Full Press Release Details
Belite Bio Presented 12-Month Interim Results
of LBS-008 Phase 1b/2 Study in Adolescent Stargardt Disease at AAO Annual Meeting 2022
SAN DIEGO, October 1, 2022- Belite Bio, Inc (NASDAQ: BLTE),
a San Diego based clinical stage biopharmaceutical drug development company targeting currently untreatable eye diseases, today presented
one-year data from their ongoing two-year Phase 2 clinical study of Tinlarebant in STGD1 as part of the oral presentation series at the
Annual Meeting of the American Academy of Ophthalmology (AAO) held during September 30 - October 3, 2022 at McCormick Place, Chicago.
"We are glad that Tinlarebant's Phase 2 results were presented
in a late-breaking oral session at the AAO annual meeting." said Dr. Tom Lin, Belite Bio's Chairman and CEO. "The Phase
2 data presented at AAO continue to support Tinlarebant's safety and efficacy profile over the one-year treatment period and reinforce
that this investigational therapy is a promising oral treatment for STGD1 patients."
Professor John Grigg, the study's principal investigator and
Head Specialty of Ophthalmology at the University of Sydney and Consultant Ophthalmologist at the Sydney Children's Hospitals Network
at Westmead and Sydney Eye Hospital provided a presentation of the interim study data.
To date, all 13 patients have completed one-year of treatment in the
ongoing two-year Phase 2 study of Tinlarebant. The results for safety and tolerability assessments, and retinal imaging data have been
collected for the evaluation of disease progression. Images from spectral-domain optical coherence tomography imaging, an imaging modality
that permits visualization of the retinal anatomy, have shown a stabilization of retinal thickness in many subjects. Fundus autofluorescence
imaging shows no autofluorescence expansion (QDAF) in 7 of 13 (53.8%) subjects, and 12 of 13 (92.3%) subjects also show no atrophic lesion
(DDAF) after one year of treatment. More importantly, 9 of 13 (69.2%) subjects show a stabilization or improvement in visual acuity throughout
the one-year treatment period. A copy of the presentation slides is available at https://investors.belitebio.com/aao-presentation-download-form.
According to the international prospective study of STGD1 (the ProgStar
Study), childhood-onset patients with no atrophic lesion (DDAF) at baseline experienced an average lesion growth rate of 0.66 mm2
for the left eyes and 0.74 mm2 for the right eyes at one year. In addition, the Prospective Cohort Study of Childhood-Onset
Stargardt Disease by Georgiou et al. reported an average atrophic lesion growth rate (DDAF) of 0.69 mm2/year for children.
Belite Bio's one-year data from ongoing Phase 2 study showed an average lesion growth rate of 0.03 mm2/year, demonstrating
a promising trend toward halting or slowing the disease progression in the study cohort.
"We are very encouraged by the 12-month treatment results from
our Phase 2 Study. While the natural progression of childhood-onset STGD1 is characterized by a rapid visual decline and fast disease
progression leading to permanent visual loss at a very young age, the Phase 2 interim data have shown the stabilization in several structural
and functional parameters." said Dr. Tom Lin.
Belite Bio is currently conducting a two-year Phase 2 study and a two-year
Phase 3 study (DRAGON) of Tinlarebant in adolescent STGD1 subjects. Belite Bio expects the next data readout in its Phase 2 STGD1 study
to occur during the second quarter of 2023 when all subjects will complete 18 months of treatment.
The two-year Phase 3 study named DRAGON is a Multi-Center, Randomized,
Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of TinlaRebant in the Treatment of StArGardt
Disease in AdOlesceNt Subjects. DRAGON study is designed to evaluate the safety and efficacy of Tinlarebant in adolescent
STGD1 patients. To date, Belite Bio has commenced the Phase 3 study in the U.S., the United Kingdom, Germany, Belgium, Switzerland, Hong
Kong, Taiwan, mainland China and Australia. Approximately 60 patients are targeted for enrollment in this study with a 2:1 randomization
(active:placebo). For more information, visit clinicaltrials.gov at https://www.clinicaltrials.gov/ct2/show/NCT05244304?term=belite+bio&draw=2&rank=1)
Tinlarebant is a novel oral therapy that prevents the buildup of
toxins in the eye that cause STGD1 and contribute to 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), a carrier protein that transports retinol to the eye. By modulating the amount of retinol entering the
eye, Tinlarebant reduces the formation of toxins that have been implicated in STGD1 and dry AMD. 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.
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 a dysfunctional 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 have helped ophthalmologists identify and monitor disease progression. Importantly, STGD1 and 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).
Dry Age-related Macular Degeneration
Dry AMD is a leading cause of vision loss in the U.S. and has no approved
treatments available. There are an estimated 11 million dry AMD patients in the U.S. and over 196 million patients worldwide with an estimated
global direct healthcare cost of US$255 billion.
Belite Bio is a San Diego based clinical stage biopharmaceutical drug
development company targeting currently untreatable eye diseases, such as atrophic age-related macular degeneration (commonly known as
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Important Cautions Regarding Forward Looking Statements
This press release contains forward-looking statements, including
statements regarding the potential implications of clinical data for patients, and Belite Bio's advancement of, and anticipated
preclinical activities, clinical development, regulatory milestones, and commercialization of its product candidates. Actual results may
differ materially from those indicated in the forward-looking statements as a result of various important factors, including but not limited
to Belite Bio's ability to demonstrate the safety and efficacy of its drug candidates; the clinical results for its drug candidates,
which may not support further development or regulatory approval; the content and timing of decisions made by the relevant regulatory
authorities regarding regulatory approval of Belite Bio's drug candidates; the potential efficacy of Tinlarebant on the treatment
of Dry AMD, as well as those risks more fully discussed in the "Risk Factors" section in Belite Bio's filings with the
U.S. Securities and Exchange Commission. All forward-looking statements are based on information currently available to Belite Bio, and
Belite Bio undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information,
future events or otherwise, except as may be required by law.
Media and Investor Relations Contact:
Jennifer Wu /ir@belitebio.com
Tim McCarthy /tim@lifesciadvisors.com