hRPC for retinal disease
During the period under review, and subsequent to it, we have made significant
progress advancing the clinical development of our human retinal progenitor cell
(hRPC) therapy candidate in the blindness-causing disease, retinitis pigmentosa
(RP). A Phase 1/2a open-label clinical trial is ongoing to evaluate the safety,
tolerability and preliminary efficacy of our hRPC stem cell therapy candidate in
patients with advanced RP. The Phase 2a element of the study, which uses a
cryopreserved hRPC formulation, enrols subjects with some remaining retinal
function and is being conducted at two clinical sites in the US: Massachusetts Eye
and Ear in Boston and Retinal Research Institute in Phoenix, Arizona.
In February 2019, we reported positive preliminary data in the first cohort of three
patients in the Phase 2a element of the study, with all three subjects in the cohort
demonstrating a rapid improvement in vision compared with their pre-treatment
baseline.
In April 2019, further data from the first patient cohort in the study were presented
at the sixth annual Retinal Cell and Gene Therapy Innovation Summit in Vancouver,
Canada, which preceded the 2019 annual meeting of the Association for Research
in Vision and Ophthalmology. In the presentation, it was reported that the first
cohort of patients in the Phase 2a element of the study had demonstrated a
sustained and further improvement in vision compared with baseline, with a mean
improvement from baseline in visual acuity of + 23 letters on the ETDRS eye chart
in the treated eye (the untreated control eyes did not show meaningful
improvement). An improvement of + 23 letters is equivalent to reading an
additional four lines of letters on the ETDRS eye chart, the standardised eye chart
used to measure visual acuity in clinical trials. An improvement of at least + 15
letters from baseline is considered to be clinically meaningful by the US Food and
Drug Administration (FDA), as stated in their recent guidance on gene therapy for
retinal disorders. In addition to these objective measurements, all three subjects
had also noted a subjective improvement in vision in their treated eye.
Dosing of the second cohort of three subjects in the Phase 2a element of the study
is complete and dosing of the remaining two cohorts is in progress. These later
cohorts comprise patients with a greater baseline level of visual acuity than those
patients earlier in the study, as we seek to assess preliminary efficacy in patient
groups with differing levels of remaining vision. The clinical protocol for the study
allows for up to 12 patients (four cohorts of three patients each) to be treated in
the Phase 2a element of the study.
We expect to treat the remaining patients in the study shortly and to report
preliminary data from all treated Phase 2a subjects in October at the American
Academy of Ophthalmology 2019 Annual Meeting in San Francisco. These results
will form the basis of our future interactions with the European and US regulatory
authorities regarding the future clinical development path of hRPC for the
treatment of RP. Our clinical programme in RP benefits from Orphan Drug
Designation in both Europe and the US, as well as Fast Track designation from the
US Food and Drug Administration (FDA).

ReNeuron Group plc (AIM: RENE), a UK-based global leader in the development of cell-based therapeutics, will webcast the analyst briefing on the day of its preliminary results, Thursday 11 July 2019.

To listen to the webcast live, please log on to the following web address approximately 5 minutes before 10.00am BST on the day of results:
https://webcasting.buchanan.uk.com/broadcast/5d011332221579216107d918

A recording of the webcast will later be made available at ReNeuron’s website, www.reneuron.com.

The analyst briefing will be held at 10.00am BST on 11 July 2019 at the offices of Buchanan, 107 Cheapside, London EC2V 6DN.

ReNeuron Group plc (AIM: RENE), a UK-based global leader in the development of cell-based therapeutics, will webcast the analyst briefing on the day of its preliminary results, Thursday 11 July 2019.

To listen to the webcast live, please log on to the following web address approximately 5 minutes before 10.00am BST on the day of results:
https://webcasting.buchanan.uk.com/broadcast/5d011332221579216107d918

A recording of the webcast will later be made available at ReNeuron’s website, www.reneuron.com.

The analyst briefing will be held at 10.00am BST on 11 July 2019 at the offices of Buchanan, 107 Cheapside, London EC2V 6DN.

