ReNeuron : 2020 Preliminary Results Presentation

2020 PRELIMINARY RESULTS PRESENTATION

20 July 2020

Olav Hellebø - Chief Executive Officer

Michael Hunt - Chief Financial Officer

Dr Rick Beckman - Chief Medical Officer

DISCLAIMER

This Presentation is being supplied to you solely for your information and may not be reproduced, further distributed to any other person or published, in wholeorin part,forany purpose. Subject to certain exceptions, this Presentation is not for distribution in the United States, Australia, Canada or Japan or any other jurisdiction whereits distributionmayconstitute a violation of the laws of such jurisdiction.

The information contained in this document ("Presentation") has been prepared by ReNeuron Group plc (the "Company") and neither this Presentation, nor theinformation contained in it should be considered a recommendation by the Company or any of its shareholders, directors, officers, agents, employees or advisers in relation to any purchase of the Company's securities, including any purchase of or subscription for any shares (or securities convertible into shares) in the capital of the Company. This Presentation hasnot been fully verified and is subject to material updating, revision and further amendment. Any person who receives this Presentation should not rely or act upon it. This Presentation should not be re-distributed,re-published, reproduced or disclosed by recipients, in whole or in part.

While the information contained herein has been prepared in good faith, neither the Company nor any of its shareholders, directors, officers, agents, employees or advisers give, have given or have authority to give, any representations or warranties (express or implied) as to, or in relation to, the accuracy, reliability or completeness of the information in this Presentation, or any revision thereof, or of any other written or oral information made or to be made available to any interested party or its advisers (all such information being referred to as "Information") and liability therefor is expressly disclaimed. Accordingly, neither the Company nor any of its shareholders, directors, officers, agents, employees or advisers take any responsibility for, or will accept any liability whether direct or indirect, express or implied, contractual, tortious, statutory or otherwise, in respect of, the accuracy or completeness of the Information or for any of the opinions contained herein or for any errors, omissions or misstatements or for any loss, howsoever arising, from the use of this Presentation.

This Presentation may contain forward-looking statements that involve substantial risks and uncertainties, and actual results and developments may differ materially from those expressed or implied by these statements and past performance is no guarantee of future performance. These forward-looking statements are statements regarding the Company's intentions, beliefs or current expectations concerning, among other things, the Company's results of operations, financial condition, prospects, revenue generation, growth, strategies and the industry in which the Company operates. By their nature, forward-looking statements involve risks and uncertainties because they relate to events and depend on circumstances that may or may not occur in the future. These forward-looking statements speak only as of the date of this Presentation and the Company does not undertake any obligation to publicly release any revisions to these forward-looking statements to reflect events or circumstances after the date of this Presentation.

This Presentation has not been approved by an authorised person in accordance with Section 21 of the Financial Services and Markets Act 2000.

In no circumstances will the Company be responsible for any costs, losses or expenses incurred in connection with any appraisal or investigation of the Company. In furnishing this Presentation, the Company does not undertake or agree to any obligation to provide the recipient with access to any additional information or to update this Presentation or to correct any inaccuracies in, or omissions from, this Presentation which may become apparent. This Presentation does not constitute an offer or invitation to subscribe for or purchase any securities and neither this Presentation nor anything contained herein shall form the basis of any contract or commitment whatsoever. In particular, this Presentation is for information purposes and does not constitute an offer or invitation to subscribe for or purchase any securities in the United States. The securities of the Company have not been and will not be registered under the US Securities Act of 1933, as amended (the "US Securities Act") or the securities laws of any state or other jurisdiction of the United States and may not be offered, sold, resold, pledged, delivered, distributed or transferred, directly or indirectly, into or in the United States except pursuant to an exemption from, or in a transaction not subject to, the registration requirements of the US Securities Act and in accordance with any applicable state securities laws. There will be no public offering of the securities of the Company in the United States.

By participating in and/or accepting delivery of this Presentation you agree to be bound by the foregoing restrictions and the other terms of this disclaimer.

