Shares of Belite Bio rose toward the upper end of analyst estimates after the company presented clinical and regulatory progress on tinlarebant at the H.C. Wainwright 28th Annual Global Investment Conference on Tuesday, Sept. 15, 2026.
Tinlarebant, an oral systemic therapy for Stargardt disease, is nearing a PDUFA decision target of February 2025, the company said. Analysts at H.C. Wainwright who cover the stock set a price-target range of $185 to $303.
The presentation centered on results from the DRAGON trial, which enrolled 104 patients across the U.S., U.K., Europe, China, Taiwan and Australia. The trial demonstrated a 35.7% reduction in daily difference-adjusted fundus autofluorescence (DDAF) lesion growth compared with placebo over 24 months. Felllow-eye analysis showed about a 33% reduction in DDAF growth, reaching statistical significance.
Placebo DDAF growth in the DRAGON trial was 0.59 square millimeters per year, lower than the 0.74 square millimeters per year observed in natural-history data from the ProgStar study, indicating a potential floor effect in the control group.
Quantitative autofluorescence measurements at month 24 remained stable to slightly reduced in the tinlarebant arm, while the placebo group showed about 20% growth over the same period. Best-corrected visual acuity was essentially unchanged in both groups over two years.
Dr. Hendrik Scholl, Belite Bio’s chief medical officer, said no subgroup differences were found by age, race, sex or geography, allowing the results to be generalized across the Stargardt patient population. Patient retention was strong — only four dropouts occurred among 104 patients over 24 months, with compliance rates above 90%.
Scholl noted the drug’s half-life of approximately four days provides a buffer against missed doses. Safety findings included delayed dark adaptation and temporary visual scene discoloration, such as yellowish or bluish tints lasting seconds to minutes during light adaptation.
Hao-Yuan Chuang, Belite Bio’s chief financial officer, said the company estimates roughly 20,000 patients are clinically diagnosed with Stargardt disease in the U.S., with about half — some 10,000 — having genetic confirmation.
Belite Bio reported $780 million in cash, cash equivalents and U.S. Treasury bills at the end of the last quarter, supporting an expected three-year operating spend of approximately $450 million. The company holds a current ratio above 33.
Belite Bio shares traded at $172.61 following a 10% pullback over the preceding week. The stock has gained 159% over the past year, giving the company a market capitalization of $6.97 billion.
