Anastrozole

精彩内容今年以来，中美华东喜讯不断：2月新产品他克莫司缓释胶囊获批上市，3月1类新药HDM1005注射液获批临床，5月1类新药迈华替尼片申报上市……公司的创新转型不断提速。2023年公司营业收入突破122亿元，净利润达到了22.99亿元，成绩可喜。目前有5款新药（含进口）、7个高端仿制药正在抓紧冲刺，公司逐步进入收获期。营业收入突破122亿！两大新品暴涨超100%据华东医药2023年年报数据显示，核心子公司中美华东整体经营继续保持稳健增长态势，全年实现销售收入122.17亿元，创出近五年新高，而净利润也达到了22.99亿元。图1：近几年中美华东的业绩情况（单位：亿元）来源：公司年报2019年中美华东的营业收入首次突破百亿，净利润也是首次达到20亿元水平；2021年受国采降价及医保谈判降价等因素影响，公司的营业收入及净利润经历了微跌；2022-2023年公司的营业收入及净利润持续高速增长，阴霾消散后公司已逐渐恢复活力。近几年，中美华东进入快速转型新阶段，在研新品陆续进入收获期，随着新产品获批及上市销售，为公司业绩增长不断注入动力，公司的整体业绩持续向好。米内网数据显示，2023年在中国城市公立医院、县级公立医院、城市社区中心及乡镇卫生院（简称中国公立医疗机构）终端，中美华东已成为免疫抑制剂市场的国内龙头企业，市场份额超过了16%，与TOP1企业诺华的差距仅为1%左右。图2：中美华东的他克莫司胶囊销售情况（单位：万元）来源：米内网中国公立医疗机构药品终端竞争格局中美华东的他克莫司胶囊2023年在中国公立医疗机构终端大卖24.1亿元，是免疫抑制剂国产冠军品牌。该产品是公司的拳头产品，2023年为公司贡献了超过23%的业绩。图3：中美华东的吡格列酮二甲双胍片(15mg/500mg)销售情况（单位：万元）来源：米内网中国公立医疗机构药品终端竞争格局中美华东也是口服复方降糖药市场TOP1企业，2023年的市场份额超过了43%，遥遥领先。公司的吡格列酮二甲双胍片(15mg/500mg)为国内首仿，近几年在中国公立医疗机构终端的销售额持续高涨，2023年突破10亿元关口，是口服降糖药TOP1品牌。目前中美华东在中国公立医疗机构终端超10亿产品有4个，合计为公司贡献了超过68%的业绩。表1：2023年在中国公立医疗机构终端中美华东部分销售大涨的产品注：销售额低于1亿元用*表示来源：米内网中国公立医疗机构药品终端竞争格局二甲双胍恩格列净片(Ⅰ)是中美华东在2021年获批上市的口服复方降糖药新品，公司是该产品国内首仿+首家过评企业。在中国公立医疗机构终端，公司的二甲双胍恩格列净片(Ⅰ)2022年的销售额仅在4000万元水平，2023年大涨超过325%，销售额突破2亿元。注射用醋酸卡泊芬净是中美华东在2020年获批上市的新品，也是公司首款全身用抗真菌药。近两年公司的注射用醋酸卡泊芬净在中国公立医疗机构终端呈现爆发式增长，2022-2023年的增长率分别为348.71%、179.73%，为公司进军新市场打下来好基础。2023年中美华东共有4个新品获批，潜力可期。马昔腾坦片为国内首仿（含同日获批），同时也是公司首款高血压用药；他克莫司软膏是公司首款皮肤病用药；利拉鲁肽注射液则是国产首家获批。随着新产品进入市场，将助力公司提升整体竞争力。60亿品种再添猛将！4大产品备战第十批国采2024年至今，中美华东喜讯不断，1个新品获批上市，1款1类新药获批临床，两个产品通过一致性评价。图4：2024年至今中美华东获批的产品来源：米内网中国申报进度（MED）数据库他克莫司是重磅品种，2023年在中国公立医疗机构终端的销售额超过了60亿元，中美华东是他克莫司缓释胶囊的国产第二家获批企业。目前中美华东在该品种已拥有胶囊剂（含缓释胶囊）、软膏剂，后续将有更多剂型获批。化药1类新药HDM1005注射液是多肽类人GLP-1受体和GIP受体的双靶点长效激动剂，临床前研究显示，HDM1005可通过激活GLP-1受体和GIP受体促进胰岛素释放，抑制食欲，发挥显著的改善糖耐量、降糖和减重效果，同时现有数据显示HDM1005具有良好的成药性和安全性。截至2024年5月25日，中美华东共有25个产品过评/视同通过一致性评价。其中阿那曲唑片中标了第三批国采，注射用泮托拉唑钠中标了第四批国采，注射用地西他滨中标了第五批国采，吗替麦考酚酯胶囊和盐酸吡格列酮片中标了第七批国采。表2：中美华东已过评的产品情况来源：米内网中国申报进度（MED）数据库在中国公立医疗机构终端，中美华东的阿那曲唑片、注射用泮托拉唑钠、注射用地西他滨、吗替麦考酚酯胶囊中标国采后市场份额得到了快速提升，公司已成为吗替麦考酚酯胶囊和注射用泮托拉唑钠的TOP2企业。目前中美华东过评且暂未纳入国采的产品中，竞争企业（原研+过评）≥5家的产品有环孢素软胶囊、马昔腾坦片、他克莫司软膏、西格列汀二甲双胍片(Ⅱ)，第十批国采正步步逼近，公司已在积极备战。5款新药、7个仿制药冲刺上市，全力抢攻千亿市场5月11日，中美华东提交了化药1类新药迈华替尼片的上市申请，备受市场瞩目。该新药是一款新型、强效、高选择性、具有口服活性的不可逆EGFR/人表皮生长因子受体-2（HER2）小分子抑制剂，申报适应症为用于EGFR 21号外显子L858R置换突变的局部晚期或转移性NSCLC成人患者的一线治疗。从目前的进度看，迈华替尼片或将成为公司首款上市的抗肿瘤化药1类新药。表3：中美华东目前报产在审的新品来源：米内网中国申报进度（MED）数据库目前中美华东有5款新药（含进口新药）报产在审，除了迈华替尼片，索米妥昔单抗注射液也为抗肿瘤药，用于治疗既往接受过1-3线系统性治疗的叶酸受体α（FRα）阳性的铂类耐药的上皮性卵巢癌、输卵管癌或原发性腹膜癌成年患者。5月24日，公司在回复投资者疑问时提到，索米妥昔单抗注射液的国内上市申请目前已进入补充资料流程。注射用利纳西普用于治疗成人和12岁及以上儿童的冷吡啉相关的周期性综合征（CAPS），包括家族性寒冷型自身炎症综合征（FCAS）和穆-韦二氏综合征（MWS），以及治疗成人和12岁及以上青少年复发性心包炎（RP）以及降低复发风险。乌司奴单抗注射液是免疫抑制剂畅销产品，中美华东是首家提交生物类似药上市申请的国内企业，公司有望成为该产品国产首家获批企业。报产在审的7个高端仿制药均为抗肿瘤和免疫调节剂，其中他克莫司颗粒、西罗莫司片、注射用卡非佐米目前暂无国内药企获得生产批文，中美华东将参与国内首仿争夺。在中国公立医疗机构终端化学药（含生物药）市场，抗肿瘤和免疫调节剂2023年的销售规模已涨至1854亿元，中美华东今明两年将陆续有新产品加入这个千亿市场。7款国产新药来势汹汹，两个重磅注射剂上市可期近几年，中美华东采用“自研+合作”的方式不断扩张公司的研发管线，陆续有项目进入了报产阶段，目前有7款国产新药在临床阶段，有望成为公司下一批申报的重磅新药。表4：中美华东部分已开展临床的新药情况注：已终止的项目不纳入统计来源：米内网中国临床试验数据库目前，公司的司美格鲁肽注射液和德谷胰岛素注射液已在III期临床。司美格鲁肽注射液是糖尿病用药TOP1产品，2023年在中国公立医疗机构终端销售额超过40亿元，德谷胰岛素注射液也达到了6.7亿元，目前两款重磅注射剂仅有原研企业获批进口，国内药企摩拳擦掌。1类新药方面，口服小分子GLP-1药物HDM1002片目前正在II期临床，超重或肥胖人群的体重管理适应症有望先于成人2型糖尿病适应症获批，助力公司开拓减肥市场。资料来源：米内网数据库、公司年报及公告注：米内网《中国公立医疗机构药品终端竞争格局》，统计范围是：中国城市公立医院、县级公立医院、城市社区中心以及乡镇卫生院，不含民营医院、私人诊所、村卫生室；上述销售额以产品在终端的平均零售价计算。数据统计截至5月25日，如有疏漏，欢迎指正！本文为原创稿件，转载请注明来源和作者，否则将追究侵权责任。投稿及报料请发邮件到872470254@qq.com稿件要求详询米内微信首页菜单栏商务及内容合作可联系QQ：412539092【分享、点赞、在看】点一点不失联哦

