"We are excited to advance our Huntington's disease program," said
The PIVOT-HD Phase 2 clinical trial is designed in two parts: an initial 12-week placebo-controlled phase focused on PTC518 pharmacology and pharmacodynamic effect, followed by a 9-month placebo-controlled phase focused on PTC518 biomarker effect.
About Huntington's Disease
Huntington's disease (HD) is a rare, inherited disease that causes the progressive degeneration of nerve cells in the brain that has broad impact on a person's motor and functional capabilities, resulting in both movement disorders and cognitive loss. While HD can present at any age, it is most prevalent in people aged 30 to 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 a Phase 1 healthy volunteer study of PTC518 for Huntington's disease, PTC518 demonstrated a dose-dependent lowering of HTT mRNA and protein to the targeted 30-50% reduction. In addition, PTC518 showed that it passes the blood brain barrier and has minimal efflux which is a key factor in the targeting the underlying cause of Huntington's disease.
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 innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC's strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Instagram, Facebook, Twitter, and LinkedIn.
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