Reneo Pharmaceuticals Announces FDA Orphan Drug Designation for REN001 in Fatty Acid Oxidation Disorders

On September 23, 2019, VCBeat (WeChat ID: vcbeat) learned from foreign media reports that pharmaceutical company Reneo Pharmaceuticals (Reneo) announced that its lead candidate drug, REN001, had received FDA Orphan Drug Designation for the treatment of fatty acid oxidation disorders (FAOD).

The FDA established the Office of Orphan Products Development to support the development of drugs for rare diseases, providing safe and effective methods for prevention, diagnosis, and treatment to patients with rare diseases worldwide.

Reneo, founded in 2014 and headquartered in California, USA, is a clinical-stage pharmaceutical company. The company primarily focuses on developing innovative therapies for hereditary mitochondrial diseases, most of which are associated with mitochondrial energy deficiency. Reneo aims to improve patients’ quality of daily life, particularly by enhancing mitochondrial function within the body to maintain muscle performance and prevent muscle damage, weakness, and degeneration. With an experienced team of drug development experts and collaborations with healthcare companies, Reneo is committed to finding effective treatments for these complex rare diseases.

FAOD is a rare and potentially life-threatening genetic disorder that impairs the mitochondria’s ability to generate energy from long-chain fatty acids. Symptoms in adult patients include muscle damage, rhabdomyolysis, cardiomyopathy, and reduced exercise tolerance, which significantly impair quality of life and may lead to frequent hospitalizations. Currently, there are no FDA-approved therapies for FAOD.

Mitochondrial fatty acid oxidation (FAO) plays a crucial role in energy production, particularly during human starvation. The mitochondrial FAO process is highly complex, involving more than 20 steps, including cellular uptake of fatty acids, activation, carnitine shuttle-mediated transport across the mitochondrial membrane, re-esterification, mitochondrial β-oxidation, electron generation and transport, and the formation of ketone bodies from acetyl-CoA in the liver. Abnormalities in any of these metabolic pathways can lead to FAO disorders, resulting in insufficient energy supply to the body. In recent years, with the widespread adoption of gas chromatography-mass spectrometry (GC-MS) and tandem mass spectrometry (MS/MS) in the diagnosis of inherited metabolic disorders, mitochondrial FAO defects have garnered increasing attention among pediatricians.

REN001 is a selective PPAR agonist currently in clinical development, primarily for the treatment of FAOD and other inherited myopathies. The drug is undergoing a 12-week Phase 1b clinical trial at several clinical centers in the United States, aimed at evaluating its safety and tolerability in adult patients with FAOD.

Dr. Niall O'Donnell, CEO of Reneo Pharmaceuticals, stated, “Patients with fatty acid oxidation disorders (FAODs) are in urgent need of effective treatments, and we believe REN001 is a promising new therapy. The orphan drug designation will help Reneo pursue novel treatment options for patients with a range of rare FAODs.”

(Compiled by Hu Yifan)