LONDON – Cell therapy specialist Reneuron Group plc has sealed a Chinese commercialization and marketing deal, agreeing to an exclusive license for both its CTX treatment for disability caused by stroke and hRPC for treating retinitis pigmentosa, with Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.

The agreement will see Fosun Pharma fully funding development of CTX and hRPC in China, while Reneuron is in line for up-front, regulatory and in market milestones payments estimated at $104.8 million. In addition, the company is eligible for tiered royalties at rates between 12% and 14% on sales.

The deal is both a validation of Reneuron's technology and a demonstration of the increasing commercial viability of cell therapy, said Michael Hunt, chief financial officer. "The deal did not happen overnight and it is gratifying Fosun [has] chosen to license both products. It validates the technology and also contributes to financing of the company in the short term," he told BioWorld.

This is the first-ever out-licensing deal for Reneuron, which has been plugging away at CTX, an off-the-shelf, cryopreserved allogeneic product based on fetally-derived neural stem cells, since it was spun out of King's College London in 1997.

Shares in the Bridgend, Wales-based company (LON:REN) rose by 31.28% to £2.24 (US$2.92) on the announcement.

Changing regulatory landscape

Regulation of cell therapies in China was lax in the past, with the responsibility for oversight resting with the Ministry of Health, which had a brief to encourage research, whilst rooting out rogue stem cell clinics. There was no clear path to national regulatory approval.

The situation is now changing, with the National Medical Product Administration (NMPA, formerly the CFDA), setting out new principles for the approval of clinical trials for cell therapies at the start of last year, as part of its effort to set higher standards and bring China closer into line with international regulatory standards. (See BioWorld, Jan. 10, 2018.)

That laid out a route for national approvals for cell therapies. Hunt said Reneuron did some initial scouting and believes that while the regulatory scene is robust, help is needed to navigate it. "That's partly why we chose Fosun, as a responsible and significant player in China, that knows its way round the regulatory framework," said Hunt.

Fosun already has started to make its mark in cell and gene therapy, securing NMPA approval for a phase I trial of Kite Pharma Inc.'s Yescarta (axicabtagene ciloleucel) in the treatment of large B-cell lymphoma, in September last year. (See BioWorld, Sept. 19, 2018.)

The product, designated FKC-876 in China, is being developed by Fosun Kite Biotechnology Ltd., a joint venture set up by Fosun and Kite (now part of Gilead Inc.) in January 2017.

The collaboration with Reneuron will help Fosun build its position in cell therapy in China, said Yifang Wu, president and CEO of Fosun. "The licensed products and unique stem cell platforms will bring a lot of strategic synergies with our current business, addressing huge unmet needs in the Chinese market," he said.

Having signed the agreement, the two companies have to set out a detailed plan for taking the products forward. "The mechanism is in place, but we have no idea how long it will take to get clinical development underway," Hunt said.

Fosun will need to treat patients in China and will take on manufacturing for its home market; however, Reneuron will be responsible for supplying CTX and hRPC cells to Fosun until technology transfer is complete.

Step forward

Clinical development of CTX took a significant step forward at the start of 2019, when the first patient was treated in the U.S. phase IIb study. The placebo-controlled trial will involve 110 patients who are in a stable condition six to 12 months after having a stroke. Patients in the active arm will receive a dose of 20 million CTX cells injected into the brain.

The primary endpoint is a reduction in disability and dependency at six months post treatment. Top-line results are expected early in 2020.

To date, CTX has been administered to 34 patients in two U.K. trials. In the single-arm phase II, all 23 patients showed clinically relevant improvements.

In its retinitis pigmentosa program, Reneuron last week reported the positive effect seen in the first cohort of three patients in the U.S. phase I part of the study of hRPC has been sustained in the first patient cohort in the phase II part of the trial.

All three of the phase I cohort had a rapid and significant improvement in vision and on average were able to read an additional three lines of five letters on EDTRS, a standardized eye chart for measuring measure visual acuity.

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