Cefotaxime Sodium

注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需要得到授权。PART 01周报概述随着全球医药行业的快速发展，新药研发与创新已成为推动行业进步的重要动力。近期，根据摩熵医药数据统计，新药申请与审批获批频繁，显示出医药创新领域的活跃态势。本文将深入分析2025年4月28日至2025年5月4日期间，国内外新药申请、临床试验批准、仿制药一致性评价等多个方面的最新进展，为用户提供全面的行业资讯。PART 02国内14款新药IND获批根据摩熵医药数据库统计，2025年4月28日至2025年5月4日期间共有56个创新药/改良型新药临床申请/上市申请获国家药品监督管理局药品审评中心（CDE）承办（按受理号统计，不含补充申请）。其中国产药品受理号47个，进口药品受理号9个。本周共计14款创新药/改良型新药临床试验申请获得“默示许可”，包括化学药9款，生物药5款。本周获批临床创新药/改良型新药（部分）信息速览(不含补充申请)注：完整数据可识别“文末”二维码下载查看PART 03本周全球TOP10创新药研发进展在全球创新药研发领域，4月29日，中国国家药监局药品审评中心（CDE）官网公示，百济神州申报的索托克拉片的上市申请获得受理。该上市申请已经被CDE正式纳入优先审评，适用于治疗既往接受过治疗的慢性淋巴细胞白血病（CLL）/ 小淋巴细胞淋巴瘤（SLL）成人患者。公开资料显示，索托克拉片（sonrotoclax）是百济神州在研的新一代BCL2抑制剂，旨在阻断可帮助肿瘤细胞存活的BCL2蛋白。Sonrotoclax在多种B细胞恶性肿瘤中表现出良好的临床活性，迄今为止共有超过1800例患者入组了该药物全球研发计划。美国FDA还已经授予该产品快速通道资格，用于治疗套细胞淋巴瘤（MCL）和华氏巨球蛋白血症（WM）患者。该产品治疗既往接受过抗CD20治疗和BTKi治疗的套细胞淋巴瘤（MCL）成人患者也已经被CDE纳入优先审评。5月2日，诺和诺德（Novo Nordisk）宣布，美国FDA已受理为Wegovy（司美格鲁肽）每日一次、25mg口服剂型递交的新药申请（NDA），拟用于患有肥胖症、或伴有一种或多种合并症的超重成人的长期体重管理，以及用于降低肥胖或超重的心血管疾病成人患者发生主要不良心血管事件（MACE）的风险。新闻稿指出，若获批准，Wegovy将成为首个用于长期体重管理的口服胰高血糖素样肽-1（GLP-1）制剂。FDA预计将在2025年第四季度完成这一新药申请的审评。本周全球 TOP10 创新药研发进展截图来源：摩熵咨询周报PART 04本周全球TOP10临床试验结果本周全球TOP10临床结果中，4月30日，德昇济医药（D3 Bio）宣布，其自主研发的KRAS G12C抑制剂D3S-001的最新临床研究成果荣登国际期刊Nature Medicine（截至发稿日最新影响因子为58.7），并获选2025年美国癌症研究协会（AACR）年会临床口头报告。研究结果证实，D3S-001凭借独特的分子作用机制、优异的安全性特征及临床疗效，在包括一代KRAS G12C抑制剂耐药患者在内的KRAS G12C突变型癌症人群中，展现出显著的差异化治疗优势和best-in-class潜力。在一项1a/1b期临床研究（NCT05410145）中，D3S-001在KRAS G12C抑制剂初治的晚期实体瘤患者中展现出73.5%的客观缓解率（ORR），其中：非小细胞肺癌（NSCLC）：66.7%，结直肠癌（CRC）：88.9%，胰腺癌（PDAC）：75.0%。此外，在20名接受过一代KRAS G12C抑制剂（包括已获批的KRAS G12C抑制剂以及其它临床试验阶段的抑制剂）治疗后出现疾病进展的NSCLC患者中，D3S-001实现了30.0%的客观缓解率（ORR）和80.0%的疾病控制率（DCR），这提供了直接的临床证据，证明其具有克服第一代G12Ci疗法产生的获得性耐药的潜力。本周全球 TOP10 积极/失败临床结果截图来源：摩熵咨询周报PART 052款品种过评，广州白云山领跑根据摩熵医药数据库统计，2025年4月28日至2025年5月4日期间共有62项仿制药申报上市/申报临床获CDE承办，其中新注册分类上市申请受理号55项（包括化药3类，4类），新注册分类临床申请受理号3项（包括化药4类），一致性评价申请4项。本周2个品种通过一致性评价（按受理号计3项），无品种视同通过一致性评价，无生物类似物注册申报动态。截图来源：摩熵咨询周报本周过评/视同过评品种主要为系统用抗感染药物；过评/视同过评产品剂型主要为注射剂。本周注射用头孢噻肟钠过评/视同过评受理号数量最多达2个，本周各品种过评/视同过评企业均为1家；本周各品种过评/视同过评品种数均为1种，本周过评/视同过评企业共包括广州白云山天心制药股份有限公司和陕西九州制药有限责任公司2家企业。截图来源：摩熵咨询周报本周无首次过评/视同过评品种，无过评/视同过评达7家企业品种。摩熵咨询本期完整周报识别二维码领取下载往期周报回顾80款新药IND获批！114款品种过评，内蒙古白医制药过评领跑…64款新药IND获批！12款品种过评，河北天成药业领跑…52款新药IND获批！52款品种过评，山东新时代过评领跑…43款新药IND获批！67款品种过评，晖致医药过评领跑…END本文为原创文章，转载请留言获取授权近期热门资源获取数据透视：中药创新药、经典验方、改良型新药、同名同方的申报、获批、销售情况-2025042023H2-2024H1中国药品分析报告-2025042024年中国1类新药靶点白皮书-202503中国AI医疗健康企业创新发展百强榜单-202502解码护肤抗衰：消费偏好洞察与市场格局分析-2025022024年FDA批准上市的新药分析报告-2025012024年NMPA批准上市的新药分析报告-2025012024年医保谈判及市场分析报告-2025.012024年中国医疗健康投融资全景洞察报告-202501小分子化药白皮书（上）-2025012024医美注射材料市场发展分析报告-202412中国放射性药物产业白皮书-202410近期更多摩熵咨询热门报告，识别下方二维码领取联系我们，体验摩熵医药更多专业服务会议合作园区服务数据库咨询定制服务媒体合作👆👆👆点击上方图片，即可开启摩熵化学数据查询点击阅读原文，申请摩熵医药企业版免费试用！

- First quarter 2025 total revenue of $111.6 million; 2025 guidance increased to $435 to $450 million - VISTAS study in PSC expected to complete enrollment in third quarter of 2025; topline data expected in second quarter of 2026 - LIVMARLI oral tablet formulation FDA approved - Conference call to provide business updates today, May 7 at 1:30 p.m. PT/4:30 p.m. ET FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for the first quarter 2025 and provided a business update. “It’s been a strong start to the year with commercial growth and multiple milestones achieved across our pipeline,” said Chris Peetz, chief executive officer of Mirum. “We’re pleased with the FDA’s approval of LIMARLI’s tablet formulation, which provides more options for the ALGS and PFIC community in the U.S. in the form of a convenient single tablet dose. Our VISTAS study of volixibat in PSC is enrolling well and we expect to complete enrollment