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Jump to Key Points Sept 22 (Reuters) - Otsuka Pharmaceutical and Ionis Pharmaceuticals said on Tuesday their experimental drug met the main goal of a late-stage study in patients with a rare inherited form of amyotrophic lateral sclerosis, or ALS.
Here are more details:
• Otsuka's ulefnersen improved function and survival compared with placebo in patients with FUS-ALS, a rare inherited form of the disease that damages nerve cells controlling movement.
• There are currently no approved treatments specifically targeting the genetic cause of FUS-ALS.
- Ulefnersen met primary endpoint in late-stage FUS-ALS trial.
- Improved function + survival vs placebo in inherited FUS-ALS.
- ↓ nerve cell damage markers; delayed disease progression.
- No approved therapies target FUS-ALS genetic cause.
- Safety profile favorable; most AEs mild/moderate.
• The drug also reduced markers linked to nerve cell damage and delayed disease progression.
• Most side effects were mild or moderate, and the companies said the drug showed a favorable safety profile.
• Otsuka and Ionis plan to discuss the results with the US FDA and other health authorities globally as they pursue potential accelerated approval pathways.
• FUS-ALS causes progressive muscle weakness that can leave patients unable to move, speak, swallow or breathe independently.
• Otsuka's ulefnersen is an experimental medicine designed to reduce production of the FUS protein linked to the disease.
• Separately, Otsuka launched a global early access program for eligible FUS-ALS patients who cannot participate in clinical trials, allowing physicians to request access to ulefnersen before any potential approval.
(Reporting by Sahil Pandey in Bengaluru; Editing by Leroy Leo)
