Ionis Pharmaceuticals Inc.’s (NASDAQ: IONS) partners Biogen has announced topline results from the placebo-controlled pivotal third phase VALOR trial of tofersen (BIIB067). Tofersen is an experimental antisense medicine under evaluation for superoxide dismutase 1 amyotrophic lateral sclerosis (ALS).
Tofersen didn’t attain the primary endpoint
While tofersen did not reach the primary objective of change from baseline to week 28 on the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), it did show positive trends on a number of secondary and exploratory disease activity and clinical function measures.
Furthermore, a pre-specified data integration from VALOR and the current open-label extension study (OLE) confirmed these findings, demonstrating that early tofersen initiation resulted in less decline across multiple measures in people with SOD1-ALS, including motor function, muscle strength, respiratory function, and quality of life. Procedure discomfort, pain in extremity, headache, fall, and back pain were all mild to moderate adverse events in both VALOR and OLE studies.
Biogen, which acquired tofersen from Ionis in 2018, revealed today that it is working closely with regulators, patient advocacy groups, the medical community, and other important stakeholders throughout the world to determine the best way to develop the drug.
VALOR study showed reduced disease progression
Ionis CEO Brett Monia said, “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.”
Chief scientific officer and neurological programs franchise leader Frank Bennett, “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.”