Summary of Agios Pharmaceuticals Conference Call Company Overview - **Company**: Agios Pharmaceuticals (AGIO) - **Focus**: Development of transformative medicines for patients with rare diseases, particularly those affecting red blood cells such as pyruvate kinase deficiency (PKD), thalassemia, sickle cell disease, and low-risk myelodysplastic syndromes (MDS) [6][7] Key Pipeline Developments - **Product Highlight**: PyraKine (Mitapivat) - **Mechanism**: Novel approach to enhance red blood cell metabolism, leading to improved energy and healthier cells [7] - **Current Status**: Commercialized for PKD; pursuing indications in thalassemia and sickle cell disease [7] Thalassemia - **Pivotal Studies**: Two phase three studies (ENERGIZE and ENERGIZE T) showed statistical significance on all primary and key secondary endpoints [8] - **Regulatory Filings**: Filed for approval in the US, Europe, Saudi Arabia, and UAE; FDA PDUFA goal date set for September 7, 2025 [8][14] - **Commercial Readiness**: Sales team fully prepared for the launch of PyraKine in thalassemia [9][35] Sickle Cell Disease - **Study Enrollment**: Fully enrolled the Rise Up phase three study, a 52-week trial [9] - **Data Readout**: Expected by year-end 2025 [9] - **Trial Design**: Focuses on hemolytic anemia and vaso-occlusion, with two primary endpoints [42][43] Financial Position - **Balance Sheet**: Approximately $1.4 billion in cash, positioning the company strongly in the current macroeconomic environment [10][12][61] - **Capital Allocation Strategy**: Focus on maximizing value from launches, advancing the pipeline, and exploring external opportunities for growth [64] Market Opportunities - **Thalassemia Patient Population**: Estimated 6,000 adult patients in the US and 70,000 in Saudi Arabia, with a prevalence rate significantly higher in the Middle East [32] - **Regulatory Engagement**: Positive interactions with regulatory bodies across multiple markets, indicating a smooth approval process [24][30] Competitive Landscape - **Treatment Paradigm**: Oral therapies like Mitapivat are positioned as accessible options compared to gene therapies, which may have more complex onboarding processes [54] - **Future Trials**: Plans to initiate a phase two trial for another PK activator, tebipivat, to provide additional treatment options for sickle cell disease [48][50] Upcoming Events - **EHA Presentations**: Agios will present data on pediatric PKD and sickle cell disease at the upcoming EHA conference, showcasing their commitment to advancing research and treatment options [58][59] Conclusion - **Strategic Vision**: Agios aims to build a multibillion-dollar franchise with PyraKine while maintaining a disciplined approach to capital allocation and exploring new opportunities for growth [64]

Summary of PTC Therapeutics Conference Call Company Overview - **Company**: PTC Therapeutics (PTCT) - **Industry**: Biotechnology, focusing on rare diseases and gene therapies Key Points and Arguments Executive Order and Market Position - The recent executive order has unclear ramifications for companies, but PTC Therapeutics expects minimal impact due to its revenue being more international than domestic [4][5] - The company is preparing for the launch of its PKU product, emphasizing a global pricing corridor to maintain product value [5][6] FDA Interactions - PTC has three applications pending with the FDA, with no significant impact from recent agency changes noted [10][12] - The PKU New Drug Application (NDA) is progressing well, with confidence in approval by the PDUFA date [11][12] Translarna Sales in Europe - Despite a negative opinion from the CHMP, PTC is pursuing a country-by-country strategy to commercialize Translarna, with about 50% of countries showing interest [16][17] - The company anticipates achieving 25-30% of European revenue for the year from Translarna [18][20] PKU Launch Strategy - PTC is ready for a global launch of its PKU product, with an early access program initiated in Germany [33][34] - The company has built a robust infrastructure to support the launch, including a patient services team to assist with prior authorizations and patient engagement [38][39] Competitive Landscape for PKU - There are approximately 17,000 individuals with PKU in the US, with a significant unmet need as 85-90% are not on current