Belite Bio (BLTE) Leerink’s Global Healthcare Conference 2025 summary

Disease background and drug mechanism

• Stargardt disease is a severe inherited juvenile macular dystrophy, with more rapid progression in children due to pathogenic genotypes.

• Tinlarebant is an oral, once-daily small molecule that antagonizes retinal binding protein 4, reducing vitamin A delivery to the eye and slowing toxic accumulation.

• Excess vitamin A in diseased eyes leads to toxin buildup and retinal tissue destruction; reducing vitamin A intake to the eye slows disease progression.

Clinical trial design and patient population

• Phase II open-label study enrolled 13 adolescent Stargardt patients, reducing their vitamin A by 80% and observing a 50% reduction in lesion growth over two years.

• Phase III DRAGON study enrolled 104 subjects globally, using the same dose and design as phase II, with interim analysis after 12-16 months.

• DRAGON 2 study in Japan leverages Sakigake designation for potential single-study approval, with 10 Japanese subjects over two years.

• The geographic atrophy (GA) phase III study targets 429 subjects, focusing on early intervention and using the same drug and endpoints.

Efficacy and endpoints

• Primary endpoint is slowing the growth of atrophic lesions, measured by retinal imaging, as lesion growth precedes vision loss.

• Secondary endpoint includes reduction in autofluorescent lesions, which precede atrophy and are linked to visual acuity loss.

• Phase II data showed about a 50% reduction in both atrophic and overall lesion growth compared to natural history.

• Interim phase III analysis indicated a strong efficacy trend, with DSMB recommending submission for regulatory review and no need to add more subjects.