US trial to fast-track Mesoblast in Japan

Stem cell therapy pioneer Mesoblast will try to shave years off approval times for several of its product candidates.

Mesoblast chief executive Silviu Itescu.

Stem cell therapy pioneer Mesoblast will try to shave years off approval times for several of its product candidates by using its late-stage clinical results in the US to get full or conditional approval in Japan.

The company will use Japan’s fast-tracked trial regimen, and potential partnerships with Japanese pharmaceutical companies, to try to craft a quick and low-cost path to new revenue streams in the world’s second-largest stand-alone pharmaceutical market.

Mesoblast chief executive Silviu Itescu told a recent conference of Japanese business leaders in Australia that the company would apply for early approval under Japan’s new streamlined clinic trial regimen for its chronic heart failure treatment.

The company hopes the Japanese move will see the heart failure therapy conditionally approved at the same time as it receives US approval if not before. Mesoblast believes Japan could become a substantial revenue stream.

“We are working on the basis we could get parallel approvals in both of the world’s major established healthcare markets — the US and Japan,” Dr Itescu told The Australian.

Earlier this year, Mesoblast, through its Japanese licensee, achieved its first commercial product, TEMCELL, treating the rare but life-threatening condition of acute graft versus host disease in Japan.

But success with a treatment for the far more common condition of chronic heart failure, which affects 1½ million Japanese, has vastly greater earning potential and would help turn around a torrid period for the company.

“It is a major unmet need — it is those patients with a high risk of recurrent hospitalisation or death,’’ Dr Itescu said.

Under the changes introduced by Japan in 2014 to accelerate approval for stem cell therapies, companies can use the results of phase two trials in the US with Japanese bridging studies to achieve conditional approval to market the product in Japan. A positive phase-three trial will lead to full approval.

Dr Itescu said the US phase-three trial for the product candidate had already recruited 300 patients and the remainder would be enrolled next year.

“The results of that trial, provided we have a stand-alone small trial in Japan as well, would support full approval we believe in Japan. It would put us many years ahead of any potential competitors. It is the same with our back pain product. We are also looking to leverage our US phase-three trial results in chronic low back pain to obtain full Japanese approval.”

Mesoblast is also hoping to confirm a partner in the US for its heart failure therapy to replace Israeli generic drug manufacturer Teva, whose decision to walk away from the remainder of the trial triggered a slump in the share price to a seven-year low of $1.02. The company last traded at $1.16, up 14 per cent slightly on that level, but well down on its historic high of $9.67 in 2011.