therapies [44][45] - PTC's PKU therapy is positioned as a better option compared to existing treatments, with a focus on diet liberalization and improved tolerability [47][50] Pricing Strategy - PTC plans to price its PKU therapy at a premium to Palynziq, justified by strong efficacy data and a favorable safety profile [54][55] - The company believes it could achieve a billion-dollar opportunity in the US alone with low double-digit market penetration [57][58] PTC 518 and Huntington's Disease - PTC 518 is positioned as a potential first-ever disease-modifying therapy for Huntington's disease, with promising early data [63][64] - The company is focused on understanding the optimal patient population for this therapy, emphasizing the importance of early-stage patients [66][68] Competitive Landscape for Huntington's Disease - PTC believes that its oral therapy will be more attractive than gene therapies due to its safety, tolerability, and ability to be administered early in the disease process [78][79] - The partnership with Novartis is expected to enhance commercialization efforts for PTC 518 [76][79] Additional Important Content - The company is optimistic about the global opportunity for its therapies, with plans for launches in Japan and other markets [59][61] - PTC is committed to addressing unmet needs in rare diseases, which is a key focus for its product development strategy [32][33]

Financial Data and Key Metrics Changes - Total revenues for Q1 2025 reached $111.6 million, representing a 61% growth compared to Q1 2024 [5] - The company updated its full-year revenue guidance to $435 million to $450 million, driven by strong growth across all commercial medicines [5][10] - Net product revenue for Q1 2025 was $111.6 million, up from $69.2 million in Q1 2024 [13] - Cash, cash equivalents, and investments at March 31, 2025, were $298.6 million, compared to $292.8 million at the beginning of the year [13] - Operating expenses for Q1 2025 totaled $126.8 million, including R&D expenses of $46 million and SG&A expenses of $57.7 million [13][15] Business Line Data and Key Metrics Changes - Total net product sales for LIVMARLI reached $73.2 million in Q1 2025, a 70% increase compared to Q1 2024 [10] - U.S. LIVMARLI sales were $49.5 million, driven by robust new patient demand [10] - International LIVMARLI sales were $23.7 million, with strong demand growth in direct European markets [10] - Bile acid products generated $38.4 million in net product sales, representing a 47% growth year-over-year [12] Market Data and Key Metrics Changes - The approval of LIVMARLI's tablet formulation is expected to enhance patient convenience and drive adoption among older children and adolescents [10][24] - The company has seen an increase in new CTX patients since the FDA approval of Citexly in February 2025 [12][66] - The market for PSC is characterized as unsatisfied, with no FDA-approved therapies currently available, positioning velixibat favorably [46] Company Strategy and Development Direction - The company is focused on advancing its commercial medicines and high-impact pipeline, with three important regulatory approvals received since the start of the year [6][17] - The VISTA study of velixibat is nearing completion of enrollment, with top-line data expected in Q2 2026 [7][18] - The company plans to initiate a Phase II study for MRM-3379 in fragile X syndrome later this year [9][18] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the strong start to the year and the growth of commercial medicines exceeding initial expectations [17] - The company is well-positioned for continued leadership in rare diseases, with a strong financial position and cash flow positive outlook for the full year [15][72] Other Important Information - The cash contribution margin from the commercial business improved from approximately 47% in Q1 2024 to approximately 53% in Q1 2025 [15] - The company is focused on raising disease awareness to improve patient identification for underdiagnosed conditions [66] Q&A Session Summary Question: Insights on the additional velexibat PBC data - Management noted that the data matured positively in the interim analysis, with strong response curves [20][21] Question: Additional IP around the Libmarli tablet formulation - The tablet formulation has resulted in novel IP, with an allowed