Belite Bio has achieved a critical milestone in its development of Tinlarebant, completing enrollment of 500 subjects in the pivotal Phase 3 PHOENIX trial evaluating the oral therapy for geographic atrophy (GA) in dry age-related macular degeneration (AMD). The global study spans sites across the United States, United Kingdom, France, Czech Republic, Switzerland, China, Taiwan, and Australia.
Addressing Critical Unmet Medical Need
Geographic atrophy represents the advanced stage of dry AMD, a leading cause of vision loss in older adults. Patients with GA develop retinal lesions that progressively expand, causing central vision loss and impairing daily activities such as reading and driving. Currently, no FDA-approved oral treatments exist for GA, and no approved therapies are available for earlier stages of dry AMD, including intermediate AMD.
"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," said Dr. Tom Lin, Chairman and CEO of Belite Bio. "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."
Trial Design and Global Participation
The PHOENIX study is structured as a 24-month, randomized, double-masked, placebo-controlled, multicenter trial designed to evaluate Tinlarebant's safety, tolerability, and potential to reduce atrophic lesion growth rate in GA patients. Dr. Hendrik Scholl, Chief Medical Officer of Belite Bio, noted that "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."
Dr. David Rhee, Principal Investigator at one of the top recruiting sites, emphasized the clinical significance: "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."
Regulatory Recognition and Development Pipeline
Tinlarebant has garnered substantial regulatory support, receiving Rare Pediatric Disease designation, Fast Track Designation, and Breakthrough Therapy Designation in the United States. The compound also holds Orphan Drug Designation in the United States, Europe, and Japan, along with Sakigake (Pioneer Drug) Designation in Japan for Stargardt disease type 1 (STGD1).
The oral therapy is intended as an early intervention for maintaining retinal tissue health and integrity in both STGD1 and GA patients. Beyond PHOENIX, Tinlarebant is being evaluated in the Phase 3 DRAGON study and Phase 2/3 DRAGON II study in adolescent STGD1 subjects.
Clinical Impact and Future Outlook
If approved, Tinlarebant would represent the first oral therapeutic option for GA patients and the first FDA-approved treatment for STGD1. The once-daily tablet is designed to reduce the accumulation of toxins in the eye, offering a non-invasive alternative to current treatment approaches.
Belite Bio plans to share interim results at the midpoint of the PHOENIX trial, with the company positioning itself to address significant unmet medical needs in degenerative retinal diseases. The completion of enrollment represents a crucial step toward potentially providing new treatment options for patients facing progressive vision loss from these debilitating conditions.
