Immunotherapy platform for ALS slows disease progression, extends survival

A new analysis of an overall survival study of an investigational immunotherapy platform to treat ALS found it slowed disease progression and extended survival, according to the manufacturer.

In a press release, Neuvivo stated it previously conducted a blinded, placebo-controlled, retrospective clinical trial to examine the survival benefits of NP001 in 268 individuals with ALS, analyzing up to 11 years of patient data following treatment. In that study, NP001 was associated with an extended survival of 4.8 months. In a subset of patients aged 65 years and younger, the extended survival interval was 10.8 months.

The most recent study followed the original participants initially given NP001 for 6 months during its phase 2a and 2b trials whose disease did not progress. 

The new analysis showed that 31% of patients treated with 2 mg/kg NP001 demonstrated no disease progression over the 6-month studies compared with 14% of those given placebo. NP001 administration also resulted in decreased presence of neurofilament light chain vs. placebo.

Per the release, NP001 significantly “slowed or halted the loss of vital lung capacity.”

The median overall survival was 58 months in the NP001 treatment group vs. 36 months in the control group, “meaning their life was extended by 22 months after a 6-month clinical trial,” the release said.

“One of the most confounding aspects of ALS research is that this disease does not uniformly progress,” Michael McGrath, MD, PhD, founder and chief scientific officer at Neuvivo, said in the release. “When I compared patients treated with NP001 vs. a control group of patients whose disease was not progressing over 6 months, the results on respiratory function and on overall survival were striking.”