Financial Data and Key Metrics Changes - For Q4 2025, R&D expenses were $14.6 million, up from $7.3 million in Q4 2024, primarily due to expenses related to the DRAGON II trial and lower Australian R&D tax incentives [9][10] - SG&A expenses increased to $13.5 million from $4.2 million in Q4 2024, driven by higher share-based compensation and professional service fees [11][12] - The net loss for Q4 2025 was $25.3 million, compared to a net loss of $10.1 million in Q4 2024 [12] - For the full year, R&D expenses were $45.4 million, up from $29.9 million in 2024, while SG&A expenses rose to $38.9 million from $10.1 million [13][14] - The full year net loss was $77.6 million, compared to a net loss of $36.1 million in 2024 [15] Business Line Data and Key Metrics Changes - The DRAGON trial met its primary efficacy endpoint with a 36% reduction in the growth rate of atrophy lesions compared to placebo, positioning the company for regulatory engagement [5] - Enrollment in the phase III PHOENIX trial was completed with 430 subjects [7] Company Strategy and Development Direction - The company aims for a transformative year in 2026, with a planned NDA submission to the FDA in Q2 2026 and commercialization preparations for Stargardt disease [8] - Key leadership positions have been filled, and the company is building its organization in various functions including sales and marketing [8] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming NDA submission and the potential for Tinlarebant to be the first approved therapy for Stargardt disease [4][8] - The company is focused on ensuring awareness among physicians and patients regarding the new treatment [33] Other Important Information - The company closed the year with $772.6 million in cash equivalents, significantly up from $145.2 million at the end of 2024, indicating a strong balance sheet [16] Q&A Session Summary Question: NDA submission and cash usage - The NDA will be a rolling submission, with DRAGON II data relevant for Japan only. The company expects R&D activities to cost about $150 million over the next three years, with commercialization costs between $150 million and $200 million [20][21] Question: Pricing guidance - While it's early to set a price, the average price for rare disease drugs in the US is around $350,000, and the company expects to perform better than that [25] Question: Launch timing and challenges - The expected launch is by Q1 2027, with a focus on genetic testing and patient awareness as key factors for success [32][33] Question: Ex-US regulatory strategy - The company prioritizes the FDA for NDA submission, with plans to follow up with EMA and other regions afterward [40][70] Question: Prevalence of Stargardt disease - The estimated prevalence of Stargardt disease in the US is about 53,000 patients, based on genetic databases [76][77]