Financial Performance - Total revenues for Q4 2024 were 16.9million,asignificantincreasefrom1.7 million in Q4 2023, and total revenues for the full year 2024 were 18.5millioncomparedto7.5 million in 2023[20]. - Total revenues for the year ended December 31, 2024, were 18.4million,asignificantincreasefrom7.53 million in 2023, representing a growth of approximately 144%[35]. - License and milestone revenues for Q4 2024 reached 15million,comparedto1 million in Q4 2023, indicating a 1400% increase[35]. - The net loss for Q4 2024 was 150.0million,or(1.26) per share, compared to a net loss of 136.9million,or(1.38) per share, in Q4 2023[23]. - The company reported a net loss of 589.5millionfortheyearendedDecember31,2024,comparedtoanetlossof526.2 million in 2023, representing an increase in losses of approximately 12%[35]. Expenses - R&D expenses for Q4 2024 were 93.6million,upfrom85.0 million in Q4 2023, with full year R&D expenses totaling 339.4millioncomparedto330.1 million in 2023[21]. - G&A expenses for Q4 2024 were 62.3million,comparedto44.1 million in Q4 2023, with full year G&A expenses reaching 215.3millionversus173.6 million in 2023[22]. Cash and Investments - As of December 31, 2024, the company had approximately 1.2billionincash,cashequivalents,andinvestments,adecreaseofapproximately60 million from 1.3billionatSeptember30,2024[17].−Cashandshort−terminvestmentsasofDecember31,2024,totaled1,076.0 million, up from 614.8millionattheendof2023,markinga751,401.7 million as of December 31, 2024, compared to 824.3millionin2023,reflectingagrowthofapproximately701,537.0 million as of December 31, 2024, compared to 1,210.6millionin2023,indicatinganincreaseofabout272,701.8 million as of December 31, 2024, from 2,112.2millionin2023,representingariseofapproximately28670 million and 710million,whichincludesinvestmentstowardcommercialreadinessforaficamten[25].−TheFDAacceptedtheNDAforaficamtenwithaPDUFAtargetactiondateofSeptember26,2025,andthecompanyispreparingforapotentialcommerciallaunchin2H2025[4][11].−ThecompanyenteredintoacollaborationandlicenseagreementwithBayerfortheexclusivedevelopmentandcommercializationofaficamteninJapan,receivingapaymentof52.4 million (€50 million) for this agreement[19]. - The company expects to report topline results from the MAPLE-HCM trial in Q2 2025 and aims to complete patient enrollment for the ACACIA-HCM trial in 2H 2025[18]. - The company aims to complete enrollment of clinical trials ACACIA-HCM, CEDAR-HCM, and AMBER-HFpEF in the second half of 2025[30]. - Future interactions with the FDA regarding drug candidates are anticipated, with a focus on regulatory approvals to enable commercialization of aficamten[30]. Vision and Goals - The company is advancing its Vision 2030 strategic objectives to become a leading muscle-focused specialty biopharmaceutical company[13].