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

Vandana Singh
2 min read
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In this article:

  • PTC Therapeutics Inc (NASDAQ: 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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This article PTC Therapeutics Stock Tumbles: Company Stops Preclinical Gene Therapy Programs After Failed Friedreich's Ataxia Study originally appeared on Benzinga.com

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