ReNeuron upbeat on retinitis pigmentosa therapy development
Cell-based therapeutics developer ReNeuron Group updated the market on its trading on Thursday, reporting that patient dosing had now started in the US in the expanded phase 2a clinical trial of its hRPC cell therapy candidate in retinitis pigmentosa (RP) patients.
The AIM-traded firm said the expanded study would enable the treatment of up to a further nine patients, beyond the 10 phase 2a patients already treated, at a higher dose level, under a revised protocol at clinical sites in both the US and the UK.
It described RP as 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 previously announced positive efficacy data from patients in the study at all time-points, including for the first patient treated, out to 18 months follow-up.
As it announced in its preliminary results on 20 July, ReNeuron said it was expecting to present further data from the expanded phase 2a clinical trial during the next 12 months.
“We expect this expanded Phase 2a study to generate sufficient data to enable the company to seek regulatory approval in the second half of 2021 to commence a single pivotal clinical study with our hRPC cell therapy candidate in RP,” the board said in its statement.
“The pivotal study will be designed to demonstrate further the efficacy of this treatment and, assuming a successful outcome, to enable ReNeuron to seek marketing approvals for its hRPC cell therapy candidate in RP in selected major markets.”
The RP programme had been granted orphan drug designation in both Europe and the US, as well as fast track designation from the US Food and Drug Administration (FDA).
“Orphan drug designation provides the potential for a significant period of market exclusivity once the therapy is approved in those territories,” the board said.
“Fast track designated products may also be eligible for accelerated approval and priority review programmes offered by the FDA.”
On 31 July, ReNeuron announced that the US Patent and Trademark office (USPTO) completed its examination of its patent application entitled ‘Phenotype profile of human retinal progenitor cells’, and issued a notification of allowance for the issue of a patent.
The allowed patent would protect the composition of the firm’s hRPC cell therapy candidate for retinal diseases, and add further intellectual property protection to the hRPC technology, which already had patent protection in a number of major territories including Europe, Japan and Australia.
“Our exosome technology is being exploited as a novel vector for delivering third party biological drugs and this partnering strategy reflects increasing industry interest in exosomes.
“We stated in our preliminary results statement that we anticipated further collaborations with pharmaceutical [and] biotechnology companies to commence over the coming months.
“We are pleased to report that we have subsequently signed a further research evaluation agreement with a leading biotechnology company in connection with the use of the company's proprietary exosomes for the delivery of novel therapeutics.”
In addition, the company said it was expecting to be able to sign further collaboration agreements, currently under negotiation, in the near term.
“Our exosomes are derived from our CTX human neural stem cell line.
“They have a natural ability to cross the blood brain barrier and can thus be used to deliver therapeutics for diseases of the brain.
“These exosomes can be produced through a fully qualified, xeno-free, scalable process and the clinical-grade source cell-line ensures consistent exosome product.”
ReNeuron said the exosomes can be loaded with a “diverse range” of potential therapeutics, such as siRNA/mRNA/miRNA, CRISPR/Cas9, antibodies, peptides and small molecules.
At 1521 BST, shares in ReNeuron Group were up 7.67% at 96.9p.