in the third quarter of this year. We’re excited to continue our strong execution across our commercial medicines and pipeline throughout the year.” Commercial: Raising full year revenue guidance to $435 to $450 million First quarter 2025 global net product sales of $111.6 million. First quarter 2025 LIVMARLI net product sales were $73.2 million representing 71% growth over first quarter 2024 net product sales. Bile Acid Medicines net product sales were $38.4 million representing 47% growth over first quarter 2024 net product sales. LIVMARLI oral tablet formulation approved. LIVMARLI approved in Japan for ALGS and PFIC. CTEXLI (chenodiol) approved in the US for cerebrotendinous xanthomatosis. Regulatory and Pipeline: Clinical milestones on track Volixibat VISTAS study in primary sclerosing cholangitis (PSC) expected to complete enrollment in third quarter of 2025; topline data expected in the second quarter of 2026. Oral presentation of 28-week interim data from volixibat in PBC (VANTAGE study) at EASL on Friday May 9th at 9:30am CEST. Volixibat VANTAGE study in primary biliary cholangitis (PBC) expected to complete enrollment in 2026. LIVMARLI EXPAND Phase 3 study for pruritus in rare cholestatic conditions expected to complete enrollment in 2026. Expect to initiate Phase 2 study for MRM-3379 in Fragile X Syndrome (FXS) in 2025. Corporate and Financial: Strong balance sheet and financial independence Total revenue for the quarter ended March 31, 2025, was $111.6 million compared to $69.2 million for the quarter ended March 31, 2024. Total operating expenses were $126.8 million for the quarter ended March 31, 2025 compared to $95.7 million for the quarter ended March 31, 2024. Total operating expenses for the quarter ended March 31, 2025 included $21.9 million of non-cash stock-based compensation, intangible amortization, and other non-cash expenses compared to $17.1 million for the quarter ended March 31, 2024. As of March 31, 2025, Mirum had unrestricted cash, cash equivalents and investments of $298.6 million compared to $292.8 million as of December 31, 2024. Business Update Conference Call Mirum will host a conference call today, May 7th at 1:30 p.m. PT/4:30 p.m. ET, to provide business updates. Join the call using the following details: Conference Call Details: U.S./Toll-Free: +1 833 470 1428 International: +1 404 975 4839 Passcode: 549600 You may also access the call via webcast by visiting the Events & Presentations section on Mirum’s website. A replay of this webcast will be available for 30 days. About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets LIVMARLI® (maralixibat) oral solution is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI.com. LIVMARLI has received orphan designation for ALGS and PFIC. LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website. IMPORTANT SAFETY INFORMATION Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein. LIVMARLI can cause side effects, including Liver injury. Changes in certain liver tests are common in patients with ALGS and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal. Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you. A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with Alagille syndrome and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects. US Prescribing Information EU SmPC Canadian Product Monograph About Volixibat Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study), and primary biliary cholangitis (PBC) (VANTAGE study). In June, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as meaningful reductions in serum bile acids and improvements in fatigue for patients treated with volixibat. