Belite Bio(BLTE)

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Belite Bio, Inc to Present at the dbVIC - Deutsche Bank ADR Virtual Investor Conference May 15th
GlobeNewswire News Room· 2025-05-12 12:35
Company Overview - Belite Bio, Inc is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for degenerative retinal diseases with significant unmet medical needs, including Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced dry age-related macular degeneration (AMD) [4] - The lead candidate, Tinlarebant, is an oral therapy aimed at reducing toxin accumulation in the eye and is currently undergoing multiple clinical trials, including a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) for STGD1, as well as a Phase 3 study (PHOENIX) for GA [4][7] Recent Developments - Dr. Nathan L. Mata, the Chief Scientific Officer, will present at the dbVIC - Deutsche Bank American Depositary Receipt (ADR) Virtual Investor Conference on May 15, 2025, providing an opportunity for investors to engage with the company [1] - The DRAGON trial, a pivotal global Phase 3 trial for Tinlarebant in adolescent STGD1 subjects, is expected to be completed by Q4 2025, with an independent DSMB recommending submission of interim analysis data for regulatory review [7] - The ongoing PHOENIX trial for Tinlarebant in GA subjects has enrolled over 460 participants [7] Event Details - The virtual investor conference will take place on May 15, 2025, at 12:30 pm ET, allowing real-time interaction between investors and the company [2] - Participation in the conference is free of charge, and an archived webcast will be available for those unable to attend live [3][2]
Belite Bio to Participate in Four Upcoming Investor Conferences
Globenewswire· 2025-05-12 12:00
Core Insights - Belite Bio, Inc is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for degenerative retinal diseases with significant unmet medical needs [2] - The company is actively participating in four upcoming investor conferences to present its developments and engage with investors [1][3] Company Overview - Belite Bio targets diseases such as Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) associated with advanced dry age-related macular degeneration (AMD) [2] - The lead candidate, Tinlarebant, is an oral therapy aimed at reducing toxin accumulation in the eye and is currently undergoing multiple clinical trials, including a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) for STGD1, as well as a Phase 3 study (PHOENIX) for GA [2] Upcoming Events - The company will present at the Deutsche Bank Depositary Receipts Virtual Investor Conference on May 15, 2025, at 12:30 pm ET [3] - A fireside chat is scheduled for May 20, 2025, at the H.C. Wainwright 3rd Annual BioConnect Investor Conference at NASDAQ [3] - Another fireside chat will take place on May 27, 2025, at the Stifel 2025 Ophthalmology Forum [3] - The Benchmark's Healthcare House Call One-on-One Conference will feature a fireside chat on May 29, 2025, at 12:45 pm ET [3]
Belite Bio to Host Webcast on May 14, 2025, to Discuss First Quarter 2025 Financial Results
Globenewswire· 2025-05-07 12:00
Core Insights - Belite Bio, Inc is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for degenerative retinal diseases with significant unmet medical needs [3] Group 1: Company Overview - Belite Bio is advancing treatments for conditions such as Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced dry age-related macular degeneration (AMD) [3] - The company's lead candidate, Tinlarebant, is an oral therapy aimed at reducing toxin accumulation in the eye and is currently undergoing multiple clinical trials, including a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) for STGD1, as well as a Phase 3 study (PHOENIX) for GA [3] Group 2: Upcoming Events - Belite Bio will host a webcast on May 14, 2025, at 4:30 p.m. Eastern Time to discuss its financial results and provide a business update for the first quarter ended March 31, 2025 [1][2] - The webcast can be accessed via a provided link and will be available for replay for approximately 90 days after the event [2]
Belite Bio Announces Poster Presentations at the Association for Research in Vision and Ophthalmology 2025 Annual Meeting
Globenewswire· 2025-04-29 12:00
