Phase 3 Trial of Jacifusen for Amyloid Lateral Sclerosis With Fused-in-Sarcoma Mutation

A phase 3 clinical trial (NCT04768972) was initiated for jacifusen (ION363; Ionis Pharmaceuticals, Calsbad, CA) for potential treatment of amyotrophic lateral sclerosis (ALS) with mutations in the fused-in-sarcoma gene (FUS). Jacifusen is an investigational antisense medicine made to reduce the production of the FUS protein from FUS mRNA. Jacifusen has the potential to reduce or prevent disease progression in individuals with FUS-ALS by targeting the root cause of FUS-ALS. 

"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 neurologic diseases," said C. Frank Bennett, PhD, 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 muscular atrophy and our intimate connection to the ALS patient community, Ionis made the decision to advance ION363 to the clinic and, ultimately, to the market because we believe we are uniquely positioned to make it available to patients living with FUS-ALS." 

The phase 3 trial of jacifusen is a global multicenter study with the enrollment of 64 participants. The first part of the trial consists of participants will be randomly assigned to receive either jacifusen (one of multiple doses being tested) or placebo for 29 weeks. The second part of the trial will be an open-label period in which all participants will be treated with jacifusen for 73 weeks. The trial will be led by Neil Shneider, MD, PhD, director of Columbia University's Eleanor and Lou Gehrig ALS Center. 

"FUS-ALS is an atypically aggressive form of the disease, involving the youngest of ALS patients. Building on our expanded access program, a controlled clinical trial is the best way to demonstrate the efficacy of ION363 and to make this therapeutic available to all patients who could potentially benefit from it," said Dr. Shneider.