What does the drug do?

NP001 acts on particular cells within the immune system in the bloodstream, which in turn influences inflammation within the brain and spinal cord. The study is focusing on people with MND who are also showing signs of an elevated immune response. By ‘damping down’ the immune system, the aim is to reduce the inflammation which damages motor neurones.

Trial Outcome

The trial did not meet its primary or secondary endpoints. However, more recent post hoc analysis has identified a subgroup that demonstrated a slower decline in ALSFRS-R score. You can read more here.

Clinical Trials

Phase 1

The phase 1 aimed to evaluate the safety and tolerability of NP001. The drug was found to be safe and well tolerated. Additionally, it was found to reduce levels of an inflammation biomarker. You can read more about the trial here.

Phase 2

The initial Phase 2 trial was randomised, double blind and placebo controlled. The trial recruited 136 participants in centres across USA and at one centre in Canada. The results suggested that the treatment was safe, but did not significantly slow the progression of MND. It did however suggest that participants with elevated levels of an inflammation biomarker called C-reaction protein might particularly benefit from the treatment. You find out more about the trial here.

Phase 2

The follow-up Phase 2 trial evaluated NP001 in 138 people with MND with evidence of increased inflammatory activity in the blood (C-reaction protein). The study didn't show slower progression of the disease or improved breathing function. You can read more about the trial here.


Post-hoc analysis identified a subset of 40-65 year old, who had high levels of inflammation, in which NP001 treated participants demonstrated slower declines in ALSFRS-R score by 36% compared to placebo. You can read more here.

Latest News


Dec 2022 - New analysis of the phase 2 data found evidence to suggest that NP001 may help to slow progression by reducing inflammation caused by gut bacteria leaking into the bloodstream. You can read more here

Last updated: 21/12/2022