ReNeuron makes solid progress with drug candidates

Cell-based therapeutic development company ReNeuron Group updated the market on its current trading on Wednesday, as investors gathered for its annual general meeting.

The AIM-traded firm said it was finalising the “relevant data packages” to enable it to submit an IND application, to commence a pivotal Phase III clinical trial with its CTX cell therapy candidate for stroke disability in the US.

“We expect to make this submission, as planned, in the final quarter of this year, with the study expected to commence in early 2018, subject to the requisite regulatory approvals,” the board said in its statement.

“We expect data from the Phase III study about two years later, in early 2020.”

As it had previously reported, the FDA specifically recommended that ReNeuron apply for a Special Protocol Assessment (SPA) for the proposed Phase III clinical trial.

The company said the SPA process was reserved for clinical studies considered “pivotal” in support of product marketing label claims.

“Further, we also plan to apply for Regenerative Medicine Advanced Therapy (RMAT) designation for our CTX cell therapy candidate for stroke disability,” the board said.

“The benefits of RMAT designation are similar to those of Breakthrough Therapy designation, including increased interactions with the FDA during development and eligibility for priority review and accelerated marketing approval.”

ReNeuron said the ongoing US Phase I/II clinical trial of its human Retinal Progenitor Cell (hRPC) cell therapy candidate for retinitis pigmentosa (RP) was also proceeding “well”.

The company said all nine patients in the Phase I element of this study had now been treated, with short term safety and tolerability data expected in the final quarter of this year.

“The final high dose cohort of patients was treated with the newly developed cryopreserved formulation of our hRPC therapeutic candidate.”

In order to garner the appropriate depth and quality of data to allow subsequent progression to a pivotal study in RP, ReNeuron said it was currently finalising the relevant protocols to enlarge the Phase II clinical development plan in that indication.

Based on that, the board said it expected additional readouts from the RP Phase I/II clinical trial in the second half of 2018, with further Phase II efficacy data from a larger cohort of patients expected in mid-2019.

“Further, we intend to file an application in the final quarter of this year to commence a Phase II clinical trial with our hRPC cell therapy candidate in patients with cone-rod dystrophy (CRD), to run concurrent with the Phase II testing of this candidate in RP.

“CRD is a group of rare eye disorders associated with a loss of cone cells in the retina that initially results in deterioration of central visual acuity and colour vision.”

ReNeuron said CRD frequently affected patients in childhood and had no cure, and it expected Phase II efficacy data from the CRD programme in the second half of 2019.

“Finally, in conjunction with our academic collaborators, we are continuing to generate preclinical data relating to our exosome development programme,” the board reported.

It said exosomes are nanoparticles secreted from cells, including its proprietary CTX stem cell line.

They are said to play a “key role” in cell-to-cell signalling, and ReNeuron said early research with ExoPrO - its first CTX-derived exosome therapeutic candidate - had demonstrated that it may have a “significant effect” in regulating cell growth and apoptosis in cancer.

“Over the past year, we have generated and presented important data relating to the characterisation, purification and in vivo biodistribution of ExoPrO, demonstrating its potential both as a novel therapeutic candidate and as a drug delivery vehicle,” the ReNeuron board explained.

On the basis of the progress made, and subject to continued success with ongoing pre-clinical development work, the company said it hoped to be able to commence clinical development with ExoPrO within the next year to 18 months, targeting solid tumours.

“Our therapeutic development programmes continue to progress to plan, with further near term milestones in prospect as our stroke programme moves into Phase III clinical development and our retinal disease programmes move into Phase II clinical development,” commented chief executive officer Olav Hellebø.

“We look forward to reporting further progress with these programmes and our exosome development programme over the coming months.”