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International Alliance of ALS/MND Associations

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Neuvivo – NP001

Background

NP001 is a small molecule first developed by Neuraltus Pharmaceuticals and currently owned by Neuvivo. The proposed mechanism of action of NP001 is to modulate macrophages within the innate immune system, aiming to restore balance and reduce uncontrolled inflammation. By targeting inflammation, this approach seeks to slow disease progression and preserve muscle function and is currently being evaluated as a potential treatment for ALS/MND. To date, NP001 underwent one Phase I and two Phase 2 trials for safety and tolerability evaluation, all studies were run in North America.

Trial Design & Results

In 2010 a randomized, double-blind placebo-controlled Phase 1 clinical trial was run by Neuraltus Pharmaceuticals to evaluate safety and tolerability of single ascending doses of NP001 (ClinicalTrials.gov ID: NCT01091142). The study enrolled 32 people living with ALS/MND in three sites within the US. The study showed that all doses of NP001, administered by 30 min infusions, were safe and generally well tolerated (Miller et al., 2014).

Based on these results, Neuraltus Pharmaceuticals initiated two Phase 2 trials. The first Phase 2 trial (Phase 2A, ClinicalTrials.gov ID: NCT01281631) ran from 2011 to 2012 and it was a multicenter, randomized, double blind placebo-controlled trial that enrolled 136 people living with ALS/MND. Participants received 2mg/kg, 1mg/kg or placebo for 6 months, through intravenous infusions. The study showed that the treatment was safe and well tolerated at all doses, with some pain at the injection site and dizziness being the most common side effects. No significant overall slowing of disease was observed in any of the groups. A post hoc analysis showed that a subset of participants with evidence of elevated systemic inflammation treated with the higher dose of NP001 showed some potential benefit, however, the trial was underpowered to make any claim regarding NP001 efficacy (Miller et al., 2015).

Building on this data, a second Phase 2 trial (Phase 2B, ClinicalTrials.gov ID: NCT02794857) was run from 2016 to 2017 and it was a randomized, double-blind, placebo-controlled study of NP001 in people living with ALS/MND who also showed evidence of elevated systemic inflammation. A total of 138 participants within 3 years of symptom onset were enrolled and randomized at a 1:1 ratio NP001 (2mg/kg) to placebo. The clinical trial found that the drug was safe and well tolerated, however, it found no difference in the primary (ALSFRS-R) or secondary (vital capacity) endpoints over six months (McGrath et al., 2023). Following these results, Neuraltus Pharmaceuticals went out of business (Pharmaceutical Technology website & ACS publications).

In 2019, Neuvivo obtained the chemical manufacturing, toxicology, and clinical records related to NP001 from Neuraltus Pharmaceuticals (ACS publications & McGrath et al., 2023). In 2023, Neuvivo carried on a post hoc analysis on the phase 2B trial that showed some effect on respiratory vital capacity in a small subgroup of people who participated in the trial, however, the study was largely underpowered (McGrath et al., 2023). Furthermore, an analysis on long-term survival was conducted for participants of both phase 2 trials. The study highlighted that people who took part in both phase 2 clinical trials, showed some benefit if under the age of 65 and treated with 2mg/kg of NP001 (Forrest et al., 2024).

In October 2024, on the basis of these post hoc analysis, Neuvivo submitted a new drug application (NDA) to the U.S. Food and Drugs Administration (FDA) seeking approval for NP001 for the treatment of ALS/MND.

Summary

NP001 is a molecule that aims to modulate systemic inflammation in people living with ALS/MND. Historically, this has been a very controversial pathway to link to neurodegeneration and a hard one to target. Results from the presented trials show weak indications that NP001 could have marginal benefits in younger people living with ALS/MND with a heightened inflammatory profile. However, it is the opinion of the Scientific Advisory Council that the data currently publicly available is not sufficient to determine whether NP001 has any effect on disease progression.

The Scientific Advisory Council encourages Neuvivo to be mindful in stating NP001’s effectiveness in slowing disease progression until clear evidence demonstrates its impact on people living with ALS/MND.

Sources

Miller et al., 2014 – https://www.tandfonline.com/doi/10.3109/21678421.2014.951940?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmed

Miller et al., 2015 – https://www.neurology.org/doi/10.1212/NXI.0000000000000100 

Miller et al., 2022 – https://pubmed.ncbi.nlm.nih.gov/35098554/ 

McGrath et al., 2023 – https://www.mdpi.com/2227-9059/12/10/2362 

Pharmaceutical Technology website – https://www.pharmaceutical-technology.com/news/neuvivo-makes-bid-to-enter-als-arena-with-immunotherapy-candidate/?utm_source=chatgpt.com&cf-view 

ACS publications – https://pubs.acs.org/doi/10.1021/cen-10024-cover4 

Forrest et al., 2024 – https://www.mdpi.com/2227-9059/12/10/2367

International Alliance of ALS/MND Associations
February 2025


The original language of communication is English and any translation cannot be guaranteed for accuracy of messaging.