© ReNeuron Group plc 2020 All rights reserved

2

A LEADER IN CELL-BASED THERAPEUTICS

Leading clinical stage cell therapy company with presence in the UK andUS

Proprietary allogeneic retinal and neural stem cell technology

platforms, including stem cell derived exosomes and pluripotent stem cells

Lead programme in Phase 2 in retinitis pigmentosa

- licensing deal expected to follow readout of positive Phase 2a data next year

Increasing focus on exosomes and iPSCs (induced pluripotent stem cells) - collaborations already underway and further collaborations anticipated

© ReNeuron Group plc 2020 All rights reserved

3

PROPRIETARY PLATFORM TECHNOLOGIES

hRPC

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

Exosome Platform

High-yielding human 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

iPSC Platform

CTX-based induced

pluripotent

stem cell platform

Technology engineers CTX neural stem cells into other forms of stem cell

Potential for new targeted cell therapeutics and for exosomes based on non- neural stem cells

CTX Cells

Immortalised neural

progenitor

stem cell line

Positive Phase 2a results in stroke disability and potential in Huntington's disease and other indications

Partnered with Fosun

Pharma for China for future

clinical development in

stroke disability

© ReNeuron Group plc 2020 All rights reserved

4

DEVELOPMENT PIPELINE

Programme

Indication	Pre-clinical	Phase 1	Phase 2	Next Milestones

hRPC

CTX cells

Exosome platform

iPSC platform

Retinitis

Pigmentosa

Stroke Disability

Neurodegeneration,

Oncology, Vaccines

(e.g. COVID-19)

Oncology, Diabetes

Further data read-outs from expanded UK/US Phase 2a study in 2020 and 2021

Future clinical development with Fosun and other potential partnerships

Proof of concept data from pharma/biotech research collaborations

Validation of technology

and publication of pre-clinicalproof-of-concept data in 2020

© ReNeuron Group plc 2020 All rights reserved

5

Operational

• financial highlights

© ReNeuron Group plc 2020 All rights reserved

6

OPERATIONAL HIGHLIGHTS

• Positive and sustained top-line efficacy data at all time-points from Phase 2a patients in ongoingUS Phase1/2a clinical

	trial in retinitis pigmentosa
hRPC	• Regulatory approval received in US and UK to expand ongoing Phase 2a study to allow for subsequent potential single
pre-approval clinical study and shorter route to market

• Further readouts from expanded study expected over next 12 months, leading to intention to file application in secondhalf of 2021 to commence pivotal clinical study

Exosome

• iPSC platforms

CTX cells

• Grant-fundedcollaboration to enable delivery of therapeutic nucleic acids using Company's exosomes
• New data presented supporting use of Company's iPSCs (induced pluripotent stem cells) to develop new immortalised cell lines as potential therapeutic agents for subsequent licensing to third parties
• Collaboration agreements signed with major pharmaceutical/biotechnology companies post year-end to explore potential of Company's exosomes to deliver therapeutic agents to the brain
• Proprietary exosome developed for the potential delivery of COVID-19 vaccines
• Positive data from PISCES II Phase 2a clinical trial of CTX in stroke disability published in peer reviewed journal
• Post year-end decision to continue stroke disability programme through regional partnerships
• • Fosun Pharma to develop and commercialise hRPC and CTX programmes in China under exclusive out-licence agreement signed in April 2019
  • PISCES III Phase 2b stroke study to remain suspended in US, following earlier COVID-19 restrictions
• CTX cell therapy candidate available for licensing in all territories in other indications
• • Publication of new positive non-clinical data with CTX cells in Huntington's disease
• Intention to reconfigure non-executive Board to reflect Company's new emphasis on retinal diseases and commercial partnerships (& approval in principle to appoint non-executive director nominated by substantial shareholder Obotritia

Capital KGaA)

