Last December, ReNeuron reported that seven-out-20 patients taking part in the PISCES II trial saw a discernible improvement in their disability.
The AIM-quoted group said the positive response rates reported at three months after treatment were sustained at 12 months after treatment, with seven people still showing a “clinically relevant improvement”.
"We are delighted that the long term follow-up data from the PISCES II clinical trial has shown the potential of our CTX cell therapy candidate to permanently improve function in patients living with chronic consequences following stroke,” said ReNeuron chief executive Olav Hellebø.
“Further, the study we plan to commence early in 2018 will allow critical placebo-controlled data with CTX in stroke disability to be available earlier than originally planned.”
IND application to be lodged “shortly”
ReNeuron confirmed it will “shortly” submit an investigational new drug application to the US Food and Drug Administration to kick off a clinical trial in the US in stroke patients.
Data from that study is expected at some point in the second half of 2019. After that, the company reckons it will need one more trial in order to get global marketing approval of the therapy.
Rare eye disease drug “progressing well”
ReNeuron is more than one-trick pony though. It also has its human retinal progenitor cell (hRPC) therapy which is used to treat the degenerative eye disease retinitis pigmentosa.
At September’s AGM Hellebø told investors that the phase I/II clinical trial was “progressing well” with all nine patients in the Phase I part of the study having now been treated.
Short term safety and tolerability data from that part of the study is expected in the final quarter of this year.
ReNeuron is also working on finalising the “relevant protocols” to enlarge the Phase II clinical development plan.
It expects additional read-outs from the RP phase I/II clinical trial in the second half of 2018, with further Phase II efficacy data from a larger group of patients expected in mid-2019.
ReNeuron has developed a new formulation of hRPC which can be cryptopreserved (frozen), extending its shelf life and allowing it to be shipped anywhere around the globe.
An application will also be filed later this year to test the same hRPC therapy in a phase II trial in cone-rod dystrophy patients. ReNeuron said it hopes to have both phase II trials (RP and CRD) running concurrently.
Cancer-fighting cell technology
Early research into the company’s Exo Pr0 therapeutic candidate has shown that it may have a “significant effect” in regulating cell growth and reducing cancer cell proliferation.
ReNeuron hopes to take ExoPr0 into the clinic within the next year to 18 months, targeting solid tumours.
Exosomes are nano-sized particles in the bloodstream and other body fluids that can be absorbed by other cells.
Interestingly they can be used as a novel treatment candidate themselves or as a drug delivery vehicle.
New research, including by ReNeuron, points to them having a major impact on the way cells behave with a number of advantages in therapeutic treatments, especially in the way they can be engineered to be strong and precise delivery mechanism
At the end of March 2017 when it published its full-year results, ReNeuron had £53mln in the bank having burnt through £12.6mln in the period.
The company still only has a market cap of around £56mln, which means just a £3mln value is placed on its pipeline.
City broker N+1 Singer thinks the current share price of around 1.7p is far too cheap and believes they are worth closer to 9p each.
On top of the cash already in the bank, ReNeuron also benefits from various grants and R&D tax credits.
In fact, it was only recently awarded another grant by the Welsh government to advance its emerging exosome therapy platform.
The grant, awarded under the SMARTExpertise scheme, will help fund a £1.2mln collaborative programme of work to be undertaken by ReNeuron, Ig Innovations and Swansea University Medical School.