To minimize risk during the COVID-19 pandemic, Reata Pharmaceuticals is implementing at-home visits to deliver omaveloxolone to Friedreich’s ataxia (FA) patients and to assess the treatment’s safety in an extension study of the MOXIe Phase 2 clinical trial.
“The COVID-19 pandemic presents an unprecedented threat to public health,” Warren Huff, president and CEO at Reata, the company developing omaveloxolone, said in a press release. “The adjustments we have made to our studies reflect our foremost concern for the safety and well-being of study participants, clinical investigators and their site staffs, our employees and communities as we face this challenging and unpredictable operating environment.”
Omaveloxolone (RTA-408) is intended to activate Nrf2, a molecule that regulates gene expression (called a transcription factor) to restore energy production in cells — an impaired process in people with FA. Preclinical studies suggested the treatment has antioxidant and anti-inflammatory properties.
The MOXie trial (NCT02255435) took place in two phases: first, a dose-escalation phase, where 69 people with FA were given increasing oral doses of omaveloxolone (between 5 and 300 mg) daily for 12 weeks. This was used to set the dose for the second part of the trial, in which 103 people with FA were randomly assigned to receive either 150 mg of omaveloxolone or a placebo once a day.
Topline results showed that, after 48 weeks of treatment, omaveloxolone led to a 2.4-point improvement in the modified Friedreich’s ataxia rating scale (mFARS, an FDA-endorsed scale to measure FA disease progression) relative to placebo among participants without pes cavus. Including participants with this foot deformity, omaveloxolone treatment led to a 1.93-point improvement in mFARS compared to placebo.
Participants in both parts of the trial were allowed to enroll in the open-label extension where all patients would receive omaveloxolone.
Because of the COVID-19 pandemic, and in line with social distancing recommendations, modifications are being made to minimize the risk to participants and investigators. Where possible, onsite visits are being replaced with at-home visits, and at-home delivery of the therapy has been arranged.
Reata does not anticipate changes to its plan to seek regulatory approval of omaveloxolone as an FA treatment in the U.S.
The company also announced similar changes to ongoing late-stage trials testing bardoxolone methyl, an anti-inflammatory agent being investigated for several conditions, including kidney disease related to Alport syndrome in the CARDINAL trial (NCT03019185), and autosomal dominant polycystic kidney disease in the FALCON study (NCT03918447). While recruitment of new participants in FALCON has been put on hold, the trials are otherwise continuing as planned, although with similar modifications to minimize interpersonal contact.
However, the Phase 3 trial CATALYST (NCT02657356), which was testing bardoxolone methyl in people with pulmonary arterial hypertension (PAH), has been stopped. Because people with PAH have an underlying respiratory disease and are often on therapies that weaken the immune system, they are particularly vulnerable to COVID-19.
As such, “continued exposure of these high-risk patients to clinic or in-person visits [as would be required for the trial] presented an unacceptable risk,” Reata stated.
An extension study of bardoxolone methyl in PAH, named RANGER (NCT03068130), has likewise been halted.
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