Plan to complete NDA submission with the mature RAMP 201 dataset, anticipated to include 12 months of follow-up, in the second half of 2024 Plan to present the mature dataset from RAMP 201 at a medical conference in the second half of 2024 Avutometinib and defactinib combination have continued to show robust and durable response rates in ongoing RAMP 201 trial in patients with recurrent low-grade serous ovarian cancer Company to host investor conference call and webcast on Friday, May 24, 2024 at 8:00 am EDT to provide update on RAMP 201 and rolling NDA submission BOSTON--(BUSINESS WIRE)-- Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, today announced that it has initiated the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval of the combination of avutometinib, a RAF/MEK clamp, and defactinib, a selective FAK inhibitor, for adult patients with recurrent KRAS mutant (KRAS mt) low-grade serous ovarian cancer (LGSOC), who received at least one prior systemic therapy. The rolling review process allows the Company to submit completed sections of an application for review by the FDA before all sections become available. The initial sections of the application will include the nonclinical and quality sections. In discussions with the FDA, Verastem reached agreement to submit a primary efficacy analysis based on the RAMP 201 study with 12 months of follow up. Based on discussions with the FDA, we understand that the proposed indication for final submission of the clinical module can be expanded in the event Verastem provides data that demonstrates a substantial improvement over available therapy in the KRAS wild-type (KRAS wt) population. FDA has accepted Verastem’s plan to submit the clinical module in the second half of 2024 to complete the NDA application. Previously, the FDA granted Breakthrough Therapy Designation (BTD) for the combination for treatment of patients with recurrent LGSOC, regardless of KRAS status, following one or more previous lines of therapy and Orphan Drug Designation (ODD) for the combination in certain LGSOC indications. The Company plans to request a priority review of the NDA. Currently, there are no FDA-approved treatments specifically for recurrent LGSOC. "The initiation of our rolling NDA submission of the avutometinib and defactinib combination for accelerated approval, is an important step towards addressing the significant unmet needs that patients face living with KRAS mutant low-grade serous ovarian cancer,"​ said Dan Paterson, president and chief executive officer of Verastem Oncology. "The data from our ongoing RAMP 201 trial continues to support our belief that the avutometinib and defactinib combination has the potential to be a new standard of care in patients with recurrent low-grade serous ovarian cancer, if approved. In the second half of this year, we anticipate completing our NDA submission with the mature data from the RAMP 201 trial and discussing with the FDA a path forward for patients with KRAS wild-type disease. We also expect to present the mature dataset at a medical meeting in the second half of 2024."​ RAMP 201 is a Phase 2 registration-directed study evaluating avutometinib and defactinib combination in patients with recurrent LGSOC. The enrollment in RAMP 201 is completed, with 115 patients being treated at the recommended Phase 2 dose (RP2D) of avutometinib 3.2 mg twice weekly and defactinib 200 mg twice daily for 3 out of every 4 weeks, and follow-up continues. Verastem expects to complete the NDA submission after obtaining mature safety and efficacy data from the RAMP 201 trial, including 12 months of follow-up, anticipated in the second half of 2024. Verastem also plans to further discuss the KRAS wt data with FDA to inform the potential path forward for approval for this patient population. The Company plans to present the mature dataset from RAMP 201 at a medical meeting in the second half of 2024.