On December 1, Belite Bio announced that its core drug Tinlarebant met its primary endpoint in the pivotal Phase 3 DRAGON trial for the treatment of Stargardt disease type 1 (STGD1).

The DRAGON trial is a randomized, double-blind, placebo-controlled global study designed to evaluate the safety and efficacy of Tinlarebant in adolescent patients with Stargardt disease.

The trial recruited 104 participants from 11 countries and regions, including the United States, the United Kingdom, Germany, France, Belgium, Switzerland, the Netherlands, mainland China, Hong Kong, Taiwan, and Australia. Participants were randomly assigned to the Tinlarebant group and the placebo group in a 2:1 ratio.

The results showed that, compared with placebo, Tinlarebant reduced the lesion growth rate by 36%, meeting the study's primary endpoint.

After using the pre-defined analysis method, the results were statistically significant (p=0.0033). Considering the progressive nature of STGD1, further post-hoc analysis provided specific data relevance, showing that the treatment effect remained significant (p<0.0001).

Regarding safety, Belite stated that most adverse events were mild and resolved during the study. Four patients discontinued treatment for treatment-related reasons. The most common drug-related ocular adverse events were xanthopsia (a color vision deficiency) and delayed dark adaptation. The most common treatment-related non-ocular adverse event was headache.

Stargardt's disease, also known as Stargardt's macular dystrophy, juvenile macular degeneration, or macular fundus, is a common cause of inherited retinal diseases. It is characterized by progressive atrophy of the macula, resulting in lesions in the eye and causing central vision loss.

Currently, there are no approved treatments worldwide, and standard treatment focuses on support and management.

Tinlarebant is an oral retinol-binding protein 4 (RBP4) antagonist designed to treat retinal diseases by reducing the accumulation of toxic vitamin A byproducts in the eye. It is the first candidate drug to demonstrate clinical efficacy in a global Phase 3 clinical trial for Stargardt disease.

Belite Bio plans to submit an NDA to the U.S. FDA in the first half of 2026.

Following the release of the data, Belite Bio's stock price surged 12%, bringing its total market capitalization to approximately $5.4 billion, reflecting market expectations that it would become the first company in the United States to launch an STGD1 therapy.

In mainland China, three hospitals participated in the DRAGON study. Currently, the NMPA has included Tinlarebant in its priority review list for the treatment of Stargardt's disease, a hereditary age-related macular degeneration in adolescents.

According to publicly available information, Belite Bio is a subsidiary of Lin Bioscience International Ltd (Taiwan, China), which focuses on developing innovative therapies for retinal degenerative and metabolic diseases.

In addition to Tinlarebant, the company also has a candidate drug, LBS-009, which is in the preclinical development stage. It is also an oral therapy that targets RBP4 and is mainly for type 2 diabetes and metabolic-related fatty liver disease.

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