Belite Bio Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Corporate Update
- Positive results from Phase 2 trial of Tinlarebant in adolescent STGD1 subjects showing sustained lower atrophic lesion growth compared to ProgStar participants
- 42% of Tinlarebant-treated subjects did not develop atrophic retinal lesions during the 24-month treatment period
- Enrollment completed for pivotal global Phase 3 trial of Tinlarebant in STGD1 subjects with interim data expected in 4Q 2024
- Initiation of pivotal global Phase 3 trial of Tinlarebant in GA subjects and approval to start PHOENIX trial in eight countries
- Strong financial position with $88.2 million in cash as of December 31, 2023
- Completion of underwritten public offering and initiation of at-the-market offering program raising significant funds for the company
- None.
Insights
The Phase 2 trial data showing sustained lower atrophic lesion growth in Tinlarebant-treated subjects is a positive indicator for Belite Bio's drug efficacy. This could potentially lead to a competitive advantage in the market for treatments of STGD1 and GA, conditions currently lacking FDA-approved therapies. The company's financial strength, with $88.2 million in cash reserves, suggests adequate funding for ongoing trials and operations. However, the increased net loss year-over-year, from $12.6 million to $31.6 million, reflects the substantial investment in R&D required for the DRAGON and PHOENIX trials. Investors should monitor the burn rate and future financing needs, as well as the potential revenue streams upon successful drug approval and commercialization.
The Fast Track and Rare Pediatric Disease designations granted by the FDA for Tinlarebant indicate a recognition of the unmet medical need in STGD1 treatment and could expedite the review process. The 42% of subjects not developing atrophic retinal lesions over 24 months is significant, suggesting Tinlarebant's potential to alter the disease course. The upcoming interim data from the DRAGON trial will be pivotal in assessing the long-term viability and safety of the drug. Given the chronic nature of GA and the aging population, a successful oral treatment could significantly impact patient quality of life and reduce the burden on healthcare systems.
Belite Bio's strategic focus on degenerative retinal diseases taps into a niche yet growing market driven by an aging global population. The lack of approved oral treatments for GA and STGD1 positions Tinlarebant to potentially capture a significant market share. The company's proactive approach in securing global trial enrollment and regulatory designations across the U.S., Europe and Japan demonstrates an understanding of the importance of market diversification. The market will be closely watching the interim results of the DRAGON trial for indications of the drug's efficacy and safety profile, which will be critical in shaping investor sentiment and the company's strategic direction.
- Tinlarebant is Belite Bio’s orally administered tablet intended to slow disease progression in patients affected with Stargardt Disease (STGD1) and Geographic Atrophy (GA) in advanced Dry Age-related Macular Degeneration (Dry AMD)
- Data from a 24-month Phase 2 trial in adolescent STGD1 subjects showed a sustained lower atrophic lesion growth in Tinlarebant-treated subjects compared to ProgStar participants possessing similar baseline characteristics (aged ≤18 years) (p<0.001)
- In the Phase 2 trial,
42% of Tinlarebant-treated subjects (5 out of 12) did not develop atrophic retinal lesions during the 24-month treatment period - Enrollment of a pivotal global Phase 3 trial of Tinlarebant in adolescent STGD1 subjects (“DRAGON”) has been completed with 104 subjects across 11 countries
- Interim data from the “DRAGON” trial expected in 4Q 2024
- Dosed first subject in pivotal global Phase 3 trial of Tinalrebant in GA subjects (“PHOENIX”) and received approval to initiate PHOENIX trial in eight countries
- As of December 31, 2023, the Company had
$88.2 million in cash. - Conference Call and Webcast on Tuesday, March 12, 2024, at 4:30 p.m. ET
SAN DIEGO, March 11, 2024 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) (“Belite Bio” or the “Company”), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced its audited financial results for full-year 2023 and the unaudited and unreviewed financial results for the quarter ended December 31, 2023, and provided a business update.
“2023 was a productive year for Belite, from the initiation of our Phase III trial in GA, enrollment completion of our Phase III Stargardt disease trial, to the exciting 24-month data from our Phase II Stargardt disease trial,” said Dr. Tom Lin, Chairman and CEO of Belite Bio. “These milestones continue to underscore the therapeutic potential of Tinlarebant, not only in Stargardt disease, for which there is still no approved treatment, but for patients suffering from GA, for which an oral treatment would be game changing. As we enter 2024, we remain focused on our mission to develop oral therapies for eye diseases with significant unmet medical needs and are well positioned to execute with our strong balance sheet. We expect to share additional analysis from our Phase 2 Stargardt disease trial at the annual meeting of The Association for Research in Vision and Ophthalmology (ARVO) in May, and to receive one-year interim data from our Phase 3 DRAGON trial later this year. We also look forward to sharing additional enrollment updates on our Phase III PHOENIX trial in GA as the year progresses.”
