Vatiquinone is under clinical development by PTC Therapeutics and currently in Phase III for Epilepsy. According to GlobalData, Phase III drugs for Epilepsy have a 67% phase transition success rate (PTSR) indication benchmark for progressing into Pre-Registration. GlobalData’s report assesses how Vatiquinone’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.
GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.
Vatiquinone (EPI-743) is under development for the treatment of Alpers-Huttenlocher syndrome, Friedreich ataxia, inherited mitochondrial disorders including Leigh syndrome, Alpers syndrome, mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), myoclonic epilepsy with ragged-red fibers (MERRF), pontocerebellar hypoplasia type 6 (PCH6), or other mitochondrial disease, mitochondrial diseases and refractory epilepsy. It is administered orally. It is a small molecule that readily crosses into the central nervous system. It works by targeting the enzyme NADPH quinone oxidoreductase 1 (NQO1) and arachidonate 15-lipoxygenase. It was also under development for the treatment of Rett syndrome, Melas syndrome, Leber’s hereditary optic neuropathy and mitochondrial diseases such as Pearson syndrome, a form of mitochondrial myopathy and Tourette syndrome. It was under development for the treatment of cobalamin C deficiency syndrome (methylmalonic acidemia), RARS2 syndrome, Parkinson's disease, metabolic disease (disorders of energy utilization or oxidation-reduction),Leigh's disease in children and noise-induced hearing loss.
PTC Therapeutics overview
PTC Therapeutics discovers, develops, and markets medicines for the treatment of rare disorders. Its marketed products include Evrysdi (risdiplam) for spinal muscular atrophy, Translarna (ataluren) and Emflaza (deflazacort) for the treatment of Duchenne muscular dystrophy. The company’s pipeline candidates target the treatment of Huntington’s disease; children with inherited mitochondrial disease; leiomyosarcoma; acute myelogenous leukemia; and aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics works in collaboration with various biotechnology and pharmaceutical companies for advancing its pipeline candidates. The company operates through its subsidiaries in the US and Europe. PTC Therapeutics is headquartered in South Plainfield, New Jersey, the US.
For a complete picture of Vatiquinone’s drug-specific PTSR and LoA scores, buy the report here.