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An experimental ALS drug may be buying patients more time. New 2-year data from the DAZALS phase 2 trial found that patients taking dazucorilant 300 mg/day had an 87% lower risk of death compared to those on placebo alone. The catch? The drug still didn't improve the functional decline measure it was originally designed to hit.
New exploratory data from the phase 2 DAZALS trial offer a striking survival signal for dazucorilant, an investigational oral drug that blocks cortisol signaling. Patients with ALS who received 300 mg/day of dazucorilant had an 87% lower risk of death over 2 years compared to placebo recipients who didn't cross over to active treatment — a finding consistent with the 84% risk reduction seen at the 1-year mark.
The drug also reduced key inflammatory markers (MCP-1 and MIP-1β) after 24 weeks, pointing to a possible biological mechanism tied to neuroinflammation. Side effects were mostly mild-to-moderate GI issues like nausea and abdominal pain.
By the Numbers:
Why it matters: ALS remains a devastating, fast-progressing disease with very few treatment options. While dazucorilant didn't slow functional decline — the trial's primary endpoint — a survival benefit of this magnitude is hard to ignore. A phase 3 trial launching in 2027 will be the real test of whether this signal holds up under rigorous randomized conditions.
