Developing stem cell technologies to improve patients' lives

ReNeuron Group plc

Annual Report for the year
ended 31 March 2020

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Our vision is to improve patients’ lives through our proprietary stem cell technologies.

As a leader in cell-based therapeutics, we develop allogeneic stem cell technology platforms, stem cell derived exosomes and induced pluripotent stem cells.

Group at a glance

hRPCs for
retinal
diseases

Exosome nanomedicine
platform

iPSCs:
expanding our therapeutic
platform

CTX stem
cell therapy
for stroke
disability

Many patients suffer from medical conditions where their needs are unmet, impacting on the quality of their lives.

Our stem cell technologies have the potential to improve the lives of patients with unmet medical needs.

Our current pipeline

Programme

Indication

Pre-clinical

Phase 1

Phase 2

Next Milestones

hRPCs

Retinitis
Pigmentosa

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

CTX cells

Stroke
Disability

Future clinical development with Fosun and other potential partnerships

Exosomes
platform

Neurodegeneration,
Oncology, Vaccines
(e.g. COVID-19)

Proof of concept data from pharma/biotech research collaborations

iPSC platform

Oncology,
Diabetes

Validation of technology and publication of pre-clinical proof-of-concept data in 2020

Pre-clinical
Phase 1
Phase 2

hRPC

CTX cells

Exosomes platform

iPSC platform

Next Milestone

hRPCs cells

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

CTX cells

Future clinical development with Fosun and other potential partnerships

Exosomes platform

Proof of concept data from pharma/biotech research collaborations

iPSC platform

Validation of technology and publication of pre-clinical proof-of-concept data in 2020

Our competitive advantages

We are positioned for success...

A year of progress towards
changing patients’ futures.

hRPC stem cell therapy candidate for retinal diseases

Our progress to date:

  • Positive interim efficacy data from patients treated in the Phase 2a segment of the ongoing Phase 1/2 study were announced in October 2019.
  • Subsequent long-term efficacy data from the study continue to show a meaningful clinical effect from the therapy at all time points post-treatment.
  • Clinical trial protocol amendment approved by the FDA and MHRA to expand the Phase 1/2a study.
  • Application approval received from the MHRA to open a site in Oxford, UK with Professor Robert MacLaren, a world-renowned leader in the treatment of retinal diseases, as Principal Investigator.

What’s coming up:

  • The Group expects to commence treating patients shortly in both the US and the UK under the revised approved study protocol, subject to a continued easing of COVID-19 related restrictions at the relevant clinical sites.
  • On this basis, the Company expects to present further data from the expanded Phase 1/2a clinical trial during the next twelve months and expects to have sufficient data from the study to enable it to seek approval in the second half of 2021 to commence a single pivotal clinical study with its hRPC cell therapy candidate in RP.

Exosome nanomedicine platform

Our progress to date:

  • Expansion of intellectual property estate via the grant of a number of key patents covering our exosome technology platform. Patents were granted in China, Korea, Japan and Europe.
  • Grant-funded collaboration initiated with European Cancer Stem Cell Research Institute to develop novel systems to enable the delivery of therapeutic nucleic acids across the blood brain barrier using our exosomes.
  • In April and June 2020, we announced separate commercial collaboration agreements to explore the potential of our exosomes to deliver therapeutic agents to the brain.

What’s coming up:

  • Targeting our exosome technology programe towards value-generating business partnerships in which exosomes may be exploited as a potential third-party drug delivery vehicle.
  • The group is developing an exosome displaying proteins characteristic of the SARSCoV-2 coronavirus with the objective of the exosome being used to deliver a vaccine against COVID-19. 

iPSCs: expanding our therapeutic platform

Our progress to date:

  • New data presented, supporting use of iPSCs to develop new immortalised cell lines as potential therapeutic agents for subsequent licensing to third parties.

What’s coming up:

  • The Group’s iPSC platform enables the derivation of different stem cell populations that can be utilised for the production of exosomes with specific tissue targeting, or as new cell-based therapeutic candidates, thus providing further scope for a wide range of industry-based partnerships.

CTX stem cell therapy candidate for stroke disability

Our progress to date:

  • During the period we continued to progress the clinical development of our CTX cell therapy candidate for stroke disability.
  • Positive data from PISCES II Phase 2a clinical trial of CTX in stroke disability was published in a peer reviewed journal.
  • Patient recruitment in the US based PISCES III Phase 2b study was put on hold due to COVID-19 related restrictions, will remain suspended in the US for the foreseeable future; clinical trial sites will be kept open and patients already treated will be followed up over time in line with the clinical trial protocol.
  • Exclusive licensing partner in China, Shanghai Fosun Pharmaceutical Industrial Development Co., Ltd. (“Fosun Pharma”), continues to pursue development of CTX cell therapy in the licensed territory (Greater China including Hong Kong, Macao and Taiwan).
  • Clinical trial applications have recently been filed by Fosun Pharma to open clinical sites in the licensed territory.

What’s coming up:

  • Clinical sites to be opened, by Fosun Pharma, in the licensed territory to build on the clinical data generated in the US.
  • The CTX cell therapy candidate will be made available for licensing in stroke disability outside China and will also be made available for licensing in other indications such as Huntington’s disease.

Performance

Chairman’s statement

ReNeuron has a clear focus to deliver value-generating data across its programmes over the next twelve months.

John Berriman
Non-executive Chairman

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Chief Executive Officer’s review of performance

During the period under review, and subsequent to it, we have continued to generate encouraging positive efficacy data from the ongoing US Phase 2a clinical trial of our hRPC cell therapy candidate in retinitis pigmentosa.

Olav Hellebø
Chief Executive Officer

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