patent expected to extend coverage [22][23] Question: Comparison of interim Phase II PBC data with competitors - Management highlighted strengths in the pelixibat program, particularly in placebo-adjusted differences [30] Question: Update on Marley access versus competitors - Access to LIVMARLI in the U.S. is strong, with no barriers anticipated for either indication [38] Question: Expectations for the VISTA study - Management is optimistic about velexibat's activity level, using VANTAGE as a benchmark [42] Question: Update on patient identification for Citexly - There has been a nice uptick in patient identification since the FDA approval, attributed to promotional efforts [66][64] Question: Sustainability of profitability into 2026 - The company expects to be cash flow positive for the full year but does not anticipate profitability in a GAAP sense soon [72]

Summary of Scinai Immunotherapeutics (SCNI) Conference Call Company and Industry Overview - **Company**: Scinai Immunotherapeutics (SCNI) - **Industry**: Biotechnology, specifically focusing on treatments for rare autoimmune diseases such as pemphigus and Stevens Johnson syndrome (SJS) Key Points and Arguments 1. **Acquisition Announcement**: Scinai has entered into a binding option agreement to acquire the Italian biotech company, Pincell, and submitted a grant application for €12 million to develop PC-one hundred eleven [1][2] 2. **Overview of Diseases**: The call discussed pemphigus, SJS, and toxic epidermal necrolysis (TEN), highlighting their symptoms, implications, and current treatment landscape [8][9] 3. **Medical Need**: There is a significant unmet medical need for effective treatments for pemphigus and SJS, with pemphigus affecting approximately 2 million people globally and SJS being a rare but severe condition [14][19] 4. **Current Treatments**: Current therapies for pemphigus include glucocorticoids and rituximab, which have severe side effects and a high relapse rate. There are no approved therapies for SJS [21][22][44] 5. **PC-one hundred eleven Mechanism**: PC-one hundred eleven is a fully human monoclonal antibody targeting soluble Fas ligand, which plays a critical role in the pathogenesis of pemphigus and SJS. It blocks keratinocyte apoptosis and acantholysis, potentially preventing blister formation [26][27][43] 6. **Efficacy Comparison**: PC-one hundred eleven is expected to have a rapid onset of action and a better safety profile compared to current treatments like rituximab, which has a medium time to remission of around six months [41][43] 7. **Development Plans**: Scinai plans to conduct parallel development for PC-one hundred eleven, focusing on chronic pemphigus and acute SJS, with an expected approval timeline of three to four years [52][53] 8. **Market Size**: The addressable patient population for pemphigus in target countries is around 164,000, with a focus on relapsing refractory patients. For SJS, the addressable population is estimated at 15,000 [56][58] 9. **Pricing and Revenue Potential**: Potential peak revenues for PC-one hundred eleven are estimated at around $500 million, with an upside of over $1 billion for both pemphigus and SJS indications [63][64] 10. **Partnership Opportunities**: There is significant interest from pharmaceutical companies in smaller indications with transformational efficacy, which could lead to partnerships for PC-one hundred eleven [66][67] Additional Important Content - **Challenges in Clinical Trials**: The company plans to address challenges in conducting clinical trials for rare diseases by collaborating with scientific societies and patient advocacy groups to identify suitable trial sites [55] - **Safety Considerations**: The non-immunosuppressive nature of PC-one hundred eleven is expected to reduce the risk of infections and complications associated with current treatments, making it a safer option for patients [70] - **Combination Therapy Potential**: PC-one hundred eleven could be used in combination with existing therapies like rituximab for pemphigus and could enhance supportive care for SJS patients [49][51] This summary encapsulates the critical insights and developments discussed during the conference call, emphasizing the potential of PC-one hundred eleven in addressing significant medical needs in the treatment of rare autoimmune diseases.

