Athersys digs for victory after stroke disappointment

Having failed in what many saw as its last chance therapy area, Athersys has picked up its shovel and is heading to the data mine in the hope of striking statistical gold. The company will be banking that an analysis of patients who received its MultiStem treatment early on during their acute ischaemic stroke will turn up a subgroup of responders.

It is not just the group’s long-term future that lies in the last refuge of failed biotechs. So too does that of its partnership with Roche's Japanese subsidiary, Chugai, which struck a deal worth more than $200m before the binary-event trial (Upcoming events: Data for Athersys in stroke, Celladon in heart failure, Heron in emesis, April 2, 2015).

Luckily for Chugai, the money it paid up front was a very modest $10m, and it now gets to save a further $7m that had been tied to the outcome of the US phase II stroke study. MultiStem bombing in stroke follows a similar explosion a year ago in a phase II trial in ulcerative colitis, an indication to which Pfizer had rights.

Always look on the bright side

As MultiStem is Athersys’s only clinical asset the stroke indication offered the only realistic way of keeping investors on side. On Friday they made their thoughts on the future of the company more than clear, sending the stock down 54% to $1.40.

In its two-step 134-patient trial MultiStem failed to show any difference compared with placebo on the Global Stroke Recovery Assessment scale, or any improvement in stroke symptoms.

MultiStem’s second study failure does not, however, appeared to have dented the optimism of the group’s management team. Gil Van Bokkelen, Athersys's chief executive, pointed to the lower mortality among patients who had received a single dose of MultiStem 24-48 hours after their stroke – despite the lack of statistical significance.

He also added that patients who received the treatment within 24-36 hours appeared to have a better outcome, and confirmed that “further analyses” of the data would be conducted.

Risky business

Athersys’s failure should act as a reminder that stem cell therapy remains a risky area, despite the recent interest in the space prompted by Celgene's investment in Mesoblast (Cautious Celegene endorsement turns Mesoblast round, April13, 2015).

The difference between the deals was not just Mesoblast’s larger size and experience compared with Athersys, but also the treatment areas at which the groups were looking. Stroke is notoriously difficult, and while that makes the discovery of any possible treatment a licence to print money it also means that it is a space littered with failure.

Athersys is studying MultiStem in other indications. But with two failed trials under its belt, it appears few investors are willing to bet that forthcoming trials in acute myocardial infarction and acute respiratory distress syndrome will yield better results.

Nevertheless, with $26.1m in the bank at the end of last year and a management team displaying the blinding optimism common to all prospectors, Athersys is likely to keep chipping away.

Project Study  Detail  Trial ID
MultiStem - 134 pts - safety and efficacy in adults who have suffered an ischaemic stroke NCT01436487

To contact the writer of this story email Lisa Urquhart in London at [email protected] or follow @LisaEPVantage on Twitter

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