Project description
Drug repurposing for the treatment of neurodegenerative diseases
Neurodegenerative diseases represent one of the biggest healthcare challenges for Western societies, with cost of care reaching into EUR 1 trillion in 2018. The EU-funded NeuroPa project addresses treatment for neurodegenerative diseases using repositioning (repurposing) of previously developed drugs for new applications. The project's lead compound SIT-161 targets the brain sigma-1 receptor, which is a critical regulator of multiple processes in neurodegeneration. The drug demonstrated excellent safety in humans, but was previously abandoned after phase 2 trials for major depression. For this compound, researchers have obtained proof-of-concept data and patent protection for the treatment of neurodegenerative diseases, including Alzheimer's disease and amyotrophic lateral sclerosis. The project investigates market positioning, clinical and regulatory feasibility, and patent and investor strategies to bring this promising drug to market implementation.
Objective
An increasingly aging population suffering from neurodegenerative diseases represents one of the biggest challenges for Western societies. Alzheimer’s disease (AD) is the most prevalent neurodegenerative disease with over 50 million patients worldwide and an estimated cost of 1 trillion euros in 2018. In addition, orphan neurodegenerative diseases such as Amyotrophic Lateral Sclerosis (ALS) have a comparably urgent need for treatment, because of the severity and life changing effect of the motor symptoms (muscle spasms, speaking and swallowing problems, full paralysis and lethality). As current treatments are only symptomatic and fail to slow down the disease progression, there is a pressing need for novel effective disease-modifying treatments.
SigmaThera is a French company that tries to address the need for effective treatments for neurodegenerative diseases by focusing on the repositioning of drug candidates: applying the proven qualities of previously developed drugs for new diseases. For our lead compound SIT-161 with an excellent safety profile in humans (the candidate was abandoned after original Phase 2 trials for major depression) we have obtained impressive proof-of-concept data and patent protection for the treatment of neurodegenerative diseases, including AD & ALS. The drug targets a brain sigma-1 receptor, a critical regulator of multiple processes in neurodegeneration and provides unique efficacy and safety compared to other competing candidates in the pipeline. The high commercial potential of the drug is reflected in the €6.2B total addressable market and projected peak sales of €2B. In the NeuroPa project, SigmaThera will investigate market positioning, clinical and regulatory feasibility, future patent strategy and investor strategy to bring this promising asset close to market implementation.
Fields of science
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques.
Programme(s)
Funding Scheme
SME-1 - SME instrument phase 1Coordinator
34980 MONTFERRIER SUR LEZ
France
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.