Arimoclomol failure leaves Biogen/Ionis exposed

Biogen is under increasing pressure – as if it needs it – to deliver with its Ionis-partnered mRNA project tofersen in amyotrophic lateral sclerosis. The asset looks increasingly exposed among pivotal-stage ALS projects after the recent failure of Brainstorm’s NurOwn and that of Orphazyme’s arimoclomol, announced this morning. Investors will have to wait another week to find out the data from arimoclomol’s pivotal ALS trial, Orarials-01, when these are presented at the Encals meeting; for now, Orphazyme says in the 245-patient study arimoclomol failed to beat placebo on either its primary measure, 76-week CAFS score, or secondary endpoints, which included the ALSFRS-R score and slow vital capacity. This comes after US FDA said Brainstorm’s unequivocally negative pivotal study of the stem cell therapy NurOwn was insufficient for filing, Orion’s levosimendan failed in the Refals trial, and Amylyx was told that it had to run a full phase 3 study with AMX0035. Tofersen’s phase 3 Valor study, ending mid-2021, is the next big readout for ALS. In addition to failing in ALS arimoclomol has also flunked trials in inclusion body myositis and Niemann-Pick disease, though Orphazyme is pursuing a filing in the last indication.