Ionis Pharmaceuticals, Inc. partner Biogen 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 (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.
In 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.
Biogen, 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.
"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 an important step forward in our commitment to find new treatments for this devastating disease. We applaud the efforts of Biogen, whose advancement of tofersen reflects the promise of our broad partnership to develop medicines for the treatment of neurological diseases," said Brett P. Monia, Ph.D., chief executive officer of Ionis.
"Given Ionis' long-standing commitment to the ALS community, we are encouraged by the results of the Phase 3 VALOR study of tofersen and its open-label extension, which showed signs of slowing disease progression in people living with SOD1-ALS," said C. Frank Bennett, Ph.D., Ionis' executive vice president, chief scientific officer and franchise leader for neurological programs. "On behalf of all of us at Ionis, I would like to express our deep gratitude to the patients, families, scientific investigators, and others who participated in the VALOR study."
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