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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

  • Natalya Rybakova, Russia

    Natalya Rybakova, Russia

  • Joanne Pratt, Diagnosed 2011 , MND Australia

    Joanne Pratt, Diagnosed 2011 , MND Australia

  • Kirsten Harley,  Diagnosed 2013,  Australia

    Kirsten Harley, Diagnosed 2013, Australia

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

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

  • Wendy Hendrickson, ALS Hope Foundation, USA

    Wendy Hendrickson, ALS Hope Foundation, USA

  • Danny Reviers, Diagnosed 1979 , ALS Liga België, Belgium

    Danny Reviers, Diagnosed 1979 , ALS Liga België, Belgium

  • David Bishop

    David Bishop

  • Phil Rossall, MND-Association, UK

    Phil Rossall, MND-Association, UK

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

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

  • Ailsa Malcolm-Hutton, Diagnosed 2013,  MND Association of England, Wales and N Ireland

    Ailsa Malcolm-Hutton, Diagnosed 2013, MND Association of England, Wales and N Ireland

  • Lombana, Spain

    Lombana, Spain

  • Margreth Burger-Saile, Diagnosed 2011,  ALS Schweiz,  Switzerland

    Margreth Burger-Saile, Diagnosed 2011, ALS Schweiz, Switzerland

  • Liam Dwyer, England

    Liam Dwyer, England

  • Angie Bordaen, Diagnosed 2014,  ALS Liga België, Belgium

    Angie Bordaen, Diagnosed 2014, ALS Liga België, Belgium

  • Andrietta

    Andrietta

  • Hans Dieter Olszewski, Diagnosed 2010 , DGM, Germany

    Hans Dieter Olszewski, Diagnosed 2010 , DGM, Germany

  • Christian Bär, Germany

    Christian Bär, Germany

  • Mauril Belanger

    Mauril Belanger

  • unnamed

    unnamed

  • Ada Garrido Benavidez, Diagnosed 2016,  FYADENMAC, Mexico

    Ada Garrido Benavidez, Diagnosed 2016, FYADENMAC, Mexico

  • Malcolm Buck, Australia

    Malcolm Buck, Australia

  • 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

  • Alan Liz Ogg 29042016 000799 lo res

    Alan Liz Ogg 29042016 000799 lo res

  • Fabio Carvalho, Associação Pró-Cura da ELA, Brazil

    Fabio Carvalho, Associação Pró-Cura da ELA, Brazil

  • JP

    JP

  • Armando González Gómez, ACELA, Colombia

    Armando González Gómez, ACELA, Colombia

  • Charlie “Hark” Dourney, Diagnosed 2007 , Hark ALS, USA

    Charlie “Hark” Dourney, Diagnosed 2007 , Hark ALS, USA

  • Sam Hayden-Harler, Motor Neurone Disease (MND) Association, UK

    Sam Hayden-Harler, Motor Neurone Disease (MND) Association, UK

  • Norm MacIsaac,  ALS Society of Canada,  ALS Society of Quebec,  Diagnosed 2014

    Norm MacIsaac, ALS Society of Canada, ALS Society of Quebec, Diagnosed 2014

  • Lachlan Terry,  MND Australia,  Diagnosed 2015

    Lachlan Terry, MND Australia, Diagnosed 2015

  • Jean Waters, Diagnosed 2004, MND Association of England, Wales and N Ireland

    Jean Waters, Diagnosed 2004, MND Association of England, Wales and N Ireland

  • Timmy, ALS Liga

    Timmy, ALS Liga

  • Guido De Mets, Belgium

    Guido De Mets, Belgium

  • Lin Yong Yi, Taiwan MND Association, Diagnosed 2004

    Lin Yong Yi, Taiwan MND Association, Diagnosed 2004

  • Dr Shelly Hoover

    Dr Shelly Hoover

  • Wilfried Leusing

    Wilfried Leusing

  • Amparo Muriel Engativa, Colombia

    Amparo Muriel Engativa, Colombia

  • Jason Goodman, Les Turner ALS Foundation, USA

    Jason Goodman, Les Turner ALS Foundation, USA

  • Horacio Fritzer, Argentina

    Horacio Fritzer, Argentina

  • H. Todd Kelly, Diagnosed 2013 , ALS Hope Foundation, USA

    H. Todd Kelly, Diagnosed 2013 , ALS Hope Foundation, USA

  • Bob Simonds and Drew O'Neil, USA

    Bob Simonds and Drew O’Neil, USA

  • Liong Ting Ngu, MND Malaysia, Diagnosed 2014

    Liong Ting Ngu, MND Malaysia, Diagnosed 2014

  • Zabun Nassar, MND Association, Diagnosed 2016, England

    Zabun Nassar, MND Association, Diagnosed 2016, England

  • Tison, USA

    Tison, USA

  • Shay Rishoni, Diagnosed 2011 - Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 – Prize4Life, Israel

  • Mike Rannie,  ALS Canada,  Diagnosed 2017

    Mike Rannie, ALS Canada, Diagnosed 2017

  • Camilla Heiberg Freiberg, Muskelsvindfonden, Denmark

    Camilla Heiberg Freiberg, Muskelsvindfonden, Denmark

  • Inta Grubb, Diagnosed 2014,  MND Australia

    Inta Grubb, Diagnosed 2014, MND Australia

  • Ana Lilia RodriguezApoyo Integral Gila A.C., Diagnosed 2018, Mexico

    Ana Lilia RodriguezApoyo Integral Gila A.C., Diagnosed 2018, Mexico

  • March of Faces Photo Submission_ALEX_ELA ARGENTINA

    March of Faces Photo Submission_ALEX_ELA ARGENTINA

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