AstraZeneca, Ionis Aim for New Drug for Rare Disease

An interim analysis of eplontersen, an experimental drug developed jointly by AstraZeneca and Ionis Pharmaceuticals, showed it met the main goals of a late-stage trial in patients with amyloid transthyretin cardiomyopathy (ATTR-CM) and amyloid transthyretin polyneuropathy (ATTR-PN). The companies plan to file for a new drug application in the US to sell and market this medication.

“Amyloid transthyretin polyneuropathy is a rare and fatal disease that can affect up to 40,000 people worldwide. These promising results show eplontersen has the potential to be a new and much needed treatment where limited options exist and significant unmet medical need remains,” said Mene Pangalos, Executive Vice President of BioPharmaceuticals R&D, AstraZeneca. In the 35-week interim analysis, eplontersen achieved a “statistically significant and clinically meaningful” reduction of serum transthyretin (TTR) production.

ATTR-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis. Without treatment, it is fatal within a decade. ATTR-CM is a systemic, progressive and fatal condition that leads to progressive heart failure and death within four years from diagnosis, said AstraZeneca.

“These encouraging data reinforce the safety profile of eplontersen and demonstrate clear evidence of its potential to provide much needed therapeutic benefit to patients living with hereditary transthyretin-mediated amyloid polyneuropathy,” said Teresa Coelho, an researcher for the trial.

Under the terms of agreement, eplontersen will be jointly developed and commercialized by both companies in the US and exclusively by AstraZeneca in the rest of the world, with the exception of Latin America. AstraZeneca paid US$200 million upfront to Ionis in December 2021 and will make additional payments of up to US$485 million following regulatory approvals. In addition, it will also pay up to US$2.9 billion of sales-related milestones.

AstraZeneca has been present in Mexico for over 60 years, impacting more than 4 million patients and training over 12,000 healthcare professionals through continuous education. Its Mexican workforce is made up of 1,100 professionals in the Mexico City offices, in the technological center in Guadalajara and the manufacturing plant in the State of Mexico, which produces around 25 million drugs every year.