​ As of February 2024, the interim data continued to show robust overall response rates (ORR) and durable responses with low discontinuation rates due to adverse events (AEs) in patients from RAMP 201 Parts A, B, C, who had a minimum follow-up of five (5) months. The FDA granted Breakthrough Therapy Designation of the investigational combination of avutometinib and defactinib for the treatment of all patients with recurrent LGSOC regardless of KRAS status after one or more prior lines of therapy, including platinum-based chemotherapy in May 2021. Avutometinib alone or in combination with defactinib was also granted Orphan Drug Designation by the FDA for the treatment of LGSOC in March 2024. The Company believes that this Orphan Drug Designation signifies that LGSOC is a rare ovarian cancer that is a distinct and different disease from other forms of ovarian cancer such as high-grade serous ovarian cancer (HGSOC). LGSOC is highly recurrent and fatal, with no FDA-approved treatment options, and the current standard of care treatments include hormonal therapy or chemotherapy, which have demonstrated an ORR between 6-13% with discontinuation due to AEs of 17-30%. ​ The Company is currently enrolling patients and activating sites for RAMP 301, an international confirmatory Phase 3 trial, evaluating the avutometinib and defactinib combination versus standard of care chemotherapy or hormonal therapy for the treatment of patients with KRAS mt and KRAS wt recurrent LGSOC. Conference Call and Webcast Information Verastem will hold an investor conference call and webcast on Friday, May 24 at 8:00 am EDT, to review the initiation of the NDA submission and limited, topline data from the RAMP 201 trial, with a minimum follow-up of five (5) months and the RAMP 205 data. The call will feature members of Verastem’s management team. To access the conference call, please dial (844) 763-8274 (local) or (412) 717-9224 (international) at least 10 minutes prior to the start time and ask to be joined into the Verastem Oncology conference call. A live audio webcast of the call, along with accompany slides, will be accessible here. The Company expects to 8-K pertaining to this update. About RAMP 201 RAMP 201 (ENGOTov60/GOG3052) is an adaptive, two-part multicenter, parallel cohort, randomized, open-label trial to evaluate the efficacy and safety of avutometinib alone and in combination with defactinib in patients with recurrent low-grade serous ovarian cancer. The first part of the study (Part A) determined the selection of the go forward regimen, which was the combination of avutometinib and defactinib versus avutometinib alone, based on overall response rates. The expansion phases of the trial (Parts B and C) are evaluating the safety and efficacy of the go forward regimen of avutometinib 3.2 mg twice weekly and defactinib 200 mg twice daily. The Part D portion of the trial is evaluating a low dose of avutometinib in combination with defactinib to inform individualized dose reduction. About RAMP 301 RAMP 301 (GOG-3097; ENGOT-ov81/NCRI) is an international collaboration between The GOG Foundation, Inc. (GOG) and the European Network of Gynaecological Oncological Trial groups (ENGOT) sponsored by Verastem Oncology. The trial is expected to enroll a total of 270 patients in the U.S., Canada, the United Kingdom, Europe, Australia and South Korea, who will be randomized to either the combination of avutometinib and defactinib or investigator’s choice chemotherapy (pegylated liposomal doxorubicin, paclitaxel, topotecan) or hormone therapy (letrozole, anastrozole). The primary endpoint is progression free survival (PFS) by Blinded Independent Central Review. Secondary endpoints include ORR, duration of response​, disease control rate, safety and tolerability, patient reported outcomes, and overall survival. About Low-Grade Serous Ovarian Cancer (LGSOC) LGSOC is a rare ovarian cancer that is insidious, persistent and ultimately fatal. LGSOC is distinct and different from high-grade serous ovarian cancer (HGSOC) and requires different treatment. LGSOC is highly recurrent and less sensitive to chemotherapy compared to HGSOC. Approximately 6,000-8,000 women in the U.S. and 80,000 worldwide are living with this disease. LGSOC affects younger women with bimodal peaks of diagnosis at ages between 20-30 and 50-60 and has a median survival of approximately ten years. The majority of patients report negative impact of LGSOC on their mental and physical health, fertility, and long-term quality of life. The current standard of care for this disease includes hormone therapy and chemotherapy, but there are no treatments specifically approved by the U.S. Food and Drug Administration to treat LGSOC. About the Avutometinib and Defactinib Combination Avutometinib is a n investigational RAF/MEK clamp that is designed to induce inactive complexes of MEK with ARAF, BRAF and CRAF potentially creating a more complete and durable anti-tumor response through maximal RAS/MAPK pathway inhibition. Avutometinib is designed to block both MEK kinase activity and the ability of RAF to phosphorylate MEK. This differentiated proposed mechanism potentially allows avutometinib to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other MEK-only inhibitors. The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation of the investigational combination of avutometinib and defactinib, a selective FAK inhibitor, for the treatment of all patients with recurrent low-grade serous ovarian cancer (LGSOC) regardless of KRAS status after one or more prior lines of therapy, including platinum-based chemotherapy. Avutometinib alone or in combination with defactinib was also granted Orphan Drug Designation by the FDA for the treatment of LGSOC. Verastem Oncology is currently conducting clinical trials with avutometinib in RAS/MAPK driven tumors as part of its Raf And Mek Program or RAMP. RAMP 301 (NCT06072781) is an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent LGSOC. RAMP 201 (NCT04625270) is a Phase 2 registration-directed trial of avutometinib in combination with defactinib in patients with recurrent LGSOC and enrollment has been completed in each of the dose optimization and expansion phases and the low-dose evaluation. Verastem Oncology has established clinical collaborations with Amgen and Mirati to evaluate LUMAKRAS™ (sotorasib) in combination with avutometinib and defactinib and KRAZATI™ (adagrasib) in combination with avutometinib in KRAS G12C mutant NSCLC as part of the RAMP 203 (NCT05074810) and RAMP 204 (NCT05375994) trials, respectively. The RAMP 205 (NCT05669482), a Phase 1b/2 clinical trial evaluating avutometinib and defactinib with