Benzinga | Oct 22, 2020 07:12AM EDT

Ionis Announces First Patients Have Been Dosed In ION541 Trial For ALS

- Trial will evaluate ION541 (BIIB105) for treatment of most forms of ALS regardless of family history

- Tofersen and IONIS-C9Rx also currently in clinical trials targeting genetic forms of the disease

CARLSBAD, Calif., Oct. 22, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), the leader in antisense therapeutics, today announced that the first patients have been dosed with ION541 (also known as BIIB105), an investigational antisense medicine being developed as a potential therapy to treat most forms of amyotrophic lateral sclerosis (ALS) regardless of family history. This is another milestone in the continuing progress of Ionis' ambitious program to develop novel treatments for ALS. Almost all cases of ALS share the pathological hallmark of 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.