Reneo Pharmaceuticals has enrolled 18 patients in an observational study (FORWARD) for patients with long-chain fatty acid oxidation disorder (LCFAOD) and has dosed 20 patients who have the disease in the open-label phase 1b REN001-102 trial, the company announced.

“We . . . made substantial progress in our long-chain fatty acid oxidation disorders studies” in the second quarter of 2021, Gregory J. Flesher, president and chief executive officer of Reneo Pharmaceuticals, said in a press release from the company.

The purpose of the FORWARD study is to collect information on disease characteristics from adults with LCFAOD. It aims to enroll 90 participants 18 years and older and is currently recruiting participants at 21 locations in the US, the UK, and Europe. The study’s estimated completion date is February 2022, with results anticipated in the second half of 2022.

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Learn more about LCFAOD treatment

REN001-102 aims to assess the safety of 2 doses of REN001 in adult patients with LCFAOD. It is currently recruiting an estimated 24 participants in the US, France, and Spain who have a confirmed genetic mutation resulting in carnitine palmitoyltransferase II deficiency (CPT2), very long-chain acyl-CoA dehydrogenase deficiency (VLCAD), long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency (LCHAD), or trifunctional protein deficiency (TFP). 

REN001 is a selective peroxisome proliferator-activated receptor delta (PPARδ) agonist. Apart from LCFAODs, it is also in clinical trials for primary mitochondrial myopathies and glycogen storage disorder type V (McArdle disease).

Research has shown that REN001 increases the transcription of genes involved in mitochondrial function and increases fatty acid oxidation. It may also increase the production of new mitochondria.

The primary endpoint of the REN001-102 study is the number of participants with adverse events. The trial is estimated to be completed in January 2022, and results are expected to be available in the first half of 2022.


Reneo Pharmaceuticals reports second quarter 2021 financial results. News release. Reneo Pharmaceuticals; August 11, 2021.

A prospective study of the disease characteristics of adult patients with long chain fatty acid oxidation disorders (FORWARD). July 22, 2020. Updated July 26, 2021. Accessed August 23, 2021.

A study of the safety of REN001 in patients with fatty acid oxidation disorders. February 6, 2019. Updated July 26, 2021. Accessed August 23, 2021.