Fosigotifator enters the HEALEY ALS platform as a 24-week Phase 2/3 regimen

Calico/Fosigotifator (ABBV-CLS-7262)

Fosigotifator, a Calico and AbbVie drug targeting the cell's stress-response pathway via EIF2b activation, entered the HEALEY ALS platform trial in March 2023 as a 24-week study of two doses in about 300 patients. The trial later missed its main goal of slowing ALS progression, though the high dose showed some benefit in slowing loss of limb strength.

• 24-week Phase 2/3 study comparing two doses in ~300 patients, enrolled 3:1 to active treatment or placebo.
• Primary outcome measured disease progression using ALS-Functional Rating Scale-Revised and mortality.
• High dose showed benefit slowing muscle strength deterioration but did not meet primary endpoint overall.
• Trial failed to show improvement in secondary endpoints of respiratory function and health-related quality of life.