SOUTH PLAINFIELD, NJ, USA I June 29, 2023 I PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the MIT-E trial of vatiquinone for the treatment of mitochondrial disease associated seizures (MDAS) failed to achieve its primary endpoint of reduction in observable motor seizures. The study showed evidence of treatment effect in reducing seizure frequency in the overall study population and in the largest subgroup of children with Leigh syndrome, in whom benefit was also observed in the key secondary endpoints of occurrence of status epilepticus and disease-related hospitalizations.

“We are incredibly grateful to the patients, their families and our investigators who participated in this important study,” said Dr. Matthew Klein, Chief Executive Officer of PTC Therapeutics, Inc. “While we are disappointed with the results of the study, we hope that there are learnings that can benefit the development of other therapies for patients with mitochondrial disease, who remain without approved treatments for this highly morbid and fatal set of diseases.”

The MIT-E study is a double-blind, placebo-controlled study of vatiquinone in pediatric patients with genetically confirmed mitochondrial disease and associated refractory seizures. The study enrolled 68 children at study sites worldwide. The study included a 24-week placebo-controlled phase followed by a 48-week open-label phase. The primary endpoint was the reduction in observable motor seizures during the 24 weeks placebo-controlled phase. All subjects who completed the placebo-controlled phase were eligible to participate in the open-label phase of the study.

While the MIT-E study results do not support the advancement of vatiquinone for the treatment of MDAS, PTC continues to plan to discuss the results of the MOVE-FA trial of vatiquinone for the treatment of Friedreich ataxia with regulatory authorities based on the important signals of clinical benefit on key subscales of the mFARS, such as upright stability.

About Vatiquinone
Vatiquinone is an investigational new drug that targets 15-lipoxygenase, a regulator of key energetic and oxidative stress pathways. Vatiquinone has been evaluated in a number of clinical studies and has demonstrated an impact on mortality risk and a number of neurological and neuromuscular disease symptoms.1,2,3 The treatment also has extensive safety data, with the longest duration of exposure being over 10 years. 

About PTC Therapeutics, Inc.
PTC is a 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 Facebook, Instagram, LinkedIn and Twitter at @PTCBio.


  1. Hinman A, et al. PloS one. 2018;13:e0201369.
  2. PTC. EPI-743 Pre-Clinical Data Deck.
  3. Kahn-Kirby AH, et al. PloS one. 2019;14:e0214250.

SOURCE: PTC Therapeutics