ALS is a rare, progress neurodegenerative disorder that affects approximately 55,000 people globally. Almost all cases of ALS display TDP-43 protein aggregation in motor neurons. ION541 targets ataxin-2 RNA (ATXN2), which has been shown to prevent or reverse TDP-43 toxicity in preclinical models of ALS.

Ionis received a payment of $10 million from Biogen for the initiation of the phase I/II clinical trial of ION541. Biogen is developing ION541 as part of a broad collaboration with Ionis to advance novel antisense therapies for the treatment of neurological disorders.

Ionis' inhaled antisense drug shows promise as CF treatment
Ionis Pharmaceuticals has released positive data from a clinical trial evaluating IONIS-ENAC-2.5Rx, an investigational antisense medicine designed to...

Ionis, Genuity Science partner to advance RNA-targeted therapies
Ionis Pharmaceuticals and Genuity Science have entered a multiyear collaboration to accelerate the discovery and development of genetically validated...