Financial Data and Key Metrics Changes - For the full year 2025, the company reported net product revenue of $155.4 million, representing a 172% increase year-over-year [6][35] - The fourth quarter net product revenue reached $52.8 million, reflecting a 37% sequential growth over the third quarter [21][35] - The company ended 2025 with over $440 million in cash and no debt, providing a strong financial foundation [9][38] Business Line Data and Key Metrics Changes - OJEMDA remains the primary revenue driver, with over 4,600 total prescriptions for the year, marking an increase of more than 180% compared to 2024 [6][22] - The company achieved double-digit sequential quarterly growth throughout 2025, with fourth quarter prescriptions exceeding 1,300, representing an 11% growth quarter-over-quarter [21][22] Market Data and Key Metrics Changes - The company anticipates global expansion for OJEMDA, with regulatory approvals being prepared for ex-U.S. markets, including Europe [8] - Coverage rates for pediatric low-grade glioma (PLGG) remain above 95%, with over 90% of patients approved on the first request [24] Company Strategy and Development Direction - The company aims to solidify OJEMDA as the second-line standard of care in PLGG, with a focus on driving new patient starts and optimizing treatment persistence [23][25] - The acquisition of Mersana Therapeutics is seen as a strategic move to enhance the pipeline, particularly with the Emi-Le program targeting adenoid cystic carcinoma [39] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth trajectory for 2026, reiterating guidance for OJEMDA net product revenue of $225 million to $250 million [6][38] - The company is focused on establishing a strong scientific basis for OJEMDA's use in frontline settings through ongoing clinical trials [7][18] Other Important Information - The company reported a median duration of therapy for commercial PLGG patients trending towards 19 months, indicating robust treatment persistence [47] - The three-year follow-up data from the FIREFLY-1 trial demonstrated a 53% objective response rate, reinforcing the clinical efficacy of OJEMDA [12][18] Q&A Session Summary Question: How does the company maintain and improve persistency for OJEMDA? - Management highlighted that persistency is currently strong, with median therapy duration trending towards 19 months, and identified opportunities for improvement through physician education and patient support programs [45][47] Question: What are the expectations for the updated Emi-Le data? - Management indicated that substantial data will be shared mid-year, focusing on antitumor activity and safety, which will support discussions with the FDA regarding accelerated clinical development [52][56] Question: What are the early trends for Q1 2026? - Management reiterated confidence in the guidance for 2026, noting that demand remains strong in Q1, although specific details about the current quarter were not disclosed [61][63] Question: Can the company break down the time to next treatment analysis further? - Management acknowledged the importance of understanding treatment patterns and indicated that ongoing analyses will provide insights into patient responses post-progression [64][66] Question: What is the expected response rate for Emi-Le in adenoid cystic carcinoma? - Management emphasized the need for a robust data set that clearly delineates the performance of Emi-Le compared to existing therapies, which currently have poor response rates [82][86]

Financial Data and Key Metrics Changes - For the full year 2025, the company reported net product revenue of $155.4 million, representing a 172% increase year-over-year, with double-digit sequential quarterly growth throughout the year [5][34] - In Q4 2025, net product revenue reached $52.8 million, reflecting a 37% sequential growth over Q3 [20][34] - The company ended 2025 with approximately $441 million in cash and no debt, providing a strong financial foundation for future growth [9][38] Business Line Data and Key Metrics Changes - OJEMDA remains the primary revenue driver, with over 4,600 total prescriptions for the year, marking an increase of over 180% compared to 2024 [5][21] - The company achieved a median duration of therapy trending towards 19 months for commercial PLGG patients, indicating strong persistency [47] Market Data and Key Metrics Changes - The company anticipates global expansion for OJEMDA, with regulatory approvals being prepared for markets outside the U.S., including Europe [8] - Coverage rates for PLGG remain above 95%, with over 90% of patients approved on the first request, facilitating quick therapy initiation [23] Company Strategy and Development Direction - The company aims to solidify OJEMDA as the second-line standard of care in pediatric low-grade glioma (PLGG) and is focused on driving new patient starts and optimizing treatment persistence [22][24] - The acquisition of Mersana Therapeutics is seen as a strategic move to enhance the pipeline, particularly with the Emi-Le program targeting adenoid cystic carcinoma [39] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth trajectory for 2026, reiterating guidance for OJEMDA net product revenue of $225 million to $250 million [5][38] - The company is optimistic about the potential of OJEMDA to become the standard of care in the second-line setting, supported by positive clinical data and physician confidence [22][24] Other Important Information - The company is advancing multiple pipeline programs, including DAY301 and Emi-Le, which target significant unmet medical needs in oncology [27][30] - The three-year follow-up data from the FIREFLY-1 trial demonstrated a 53% objective response rate for OJEMDA, reinforcing its clinical efficacy [12][18] Q&A Session Summary Question: How does the company maintain and improve persistency for OJEMDA? - Management highlighted that current persistency is strong, with median therapy duration trending towards 19 months, and identified opportunities to enhance persistency through targeted patient support programs and physician education [47][48] Question: What are the expectations for the upcoming Emi-Le data? - Management indicated that they expect to present substantial data supporting the move into registrational trials, focusing on the antitumor activity and safety profile of Emi-Le [52][54] Question: What early trends are observed in Q1 2026? - Management noted that demand remains strong in Q1, with confidence in achieving the reiterated revenue guidance for the year [62][63] Question: Can the company provide insights on the time to next treatment analysis? - Management explained that the analysis shows that many patients continue therapy despite radiographic progression, emphasizing the importance of clinical signs over imaging results in treatment decisions [66][68] Question: What is the expected response rate for Emi-Le in ACC? - Management stated that they aim for a robust data package that clearly delineates the performance of Emi-Le compared to existing therapies, which have poor response rates [85][86]