Company announces phase 4 LEROS trial with Raxone (idebenone) met primary endpoint in patients with Leber’s hereditary optic neuropathy

In trial (n=199), after 12 months of treatment, more patients treated with Raxone achieved clinically relevant benefit vs. control group (43.1 vs. 20.7%; OR 2.286; 95% CI 1.352-3.884; p=0.002). Beneficial treatment effect was maintained at 24 months (2.082; 1.074-4.099; p=0.0297)

SPS commentary:

Idebenone is a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1). In patients with Leber’s hereditary optic neuropathy (LHON), the mitochondria do not produce sufficient energy for the retina to work effectively. Idebenone is thought to help support the mitochondria and allow the retina to function more effectively, which may improve vision or prevent blindness. In 2020, NHS England reviewed the evidence and concluded that there is not enough evidence to consider making idebenone available for treating people over 12 years of age with LHON.

Biospace Inc.