ReNeuron Outlines First-To-Market Plans For Stroke Disability Cell Therapy

ReNeuron Group PLC is planning "world domination" for stem cell therapy use in ischemic stroke, says CEO Olav Hellebø; the UK biotech wants to commercialize its potential blockbuster therapy alone in the US and Europe, but seek a local partner to help conquer the market in Japan.

ReNeuron's lead compound, CTX (ReN001), is being developed as a cell therapy treatment to reverse the functional deficits associated with stroke disability. The company expects to launch a pivotal Phase III trial in the first half of 2018, once it has secured a Regenerative Medicine Advanced Therapy (RMAT) designation for the product in the US. This is a slight delay from previously announced plans to launch a pivotal trial for CTX in late-2017, but ReNeuron's chief Hellebø told Scrip he was more concerned with getting the trial design right than sticking to a former schedule.

"We want to make sure we have a perfectly good protocol and a great Phase III setup before we pull the trigger," Hellebø said.

ReNeuron was advised by the FDA to seek a Special Protocol Assessment (SPA) for the Phase III trial, as well as a RMAT designation, after an end of Phase II meeting with the regulator. The benefits of RMAT designation are similar to those of Breakthrough Therapy designation, and include increased interactions with the FDA during development and eligibility for priority review and accelerated marketing approval.

Hellebø noted that while an SPA for the therapy was an asset, it takes time to put in place. "We have planned for a lot of back and forth with the FDA before we are able to start the pivotal study for CTX. We are being realistic with the time for that trial to start." The Phase III trial for CTX, expected to last two years, has a target enrolment of 220 patients.

In the previous Phase II PISCES trial, ReNeuron's cell therapy did not quite meet its primary endpoint. However, data from the study were considered promising by the FDA – especially when added to strongly positive results in its other endpoints and the large unmet medical need for treatments able to reverse stroke disability. (Also see "ReNeuron Stroke Stem Cell Therapy Advances" - Scrip, 6 Dec, 2016.)

ReNeuron does not expect to seek a partner for commercialization of CTX in the US and believes it can also launch the product in Europe; however, the company would consider a marketing deal in Europe if the right offer came along, Hellebø noted.

"Our plan is world domination," Hellebø said, adding that the number commercial sales staff for needed CTX in the US is "quite low" at around 100 employees, and the job will be "very doable, even for a small company."

Approximately 800,000 suffer from stroke in the US each year.

Meanwhile, in Japan ReNeuron is actively seeking a partner for potential commercialization of CTX in the country.

Few Competitors For A Big Market

ReNeuron's closest competitor is SanBio Co. Ltd., which, in partnership with Sumitomo Dainippon Pharma Co. Ltd., is also developing a stem cell treatment, SB623, for stroke disability.

Hellebø told Scrip that the two products were similar in their mechanisms of action, but that CTX was slightly ahead of SB623 in the clinic. SanBio's drug, which is an allogeneic neural stem cell therapy, is currently being tested in Phase IIb trials.

The injection sites for the two companies' products are also slightly different, with ReNeuron injecting the treatment into the area of the brain with the stroke damage, while SanBio's therapy is targeted to the subcortical peri-infarct area of the brain.

With no current therapeutic options available for stroke disability reversal, Hellebø said there was "room for both of us on the market". Still, he believes ReNeruon has the opportunity to be the first to market in this space.

Looking Ahead

Next year will be a busy year of clinical development for ReNeuron as the company also plans to initiate a Phase II trial for its cell therapy treatment ReN003 for the treatment of retinitis pigmentosa – an orphan disease. Topline results for ReN003 from a Phase I/II trial are expected in the fourth quarter of this year.

In 2018, ReNeuron also hopes to run a Phase II study for ReN003 in cone-rod dystrophy (CRD), a group of related eye disorders that cause vision loss, alongside the retinitis pigmentosa program.

Despite the company’s expanding clinical trial programs and the delay of its pivotal stroke study until 2018, analysts at Stifel said in a June 29 note that "ReNeuron's cash is still likely to last until 1H FY19."

ReNeuron announced its FY17 results on June 29, with a loss for the period of £15.57m – a figure significantly lower than analysts' expectations of a loss of £24.7m, largely due to the pivotal stroke trial now being launched in 2018. Total available cash for the company sits at £53.5m.