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate. Volixibat has been granted breakthrough therapy designation for the treatment of PBC. About CHOLBAM® (cholic acid) capsules The FDA approved CHOLBAM (cholic acid) capsules in March 2015, the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for adjunctive treatment of patients with peroxisome biogenesis disorder-Zellweger spectrum disorder. The effectiveness of CHOLBAM has been demonstrated in clinical trials for bile acid synthesis disorders and the adjunctive treatment of peroxisomal disorders. An estimated 200 to 300 patients are current candidates for therapy. CHOLBAM® (cholic acid) Indication CHOLBAM is a bile acid indicated for Treatment of bile acid synthesis disorders due to single enzyme defects. Adjunctive treatment of peroxisomal disorders, including Zellweger spectrum disorders, in patients who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption. LIMITATIONS OF USE The safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects or peroxisomal disorders, including Zellweger spectrum disorders, have not been established. IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS – Exacerbation of liver impairment Monitor liver function and discontinue CHOLBAM in patients who develop worsening of liver function while on treatment. Concurrent elevations of serum gamma glutamyltransferase (GGT) and alanine aminotransferase (ALT) may indicate CHOLBAM overdose. Discontinue treatment with CHOLBAM at any time if there are clinical or laboratory indicators of worsening liver function or cholestasis. ADVERSE REACTIONS The most common adverse reactions (≥1%) are diarrhea, reflux esophagitis, malaise, jaundice, skin lesion, nausea, abdominal pain, intestinal polyp, urinary tract infection, and peripheral neuropathy. Please see full Prescribing Information for additional Important Safety Information. About CTEXLI™ (chenodiol) tablets CTEXLI™ (chenodiol) tablets is FDA-approved for the treatment of adults with cerebrotendinous xanthomatosis (CTX). Chenodiol is another name for chenodeoxycholic acid (CDCA). CDCA is a naturally occurring bile acid that was originally approved for the treatment of people with radiolucent stones in the gallbladder. CTEXLI was evaluated as part of the Phase 3 RESTORE study, the first and only clinical trial for CTX. CTX is a rare progressive disease that can affect the brain, spinal cord, tendons, eyes and arteries. IMPORTANT SAFETY INFORMATION CTEXLI can cause side effects, including: Liver Injury: You will need to undergo laboratory testing before starting and while taking CTEXLI to check your liver function. Changes in certain liver tests may occur during treatment and may be a sign of liver injury. This can be serious. Stop taking CTEXLI immediately and tell your healthcare provider right away if you get any signs or symptoms of liver problems, including, stomach (abdomen) pain, bruising, dark-colored urine, feeling tired (fatigue), bleeding, yellowing of the skin and eyes, nausea, and itching. Most Common Side Effects: Diarrhea, headache, stomach pain, constipation, high blood pressure, muscular weakness, and upper respiratory tract infection. Tell your healthcare provider about all the medications that you take, as CTEXLI may interact with other medicines. US Prescribing Information About Mirum Pharmaceuticals Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum has initiated the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X). Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, significant increase or continued commercial results for our approved medicines, including continued financial growth, the potential achievement of our yearly financial guidance, continued strong execution across our approved medicines and pipeline, the results, enrollment, conduct and progress of our ongoing and planned studies for our product candidates, the timing and results of interim analyses of our ongoing studies and the regulatory approval path for our product candidates in any indication or any specific territory. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “expected,” “will,” “could,” “would,” “guidance,” “potential,” “continue” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum’s business in general, the impact of geopolitical and macroeconomic events, and the other risks described in Mirum’s Annual Report for the year ended December 31, 2024, filed with the Securities and Exchange Commission on February 26, 2025, and subsequent filings with the Securities and Exchange Commission, which are available at www.sec.gov. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Mirum Pharmaceuticals, Inc. Condensed Consolidated Statement of Operations Data (in thousands, except share and per share amounts) (Unaudited) Three Months Ended March 31, 2025 2024 Revenue: Product sales, net $ 111,585 $ 68,917 License and other revenue — 305 Total revenue 111,585 69,222 Operating expenses: Cost of sales (1) 23,018 17,830 Research and development 46,044 32,222 Selling, general and administrative 57,706 45,638 Total operating expenses (2) 126,768 95,690 Loss from operations (15,183 ) (26,468 ) Other income (expense): Interest income 3,023 3,633 Interest expense (3,596 ) (3,577 ) Other income, net 2,108 1,757 Net loss before provision for income taxes (13,648 ) (24,655 ) Provision for income taxes 1,029 624 Net loss (14,677 ) (25,279 ) Net loss per share, basic and diluted $ (0.30 ) $ (0.54 ) Weighted-average shares of common stock outstanding, basic and diluted 48,889,058 46,927,550 (1) Amounts include intangible amortization expense as follows: Intangible amortization $ 5,893 $ 5,402 (2) Amounts include stock-based compensation expense as follows: Cost of sales $ 331 $ — Research and development 5,229 3,861 Selling, general and administrative 10,243 7,589 Total stock-based compensation $ 15,803 $ 11,450 Mirum Pharmaceuticals, Inc. Condensed Consolidated Balance Sheet Data (in thousands) (Unaudited) March 31, December 31, 2025 2024 Assets Current assets: Cash and cash equivalents $ 211,822 $ 222,503 Short-term investments 65,852 57,812 Accounts receivable 95,852 78,286 Inventory 22,418 22,403 Prepaid expenses and other current assets 14,874 11,784 Total current assets 410,818 392,788 Restricted cash 425 425 Long-term investments 20,912 12,526 Intangible assets, net 243,845 249,819 Other noncurrent assets 14,245 15,196 Total assets $ 690,245 $ 670,754 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $ 11,639 $ 14,618 Accrued expenses and other current liabilities 115,938 111,933 Total current liabilities 127,577 126,551 Operating lease liabilities, noncurrent 7,003 7,972 Convertible notes payable, net, noncurrent 308,509 308,082 Other liabilities 13,900 2,509 Total liabilities 456,989 445,114 Commitments and contingencies Stockholders’ equity: Preferred stock — — Common stock 5 5 Additional paid-in capital 892,367 870,189 Accumulated deficit (658,858 ) (644,181 ) Accumulated other comprehensive loss (258 ) (373 ) Total stockholders’ equity 233,256 225,640 Total liabilities and stockholders’ equity $ 690,245 $ 670,754 Investor Contact: Andrew McKibben ir@mirumpharma.com Media Contact: Erin Murphy media@mirumpharma.com