Stages of Development for Vatiquinone (PTC-743)

The drug development process can be thought of as a series of stages, and successful drugs must pass through each stage to become available to patients.

Link to PTC Therapeutics

2012: Several publications report on open-label clinical studies of EPI-743 (aka PTC-743) in mitochondrial diseases, such as Leigh syndrome and Leber Hereditary Optic Neuropathy. In both studies, there was evidence that patients receiving EPI-743 had clinical improvements and no adverse events were observed. An earlier publication reports on experience in treating 14 patients with various mitochondrial conditions with EPI-743. This open-label study reports “EPI-743 has modified disease progression in > 90% of patients in this open-label study as assessed by clinical, quality-of-life, and non-invasive brain imaging parameters” and controlled trials to follow-up these findings.

January 2013: Edison Pharmaceuticals announced the launch of a Phase 2 clinical trial of EPI-743 in adults with FA. There were three sites – University of South Florida, Children’s Hospital of Philadelphia and University of California Los Angeles. Subjects were recruited through the FARA Patient Registry, and all subjects were enrolled by the fall of 2013.

March 2014: Edison announced that FDA granted EPI-743 Fast Track designation.

April 2016: Results were reported from the open-label study of EPI-743 in FA patients with point mutations and can be accessed here.

September 2016: Study investigators reported the results of the EPI-743 phase 2 trial at the FA educational symposium. A total of 63 subjects were enrolled in the trial. EPI-743 was found to be safe and well-tolerated over the entire 24 months study period. There were no significant differences between EPI-743 treatment groups and placebo at 6 months on the primary endpoint, a measure of vision; however, there was a trend towards improvement in neurological function as assessed by the FARS neuro scale. In the analysis of the entire 24-month trial there was suggested evidence of a significant improvement in neurological function based on the FARS score in EPI-743 treated subjects when compared to an age, sex and disease-severity matched natural history cohort followed as part of the Friedreich’s Ataxia-Clinical Outcomes Measures Study (FA-COMS). Results of this clinical trial have been published and can be accessed here.

Early 2017: Edison Pharmaceuticals announced name change to BioElectron Technology Corporation.

Late 2019: PTC Therapeutics acquired assets from BioElectron Technology Corporation which included EPI-743.

2022: PTC started a Phase II EPI-743 Clinical trial – An Open-Label Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Vatiquinone in Children With Friedreich Ataxia Younger Than 7 Years of Age

January 2021: PTC initiates a registration-directed Phase III trial, MOVE-FA study, evaluating vatiquinone in children and young adults with Friedreich ataxia (FA). The Phase 3 MOVE-FA trial is an 18-month parallel arm, placebo-controlled study evaluating vatiquinone versus placebo in children and young adults with FA. The primary endpoint is the change from baseline in mFARS, with key secondary endpoints assessing ambulation and activities of daily living.

November 2021: The study completed enrollment with >140 subjects enrolled across 14 sites in 9 different countries.

PTC Therapeutics would like to express their gratitude to the Friedreich ataxia community, patients, and families. We appreciate the commitment and dedication of those who are participating in, or considered participating in, the MOVE-FA study. PTC remains focused on those who are impacted by this condition and values the partnership of the community.

May 23, 2023: PTC reported topline results from the MOVE-FA trial of vatiquinone in patients with FA. The study did not meet its primary endpoint of statistically significant change in mFARS score at 72 weeks in the primary analysis population (all patients enrolled, ages 7-21yrs, N=123). However, vatiquinone treatment did demonstrate significant benefit on key secondary endpoints that are clinically significant to individuals with FA.  There was significant improvement in the patient reported outcome measure of fatigue, the Modified Fatigue Scale (nominal p value of 0.025). The vatiquinone treated group demonstrated less decline in the upright stability of mFARS (measures standing and walking) compared to the placebo group (p<0.021). The study experienced a higher number of early withdrawals, mostly due to COVID-related issues, so there was an additional prespecified analysis with the population of subjects who completed all visits through week 72 (n=96).  In the population of subjects that completed the study protocol, significance was reached in the mFARS endpoint (p <0.05), upright stability subscale of mFARS (p<0.026), and modified fatigue rating scale (p<0.042). The placebo corrected difference in mFARS was 2.31, which represents a 75% slowing in disease progression over 72 weeks. PTC stated that they will discuss the potential path to registration with FDA and EMA based on these results, the unmet need in children with FA and the favorable safety data for vatiquinone.

January 8, 2024: PTC Therapeutics provided an update on commercial progress and R&D pipeline at the 42nd Annual J.P. Morgan Healthcare Conference. They announced a FDA meeting for vatiquinone to discuss how the MOVE-FA data along with additional clinical and preclinical data could support an NDA submission in FA is scheduled for the first quarter. Additionally, scientific advice feedback from the EMA on a potential submission of vatiquinone for conditional marketing authorization for Friedreich ataxia is expected in the first quarter.

April 25, 2024: In their corporate update and report on first quarter 2024 financial results, PTC announced plans to submit an NDA for vatiquinone for the treatment of FA in late 2024 based on FDA feedback.

News and Press Releases

| May 23, 2023

PTC Therapeutics Announces Topline Results from Vatiquinone MOVE-FA Registration-Directed Trial

Industry News
| Nov 30, 2020

PTC Therapeutics Announces Initiation of Global Phase 3 Clinical Trial to Evaluate Vatiquinone in Friedreich Ataxia

Industry News
| Mar 17, 2014

FDA Awards Fast Track Status to Edison Pharmaceuticals' EPI-743 for Friedreich's Ataxia

Industry News
| Feb 4, 2014

FDA Grants Edison Pharmaceuticals' EPI-743 Orphan Status for Friedreich's Ataxia

Industry News

Vatiquinone PTC-743 Clinical Trials

Improve Mitochondrial Function & Reduce Oxidative Stress
Clinical Trial | Ages 7-21

A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study With Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich Ataxia (MOVE-FA)

PTC Therapeutics
Phase III | In-person
Improve Mitochondrial Function & Reduce Oxidative Stress
Clinical Trial | Age <7 years

A Study of Vatiquinone for the Treatment of Participants with Friedreich’s Ataxia

PTC Therapeutics
Phase II | In-person