Financial Data and Key Metrics Changes - BioMarin reported a revenue growth of 15% in Q1 2025, totaling $745 million compared to the same period last year [12] - Non-GAAP earnings per share reached $1.13, representing a 59% year-over-year increase, indicating significant profitability expansion [5][19] - Non-GAAP operating margin expanded to 35.7%, an increase of 11.9 percentage points year-over-year [15] Business Line Data and Key Metrics Changes - Voxzogo's global revenue reached $214 million, a 40% increase year-over-year, continuing its strong growth trajectory since approval [13] - Revenue from the Enzyme Therapies business unit grew 8% year-over-year to $484 million, with Palynziq contributing a 22% increase compared to Q1 2024 [13][22] - Full-year revenue expectations for Voxzogo are between $900 million and $950 million, representing a 26% growth at the midpoint [14] Market Data and Key Metrics Changes - Approximately two-thirds of BioMarin's total revenues originate from outside the United States, providing insulation from U.S. macroeconomic conditions [7][9] - The company is analyzing potential exposure to pharmaceutical tariffs and believes its global revenue base offers protection against such impacts [8][9] Company Strategy and Development Direction - BioMarin is focused on implementing changes to its strategy and operating model announced in September 2024, aiming for record full-year performance in 2025 [6][11] - The company is advancing its innovation strategy, with pivotal studies for Voxzogo and plans for new product applications in the second half of 2025 [10][24] - BioMarin aims to leverage its established global capabilities to support growth and innovation, particularly in rare disease treatments [11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in BioMarin's ability to navigate market uncertainties due to its focus on rare conditions and strong cash generation [6][7] - The company anticipates continued strong performance and innovation in the coming quarters, supported by its strategic transformation [11][19] Other Important Information - Non-GAAP R&D expenses were $147 million, lower than the same quarter in 2024, reflecting a reprioritization of R&D efforts [14] - Non-GAAP SG&A expenses decreased to $183 million due to cost transformation initiatives [15] Q&A Session Summary Question: Will Q2 revenue for Voxzogo decline? - Management indicated that while Q1 showed growth, Q2 revenue may appear flat due to global order dynamics, with expectations for stronger growth in the second half of the year [35][36] Question: How could potential U.S. tariffs impact financials? - Management stated that current guidance includes modest impacts from existing tariffs, but they are modeling various scenarios for potential future tariffs [36][37] Question: What strategies are being implemented to drive further adoption of Voxzogo? - The company is focusing on increasing awareness and expanding the prescriber base, particularly among pediatric endocrinologists, to drive adoption across all age groups [55][56] Question: What is the timeline for BMN 333 and its potential indications? - BMN 333 is in a Phase I study, with plans for pivotal studies in 2027, and the company is evaluating its future role in additional indications [84] Question: How have U.S. versus ex-U.S. sales evolved for Voxzogo? - Approximately 75% of Voxzogo revenues come from outside the U.S., and this split is expected to fluctuate as the brand matures [88]

Financial Data and Key Metrics Changes - In Q1 2025, net PyroKine revenue was $8.7 million, a 6% increase compared to $8.2 million in Q1 2024 [30] - Revenue decreased by 19% compared to Q4 2024, primarily due to year-end stocking benefits and adjustments to revenue reserves [33] - Cost of sales for the quarter was $1.1 million, R&D expenses were $72.7 million, an increase of $4.1 million compared to Q1 2024, and SG&A expenses were $41.5 million, an increase of $10.5 million compared to the prior year [36] Business Line Data and Key Metrics Changes - The company reported a 5% increase in new prescriptions and new patient starts in Q1 2025, translating to 136 net patients on therapy, also a 5% increase versus the prior quarter [31] - The company expects growth in net revenue to be in the 10% to 20% range on an annual basis, consistent with other rare disease launches [34] Market Data and Key Metrics Changes - The thalassemia market is characterized by a well-defined patient population, with approximately 6,000 adults diagnosed in the U.S., and the company estimates that PyroKine's initial launch focus will address about 65% of the adult thalassemia patient population [27][28] - The sickle cell disease community is estimated to include 120,000 to 135,000 individuals in the U.S. and EU5, with a global prevalence exceeding 3 million [13] Company Strategy and Development Direction - The company aims to maximize the potential of the PyroKine franchise, advance and diversify key pipeline programs, and strategically focus capital deployment to sustain growth [7] - The company is preparing for a potential U.S. approval and launch of PyroKine