Summary of Belite Bio (BLTE) Conference Call Company Overview - **Company**: Belite Bio - **Location**: San Diego, California - **Focus**: Biotech company developing an oral treatment for Stargardt disease and age-related macular degeneration (AMD) [2][3] Core Points and Arguments - **Treatment Mechanism**: The drug, tinlaribat, is designed to reduce the amount of vitamin A entering the eye, thereby slowing the accumulation of toxic byproducts implicated in disease progression [3][4] - **Target Diseases**: - Stargardt disease: A juvenile inherited macular dystrophy - Geographic atrophy: A form of AMD associated with aging [2][4] - **Clinical Trials**: - **Stargardt Disease**: - Completed a two-year open-label study showing promising safety and efficacy [5] - Phase three study (DRAGON) initiated with 104 adolescent patients, showing positive interim analysis results [5][6] - Another phase three study (DRAGON 2) focusing on Japan, US, and UK, leveraging a Japanese regulatory designation for expedited development [7][8] - **Geographic Atrophy**: - Phase three trial (PHOENIX) enrolling 500 subjects, expected to close enrollment by July [9][10] Important Data and Metrics - **Efficacy and Safety**: - DRAGON study showed a dropout rate of less than 10% and positive efficacy signals [6] - The five-milligram dose of tinlaribat reduced retinal binding protein four by approximately 80%, which is linked to slowing lesion growth [10][11] - **Market Potential**: The market for both Stargardt disease and geographic atrophy is described as significant, with no approved oral treatments currently available [13] Regulatory Designations - The company has received various orphan drug designations and breakthrough therapy designations from regulatory agencies in the US, EU, and Japan [12][56] Competitive Advantage - **Differentiation**: Tinlaribat is an oral treatment, unlike currently approved injectable therapies for geographic atrophy, which may enhance patient compliance [11][36] - **Focus on Early Disease Stages**: The company aims to treat patients with earlier-stage diseases to maximize treatment efficacy [12][31] Future Outlook - **Upcoming Milestones**: - Completion of the DRAGON trial by the end of the year, with potential regulatory submissions based on interim analysis results [52][56] - Expected top-line results from the PHOENIX trial in July 2027 [55] Additional Considerations - **Patient Enrollment**: Enrollment for trials has been robust, with patients preferring oral treatments over injections due to lower treatment burden [36][37] - **Market Dynamics**: The company plans to adjust pricing strategies post-approval based on market size and demand for orphan drugs versus broader indications [49] This summary encapsulates the key points discussed during the conference call, highlighting the company's focus, clinical progress, regulatory strategies, and market potential.

Core Points - Belite Bio, Inc. has received Breakthrough Therapy Designation from the FDA for Tinlarebant, aimed at treating Stargardt disease (STGD1), which currently has no approved therapies [1][2] - The designation is based on interim data from the Phase 3 DRAGON trial, indicating substantial improvement over existing therapies and a favorable safety profile [2][6] - The DRAGON trial is a global study involving 104 subjects across 11 jurisdictions, designed to evaluate the safety and efficacy of Tinlarebant [5][6] Company Overview - Belite Bio is focused on developing novel therapeutics for degenerative retinal diseases, including STGD1 and Geographic Atrophy (GA) [8] - Tinlarebant is an orally administered tablet intended for early intervention in maintaining retinal health, with no current FDA-approved treatments for STGD1 or GA [7][8] - The company is also conducting a Phase 2/3 study (DRAGON II) and a Phase 3 study (PHOENIX) for other related conditions [8] Clinical Trial Insights - The Phase 3 DRAGON trial is randomized, double-masked, and placebo-controlled, with a primary efficacy endpoint focused on the growth rate of atrophic lesions [5] - The trial's completion is expected by Q4 2025, including a three-month follow-up period [6] - Tinlarebant has previously received multiple designations, including Fast Track and Rare Pediatric Disease Designations in the U.S. [6]

