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June 25, 2021
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Phase 4 trial shows long-term efficacy of Raxone for Leber’s hereditary optic neuropathy

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A long-term phase 4 trial investigating Raxone for the treatment of Leber’s hereditary optic neuropathy met its primary endpoint, according to a press release from Santhera Pharmaceuticals.

LEROS is an externally controlled open-label intervention study intended to confirm the efficacy of Raxone (idebenone) in patients with Leber’s hereditary optic neuropathy (LHON) after 12 months. Additionally, the study is investigating long-term efficacy and safety data over 24 months.

The primary endpoint was the proportion of eyes to experience clinically relevant benefits after 12 months of treatment with Raxone vs. untreated patients. After 12 months, 43.1% of patients receiving Raxone achieved a statistically significant benefit compared with 20.7% of patients in the external control group (P = .002), and the benefit was maintained at 24 months (P = .0297), the release said.

The study also showed positive results in patients who started treatment 1 year or more after symptoms began (P = .0058).

“These results confirm the efficacy of idebenone in the treatment of LHON by significantly increasing the chances for vision recovery and/or for prevention of further vision loss,” Thomas Klopstock, MD, professor for neurology at the University of Munich, LHON researcher and principal LEROS study investigator, said in the release.