for thalassemia in September 2025, followed by sickle cell disease in 2026 [6][16] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming PDUFA date and the potential to serve patients with unmet needs in thalassemia and sickle cell disease [46][66] - The company is confident in its strong balance sheet, which provides financial independence to support potential launches and advance existing programs [37][39] Other Important Information - The company has a robust early and mid-stage pipeline, with plans to file an investigational new drug application for AG-236 targeting polycythemia vera in mid-2025 [9][20] - The company has engaged in disease state education campaigns to prepare for the launch of PyroKine, focusing on both patients and healthcare providers [24] Q&A Session Summary Question: Has the mid-cycle review happened, and what are the next steps? - Management confirmed that no advisory committee meeting is planned and that the regulatory review is ongoing, with strong engagement with the FDA [45][46] Question: How has the withdrawal of Oxbreda impacted trial recruitment? - Management noted that while the withdrawal was disappointing, it has not affected their clinical trial conduct, and they see a significant opportunity in the sickle cell market [48][49] Question: What are the plans for launching mitapivat ex-U.S.? - The company plans to prioritize the Gulf region and Europe for launches, leveraging partnerships for effective execution [54][57] Question: Any changes in communication frequency with the FDA? - Management stated that communication with the FDA remains consistent, with no changes due to recent agency reshuffling [65][66] Question: Rationale for starting the Phase II tebapivat study in sickle cell? - Management expressed confidence in the unmet need for therapies in sickle cell disease and the potential to grow the patient population with multiple products [72][73] Question: Plans for marketing to non-transfusion dependent patients? - The company plans to focus on both transfusion-dependent and non-transfusion-dependent patients, emphasizing the value proposition of PyroKine for both groups [78] Question: Anticipated evolution of PyroKine's pricing? - Pricing decisions will be based on the value proposition of the label received, with positive feedback from payers regarding the product profile [98]

Financial Data and Key Metrics Changes - Total revenue for Q1 2025 reached $125.2 million, a 13% increase year-over-year, with a 15% growth at constant exchange rates [33][34] - GAAP net loss for Q1 2025 was $21.7 million, or $0.07 per share, compared to a net loss of $48.4 million, or $0.16 per share in Q1 2024 [34] - Non-GAAP net income for Q1 2025 was $9 million, or $0.03 per share, compared to a non-GAAP net loss of $4.6 million, or $0.02 per share in the same period last year [34] Business Line Data and Key Metrics Changes - Galafold revenue reached $104.2 million, up 6% at constant exchange rates, with new patient starts globally increasing by 14% [9][11] - Pombility revenue for Q1 2025 was $21 million, up 92% at constant exchange rates, primarily driven by the US, Germany, and Spain [13][14] - Adjusted revenue growth guidance for Pombility and Upholda was revised from 65%-85% to 50%-65% at constant exchange rates [36] Market Data and Key Metrics Changes - The US contributed significantly to Galafold's growth, with an 18% increase in sales, while UK sales were impacted by higher VPAG rebates [11][45] - The company achieved a record number of patients on Galafold in Australia following its first-line listing [10] - Revenue was recorded in seven countries, with new patient starts anticipated in four additional countries in Q2 2025 [18] Company Strategy and Development Direction - The company entered a strategic collaboration with Dimerix to commercialize DMX200, a treatment for FSGS, enhancing its late-stage pipeline [24][26] - The company is focused on diversifying its supply chain and has onshored a portion of drug product manufacturing to the US [6][19] - The long-term outlook for Galafold and Pombility remains positive, with expectations of reaching combined sales of $1 billion by 2028 [7] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving GAAP profitability in the second half of 2025, despite challenges faced in Q1 [7][36] - The management acknowledged the impact of unexpected factors on quarterly revenues but remains optimistic about sales acceleration for the remainder of the year [7][15] - The company is well-positioned to create substantial value for shareholders and deliver on its mission for patients [7][41] Other Important Information - The company reiterated its financial discipline and updated its full-year revenue growth guidance to 15%-22% from the previous 17%-24% [36] - The company is committed to maintaining high compliance and adherence rates for its therapies, which are crucial for long-term growth [16] Q&A Session Summary Question: What led to the higher-than-anticipated rebate for Galafold and Pombility? - Management explained that the rebate was a negotiated rate by the industry association in the UK, which was