Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as REN001
Mavodelpar · 3 trials · 3 indications
Distance walked in meters
Number of participants with Adverse Events (AEs) as a measure of safety and tolerability
| Arm | Type | Description |
|---|---|---|
| Mavodelpar | EXPERIMENTAL | Once daily |
| Matched placebo | PLACEBO_COMPARATOR | Once daily |
| REN001 | EXPERIMENTAL | Oral |
| Group 1 - Part A | EXPERIMENTAL | REN001 Low Dose oral once daily x 12 weeks |
| Group 2 - Part A | EXPERIMENTAL | REN001 High Dose oral once daily x 12 weeks |
| Group 3 - Part B | EXPERIMENTAL | REN001 High Dose oral once daily x 12 weeks |
| Name | Type | Description |
|---|---|---|
| Mavodelpar | DRUG | Once daily |
| Placebo | DRUG | Once daily |
| REN001 | DRUG | Once daily for 12 weeks |
| Low Dose REN001 | DRUG | Oral |
| High Dose REN001 | DRUG | Oral |
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 developed by OnKure Therapeutics, Inc. for the treatment of primary mitochondrial myopathy, a genetic disorder affecting mitochondrial function. It is being studied as a potential therapy for adults with this condition and has been evaluated in a completed Phase 2 clinical trial.
Mavodelpar is being developed for the treatment of primary mitochondrial myopathy in adults. This condition involves genetic mutations that impair mitochondrial energy production, leading to muscle weakness and exercise intolerance. The drug has been studied in a Phase 2 trial but is not yet approved for any indication.
Mavodelpar is being developed by OnKure Therapeutics, Inc., a biopharmaceutical company that trades under the ticker symbol OKUR. The company is responsible for the clinical development of the drug, including the completed Phase 2 trial in primary mitochondrial myopathy.
Mavodelpar is in Phase 2 development. A Phase 2 clinical trial evaluating the drug in patients with primary mitochondrial myopathy has been completed. The drug is investigational and has not been approved by the FDA or any other regulatory agency for commercial use.
Mavodelpar has been evaluated in one completed Phase 2 clinical trial, NCT04535609, titled "An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients." The trial enrolled 213 participants across the United States, Australia, Belgium, Canada, Czechia, Denmark, France, Germany, Hungary, Italy, Netherlands, New Zealand, Norway, Spain, and the United Kingdom.
Yes, Mavodelpar is also known as REN001. The Phase 2 clinical trial for primary mitochondrial myopathy was conducted under the name REN001, as reflected in the trial title. Both names refer to the same investigational small molecule developed by OnKure Therapeutics, Inc.