Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Mavodelpar · 1 trial · 1 indication
Distance walked in meters
| Arm | Type | Description |
|---|---|---|
| Mavodelpar | EXPERIMENTAL | Once daily |
| Matched placebo | PLACEBO_COMPARATOR | Once daily |
| Name | Type | Description |
|---|---|---|
| Mavodelpar | DRUG | Once daily |
| Placebo | DRUG | Once daily |
Inclusion Criteria: 1. Subjects age 18 years or older with PMM as defined by the International Workshop: Outcome measures and clinical trial readiness in primary mitochondrial myopathies in children and adult (Mancuso et al 2017). 2. A confirmed PMM diagnosis due to known pathogenic gene mutation o...
Mavodelpar is an investigational small molecule being developed for Primary Mitochondrial Myopathy, a condition that affects muscle function due to mitochondrial dysfunction. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities.
Mavodelpar is being developed by OnKure Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol OKUR. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Primary Mitochondrial Myopathy.
Mavodelpar is in Phase 2 clinical development. A Phase 2 trial has been completed, and the drug remains investigational, meaning it has not yet received regulatory approval for commercial use.
Mavodelpar has one completed Phase 2 clinical trial, identified as NCT04535609. This randomized, double-blind, placebo-controlled study evaluated 24 weeks of treatment with Mavodelpar in 213 patients with Primary Mitochondrial Myopathy across multiple countries.
Yes, Mavodelpar is also known as REN001. The completed Phase 2 clinical trial NCT04535609 refers to the drug as REN001, confirming that both names refer to the same investigational compound.