Belite Bio (BLTE) H.C. Wainwright 6th Annual Ophthalmology Virtual Conference summary

Key advances in inherited retinal disorder therapies

• Disease-modifying therapies and gene therapy are most effective in early to intermediate stages, while restorative approaches like optogenetics are suited for advanced disease with severe photoreceptor loss.

• Functional benefit is the ultimate goal, but structural preservation is accepted as a surrogate endpoint due to slow disease progression; longer-term follow-up is needed to confirm functional gains.

• In advanced retinitis pigmentosa, endpoints such as mobility, object localization, and navigation under varied lighting are more meaningful than visual acuity alone.

• No reliable clinical selection algorithm exists for optogenetics, but preserved inner retinal tissue and optic nerve integrity are considered favorable.

Clinical trial insights and efficacy outcomes

• The DRAGON trial for Stargardt disease showed a 35.7% reduction in annualized macular lesion growth, considered substantial compared to other therapies for geographic atrophy.

• Visual acuity changes are minimal over two years, but structural preservation is expected to yield functional benefits over decades.

• Treatment effect in DRAGON was not strongly dependent on baseline lesion size or ABCA4 genotype, supporting broad applicability.

• Early intervention is recommended, as patients with more preserved photoreceptors benefit most, but even those at legal blindness may see slowed progression.

Novel gene therapy and optogenetics developments

• MCO-010 targets inner retinal cells, restoring vision from light perception to up to 20/50 in some patients, with improvements in mobility and daily activities.

• All treated patients showed improvement in at least one efficacy endpoint, with thicker retinas correlating with better outcomes.

• Durability data for MCO-010 show up to four years of sustained benefit, with expectations for decades-long efficacy due to the mechanism of action.

• Bilateral functional improvement has been observed after unilateral administration, attributed to vector transfer via the optic chiasm.