Positive Pipeline Updates for Friedreich’s Ataxia

PTC Therapeutics reported recent advance in the development of vatiquinone, a drug aimed at treating Friedreich's ataxia (FRDA), a rare degenerative disease that affects the nervous system.

Coming off of clinical trial setbacks in 2023, PTC Therapeutics reported promising long-term data that showed a significant reduction in disease progression as measured by the modified Friedreich ataxia Rating Scale (mFARS).

In two long-term extension studies that were clinically and statistically significant according to a PTC Therapeutics press release, the drug candidate hit key end points. The data came from the MOVE-FA long-term extension study that evaluated 144 weeks of vatiquinone treatment.

The MOVE-FA study evaluated vatiquinone in children and young adults with FRDA in a phase 3 trial, an 18-month, placebo-controlled study. The primary endpoint was a change from baseline in mFARS, with secondary endpoints assessing ambulation and activities of daily living.

The results indicated a 3.7-point benefit on mFARS at 144 weeks compared to a matched natural history cohort from the Friedreich Ataxia Clinical Outcome Measures (FACOMS) disease registry. The company explained that the achieved benefit can be viewed as a clinically meaningful 50% slowing of disease progression over three years compared to the natural course of diease.

In May 2023, PTC Therapeutics reported that the study did not meet its primary end point of a statistically significant change in mFARS score at 72 weeks but that treatment with vatiquinone achieved clinically meaningful benefits on key secondary endpoints.

According to the release, PTC plans to submit an NDA to the FDA for vatiquinone's approval by December 2024. The NDA will include results from the placebo-controlled portion of the MOVE-FA study. The company said it will also include confirmatory evidence from the two long-term treatment analyses and mechanistic data demonstrating the treatment's effect on biomarkers of disease pathology.

Vatiquinone is a first-in-class selective inhibitor of 15-Lipoxygenase (15-LO). The enzyme regulates the energetic and oxidative stress pathways that contribute to the pathophysiology observed in Friedreich ataxia. 15-LO inhibition helps mitigate the effects of mitochondrial dysfunction and oxidative stress, ensuring neuronal survival.

If approved, vatiquinone could be an additional treatment option, offering patients a chance to improve their quality of life and a potential pathway for further research into similar therapies. The promising results from the MOVE-FA study, supported by accompanying analyses of disease biomarkers, suggest that PTC has addressed previous concerns related to the drug's efficacy.

If the FDA grants approval, the approval would increase treatment options for this rare condition, improving outcomes for many affected individuals.