Targeting the brain sigma-1 receptor: a paradigm shift for the treatment of neuro-degenerative disorders
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.