- Following positive topline results from the pivotal, global Phase 3 DRAGON trial of tinlarebant in adolescents with Stargardt disease type 1 (STGD1), the Company is on track to submit a New Drug Application (NDA) to the
U.S. Food and Drug Administration (FDA) in Q2 2026 - Completed enrollment in the Phase 2/3
DRAGON II trial in STGD1 - Completed a
$402 million underwritten public offering of American Depositary Shares - Conference call and webcast on
Monday, March 2, 2026 , at4:30 p.m. ET
“2025 marked a defining year for Belite, highlighted by positive topline results from our pivotal Phase 3 DRAGON trial, establishing tinlarebant as a potential first-in-class therapy for Stargardt disease,” said Dr.
Full Year 2025 Business Highlights and Upcoming Milestones
Clinical Highlights
Tinlarebant is an oral, potent, once-daily, retinol binding protein 4 (RBP4) antagonist that decreases RBP4 levels in the blood and reduces vitamin A (retinol) delivery to the eye without disrupting systemic retinol delivery to other tissues. Vitamin A is critical for normal vision but can accumulate as toxic byproducts in individuals affected with STGD1 and geographic atrophy (GA), the advanced form of dry age-related macular degeneration (AMD), leading to retinal cell death and loss of vision.
Stargardt disease (STGD1): Accumulation of cytotoxic vitamin A byproducts (bisretinoids) compounds has been implicated in the onset and progression of STGD1, for which there is no approved treatment. Tinlarebant has been granted Breakthrough Therapy,
- DRAGON Trial: Completed, 24-month, 104 subjects, aged 12 to 20 years old, randomized (2:1, active: placebo), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in adolescent STGD1 patients
- Met its primary efficacy endpoint, demonstrating a statistically significant and clinically meaningful 35.7% reduction in the growth rate of macular lesions, measured as definitely decreased autofluorescence (DDAF) by fundus autofluorescence imaging, compared with placebo.
- Achieved statistical significance when applying the pre-specified analysis (p-value = 0.0033).
- Demonstrated in a post-hoc analysis that the treatment effect remained (35.4%) consistent with a p-value < 0.0001 when considering the progressive nature typically seen in STGD1.
- On track to submit an NDA to the FDA in the second quarter of 2026.
- DRAGON II Trial: Combination of a Phase 1b open-label trial to evaluate the pharmacokinetics and pharmacodynamics of tinlarebant in adolescent Japanese STGD1 patients and a Phase 2/3, 24-month, randomized (1:1, active: placebo), double-masked, placebo-controlled, multi-center trial in adolescent STGD1 patients aged 12 to 20 years old across
Japan , theU.S. , and theUnited Kingdom .- Targeted enrollment of 60 subjects was reached in
January 2026 . - Subjects who passed screening before enrollment cut off are allowed to be enrolled in the trial through early March. 72 subjects were enrolled as of
February 27, 2026 . - Trial design and inclusion of Japanese patients are intended to facilitate a future NDA in
Japan . - Primary efficacy endpoint is the growth rate of atrophic lesions; safety and tolerability will also be assessed.
- Targeted enrollment of 60 subjects was reached in
Geographic Atrophy (GA): GA is a chronic degenerative disease of the retina that leads to blindness in the elderly. Accumulation of bisretinoids has been implicated in the progression of GA. There are currently no FDA-approved, orally administered treatments for GA.
PHOENIX Trial: Ongoing, 24-month, randomized (2:1, active: placebo), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in GA patients- Completed enrollment with 530 subjects.
- Primary efficacy endpoint is the growth rate of atrophic lesions; safety and tolerability will also be assessed.
- The Company expects to conduct an interim analysis.
Corporate Highlights
- Completed a
$402 million underwritten public offering of American Depositary Shares with overallotment fully exercised by the underwriters, with net proceeds intended to support commercialization preparation, development and expansion of pipelines, and general corporate purposes.
