Belite Bio Presented 18-Month Interim Data from a 24-Month Phase 2 Study of Tinlarebant in Adolescent Stargardt Disease at the 2023 ARVO Meeting Tinlarebant (aka LBS-008) continues to be safe and well tolerated in adoles

Belite Bio Presented 18-Month Interim Data from

a 24-Month Phase 2 Study of Tinlarebant in Adolescent Stargardt Disease at the 2023 ARVO Meeting

SAN DIEGO, Calif., April 25, 2023- Belite Bio, Inc (NASDAQ:

BLTE), a San Diego based clinical stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting

retinal degenerative eye diseases which have significant unmet medical needs, today presented 18-month data from its ongoing two-year,

open-label Phase 2 clinical study of Tinlarebant in adolescent STGD1 patients ("LBS-008-CT02") as part of the poster presentation

at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting (Presentation: April 25th 8:45 am -

Professor John Grigg, the study's Principal Investigator and

Head of Specialty Clinical Ophthalmology at the University of Sydney and Consultant Ophthalmologist at the Sydney Children's Hospitals

Network at Westmead and Sydney Eye Hospital was the presenter of this interim study data. "It is great to observe that the 18-month

interim data showed a consistent safety profile in patients treated with Tinlarebant." said Dr. John Grigg. "Belite Bio's

current Phase 2 data also continued to demonstrate a promising trend toward slowing the disease progression in the study cohort."

To date, twelve patients*

have completed 18 months of treatment in the ongoing two-year Phase 2 study of Tinlarebant. Routine assessments were performed to evaluate

safety and tolerability of Tinlarebant. Retinal imaging data have been collected for the evaluation of disease progression in all subjects.

At Month 18, data from fundus autofluorescence (FAF) imaging revealed that nearly 60% of subjects (seven out of 12) showed no incident

atrophic retinal lesions. Mean visual acuity was stabilized in the study cohort throughout the 18-month treatment period. Nine of 12

patients experienced mild xanthopsia/chromatopsia and delayed dark adaptation and one of 12 patients experienced night vision impairment,

all of which were mild in severity and well-tolerated. A copy of the poster is available here (LINK).

* Our Phase 2 clinical

trial of Tinlarebant in adolescent STGD1 initially enrolled 13 subjects in Australia and Taiwan. Among the 13 subjects, one subject in

Australia was lost to follow up and therefore 12 subjects with complete 18-month data were evaluated.

"It is important to note that when compared with ProgStar study

participants with only questionably decreased autofluorescence lesions at baseline, LBS-008-CT02 subjects exhibited a smaller increase

(0.2 0.1 mm2) in definitely decreased autofluorescence (DDAF) lesion size compared to ProgStar subjects (0.4

0.3 mm2) after 18 months on study." said Dr. John Grigg.

"We are very encouraged by the 18-month treatment results from

our Phase 2 study as a majority of the subjects showed no transition to atrophic (DDAF) lesions, and in those subjects with transition

to DDAF lesions, the DDAF lesion progression rate was slowed compared to a study of the natural history of disease." said Dr. Nathan

L. Mata, Chief Scientific Officer of Belite Bio. "The Phase 2 data presented at ARVO continue to reinforce that this investigational

therapy could be a promising oral treatment for STGD1 patients."

Belite Bio is currently conducting a two-year Phase 2 study and is

enrolling patients in a two-year Phase 3 study (DRAGON) of Tinlarebant in adolescent STGD1 subjects and plans to begin enrolling patients

in a two-year Phase 3 study (PHOENIX) of Tinlarebant in Geographic Atrophy (GA) in mid 2023. Belite Bio expects the next data readout

in the Phase 2 STGD1 study to occur during the fourth quarter of 2023 when all subjects are expected to complete two years of treatment.

The two-year Phase 3 study (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 is designed to evaluate the safety and efficacy of Tinlarebant in adolescent STGD1

patients. Approximately 90 patients are targeted for enrollment in this study with a 2:1 randomization (active:placebo). To date, Belite

Bio has commenced this Phase 3 study in the U.S., the United Kingdom, Germany, Netherlands, France, Belgium, Switzerland, China, Hong

Kong, Taiwan, and Australia. For more information, visit clinicaltrials.gov at https://www.clinicaltrials.gov/ct2/show/NCT05244304)

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.

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).

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.

Belite Bio is a clinical-stage biopharmaceutical drug development

company focused on advancing novel therapeutics targeting retinal degenerative eye diseases which have significant unmet medical needs,

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Important Cautions Regarding Forward Looking Statements

This press release contains forward-looking statements, about future

expectations, plans and prospectus, as well as any other statements regarding matters that are not historical facts. These statements

include but are not limited to statements regarding the potential implications of clinical data for patients, clinical development, regulatory

milestones, and commercialization of its product candidates, and any other statements containing the words "expect," "will,"

"target," and other similar expressions. 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, 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