For more information, please contact Buchanan on reneuron@buchanan.uk.com
or 020 7466 5000.

ReNeuron Group plc (AIM: RENE), a UK-based global leader in the development of
cell-based therapeutics, will webcast the analyst briefing on the day of its
preliminary results, Thursday 11 July 2019.
To listen to the webcast live, please log on to the following web address
approximately 5 minutes before 10.00am BST on the day of results:
https://webcasting.buchanan.uk.com/broadcast/5d011332221579216107d918
A recording of the webcast will later be made available at ReNeuron’s website,
www.reneuron.com.
The analyst briefing will be held at 10.00am BST on 11 July 2019 at the offices of
Buchanan, 107 Cheapside, London EC2V 6DN.
For more information, please contact Buchanan on reneuron@buchanan.uk.com

hRPC moves to centre stage

The recent early but striking data on ReNeuron's hRPC product for treating RP has become the focus of investor attention. This is because the data reported so far are good, the study has a placebo-controlled element (with one of the patient’s eyes untreated) and the results for all 12 of the patients in the Phase IIa study are expected to be reported in H219. We have not changed the timelines for the first regulatory approval for the hRPC product, which we had estimated to be in 2023, but the possibility exists that this timeline could accelerate if the data continue to prove compelling. In a similar way, the clinical trial results to date have raised the profile of this programme and, bearing in mind the cash-rich, innovative product-poor status of many big pharmaceutical and biotechnology companies, we would not be surprised to see another licensing transaction for the hRPC product.

ReNeuron Group Plc (RENE) Insider Buys £180,000 in Stock
Posted by Anthony Bellafiore on Apr 27th, 2019

ReNeuron Group logoReNeuron Group Plc (LON:RENE) insider John Edward Berriman bought 80,000 shares of the company’s stock in a transaction dated Thursday, April 11th. The shares were purchased at an average price of GBX 225 ($2.94) per share, with a total value of £180,000 ($235,201.88).

Shares of RENE opened at GBX 310 ($4.05) on Friday. ReNeuron Group Plc has a one year low of GBX 29.50 ($0.39) and a one year high of GBX 257 ($3.36). The stock has a market capitalization of $98.14 million and a price-to-earnings ratio of -7.33.

stem cell researcher Reneuron up 22% due to positive clinical trials for a blindness treatment. That brought year-to-date returns to 520% making it the second largest riser in the FTSE Aim All Share Index this year, just behind 541.7% returns from MX Oil, according to Bloomberg data

