Neuraltus Signals It Is Moving Toward Analysis Stage of Phase 2 Trial of ALS Treatment

Neuraltus Signals It Is Moving Toward Analysis Stage of Phase 2 Trial of ALS Treatment

Neuraltus Pharmaceuticals signaled that it was moving toward the analysis stage of a Phase 2 clinical trial of its ALS therapy NP001.

The signal was its announcement that it had completed the last patient visit in the study.

Neuraltus conducted the trial (NCT02794857) to confirm the results of a previous Phase 2 trial (NCT01281631) showing that NP001 slowed the progression of ALS in patients with chronic inflammation.

The first study, completed in 2012, assessed the effectiveness of two intravenous doses of NP001 in 136 patients, compared with a placebo. The yardstick researchers used was the ALS Functional Rating Score Revised, which evaluates patients’ function and ability to carry out daily activities.

NP001 decreased levels of patients’ inflammatory markers, and was safe, researchers said. Importantly, the data suggested that patients with the highest levels of C-reactive protein, a biomarker for chronic  inflammation, responded best to the drug.

Researchers published their study, “Randomized phase 2 trial of NP001, a novel immune regulator,” in the journal Neurology: Neuroimmunology & Neuroinflammation.

“The outcome of the confirmatory Phase 2 study of NP001 for the treatment of ALS is highly anticipated by researchers, patients and caregivers, as it has the potential to reaffirm the preliminary evidence from the company’s first Phase 2 clinical trial of this investigational compound,” Dr. Robert Miller, the study’s principal investigator, said in a press release. He is clinical professor of Neurology & Neurological Sciences at Stanford Medicine and director of the Forbes Norris MDA/ALS Research & Treatment Center at California Pacific Medical Center.

Although the cause of ALS remains unknown, growing evidence indicates that inflammation drives the disease’s progression. This includes findings that high levels of inflammation-generating, activated macrophages — a type of white blood cell — lead to the nerve cell damage in the disease. NP001 works by switching off macrophages’ activated state, preventing the immune cells from entering the brain, Neuraltus said.

The confirmation trial involved 138 ALS patients with chronic inflammation. It was held at a number of  locations across North America. Patients received either intravenous NP001 or a placebo for six months. Doses were given over five consecutive days in the first month, and three consecutive days in each of the  following months.

The trial’s main objective was to see whether NP001 improved patients’ ALS Functional Rating Scale-Revised score. Secondary objectives included seeing if it could show the progression of patients’ lung function and lower markers of inflammation in their blood.

Patient enrollment was completed in July 2017. Neuraltus said last year that it expected to announce key results of the study at the end of the first quarter of 2018.

“If the study results confirm the previous study’s findings, NP001 could be one step closer to regulatory review,” said Rich Casey, the company’s CEO.

6 comments

  1. Charlie says:

    We may find a cure,or rather, researchers may find a cure,soon, but this would only be the result of shockingly fantastically good luck. I hope someone has this good luck in the labs.

    We all know that:-
    i) the cause of ALS is not yet known, and
    ii) research is 100% aimed at finding a treatment (not a cure).

    These two features are inextricably linked.

    If they find the ALS cause, the move towards creating a cure would be relatively quick. As no-one knows what causes ALS, research today focuses on trying to find a treatment that slows down progressing decline.

    If declines can be slowed, or even stopped, then the extended longevity would potentially allow some pALS to survive until the cause is ultimately found.

    So all research results are helpful, even the failures, like Tirasemtiv, because they tell us what will not work, and so an unproductive avenue of research can be ditched.In a way we need more and more and more failures (but quickly, please Science) so that the field of possibilities can be narrowed down dramatically.
    The field needs to be strongly culled for all the right reasons, because current research is scattered over innumerable possibilities.

    • Paul says:

      Finding all the causes would be ideal (there are probably many causes) but I think there needs to be more research into treatment that can stop progression. We really need to save people now. This needs to be a treatable condition.

      This drug has been in trial for way too long.

  2. Charlie says:

    “If the study results confirm the previous study’s findings, NP001 could be one step closer to regulatory review,” said Rich Casey, the company’s CEO.”
    The step that Mr. Casey refers to is a Phase 3 trial. If this current Phase 2 is promising (If If If) then 2018 would be the year in which that trial could be designed, with perhaps a start in 2019. Phase 3 means more patients, more data to be analysed, and more care and attention, so we might see published results, good, bad or ugly in 2021.

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