Core Insights - Belite Bio, Inc. is focused on developing novel therapeutics for degenerative retinal diseases, specifically targeting Stargardt disease and geographic atrophy, which have significant unmet medical needs [1][5] Group 1: Presentations at ARVO Annual Meeting - The company will present two posters at the ARVO Annual Meeting from May 4-8, 2025, in Salt Lake City, Utah [1] - The first presentation will cover baseline characteristics of adolescent subjects in the Phase 3 DRAGON trial of Tinlarebant [2] - The second presentation will discuss pharmacokinetic and pharmacodynamic properties of Tinlarebant in adolescent Japanese patients [2] Group 2: Tinlarebant Overview - Tinlarebant is an oral therapy designed to reduce the accumulation of vitamin A-based toxins (bisretinoids) that lead to retinal diseases [3] - The drug works by modulating serum retinol binding protein 4 (RBP4) levels, thereby reducing bisretinoid formation [3] - Tinlarebant has received multiple designations, including Fast Track and Orphan Drug Designation in the U.S., and Sakigake Designation in Japan for treating STGD1 [3] Group 3: Stargardt Disease (STGD1) - STGD1 is the most common inherited macular dystrophy, leading to central vision loss due to mutations in the ABCA4 gene [4] - There are currently no FDA-approved treatments for STGD1, highlighting the need for effective therapies [4] - The disease progression is monitored through the accumulation of bisretinoids, which also affects patients with geographic atrophy [4] Group 4: Company Background - Belite Bio is a clinical-stage biopharmaceutical company dedicated to developing therapies for degenerative retinal diseases and specific metabolic diseases [5] - The lead candidate, Tinlarebant, is being evaluated in multiple Phase 3 studies, including DRAGON and PHOENIX, targeting both STGD1 and geographic atrophy [5]
Belite Bio(BLTE) - 2024 Q4 - Earnings Call Transcript
2025-03-18 02:58
Financial Data and Key Metrics Changes - In 2024, R&D expenses were $29.9 million, up from $28.8 million in 2023, primarily due to increased royalty payments and share-based compensation [33] - G&A expenses rose to $10.1 million in 2024 from $6.8 million in 2023, driven by share-based compensation [34] - The net loss for 2024 was $36.1 million, compared to $31.6 million in 2023 [34] - Cash and investments at the end of 2024 totaled $145.2 million, up from $88.2 million at the end of 2023 [34] Business Line Data and Key Metrics Changes - The company is advancing Tinlarebant in Phase 3 trials for Stargardt disease and geographic atrophy, with significant progress reported in both trials [5][9] - The Phase 3 DRAGON trial for Stargardt disease has maintained a sample size of 104 subjects after an interim analysis recommended no modifications [9][21] - The PHOENIX trial for geographic atrophy has enrolled over 400 subjects, with plans to increase enrollment to 500 [11][31] Market Data and Key Metrics Changes - Tinlarebant has received multiple designations, including Rare Pediatric Disease and Fast Track in the U.S., and Pioneer Drug designation in Japan, indicating a significant unmet need in the market [7] - The company is uniquely positioned with ongoing global Phase 3 trials, which may lead to the first oral treatment for degenerative retinal diseases [12] Company Strategy and Development Direction - The company aims to position Tinlarebant as a first-in-class oral therapy for Stargardt disease and geographic atrophy, focusing on early intervention [5][6] - The strategy includes expanding the sample size in the PHOENIX trial to enhance the chances of success [41] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism regarding regulatory reviews following the DSMB's recommendations, anticipating alignment with regulatory agencies [39] - The company remains focused on completing trials and maintaining a full-year cash runway, with expectations for continued progress in clinical studies [35] Other Important Information - The withdrawal rate in the DRAGON trial was 9.6%, with ocular adverse events accounting for 3.8% of withdrawals [20] - The company raised $15 million in gross proceeds in February 2025, enhancing its financial position [35] Q&A Session Summary Question: Potential outcomes from regulatory interactions regarding Stargardt - Management believes the regulatory agencies will likely align with the DSMB's positive recommendation for drug approval [39] Question: Reason for increasing sample size in GA trial - The decision was made to boost chances of success based on smooth enrollment progress [41] Question: Current discontinuation rates in the GA trial - The dropout rate for the PHOENIX trial is approximately 20%, which is common for elderly populations [53] Question: Approval potential for broader patient populations - Management indicated that efficacy in adolescents could facilitate approval for adults as well [57] Question: Difference in lesion growth between placebo and treatment in DRAGON study - The study is powered for a 40% treatment effect with 80% power to detect that effect at the second year [84]
