Dosing Begins in Phase 1 Trial for PTC’s Experimental Parkinson’s Therapy PTC857

The first participant has been dosed in a Phase 1 clinical trial evaluating the safety of PTC857, PTC Therapeutics‘ investigational therapy for Parkinson’s disease. In addition to safety, the trial, which is being conducted in healthy volunteers, will evaluate how the therapy affects and is processed by the body. If the study concludes that the treatment has a reasonable safety profile, the results will be used to inform further investigations in people with Parkinson’s. Participants in the trial will receive either a single dose of PTC857 or they will get multiple ascending doses, in which the dosage increases each time. “We are excited about the initiation of this study for PTC857, which was developed from our novel Bio-e platform,” Stuart W. Peltz, PhD, CEO of PTC Therapeutics, said in a press release. The Bio-e platform uses biochemical knowledge to aid in the development of therapies that target oxidoreductases — a family of enzymes believed to play a central role in the diseases of the nervous system. PTC857 is the second compound developed through the Bio-e platform to enter clinical trials, the other being PTC743, which is being evaluated as a potential treatment for Frederich’s ataxia. Both PTC857 and PTC743 work by targeting the same enzyme: 15-lipoxygenase. By targeting this enzyme, it is believed that these treatments could reduce inflammation in the brain. In addition, targeting 15-lipoxygenase could reduce oxidative stress — an overproduction of highly reactive compounds that can damage cell structures. Since both inflammation and oxidative stress in the brain are thought to contribute to the development of neurological diseases, including Parkinson’s, reducing these processes by targeting 15-lipoxygenase with PTC857 could be therapeutic. PTC857 is first being developed specifically as a treatment for people with Parkinson’s who have a mutation in the GBA gene, a subtype known as GBA Parkinson’s disease, which is one the most common genetic subtypes of Parkinson’s disease. According to PTC Therapeutics, the investigational therapy has shown promise in animal models of GBA Parkinson’s disease, as well as in models using cells in dishes. “PTC857 is an inhibitor of the key enzyme whose products modulate fundamental pathways that are involved in neuroinflammation and oxidative stress that lead to multiple disorders including GBA Parkinson’s disease,” Peltz said. “We look forward to completing the healthy volunteer study and advancing PTC857 into patients.”