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]