
Retinitis | expanded Phase 2a study over next | ||||
twelve months | |||||
Pigmentosa | |||||
• Pivotal trial to commence in H2 | |||||
2022, subject to Phase 2a data | |||||
Neurodegeneration, | • Proof of concept data from current | ||||
research collaborations expected in | |||||
Oncology, Vaccines (e.g. | H1 2021 | ||||
COVID-19) | • | Additional collaborations expected | |||
over the next 12 months | |||||
• | Validation of technology | ||||
Oncology, Diabetes | and publication of pre-clinical | ||||
proof-of-concept data |
• Currently partnered in China with
Stroke Disability
• Open for partnerships outside
China
© ReNeuron Group plc 2021 All rights reserved | 6 |
INTERIM RESULTS - HIGHLIGHTS
Six months | Six months | ||
ended 30 | ended 30 | Year ended | |
September | September | ||
2020 | 2019 | 31 March | |
(£'m) | 2020 | ||
(Unaudited) | (Unaudited) | (Audited) | |
Revenue and other operating income | 0.1 | 6.1 | 6.1 |
Research and development costs | (5.9) | (9.2) | (16.3) |
General and administrative costs | (1.9) | (2.6) | (4.2) |
Operating loss | (7.7) | (5.7) | (14.4) |
Net finance (expense)/income | (0.3) | 0.6 | 0.6 |
Taxation | 0.9 | 1.2 | 2.4 |
Loss for the period | (7.1) | (3.9) | (11.4) |
Net decrease in cash and deposits | (2.8) | (5.1) | (13.8) |
Cash and deposits at start of period | 12.6 | 26.4 | 26.4 |
Cash and deposits at period end | 9.8 | 21.3 | 12.6 |
© ReNeuron Group plc 2021 All rights reserved | 7 |
MATERIAL VALUE INFLECTION POINTS TARGETED OVERNEXT12MONTHS AND BEYOND(1)
RP Phase 2a
extended study -
first 'non-US'
subject treated
RP Phase 2a
extended study - 6
month data
Potential out- licensing deals on full Phase 2a data
RP Phase 2a | |
extended study - | |
first subject treated | |
hRPC | in US |
RP Phase 2a initial study - 12 month data on all subjects
RP Phase 2a
extended study -
last subject
treated
RP Phase 2a | FDA meeting |
extended study - | |
re pivotal | |
3 month data | |
study design | |
RP pivotal study | RP pivotal study |
commences | top-line data |
Q3 2020Q4 2020 Q1 2021Q2 2021 Q3 2021 Q4 2021 H1 2022 H2 2022 2023 2024
Potential out- | ||||
licensing deals on | ||||
Further pharma | POC data for delivery | POC data | ||
collaborations | of functional | |||
Exosome and iPSC | proteins by CTX- | |||
POC data for | derived exosomes | |||
Platforms | ||||
delivery of siRNA | ||||
by CTX-derived | ||||
exosomes | (1) Indicative clinical timelines subject to successful recruitment of patients | |||
© ReNeuron Group plc 2021 All rights reserved | 8 |
Lead
Programme
hRPC in retinitis
pigmentosa
© ReNeuron Group plc 2021 All rights reserved | 9 |
RETINITIS PIGMENTOSA: AN UNMET NEED
RP is an inherited, degenerative eye disease1,2,3
• Incidence of 1:4,000 in U.S. and worldwide
>100 genes identified containing mutations leading to RP4
Normal View
Treatment available only for patients with a single gene defect (RPE65)
Patients with all other types of RP (c98% of patients5) have declining vision eventually leading to severe visual disability in most
Therapeutic benefit of hRPC approach not dependent on genetic cause | View with Retinitis Pigmentosa | |
- Hamel (2006) Orphanet J Rare Disease 1, 40;
- https://nei.nih.gov/health/pigmentosa/pigmentosa_facts;
- NORD
- https://www.genome.gov/13514348/learning-about-retinitis-pigmentosa/
- www.nice.org.uk/guidance/hst11/chapter/2-The-condition
© ReNeuron Group plc 2021 All rights reserved | 10 |
HUMAN RETINAL PROGENITOR CELLS (hRPC)
hRPC: allogeneic cell-based
therapeutic approach to retinal disease
hRPCs differentiate into functional
photoreceptors and integrate into retinal layers in pre-clinical models; integration may also enable durable trophic support
Broad potential across a range of eye diseases, initially targeting inherited retinal degenerative diseases
Orphan Drug Designation in EU and US in RP
and FDA Fast Track Designation
Proprietary manufacturing process
and controls allow for stable, high quality
and high quantity GMP production
Collaborations with Schepens Eye Research Institute (Harvard) and University College London
Proprietary technology enabled development
of GMP manufacturing process
Cryopreserved formulation provides nine- month shelf life and enables local treatment worldwide
© ReNeuron Group plc 2021 All rights reserved
High commercial potential
RP is a large orphan market.
