"We are excited about the initiation of this study for PTC857, which was developed from our novel Bio-e platform," said
The Phase 1 study will evaluate PTC857 in healthy human volunteers with single and multiple ascending doses that will inform safety and pharmacology, and the design for the Phase 2 program. PTC857 has demonstrated efficacy in a number of GBA Parkinson's disease-relevant in vitro and in vivo test systems.
PTC857 is an investigational, novel small molecule in development for orphan neurodegenerative diseases of oxidative stress and inflammation from PTC's Bio-e platform. PTC857 targets the enzyme 15-lipoxygenase, a key enzymatic hub that regulates the inflammation and oxidative stress that underpin CNS pathology. PTC857 is currently being studied in GBA subtype Parkinson's disease (PD) , which is one of most common genetic risk factors for PD.[i]
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.
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