Otsuka and Ionis' ulefnersen hits main goal in late-stage FUS-ALS trial
Otsuka and Ionis' ulefnersen met its primary endpoint in a late-stage FUS-ALS study, improving function and survival with a favorable safety profile.
Otsuka Pharmaceutical and Ionis Pharmaceuticals announced on Tuesday that their experimental drug ulefnersen met the primary endpoint in a late-stage clinical trial involving patients with FUS-ALS, a rare inherited form of amyotrophic lateral sclerosis.
The trial showed that ulefnersen improved function and survival compared with placebo in patients with the condition, which progressively damages nerve cells that control movement. The drug also reduced markers associated with nerve cell damage and slowed disease progression.
FUS-ALS currently has no approved treatments that specifically target its genetic cause. The disease leads to worsening muscle weakness that can eventually leave patients unable to move, speak, swallow or breathe without assistance.
Ulefnersen is designed to lower production of the FUS protein, which is linked to the disease. In the study, most side effects were mild or moderate, and the companies described the drug's safety profile as favorable.
Otsuka and Ionis said they plan to discuss the results with the US Food and Drug Administration and other health authorities worldwide as they pursue potential accelerated approval pathways.
Separately, Otsuka has launched a global early access program for eligible FUS-ALS patients who cannot take part in clinical trials. The program allows physicians to request access to ulefnersen before any potential approval.