Three drug candidates from an amino acid specific molecular imprinted polymer platform for the treatment of rare metabolic diseases

The grant consortium will further development of their innovative molecular imprinted polymer (MIP) platform by finalising manufacturing of the phenylketonuria (PKU) MIP for use in clinical studies, as well as developing and testing MIPs in other metabolic disease animal models. Together, the consortium will utilise their individual expertise in MIP development and manufacturing, clinical development services (including regulatory), animal modelling, clinical treatment and recruitment into clinical studies to successfully move the proposed MIP platform closer to commercialisation. The use of MIPs for the treatment of patients with rare metabolic diseases has not been implemented previously anywhere in the world. Mipsalus has exhibited proof of concept of their innovation in an animal PKU model. PKU is a severe, inherited disease causing protein intolerance. Untreated, children get irreversible brain damage. For >70% of PKU patients there is no treatment except a very strict diet. Low compliance is common and generate many health issues. As the project is based on a platform generally enabling sequestering of specific amino acids from food intake, similar drug candidates to treat other rare amino acid metabolism disorders will be developed, such as a tyrosine-binding MIP intended for alkaptonuria (AKU) and hypertyrosinemia type 1 (HT1); and a leucine-binding MIP intended for treatment of maple syrup urinary disease (MSUD).