New proprietary drug

MipSalus is developing a new proprietary drug – the first in a new class of medicines – for the treatment of all inborn genetic disorders that results in elevated concentrations of the amino acid phenylalanine in the blood. The collective term for these disorders is Hyperphenylalaninemia (known as HPA) – i.e. increased level of phenylalanine (Phe) in the blood. The most common variant is Phenylketonuria, or simply PKU.

People with PKU and other types of HPA cannot metabolize Phe which is found in all kinds of dietary protein, and with the treatment currently available they have to adhere to lifelong, low-protein diet. The treatment developed by MipSalus will allow patients to eat normally – a substantial increase in their quality of life.

MipSalus’ new drug is based on a well-established technology: Molecular Imprinted Polymers or MIPs. MipSalus has demonstrated that the company’s Phe binding MIPs, called Phelimin, very efficiently reduces the uptake of surplus Phe from dietary proteins in mice suffering from PKU. The effect of Phelimin is dose dependent, and this allows patients to tailor-make their treatment to fit individual meal patterns. In other words, by dosing Phelimin accordingly, HPA patients are free to consume smaller and larger meals made of normal food as they please.

Phelimin is intended to act as a mono-therapy and will be taken orally together with a meal. As Phelimin binds Phe, the amino acid cannot enter the blood stream via the gastrointestinal tract. Consequently, the build-up of toxic levels of Phe in the blood is prevented. Studies have documented that Phelimin is both inert and non-systemic, i.e. the drug will not be altered nor react with other substances and it works solely within the stomach and intestines. Due to its non-systemic and inert nature, Phelimin is expected to have very few side-effects.

To investigate the effect of Phelimin, MipSalus has used a human predictive mouse model. The mice – of the type BTBR Pah^enu2/j – have PKU and their enzyme defect and pathogenic response pattern are identical to human classical PKU patients. This is highly beneficial as it enables MipSalus to mitigate significant risks at a very early stage in the development process by conducting animal studies.

MipSalus has success­fully concluded its first scientific advice meeting with the Danish Health and Medicines Agency (today the Danish Medicines Agency). Due to the Phe binding MIPs particles’ non-systemic way of action, the Agency agrees to a very limited non-clinic plan and accepts blood Phe concentration as the clinical endpoint in the clinical studies to come.

Furthermore, both the European Medicines Agency (EMA) and the U.S. Food & Drug Administration (FDA) has granted Phelimin an Orphan Drug Designation.