Belite Bio, Inc. announced patient enrollment has concluded for its upcoming phase 3 pivotal trial evaluating the potential of tinlarebant to treat geographic atrophy (GA) in dry age-related macular degeneration (AMD).
I’m not familiar with this company …
A quick rundown: Based in San Diego, California, the clinical-stage biopharmaceutical drug development company is developing novel therapeutics intended for the treatment of degenerative retinal diseases with unmet needs.
- Those diseases: Include GA in advanced dry AMD; Stargardt disease type 1 (STGD1); and metabolic diseases (non-fatty liver disease and type 2 diabetes)
And its pipeline?
Belite’s investigational therapies are based on a retinol binding protein 4 (RBP4) intellectual property portfolio.
- Refresh: RBP4 is the primary protein responsible for carrying Vitamin A (retinol) in the bloodstream—and is critical for maintaining adequate Vitamin A levels in the retina to ensure healthy vision.
As for the company’s lead asset: That would be the aforementioned tinlarebant.
Tell me about it.
As a once-daily, orally-administered tablet, tinlarebant is under clinical investigation for early intervention in maintaining retinal tissue health among GA and STGD1 patients.
- How it works: The oral therapy is intended to reduce toxin accumulation within the eye that causes STGD1 and contributes to GA.
Its regulatory status: Though still investigational, the tablet has already received several FDA designations for its SGT1 indication:
- Breakthrough Therapy (in May 2025)
- Fast Track
- Orphan Drug
- Rare Pediatric Disease
Gotcha. So what has tinlarebant’s clinical performance looked like so far?
On the STGD1 front: The therapeutic has undergone clinical evaluation via:
A phase 1b/2 trial (NCT05266014):
- Phase 1: Identified an optimal dosage of tinlarebant among adolescent patients
- Phase 2: Extended study to examine the safety and efficacy of tinlarebant among patients
The findings thus far:
- Tinlarebant resulted in visual acuity (VA) stabilization, particularly among patients with significant vision loss prior to study enrollment
- 75% of patients with atrophic lesion involvement within the macula at baseline saw growth halted in the second year
- See here for more details
Any other ongoing trials?
Still sticking with this STGD1 indication … the phase 3 DRAGON trial (NCT05244304) is evaluating the safety and efficacy of tinlarebant versus a placebo (randomized 2:1) among STGD1 patients aged 12 to 20 over a 24-month period.
And a separate phase 1b and phase 2/3 trial (dubbed DRAGON II; NCT05949593) is also underway.
Now to this phase 3 GA trial.
Its purpose: To evaluate the efficacy of tinlarebant in slowing the growth of atrophic lesions—a notable key clinical marker for GA progression.
With the study being conducted at clinical sites across the United States, United Kingdom, France, the Czech Republic, Switzerland, China, Taiwan, and Australia, here are a few details to know:
- The design: Multicenter, double-masked, parallel-group, placebo-controlled, randomized, and fixed-dose
- The participants: Estimated 429 patients (aged 60 to 85) diagnosed with GA with atrophic lesions in one or both eyes
- The setup: Patients randomized 2:1 to receive either tinlarebant (5 mg) or a placebo administered orally once daily from baseline (Day 1) to the final day of Month 24
And what’s being measured?
Measured from baseline to Month 24:
- Primary endpoint: Rate of change (Growth rate slope) in GA lesion size
- Secondary endpoints:
- Change in best-corrected visual acuity (BCVA) via Early Treatment Diabetic Retinopathy (ETDRS) scale
- Changes in area and size of the inner/outer segment junction of photoreceptors by spectral-domain optical coherence tomography (SD-OCT)
So … when can we expect data to be released?
Per Clinical Trials, the study is estimated to conclude in November 2027 (with a primary completion by August 2027).
Stay tuned in the meantime for interim findings (not to mention data from those aforementioned SDTG1-targeted DRAGON trials).
We remain committed to advancing Tinlarebant through late-stage development and look forward to sharing interim results at the midpoint of the PHOENIX trial.”
And the big-picture perspective on this?
As David Rhee, MD, principal investigator of the PHOENIX trial, noted: “(GA) is a progressive and debilitating condition that can severely impact a person’s vision and therefore quality of life, yet treatment options remain limited.”
Case in point: There’s currently an unmet need for both AMD and GA patient bases, with:
- No approved orally administered treatments for GA
- No approved therapies for early stages of dry AMD (such as intermediate AMD)