Phase 3 PHOENIX trial for oral GA treatment completes enrollment

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Belite Bio, Inc. has completed enrollment in the PHOENIX trial. The PHOENIX study is a 24-month, randomized, double-masked, placebo-controlled, multicenter, pivotal Phase 3 trial for this oral medication. This trial will evaluate the safety and tolerability of Tinlarebant and its potential to reduce atrophic lesion growth rate in patients diagnosed with geographic atrophy (GA) in dry age-related macular degeneration (AMD).¹

The study is ongoing across sites in the United States, the United Kingdom, France, Czech Republic, Switzerland, China, Taiwan, and Australia.¹

Dr. Tom Lin, Chairman and CEO of Belite Bio, is quoted in the company’s press release¹ on this news, saying, “Completing enrollment in the PHOENIX trial marks an important milestone for Belite Bio as we advance our lead candidate, Tinlarebant, for the treatment of geographic atrophy. This achievement brings us one step closer to evaluating the potential of Tinlarebant to slow atrophic lesion growth in this serious and progressive disease for which there are no approved oral treatments. There remains a significant unmet need for this patient population. We remain committed to advancing Tinlarebant through late-stage development and look forward to sharing interim results at the midpoint of the PHOENIX trial.”

Similarly, Dr. Hendrik Scholl, Chief Medical Officer of Belite Bio also celebrated the global aspect of this trial and the potential it has to advance GA treatment options. He said, “We are pleased to announce the completion of enrollment in our pivotal clinical trial in geographic atrophy, a milestone that reflects strong global interest in our investigational therapy. The United States led enrollment by a significant margin, underscoring the appeal of a convenient, once-daily oral tablet compared to existing invasive treatment options.”¹

“Geographic atrophy is a progressive and debilitating condition that can severely impact a person’s vision and therefore quality of life, yet treatment options remain limited,” said Dr. David Rhee, Principal Investigator at one of the top recruiting sites for the PHOENIX trial. “The PHOENIX study is designed to evaluate the efficacy of Tinlarebant in slowing the growth of atrophic lesions, a key clinical marker of disease progression. Completing enrollment is a meaningful step in advancing research for this patient population, and I look forward to seeing the results from this important study.”¹

Previously, Ophthalmology Times reported on the DRAGON trial including that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for the company’s treatment for Stargardt disease (STDG1), Tinlarebant.²

About Tinlarebant

Tinlarebant is an orally administered, once-a-day tablet intended as an early intervention for maintaining the health and integrity of retinal tissues in Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) patients. Currently, there are no FDA approved treatments for STGD1 and no approved orally administered treatments for GA. Therefore, if approved, Tinlarebant would be a novel oral therapeutic addressing an unmet medical need in both STGD1 and GA. Tinlarebant has been granted Rare Pediatric Disease designation, Fast Track Designation, and Breakthrough Therapy Designation in the United States as well as Orphan Drug Designation in the United States, Europe, and Japan. It has also received Sakigake (Pioneer Drug) Designation in Japan for STGD1.¹

References:

1. Belite Bio Announces Completion of Enrollment in the Pivotal Global Phase 3 PHOENIX Trial Evaluating Oral Tinlarebant in Geographic Atrophy. Belite Bio, Inc. July 2, 2025. Accessed July 7, 2025. https://investors.belitebio.com/news-releases/news-release-details/belite-bio-announces-completion-enrollment-pivotal-global-phase

2. Filkins, K. FDA grants Breakthrough Therapy Designation for Tinlarebant. Ophthalmology Times. May 21, 2025. Accessed July 7, 2025. https://www.ophthalmologytimes.com/view/fda-grants-breakthrough-therapy-designation-for-tinlarebant