Primary Sidebar

Drugs in Development

  • AB Science – Masitinib
  • BrainStorm Cell Therapeutics – NurOwn
  • Clene Nanomedicine – CNM-Au8
  • Collaborative Medicinal Development – CuATSM
  • ILB – Tikomed
  • Kadimastem – AstroRx
  • Methylcobalamin
  • Mitsubishi Tanabe Pharma America – Oral Edaravone
  • Neuronata-R/Lenzumestrocel
  • NeuroSense – PrimeC
  • Neuvivo – NP001
  • T Regulatory Cell Therapies
  • Prilenia Therapeutics – Pridopidine
  • SOD1 Therapies & Trials

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    Jo Knowlton and her dog, Scotland

  • Luis Antonio Pimenta Lima, Brazil

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  • Horacio Fritzer, Argentina

    Horacio Fritzer, Argentina

  • Claudette Sturk, ALS Society of Canada

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  • Teddy Hanono Annie, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

    Teddy Hanono Annie, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

  • Hanne Stenmose, Muskelsvindfonden, Denmark

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  • Mahmood Anwar, UK

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  • Hollister

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  • Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

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  • Timmy, ALS Liga

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  • Jette Odgaard Villemoes, Muskelsvindfonden, Denmark

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  • Eric Von Schaumburg, USA

    Eric Von Schaumburg, USA

  • Marco Antonio Alvarez Mercado, Mexico

    Marco Antonio Alvarez Mercado, Mexico

  • Brian Parsons

    Brian Parsons

  • Willi Klein

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  • Mary Thomas, Diagnosed 2013 , MND Australia

    Mary Thomas, Diagnosed 2013 , MND Australia

  • Ian Gale, MND Australia

    Ian Gale, MND Australia

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    IMG_2658

  • Colm Francis Davis, Ireland

    Colm Francis Davis, Ireland

  • Natalya Rybakova, Russian Charity ALS Foundation

    Natalya Rybakova, Russian Charity ALS Foundation

  • Orlando Ruiz, Diagnosed 2001,  ACELA, Colombia

    Orlando Ruiz, Diagnosed 2001, ACELA, Colombia

  • Emilienne Verhaegen, ALS Liga Belgium, Diagnosed 2014

    Emilienne Verhaegen, ALS Liga Belgium, Diagnosed 2014

  • Enzo Maccarrone, AISLA ONLUS, Italy

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  • Irene McCaughey, Diagnosed 2011,  MND Australia

    Irene McCaughey, Diagnosed 2011, MND Australia

  • Ian and Teresa Roberts

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  • Richard Clark, MND New Zealand,  Diagnosed 2011

    Richard Clark, MND New Zealand, Diagnosed 2011

  • Alan Liz Ogg 29042016 000799 lo res

    Alan Liz Ogg 29042016 000799 lo res

  • Susan Keldani, Les Turner ALS Foundation, USA

    Susan Keldani, Les Turner ALS Foundation, USA

  • John Dinon, MND Australia

    John Dinon, MND Australia

  • Daniel Hare

    Daniel Hare

  • Feng Gin Sun, Diagnosed 2014 , Shaanxi ALS Association, China

    Feng Gin Sun, Diagnosed 2014 , Shaanxi ALS Association, China

  • Michel Perrozzo, ARSLA, Diagnosed 2015, France

    Michel Perrozzo, ARSLA, Diagnosed 2015, France

  • Yolanda Armendariz, Diagnosed 2017 , FYADENMAC, Mexico

    Yolanda Armendariz, Diagnosed 2017 , FYADENMAC, Mexico

  • Anthony (Tony) Lynch, MND New South Wales, Diagnosed 2016, Australia

    Anthony (Tony) Lynch, MND New South Wales, Diagnosed 2016, Australia

  • Animesh Kumar, Diagnosed 2013 , Asha Ek Hope Foundation, India

    Animesh Kumar, Diagnosed 2013 , Asha Ek Hope Foundation, India

  • Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

    Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

  • Glen Elison,  ALS Hope Foundation,  Diagnosed 2019,  USA

    Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

  • Graham Johnson, MND Australia

    Graham Johnson, MND Australia

  • Dick Dayton, USA

    Dick Dayton, USA

  • Mark Miller

    Mark Miller

  • Cath Muir

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  • Camilla Heiberg Freiberg, Muskelsvindfonden, Denmark

    Camilla Heiberg Freiberg, Muskelsvindfonden, Denmark

  • Verónica Isabel Castro Molina, Diagnosed 2014, Argentina

    Verónica Isabel Castro Molina, Diagnosed 2014, Argentina

  • Fabrice Kamp, Germany

    Fabrice Kamp, Germany

  • Anita Forte, Les Turner ALS Foundation, USA

    Anita Forte, Les Turner ALS Foundation, USA

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    March of Faces Photo Submission_OLGA_ELA ARGENTINA

  • Stephanie Christiansen Hall, Canada

    Stephanie Christiansen Hall, Canada

  • Inta Grubb, Diagnosed 2014,  MND Australia

    Inta Grubb, Diagnosed 2014, MND Australia

  • Aida Trzmiel de Guterman, Asociacion ELA Argentina, Diagnosed 2007, Argentina

    Aida Trzmiel de Guterman, Asociacion ELA Argentina, Diagnosed 2007, Argentina

  • Imelda Arenas, ACELA, Colombia

    Imelda Arenas, ACELA, Colombia

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