Reneuron Share Discussion Threads

[cottoner] 25/2/2019 22:08

What happened
Seemingly every company that mentions the term "gene therapy" or "gene editing" is rising today after Roche announced it would acquire Spark Therapeutics for $4.8 billion. That represents a premium of more than 120% for the gene therapy pioneer, which is bringing a treatment for a rare eye disease to the market. The genetic medicine, branded as Luxturna, will cost $425,000 per eye. It has gotten off to a slow start.

The acquisition of Spark Therapeutics shows there's an appetite among established pharma companies for promising genetic cures and treatments. Or at least that's what investors and Wall Street analysts are hoping. The excitement has sent shares of many related companies higher today.


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31 January 2019

ReNeuron Group plc

("ReNeuron" or the "Company")

New exosome production scale-up data presented

ReNeuron Group plc (AIM: RENE), a UK-based global leader in the development of cell-based therapeutics, is pleased to announce that new data relating to the manufacturing scale-up of its GMP produced, CTX-derived exosomes will be presented today at a leading US forum on the manufacture of cell and gene therapies.

Commenting on the new data, Dr Randolph Corteling, Head of Research at ReNeuron, said: "Exosomes are biological nanoparticles ideally suited to the delivery of oligonucleotide and gene-based therapies due to their natural occurrence and abundance, their ability to protect their cargo from degradation, and their potential for favourable bio-distribution. The ability to scale-up production of our CTX-derived exosomes, as demonstrated by these new results, further exemplifies the capability of ReNeuron's exosome platform."

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Will that do? :-)

[pdt] 25/2/2019 20:52

I see Roche is buying Spark Therapeutics for $4.8 billion (Spark have Luxturna to treat RP patients with a particular faulty gene).

This had led to other gene therapy companies stock jumping today such at Nightstar Therapeutics up 15%, they have gene therapy trials running in retinal diseases. They have a relatively small market cap of $500m. And MeiraGTx up 10%.



I remember a quote from RENE saying that Spark treat 2% of the RP population with this faulty gene and they are trying to treat the other 98% because the stem cell therapy approach is gene independent.

[philh75] 23/2/2019 17:36

I'd say they will get a partner by August in RP with upfront fee and trial costs covered by the partner, a discussion with FDA will shorten the trial required, submission for marketing approval in early 2021 or before.

[philh75] 23/2/2019 16:44

Oh dear rocket fuel, looks like you've been burnt by buying too many of these too early.

Even if they do dilute to enable us to take these drugs all the way through, it would still be worth it.

Why don't you join us for the ride?

[cottoner] 23/2/2019 16:43

Cash equivalents and bank deposits at 30 September 2018 of £30.67 million

Last placing was at a premium.

[rocket fuel] 23/2/2019 16:32

Watch out for the next mega placing!.. coming to a place near you!..

..these boys know how to dilute and burn the cash!

What's left in the bank won't last you a year! They love an upfront placing even with plenty cash showing.

MikeHunt will be bending you boys over soon!

[cottoner] 23/2/2019 14:32

Worth noting that RENE has been granted Fast Track designation for its human Retinal Progenitor Cell (hRPC) therapy candidate for retinitis pigmentosa (RP) and previously granted Orphan Drug Designation in both Europe and the US by the European Commission and the FDA, respectively.

Products with Orphan Drug Designation benefit from potential market exclusivity post-approval for up to 7 years in the US and up to 10 years in Europe.

[cottoner] 23/2/2019 14:26

Article about this weeks announcement posted on the Foundation Fighting Blindness blog,an American organisation which has supported RENE.



includes the comments

“We are very excited by the remarkable early results that we have seen now that we have moved into a patient population with some remaining vision,” says Richard Beckman, MD, chief medical officer, ReNeuron. “If these gains can be maintained, and similar results achieved in other patients with even better remaining vision, we will be well on the path towards having a huge impact on the lives of patients with this devastating disease.”

“We are encouraged by ReNeuron’s early results of vision improvement for RP patients. We look forward to learning if the vision improvements are sustained over the long term,” says Stephen Rose, PhD, chief scientific officer, Foundation Fighting Blindness. “ReNeuron’s therapy holds promise for restoring vision in people with RP and related conditions, independent of their gene mutation.”

[philh75] 23/2/2019 10:35

Michael hunt on RP data. Nice guy, I know he's trying not to over egg it but a little more enthusiasm in his voice would be good! Needs to switch to 'sales mode'

[pdt] 23/2/2019 09:23

Super - I am actually in a similar position to you, they have been on my watchlist for years. The RP results made me sit up and take notice.

The only other useful link I have come across was the last Corporate Presentation in December 2018 which gives a summary of where they currently are in their developments.



I would be interested to know your thoughts once you have had a closer look.

[supernumerary] 23/2/2019 00:01

Thanks - no I hadn't seen that - I've had rene on a watchlist for a long time without feeling the need to look too closely, but I was struck by the recent RP results and decided some proper dd was required.

From that presentation it seems that they're only using 1M cells compared to jCyte's 3M cells, so the sight improvement is outstanding - far better than I'd imagined. A better outcome in a shorter timescale with a lower dose - can't complain about that!

I'll dig a bit deeper over the next week or so. Do you have any further useful links? It's a pity the thread owner seems to have departed advfn - it would be good to have a more informative header.

[pdt] 22/2/2019 20:32

Supernumerary - Rene are using a dose of 1 million cells.

This is a good video presentation if you have not seen it;

[supernumerary] 22/2/2019 19:40

Thanks - that's what I saw, and found it hard to make a comparison. The rene results look much better - they do have an exceptionally strong signal for a clinical trial - I'm used to oncology trials where they seem to require a thousand patients before any relevant difference can be distinguished :( - but I'm not sure how deep that goes. If jCyte can just agree a new arm with increased dosing in their current trial (unlikely, but not impossible), then maybe they would achieve the same outcomes?

