This pharmaceutical company develops targeted therapies for neurodegenerative diseases. Their treatments aim to slow disease progression, extend patient survival, and improve quality of life.
Funding
Funding not disclosed
Founders
Product
Problem
Neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD), Huntington's disease (HD), and frontotemporal dementia (FTD), are characterized by the progressive loss of neurons, leading to a decline in cognitive and/or motor function. Currently, there are no cures or effective treatments available to halt or reverse the progression of these diseases.
Solution
Neuropeutics is developing targeted therapies designed to address TDP-43 pathology, a shared feature among various neurodegenerative diseases, to extend patient survival and improve their quality of life. Their approach focuses on preventing and reversing the mislocalization, accumulation, and aggregation of the TDP-43 protein, which is associated with neuronal toxicity and neurodegeneration. The company utilizes proprietary cellular and animal models to identify and validate therapeutic strategies. Their lead small molecule, JRMS-22, aims to prevent and reverse toxic protein aggregation with strong efficacy.
Target Audience
The primary target audience includes patients suffering from neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), Alzheimer's disease (AD), Parkinson's disease (PD) and Huntington's disease (HD).
Features
- JRMS-22: A small molecule designed to prevent and reverse TDP-43 aggregation.
- Targets a non-canonical function of a proprietary protein to prevent and reverse TDP-43 aggregation.
- Demonstrated efficacy in human cells, mouse primary neurons, and mouse models, reducing TDP-43 aggregation by approximately 30% after two weeks of treatment in mice.
- Aims to be a disease-modifying treatment by targeting the root pathology of TDP-43 aggregation.