Belite Bio (BLTE) Morgan Stanley 24th Annual Global Healthcare Conference summary

Pipeline and regulatory path

• Tinlarebant’s Stargardt phase III DRAGON study is complete; the FDA accepted the NDA for priority review, with a Feb. 12 PDUFA date. DRAGON II is running in parallel, while GA phase III PHOENIX is fully enrolled and ongoing.

• FDA correspondence leaves single-study approval as a possibility but a review issue; the NDA includes confirmatory evidence such as ProgStar natural-history data.

• DRAGON II is a Japan-focused phase Ib/PK/PD/phase III trial, fully enrolled since January with 73 patients, including 15 in Japan; it is not expected to support the U.S. NDA.

• A Japan NDA based essentially on DRAGON I data was submitted; the PMDA is reviewing the package with the goal of making Japan the first country to approve tinlarebant.

Stargardt disease and patient care

• Estimated U.S. prevalence is 47,000–59,000; 20,000 patients are clinically diagnosed, and more than half of those have at least one detected ABCA4 mutation.

• Disease typically begins in childhood or early adulthood and worsens over time, often leading to legal blindness; precise diagnosis is achievable with retinal imaging, with specialist referral expected for treatment.

• Commercial preparation will focus on IRD and retinal specialists, broader disease awareness among ophthalmologists and low-vision optometrists, genetic testing or referral, and patient advocacy outreach.

Mechanism and clinical evidence

• ABCA4 dysfunction drives toxic bisretinoid and A2E accumulation; tinlarebant targets RBP4/STRA6 to reduce vitamin A influx into the eye, lowering visual-cycle substrate and bisretinoid accumulation.

• In DRAGON, tinlarebant slowed DDAF lesion progression by 36% vs placebo over two years; fellow-eye lesion growth was slowed by 33%, also statistically significant.

• DDAF is an FDA-recognized approvable endpoint; ProgStar found visual-acuity loss of 0.55 letters/year, while acuity testing has 8-letter intersession variability, limiting detection of change in a two-year trial.