ReNeuron Group plc
(“ReNeuron” or the “Company”)
Clinical update and conference presentation
Latest results show sustained and further improvement in vision at 60 and 120 days in first
patient cohort of Phase 2a study of hRPC cell therapy in retinitis pigmentosa
ReNeuron Group plc (AIM: RENE), a global leader in the development of cell-based
therapeutics, is pleased to announce updated positive preliminary data in the Company’s
ongoing Phase 1/2a clinical trial of its human retinal progenitor cell (hRPC) therapy candidate
in the blindness-causing disease, retinitis pigmentosa (RP). All three subjects in the first
cohort of the Phase 2a element of the study have demonstrated a sustained and further
improvement in vision compared with their pre-treatment baseline.
These latest results are being presented today by Jason Comander MD, PhD, Associate
Director, Inherited Retinal Disorders Service, Massachusetts Eye and Ear, and Assistant
Professor, Harvard Medical School, at the sixth annual Retinal Cell and Gene Therapy
Innovation Summit in Vancouver, Canada, which precedes the 2019 annual meeting of the
Association for Research in Vision and Ophthalmology (ARVO) taking place on 28 April – 2
May.
Summary of the preliminary efficacy data (visual acuity measured using the standardised
ETDRS chart, five letters per line):
At most recent follow-up, subjects in the study showed a mean improvement from baseline
in visual acuity of + 23 letters in the treated eye. The untreated control eyes did not show
meaningful improvement (mean change from baseline of + 5 letters, range - 2 to + 12 letters).
First subject 9 letters + 20 letters (60 day follow-up ) + 21 letters (120 day follow-up)
Second subject 9 letters + 15 letters (18 day follow-up) + 25 letters (60 day follow-up)
Third subject 32 letters + 14 letters (18 day follow-up) + 23 letters (60 day follow-up)
An improvement of + 23 letters is equivalent to reading an additional four lines of letters on
the ETDRS eye chart, the standardised eye chart used to measure visual acuity in clinical trials.
An improvement of at least + 15 letters from baseline on the ETDRS chart is considered to be
clinically meaningful by the US Food and Drug Administration (FDA), as stated in their recent
guidance on gene therapy for retinal disorders. As a comparator, the difference between a
patient with 20/20 vision and 20/200 vision (the latter being the legal definition for blindness
in terms of central visual acuity) would be the equivalent of being able to read an extra ten
lines on the ETDRS chart.
In addition to these objective measurements, all three subjects have also noted a subjective
improvement in vision in their treated eye.
Pravin Dugel MD, Managing Partner, Retinal Consultants of Arizona, Phoenix, Arizona, and
Clinical Professor, Roski Eye Institute, USC Keck School of Medicine, Los Angeles, California,
and study investigator, commented:
“I am excited that the rapid and remarkable visual improvement in these RP patients has been
sustained and even improved upon at 60 and 120 days. It is especially gratifying to feel the
excitement and joy in my patients where this objective and subjective improvement in their
vision has been a source of hope following years of slow but steady progression towards
blindness.”
RP is a group of hereditary diseases of the eye that lead to progressive loss of sight due to
cells in the retina becoming damaged and eventually dying. The Company’s RP clinical
programme has been granted Orphan Drug Designation in both Europe and the US, as well as
Fast Track designation from the FDA.
The Phase 1/2a clinical trial is an open-label study to evaluate the safety, tolerability and
preliminary efficacy of ReNeuron’s hRPC stem cell therapy candidate in patients with
advanced RP. The Phase 2a element of the study, which uses a cryopreserved hRPC
formulation, enrols subjects with some remaining retinal function and is being conducted at
two clinical sites in the U.S. – Massachusetts Eye and Ear in Boston and Retinal Research
Institute in Phoenix, Arizona.
The Company notes that these data remain early and it will continue to generate further data,
including regular ongoing monitoring of the treated subjects, to continue to assess durability
of effect and efficacy over a longer period of time and in a larger number of patients.
Olav Hellebø, Chief Executive Officer of ReNeuron, commented:
“The extent of vision improvement observed in this patient cohort demonstrates the
potential for our hRPC cell therapy candidate to make an enormous difference in the lives of
patients with RP. Treatment has already begun in the next cohort of patients, who have a
greater baseline level of visual acuity than those treated so far. The results from this cohort
will be presented in due course.”

“The business remains significantly undervalued, and we look forward to further clinical data on the retinal programme at the end of April, and potential further licensing activity, as near-term catalysts to build on recent momentum,” it said in a note earlier this month.

Scientists on brink of CURING blindness after patient has eyesight RESTORED

BRITISH scientists are on the brink of curing blindness following the success of a procedure which repairs damaged retina.

Scientists have successfully improved the vision of three legally blind patients, with one having their eyesight restored. All three patients had retinitis pigmentosa, an inherent disease which slowly constricts vision. The disease currently has no cure but a British firm has reported early success with a procedure which helps to repair the damaged retina.

Before the procedure, the patients were legally blind and could only read the largest group of letters on a special eye test chart.

But 18 days after being injected with stem cells, their sight had improved to the point where they could read three letter sizes smaller.

One patient is no longer classified as legally blind and another said she was able to see the food on her plate for the first time in years.

Olav Hellebo, chief executive of UK biotech firm ReNeuron, said the women went from being capable of seeing nine letters on the eye test chart to 29.

People are considered legally blind if they can read less than 36 letters on the 100-letter chart.

Mr Hellebo said: “She said she could now see the food on her plate, which is really motivating for us to hear.”

The two men improved their eyesight from nine to 24 and from 31 to 45.

The treatment for retinitis pigmentosa requires growing billions of progenitor stem cells in a laboratory.

