2016
by: Andrew Ward, Pharmaceuticals Correspondent
A Welsh cell therapy company founded by a Nobel laureate has agreed a partnership with Daiichi Sankyo, the Japanese pharmaceuticals group, in a sign of the growing momentum behind advanced therapies that repair faulty cells and tissue.
Cardiff-based Cell Therapy will receive an upfront payment of £12.5m from its new partner for the Japanese rights to aregenerative medicine for heart disease, with further potential payments subject to development milestones and commercial success.
The deal will add credibility to a company founded by Sir Martin Evans, winner of the2007 Nobel Prize for medicine for stem cell research, and chaired by Lord Digby Jones, former UK trade minister.
The company’s Heartcel product involves the injection of modified cells into a patient’s heart tissue to repair the scarring associated with heart failure.
Regenerative therapies have been slow to emerge since first being touted in the 1990s as the next big frontier in medical science.
However, products are now beginning to reach market. The European Medicines Agency approved its first cell therapy — a treatment for damaged corneas developed by Chiesi of Italy — in 2014.
Lord Jones said the deal with Daiichi Sankyo opened a path to market in Japan while providing more funds for Cell Therapy to advance its product in Europe and, eventually, the US. “It validates our science and increases the confidence we have going into our next stage of development,” he said.
Cell Therapy received the go-ahead from the EMA last year to begin the application process for conditional approval based on promising early trial data. This would allow the company to start selling its product while collecting further evidence of its safety and efficacy.
So far, Heartcel has been tested on only 11 patients with severe heart failure but all of them remain alive after 36 months. This compares with an average 70 per cent annual mortality rate for such patients using existing treatments.
Under the deal announced on Tuesday, Daiichi Sankyo will take responsibility for development, regulatory approval and marketing of Heartcel in Japan. Cell Therapy will retain full rights in the rest of the world.
11
Patients to have received Heartcel therapy remain alive 36 months on, against an average 70% annual mortality rate for existing treatments
Like the EMA, Japanese drugs regulators are offering fast-track approval processes for cell therapies as part of wider efforts by the Japanese government to promote development of regenerative medicines.
Ajan Reginald, chief executive and co-founder of Cell Therapy, said he hoped Heartcel would be on the market in Europe and Japan by late 2017 or early 2018, subject to further positive trial results.
Sir Martin, who was the first scientist to cultivate mice stem cells in a laboratory in 1981, remains active in the company as chief scientific officer and serves as chancellor of Cardiff university.
Cell Therapy is one of two companies developing stem cell treatments from a base in south Wales. The other, Aim-listed ReNeuron, was lured from the south-east of England by incentives from the Welsh government, which is trying to develop a life science cluster in the region.
Lord Jones said an initial public offering of Cell Therapy was possible in future but not this year and that the company had enough cash to keep going for at least two years......................................................................................................................................................................................2 Eye gene and cell therapy advances in battle on blindness
Gene and cell therapy R&D makes advances 2016
by: Clive Cookson
The eye is a tempting target for researchers developing innovative stem cell and gene therapies. It is small, easily accessible, biologically well understood and transparent, so the effect of treatment can be evaluated non-invasively.