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Also known as Low Dose REN001, Mavodelpar
REN001 · 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...
REN001 is an investigational small molecule developed by OnKure Therapeutics, Inc. It is being studied for Fatty Acid Oxidation Disorders and McArdle Disease, two rare inherited conditions that affect how muscles produce energy. REN001 has completed Phase 1 clinical testing and is not approved for any indication.
REN001 targets PPAR, a class of nuclear receptors that regulate gene expression involved in energy metabolism. By acting on PPAR, REN001 is designed to influence how cells process fatty acids and generate energy, which is relevant to the metabolic defects seen in Fatty Acid Oxidation Disorders and McArdle Disease.
REN001 is being developed by OnKure Therapeutics, Inc., which trades under the ticker OKUR. The company is the sponsor of the clinical program evaluating REN001 in Fatty Acid Oxidation Disorders and McArdle Disease.
REN001 is in Phase 1 clinical development. It has completed Phase 1 testing and is investigational, meaning it has not been approved by the FDA or any other regulatory agency for Fatty Acid Oxidation Disorders, McArdle Disease, or any other indication.
REN001 has been evaluated in two completed Phase 1 trials. NCT04226274 studied its safety in patients with McArdle Disease across Spain and the United Kingdom, enrolling 19 participants. NCT03833128 studied its safety in patients with Fatty Acid Oxidation Disorders across the United States, France, and Spain, enrolling 24 participants.
Yes, Low Dose REN001 is an alternative name for REN001. Both names refer to the same investigational small molecule developed by OnKure Therapeutics, Inc. for Fatty Acid Oxidation Disorders and McArdle Disease.