Otsuka Pharmaceutical and Ionis Pharmaceuticals announce that their experimental drug ulefnersen met its primary endpoint in the Phase 3 FUSION trial. Involving 73 patients with FUS-ALS, a rare and rapidly progressive genetic form of ALS, the trial demonstrates statistically significant improvements in function and survival compared to placebo (p=0.0005). The partners plan to discuss expedited regulatory approval pathways with the FDA.
Phase 3 FUSION trial results
- ▪The Phase 3 FUSION trial of ulefnersen met its primary endpoint in 73 FUS-ALS patients, yielding a p-value of 0.0005 and demonstrating statistically significant improvements in function and survival compared to placebo.
- ▪The Phase 3 FUSION trial of ulefnersen met several secondary endpoints, including reducing serum neurofilament light chain levels and delaying time to death, permanent ventilation, rescue, or disease-related withdrawal.
Regulatory and access plans for ulefnersen
- ▪Otsuka Pharmaceutical launched a global early access program allowing physicians to request ulefnersen for eligible FUS-ALS patients who are unable to participate in clinical trials prior to regulatory approval.
- ▪Otsuka Pharmaceutical plans to discuss the Phase 3 FUSION trial results with the U.S. Food and Drug Administration and other global health authorities to explore potential expedited regulatory approval pathways for ulefnersen.
Ulefnersen and Ionis' ALS portfolio
- ▪Ulefnersen expands Ionis Pharmaceuticals' genetic amyotrophic lateral sclerosis portfolio, which also includes Qalsody, an FDA-approved drug targeting SOD1-related ALS developed in partnership with Biogen.
- ▪Ulefnersen is an experimental antisense oligonucleotide medicine designed to target the genetic cause of FUS-ALS by reducing the production of the disease-linked fused in sarcoma protein.
Characteristics of FUS-ALS
- ▪FUS-ALS is a rare, rapidly progressive, and fatal genetic form of amyotrophic lateral sclerosis caused by mutations in the fused in sarcoma gene, accounting for an estimated 0.6% of all ALS cases.
- ▪Mutations in the fused in sarcoma gene are highly prevalent in juvenile and pediatric amyotrophic lateral sclerosis, accounting for an estimated 43% to 52% of those cases.
Otsuka and Ionis licensing agreement
- ▪Under the 2024 licensing agreement, Ionis Pharmaceuticals remains eligible to receive regulatory and commercial milestone payments as well as tiered royalties on future net sales of ulefnersen.
- ▪Otsuka Pharmaceutical acquired exclusive global rights to ulefnersen from Ionis Pharmaceuticals in November 2024, paying an upfront fee of $10 million.
Debatable claims
- ▪Otsuka's early access program for ulefnersen is justified before regulatory approval
- ▪The FDA should grant expedited approval to ulefnersen for FUS-ALS
Story comments
Loading comments…