An interim analysis of AstraZeneca and partner Ionis Pharmaceuticals' eplontersen showed the investigational drug met its co-primary endpoints in a late-stage trial in patients with a rare, fatal disease.
Last December, AstraZeneca 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 commercialize eplontersen. Eplontersen, formerly known as IONIS-TTR-LRx, is a ligand-conjugated antisense (LICA) investigational medicine designed to reduce the production of TTR protein at its source to treat both hereditary and non-hereditary forms of transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) — a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade.
According to the partners, the positive high-level results from the NEURO-TTRansform phase 3 trial in patients with hereditary ATTRv-PN showed eplontersen met its co-primary endpoints in a planned interim analysis at 35 weeks. In the trial, eplontersen reached a statistically significant and clinically meaningful change from baseline for its co-primary endpoint of percent change in serum transthyretin concentration, reducing TTR protein production.
Based on the trial results, the companies plan to file an NDA with the U.S. FDA later this year for patients with hereditary ATTRv-PN.
ATTRv-PN is expected to be the first indication for which AstraZeneca and Ionis will seek regulatory approval for eplontersen. The drug is also currently being evaluated in a phase 3 trial for amyloid transthyretin cardiomyopathy (ATTR-CM).