Financial Performance - The company reported a net loss of 28.8million,or0.45 per share, for Q4 2024, compared to a net loss of 13.0million,or0.30 per share, for Q4 2023[7]. - For the full year 2024, the company reported a net loss of 80.6million,or1.32 per share, compared to a net loss of 36.9million,or0.84 per share, for 2023[10]. - Net loss for the year was 80,604,000in2024comparedto36,949,000 in 2023, reflecting a 118.5% increase in losses[21]. - The company reported a net loss per share of 1.32in2024,comparedto0.84 in 2023, indicating a worsening loss per share by 57.1%[21]. Research and Development - Research and development expenses for Q4 2024 were 26.7million,asignificantincreasefrom10.6 million in Q4 2023, primarily due to a 15.0millionriseinnomlabofuspmanufacturingcosts[8].−Researchanddevelopmentexpensesforthefullyear2024were73.3 million, up from 27.7millionin2023,drivenbya36.1 million increase in nomlabofusp manufacturing costs[11]. - Research and development expenses rose significantly to 73,278,000in2024from27,670,000 in 2023, an increase of 164.5%[21]. Cash and Assets - As of December 31, 2024, the company had cash, cash equivalents, and marketable securities totaling 183.5million,withaprojectedcashrunwayintothesecondquarterof2026[5].−Totalassetsincreasedto200,225,000 in 2024 from 95,935,000in2023,representingagrowthof108.5195,304,000 in 2024, up from 90,175,000in2023,markinga116.5171,812,000 in 2024 from 81,720,000in2023,agrowthof109.828,413,000 in 2024 from 14,215,000in2023,anincreaseof100.010.3 million, compared to 4.8millionin2023,primarilyduetoincreasedinterestincome[13].−Otherincome,net,improvedto10,286,000 in 2024 from 4,809,000in2023,anincreaseof114.517,612,000 in 2024 from $14,088,000 in 2023, a rise of 25.5%[21]. Future Plans and Studies - The company plans to submit a Biologics License Application (BLA) seeking accelerated approval by the end of 2025[5]. - The ongoing open label extension (OLE) study continues to enroll participants, with data from the 50 mg dose expected to be reported in September 2025[6]. - The company is on track to initiate a global Phase 3 study in mid-2025, with feedback obtained from both FDA and EMA on the study protocol[6]. - The FDA has indicated openness to considering skin FXN concentration as a surrogate endpoint for accelerated approval, which could support the BLA submission[6]. Shareholder Information - The weighted average common shares outstanding increased to 61,256,084 in 2024 from 43,901,241 in 2023, a growth of 39.5%[21].