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

  1. Hamel (2006) Orphanet J Rare Disease 1, 40;
  2. https://nei.nih.gov/health/pigmentosa/pigmentosa_facts;
  3. NORD
  4. https://www.genome.gov/13514348/learning-about-retinitis-pigmentosa/
  5. 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