Pharmasum plans a Proof-of-Concept (PoC) clinical study in persons with Down syndrome and dementia where the genetic link to our drug target DYRK1A is strong and the clinical chance of success is high due to a less heterogenous population compared to Alzheimer´s disease in general.
Down syndrome individuals are especially prone to developing dementia and represent the largest group of dementia patients below 50 years of age (Hanney et al 2012; Lancet). Down syndrome (DS; Trisomy 21) is the most common genetic disorder causing cognitive problems, affecting as many as 1 out of 800 live born children, with almost 6 million individuals worldwide.
Common to all who have DS is that they have an extra copy of chromosome 21. This chromosome codes for the protein kinase enzyme DYRK1A, which is considered the key brain factor that leads to the primary developmental cognitive impairment (problems with cognition, learning and memory). Due to trisomy 21, those with DS have an overdose of DYRK1A, which is the cause of DS and the development of dementia.
Pharmasum Therapeutics has developed an inhibitor to the DYRK1A target, PST-674, that represents a unique innovation in a global market, serving an extremely high unmet medical need. The value creation of the technology for society and patients is potentially vast.
Because there is a close link between DYRK1A and dementia in Down syndrome, this is an ideal group to demonstrate the effect of a new medicine for the treatment of dementia.