Financial Data and Key Metrics Changes - The company currently has USD 157 million available, which is sufficient to complete all ongoing Phase III clinical trials [32] Business Line Data and Key Metrics Changes - The lead candidate, telarabant, is being evaluated in two Phase III trials named Dragon and Dragon II, with the first Dragon trial expected to complete in Q4 2025 [13][18] - The Dragon II trial is currently enrolling patients, with 16 out of a target of 60 patients already enrolled [19] Market Data and Key Metrics Changes - The estimated prevalence of Stargardt disease in the U.S. is between 43,000 and 59,000 patients, indicating a significant market opportunity [12] - The prevalence of Stargardt disease is approximately 1 in 6,500 for populations of European descent and 1 in 11,000 to 12,000 for East Asians [11] Company Strategy and Development Direction - The company aims to expedite the approval process for telarabant, having received breakthrough designation from the FDA based on interim data [15][17] - The company is also exploring opportunities in Japan, with the PMDA showing interest in telarabant's market authorization [21] Management's Comments on Operating Environment and Future Outlook - Management expressed high expectations for the final study data from the Dragon trial, which is anticipated by the end of the year [15] - The management is closely monitoring the regulatory environment and market conditions, particularly in relation to pricing policies [24] Other Important Information - Telarabant's mechanism of action is unique as it addresses vitamin A availability in photoreceptors, which is crucial for treating Stargardt disease [7][9] - The company is aware of competitors in the geographic atrophy space but believes telarabant could be transformative due to its oral administration [31] Q&A Session Summary Question: Does BELIBA currently have sufficient capital to complete all ongoing Phase III trials? - The company confirmed it has USD 157 million available, which is sufficient to complete all clinical trials currently running [32]

Belite Bio (BLTE) Conference May 15, 2025 12:30 PM ET Speaker0 Hello, and welcome to the Deutsche Bank Depository Seats Virtual Investor Conference, DBVIK. I'm Zafra Ziz from the Deutsche Bank team. I'm pleased to announce our next presentation will be from Elote Bio. Before I introduce our speaker, a few points to note. Please submit your questions in the questions box. Also, all of those presentations will be recorded and can be accessed by the Deutsche Bank website, adr.db.com. At this point, I'm very pl ...

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Belite Bio (BLTE) Q1 2025 Earnings Call May 14, 2025 04:30 PM ET Company Participants Julie Fallon - Managing DirectorYu-Hsin Lin - Chairman of the Board of Directors & CEONathan L. Mata - Chief Scientific OfficerHendrik Scholl - CMOHao-Yuan Chuang - CFO & DirectorBasma Radwan - Vice PresidentJennifer Kim - Equity Research Director Conference Call Participants Yi Chen - Managing Director & Senior AnalystBruce Jackson - Senior AnalystMichael Okunewitch - Senior Biotechnology Analyst Julie Fallon Hello and th ...

