RARE Daily

Reneo Raises $50 Million to Develop Therapies for Mitochondrial Diseases

May 21, 2019

San Diego-based Reneo Pharmaceuticals has completed a $50 million series A financing to develop therapies for diseases associated with deficits in cellular metabolism and energy production.

New Enterprise Associates led the round with participation from Lundbeckfonden Ventures, Pappas Capital, and RiverVest Venture Partners. Reneo was founded by Mike Grey and Niall O’Donnell, both of whom were part of the management team of rare liver disease specialist Lumena before it was acquired by Shire in 2014.

Reneo’s goal is to improve daily function and quality of life for patients suffering from these diseases by improving how their mitochondria work, and thus preserving muscle function and preventing muscle injury, weakness, and wasting.

The biotech’s most advanced candidate, REN001, activates the PPAR delta receptor that is believed to regulate certain metabolic processes. Proceeds from the funding will be used to support ongoing phase 1b clinical trials, as well as other clinical and preclinical studies.

REN001 has demonstrated potential for both fatty acid oxidation disorders (FAODs) and primary mitochondrial myopathies (PMMs), according to Niall O’Donnell, president and CEO of Reneo.

“The compound increases fatty acid metabolism, which has direct implications for fatty acid oxidation disorders,” he said. “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.”

Mitochondria are known as the powerhouses of the cell where carbohydrates, fats and proteins are used to generate the energy the body needs. Inherited mutations in mitochondria result in impaired energy metabolism that result in multisystem disorders.

FAODs are rare autosomal recessive disorders that affect the body’s ability to use fats from food as an energy source. If undiagnosed and untreated, these disorders can lead to serious complications resulting in hospitalizations and in some cases death. PMMs are a group of complex, rare, and often life-threatening diseases caused by mutations in genes of the mitochondria that result in energy deprivation in different tissues, particularly nerves and muscles. Common symptoms of these diseases can include muscle pain, weakness, cramps and muscle wasting, that can be accompanied by activity intolerance, and sometimes severe muscle tissue breakdown known as rhabdomyolysis.

“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,” said Alejandro Dorenbaum, chief medical officer of Reneo.

Photo: Alejandro Dorenbaum, chief medical officer of Reneo

Author: Rare Daily Staff

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