Financial Data and Key Metrics Changes - Total net product sales for 2024 were $336.4 million, a 88% increase from $178.9 million in 2023 [24][27] - Fourth quarter 2024 net product sales were $99.4 million, representing a 43% year-over-year increase from $69.5 million [24][25] - The company ended 2024 with cash, cash equivalents, and investments of $293 million, an increase of approximately $7 million from the start of the year [27] Business Line Data and Key Metrics Changes - LIVMARLI's total net product sales for 2024 were $213.3 million, a 50% increase compared to 2023 [25] - Bile acid medicines achieved total net product sales of $123.1 million for 2024, with fourth quarter sales of $35 million, representing approximately 25% growth over the fourth quarter of 2023 [25][26] Market Data and Key Metrics Changes - The company expects to add close to $100 million to its top-line in 2025, with anticipated net product sales between $420 million and $435 million [12][27] - The market for MRM-3379 in Fragile X syndrome is estimated at $1 billion, primarily focusing on the 50,000 male patients in the US [39][40] Company Strategy and Development Direction - The strategic priorities for 2025 include global growth of commercial medicines, advancing the pipeline, selectively pursuing product acquisition opportunities, and maintaining scientific and financial discipline [8][9] - The company aims to expand LIVMARLI's label and drive Volixibat towards pivotal data in adult cholestasis [8][14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong demand for LIVMARLI in Alagille syndrome and PFIC, anticipating continued growth [10][12] - The company is positioned for sustained growth in the rare disease market, leveraging its financial position for potential acquisitions [33][34] Other Important Information - The FDA approved CTEXLI for cerebrotendinous xanthomatosis (CTX) in adults, which is expected to enhance promotional efforts and patient reach [12][51] - The company reported being cash flow positive in 2024 and expects to maintain this status in 2025 [27] Q&A Session Summary Question: Strategic implications of convertible notes and capital allocation - Management highlighted the unique position of the company in the rare disease market, allowing for potential acquisitions and value creation opportunities [33][34] Question: Market penetration and competition for MRM-3379 - The market potential for MRM-3379 is based on a 50,000-patient market in the US, with substantial upside beyond the initial estimate [39][40][42] Question: Alagille market penetration and growth drivers - Current penetration for eligible treatment population in the US is around 40%, with growth driven by new diagnoses and increased patient starts [46][48] Question: Impact of CTX approval on sales trajectory - Management expects a gradual increase in patient diagnoses and treatment conversions to CTEXLI, impacting sales positively over time [51][52] Question: Patient mix for LIVMARLI in Q4 2024 - Most LIVMARLI patients are currently from Alagille syndrome, but there has been a notable increase in PFIC patient starts [56][58] Question: Commercial team expansion for LIVMARLI - No significant expansion of the commercial team is anticipated, as the same prescribing universe will be leveraged for the potential label expansion [62] Question: Timeline for topline data from Volixibat study - Topline data is expected approximately six months after enrollment completion, which is targeted for the second half of 2025 [84][85] Question: Patient experience in the EXPAND study - Positive treatment outcomes have been observed in biliary atresia patients treated through compassionate use, supporting the need for the EXPAND study [90][91]

舒泰神（北京）生物制药股份有限公司 向特定对象发行股票的审核问询函的回复 天衡专字(2023) 00831 号 您可使用手机"扫一扫"或进入"注册会计师行业统一监管平台（http://ac.mcf.mor.cn）"进行营" "进行" 舒泰神(北京)生物制药股份有限公司 向特定对象发行股票的审核问询函的回复 天衡专字(2023) 00831 号 深圳证券交易所: 根据贵所于 2023年3月 16 日出具的《关于舒泰神(北京)生物制药股份 有限公司申请向特定对象发行股票的审核间询函》(审核函(2023)020046号) 所列的问题,天衡会计师事务所(特殊普通合伙)(以下简称"本所"或"申报 会计师")作为舒泰神(北京)生物制药股份有限公司(以下简称"舒泰神"、"公 司"、"发行人"或"申请人")向特定对象发行股票的申报会计师,我们对反馈意见 中需申报会计师核查的相关问题进行了认真核查,现将有关情况汇报说明如下: 一、申报材料显示,本次发行拟募集资金总额不超过58,000万元,全部用 于创新药物研发项目,具体包括 BDB-001 注射液、注射用 STSP-0601、 STSA-1002和STSA-1005联合用药三 ...

CANbridge Pharmaceuticals (01228) H2 2022 Earnings Call March 31, 2023 12:00 PM ET Speaker0 Good morning, everyone. Welcome to Cambridge Pharmaceuticals twenty twenty two twelve Month Annual Results Conference Call. Today, it's a great honor to have Doctor. James Shre, Founder, Chairman and CEO of Cambridge Pharmaceuticals Doctor. Gerald Cox, Chief Medical Officer and Chief Development Strategist Glenn Hassan, Chief Financial Officer and Sophie Hsieh, Director of Investor Relations. Management team will giv ...