Readers of this blog probably know the story of the drug NP001. One phase I and two phase II trials were conducted by Neuraltus in ALS patients (NCT01281631 and NCT02794857). These were both 6-month studies, the usual clinical trial duration. Phase 2A was completed in 2012, and Phase 2B in 2017. Both failed. Now some people want to revive this drug, through a new biotech named Neuvivo.
What the authors of a new publication did was to try to find a subset of patients that showed some longer survival with NP001 in those old trials.
This is a widely used technique by unsuccessful companies, but it is statistically meaningless.
The authors found a subset of patients who had inflammation and a longer survival. Yet this analysis would be comically wrong if we were not speaking of dying people.
For example, the authors claim that survival of ALS patients with inflammation is 16 months longer than in the placebo arm. At 72 months there were only 3 people in the inflammation subset and two people in the placebo arm. Nobody can say anything about these numbers. If we use the same criteria, it shows that NP001 worsened the condition in all pALS with respect to the placebo branch, as starting from 72 months there were fewer survivors in the NP001 arm than in the placebo.
In addition, one could read the subset of pALS with inflammation as them having a comorbidity. If you prescribe an inflammation therapy to people having inflammation, they will improve a bit, which would be reflected in longer survival to ALS.
The proposed mechanism of action is vague (as usual, at least here there is a proposal): They said that NP001 is converted by macrophages to taurine chloramine, a regulator of inflammation. As the authors assert that persistent immune activation in patients with ALS is the cause of loss of muscle, an anti-inflammatory drug would help.
There are few publications about taurine chloramine, but, when taurine is in the presence of highly toxic hypochlorous acid, it generates the less toxic taurine chloramine. It's the result of the body's mitigation to a poison.