PTC Therapeutics' Seizure Study Fails To Meet Primary Goal, Analyst Says Minimal Impact On Investment Thesis

PTC Therapeutics Inc PTCT said its MIT-E trial of vatiquinone for 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 the largest subgroup of children with Leigh syndrome, in whom the benefit was also observed in the key secondary endpoints of occurrence of status epilepticus and disease-related hospitalizations.

Also Read: PTC Therapeutics Says Interim Data For Huntington's Disease Candidate Shows Promise In Mid-Stage 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 Friedreich ataxia (FA) with regulatory authorities based on the important signals of clinical benefit on key subscales of the mFARS, such as upright stability.

William Blair says the update is unfortunate, and it previously thought a 50%reduction in seizure frequency was a high bar to meet. 

Furthermore, based on the small addressable population (estimated global prevalence of 20,000 people), the program was not central to the analyst's valuation thesis. It keeps the Outperform rating.

The analyst still sees PTC Therapeutics' PKU program as undervalued based on the responses seen in the classical PKU subpopulation of patients and looks forward to the company's interactions with the FDA regarding vatiquinone for FA. 

Price Action: PTCT shares are trading lower by 2.98% at $40.00 premarket on the last check Friday.