gemcitabine/Nab-paclitaxel in patients with front-line metastatic pancreatic cancer, is supported by a PanCAN Therapeutic Accelerator Award. About Verastem Oncology Verastem Oncology (Nasdaq: VSTM) is a late-stage development biopharmaceutical company committed to the development and commercialization of new medicines to improve the lives of patients diagnosed with cancer. Our pipeline is focused on RAS/MAPK-driven cancers, specifically novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition and FAK inhibition. For more information, please visit and follow us on LinkedIn. Forward Looking Statements This press release includes forward-looking statements about, among other things, Verastem Oncology’s programs and product candidates, strategy, future plans and prospects, including statements related to the expected timing of the planned rolling New Drug Application (NDA) submission for the avutometinib and defactinib combination in low-grade serous ovarian cancer, the potential clinical value of various of the Company’s clinical trials, including the RAMP 201, RAMP 205 and RAMP 301 trials, the timing of commencing and completing trials, including topline data reports, interactions with regulators, the potential for and timing of commercialization of product candidates and potential for additional development programs involving Verastem Oncology’s lead compound. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," “can,” “promising” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement. Applicable risks and uncertainties include the risks and uncertainties, among other things, regarding: the success in the development and potential commercialization of our product candidates, including avutometinib in combination with other compounds, including defactinib, LUMAKRAS™ and others; the uncertainties inherent in research and development, such as negative or unexpected results of clinical trials, the occurrence or timing of applications for our product candidates that may be filed with regulatory authorities in any jurisdictions; whether and when regulatory authorities in any jurisdictions may approve any such applications that may be filed for our product candidates, and, if approved, whether our product candidates will be commercially successful in such jurisdictions; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the scope, timing, and outcome of any legal proceedings; decisions by regulatory authorities regarding trial design, labeling and other matters that could affect the timing, availability or commercial potential of our product candidates; whether preclinical testing of our product candidates and preliminary or interim data from clinical trials will be predictive of the results or success of ongoing or later clinical trials; that the timing, scope and rate of reimbursement for our product candidates is uncertain; the market opportunities of our drug candidates are based on internal and third-party estimates which may prove to be incorrect; that third-party payors (including government agencies) may not reimburse; that there may be competitive developments affecting our product candidates; that data may not be available when expected; that enrollment of clinical trials may take longer than expected, which may delay our development programs, including delays in submission or review by the FDA of our NDA submission in recurring KRAS mutant LGSOC if enrollment in our confirmatory trial is not well underway at the time of submission, or that the FDA