© ReNeuron Group plc 2020 All rights reserved

7

PRELIMINARY RESULTS for the year ended 31 March2020

Highlights

(£'m)	Year ended 31 March 2020 (Audited)	Year ended 31 March 2019 (Audited)
Revenues and other income	6.1	2.7
Research and development costs	(16.3)	(16.2)
General and administrative costs	(4.2)	(4.8)
	(14.4)	(18.3)
Net finance income	0.6	1.1
Taxation	2.4	2.9
Loss for the year	(11.4)	(14.3)
Net decrease in cash and deposits	(13.8)	(11.0)
Cash and deposits at start of period	26.4	37.4
Cash and deposits at period end	12.6	26.4
	© ReNeuron Group plc 2020 All rights reserved	8

Lead

Programme

hRPC in retinitis

pigmentosa

© ReNeuron Group plc 2020 All rights reserved

9

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 2020 All rights reserved

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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 2020 All rights reserved

11

RETINITIS PIGMENTOSA: THERAPY LANDSCAPE

Company

ReNeuron

(AIM, market cap: £45.8m*)

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 $467.8m*)

AGTC

(Nasdaq, market cap $142.7m*)

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

Fresh formulation

Addresses only about 2%** of RP patients

UK company co-founded by Prof Robert MacLaren

-

-

• Market capitalisations as at 30 June 2020
• www.nice.org.uk/guidance/hst11/chapter/2-The-condition

© ReNeuron Group plc 2020 All rights reserved

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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 2020 All rights reserved

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LATEST PHASE 2a EFFICACY RESULTS

Mean changes in ETDRS letters read (treated eye vs untreated eye)

		1 Month	2 Months	3 Months	6 Months	9 Months	12 Months	18Months
		(n=9)	(n=9)	(n=9)	(n=9)	(n=8)	(n=5)	(n=1)
Mean Change*	Treated Eye	+7.9	+8.0	+10.8	+9.6	+7.1	+11.9	+17
(per time point)
	Untreated Eye	+0.2	+1.2	+4.4	+3.4	+1.2	+4.3	+1
	Difference	+7.7	+6.8	+6.4	+6.2	+5.9	+7.6	+16

ETDRS letters read (mean change from baseline)

Days post-treatment

*excluding 1 patient with surgery-related vision loss

© ReNeuron Group plc 2020 All rights reserved

14

CLINICAL DEVELOPMENT

Phase 2a Extension

Phase 2a Extension

9 additional subjects with established RP

• Patients selected with capability to perform micro-perimetry - should allow retinal sensitivity to be an indicator of efficacy
• Dose escalation: from 1m to 2m cell dose
• Modified surgical technique to target bleb placement

Primary endpoint

• Safety

Secondary measures

• Visual acuity, micro-perimetry, visual field, retinal sensitivity and retinal structure

Additional Sites	Oxford Eye Hospital, Oxford, UK		Two further sites planned,
(Prof Robert MacLaren)		one in Europe and one in the US

© ReNeuron Group plc 2020 All rights reserved

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hRPC PLATFORM NEXT STEPS

Expanded Phase 2a study to generate further and longer-term follow up efficacy data

• UK and US regulatory approvals obtained in April and May 2020 respectively
• Modifications in patient selection, dose, surgical technique and efficacy assessments to amplify current efficacy signal

Further top-line efficacy data expected to be presented later in 2020 (subject to continued easing of COVID-19 restrictions) and during 2021

• Expecting to file for approval for potential single pivotal study during H2 2021

© ReNeuron Group plc 2020 All rights reserved

Partnering strategy

to be based on Phase 1/2a data

Assess other

indications alongside

RP (e.g. Cone Rod

Dystrophy)

16

Exosome

and iPS cell

platforms

© ReNeuron Group plc 2020 All rights reserved

17

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
• Programmes including COVID-19 viral vaccines
• Collaborations in place and further ones under negotiation

Increasing industry interest in and commercial value of exosome deals

© ReNeuron Group plc 2020 All rights reserved

Lipid bilayer

Surface proteins (tetraspanins CD63, CD81)

Internal proteins

(Hsp70, Tsg101)

Specific nucleic acids (miRNAs)

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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 2020 All rights reserved	19