higher than expected, impacting revenue throughout the year [45][46] Question: What has changed regarding the cadence of ex-US launches and patient starts? - Management noted that reimbursement settlements led to a delay in patient starts, which are now expected to accelerate in the second half of the year [49][51] Question: Can you elaborate on the diligence process for the DMX200 licensing agreement? - Management highlighted that the diligence process involved careful review of data, market research, and discussions with physicians, leading to confidence in the asset's differentiation [58][60] Question: What is the expected timeline for the next interim analysis for DMX200? - Management indicated that the timeline for the next interim analysis could be around nine months, depending on the collaboration with Parasol and the FDA [72][74] Question: Will the VPAG rebate impact be recurring? - Management clarified that while the rebate will impact revenue this year, it is not expected to be a recurring event [86][87]

Financial Data and Key Metrics Changes - The company achieved total revenue of $125.2 million for Q1 2025, representing a 13% increase year-over-year, and a 15% increase at constant exchange rates [33] - GAAP net loss for Q1 2025 was $21.7 million, or $0.07 per share, compared to a net loss of $48.4 million, or $0.16 per share in Q1 2024 [34] - Non-GAAP net income was $9 million, or $0.03 per share, compared to a non-GAAP net loss of $4.6 million, or $0.02 per share in the same period last year [35] - Cash, cash equivalents, and marketable securities were $251 million as of March 31, 2025, compared to $250 million at December 31, 2024 [35] Business Line Data and Key Metrics Changes - Revenue for Galafold reached $104.2 million, up 6% at constant exchange rates, with a 14% increase in new patient starts globally [10] - Revenue for Pombility reached $21 million, up 92% at constant exchange rates, driven primarily by the US, Germany, and Spain [14] - The company adjusted its revenue growth guidance for Pombility and Upholda from 65%-85% to 50%-65% at constant exchange rates [36] Market Data and Key Metrics Changes - The US contributed significantly to growth, with 60% of total revenue generated outside the US and 40% from within [33] - The UK sales were impacted by higher VPAG rebates than previously anticipated, with the negotiated rate increasing from 15% to 22% [46] - The company recorded revenue in Q1 across seven countries, including the US, Germany, Austria, Spain, the UK, Switzerland, and Sweden [18] Company Strategy and Development Direction - The company entered a strategic collaboration with Dimerix to commercialize DMX200, a treatment for FSGS, enhancing its late-stage pipeline [25] - The company aims to achieve GAAP profitability in the second half of 2025 while maintaining financial discipline [9] - The company is focused on expanding its portfolio in rare diseases, particularly in neuromuscular and rare kidney diseases [92] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term growth trajectory, expecting continued double-digit revenue growth in core business areas [6] - The management acknowledged challenges in the UK market due to unexpected rebate increases but remains optimistic about patient growth compensating for this [46] - The company anticipates a significant acceleration in patient starts in the second half of 2025, particularly in new launch markets [52] Other Important Information - The company is onshoring a portion of its drug product manufacturing to enhance supply chain diversification [8] - The company reiterated its commitment to maintaining high compliance and adherence rates for its therapies [13] - The company is actively enrolling in pediatric clinical trials and generating real-world data to support its therapies [24] Q&A Session Summary Question: What led to the higher-than-anticipated rebate for Galafold and Pombility? - Management explained that the rebate was a negotiated rate by the industry association in the UK, which unexpectedly increased from 15% to 22% [46] Question: What has changed regarding the cadence of ex-US launches and patient starts? - Management noted that reimbursement settlements led to delays in patient starts, which are now expected to accelerate in the second half of the year [50][52] Question: Can you elaborate on the diligence process for the DMX200 licensing agreement? - Management highlighted that the diligence included careful review of data, market research, and discussions with physicians, leading to confidence in the asset's differentiation [60] Question: What is the expected timeline for the next interim analysis for DMX200? - Management indicated that the timeline for the next interim analysis could be around nine months, depending on the collaboration with Parasol and the FDA [71] Question: How does the company plan to position Pombility against traditional ERT in new markets? - Management emphasized the importance of demonstrating the product's value proposition and differentiated data to drive adoption in new markets [90]

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 ...