Calico has reported initial topline results from Regimen F of the HEALEY ALS Platform Trial for fosigotifator (ABBV‑CLS‑7262), a potential treatment for amyotrophic lateral sclerosis (ALS), according to the company.
The trial evaluated primary and exploratory high doses over a 24‑week period against a shared placebo cohort. The company said that fosigotifator did not meet the primary endpoint assessing disease progression—combining functional decline (ALSFRS‑R) and survival—at either dose. Secondary measures related to respiratory function, quality of life and overall muscle strength also failed to reach statistical significance for the primary dose.
However, data from the exploratory high‑dose cohort showed slower deterioration in muscle strength for both upper and lower extremities compared to placebo. There was also a potential signal for reduced respiratory decline. Further analysis of hand‑held dynamometry and biomarker results is ongoing and will be presented at a future medical meeting.
Safety findings were consistent across groups, with treatment‑emergent adverse event rates similar between active and placebo arms. The company said that fosigotifator was safe and well‑tolerated during the trial period.
Calico claims that while these results are disappointing, the observed signals support further exploration of fosigotifator in ALS and other CNS disorders, including vanishing white matter disease and major depressive disorder.
The findings underscore the complexity of developing effective ALS therapies but suggest potential with targeted high‑dose regimens in future studies.