Full Year 2023 Business Highlights and Upcoming Milestones:
Clinical Highlights
Tinlarebant (LBS-008) is designed to be 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 GA (the advanced form of Dry AMD) leading to retinal cell death and loss of vision.
- Stargardt disease (STGD1): Accumulation of cytotoxic vitamin A byproducts (bisretinoids) has been implicated in the onset and progression of STGD1. Tinlarebant has been granted Fast Track and Rare Pediatric Disease (RPD) designations by the U.S. Food and Drug Administration (FDA) and orphan drug designation (ODD) in the U.S., Europe, and Japan for STGD1. There are currently no FDA-approved treatments for STGD1.
- LBS-008-CT02 Trial: Completed, open label, 24-month Phase 2 trial in adolescent STGD1 subjects and presented end of trial data at the American Association of Ophthalmology (AAO) annual meeting in November 2023
- Tinlarebant was safe and well tolerated with no withdrawals due to adverse events
- The 24-month data showed a sustained lower atrophic lesion growth in Tinlarebant-treated subjects compared to ProgStar participants possessing similar baseline characteristics (aged ≤18 years) (p<0.001)
42% of Tinlarebant-treated subjects (5 out of 12) did not develop atrophic retinal lesions- Visual acuity was stabilized in the majority of subjects during the trial with a mean loss of five letters following 24 months of treatment (a loss of <10 letters is not considered clinically significant)
- DRAGON Trial: Ongoing, 24 month, randomized (2:1, active: placebo), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in adolescent STGD1 subjects
- Completed enrollment with 104 subjects in 11 countries
- Primary efficacy endpoint is slowing of atrophic lesion growth rate; safety and tolerability will also be assessed
- Interim data expected in 4Q 2024
- Geographic Atrophy (GA): GA, the advanced form of Dry AMD, is a chronic degenerative disease of the retina that leads to blindness in the elderly. Accumulation of toxic vitamin A byproducts (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; n~430 subjects), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in patients with GA
- Primary efficacy endpoint is slowing of atrophic lesion growth rate; safety and tolerability will also be assessed
- First patient dosed in the third quarter of 2023
- Has enrolled 56 subjects
- Interim data expected at mid-point of the trial
- PHOENIX Trial: Ongoing, 24-month, randomized (2:1, active: placebo; n~430 subjects), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in patients with GA
Corporate Highlights
- Completed an underwritten public offering of American Depositary Shares of the Company (“ADSs”) and warrants (the “Follow-on Offering”) for gross proceeds of
$30 million in June 2023 - Initiated an at-the-market offering program of additional ADSs (the “ATM Offering”) with an aggregate offering price of up to
$100 million in June 2023 - Raised
$22 million from the exercise of warrants issued in the Follow-on Offering and$29 million from ATM Offering as of December 31, 2023
Audited Full Year 2023 and Unaudited and Unreviewed Fourth Quarter 2023 Financial Results:
Cash: As of December 31, 2023, the Company had
R&D Expenses:
For the three months ended December 31, 2023, research and development expenses were
G&A Expenses:
For the three months ended December 31, 2023, general and administration expenses were
Net Loss:
For the three months ended December 31, 2023, the Company reported a net loss of
Webcast Information
Belite Bio will host a webcast on Tuesday, March 12, 2023, at 4:30 p.m. Eastern Time to discuss the Company’s financial results and provide a business update. To join the webcast, please visit https://wsw.com/webcast/cc/blte2/1419894. A replay will be available for approximately 90 days following the event at the Company’s Investor Relations website at https://investors.belitebio.com/presentations-events/events.
About Belite Bio
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 such as (i) atrophic age-related macular degeneration (AMD), commonly known as Geographic Atrophy (GA) in advanced dry AMD, and (ii) autosomal recessive Stargardt disease type 1, or STGD1, in addition to specific metabolic diseases. For more information, follow us on Twitter, Instagram, LinkedIn, Facebook, or visit us at www.belitebio.com.
