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- 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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PTCTPTC Therapeutics Inc
$47.00-3.15%
Edge Rankings
Momentum
84.74
Growth
N/A
Quality
N/A
Value
43.70
Price Trend
Short
Medium
Long
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