Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
REN001 · 1 trial · 1 indication
Number of participants with Adverse Events (AEs) as a measure of safety and tolerability
| Arm | Type | Description |
|---|---|---|
| REN001 | EXPERIMENTAL | Oral |
| Name | Type | Description |
|---|---|---|
| REN001 | DRUG | Once daily for 12 weeks |
Inclusion Criteria: * Subjects must give written, signed and dated informed consent * Confirmed diagnosis of McArdle Disease * Subjects must be able to remain on stable medication through the study and specifically must not commence or have changes to agents that affect metabolism such as medicatio...
Low Dose REN001 is an investigational small molecule being developed for Fatty Acid Oxidation Disorders and McArdle Disease. It is currently in Phase 1 clinical development and is not yet approved by the FDA.
Low Dose REN001 is being developed by OnKure Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol OKUR.
Low Dose REN001 is in Phase 1 clinical development. Two Phase 1 studies have been completed, one in Fatty Acid Oxidation Disorders and one in McArdle Disease. It remains investigational and has not been approved.
Low Dose REN001 has completed two Phase 1 trials. NCT03833128 studied safety in 24 patients with Fatty Acid Oxidation Disorders in the United States, France, and Spain. NCT04226274 studied safety in 19 patients with McArdle Disease in Spain and the United Kingdom.
Low Dose REN001 is a formulation of REN001, the same investigational small molecule. The clinical trials for Fatty Acid Oxidation Disorders and McArdle Disease used the REN001 name, and Low Dose REN001 refers to a lower dose of this compound.