REN001 is safe and well tolerated in patients with long-chain fatty acid oxidation disorder (LCFAOD), according to results from a recently completed phase 1b clinical trial. 

“We are very encouraged by the results of the LCFAOD phase1b study,” Alejandro Dorenbaum, MD, chief medical officer of Reneo Pharmaceuticals, the developers of REN001, said in a press release. “Safety and tolerability of REN001 in this study was consistent with prior REN001 studies.”

 The 12-week, open-label study recruited 24 patients aged 18 or more years with different types of LCFAOD.

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Participants were given either a low or high dose of oral REN001. The study’s primary outcome measure was the number of participants with adverse events, as a measure of safety and tolerability. The study also explored potential efficacy measurements for future clinical trials.

Read more about the etiology of different types of LCFAOD

The results showed that the treatment was safe and well tolerated, with the most common adverse events being rhabdomyolysis and myalgia. The researchers also found that the 12-minute walk distance increased in patients with long chain 3-hydroxy acyl-CoA dehydrogenase (LCHAD) deficiency and carnitine palmitoyltransferase 2 (CPT2) deficiency following treatment.

“The increase in walk distance observed in both the LCHAD and CPT2 subgroups supports the use of the 12-minute walk test as key efficacy measurement for future LCFAOD clinical trials,” Dr. Dorenbaum said. “We look forward to meeting with regulatory agencies to discuss next steps.”

Reneo also conducted a 16-week, observational study on 58 participants with different types of LCFAOD to better understand the natural history of the disease. 

Based on results from both studies, the company intends to continue the development of REN001 as a potential treatment for LCFAOD.

REN001 is a selective peroxisome proliferator-activated receptor delta (PPARδ) agonist. It can control a number of genes involved in the activity of mitochondria.


Reneo Pharmaceuticals reports positive results from REN001 long-chain fatty acid oxidation disorders (LC-FAOD) study and provides additional development updates. News release. Reneo Pharmaceuticals, Inc; July 26, 2022.

A study of the safety of REN001 in patients with fatty acid oxidation disorders. February 6, 2019. Updated January 11, 2022. Accessed August 8, 2022.