An interim analysis of AstraZeneca and partner Ionis Pharma’s eplontersen showed the experimental drug met the main goals in a late-stage trial in patients with a rare, fatal disease, the Anglo-Swedish drugmaker said yesterday.
Based on the results, the companies plan to file an application to market the therapy in the US later this year in patients with hereditary transthyretin-mediated amyloid polyneuropathy.
The disease – which affects an estimated 40,000 patients globally – leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade, according to AstraZeneca.
The Cambridge, UK-based company agreed to pay California-based Ionis $200 million upfront, with up to $485 million in conditional payments following regulatory approvals as well as up to $2.9 billion in sales-related milestone payments in a deal to jointly develop and commercialise the drug last year.
Edits by EP News Bureau