Ionis initiates Phase 3 trial of novel antisense medicine to treat leading cause of juvenile-onset ALS

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[{"type":"text","content":"- ION363, the first medicine to specifically target FUS-ALS, is among Ionis' wholly owned assets the company plans to commercialize\n - 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\n - 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 University\n\n\nCARLSBAD, Calif., April 5, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today 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 gene (FUS). 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.\n\n \n \n \n \n \n \n\n \nALS is a rare, rapidly progressing and fatal neurodegenerative disorder that affects approximately 55,000 people globally.i FUS-ALS is the third most common genetic cause of ALS. People with ALS experience muscle weakness, loss of movement, and difficulty breathing and swallowing, resulting in a severely declining quality of life and eventually death.\n\"There is an urgent need for novel treatments for all forms of ALS, a devastating disease that affects far too many patients and their families. Advancement of ION363 to a pivotal trial is the latest example of the power of Ionis' antisense technology to potentially target the root causes of neurological diseases,\" said C. Frank Bennett, Ph.D., Ionis' chief scientific officer and franchise leader for neurological programs. \"Driven by our experience in developing medicines for motor neuron diseases such as ALS and spinal...

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