The global healthcare market is growing rapidly—it is expected to climb at a 6.9% CAGR over the next eight years, reaching more than $22.3 trillion by 2033. Its appeal to investors in the current market environment may also lie in its non-cyclical nature, thanks to resilient demand that is more related to health needs and demographics than to external economic conditions. However, finding healthcare names poised for major growth may be difficult, considering that many of the largest legacy firms in the spac ...

Tinlarebant has been granted Breakthrough Therapy, Fast Track, and Rare Pediatric Disease Designations in the U.S.; Orphan Drug Designation in the U.S., Europe, and Japan; and Pioneer Drug Designation in Japan for Stargardt diseaseLast subject visit completed in the pivotal Phase 3 DRAGON trial of Tinlarebant in Stargardt diseaseTopline data expected in Q4 2025 SAN DIEGO, Sept. 12, 2025 (GLOBE NEWSWIRE) -- Belite Bio Inc (NASDAQ: BLTE) (“Belite” or the “Company”), a clinical-stage drug development company f ...

Summary of the Conference Call on Belite Bio and Tinlarebant Company Overview - **Company**: Belite Bio - **Product**: Tinlarebant - **Indication**: Stargardt disease Key Points Development Status - The Dragon trial is a two-year international registration trial involving 104 patients with Stargardt disease, with top-line data readout expected by the end of Q4 2023 and an NDA submission planned for the first half of 2026 [3][6] Efficacy and Safety Data - Tinlarebant has shown efficacy in preventing the development of atrophic lesions (DDAF) and slowing their growth by almost 50% compared to natural history controls [4] - The overall withdrawal rate due to ocular adverse events is less than 4%, indicating an excellent safety profile [5] Regulatory Strategy - The FDA granted breakthrough designation based on interim analysis data, allowing for expedited regulatory review [6] - Full approval will be sought in the U.S., Europe, Japan, and other markets, with a second registration trial (Phoenix trial) for geographic atrophy (GA) already fully enrolled with 530 patients [14][22] Market Potential - The estimated prevalence of Stargardt disease is approximately 1 in 7,000, translating to about 47,000 to 59,000 patients in the U.S. alone, with a potential market penetration of around 40% [8][9] - The company anticipates that Tinlarebant will be beneficial for both pediatric and adult patients, with no age restrictions on the label [8] Competitive Landscape - NanoScope Therapeutics' gene therapy is viewed as a complementary approach rather than a direct competitor, as it targets different aspects of vision restoration [10][11][13] Future Catalysts - Key upcoming milestones include: - Q4 2025: Full enrollment of the Dragon 2 trial - End of Q4 2025: Top-line data readout for the Dragon trial - First half of 2026: NDA submission anticipated [23] Financial Position - Belite Bio has approximately $160 million in cash, providing a four-year runway for ongoing programs, bolstered by a recent private placement financing of up to $275 million [22] Patent Protection - The company holds 14 active patent families, primarily composition of matter patents, which will last until at least 2040 [21] Go-to-Market Strategy - The company plans to prioritize the commercialization of Tinlarebant for Stargardt disease due to its orphan indication status, which allows for higher pricing and easier access to patients through specialized networks [18] Correlation with Geographic Atrophy - There is a belief that Tinlarebant's efficacy in Stargardt disease may correlate positively with its efficacy in GA, as both conditions share common pathological features [16][17] Additional Insights - The company is strategically positioned to leverage its connections with retinal specialists and inherited retinal disease specialists to facilitate the commercialization of Tinlarebant [18]

Transaction led by RA Capital Management with participation from Eventide Asset Management, Marshall Wace, RTW Investments, Soleus Capital and Vestal Point Capital$125 million financing upfront with up to an additional $150 million tied to exercise of warrantsWarrants exercisable at a 20% premium to purchase price SAN DIEGO, Sept. 08, 2025 (GLOBE NEWSWIRE) -- Belite Bio, Inc ("Belite Bio" or the "Company") (Nasdaq: BLTE), a clinical-stage drug development company focused on advancing novel therapeutics targ ...

Company Overview - Belite Bio, Inc is a clinical-stage drug development company focused on novel therapeutics for degenerative retinal diseases with significant unmet medical needs, including Stargardt disease type 1 and Geographic Atrophy in advanced dry age-related macular degeneration [2] - The lead candidate, Tinlarebant, is an oral therapy aimed at reducing toxin accumulation in the eye, currently undergoing Phase 3 and Phase 2/3 studies [2] Upcoming Events - The executive management team will participate in three investor conferences: - Cantor Global Healthcare Conference on September 5, 2025, at 8:00 am ET - Morgan Stanley 23rd Annual Global Healthcare Conference on September 8, 2025, at 5:35 pm ET - H.C. Wainwright 27th Annual Global Investment Conference on September 9, 2025, at 8:00 am ET [3] - Webcasts of the presentations will be available on the investor relations section of the Belite Bio website, with replays archived for 90 days [1]

