Biotech Values

[iwfal]

AVXS IONS BIIB

AveXis Inc. today announced that the planned pivotal study of AVXS-101 in spinal muscular atrophy (SMA) Type 1 will reflect a single-arm design, using natural history of the disease as a comparator, and enroll approximately 20 patients. This update is based on the receipt of the minutes following the Type B meeting with the U.S. Food and Drug Administration (FDA) held on September 30, 2016.

FWIW I think their drug likely has enough efficacy to show benefit against historical - but in aggregate it is the wrong choice, albeit from a set of choices of which none look really good.

1) Without question the immanent approval of Nusinersen is making things difficult at AVXS.

1a) if they wait until after approval they either have to go head-to-head or run a trial as an additive therapy - and both have high $ and risk costs

1b) if they somehow get a trial started now they run the substantial risk that they cannot complete enrollment - and even if they do, they cannot possibly prevent the kids from taking Nusinersen thus tainting the historical comparison. About the only possible hope they have here is to somehow roll out testing kits etc before IONS/BIIB does (BIIB/IONS are trying, even now, to find all the SMA kids ASAP to enroll them in Compassionate Use - and not only is that the right thing ethically, it also steals all AVXS' air, perhaps forcing them to the trial path that, in the long term, is better for patients.)

2) Unlike most historical comparison trials, I do think that they probably have enough efficacy to, eventually, be successful. That said, I think that the focus on historical comparisons in naive - probably chosen because it sounds faster. But the reality is that it probably is slower because the separation needed vs historicals to rule out chance is a lot larger.

Altogether they don't really have any good options, but I think a historical comparison is even worse than normal because you NEVER, EVER run a historical comparison trial thru a giant change in SOC.