– ION363, the first medicine to specifically target FUS-ALS, is among Ionis’ wholly owned assets the company plans to commercialize– ALS portfolio now includes four clinical-stage investigational antisense medicines designed to treat the root causes of genetic and non-genetic forms of the disease– Journey to pivotal clinical study began with Ionis’ commitment to Jaci Hermstad, the first patient treated with ION363 under a ‘compassionate use’ protocol led by Dr. Neil Shneider of Columbia UniversityIonis Pharmaceuticals, Inc.
announced the initiation of a Phase 3 clinical trial of ION363 in patients with amyotrophic lateral sclerosis (ALS) with mutations in the fused in sarcoma
). Patients with a mutation in the FUS
gene develop a rare form of ALS, referred to as FUS-ALS, which is the most common cause of juvenile onset ALS. There is substantial evidence that mutations in the FUS
gene are responsible for a toxic gain of function that can lead to rapid, progressive loss of motor neurons in patients with FUS-ALS. ION363 is an investigational antisense medicine targeting the FUS RNA to reduce the production of the FUS protein. Antisense-mediated reduction of mutant FUS protein in a FUS-ALS mouse model prevents motor neuron loss. By targeting the root cause of FUS-ALS, ION363 has the potential to reduce or prevent disease progression in FUS ALS patients.