BREADTH OF OPPORTUNITIES

Product

ExoBDNF

Indication

Neurodegeneration (hearing loss, Parkinson's, Alzheimer's)

Technology	Discovery	POC/Development	Milestones
Expresses the
neuroprotective growth			Pivotal pre-clinical
factor BDNF (brain derived			2020
neurotrophic factor)

Exosome Platform

iPSC Platform

ExoKRAS

ExoSPIKE

ExoXX

CTX-HSC

CTX-PP

Oncology

COVID-19 prevention

Diseases of the brain

Haematological cancers

Diabetes

Engineered to deliver siRNA (small inhibitory RNA) against KRAS G12D mutation

Expresses SARS-Cov-2 spike protein for potential delivery of COVID-19 vaccines

Exosomes loaded with therapeutic of partners choice

Immortalised hematopoietic stem cells - for scaled production of allogeneic T or NK cells

For generation of phenotypically stable human pancreatic progenitor cells

Pivotal pre-clinical 2020

Grant application - awaiting decision

Collaborations ongoing

Validation and further characterisation 2020

Validation and further characterisation 2020

© ReNeuron Group plc 2020 All rights reserved

20

PROGRESS TOWARDS PROOF OF CONCEPT DATA

hNSC-ExosomePlatform (for delivery across the blood brain barrier)

Research collaborations ongoing

• Undisclosed industry leading partners
• Focused on delivery of oligonucleotides
• Goal to deliver proof of concept data

Further research collaborations in negotiation, focused on delivery of other novel therapeutics including antibodies

Strategy to progress to licensing deals

Recent exosome deals, based on POC data, underline potential*:

Codiak/	Evox/ Lilly	Evox/ Takeda	Codiak/ Jazz
Sarepta	Upfront: $20m	Total: $882m	Upfront: $56m
Total: $72.5m	Total: $1,230m	rare diseases	Total $1,076m
neuro-muscular	neurological targets		cancer
targets

© ReNeuron Group plc 2020 All rights reserved

*Source - public announcements

21

INDUCED PLURIPOTENT STEM CELLS

ReNeuron's iPSC platform technology is for reprogramming proprietary neural stem cells into a pluripotent state able to differentiate into any other form of stem cell

iPSCs retain the immortalisation characteristic of the stem cells from which they are derived, resulting in highly stable cell lines

Technology has the potential to lead to off the shelf therapeutics, such as T cells for CAR-T therapy

Also has the potential to produce exosomes with tissue-specific targeting ability

Two programmes - currently underway in the validation and characterisation stage

© ReNeuron Group plc 2020 All rights reserved

Conditionally immortalised derivatives from CTX-iPSCs

22

CTX cell line

© ReNeuron Group plc 2020 All rights reserved

23

CTX CELL THERAPY

CTX: Allogeneic, Cryopreserved, Human Neural Stem Cell Product

Promotes anatomical plasticity in the brain

Excellent safety profile - no immunogenicity

issues post-administration

Manufactured under cGMP with

a 12 month shelf life

Positive Phase 2a data published

in peer-reviewed journal*

Commercially

Attractive

Cryo-shipped product can be easily ordered,

delivered and stored at the hospital

Commercial scale manufacturing

at attractive COGs

Licensing

Strategy

Stroke disability: licensed to Fosun Pharma in

China with other territories available

Out-licensing strategy for other potential indications such as Huntington's disease

*Journal of Neurology, Neurosurgery, and Psychiatry http://jnnp.bmj.com/cgi/content/full/jnnp-2019-322515f

© ReNeuron Group plc 2020 All rights reserved

24

Summary

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25

SUMMARY

A global leader in cell-based therapeutics - presence in UK and US

Phase 2a study in RP expanded with protocol changes to enable fastest route to market - further significant data readouts over next 18 months

Allogeneic stem cell technology platforms - patented, scalable & cost effective

High level of industry interest in exosomes - near/medium term opportunities for value-generating partnering/collaboration deals

© ReNeuron Group plc 2020 All rights reserved

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