Belite Bio(BLTE) - 2024 Q4 - Earnings Call Transcript
2025-03-17 22:40
Financial Data and Key Metrics Changes - In 2024, the company reported R&D expenses of $29.9 million, an increase from $28.8 million in 2023, primarily due to higher royalty payments and increased share-based compensation [32] - General and administrative (G&A) expenses rose to $10.1 million in 2024 from $6.8 million in 2023, driven by increased share-based compensation [33] - The net loss for 2024 was $36.1 million, compared to $31.6 million in 2023 [33] - Cash and investments at the end of 2024 totaled $145.2 million, up from $88.2 million at the end of 2023 [33] Business Line Data and Key Metrics Changes - The company is advancing its lead product, Tinlarebant, in Phase 3 trials for Stargardt disease and geographic atrophy, with significant progress reported in both trials [4][10] - The Phase 3 DRAGON trial for Stargardt disease has maintained a sample size of 104 subjects after an interim analysis recommended proceeding without modifications [8][20] - The PHOENIX trial for geographic atrophy has enrolled over 400 subjects, with plans to increase enrollment from 450 to 500 subjects [10][30] Market Data and Key Metrics Changes - Tinlarebant has received multiple designations, including Rare Pediatric Disease and Fast Track in the U.S., and Pioneer Drug designation in Japan, indicating a significant unmet medical need [6] - The company is uniquely positioned with ongoing global Phase 3 trials, which may lead to the first oral treatment for degenerative retinal diseases [11] Company Strategy and Development Direction - The company aims to position Tinlarebant as a first-in-class oral therapy for Stargardt disease and geographic atrophy, focusing on early intervention to slow disease progression [4][5] - The strategy includes expanding patient enrollment in clinical trials to enhance the chances of regulatory approval and market success [40] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism regarding the regulatory review process following the DSMB's recommendation for drug approval based on interim results [38] - The company plans to expedite a second Phase 3 trial for geographic atrophy if positive signals are observed in the ongoing PHOENIX trial [44] Other Important Information - The company raised $15 million in gross proceeds through a registered direct offering in February 2025, ensuring a full year's cash runway [34] - The dropout rate in the PHOENIX trial is approximately 20%, which is considered manageable given the elderly population involved [52] Q&A Session Summary Question: Potential outcomes from regulatory interactions regarding Stargardt disease - Management believes that regulatory agencies will likely align with the DSMB's positive recommendation for drug approval, but if not, they will continue with the study [38] Question: Reason for increasing sample size in the GA trial - The decision to increase the sample size to 500 patients was driven by smooth progress in the GA study and the desire to enhance the chances of success [40] Question: Update on discontinuation rates in the GA trial - The dropout rate for the PHOENIX trial is approximately 20%, which is lower than historical rates for similar studies [52] Question: Approval expectations for Stargardt disease patients - Management indicated that if efficacy is demonstrated in adolescents, it should facilitate approval for adults as well [56] Question: Current status of regulatory submissions - The company is working with multiple CROs for regulatory submissions, and the process is currently underway [78] Question: Expected treatment effect in the DRAGON study - The DRAGON study is powered for a 40% treatment effect with 80% power to detect that effect at the second year [84]
Belite Bio(BLTE) - 2024 Q4 - Annual Report
2025-03-17 10:05
Exhibit 99.1 · Following a pre-specified interim analysis, the Data Safety Monitoring Board (DSMB) recommended the DRAGON trial, a pivotal Phase 3 trial in adolescent Stargardt 1 (STGD1) subjects, proceed without any modification; trial completion expected Q4 2025 (including a three-month follow-up period) · Pivotal global Phase 3 PHOENIX trial of Tinlarebant in geographic atrophy (GA) subjects is ongoing with 406 subjects enrolled · Raised $15 million in gross proceeds in a registered direct of ering on Fe ...
