Ionis partner Biogen announces that results from Phase 3 VALOR study and open-label extension of tofersen showed clinical benefit in SOD1-ALS patients

Twelve-month data show that earlier initiation of tofersen slowed decline across measures of clinical and respiratory function, strength and quality of life Tofersen also led to robust and sustained reductions in neurofilament, a marker of neurodegeneration SOD1-ALS is a rare, progressive and fatal genetic form of the disease leading to loss of everyday functions and affecting approximately 2% of people with ALS CARLSBAD, Calif., June 3, 2022 /PRNewswire/ - Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) partner Biogen today announced results from the Phase 3 VALOR study and its open-label extension (OLE) of tofersen, an investigational antisense medicine being evaluated for people with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). The data show that earlier initiation of tofersen, compared to delayed initiation, slowed declines in clinical function, respiratory function, muscle strength and quality of life. At the time of the analysis, because the majority of participants survived without permanent ventilation (PV), the median time to death or PV could not be estimated. However, early survival data suggest a lower risk of death or PV with earlier initiation of tofersen. These results are based on new integrated data from the VALOR pivotal study and its OLE study.

The release as fetched from its publisher. ionis.com ↗