Positive results from long-term treatment studies and updates on regulatory progress for vatiquinone for Friedreich's ataxia program-PTC Therapeutics

The pre-specified endpoint for two different FA long-term extension studies was met, with highly statistically significant evidence of durable treatment benefit on disease progression. In addition, PTC recently aligned with FDA on key aspects of the planned NDA submission for vatiquinone. "The results of the extension studies provide further evidence of the potential benefit of vatiquinone in slowing disease progression," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "In addition, the strong safety profile of vatiquinone positions it to be a potentially meaningful therapy for all Friedreich ataxia patients, particularly children and adolescents for whom there are no approved therapies. We look forward to submitting the NDA by the end of the year."

Analysis of the MOVE-FA long-term extension study demonstrated 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) relative to a matched natural history cohort from the FACOMS (Friedreich Ataxia Clinical Outcome Measures) disease registry. This treatment difference on the primary endpoint represents a clinically meaningful 50% slowing in disease progression over 3 years. These results confirm that the slowing of disease progression recorded in the 72-week placebo-controlled MOVE-FA trial are maintained over 144 weeks of treatment. In addition, vatiquinone continued to be safe and well tolerated without any treatment-related serious adverse events reported.

In addition, PTC analyzed long-term open-label data from an earlier study of vatiquinone in adults with FA. Following 24-months of treatment with vatiquinone, subjects had a 4.8-point benefit on the mFARS relative to a matched natural history population (p<0.0001, N=41).

"MOVE-FA was a well-conducted international clinical trial in children and adults with Friedreich's ataxia. Both this trial data and the open-label extension data are compelling with positive results in clinical endpoints that are meaningful to the FA community," said Jennifer Farmer, Chief Executive Officer of the Friedreich's Ataxia Research Alliance (FARA). "We are also encouraged that vatiquinone treatment continues to be safe and well-tolerated. Given the high unmet need, especially in the pediatric population, we are excited that PTC Therapeutics is submitting an NDA."

PTC plans to submit the vatiquinone NDA in December 2024. The NDA will include results from the placebo-controlled portion of the MOVE-FA study in which there was significant benefit recorded on the Upright Stability Subscale of the mFARS. This subscale is the most relevant and sensitive mFARS component for pediatric and young adults. The NDA will also include confirmatory evidence from the two long-term treatment analyses discussed above, as well mechanistic data demonstrating treatment effect on biomarkers of disease pathology.

Vatiquinone is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich ataxia. Inhibition of 15-LO helps to alleviate the consequences of mitochondrial dysfunction and oxidative stress, ultimately preventing ferroptosis and aiding neuronal survival. Vatiquinone has been evaluated in a number of clinical studies, many focused on pediatric patients, and has demonstrated an impact on mortality risk and a number of neurological and neuromuscular disease symptoms.