Business
Ionis' partner Biogen provides update on tofersen Phase 3 VALOR study in SOD1-ALS
- In the Phase 3 VALOR study, the primary endpoint as measured by the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) did not reach
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"- In the Phase 3 VALOR study, the primary endpoint as measured by the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) did not reach statistical significance; however, signs of reduced disease progression across multiple secondary and exploratory endpoints were observed\n - The totality of evidence from VALOR and its ongoing open-label extension showed that participants who started tofersen earlier experienced better outcomes, further suggesting a positive clinical effect\n - Given the high unmet medical need, Biogen will expand its ongoing early access program (EAP) to the broader SOD1-ALS population\n - Topline data being presented today at the American Neurological Association 2021 Annual Meeting\n\n\nCARLSBAD, Calif., Oct. 17, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) partner Biogen today announced topline results from its placebo-controlled pivotal Phase 3 VALOR study of tofersen (BIIB067), an investigational antisense medicine being evaluated for people with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). While tofersen did not meet the primary endpoint of change from baseline to week 28 in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), trends favoring tofersen were seen across multiple secondary and exploratory measures of disease activity and clinical function. \n\n \n \n \n \n \n \n\n \nIn addition, a pre-specified integration of data from VALOR and its ongoing open-label extension study (OLE) reinforced these findings and showed that early tofersen initiation led to less decline across multiple measures including motor function, respiratory function, muscle strength, and quality of life in people with SOD1-ALS. Most adverse events in both VALOR and OLE were mild to moderate in severity, including procedural pain, headache, pain in extremity, fall and back pain.\nBiogen, which licensed tofersen from Ionis in 2018, also announced today that it is actively engaging with regulators, the medical community, patient advocacy groups and other key stakeholders around the world to determine next steps.\n\"The topline results of the Phase 3 VALOR study showed signs of reduced disease progression across key secondary and exploratory endpoints, including biomarker data, clinical outcomes and quality of life measures. These data represent...