Belite Bio submits application to expand PHOENIX phase 3 clinical trial to Taiwan

Tinlarebant, a novel oral therapy, is intended to reduce the accumulation of toxins in the eye that cause STGD1 and contribute to geographic atrophy (GA). Image credit: Nina Lawrenson/peopleimages.com – stock.adobe.com

Belite Bio, Inc has submitted an application to the Taiwan Food and Drug Administration to initiate a Phase 3 clinical trial of Tinlarebant in GA (the PHOENIX study) in Taiwan. This application joins those submitted by the company in Australia and South Korea, the latter of which has received approval to initiate.

About PHOENIX Study

The PHOENIX study is a phase 3, multicenter, double-masked, placebo-controlled, randomized, fixed-dose clinical study designed to evaluate the efficacy and safety of tinlarebant in patients with GA associated with dry AMD. Approximately 430 subjects are targeted for enrollment in this study with a 2:1 randomization (tinlarebant:placebo).

About Tinlarebant (a/k/a LBS-008)

Tinlarebant is a novel oral therapy which is intended to reduce the accumulation of toxins in the eye that cause STGD1 and contribute to GA, or advanced Dry AMD. These toxins are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of these toxins. Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease designation in the U.S., and Orphan Drug Designation in the U.S. and Europe for the treatment of STGD1.

Stargardt Disease (STGD1)

STGD1 is the most common inherited retinal dystrophy (causing blurring or loss of central vision) in both adults and children. The disease is caused by mutations in a retina-specific gene (ABCA4) which results in massive accumulation of toxic vitamin A byproducts (known as “bisretinoids”) in the retina leading to retinal cell death and progressive loss of central vision. The fluorescent properties of bisretinoids and the development of retinal imaging systems have helped ophthalmologists identify and monitor disease progression. Currently, there are no FDA approved treatments for STGD1.

Importantly, STGD1 and GA, or advanced Dry AMD, share a similar pathophysiology which is characterized by the excessive accumulation of cytotoxic bisretinoids, retinal cell death, and loss of vision. Vision loss occurs slowly, despite peripheral expansion of “dead retina”, until the disease reaches the center of the eye (the macula). Therefore, Belite Bio intends to evaluate safety and efficacy of Tinlarebant in GA patients in its Phase 3 study (PHOENIX).

GA in advanced Dry Age-related Macular Degeneration (Dry AMD)

Dry AMD is a leading cause of vision loss in older adults. Geographic Atrophy, or GA, is the advanced stage of AMD. Currently, there are no FDA approved orally administered treatments for GA and no FDA approved therapies for the other stages of Dry AMD other than GA. There are an estimated 20 million AMD patients in the U.S. and over 196 million patients worldwide with an estimated global direct healthcare cost of US$255 billion.

Reference:

Belite Bio Announces TFDA Submission for Tinlarebant Phase 3 GA Clinical Trial in Taiwan. Belite Bio, Inc. July 6, 2023. Accessed July 6, 2023. https://investors.belitebio.com/news-releases/news-release-details/belite-bio-announces-tfda-submission-tinlarebant-phase-3-ga