- Rare disease drug eplontersen delivers positive trial results
- FDA application expected in 2022
- Multi-billion-dollar market potential
Pharmaceutical giant AstraZeneca (AZN) said a Phase III study of eplontersen to treat a rare heart disease had delivered positive results. The drug achieved statistically significant and clinically meaningful reduction of the transthyretin protein.
AstraZeneca and joint development and commercialization partner Ionis (IONS:NASDAQ) now plan to file for application with the US Food and Drug Administration in 2022.
AstraZeneca shares ticked-up 0.2% to £100.67 on Tuesday.
In December 2021 the companies signed a deal whereby AstraZeneca paid Ionis $200 million up-front with additional conditional payments of up to $485 million following regulatory approvals.
It also agreed to pay up to $2.9 billion of sales-related milestones for sales thresholds of between $500m and $6bn, plus royalties in the range of low double-digit to mid-twenties percentage depending on the region.
Eplontersen will be jointly developed and commercialised by both companies in the US and will be developed and commercialised in the rest of the world by AstraZeneca except for Latin America.
The two companies have a history of working together dating from 2012. In 2015 they formed a strategic collaboration to work on developing therapies for cardiovascular, metabolic and kidney diseases.
RARE AND DANGEROUS DISEASE
The drug is designed to inhibit the production of transthyretin, or TTR protein which can build up to dangerous levels for those with the disease, causing heart problems and nerve damage.
Vice president of BioPharmaceuticals at Astrazeneca Mene Pangalos commented: ‘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.’
