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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 (SAC) 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.

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.

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

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

  • Sanjay Kumar Srivastava, Asha Ek Hope Foundation for ALS/MND, Diagnosed 2018, India

    Sanjay Kumar Srivastava, Asha Ek Hope Foundation for ALS/MND, Diagnosed 2018, India

  • Irene McCaughey, Diagnosed 2011,  MND Australia

    Irene McCaughey, Diagnosed 2011, MND Australia

  • David Watson,  MND Scotland,  Diagnosed 2018

    David Watson, MND Scotland, Diagnosed 2018

  • Bayley, Australia

    Bayley, Australia

  • Karl Hughes, Diagnosed 2010 , IMNDA,  Ireland

    Karl Hughes, Diagnosed 2010 , IMNDA, Ireland

  • Horacio Fritzer, Argentina

    Horacio Fritzer, Argentina

  • Jose Rivero Muñoz, Diagnosed 2015, FYADENMAC, Mexico

    Jose Rivero Muñoz, Diagnosed 2015, FYADENMAC, Mexico

  • Willi Klein

    Willi Klein

  • Josée Kolijn-de Man, Diagnosed 2015 , ALS Patients Connected, The Netherlands

    Josée Kolijn-de Man, Diagnosed 2015 , ALS Patients Connected, The Netherlands

  • Oliver Juenke, DGM, Germany

    Oliver Juenke, DGM, Germany

  • Maria Santos Garcia Tellez, Diagnosed 2017 , FYADENMAC, Mexico

    Maria Santos Garcia Tellez, Diagnosed 2017 , FYADENMAC, Mexico

  • Alejandro Aquino, Diagnosed 2011 , Asociación ELA Argentina

    Alejandro Aquino, Diagnosed 2011 , Asociación ELA Argentina

  • Frank "Papa" Taylor, USA

    Frank “Papa” Taylor, USA

  • Hans Dieter Olszewski, Diagnosed 2010 , DGM, Germany

    Hans Dieter Olszewski, Diagnosed 2010 , DGM, Germany

  • Art Eggert, USA

    Art Eggert, USA

  • Ian Gale, MND Australia

    Ian Gale, MND Australia

  • Claudette Sturk, ALS Society of Canada

    Claudette Sturk, ALS Society of Canada
    Picture2

  • Sally Pauls, Diagnosed 2006 , Les Turner ALS Foundation

    Sally Pauls, Diagnosed 2006 , Les Turner ALS Foundation

  • Maurice Leclerc, Canada

    Maurice Leclerc, Canada

  • Joanne Pratt, Diagnosed 2011 , MND Australia

    Joanne Pratt, Diagnosed 2011 , MND Australia

  • Camilla Heiberg Freiberg, Muskelsvindfonden, Denmark

    Camilla Heiberg Freiberg, Muskelsvindfonden, Denmark

  • Emilienne Verhaegen, ALS Liga Belgium, Diagnosed 2014

    Emilienne Verhaegen, ALS Liga Belgium, Diagnosed 2014

  • Orlando Ruiz, Diagnosed 2001,  ACELA, Colombia

    Orlando Ruiz, Diagnosed 2001, ACELA, Colombia

  • Alan Liz Ogg 29042016 000799 lo res

    Alan Liz Ogg 29042016 000799 lo res

  • Ana María Zavala, FYADENMAC, Diagnosed 2019, Mexico

    Ana María Zavala, FYADENMAC, Diagnosed 2019, Mexico

  • Dorette Lüdi, Diagnosed 2014 , ALS Schweiz, Switzerland

    Dorette Lüdi, Diagnosed 2014 , ALS Schweiz, Switzerland

  • David Solomon, Diagnosed 2015, MND Association of England, Wales and N Ireland

    David Solomon, Diagnosed 2015, MND Association of England, Wales and N Ireland

  • Shay Rishoni, Diagnosed 2011 , Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 , Prize4Life, Israel

  • Oliver Juenke, Germany

    Oliver Juenke, Germany

  • Laurie Petit-Jean, Diagnosed 2012 , ARSLA, France

    Laurie Petit-Jean, Diagnosed 2012 , ARSLA, France

  • Erwin Coppejans, Diagnosed 2007 , ALS Liga België, Belgium

    Erwin Coppejans, Diagnosed 2007 , ALS Liga België, Belgium

  • Dick Dayton, USA

    Dick Dayton, USA

  • Armando González Gómez, ACELA, Colombia

    Armando González Gómez, ACELA, Colombia

  • Debbie Craghill, USA

    Debbie Craghill, USA

  • David Bishop

    David Bishop

  • Steven Gallagher, Canada

    Steven Gallagher, Canada

  • Valdomiro Xavier Honório, Brazil

    Valdomiro Xavier Honório, Brazil

  • Imelda Arenas, ACELA, Colombia

    Imelda Arenas, ACELA, Colombia

  • Timothy Holman, Switzerland

    Timothy Holman, Switzerland

  • Nicholas (Nic) Bowman, MND Association of South Africa,  Diagnosed 2016,  Australia

    Nicholas (Nic) Bowman, MND Association of South Africa, Diagnosed 2016, Australia

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

    Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • Susan Anderson, Diagnosed 2014 , Hope Loves Company,  USA

    Susan Anderson, Diagnosed 2014 , Hope Loves Company, USA

  • Peng Yi-Wen

    Peng Yi-Wen

  • Chih Ching Darren Wong, MND Malaysia

    Chih Ching Darren Wong, MND Malaysia

  • Denis Blais, Diagnosed 2015 , ALS Canada

    Denis Blais, Diagnosed 2015 , ALS Canada

  • Shay Rishoni, Diagnosed 2011 - Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 – Prize4Life, Israel

  • Andrietta

    Andrietta

  • Claudia Gotti, Brazil

    Claudia Gotti, Brazil

  • Fabio Correia

    Fabio Correia

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