Belite Bio(BLTE) - 2024 Q4 - Annual Report
2025-03-17 10:02
Product Development and Regulatory Approval - The company is highly dependent on the success of its lead product candidate, Tinlarebant, which is currently in clinical trials for the treatment of autosomal recessive Stargardt disease (STGD1) [28]. - The company currently has no products approved for commercial sale and may never be able to develop marketable products, making investment riskier compared to companies with multiple late-stage product candidates [28]. - Significant delays in obtaining regulatory approvals for Tinlarebant could adversely affect the company's business and financial condition [42]. - The success of Tinlarebant will depend on successful completion of ongoing clinical trials and timely receipt of marketing approvals from regulatory authorities [30]. - The company faces risks related to the regulatory approval processes, which are time-consuming and may evolve over time [25]. - The regulatory approval process for the company's product candidates is expensive and may take many years, with potential delays due to additional required clinical trials [72]. - The company has never obtained marketing approval for any product candidate, and regulatory authorities may require additional trials or studies before reconsidering applications [73]. - The company may need to conduct additional clinical trials if initial results are not positive, which could delay regulatory approval and commercialization prospects [58]. - The inability to obtain marketing approvals could prevent the commercialization of product candidates, significantly harming the business [74]. - Regulatory authorities may impose additional restrictions or require recalls if product candidates are found to be less effective or cause undesirable side effects post-approval [75]. - The company may face challenges in obtaining regulatory approvals from authorities such as the FDA, TGA, NMPA, EMA, and PMDA, which can be time-consuming and uncertain [121]. - Regulatory approval processes may require additional preclinical or clinical data, potentially delaying commercialization plans [124]. - Ongoing regulatory obligations post-approval may lead to significant additional expenses and potential penalties for non-compliance [138]. Financial Performance and Funding - The company has incurred significant net operating losses of approximately US$12.6 million, US$31.6 million, and US$36.1 million for the years ended December 31, 2022, 2023, and 2024, respectively, with an accumulated deficit of approximately US$107.6 million as of December 31, 2024 [115]. - The company expects to continue incurring substantial expenses related to ongoing clinical trials and research and development efforts, which will require additional funding [107]. - The net cash used in operating activities was approximately US$11.5 million, US$29.8 million, and US$29.2 million for the years ended December 31, 2022, 2023, and 2024, respectively [106]. - The company has no products approved for commercial sale and has not generated any revenue from product sales, indicating a high level of uncertainty regarding future profitability [112]. - The company anticipates that its expenses will increase significantly as it seeks regulatory approvals and commercializes its product candidates [116]. - The company has recorded net cash outflow from operating activities since its inception, indicating a need for additional financing to fund operations [105]. - The company may seek additional funding through equity offerings, debt financings, collaborations, and licensing arrangements, which could dilute existing shareholders' ownership [119]. Clinical Trials and Patient Enrollment - The timely completion of clinical trials is contingent on the ability to enroll a sufficient number of patients, which may be affected by various factors [48]. - Clinical trials may face competition for patient enrollment, which could limit the number and types of patients available for the company's trials [53]. - The company may incur additional costs or experience delays if clinical trials do not demonstrate safety and efficacy to the satisfaction of regulatory authorities [54]. - Manufacturing issues, including supply quality and compliance with good manufacturing practices, could significantly impact the company's clinical trials and product availability [71]. Intellectual Property and Competition - The company holds a portfolio of 23 issued U.S. patents and 9 pending U.S. patent applications, along with 23 issued foreign patents and 44 pending foreign patent applications, providing protection in multiple regions [151]. - The patent position of the company is uncertain, and challenges to patent validity could limit competitive advantages [152]. - The company may face increased competition if a competitor's drug is approved earlier for the same condition, potentially impacting market share despite orphan drug exclusivity [134]. - Competition in the biopharmaceutical industry is intense, with competitors potentially achieving regulatory approval and market penetration faster [82]. - The company