PTC Therapeutics has announced positive results from long-term extension studies of vatiquinone in patients with Friedreich Ataxia (FA), showing durable treatment benefits and supporting the planned NDA submission to the FDA by the end of 2024. The data indicate a significant slowing of disease progression, with a favorable safety profile.
Long-Term Efficacy Confirmed
Analysis of the MOVE-FA long-term extension study revealed that 144 weeks of vatiquinone treatment resulted in a 3.7-point benefit (p<0.0001, N=70) on the modified Friedreich Ataxia Rating Scale (mFARS) compared to a matched natural history cohort from the FACOMS registry. This represents a clinically meaningful 50% slowing in disease progression over three years, confirming the benefits observed in the initial 72-week MOVE-FA trial.
In an earlier study, adults treated with vatiquinone for 24 months experienced a 4.8-point benefit on the mFARS relative to a matched natural history population (p<0.0001, N=41). These results further support the long-term efficacy of vatiquinone in managing FA progression.
Safety and Tolerability
Vatiquinone has demonstrated a consistent safety profile across multiple clinical studies. The long-term extension studies reported no treatment-related serious adverse events, reinforcing its potential as a safe therapeutic option for FA patients, including children and adolescents.
Regulatory Pathway
PTC Therapeutics is preparing to submit an NDA to the FDA in December 2024. The submission will include data from the placebo-controlled MOVE-FA study, which showed a statistically significant effect (p=0.021) on the mFARS upright stability subscale, particularly relevant for pediatric and young adult patients. The NDA will also incorporate confirmatory evidence from the long-term treatment analyses and mechanistic data demonstrating the treatment's effect on biomarkers of disease pathology.
Vatiquinone Mechanism of Action
Vatiquinone is a first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme involved in regulating energetic and oxidative stress pathways disrupted in Friedreich Ataxia. By inhibiting 15-LO, vatiquinone helps mitigate the consequences of mitochondrial dysfunction and oxidative stress, preventing ferroptosis and promoting neuronal survival.
Friedreich Ataxia: An Unmet Need
Friedreich Ataxia is a rare, debilitating, and life-shortening neuromuscular disorder affecting approximately 25,000 people globally. It is characterized by progressive loss of coordination and muscle strength, leading to impaired balance, speech difficulties, and cardiac complications. The condition is typically diagnosed in childhood or adolescence. Currently, there are limited treatment options available, particularly for pediatric patients, highlighting the urgent need for effective therapies like vatiquinone.
