In short
Mirivadelgat is an investigational small-molecule medicine being studied in Parkinson's disease. It is a chemical substance taken by mouth as a capsule. A Phase 2 trial with 45 participants is assessing its safety and whether it reaches the brain, where it may reduce harmful oxidative stress.
Key points
- Mirivadelgat is an investigational small molecule taken orally as a capsule.
- It is being studied in a Phase 2 trial for Parkinson's disease.
- The trial includes 45 participants and is currently authorised.
- The study measures safety, brain exposure, and a marker of oxidative stress in spinal fluid.
- Mirivadelgat has no marketing authorisation and is not yet an approved medicine.
What is mirivadelgat?
Mirivadelgat is an investigational small-molecule medicine that has not received marketing authorisation. It is a chemically synthesised substance developed by Foresee Pharmaceuticals and is also known by the development codes FP-045 and FRS 045. Mirivadelgat is classified as a new molecular entity, meaning its active structure has not previously been approved in a medicine. In the product records for the current trial, the authorisation holder is listed as Helse Bergen HF. The substance belongs to the therapeutic area of nervous system diseases.
How does mirivadelgat work?
Mirivadelgat is described as a stimulant of the enzyme aldehyde dehydrogenase 2. This enzyme is involved in breaking down certain reactive aldehydes that can damage cells. In Parkinson's disease, oxidative stress leads to the formation of harmful compounds such as 4-hydroxy-2-nonenal (4-HNE). By activating aldehyde dehydrogenase 2, mirivadelgat is expected to help clear these damaging aldehydes and reduce oxidative stress in the brain. The trial measures 4-HNE protein adducts in cerebrospinal fluid (CSF), the fluid surrounding the brain and spinal cord, to see whether the medicine has this effect.
Which conditions is it studied for?
- Parkinson's disease — a progressive condition of the nervous system that affects movement, causing tremor, stiffness, and slowness. The current Phase 2 trial is studying mirivadelgat in people with this condition.
According to a drug development profile, mirivadelgat has also been studied in earlier phases for other conditions, including pulmonary hypertension and Fanconi anaemia.
How is mirivadelgat given?
Mirivadelgat is taken by mouth as a capsule. In the current trial, participants receive a dose of 300 mg once daily for 12 weeks. The trial also includes a placebo arm, in which participants receive a capsule that looks the same but contains no active medicine.
What do the clinical trials show?
One trial of mirivadelgat is listed in the EU Clinical Trials Information System. It is a Phase 2, interventional study in Parkinson's disease with a planned enrolment of 45 participants. The trial is currently authorised, meaning it has received regulatory approval to begin.
The study has several objectives. It aims to assess the safety and tolerability of mirivadelgat by recording adverse events and serious adverse events, as well as how well participants stick to the dosing schedule. It also measures how much of the active metabolite AD-835 reaches the brain by comparing its concentration in cerebrospinal fluid with its concentration in blood after 12 weeks of treatment. Finally, the trial measures the change in CSF levels of 4-HNE protein adducts, a marker of oxidative stress, from the start of the study to week 12.
What side effects are known?
The current trial is designed to collect information on side effects, but no results on adverse events are available yet. The drug development profile lists an adverse events section, but specific side effects of mirivadelgat have not been reported in the provided sources.