MOVE-FA Study Initiated for Friedriech’s Ataxia in Children and Young Adults


PTC Therapeutics announced the initiation of its third study of 2020 investigating vatiquinone.

Stuart Peltz, PhD,  founder and Chief Executive Officer, PTC Therapeutics

Stuart Peltz, PhD

PTC Therapeutics has announced the initiation of the global phase 3 MOVE-FA study (NCT04577352) of vatiquinone (PTC743) for Friedreich’s ataxia (FA). The study is currently recruiting children and young adults.1 Initiation of the trial was delayed by COVID-19.2

Vatiquinone was granted orphan drug designation and fast track designation for FA by the US FDA in 2014. There are currently no approved disease modifying therapies for FA, despite the estimated 25,000 patients with FA worldwide. Vatiquinone previously demonstrated a statistically significant effect on disease severity at 24 months in its phase 2 trial (NCT01962363), as assessed by the modified Friedreich ataxia rating scale (mFARS). While these results were promising, this trial only enrolled 4 participants, warranting further study.3

Stuart W. Peltz, PhD, founder and Chief Executive Officer, PTC Therapeutics, said in a statement that "this is the third clinical trial initiated this year based on compounds using our Bio-e platform. Vatiquinone is an exciting orally bioavailable small molecule that targets 15-lipoxygenase, the key enzyme regulating signaling pathways that control neuroinflammation and oxidative stress. Both pathways are important players in determining the pathology and disease progression seen in Friedreich ataxia patients. Previous clinical trial results give us confidence in vatiquinone's potential in treating patients living with this devastating disease."

The double-blind MOVE-FA trial will evaluate vatiquinone versus placebo in approximately 110 children and young adults with FA in parallel arms over 18 months. Patients will be enrolled from the US, EU, Australia, and Latin America and stratified by baseline mFARS score (<40 versus ≥40), age of disease onset (<14 versus ≥14), and age at screening (≤21 years or >21 years) and randomized to receive either vatiquinone or placebo.

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Doses will be either 200 mg or 400 mg orally 3 times per day depending on weight and age. A 24-week open-label extension will follow the 72 weeks of the placebo-controlled phase in which all participants will receive vatiquinone. A safety follow-up will be performed 10–30 days after the last dose.4

MOVE-FA's parallel endpoint will evaluate the change from baseline according to mFARS. Secondary endpoints include assessing change from baseline in Friedreich Ataxia Rating Scale Activities of Daily Living (FARS-ADL), 1-Minute Walk Test (1MWT), and number of falls. The estimated study completion date is currently July 6, 2023, although the company has stated that further delays may be experienced due to the uncertain impact that COVID-19 continues to pose.

Jennifer Farmer, Chief Executive Officer, FA Research Alliance, said in a statement that “we are excited for the PTC743 program to reach this milestone of opening enrollment of the MOVE-FA clinical trial and we look forward to assisting PTC with recruitment in the US and internationally."

PTC Therapeutics has initiated two other trials this year investigating vatiquinone, a Phase 2/3 trial (NCT04378075) of vatiquinone in children and adolescents with refractory mitochondrial epilepsy, and a Phase 1 study of PTC857 for Parkinson disease.5

1. PTC Therapeutics announces initiation of global phase 3 clinical trial to evaluate vatiquinone in friedreich ataxia. News release. PTC Therapeutics. November 30, 2020. Assessed December 22, 2020.
2. PTC Therapeutics reports first quarter 2020 financial results and provides a corporate update. News release. PTC Therapeutics. April 30, 2020. Assessed December 22, 2020.
3. EPI-743 in Friedreich's Ataxia Point Mutations. June 29, 2016. Accessed December 22, 2020.
4. A Study to assess the efficacy and safety of vatiquinone for the treatment of participants with friedreich ataxia (MOVE-FA). December 19, 2020. Assessed December 22, 2020.
5. Figueiredo M. PTC Therapeutics launches trial testing vatiquinone in children, young adults. Freidreich Ataxia News. December 3, 2020. Accessed December 22, 2020.
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