HPP593 is a functionally selective peroxisome proliferator-activated receptor delta (“PPAR-delta”) agonist that does not exhibit off-target activity. In December 2017, we licensed worldwide rights to develop and commercialize HPP593 to Reneo Pharmaceuticals.
In June 2020, Reneo completed a 12-week clinical study (NCT03862846) in primary mitochondrial myopathies (PMM) patients with mitochondrial gene defects and a history of myopathy. Safety data from the study suggests that REN001 was well tolerated in PMM patients. Preliminary walk test data from this study is aiding in the design of a 200-patient clinical trial.
Reneo is also conducting an open label study in the United States to determine the safety and tolerability of REN001 in patients with fatty acid oxidation disorders. For additional information on the study, refer to NCT03833128 at Clinicaltrials.gov.
Finally, Reneo is conducting an open label study in the United Kingdom and Spain to determine the safety and tolerability of REN001 in patients with McArdle disease.
In June 2020, the FDA Office of Orphan Products Development granted Orphan Drug Designation to REN001 for the treatment of PMM. Last year, Reneo received FDA Orphan Drug Designation for REN001 for the treatment of fatty acid oxidation disorders.
Prior to its partnership with Reneo, vTv completed three phase 1 studies of up to 28 days in duration with HPP593. HPP593 was well tolerated at all doses tested.
Mechanism of Action
PPAR-delta, a member of the nuclear regulatory superfamily of ligand-activating transcriptional regulators, is expressed in most metabolically active tissues throughout the body. Studies in mice have shown that PPAR-delta agonists induce genes related to mitochondrial biogenesis. PPAR-delta activation by agonists also reduces blood glucose, improves insulin insensitivity and regulates inflammation.