MipSalus is developing a new proprietary drug – the first in a new class of medicines – for the treatment of all variants of HPA (Hyperphenylalaninemia – i.e. increased level of phenylalanine in the blood), including PKU (Phenylketonuria) which makes up the vast majority of HPA patients.
MipSalus has demonstrated that the company’s phenylalanine (Phe) binding MIPs, Phelimin, very efficiently reduces the uptake of Phe from dietary proteins in PKU mice. The effect of Phelimin is dose dependent, which means that the patient has the possibility to tailor-make his or her Phelimin treatment to fit the individual meal pattern. The patient has in other words the freedom to eat small or large meals of normal food by dosing Phelimin accordingly.
This new treatment approach is a medicinal product intended to act as a mono-therapy and due to its non-systemic way of action the product is expected to have very few or no side-effects. The product, to be taken orally together with an ordinary meal, prevents Phe in the food from entering the blood stream via the gastro-intestinal tract and hence prevents an elevated blood Phe level. The patient will thus be able to eat normal meals, which will significantly improve the quality of life and sense of freedom for the patient, but still keep the blood Phe level low.
MipSalus has successfully concluded a first scientific advice with the Danish Health and Medicines Agency, which agrees in a very limited non-clinic plan, due to Phe-MIPs’ non-systemic way-of-action, as well as blood Phe concentration as the clinical endpoint in the clinical studies to come. Furthermore, MipSalus has been granted an Orphan Drug Designation from the European authorities on Phelimin for treatment of PKU.
Based on correlations between high levels of blood Phe and neurological outcome in patients with HPA, the reduction in blood Phe compared to placebo is accepted as the only clinical endpoint for the pivotal trials within the clinical development program.
The use of a human predictive mouse model (PKU mouse (BTBR-pahenu2/J)), that has an enzyme defect and pathogenic response pattern identical to human PKU-patients, makes MipSalus able to mitigate significant risk elements with simple animal studies at a very early stage in the development process.