Attractive pricing precedent set by Luxturna
Mechanism of action independent of genetic
cause
Commercially viable formulation
11
CLINICAL DEVELOPMENT
Phases 1 and 2a
Phase 1
Single ascending dose in subjects with established RP
- Subjects with very poor visual potential
- Four cohorts, three subjects each (n=12)
- Formulation changed from fresh to cryopreserved cells
Established safety in cryopreserved formulation
Phase 2a
10 subjects with established RP
- Patients with better visual potential
- 1m cell dose
Primary endpoint
- Safety
Secondary measures
-
Visual acuity, visual field, retinal sensitivity and retinal structure
Established efficacy signal, continued safety
Existing Clinical Sites | Massachusetts Eye & Ear Infirmary, Boston | Retinal Research Institute, Phoenix | |
© ReNeuron Group plc 2021 All rights reserved | 12 |
SURGICAL TECHNIQUE
Sub-retinal Injection
Well established technique used commercially with Luxturna®
Allows correct anatomic placement of cells for integration into the retina
- Can serve as a depot for prolonged production of trophic factors
- Can allow for differentiation into photoreceptors with proper connections to other cells needed for vision
© ReNeuron Group plc 2021 All rights reserved | 13 |
PHASE 2a EFFICACY RESULTS
Mean changes in ETDRS letters read (treated eye vs untreated eye)
Day 30 | Day 60 | Day 90 | Day 180 | Day 270 | Day 365 | |||
(n=9) | (n=9) | (n=9) | (n=9) | (n=8) | (n=7) | |||
Treated Eye | +7.9 | +8.0 | +10.8 | +9.6 | +7.1 | +9.9 | ||
Untreated Eye | +0.2 | +1.2 | +4.4 | +3.4 | +1.2 | -2.4 | ||
Difference | +7.7 | +6.8 | +6.4 | +6.2 | +5.9 | +12.3 | ||
+12.0 | ||||||||
+10.0 | ||||||||
ETDRS letters read | (mean change from baseline) | +8.0 | ||||||
+6.0 | ||||||||
+4.0 | ||||||||
+2.0 | ||||||||
0 | Day 30 | Day 60 | Day 90 | Day 180 | Day 270 | Day 365 | ||
-2.0 | Days post-treatment | |||||||
-4.0 | ||||||||
Treated Eye | Untreated Eye |
Additional Notes:
*excluding 1 patient (6003) with surgery-related vision loss
**Two patients have so far been assessed at 18 months. One patient has gained 17 letters from baseline in the study eye and one letter in the non-study eye. The second patient has gained six letters from baseline in the study eye and 22 letters in the non-study eye.
© ReNeuron Group plc 2021 All rights reserved | 14 |
INDIVIDUAL PATIENT IMPROVEMENTS AT 12 MONTHS
ETDRS change from baseline 12 months post treatment (n=7)
+25.0
Mean = +9.9 letters
+20.0
+20.0
+15.0
+15.0
+10.5
+10.0
+8.0+8.0
+6.0
+5.0
+2.0
0
1 | 2 | 3 | 4 | 5 | 6 | 7 |
© ReNeuron Group plc 2021 All rights reserved | 15 |
CLINICAL DEVELOPMENT: PHASE 2A EXTENSION
Modifications to better hone efficacy signal
Phase 2a Extension
9 additional subjects with established RP
- Dose escalation: from 1m to 2m cells
- Require ability to perform micro-perimetry - should allow retinal sensitivity to be an indicator of efficacy
- Additional baseline VA's to ensure patient reliability
- Modified surgical technique to target bleb placement: injection sites chosen to avoid areas of viable retina
Primary endpoint
- Safety
Secondary measures
- Visual acuity, micro-perimetry, visual field, retinal sensitivity and retinal structure
Additional Sites Added
Oxford Eye Hospital, Oxford, UK | Two further sites planned, | |
(Prof Robert MacLaren) | one in Europe and one in the US | |
© ReNeuron Group plc 2021 All rights reserved | 16 |
RETINITIS PIGMENTOSA: THERAPY LANDSCAPE
Company
ReNeuron
(AIM, market cap: £26m*)
jCyte Inc
(US, private)
Spark Therapeutics (acquired by Roche in 2019 for $4.3bn)
Nightstar Therapeutics (acquired by Biogen in 2019 for $800 million)
MeiraGTx
(Nasdaq, market cap $585m*)
AGTC
(Nasdaq, market cap $118m*)
Technology
Cell therapy
Cell therapy
Gene therapy
Gene therapy
Gene therapy
Gene therapy
Stage
Phase 1/2a
Phase 2b
Approved and marketed, Luxturna for RPE65
Phase 2/3
Phase 1/2
Phase 1/2
Comment
Cryopreserved formulation
Not cryopreserved; used to date in California and Massachusetts
Addresses only about 2%** of RP patients
UK company co-founded by Prof Robert MacLaren
-
-
- Market capitalisations as at 7 December 2020
- www.nice.org.uk/guidance/hst11/chapter/2-The-condition
© ReNeuron Group plc 2021 All rights reserved | 17 |
RETINAL PLATFORM NEXT STEPS
Material newsflow and value inflection points over the next 15 months and beyond
Collect long term data in normal dose subjects
- Most patient visits restarted post-Covid restrictions
