Eplontersen, formerly known as IONIS-TTR-LRx and AKCEA-TTR-LRx, is an investigational ligand-conjugated antisense (LICA) medicine designed to inhibit the production of TTR protein. We are developing eplontersen as a monthly self-administered subcutaneous injection to treat all types of ATTR.
About Hereditary Transthyretin Amyloidosis
Hereditary transthyretin amyloidosis (ATTRv-PN) is caused by the accumulation of misfolded mutated TTR protein in the peripheral nerves. Patients with ATTRv-PN experience ongoing debilitating nerve damage throughout their body resulting in the progressive loss of motor functions, such as walking. These patients also accumulate TTR in other major organs, which progressively compromises their function and eventually leads to death within five to fifteen years of disease onset. There are an estimated 40,000 addressable patients, which includes those with ATTRv-PN and those with ATTRv-mixed phenotype worldwide.
Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.