Financial Data and Key Metrics Changes - For Q1 2025, the company reported R&D expenses of $9.4 million, an increase from $6.8 million in the same period last year, primarily due to share-based compensation and higher clinical trial expenses related to the PHOENIX trial [22] - General and administrative expenses rose to $6.1 million from $1.6 million year-over-year, also attributed to share-based compensation [22] - The net loss for the quarter was $14.3 million, compared to a net loss of $7.9 million in the prior year [22] - Operating cash outflow was approximately $8.3 million, with a cash increase of $12.3 million for the quarter, leaving the company with $157.4 million in cash and equivalents at the end of Q1 [22] Business Line Data and Key Metrics Changes - The company is advancing its lead product, Tenereband, which is in global Phase III trials for Stargardt disease and geographic atrophy, with promising interim results reported [3][4] - The DRAGON trial for Stargardt disease has maintained its sample size at 104 subjects after an interim analysis, with a completion expected by the end of the year [4][12] - The PHOENIX trial for geographic atrophy has enrolled 464 subjects to date, with full enrollment of 500 expected in Q3 [5] Market Data and Key Metrics Changes - The company has received multiple designations for Tenereband, including rare pediatric disease and fast track designations in the US, and pioneer drug designations in Japan, highlighting the significant unmet need in the market [3][4] Company Strategy and Development Direction - The company is focused on advancing Tenereband as a potential first oral treatment for degenerative retinal diseases, with ongoing Phase III trials [6] - The strategy includes maintaining a strong balance sheet to support clinical trials, with expectations of a four-year cash runway [22] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism regarding the interim results from the DRAGON trial, indicating a trend for efficacy and a well-tolerated safety profile [12][41] - The company is monitoring regulatory changes and their potential impact on drug pricing strategies, particularly in the context of US and international markets [45] Other Important Information - The company reported a low dropout rate of approximately 20% in the PHOENIX trial, which is significantly lower than rates reported in other studies [30] - The interim analysis of the DRAGON trial indicated a withdrawal rate of less than 10%, with ocular adverse events being mild and transient [12][41] Q&A Session Summary Question: Can you provide data about the discontinuation rates and enrollment in the PHOENIX trial? - The dropout rate is approximately 20%, which is below rates reported in previous studies [30] Question: Any updates on regulatory meetings for Stargardt disease trial requirements? - Meetings with regulators are scheduled, but no updates are available at this time [33] Question: Thoughts on regulatory risk given changes at the FDA? - Management does not perceive any regulatory risk due to ongoing guidance from the FDA [38] Question: What is the goalpost for efficacy and safety in the Phase III data? - The study is powered to detect a 35% treatment effect between placebo and active [40] Question: Will operating expenses continue to rise during 2025? - Operating expenses are expected to be slightly higher than Q1 levels due to upcoming milestones in clinical studies [46] Question: How is the supply chain structured geographically? - Tenereband is manufactured in the US and other geographies, mitigating tariff impacts [56]

Core Viewpoint - Belite Bio, Inc is advancing its clinical development of Tinlarebant, targeting degenerative retinal diseases, with positive interim results from the Phase 3 DRAGON trial and plans for trial completion by the end of 2025 [2][4]. Clinical Highlights - Tinlarebant is an oral, once-daily RBP4 antagonist that reduces RBP4 levels in the blood, decreasing vitamin A delivery to the eye, which is crucial for normal vision but can lead to toxicity in certain retinal diseases [3]. - The independent Data Safety Monitoring Board (DSMB) recommended the continuation of the DRAGON trial without modifications, with completion expected in Q4 2025 [4]. - The ongoing Phase 3 trial (PHOENIX) for Tinlarebant in geographic atrophy (GA) patients has enrolled 464 out of the targeted 500 subjects [4][14]. Financial Highlights - As of March 31, 2025, the company reported current assets of $157.4 million, including cash and liquidity funds [7]. - Research and development expenses for Q1 2025 were $9.4 million, up from $6.8 million in Q1 2024, primarily due to increased share-based compensation and clinical trial expenses [8]. - General and administrative expenses rose to $6.1 million in Q1 2025 from $1.6 million in Q1 2024, attributed to share-based compensation [9]. - The company reported a net loss of $14.3 million for Q1 2025, compared to a net loss of $7.9 million in the same period of 2024 [11][17]. Corporate Highlights - In February 2025, Belite Bio raised $15 million in gross proceeds through a registered direct offering, with potential additional proceeds from warrant exercises [6]. - The company is focused on maintaining strong execution across its late-stage clinical programs to deliver new treatment options for degenerative retinal diseases [2]. Upcoming Milestones - A conference call and webcast is scheduled for May 14, 2025, to discuss financial results and provide a business update [12]. - The primary efficacy endpoint for the DRAGON trial is the growth rate of atrophic lesions, with safety and tolerability also being assessed [5][14].

SAN DIEGO, May 12, 2025 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced that CSO, Dr. Nathan L. Mata will present at the dbVIC - Deutsche Bank American Depositary Receipt (ADR) Virtual Investor Conference on May 15, 2025. This virtual investor conference is aimed exclusively at introducing global companies wi ...

SAN DIEGO, May 12, 2025 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced that the executive management team will participate in four upcoming investor conferences. Details of the presentations are as follows: Deutsche Bank Depositary Receipts Virtual Investor Conference (Virtual) May 15, 2025, at 12:30 pm ET, ...