The Huntington Disease Deep Dive will provide an overview of PTC's splicing platform and the identification of molecules that have selectivity and specificity. PTC will highlight its Huntington program and the potential advantages of an orally bioavailable compound. PTC will discuss in detail the splicing modifier, PTC518, that is in an ongoing Phase 1 clinical trial. In preclinical studies, PTC518 has demonstrated broad biodistribution that uniformly reaches all regions of the brain and periphery and has shown reduction in Huntingtin protein (HTT) levels with a 1:1 correlation between blood and brain. PTC will also present preliminary results from the Phase 1 study of PTC518.
The webinar can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the webinar and providing the passcode 5190575. A live, listen-only webcast can be accessed on the Events and Presentations page under the investor relations section of
About Huntington Disease
Huntington disease (HD) is a rare, inherited disease that causes the progressive degeneration of nerve cells in the brain, impacting a person's functional abilities.1 While HD can present at any age, it is most prevalent in people aged 30 – 50, and it affects approximately 45,000 people in
PTC518 is a small molecule splicing modifier that acts via a unique mechanism to promote the inclusion of a novel pseudoexon containing a premature termination codon, thus triggering HTT mRNA degradation and subsequent reduction in HTT protein levels. In preclinical studies using cells isolated from patients with Huntington disease (HD), PTC518 reduced both huntingtin mRNA and protein levels with high potency in a dose-dependent manner. In addition, oral administration of PTC518 in the BACHD mouse model of HD achieved dose dependent and equitable lowering of HTT protein throughout the body, including the brain, muscle, and blood.
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. The Company's strategy is to leverage its strong scientific expertise and global commercial infrastructure to maximize value for its patients and other stakeholders. 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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1 Huntington's disease. (2020,
2 Fisher ER, Hayden MR. Multisource ascertainment of Huntington disease in
3 Overview of Huntington's disease. (n.d.). Retrieved