Core Insights - Belite Bio presented topline data from its Phase III trial of Tinlarebant for Stargardt disease, known as the DRAGON study, which is the largest interventional trial conducted in adolescent patients with this condition [2] Group 1: Study Overview - The DRAGON study is a pivotal 2-year trial focusing on the efficacy of Tinlarebant in treating Stargardt disease [2] - The trial involved a significant number of adolescent patients, highlighting its scale and importance in the field of retinal diseases [2] Group 2: Leadership and Expertise - The conference call featured key executives from Belite Bio, including Dr. Tom Lin (Chairman and CEO) and Dr. Hendrik Scholl (Chief Medical Officer), who provided insights into the study [4] - Dr. Hendrik Scholl, a notable figure in retinal disease research, served as the coordinating principal investigator for the ProgStar Study, adding credibility to the DRAGON study [4]

Financial Data and Key Metrics Changes - The company's cash, cash equivalents, and restricted cash totaled $32.9 million as of September 30, 2025, down from $58.8 million as of December 31, 2024 [13] - Total operating expenses for the three months ended September 30, 2025, were $19.4 million, compared to $14.4 million for the same period in 2024 [14] Business Line Data and Key Metrics Changes - The OCU400 phase three LI M EL IGHT clinical trial is on track for BLA and MAA submissions in 2026, addressing multiple genetic mutations with a single therapeutic approach [7] - OCU410 is designed to address multiple pathways implicated in dry age-related macular degeneration, showing a 23% reduction in lesion growth at 12 months [12] Market Data and Key Metrics Changes - Approximately 300,000 people in the U.S. and Europe combined live with Retinitis Pigmentosa (RP), with OCU400 targeting the remaining 98-99% of RP patients [7][8] - There are an estimated 7,000 individuals in South Korea with RP, representing about 7% of the U.S. market [9] Company Strategy and Development Direction - The company aims to file three BLAs in the next three years, with a focus on addressing unmet medical needs for patients facing vision loss [5] - Ocugen is pursuing regional partnerships to maximize patient reach while generating returns for shareholders, including an exclusive licensing agreement with Kwang Dong Pharmaceutical for OCU400 in South Korea [9] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the progress of clinical trials and the potential for OCU400 to provide a therapeutic option for a large patient population [8] - The company anticipates commercialization of OCU400 in 2027, with brand planning and marketing initiatives already underway [8] Other Important Information - The company closed a registered direct offering with Janus Henderson in August, raising approximately $20 million to extend its runway through the second quarter of 2026 [6] - The interim data from the ongoing phase two three study for OCU14ST is expected mid-2026, further advancing the goal of bringing the treatment to patients [11] Q&A Session Summary Question: Timing of BLA completion for OCU400 - Management indicated that resources are ready to turn around pivotal data to the FDA in weeks [15] Question: Manufacturing readiness for commercial production - The company confirmed that process validation runs are on target and materials for registration can be commercialized [16] Question: Endpoints for OCU410ST interim readout - The primary endpoint is lesion growth compared to untreated controls, with visual acuity as a secondary endpoint [17] Question: Statistical design of the LIMELIGHT study for OCU400 - The study includes a control arm and has a planned enrollment of 150 subjects, with a 97% power for the study [19] Question: Enrollment progress for OCU410ST - Enrollment is on track with no geographical restrictions, covering a pediatric population [24] Question: Commercial strategy for OCU400 - The company is targeting a broad indication for RP, including various mutations to maximize treatment potential [25] Question: Regulatory path for OCU400 in South Korea - Approval in South Korea is expected to follow FDA approval without the need for additional clinical trials [29] Question: Manufacturing capacity for larger indications - The company has a facility in Pennsylvania and plans to produce all U.S. supply from this site by 2027 [30]

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]