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.

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]