Problem: Neurodegenerative diseases remain with no cure or effective treatment.
Neurodegenerative diseases are characterized by the progressive loss of neurons, the building blocks of the nervous system, leading to a decline in cognitive function and/or motor skills.
Neurodegenerative diseases refers to a group of disorders such as amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD), Huntington's disease (HD), and frontotemporal dementia (FTD).
Neurodegenerative diseases remains with no cure or effective treatment, creating great urgency in developing new disease modifying treatments.
Neurodegenerative diseases refers to a group of disorders such as amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD), Huntington's disease (HD), and frontotemporal dementia (FTD).
Neurodegenerative diseases remains with no cure or effective treatment, creating great urgency in developing new disease modifying treatments.
Solution: Alleviating TDP-43 pathology to treat neurons, extending patients' lives and improving their quality of life.
A shared feature of neurodegenerative diseases is TDP-43 pathology. TDP-43 pathology refers to the mislocalization, cytoplasmic accumulation and aggregation of TDP-43 protein (in green in figure), which is associated with causing neuronal toxicity and neurodegeneration.
Using Neuropeutics' proprietary cellular and animal models, we identified and validated a pipeline comprising of three distinct strategies with different mechanisms of action to prevent and reverse TDP-43 aggregation, alleviating TDP-43 pathology and treating neurons.
Our primary disease indication is that of amyotrophic lateral sclerosis (ALS) given the 97% occurrence of TDP-43 pathology, followed by frontotemporal dementia (FTD), Alzheimer's disease (AD), Parkinson's disease (PD) and Huntington's disease (HD).
Using Neuropeutics' proprietary cellular and animal models, we identified and validated a pipeline comprising of three distinct strategies with different mechanisms of action to prevent and reverse TDP-43 aggregation, alleviating TDP-43 pathology and treating neurons.
Our primary disease indication is that of amyotrophic lateral sclerosis (ALS) given the 97% occurrence of TDP-43 pathology, followed by frontotemporal dementia (FTD), Alzheimer's disease (AD), Parkinson's disease (PD) and Huntington's disease (HD).
JRMS-22: Small Molecule To Revolutionize Amyotrophic Lateral Sclerosis Treatment
Neuropeutics' leading small molecule 'JRMS-22' prevents and reverses toxic protein aggregation with strong efficacy in human cells, mouse primary neurons, and mouse models.
JRMS-22 harnesses a non-canonical function of an undisclosed proprietary protein to prevent and reverse TDP-43 aggregation. JRMS-22 is highly efficacious, reducing TDP-43 aggregation by about 30% after only 2 weeks of treatment in mice.
Given that TDP-43 aggregation occurs in over 97% of all amyotrophic lateral sclerosis (ALS) cases, JRMS-22 is a patient inclusive therapeutic.
Neuropeutics' JRMS-22 is a disease modifying treatment targeting the pathology at its root, with the potential to extend patients' lives and improve their quality of life.
JRMS-22 harnesses a non-canonical function of an undisclosed proprietary protein to prevent and reverse TDP-43 aggregation. JRMS-22 is highly efficacious, reducing TDP-43 aggregation by about 30% after only 2 weeks of treatment in mice.
Given that TDP-43 aggregation occurs in over 97% of all amyotrophic lateral sclerosis (ALS) cases, JRMS-22 is a patient inclusive therapeutic.
Neuropeutics' JRMS-22 is a disease modifying treatment targeting the pathology at its root, with the potential to extend patients' lives and improve their quality of life.
There are three currently FDA approved drugs for ALS (i.e., riluzole, edaravone and PB-TURSO), all of which target secondary pathomechanisms and have deemed to be have limited benefits, emphasizing the need for new treatments.
Therapy |
Target |
Mechanism |
Target Primary Pathology |
Effect on TDP-43 Aggregates? |
Effect on Lifespan |
JRMS-22 |
(Proprietary) |
Endogenous |
✓ |
✓ |
(Years) |
Riluzole |
Glutamate Excitotoxicity |
Exogenous |
X |
X |
6-19 months * |
Edaravone |
Oxidative Stress |
Exogenous |
X |
X |
No effect ** |
PB-TURSO |
ER Stress & Mitochondria |
Endogenous |
X |
X |
No effect *** |
* Andrews, et al. (2020). Real-world evidence of riluzole effectiveness in treating amyotrophic lateral sclerosis. Amyotrophic lateral sclerosis & frontotemporal degeneration, 21(7-8), 509–518.
** Ferrer reports top-line results from Phase III ADORE study in ALS - www.ferrer.com/en/results-study-ADORE-ALS
*** PHOENIX Trial of AMX0035 did not meet endpoints https://www.amylyx.com/news/amylyx-pharmaceuticals-announces-topline-results-from-global-phase-3-phoenix-trial-of-amx0035-in-als#:~:text=PHOENIX%20did%20not%20meet%20the,met%20the%20CENTAUR%20trial%20criteria.
** Ferrer reports top-line results from Phase III ADORE study in ALS - www.ferrer.com/en/results-study-ADORE-ALS
*** PHOENIX Trial of AMX0035 did not meet endpoints https://www.amylyx.com/news/amylyx-pharmaceuticals-announces-topline-results-from-global-phase-3-phoenix-trial-of-amx0035-in-als#:~:text=PHOENIX%20did%20not%20meet%20the,met%20the%20CENTAUR%20trial%20criteria.
