In short
REN001, also known as mavodelpar, is an investigational chemical substance with no marketing authorisation. It is being studied as a treatment for mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS), a rare inherited disease. One completed Phase 1 trial evaluated how impaired liver function affects the drug's pharmacokinetics in 32 participants.
Key points
- REN001 (mavodelpar) is an investigational chemical substance with no marketing authorisation.
- It has orphan designation from the European Commission for the treatment of MELAS.
- One Phase 1 trial has been completed, studying the drug in people with impaired hepatic function.
- The trial enrolled 32 participants across four groups with different levels of liver function.
- The trial measured pharmacokinetics — how the drug is absorbed, distributed, and eliminated by the body.
- REN001 is given as an oral capsule.
What is REN001?
REN001, also known as mavodelpar, is a chemically synthesised substance under investigation for the treatment of mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS). Its full chemical name is sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate. REN001 is an investigational substance with no marketing authorisation in the product records. The product is supplied by Reneo Pharma Limited.
How does REN001 work?
The supplementary sources do not describe the specific mechanism of action of REN001. MELAS is caused by genetic abnormalities in the mitochondria, the energy-producing components within cells, leading to reduced energy production. REN001 has been granted orphan designation by the European Commission for the treatment of this condition.
Which conditions is REN001 studied for?
- Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS) — an inherited disease caused by genetic abnormalities in mitochondria that leads to muscle weakness, pain, headaches, seizures, and stroke-like episodes.
- Impaired hepatic function — the completed Phase 1 trial studied how REN001 behaves in people with mild, moderate, or severe liver impairment compared to healthy volunteers.
How is REN001 given?
REN001 is formulated as an oral capsule. In the Phase 1 trial, a single dose of 25 mg was administered orally.
What do the clinical trials show?
One Phase 1 trial of REN001 has been completed. The trial enrolled 32 participants across four groups: healthy subjects with normal hepatic function (10 participants), mild hepatic impairment (8 participants), moderate hepatic impairment (8 participants), and severe hepatic impairment (6 participants). The primary objective was to evaluate the effect of impaired hepatic function on the pharmacokinetics of a single 25 mg oral dose of REN001. The trial measured AUClast (total drug exposure up to the last measurable time point), AUCinf (total drug exposure extrapolated to infinity), and Cmax (maximum concentration of the drug in the blood).
What side effects are known?
The supplementary sources do not provide information on side effects of REN001.