Summary of Belite Bio (BLTE) FY Conference - August 13, 2025 Company Overview - **Company**: Belite Bio - **Focus**: Development of oral treatments for retinal diseases, specifically Stargardt disease and geographic atrophy secondary to age-related macular degeneration (AMD) [12][31] Key Points and Arguments 1. **Leadership Team**: - Doctor Tom Lin, Chairman and COO, has over 10 years of executive management experience in biotech and has been involved in over 10 new drug developments [4] - Doctor Hendrick Scholl, Chief Medical Officer, has 25 years of experience in treating retinal diseases and has led significant research initiatives [5][6] - Doctor Nathan Mata, Chief Science Officer, has over 15 years of expertise in ophthalmic drug development [7] 2. **Pipeline Overview**: - **Tinlariband**: A novel once-daily oral tablet targeting serum retinal binding protein 4 (RBP4) to reduce retinol delivery to the eye, aimed at slowing or halting disease progression in Stargardt disease and geographic atrophy [11][17] - Ongoing clinical trials include: - **Dragon Trial**: A global registration trial for Stargardt disease with 104 subjects, focusing on slowing atrophic lesions [9][25] - **PHOENIX Trial**: A phase three trial for geographic atrophy, recently completed enrollment of 500 subjects [10][32] 3. **Market Opportunity**: - Significant unmet medical need for Stargardt disease, with no FDA-approved treatments available [12] - Breakthrough therapy, Fast Track, and orphan drug designations received in the US and EU for Stargardt disease [12] 4. **Clinical Trial Results**: - Phase two trial showed that tinlariband slowed lesion growth in Stargardt disease, with a significant reduction in RBP4 levels [21][24] - Interim analysis of the Dragon trial indicated that tinlariband is safe and well-tolerated, with a withdrawal rate of less than 10% [27] 5. **Safety Profile**: - Common adverse events included mild xanthopsia and delayed dark adaptation, with no severe treatment-related adverse events reported [29] - Overall, the treatment was well tolerated with no significant findings related to vital signs or organ functions [29] 6. **Visual Acuity Data**: - No significant decline in visual acuity was observed over the two-year duration of the Dragon trial, with average changes being minimal [30] 7. **Future Directions**: - Focus on early intervention for patients with small lesions to potentially slow disease progression [31] - Plans for further regulatory submissions based on interim analysis data [28] Additional Important Information - **Pathophysiology**: Stargardt disease and geographic atrophy share similar mechanisms involving the accumulation of toxic bisretinoids leading to retinal cell death [16][17] - **Regulatory Engagement**: Data from the interim analysis has been presented to various regulatory agencies, including the PMDA in Japan and the FDA [28] This summary encapsulates the critical insights from the conference, highlighting Belite Bio's strategic focus on innovative treatments for retinal diseases and the promising results from ongoing clinical trials.

Financial Data and Key Metrics Changes - For Q2 2025, the company reported R&D expenses of $11 million, an increase from $9.1 million in the same period last year, primarily due to higher expenses related to the PHOENIX trial and manufacturing costs [8][9] - G&A expenses rose to $6.5 million from $1.4 million year-over-year, mainly due to increased share-based compensation [9] - The net loss for the quarter was $16.3 million, compared to a net loss of $9.5 million in the same period last year, with a significant portion of the increase attributed to non-cash share-based compensation of $7.6 million [9][10] - As of the end of Q2, the company had $149.2 million in cash and equivalents, with a projected four-year cash runway [10] Business Line Data and Key Metrics Changes - The company is advancing its drug teneraband, which is in global phase three trials for Stargardt's disease and geographic atrophy, with significant progress reported in both trials [4][5] - The Dragon trial for Stargardt's disease has completed interim analysis, and the FDA has recommended proceeding without modifications, with completion expected in Q4 2025 [6][15] - The global phase three study for geographic atrophy has completed enrollment with 529 subjects [7] Market Data and Key Metrics Changes - The company has received multiple designations for teneraband, including breakthrough therapy and orphan drug designations in the US, Europe, and Japan, indicating a significant unmet medical need [5] Company Strategy and Development Direction - The company aims to position teneraband as the first oral treatment for degenerative retinal diseases, focusing on completing its phase three trials and preparing for potential regulatory submissions [8][10] - The management is strategically expanding the Dragon two trial to additional countries to accelerate enrollment [41] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing trials and the potential for accelerated approval based on robust statistical significance from the interim data [15][26] - The company anticipates a cash burn of approximately $40 to $45 million over the next two years as it approaches key clinical milestones [46] Other Important Information - The company raised $15 million in gross proceeds from a registered direct offering on August 8, which supports its ongoing operations and trial commitments [7][10] Q&A Session Summary Question: Status of FDA discussion regarding interim data from the Dragon trial - Management confirmed that they have met with the FDA and are on track to potentially file for accelerated approval based on interim data, but a confirmatory follow-up study will still be required [14][15] Question: Upcoming presentations or data readouts - Management indicated that they plan to present eye data at the AAO conference in late October, but will keep efficacy data confidential until after submission [27][28] Question: Timing of data release after the Dragon trial finishes - Management expects to show statistically significant differences in lesion growth rates between treatment and placebo groups, which is critical for approval [36] Question: Current estimate timeline for reaching target enrollment in the Dragon-two trial - Management stated that enrollment is expected to complete by the end of the year, with strategic timing to avoid competition with the Dragon one trial [42] Question: Drivers for rising operating expenses - Management noted that the majority of the increase in operating expenses is due to share-based compensation, which is non-cash related, and expects cash burn to increase as they approach key milestones [46]

