
Financial Performance - The net loss for the year ended December 31, 2024 was 1.65 per share, compared to a net loss of 2.38 per share for 2023[7]. - The company reported a net loss of 0.18 per share, for the fourth quarter of 2024, compared to a net loss of 0.60 per share, for the same period in 2023[7]. - The net loss for the three months ended December 31, 2024, was 26,958 million in the same period of 2023, indicating improved financial performance[17]. - Comprehensive loss for the year ended December 31, 2024, was 103,548 million in 2023, indicating a reduction in overall losses[17]. Revenue and Income - Revenue recognized from the License and Collaboration Agreement with Acadia Pharmaceuticals for the year ended December 31, 2024 was 8.8 million in 2023[7]. - Revenue for the three months ended December 31, 2024, was 2,801 million for the same period in 2023, representing a significant increase[17]. - Total other income for the three months ended December 31, 2024, was 2,629 million in 2023, showing an increase of 20.7%[17]. Expenses - Research and development expenses for the year ended December 31, 2024 were 82.2 million in 2023[7]. - General and administrative expenses for the year ended December 31, 2024 were 41.3 million in 2023[7]. - Total operating expenses for the three months ended December 31, 2024, were 32,388 million in 2023, reflecting a year-over-year increase of 5.4%[17]. - Research and development expenses for the year ended December 31, 2024, totaled 82,231 million in 2023, highlighting ongoing investment in innovation[17]. - General and administrative expenses for the year ended December 31, 2024, were 41,322 million in 2023, reflecting a year-over-year increase of 18.1%[17]. Cash and Funding - As of December 31, 2024, the company had 165 million upfront payment from the Biogen collaboration, is expected to fund operations to mid-2028[1][7]. - The company is well-capitalized to advance through Phase 3 and prepare for launch readiness of zorevunersen[2]. Research and Development - The company plans to initiate the Phase 3 EMPEROR study of zorevunersen in the second quarter of 2025, following successful alignment with regulatory agencies[2][3]. - The FDA granted Breakthrough Therapy Designation for zorevunersen for the treatment of Dravet syndrome with a confirmed mutation in the SCN1A gene[4]. - The company retains exclusive rights for zorevunersen in the United States, Canada, and Mexico, while Biogen has exclusive commercialization rights for the rest of the world[3]. Share Information - The weighted average common shares outstanding for the three months ended December 31, 2024, increased to 57,029,296 from 44,958,894 in 2023, indicating a rise in share issuance[17]. Marketable Securities - The company reported an unrealized loss on marketable securities of 122 million in the same period of 2023[17].