PTC Therapeutics Stock Tumbles: Company Stops Preclinical Gene Therapy Programs After Failed Friedreich's Ataxia Study

  • PTC Therapeutics Inc PTCT reported topline results from the MOVE-FA trial of vatiquinone in patients with Friedreich ataxia
  • The study did not meet its primary endpoint of statistically significant change in mFARS score at 72 weeks in the primary analysis population.
  • The FARS scale is a series of physical examination assessments to measure disease progression.
  • The company says that vatiquinone treatment did demonstrate significant benefits on key disease subscales and secondary endpoints. 
  • In addition, in the population of subjects that completed the study protocol, significance was reached in the mFARS endpoint and several secondary endpoints.
  • The MOVE-FA trial enrolled 146 pediatric and adult patients.
  • The mean placebo-corrected change in the mFARS score in the primary analysis population was 1.6 (p=0.14). There was a significant benefit recorded in the bulbar and upright stability subscales, reflective of key aspects of disease morbidity and predictive of loss of time to loss of ambulation. 
  • In addition, a statistically significant difference was recorded on the Modified Fatigue Scale. 
  • Overall, vatiquinone was well tolerated.
  • Also Read: Why PTC Therapeutics Shares Are Gaining Today.
  • Concurrently, PTC Therapeutics discontinued preclinical and early research programs in gene therapy as part of a strategic portfolio prioritization
  • PTC will continue its development and global commercialization of Upstaza.
  • Estimated reductions of approximately 15% in residual 2023 operating expenses are expected.
  • PTC Therapeutics' discontinued gene therapy programs include preclinical stage programs in Friedreich ataxia and Angelman syndrome and several other programs targeting rare CNS and ophthalmological disorders. 
  • The prioritization decision will allow for additional focus on PTC's proprietary splicing platform and additional CNS and metabolic disorders.
  • In addition, PTC's CFO, Emily Hill, will leave the organization.
  • Price Action: PTCT shares are down 23.8% at $44.54 on the last check Wednesday.
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