- All 22 patients will be followed to at least 24 months post treatment
Recruit high dose expansion study
- First patient treated September 2020
- Enhancements in patient selection, dose, surgical technique and efficacy assessments
Further efficacy data to be presented at retinal conferences over the coming twelve months
- AAO/ASRS/ARVO are the key conferences in ophthalmology
- 3 months data on all 9 Phase 2a extension patients to be presented at ASRS in July
A single further clinical trial is planned before filing for marketing authorisation
- Randomised, not placebo controlled
- Three patient groups (high dose, low dose and observational cohort)
© ReNeuron Group plc 2021 All rights reserved
Assess other indications alongside RP (e.g. Cone Rod Dystrophy)
Partnering strategy to be based on full Phase 2a data
18
Exosome platform
© ReNeuron Group plc 2021 All rights reserved | 19 |
EXOSOMES: BIOLOGICAL NANOPARTICLES
Nano-scale vesicles released by most cell types as a means of intercellular communication
Naturally occurring liposomal delivery system
Contain and transport bio-active lipids, proteins and nucleic acids
Potential as a drug delivery vehicle and as a therapeutic
- Current focus is on drug delivery
- Collaborations in place and further ones under negotiation
Increasing industry interest in and commercial value of exosome deals
© ReNeuron Group plc 2021 All rights reserved
Lipid bilayer
Surface proteins (tetraspanins CD63, CD81)
Internal proteins
(Hsp70, Tsg101)
Specific nucleic acids (miRNAs)
20
ADVANTAGES OF RENEURON'S EXOSOME TECHNOLOGY
Favourable distribution | Proven ability | Stable, consistent, | Fully qualified xeno-free, |
across the blood | to load miRNA | high-yield, | optimised, scalable |
brain barrier | and proteins | clinical-grade product | GMP process |
Established | Modifiable to carry siRNA/mRNA, | Engineered to target |
analytics | CRISPR/Cas9 proteins, | particular tissues |
small-molecule inhibitors | ||
© ReNeuron Group plc 2021 All rights reserved | 21 |
PROOF OF CONCEPT DATA EXPECTED IN H1 2021
hNSC-ExosomePlatform (for delivery across the blood brain barrier)
Significant research collaborations ongoing
- Three current undisclosed industry leading partners
- Focused on delivery of siRNA and mRNA
- Goal to deliver in-vivo proof of concept data
- Trials financed by partners
Further research collaborations under review, focused on delivery of other novel therapeutics including antibodies
ReNeuron owns equal co-development rights to new therapeutic modalities
© ReNeuron Group plc 2021 All rights reserved | *Source - public announcements | 22 |
Summary
© ReNeuron Group plc 2021 All rights reserved | 23 |
VALUE REALISATION
Recent deals in cell therapy for retinitis pigmentosa and exosomes research
Santen deal based on Phase 2 data in RP* | Exosome deals based on pre-clinical POC data* | |
- jCyte Inc signed an ex-US licensing deal for its jCell product in May 2020 with Santen Pharmaceutical
- jCell is a Phase 2b retinal progenitor cell suspension for RP
Deal terms
- $50m upfront
- $12m convertible note
- $190m of milestones
- Double-digitroyalties
Total: $72.5m | Upfront: $20m | Total: $882m | Upfront: $56m | |
neuro-muscular | Total: $1,230m | rare diseases | Total $1,076m | |
targets | neurological | cancer | ||
Codiak listed on | targets | |||
Nasdaq in October | ||||
2020, raising $83m | ||||
© ReNeuron Group plc 2021 All rights reserved | *Source - public announcements | 24 |
SUMMARY
Major value creation opportunities in the coming 12 months for hRPC
New data from expanded Phase 2a study of hRPC in retinitis pigmentosa to be presented at major conferences next year
Competitor data support ReNeuron's approach in RP
Potential of cell therapy in ophthalmologyunderlinedby recent JCyte/Santen Pharmaceutical licensingdeal
$252m in upfronts and milestones for ex-US rightsplusdouble digit royalties
Exosome programme being advanced through partners while retaining rights
High level of industry interest in exosomes reflected in recent licensing deals
Deals totaling more than $2bn in upfronts and milestones based on proof of concept data
© ReNeuron Group plc 2021 All rights reserved | 25 |
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Ticker: RENE.L
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ReNeuron Group plc published this content on 06 January 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 06 January 2021 16:37:06 UTC