What the company does
Human retinal progenitor cells (hRPC)
Human retinal progenitor cells are cells that differentiate into components of the retina.
Reneuron has developed the ability to scale up the manufacturing of the hRPCs using a patented low-oxygen cell expansion technology.
The hRPC cell therapy candidate is being evaluated in an ongoing phase I/IIa clinical trial in the US in subjects with a blindness-causing inherited retinal disease, retinitis pigmentosa (RP).
CTX cell therapy candidate is a treatment for patients left disabled by the effects of a stroke.
Reneuron’s product is a standardised, clinical and commercial-grade cell therapy product capable of treating all eligible patients presenting with the diseases targeted, without the need for additional immunosuppressive drug treatments.
Data from the Phase II PISCES trial indicated CTX therapy was safe and well-tolerated and produced clinically meaningful and sustained improvement in the level of disability and dependence as well as motor function.
Exosomes are nanoparticles, released by cells, and contain a number of active proteins and micro RNAs, which are short non-coding RNAs capable of regulating gene expression, that are believed to play a key role in cell-to-cell communication.
ExoPr0, Reneuron’s first CTX-derived exosome therapeutic candidate, has demonstrated potential as both a novel therapeutic candidate as well as a drug delivery vehicle
How is it doing
Its human retinal progenitor cells (hRPC), meanwhile, have scored some early success.
A Phase I/II assessment of a very small group of sufferers of a blindness-causing disease called retinitis pigmentosa saw a significant improvement in vision after treatment.
Six months after treatment there was a mean improvement of 18.5 per treated eye, with a mean improvement of 12 letters per treated eye after nine months, whereas inexorable disease progression is the norm for this disease.
With a total of 22 patients now treated and the study still ongoing, ReNeuron said the efficacy in subsequent patients was seen but at a lower rate and magnitude, with improvement in visual acuity ranging from +5 to +11 letters in the treated eye three months after treatment.
In February, clinical data from the PISCES II clinical trial were published in peer-reviewed Journal of Neurology, Neurosurgery, and Psychiatry.
In the six months to 30 September, losses were narrowed to £3.9mln from £5.36mln, while cash burn for the six months fell to £5.15mln from £7.54mln.
Cash at the end of the period was just over £21mln, giving it two years’ runway at the current burn rate.
What the boss says: Michael Hunt, CFO
“We are very encouraged by the data as it matures, it needs to mature further in the patients that we have treated more recently, but the bottom line is all systems go for that programme and we are looking forward to discussing with the regulatory authorities where we go next in terms of the next trial we would like to do.”
“It’s fair to say that the responses we got in the first three patients were so extraordinarily good that it was going to be quite an achievement to try and replicate that in all patients that we treated, but what we are seeing is a nice improvement in all patients that we have treated who have responded to the treatment and the aggregate scores that we are seeing, if you like, for visual accuracy are extremely encouraging.”
“We have a product here and I think you’ll find that view is backed up by the investigators working with us on the study.”
- The company presented data at the Annual Congress of the European Society of Gene and Cell Therapy that showed for the first time that its CTX treatment could be used to produce new, non-donor-specific stem cells to develop therapies for diseases that currently have an unmet medical need
- ReNeuron's hRPC therapy was hailed as a "major step forward" by Benjamin Yerxa, chief executive of the Foundation Fighting Blindness, as he addressed the American Academy of Ophthalmology's Annual Meeting in San Francisco in mid-October
- The RP trial results were presented in detail at the American Academy of Ophthalmology Annual Meeting in San Francisco and at the Ophthalmology Innovation Summit.
- On the CTX front, a Phase IIb trial, called PISCES III, is underway.
- The results from this trial are expected in the first half of 2021 because patient recruitment has been slower than anticipated due to the nature and complexity of the study.
- ReNeuron remains in negotiations with potential partners for all of its key programmes.