Belite Bio (BLTE) investor relations material

Mechanism of action and therapeutic rationale

• Tinlarebant targets RBP4 to reduce vitamin A in photoreceptors, limiting toxic bisretinoid accumulation in the retina, which drives disease progression in Stargardt and geographic atrophy.

• The approach allows selective reduction of vitamin A in the eye without systemic safety concerns, leveraging the unique expression of the RBP receptor in ocular tissue.

• Tinlarebant is not a visual cycle modulator but reduces the substrate for toxic deposit formation, aiming for a therapeutic effect without disrupting normal vision processes.

Clinical trial data and regulatory pathway

• The DRAGON trial used DDAF lesion growth as the primary endpoint, showing a significant reduction in progression rate with tinlarebant (0.38 vs. 0.59 mm²/year, p<0.0001).

• Key secondary endpoints, such as decreased autofluorescence, also showed statistically significant treatment effects.

• Safety profile was favorable, with few serious adverse events and none related to treatment; most ocular adverse events were mild and resolved on treatment.

• The DRAGON II trial is designed primarily for Japanese regulatory submission but could support FDA approval if needed.

Market access, patient population, and commercial readiness

• Estimated 49,000–57,000 Stargardt patients in the U.S., with efforts underway to increase genetic confirmation rates for diagnosis and reimbursement.

• Initiatives include education for physicians and patients, leveraging existing genetic testing programs, and preparing for a commercial launch event.

• Commercialization budget is $300 million for the U.S. launch, with $800 million in cash reserves and a projected total burn rate of $450–$500 million over three years.

• Potential eligibility for a rare pediatric disease priority review voucher (PRV) could add $100–$200 million in proceeds, not included in current financial planning.