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PES2020-Prosj.etabl.støtte H2020

MIPdrug: Phelimin – a new drug to treat PKU, a severe disease: from lab to clinic.

Tildelt: kr 69 999

PKU is a severe, inherited disease causing protein intolerance. Untreated, children get irreversible brain damage. For >75% of PKU patients there is no treatment except a very strict diet. AAM disorders are individually rare but collectively numerous with high human and societal costs. In collaboration with Aarhus University (AU), the SME partners have spent 10 years developing a new type of medicine to treat these diseases. The technology builds on the idea of oral administration and non-systemic action i.e. removing the disease causing component before it enters into the blood. By binding the problematic amino acid – for PKU patients it is phenylalanine (Phe) – in the food in the gastro-intestinal (GI) tract, we prevent Phe from entering into the patient’s blood causing serious health issues. Phelimin will be a breakthrough product, as it is a completely new type of medicinal product. It will be “first in class” of a range of products to treat a series of AAM rare diseases where specific amino acids in the food cause disease in patients. All based on the same, proprietary technology platform. Today, only few treatments exist and for the vast majority of those diseases where the only treatment is a complicated and very strict diet with low compliance. Approach The planning phase, covered by the PES application, will clarify details of the final work packages for the EUROSTARS application. At present, our idea is that there will be a total of 8 Work Packages: 1. Consortium administration, management and reporting 2. Process optimisation and production of Phelimin for 3 days’ study in PKU mice 3. Prepare test animals and delivery method and conduct protein challenge studies 4. Conduct 5+ days’ pre-clinical Phelimin studies 5. Finalise up-scaling of production process 6. Regulatory and clinical development planning 7. Pilot clinical study in PKU patients 8. Business development

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PES2020-Prosj.etabl.støtte H2020