may face claims regarding the wrongful use of trade secrets from former employers of its employees, which could lead to litigation and loss of intellectual property rights [185]. - There is a risk of costly litigation if third parties claim that the company's products infringe on their intellectual property rights [193]. Operational Risks and Management - The company must maintain an effective system of internal controls to accurately report its results and prevent fraud, as failure to do so could adversely affect investor confidence [25]. - The adequacy of the company's internal control over financial reporting is crucial for maintaining investor confidence and preventing material misstatements [205]. - The company may experience difficulties in managing growth as it plans to significantly increase the number of employees and consultants [216]. - The company is exposed to risks of misconduct by employees and contractors, which could harm its reputation and financial standing [215]. - The company may not be able to effectively manage outsourced activities, potentially leading to delays in regulatory approvals [217]. Market and Commercialization Challenges - Market acceptance of product candidates is crucial for commercial success, and failure to achieve this could result in insufficient revenue generation [79]. - Reimbursement for approved product candidates may be limited or unavailable, impacting the ability to commercialize successfully [89]. - The potential market opportunity for the company's product candidates may be limited, depending on regulatory approvals and market acceptance [98]. - Legislative changes, such as the ACA, may impose additional costs and requirements on the company, affecting marketing approvals and pricing strategies [146]. Risks Related to Manufacturing and Supply Chain - The company relies entirely on third-party contractors for cGMP manufacturing capabilities, which poses risks of delays in clinical supply and commercialization if issues arise [70]. - The company relies on third parties for conducting clinical trials and manufacturing, and any failure in these relationships could harm its business [25]. - The company maintains liability insurance for clinical trials, but coverage may be insufficient for product liability claims [219].
Belite Bio Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Corporate Update
Newsfilter· 2025-03-17 10:00
Core Insights - Belite Bio, Inc is advancing its clinical development of Tinlarebant, a novel therapeutic for degenerative retinal diseases, with significant progress in the Phase 3 DRAGON trial for Stargardt disease type 1 (STGD1) [2][3][4] Financial Results - As of December 31, 2024, the company reported cash of $31.7 million, a decrease from $88.2 million on December 31, 2023 [8][20] - The company had $113.5 million in investments as of December 31, 2024, compared to none on December 31, 2023 [8][20] - For the year ended December 31, 2024, research and development expenses were $29.9 million, up from $24.8 million in 2023 [10][18] - General and administrative expenses for the year ended December 31, 2024, were $10.1 million, compared to $6.8 million in 2023 [11][18] - The net loss for the year ended December 31, 2024, was $36.1 million, or ($1.18) per share, compared to a net loss of $31.6 million, or ($1.19) per share in 2023 [13][18] Clinical Development Highlights - The DRAGON trial for STGD1 subjects is ongoing, with completion expected in Q4 2025, following a positive interim analysis by the Data Safety Monitoring Board (DSMB) [4][5] - The PHOENIX trial for geographic atrophy (GA) subjects is also ongoing, with 406 subjects enrolled and an increased sample size target of 500 [14] - Tinlarebant has received multiple designations, including Fast Track and Orphan Drug Designation in the U.S. for STGD1 [5][16] Corporate Actions - The company raised $15 million in gross proceeds through a registered direct offering on February 5, 2025, with potential additional proceeds from warrant exercises [4][7] - A conference call and webcast were scheduled for March 17, 2025, to discuss financial results and business updates [15]
Belite Bio Announces Availability of Annual Report on Form 20-F Through Company Website
Globenewswire· 2025-03-17 10:00
Group 1 - Belite Bio, Inc is a clinical-stage biopharmaceutical company focused on developing therapies for degenerative retinal diseases and specific metabolic diseases [2] - The company has filed its annual report on Form 20-F for the year ended December 31, 2024, with the Securities and Exchange Commission [1] - Belite's lead candidate, Tinlarebant, is currently undergoing multiple clinical trials, including a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) for Stargardt disease type 1, as well as a Phase 3 study (PHOENIX) for Geographic Atrophy [2] Group 2 - The annual report is available on the company's website, and shareholders can request a hard copy free of charge [1] - Belite Bio targets significant unmet medical needs in retinal diseases, such as Stargardt disease type 1 and Geographic Atrophy in advanced dry age-related macular degeneration [2]