Preliminary and Unaudited Fourth Quarter and Full Year 2025 Financial Results
Cash and Cash Equivalents: As of
Investments: As of
R&D Expenses:
For the three months ended
On a non-GAAP basis, excluding share-based compensation expenses, non-GAAP research and development expenses for the three months and year ended
Selling, G&A Expenses:
For the three months ended
On a non-GAAP basis, excluding share-based compensation expenses, non-GAAP selling, general and administrative expenses for the three months ended
Other Income:
For the three months ended
Net Loss:
For the three months ended
On a non-GAAP basis, excluding share-based compensation expenses, the Company reported a non-GAAP net loss of
Webcast Information
About
Important Cautions Regarding Forward Looking Statements
This press release contains forward-looking statements regarding future expectations, plans and prospectus, as well as 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, and Belite Bio’s advancement of, and anticipated preclinical activities, clinical development, regulatory milestones, and commercialization of its product candidates, the ability of tinlarebant to treat STGD1 and GA, the timing to complete relevant clinical trials and/or to receive the interim/final data of such clinical trials; the timing to submit trial data to regulatory authorities for drug approval, as well as any other statements regarding matters that are not historical facts, and any other statements containing the words “expect”, “believe”, “target”, “plan”, “hope” “potential” and other similar expressions. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors related to Belite Bio’s business, 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; expectations for the timing of initiation, enrollment and completion of, and data relating to, its clinical trials; the timing to complete any ancillary clinical trials and/or to receive the interim/final data of such clinical trials; the timing to communicate with and submit trial data to regulatory authorities for drug approval in various jurisdictions; the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approval of Belite Bio’s drug candidates; timing for
Discussion of Non-GAAP Financial Measures
To supplement the Company’s unaudited condensed consolidated financial results prepared in accordance with GAAP, the Company discloses certain non-GAAP financial measures that exclude share-based compensation, including research and development (non-GAAP), selling, general and administrative (non-GAAP), total operating expenses (non-GAAP), loss from operations (non-GAAP), net loss (non-GAAP), weighted average number of ordinary shares used in per share (non-GAAP) and net loss per ordinary share basic and diluted (non-GAAP).
The Company believes that these non-GAAP measures provide supplemental information that may be helpful in understanding period-to-period trends in operating expenses and results when considered together with, and not as a substitute for, the corresponding GAAP financial measures. These measures are intended to increase transparency into expense items that may vary from period to period for reasons such as the timing, structure, and valuation of equity awards. These measures are not intended to replace GAAP financial information and are not considered by management to be superior to GAAP measures.
At the Company’s current stage of development as a clinical-stage biotechnology company, the primary expenditures relate to the execution of clinical trials, regulatory activities (including preparation for potential NDA submissions), and the management of ongoing operations. In this context, management believes that the supplemental presentation of operating expenses excluding certain non-cash charges, such as share-based compensation, may assist users in understanding the nature and scale of cash-based operating activities by reducing period-to-period volatility from non-cash items. However, these non-GAAP measures are not intended to represent, and should not be viewed as, measures of liquidity, cash burn rate, or cash flows.
Non-GAAP measures have inherent limitations and may differ from similarly titled measures used by other companies. Accordingly, these measures should be viewed as supplemental and evaluated together with the Company’s GAAP results and the reconciliations to the most directly comparable GAAP measures presented in this release.
Explanation of Adjustment – Share-based compensation:
Share-based compensation expense consists of non-cash charges related to the fair value of equity awards awarded to employees and other non-employees. The amount recognized in any period may vary based on factors such as grant timing, award structure, and valuation assumptions, which may not be directly correlated with the timing or magnitude of cash payments related to the Company’s clinical, regulatory, and operational activities. The exclusion of share-based compensation in the Company’s non-GAAP measures is intended to supplementally illustrate operating expense trends and facilitate period-to-period comparisons of cash-based expenditures. The Company recognizes that share-based compensation is an important component of total compensation, and does not view non-GAAP measures as a replacement for GAAP results, which include the full impact of share-based compensation.
UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (Amounts in thousands of US Dollars, except share and per share amounts) | ||||||||||||||||
| For the Three Months | For the Year | |||||||||||||||
| Ended | Ended | |||||||||||||||
| 2024 | 2025 | 2024 | 2025 | |||||||||||||
| Expenses | ||||||||||||||||
| Research and development | 7,254 | 14,624 | 29,939 | 45,377 | ||||||||||||
| Selling, general and administrative | 4,203 | 13,453 | 10,057 | 38,831 | ||||||||||||
| Total operating expenses | 11,457 | 28,077 | 39,996 | 84,208 | ||||||||||||
| Loss from operations | (11,457 | ) | (28,077 | ) | (39,996 | ) | (84,208 | ) | ||||||||
| Other income: | ||||||||||||||||
| Total other income, net | 1,357 | 2,753 | 3,858 | 6,597 | ||||||||||||
| Loss before income tax | (10,100 | ) | (25,324 | ) | (36,138 | ) | (77,611 | ) | ||||||||
| Income tax expense | — | — | 6 | — | ||||||||||||
| Net loss | (10,100 | ) | (25,324 | ) | (36,144 | ) | (77,611 | ) | ||||||||
| Other comprehensive income (loss) | ||||||||||||||||
| Foreign currency translation adjustments, net of nil tax | (259 | ) | (104 | ) | (286 | ) | 124 | |||||||||
| Total comprehensive loss | (10,359 | ) | (25,428 | ) | (36,430 | ) | (77,487 | ) | ||||||||
| Weighted average number of ordinary shares used in per share | ||||||||||||||||
| calculation: | ||||||||||||||||
| - Basic and Diluted | 31,453,211 | 36,218,289 | 30,538,378 | 33,538,160 | ||||||||||||
| Net loss per ordinary share | ||||||||||||||||
| - Basic and Diluted | $ | (0.32 | ) | $ | (0.70 | ) | $ | (1.18 | ) | $ | (2.31 | ) | ||||
RECONCILIATION OF GAAP TO NON-GAAP UNAUDITED OPERATING RESULTS (Amounts in thousands of US Dollars, except share and per share amounts) | ||||||||||||||||
| For the Three Months | For the Year | |||||||||||||||
| Ended | Ended | |||||||||||||||
| 2024 | 2025 | 2024 | 2025 | |||||||||||||
| Expenses | ||||||||||||||||
| 7,254 | 14,624 | 29,939 | 45,377 | |||||||||||||
| Share-based compensation expense | (1,510 | ) | (2,452 | ) | (3,775 | ) | (9,147 | ) | ||||||||
| Non-GAAP research and development | 5,744 | 12,172 | 26,164 | 36,230 | ||||||||||||
| GAAP Selling, general and administrative | 4,203 | 13,453 | 10,057 | 38,831 | ||||||||||||
| Share-based compensation expense | (2,696 | ) | (9,263 | ) | (5,212 | ) | (29,772 | ) | ||||||||
| Non-GAAP selling, general and administrative | 1,507 | 4,190 | 4,845 | 9,059 | ||||||||||||
| GAAP Total operating expenses | 11,457 | 28,077 | 39,996 | 84,208 | ||||||||||||
| Share-based compensation expense | (4,206 | ) | (11,715 | ) | (8,987 | ) | (38,919 | ) | ||||||||
| Non-GAAP Total operating expense | 7,251 | 16,362 | 31,009 | 45,289 | ||||||||||||
| GAAP Loss from operations | (11,457 | ) | (28,077 | ) | (39,996 | ) | (84,208 | ) | ||||||||
| Share-based compensation expense | 4,206 | 11,715 | 8,987 | 38,919 | ||||||||||||
| Non-GAAP Loss from operations | (7,251 | ) | (16,362 | ) | (31,009 | ) | (45,289 | ) | ||||||||
| GAAP Net loss | (10,100 | ) | (25,324 | ) | (36,144 | ) | (77,611 | ) | ||||||||
| Share-based compensation expense | 4,206 | 11,715 | 8,987 | 38,919 | ||||||||||||
| Non-GAAP Net Loss | (5,894 | ) | (13,609 | ) | (27,157 | ) | (38,692 | ) | ||||||||
| Weighted average number of ordinary shares used in per share | ||||||||||||||||
| Calculation GAAP and Non-GAAP: | ||||||||||||||||
| - Basic and Diluted | 31,453,211 | 36,218,289 | 30,538,378 | 33,538,160 | ||||||||||||
| Net loss per ordinary share | ||||||||||||||||
| - Basic and Diluted GAAP | $ | (0.32 | ) | $ | (0.70 | ) | $ | (1.18 | ) | $ | (2.31 | ) | ||||
| - Basic and Diluted Non-GAAP | $ | (0.19 | ) | $ | (0.38 | ) | $ | (0.89 | ) | $ | (1.15 | ) | ||||
UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS (Amounts in thousands of US Dollars, except share amounts) | ||||||
| 2024 | 2025 | |||||
| Current assets | $ | 147,073 | $ | 494,272 | ||
| Other assets | 5,059 | 286,284 | ||||
| TOTAL ASSETS | $ | 152,132 | $ | 780,556 | ||
| TOTAL LIABILITIES | $ | 6,311 | $ | 10,070 | ||
| TOTAL SHAREHOLDERS’ EQUITY | 145,821 | 770,486 | ||||
| TOTAL LIABILITIES AND SHAREHOLDERS’ EQUITY | $ | 152,132 | $ | 780,556 | ||
| Ordinary shares authorized | 400,000,000 | 400,000,000 | ||||
| Ordinary shares issued | 31,857,802 | 39,353,365 | ||||
| Ordinary shares outstanding | 31,826,549 | 39,339,960 | ||||
Media and Investor Relations Contact:
Jennifer Wu
ir@belitebio.com
Julie Fallon
belite@argotpartners.com
