PTC Therapeutics Inc (NASDAQ: PTCT), a United States-based pharmaceutical company, has dosed its first patient in the phase one clinical trial of PTC857 in healthy volunteers, it was reported on Wednesday.
The product, which was discovered through the company's Bio-e platform, is an investigational small molecule drug that inhibits 15-Lipoxygenase, an enzyme that is a key regulator of the oxidative stress, protein aggregation and inflammation response pathways that underpin neurodegenerative disease pathologyj. The first indication proposed for the product is Parkinson's disease caused by a mutation in the GBA gene.
The Phase one study is to assess the product in healthy human volunteers with single and multiple ascending doses that will inform safety and pharmacology, and the design for the Phase two program. The product has indicated efficacy in various GBA Parkinson's disease-relevant in vitro and in vivo test systems.
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