I suspect rene also has an advantage in manufacturing at scale, which seems to be an issue with these progenitor stem cells, but again, find it hard to pin it down. More research needed.

It would also be interesting to know more about why the putative partner walked. $2.5M is a decent sum to just throw away...

[pdt] 22/2/2019 19:19

The jCyte result I was looking at;



"To evaluate changes in visual acuity, the study assessed mean difference in BCVA change between treated and untreated eyes at specified time points throughout the study. In the lowest dose group (500,000 cells) the mean difference was 1 letter at 12 months after treatment, while at the highest dose (3 million cells), the mean difference was 9 letters"

Rene after a short period of time (18 days and 2 months) achieved a mean difference of 16 letters I believe. But it is only 3 people.

[supernumerary] 22/2/2019 19:06

They really need to push on with the pivotal trial. As far as I can see jCyte are marginally ahead in that they're already in their PH1/2, and as both have orphan drug status for very similar drugs, the winner - first to approval - could take all.

Somebody commented that these results look better than jCyte, but I don't think it's very clear. First 3 patients aren't statistically very strong, and secondly I suspect it's dose-related, and jCyte have chosen lower trial doses.

Does anybody have clarity on dosing for RENE? They're being very cagey about it - I can't find any mention of the 3 levels in the dose-ranging trial on clinicaltrials.gov, which is unusual, nor is there anything in the presentations. Is there a paper describing the dose-ranging trial?

[cottoner] 22/2/2019 17:53

Their valuation unchanged at 608p.

They see a wee bit of upside then. :-)

[philh75] 22/2/2019 17:47

Update from Edison:

Pretty upbeat.






ReNeuron’s announcement of early but exciting data on three patients in the clinical study of its human retinal progenitor cell (hRPC) product was well received. Although the patient numbers are small and the follow-up time points are short (two months and 18 days), the improvements in visual acuity are striking and may enable an earlier start to the pivotal programme.

We have made no changes to our valuation or probabilities at this stage. However, we recognise that early evidence of efficacy may enable ReNeuron to advance to a pivotal study more quickly, subject to discussions with regulators.

[philh75] 21/2/2019 17:00

No problem PDT,

I think you've probably made a sound investment.

[cottoner] 21/2/2019 14:29

For info :




First Patients in Arizona Receive Experimental Stem Cell Treatment for Eye Disease


Phoenix, AZ, February 11, 2019 --(PR.com)-- Retinal Consultants of Arizona & the Retinal Research Institute – one of the nation’s leading retina & vitreous clinical research and patient treatment facilities – is proud to announce that Dr. Pravin U. Dugel has conducted the first stem cell therapy treatment for patients with retinitis pigmentosa (RP) in Arizona.

The stem cell therapy – human retinal progenitor cells (hRPC) – is being investigated for the treatment of RP. RP is a disease affecting the nerve cells involved in vision that are found in the retina (tissue of the eye used to see). hRPC is being investigated in subjects with RP to see if hRPC, when injected into the retina, can slow the degeneration of, or even possibly replace these retinal cells that are used for vision.

“This clinical trial is an exciting step forward in stem cell therapy,” said Dr. Pravin U. Dugel, managing partner, Retinal Consultants of Arizona & Retinal Research Institute, Phoenix, AZ; clinical professor, USC Roski Eye Institute, Keck School of Medicine, University of Southern California, Los Angeles, CA. “Our goal as physicians is to advance the diagnosis and treatment of retina diseases affecting millions of people globally. Advancements like hRPC are critical to the treatment of patients who experience vision loss or blindness due to retinitis pigmentosa and are currently without hope."

According to the trial sponsor, UK biotechnology company ReNeuron, pre-clinical studies have demonstrated that retinal implantation of human retinal progenitor cells (hRPC) have the potential to preserve existing photoreceptors, potentially reducing or halting further deterioration of vision. In addition, the progenitor cells have been shown to mature into functional photoreceptors that engraft into the photoreceptor layer, bringing the possibility of restored vision.

The hRPC used in this study is investigational. “Investigational” means the hRPC is a study agent being tested and has not yet been approved for use as a medicine by the United States Food and Drug Administration (FDA). See full study details at ClinicalTrials.gov:

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[cottoner] 21/2/2019 14:13

extract

Pravin Dugel, managing partner, retinal consultants of Arizona and a first Phase II cohort’s investigator, said: “When I heard about the rapidity and magnitude of the visual gain in the first patient that Jason treated in this cohort, I was hopeful, but sceptical. Now that I saw this repeated in my first two patients, I am very excited indeed.”

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Pravin Dugel will be speaking today Thursday Feb 21st at the first GENE THERAPY FOR CHRONIC RETINAL DISEASES SUMMIT IN NEW YORK




He is obviously very eminent in the field of retinal diseases and so for him to be 'very excited' with the initial results of RENE's trial surely speaks volumes.

[pdt] 21/2/2019 13:42

philh75 - thanks for posting a link to the evaluate article.

I bought in yesterday and today on the back of the very impressive early eye results.

Compares very well to the Jcyte results.

[cottoner] 20/2/2019 23:16

cash equivalents and bank deposits at 30 September 2018 of £30.67 million

[robizm] 20/2/2019 22:55

Very heavy cash burn on this one. Raised money at £5 A share so way down and a long way to go to making money.

[philh75] 20/2/2019 20:06

Good to get a mention too on twitter from Prof Sir Chris Evans:

Delighted at the breaking news that 3 US patients with blindness from serious retinitis pigmentosa in Boston and Arizona now have substantially improved vision following treatment in a trial with our new hRPC stem cell therapy developed by ReNeuron (1/2)