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PTC Therapeutics Announces First Patient Dosed in Phase 1 Trial for PTC857

- First in human trial for PTC857, the second compound in the Bio-e platform to enter clinical trials -


SOUTH PLAINFIELD, N.J., July 8, 2020 – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the first subject in the Phase 1 clinical trial evaluating PTC857 in healthy volunteers has been dosed. PTC857, discovered through PTC’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 pathology. The first indication planned for PTC857 is Parkinson's disease caused by a mutation in the GBA gene (“GBA Parkinson’s disease”), which is one of the most common genetic subtypes of Parkinson’s disease. 

“We are excited about the initiation of this study for PTC857, which was developed from our novel Bio-e platform,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. “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. We look forward to completing the healthy volunteer study and advancing PTC857 into patients.”


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.


About PTC857

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]



About PTC Therapeutics, Inc.

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 and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.


[i] GBA Mutation May Lead to Novel Therapeutics (2020). Parkinson’s Foundation. Accessed June 30, 2020.

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Last Updated: 08-Jul-2020