Collaboration with Liverpool University and The Royal Liverpool University Hospital
September 2017: Together with Liverpool University and The Royal Liverpool University Hospital, Mipsalus has conducted a series of mice studies which indicate that Phelimin can very likely be used to treat children and adolescents suffering from Alkaptunoria (AKU) – a rare inborn error of amino acid metabolism similar to PKU. The studies also suggest that Phelimin could potentially be used to lessen serious side effects resulting from the current treatment of adult AKU patients. Furthermore, results from the studies also suggest that Phelimin can be used to treat yet another inborn error of amino acid metabolism called Tyrosinemia type1 (HT-1).
Promising Results from Preliminary Safety Study
July 2017: MipSalus has conducted a preliminary 2-week safety study. By oral administration, four groups of five BALB/c mice each were given MIPs particles three times a day for both one and two weeks. No difference was observed between the treated and the non-treated control groups at all parameters tested:
The results confirm the assumption that both the polymer of which Phelimin is made and the particles themselves behave inert in the mouse gastrointestinal tract when dosed at a level suitable to prevent accumulation of phenylalanine from a normal diet in a PKU mouse.
Multi-dose studies with Phelimin show high efficacy and flexibility
April 2017: Mipsalus has concluded a series of multi-dose studies documenting Phelimin to be very effective in regulating phenylalanine uptake from dietary proteins. Over the course of two days, mice with PKU were treated with three administrations of Phelimin and dietary proteins per day.
Three different dosing levels of Phelimin were tested, and the lowest Phelimin dose was able to reduce and stabilize the blood phenylalanine at a normal, low level. The control group, which received dietary proteins but no Phelimin, had a significant accumulation of phenylalanine in their blood over the course of the two days the studies lasted.
The studies thus document a strong Phelimin dose-response effect, meaning that the phenylalanine uptake can be regulated at the individual meal-level. Hence, patients can regulate their blood phenylalanine very efficiently by dosing Phelimin according to each meal, which then gives them the freedom to eat many smaller meals or a few large meals of normal food.
Correction of information published on GeneFo’s website
April 2017: On April 14, GeneFo announced that MipSalus’ upcoming medicinal product Phelimin for treatment of PKU had been approved by EU authorities and awaited approval in US. This is not correct and GeneFo has removed the information from their website. It is, however, true that preclinical studies with Phelimin has been very promising and we expect to initiate clinical trials within 24 months.
Following GeneFo’s publication, MipSalus received numerous inquiries from patients and PKU associations who were all of the impression that a Phelimin treatment was now available. We highly appreciate the PKU patients’ interest in Phelimin and regard the interest as an additional confirmation of the de facto unmet medical need existing for PKU patients. It encourages us to work even harder to transform the very promising preclinical data into clinical trials.
MipSalus to collaborate with at Liverpool University
February 2017: CEO Nicolas Krogh and CSO Klaus Gregorius visited Liverpool University to meet with a research group dedicated to the study of alkaptonuria (AKU), a rare inborn error of amino acid metabolism similar to PKU. The research group has been selected for a EU grant of GBP 8 mio.
MipSalus presented the result of its studies with Phelimin and PKU, and the research group concluded that Phelimin could likely be used to treat AKU patients. To test this hypothesis, MipSalus and Liverpool University made an agreement to conduct an animal study.
MipSalus documents proof of concept for its Home Sensor
February 2017: As the results of a feasibility study, MipSalus has now gotten in-vitro proof of concept for the home sensor which will allow PKU patients to monitor the concentration of blood phenylalanine. To further develop the home sensor, additional investments are needed and we will work identifying relevant investors.