
Financial Performance - Cash and cash equivalents totaled $79.4 million as of December 31, 2024, compared to $49.9 million at December 31, 2023, representing a 59% increase[9] - Research and development expenses for the year ended December 31, 2024, were $48.7 million, a decrease of approximately 10% from $53.9 million for the year ended December 31, 2023[9] - General and administrative expenses increased to $56.0 million for the year ended December 31, 2024, from $20.6 million in 2023, marking an increase of approximately 172%[9] - The net loss for the year ended December 31, 2024, was $105.6 million, or $0.76 per share, compared to a net loss of $119.8 million, or $1.42 per share, for the year ended December 31, 2023[9] - Total revenue for the year ended December 31, 2024, was $455,000, a significant decrease from $9.99 million in 2023[16] - The accumulated deficit increased to $574.2 million as of December 31, 2024, compared to $468.6 million as of December 31, 2023[14] Regulatory and Strategic Developments - The company received a Complete Response Letter from the FDA regarding the New Drug Application for govorestat for Classic Galactosemia, and is evaluating its response[3] - The company is focused on preparing for potential regulatory interactions regarding govorestat in both Classic Galactosemia and SORD Deficiency[2] - The company appointed key senior executives to enhance its regulatory, medical, and quality affairs capabilities, including Todd F. Baumgartner as Chief Regulatory Officer[3] Asset Management - The total assets increased to $86.7 million as of December 31, 2024, from $54.8 million as of December 31, 2023[14]