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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