Belite Bio (BLTE) Deutsche Bank ADR Virtual Investor Conference summary

Key presentations and clinical updates

• Tinlarebant, an oral once-daily therapy, is in phase 3 trials for Stargardt disease and geographic atrophy, targeting toxic vitamin A byproducts to slow disease progression.

• Phase 2 Stargardt trial showed significant slowing of lesion growth and strong safety, leading to phase 3 trials (Dragon and Dragon 2), with Dragon's interim analysis indicating promising efficacy and safety.

• Dragon 2 leverages Japan's SAKIGAKE designation for expedited regulatory access, aiming for first oral ophthalmology approval in Japan.

• Phoenix phase 3 trial for geographic atrophy is ongoing, with enrollment expected to close in summer; trial design mirrors Stargardt studies.

• Tinlarebant's mechanism reduces vitamin A delivery to the eye, addressing early-stage disease where no approved oral therapies exist.

Regulatory and financial outlook

• Multiple orphan, fast track, and rare pediatric disease designations secured in the US, EU, and Japan, with strong patent protection through at least 2040.

• Interim Dragon trial data prompted DSMB to recommend regulatory submission, suggesting strong efficacy; global regulatory engagement is underway.

• No additional capital needed to complete current phase 3 trials; four-year cash runway supports all ongoing studies except a second confirmatory GA trial.

• Approval for Stargardt disease expected in 2026 or 2027, with premium pricing anticipated until GA approval; profitability projected in both base and best-case scenarios.

• Focus remains on execution and expedited approval, with ongoing discussions with regulatory authorities in the US, Japan, China, Switzerland, Australia, and Europe.

Safety, efficacy, and differentiation

• Tinlarebant is oral, not intravitreal, and does not cross the blood-retinal barrier; its effect is systemic, reducing vitamin A delivery to the eye.

• Safety profile is strong, with less than 10% withdrawal and under 4% due to ocular adverse events; main side effects are mild, transient visual disturbances and occasional headache.

• Visual acuity stabilized over two years in Stargardt patients, with slowed lesion growth compared to natural history data.

• Drug targets early-stage disease, unlike current GA therapies that address late-stage inflammation; no approved treatments exist for Stargardt disease.

• Same 5 mg dose effective in both adolescent and older GA patients, supporting broad applicability.