may require the Company to have completed enrollment or to enroll additional patients in the Company’s ongoing RAMP-301 confirmatory Phase 3 clinical trial prior to Verastem submitting or the FDA taking action on our NDA seeking accelerated approval; that our product candidates will cause adverse safety events and/or unexpected concerns may arise from additional data or analysis, or result in unmanageable safety profiles as compared to their levels of efficacy; that we may be unable to successfully validate, develop and obtain regulatory approval for companion diagnostic tests for our product candidates that require or would commercially benefit from such tests, or experience significant delays in doing so; that our product candidates may experience manufacturing or supply interruptions or failures; that any of our third party contract research organizations, contract manufacturing organizations, clinical sites, or contractors, among others, who we rely on fail to fully perform; that we face substantial competition, which may result in others developing or commercializing products before or more successfully than we do which could result in reduced market share or market potential for our product candidates; that we will be unable to successfully initiate or complete the clinical development and eventual commercialization of our product candidates; that the development and commercialization of our product candidates will take longer or cost more than planned, including as a result of conducting additional studies; that we may not have sufficient cash to fund our contemplated operations; that we may not attract and retain high quality personnel; that we or Chugai Pharmaceutical Co., Ltd. will fail to fully perform under the avutometinib license agreement; that our target market for our product candidates might be smaller than we are presently estimating; that Secura Bio, Inc. will fail to fully perform under the asset purchase agreement with Secura Bio, Inc., including in relation to milestone payments; that we will not see a return on investment on the payments we have and may continue to make pursuant to the collaboration and option agreement with GenFleet Therapeutics (Shanghai), Inc. (GenFleet), or that GenFleet will fail to fully perform under the agreement; that we may be unable to obtain adequate financing in the future through product licensing, co-promotional arrangements, public or private equity, debt financing or otherwise; that we will not pursue or submit regulatory filings for our product candidates; and that our product candidates will not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients. As a result of these and other factors, we may not achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Other risks and uncertainties include those identified under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 as filed with the Securities and Exchange Commission (SEC) on March 14, 2024 and in any subsequent filings with the SEC. The forward-looking statements contained in this press release reflect Verastem Oncology’s views as of the date hereof, and the Company does not assume and specifically disclaims any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by law. View source version on businesswire.com: Contacts For Investor and Media Inquiries: Julissa Viana Vice President, Corporate Communications and Investor Relations investors@verastem.com or media@verastem.com Source: Verastem Oncology View this news release online at: