Financial Data and Key Metrics Changes - The company ended 2024 with just under $103 million in cash and cash equivalents, which includes the EUR 28.5 million received from Neurogene and the expected financial impact of the strategic restructuring [17] - Net revenues for Zolremdi were $1.4 million for Q4 and $2.6 million for the full year 2024 [17] - R&D expenditures totaled $21.7 million for Q4 and $81.6 million for the full year, with SG&A expenses at $15.1 million for Q4 and $61.5 million for the full year [17] - The net loss was $39.8 million for Q4 and $37.5 million for the full year, reflecting a one-time sale of a priority review voucher for $105 million [17] Business Line Data and Key Metrics Changes - Zolremdi sales topped $2.5 million, covering the 7.5 months since its launch in mid-May [8] - The company has engaged with top-tier immunologists and hematologists, expecting continued patient finding and shorter time to prescription [8] Market Data and Key Metrics Changes - The company has made significant progress in expanding the global reach of MAVERICKS-four, with an MAA submitted to the EMA and accepted for review [9] - Two international partnerships were established, one with Neurogene for commercialization in Europe, Australia, and New Zealand, and another with TABA Rare for the MENA region [10][11] Company Strategy and Development Direction - The company aims to maximize the opportunity for MAVERICKS-four in chronic neutropenia, believing it represents a larger market opportunity [16] - A strategic restructuring was announced to decrease spending by approximately $30 million to $35 million annually [16] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the engagement with regulatory agencies, indicating a supportive environment for trial design and success [20][24] - The company is focused on building awareness and education around WIM syndrome, with plans to pivot efforts towards the patient community [62] Other Important Information - The FORWARD trial for MAVERICKS-four has been activated at about 90% of targeted sites worldwide, with expectations for full enrollment in Q3 or Q4 of this year [15] - A slight price increase of 7% for Zolremdi was implemented at the start of 2025 [37] Q&A Session Summary Question: Regulatory conversation regarding ANC threshold - Management indicated that the FDA is supportive of the trial design focusing on moderate to severe patients, and the change in ANC threshold was aligned with this [20][21] Question: Impact of tightening eligibility criteria on enrollment pace - Management does not expect the tightening of eligibility criteria to slow down enrollment, as the targeted patient population remains consistent [25][26] Question: Current inventory status and discounting trends - The company noted that inventory metrics reflect stocking up in Q4, and there is no discounting occurring at this time [28][33] Question: Patient numbers and price increase for Zolremdi - Management refrained from disclosing specific patient numbers but confirmed a 7% price increase for the new year [36][38] Question: Screening progress for Phase III trial - Management reported that site activations are at about 90%, and screening is ramping up as expected [43][44] Question: Commercialization experiences since launch - The company has focused on building awareness and engaging physicians, successfully enrolling patients from the clinical trial onto the product [50][51] Question: Sales guidance for 2025 - Management is striving for continued demand increase but is not providing formal sales guidance [60][62] Question: Impact of primary outcome change on labeling for chronic neutropenia - Management does not believe the change in primary outcome will affect the labeling for chronic neutropenia [66]