These have the capability to transform themselves into other types of cells depending on where they are located in the body.



One million stem cells are injected into the back of the patient’s eyeball.

Once there, they transform into new light-sensitive cells called rods and cones which replace ones that have been lost prematurely because of genetic flaws.

Olav Hellebo said the tests on the patients had produced “exciting” results.

“We are obviously very excited. We have to bear in mind all the caveats – that these are results in only three patients and it is early days – but the reaction from ophthalmologists has been very encouraging,” Mr Hellebo told the Mail on Sunday.

Development of the technology has been led by experts at ReNeuron in Bridgend, Wales.

A further nine patients will now have the procedure.

Retinitis pigmentosa, which affects around 25,000 people in the UK, is caused by about 100 inherited genetic defects.

The loss of vision can start in childhood, adolescence or adulthood.

It starts with deteriorating night vision and peripheral vision which then narrows and leaves people with only hazy tunnel vision.

Total blindness usually follows.

Tina Houlihan, of the Retina UK charity which supports people with inherited sight loss, said: “These early results are encouraging and will provide hope to those living with retinitis pigmentosa.

“However, while the trial is at this very early stage, with only a very small number of patients involved, we are cautious in our optimism.”

Foundation Fighting Blindness to Host 6th Annual Retinal Cell & Gene Therapy Innovation Summit Before ARVO
SOURCE Foundation Fighting Blindness

The Summit, which will bring physicians, scientists, and industry together to collaboratively advance innovation for emerging retinal therapies, will be held Friday, April 26, 2019.

COLUMBIA, Md., April 23, 2019 /PRNewswire/ -- The Foundation Fighting Blindness, a national nonprofit focused on funding cutting-edge retinal disease research, will co-host the sixth annual Retinal Cell and Gene Therapy Innovation Summit, in partnership with the Casey Eye Institute at Oregon Health & Science University. The Summit – one of the world's most comprehensive reviews (three dozen presenters) of ongoing and planned clinical research for retinal degenerative diseases – will be held in Vancouver, British Columbia, on Friday, April 26, 2019, prior to the 2019 annual meeting of the Association for Research in Vision and Ophthalmology (ARVO).

At the summit, representatives from biotech and pharma industries will come together with members of the physician and scientist communities to discuss rapidly emerging retinal gene, cell and novel therapies and strategize how to move the emerging options forward. The summit features presentations by leading retinal disease experts on potential gene and stem-cell therapies and how best to deliver them to patients.

"The purpose of the Summit is to create visibility for the many projects based on gene or cell therapy approaches that are in or entering the clinic," said Brian Mansfield, PhD, executive vice president, interim chief scientific officer. "These approaches, many of which were initiated or supported with funding from the Foundation, hold strong promise for the treatment of diseases of the retina, such as age-related macular degeneration and inherited conditions such as retinitis pigmentosa and Stargardt disease. This progress is occurring due to the expertise and collaboration of companies, clinicians, and scientists, many of whom will attend the summit."

Summit registration is open to academic, physician, biotech and pharmaceutical representatives, as well as industry media interested in learning more about the collaborative advancements featured at the Summit. For additional information, please visit https://www.regonline.com/InnovationSummitVancouver.

Presentations of Note
FRIDAY, APRIL 26

1:45 PM – 2:00 PM: Subretinal implantation of human retinal progenitor stem cells
Dr. Jason Comander, Massachusetts Eye and Ear

Here, Dr. Comander will review early, encouraging vision improvements for the ReNeuron Phase 2 retinal progenitor clinical trial for patients with retinopathy of prematurity.

ReNeuron Group Plc (LON:RENE) insider John Edward Berriman acquired 80,000 shares of the company’s stock in a transaction dated Thursday, April 11th. The shares were bought at an average price of GBX 225 ($2.94) per share, for a total transaction of £180,000 ($235,201.88).

“The business remains significantly undervalued, and we look forward to further clinical data on the retinal programme at the end of April, and potential further licensing activity, as near-term catalysts to build on recent momentum,” it said in a note earlier this month.