Reneo Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted mavodelpar (REN001) Fast Track designation for long-chain 3-hydroxyacyl-CoA dehydrogenase (LCHAD) deficiency, one of the predominant genotypes in patients with long-chain fatty acid oxidation disorder (LC-FAOD). The FDA's Fast Track regulatory process is designed to facilitate and expedite the development of investigational treatments that demonstrate a potential to address unmet medical needs in serious or life-threatening conditions. Programs with FDA Fast Track designation can benefit from early and frequent communication with the FDA in addition to a rolling submission of the marketing application.

The company recently completed a natural history study (FOR WARD) and an open label study evaluating mavodelpar in patients with LC-FAOD that included multiple genotypes, including the LCHAD genotype. Mavodelpar is also being evaluated in a pivotal clinical trial (STRIDE study) in patients with primary mitochondrial myopathies (PMM) with mitochondrial DNA (mtDNA) defects. The company expects topline data from the STRIDE study in the fourth quarter of this year.

About Reneo Pharmaceuticals Reneo is a clinical-stage pharmaceutical company focused on the development and commercialization of therapies for patients with rare genetic mitochondrial diseases, which are often associated with the inability of mitochondria to produce adenosine triphosphate (ATP). The company's lead product candidate, mavodelpar (REN001), is a potent and selective agonist of the peroxisome proliferator-activated receptor delta (PPAR). Mavodelpar has been shown to increase transcription of genes involved in mitochondrial function and increase fatty acid oxidation and may increase production of new mitochondria.

Mavodelpar (REN001) is a potent and selective peroxisome proliferator-activated receptor delta (PPAR) agonist currently in clinical development for two rare genetic mitochondrial diseases that typically present with myopathy and have high unmet medical needs: primary mitochondrial myopathies (PMM) and long-chain fatty acid oxidation disorders (LC- FAOD). PMM are a group of rare, genetic metabolic disorders caused by mutations or deletions in the mitochondrial DNA (mtDNA) or nuclear DNA (nDNA). These genetic alterations hamper the ability of mitochondria to generate energy from nutrient sources, resulting in energy deficits that are most pronounced in tissues with high energy demand such as muscle, brain, and heart.

The symptoms of PMM include muscle weakness, exercise intolerance, movement disorder, deafness, blindness, and droopy eyelids among others. The prognosis for these disorders ranges in severity from progressive weakness to death. About LC-FAOD LC-FAOD are a group of rare, genetic metabolic disorders caused by mutations or deletions in the nuclear DNA (nDNA).

These genetic alterations prevent the body from breaking down long-chain fatty acids during metabolism. The most severe cases of LC-FAOD are diagnosed within the first few days or weeks of life. Young patients often present with a severe energy deficit that results in lethargy, liver dysfunction, hypoglycemia, encephalopathy, and high risk for sudden death.

Older patients usually present with muscle weakness, exercise intolerance, muscle aches, or rhabdomyolysis which can damage the heart and kidneys and cause permanent disability or even death.