Dive Brief:

• An experimental drug from Belite Bio succeeded in a Phase 3 trial in the most common form of Stargardt disease, positioning the company to seek regulatory approval next year of what could be the first marketed medicine for the condition.
• According to Belite, treatment with its drug, known as tinlarebant, was associated with a roughly 36% reduction in the growth rate of retinal lesions compared to a placebo over the course of two years, meeting the trial’s main goal. Both study groups had a minimal overall change in visual acuity, but Belite said that finding was “consistent” with historical data.
• Belite said tinlarebant was “well tolerated,” with only four patients stopping treatment due to adverse events. The most common eye side effects related to treatment were a type of color vision deficiency and issues seeing at night or adjusting to a dark environment. The majority of those cases were mild, and most resolved during the trial, the company said.

Dive Insight:

Though it raised only $36 million in an initial public offering three years ago, Belite has quietly been one of the biopharmaceutical sector’s top performers ever since, according to BioPharma Dive data. Company shares, which debuted at $6 apiece, opened Monday trading at nearly $140. Belite is currently valued at around $5 billion.

The reason is tinlarebant, an oral drug with the chance to become the first marketed medicine for Stargardt. In Stargardt, a genetic defect changes how the body processes the vitamin A used to make retinal cells. That defect causes yellowish clumps to form in the eyes, destroying cells and triggering progressive vision loss. There are no available treatments for the roughly 50,000 people in the U.S. Belite estimates to have the disease.

Tinlarebant is designed to reduce the accumulation of toxic vitamin A byproducts in the eye. The drug was first studied at Columbia University over a decade ago, and then put up for bid in a National Institutes of Health program in 2016. Belite’s CEO and chairman, Tom Lin, licensed it and formed Belite around the program two years later. Belite has since brought tinlarebant into late-stage testing in Stargardt as well as geographic atrophy, a more common type of vision loss.

Belite’s value had already doubled this year in anticipation of the results in Stargardt. The company also received a positive recommendation from trial monitors that the 104-patient study continue without modifications, and noted that China’s drugs regulator had begun reviewing an application based on interim results showing statistical significance on the study’s main measure. The likelihood of success was “fairly high” given those updates, Leerink Partners analyst Marc Goodman wrote last month. Goodman has forecast anywhere from $1.4 billion to $3.6 billion in peak annual sales, depending on the drug’s price.

With the positive results, Belite intends to discuss an approval application with regulatory authorities in the U.S. and elsewhere in first half of 2026. It could face competition soon, however. Alkeus Pharmaceuticals, a privately held company, is preparing a submission for a new drug that’s similarly designed to reduce the buildup of harmful vitamin A byproducts in the eye. Ocugen, AAVantgarde, Splice Bio and others are advancing genetic medicines for the condition, too.

Stargardt experts believe gene therapy will eventually “be the preferred treatment approach” for the condition, wrote Mizuho Securities’ Graig Suvannavejh. But for now, the lack of other available therapies should mean “enthusiastic market uptake” for a drug proven to slow disease progression, he wrote in November.

Belite shares climbed by double digits before reversing course and dipping a few percentage points early Monday.