SOUTH PLAINFIELD, NJ, USA I October 18, 2022 I PTC Therapeutics, Inc. (NASDAQ: PTCT) confirmed today that enrollment is active and ongoing for its Phase 2 PIVOT-HD trial of PTC518 for the treatment of Huntington’s disease at study sites globally. To date, PTC has received approval for conduct of the planned 12-month study in several European countries as well as Australia.

The Phase 2 PIVOT-HD study consists of two parts; an initial 12-week placebo-controlled part focused on PTC518 pharmacology and pharmacodynamic effect, followed by a 9-month placebo-controlled part, during which blood, CSF, and radiographic biomarker data will be collected.

The study will initially include two dose levels, 5 milligrams and 10 milligrams, with the potential to study a third dose leveraging the titratability of the drug. The approvals received to date for the 12-month study include both the 5mg and 10mg dose levels, as well as a potential third dose pending the results of the first two dosing cohorts.

Enrollment in the 12-week portion of the study began first in the United States and continues globally. To date there have been no treatment-related adverse events reported in the US or outside of the US. However, the FDA has requested additional data to allow the study to proceed in the US. Accordingly, enrollment in the US has been paused while discussions with the FDA continue towards resuming US enrollment. 

PTC is working towards sharing data from the 12-week portion of PIVOT-HD within the first half of 2023.

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 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 and follow us on Instagram, Facebook, Twitter, and LinkedIn.

SOURCE: PTC Therapeutics