Financial Data and Key Metrics Changes - For Q2 2025, the company reported R&D expenses of $11 million, an increase from $9.1 million in the same period last year, primarily due to higher expenses related to the PHOENIX trial and manufacturing costs [10] - G&A expenses rose to $6.5 million from $1.4 million year-over-year, mainly due to increased share-based compensation [10] - The net loss for the quarter was $16.3 million, compared to a net loss of $9.5 million in the same period last year, with a significant portion of the increase attributed to non-cash share-based compensation [11] - As of the end of Q2, the company had $149.2 million in cash and equivalents, with a projected four-year cash runway [12] Business Line Data and Key Metrics Changes - The company is advancing its drug teneraband, which is in global phase three trials for Stargardt's disease and geographic atrophy, with significant progress reported in both trials [5][6] - The Dragon trial for Stargardt's disease has completed interim analysis, and the FDA has recommended proceeding without modifications, with completion expected in Q4 2025 [7] - Enrollment for the Dragon two trial is ongoing, with 17 out of 60 subjects enrolled, including 10 Japanese subjects [8] Market Data and Key Metrics Changes - The company has received multiple designations for teneraband, including breakthrough therapy and orphan drug designations in the US, Europe, and Japan, indicating a significant unmet medical need [6] - The global phase three study for geographic atrophy has completed enrollment with 529 subjects [8] Company Strategy and Development Direction - The company aims to position teneraband as the first oral treatment for degenerative retinal diseases, focusing on completing its phase three trials and preparing for potential regulatory submissions [9] - The management is strategically expanding the Dragon two trial to additional countries to accelerate enrollment [43] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing trials and the potential for accelerated approval based on interim data, with a focus on achieving robust statistical significance [17][55] - The company anticipates higher cash burn in the coming years as it approaches key clinical milestones, but expects to maintain a four-year cash runway [47] Other Important Information - The company raised $15 million in gross proceeds from a registered direct offering on August 8 [8] - Management emphasized the importance of confidentiality regarding efficacy data until the submission to the FDA [29] Q&A Session Summary Question: Status of FDA discussion regarding interim data from the Dragon trial - The company has met with the FDA and is on track to potentially file for accelerated approval based on interim analysis data, with a recommendation to complete the 24-month study [16][19] Question: Plans for interim analysis of the GA trial - An interim analysis is planned for the GA trial, but specific timelines are not yet available [22] Question: Educational efforts for the medical community - The company plans to present data at the AAO conference but will keep efficacy data confidential until submission [29][30] Question: Timeline for data release and filing - The company expects to have sufficient long-term safety data by the first quarter of next year and is on track with CMC discussions with the FDA [63][66]

Financial Performance - Total operating expenses increased to $17596 thousand in Q2 2025 from $10471 thousand in Q2 2024[13] - Research and Development (R&D) expenses increased to $11049 thousand in Q2 2025 from $9078 thousand in Q2 2024[13] - General and Administrative (G&A) expenses increased significantly to $6547 thousand in Q2 2025 from $1393 thousand in Q2 2024[13] - Net loss increased to $16320 thousand in Q2 2025 from $9494 thousand in Q2 2024[13] - The company had $1492 million in cash, liquidity fund, time deposits and U S treasury bills[13] - The company raised approximately $15 million in gross proceeds in a registered direct offering on August 8, 2025[13] Pipeline and Clinical Trials - Belite Bio is conducting a Phase 3, 2-year global trial ("PHOENIX" Study) for Stargardt Disease (STGD1) with completed enrollment of 529 subjects[10] - A Phase 3, 2-year global trial ("DRAGON" Study) is ongoing for Tinlarebant with 104 subjects aged 12-20, with completion expected in Q4 2025[10] - A Phase 2/3, 2-year global trial ("DRAGON II" Study) is ongoing for Tinlarebant with 60 subjects aged 12-20[10] Tinlarebant - Tinlarebant is a novel, once-daily oral tablet designed to reduce retinol delivery to the eye by binding to serum retinol binding protein 4 (RBP4)[11] - Belite Bio believes early intervention targeting emerging retinal pathology is the best approach to slow disease progression in STGD1 & GA[11]

SAN DIEGO, Aug. 04, 2025 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE), a clinical-stage drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced that it will host a webcast on Monday, August 11, 2025, at 4:30 p.m. Eastern Time to discuss the Company's financial results and provide a business update for the second quarter ended June 30, 2025. Media and Investor Relations Contact: Webcast Informati ...