Important Cautions Regarding Forward Looking Statements
This press release contains forward-looking statements about future expectations and plans, 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, and any other statements containing the words “expect”, “hope” and 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 timing to complete relevant clinical trials and/or to receive the interim/final data of such clinical trials; 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.
| BELITE BIO, INC CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (Amounts in thousands of US Dollars, except share and per share amounts) | ||||||||
| For the Year | ||||||||
| Ended December 31, | ||||||||
| 2022 | 2023 | |||||||
| (Audited) | (Audited) | |||||||
| Expenses | ||||||||
| Research and development | 8,869 | 24,844 | ||||||
| General and administrative | 3,952 | 6,824 | ||||||
| Total operating expenses | 12,821 | 31,668 | ||||||
| Loss from operations | (12,821 | ) | (31,668 | ) | ||||
| Other income | ||||||||
| Total other income, net | 173 | 45 | ||||||
| Loss before income tax | (12,648 | ) | (31,623 | ) | ||||
| Income tax expense | - | 9 | ||||||
| Net loss | (12,648 | ) | (31,632 | ) | ||||
| Other comprehensive income (loss) | ||||||||
| Foreign currency translation adjustments, net of nil tax | (196 | ) | 18 | |||||
| Total comprehensive loss | (12,844 | ) | (31,614 | ) | ||||
| Weighted average number of ordinary shares used in | ||||||||
| per share calculation: | ||||||||
| - Basic and Diluted | 19,976,596 | 26,593,673 | ||||||
| Net loss per ordinary share | ||||||||
| - Basic and Diluted | $ | (0.63 | ) | $ | (1.19 | ) | ||
| For the Three Months | ||||||||
| Ended December 31, | ||||||||
| 2022 | 2023 | |||||||
| (Unaudited and Unreviewed) | (Unaudited and Unreviewed) | |||||||
| Expenses | ||||||||
| Research and development | 5,226 | 4,862 | ||||||
| General and administrative | 1,495 | 2,093 | ||||||
| Total operating expenses | 6,721 | 6,955 | ||||||
| Loss from operations | (6,721 | ) | (6,955 | ) | ||||
| Other expense | ||||||||
| Total other expense, net | (62 | ) | (36 | ) | ||||
| Loss before income tax | (6,783 | ) | (6,991 | ) | ||||
| Income tax expense | - | - | ||||||
| Net loss | (6,783 | ) | (6,991 | ) | ||||
| Other comprehensive income (loss) | ||||||||
| Foreign currency translation adjustments, net of nil tax | 127 | 133 | ||||||
| Total comprehensive loss | (6,656 | ) | (6,858 | ) | ||||
| Weighted average number of ordinary shares used in | ||||||||
| per share calculation: | ||||||||
| - Basic and Diluted | 24,889,136 | 28,316,251 | ||||||
| Net loss per ordinary share | ||||||||
| - Basic and Diluted | $ | (0.27 | ) | $ | (0.25 | ) | ||
| BELITE BIO, INC CONSOLIDATED BALANCE SHEETS (Amounts in thousands of US Dollars, except share amounts) | ||||||||
| December 31 | ||||||||
| 2022 | 2023 | |||||||
| (Audited) | (Audited) | |||||||
| ASSETS | ||||||||
| Current Assets | ||||||||
| Cash | $ | 42,089 | $ | 88,157 | ||||
| Other receivables | — | 818 | ||||||
| Prepayments and other current assets | 716 | 947 | ||||||
| Other receivables due from related parties | 2 | 18 | ||||||
| Total current assets | 42,807 | 89,940 | ||||||
| Property and equipment, net | 541 | 490 | ||||||
| Prepayments and other non-current assets | 31 | 3,297 | ||||||
| Security deposits | 88 | 104 | ||||||
| Operating lease right-of-use asset, net | 806 | 811 | ||||||
| TOTAL ASSETS | $ | 44,273 | $ | 94,642 | ||||
| LIABILITIES AND SHAREHOLDERS’ EQUITY | ||||||||
| Current liabilities | ||||||||
| Accrued expenses and other liabilities | 1,906 | 3,325 | ||||||
| Operating lease liabilities – current | 198 | 308 | ||||||
| Total current liabilities | 2,104 | 3,633 | ||||||
| Non-current liabilities | ||||||||
| Operating lease liabilities –non – current | 668 | 578 | ||||||
| TOTAL LIABILITIES | 2,772 | 4,211 | ||||||
| Shareholders’ equity | ||||||||
| Ordinary shares, par value of US | 3 | 3 | ||||||
| Additional paid-in capital | 81,761 | 162,305 | ||||||
| Accumulated other comprehensive loss | (392 | ) | (374 | ) | ||||
| Accumulated deficit | (39,871 | ) | (71,503 | ) | ||||
| Total shareholders’ equity | 41,501 | 90,431 | ||||||
| TOTAL LIABILITIES AND SHAREHOLDERS’ EQUITY | $ | 44,273 | $ | 94,642 | ||||
Media and Investor Relations Contact:
Jennifer Wu
ir@belitebio.com
Julie Fallon
belite@argotpartners.com
FAQ
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