Ionis Pharmaceuticals Inc. (NASDAQ:IONS) has announced the dosing of the first patient with its investigational antisense ION541, also called BIIB105. The company is developing the medicine as a potential therapy for treating most amyotrophic lateral sclerosis (ALS) forms irrespective of family history.
Commencement of trial vital in developing ALS treatment
This is a huge milestone in the on-going progress of the company’s ambitious program of developing proprietary ALS treatments. Almost all ALS cases share the pathological TDP-43 protein aggregation hallmark in motor neurons. The treatment that targets ataxin-2 RNA (ATXN2) has shown the potential to prevent or reverse TDP-43 toxicity in the preclinical ALS model.
Amyotrophic lateral sclerosis is a rare, fatal, and progressive neurodegenerative disease affecting around 55,000 people globally. Usually, around 90% of the cases happen to people that don’t have a family history of the disease. ALS patients experience loss of movement, muscle weakness, and difficulties in breathing and swallowing, leading to declining quality of life and eventually death.
Frank Bennett, the company’s chief scientific officer and franchise leader for neurological programs, said that ION541 is the company’s third medicine designed for treating various ALS forms that is in clinical trials. He said that ION541 represents another example of the company’s antisense technology’s strength in targeting the root cause of debilitating neurodegenerative diseases. Benett added that the ION541 clinical trial commencement is a huge milestone for Ionis’ ALS program and reaffirms the commitment of the company to the ALS community.
Ionis received $10 million to commence a clinical trial
The company received a $10 million payment from Biogen to commence this Phase 1/2 ION541 clinical study. Biogen is developing ION541 as part of the strategic partnership with Ionis in advancing proprietary antisense therapies to treat various neurological disorders.
Other leading experimental therapies from Ionis for ALS treatment are IONIS-C9RX (BIIB078) and tofersen (BIIB067) both of which are being developed through the partnership Biogen.Tofersen has been designed to minimize superoxide dismutase 1 production which is the cause of genetic ALS while IONIS-C9RX reduces mutant C9ORF72 RNA and related neurotoxicity.