Reneo Pharmaceuticals Raises $50M to Support Development of Therapies for Mitochondrial Diseases

Joana Carvalho, PhD avatar

by Joana Carvalho, PhD |

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Reneo Pharmaceuticals has raised $50 million to support the development of therapies for mitochondrial diseases associated with defects in cells’ metabolism and energy production.

The company’s fundraising includes contributions from several large investors, including Lundbeckfonden Ventures, Pappas Capital and RiverVest Venture Partners.

Part of the money will be used to support the clinical testing of REN001, Reneo’s investigational compound for the treatment of rare mitochondrial disorders, including fatty acid oxidation disorders (FAODs) and primary mitochondrial myopathies (PMMs), including two ongoing Phase 1 trials (NCT03862846 and NCT03833128), as well as future preclinical and clinical studies.

FAODs are disorders that affect the body’s ability to break down fatty molecules from food and use them as an energy source. PMMs prevent certain cells in the body, especially nerves and muscle cells, from using oxygen and food nutrients to produce energy.

Both conditions are caused by rare genetic defects and may be life-threatening if patients remain undiagnosed and fail to receive appropriate treatment.

REN001 is a peroxisome proliferator-activated receptor (PPAR) agonist, a class of drugs normally used to control symptoms of metabolic syndrome (a series of conditions that increase patients’ risk of developing heart disease, stroke, or type 2 diabetes), including lowering the levels of sugar and triglycerides in the blood.

Essentially, the compound works by enhancing the activity, and potentially increasing the number of mitochondria — the cell compartments responsible for the production of energy — in cells, boosting the production of energy from oxygen and food sources in all cells in the body.

“REN001 has demonstrated potential for both fatty acid oxidation disorders and primary mitochondrial myopathies,” Niall O’Donnell, PhD, president and CEO of Reneo, said in a press release. “The compound increases fatty acid metabolism, which has direct implications for fatty acid oxidation disorders. In addition, an increase in fatty acid metabolism has the potential to increase the amount of ATP within cells, which could in turn improve symptoms for patients afflicted with mitochondrial myopathies.”

Alejandro Dorenbaum, MD, CMO of Reneo, said: “People with FAODs or with PMMs have no approved medicines to help manage their disease. We are hopeful that our approach will generate meaningful data and allow us to provide a much-needed treatment option for people suffering from these diseases.”