Nusinersen sodium

每年的医保谈判，创新药企们都希望通过合理区间的价格降幅，争取到更大院内市场。但药品“入保”后，能否带来销售的快速增长？ 对于这个问题，在全国一些地区医保基金吃紧、“进院”和“双通道”路径不畅以及医保基金对创新支持力度不足的背景下，部分创新药企和资本市场投资人持审慎乃至较为悲观的态度。 01 创新药“入保”第一年 “进院”情况不容乐观 2024年的医保谈判已收官，对于那些想要抓住市场增量机遇的企业来说，考验才刚刚开始。 据统计，2019-2023年，共有115个创新药获批，有75个创新药进入医保目录，占比65.2%。其中，包含56个化药占进医保药品总数的74.7%，占上市化药创新药总数的86.2%；10个生物药占进医保药品总数的13.3%，占上市生物药创新药总数的28.6%；9个中药占进医保药品总数的12%，占上市中药创新药总数的60%。 来源：公开资料，新康界整理 与去年相比，今年企业申报药品数量均有所增加。可见，国家医保目录的吸引力正在逐年提升，已经成为创新药商业化过程中至关重要的门槛。但不可否认，“入保”只是敲门砖，“进院”才是创新药实现销售的“最后一公里”，也是“最难一公里”。 一般而言，创新药入院需要经历科室申请、药学部门审核、药事委员会投票等程序，往往长达一年之久。2024年以来，不少省份发文明确定点医疗机构召开药事委员会的时限和频次，要求新增谈判药品“应配尽配”。 有的省份提出必要时随时开，大力推动创新药入院。例如，广东省允许医疗机构通过一定程序，将临床优势明显、安全性高、不可替代、患者有需求的国家医保谈判药品直接纳入用药目录。 但创新药研发的投入和耗时巨大，价格比普通药品更高。因此在DRG/DIP支付方式改革背景下，医疗机构还面临着基本用药供应目录品种数、药占比以及医保总额预算等约束，使用创新药的积极性不高。加上一些医疗机构对创新药的认识不足，有的医生甚至有药不敢用。 一项根据国家医保服务平台公示数据梳理的统计研究显示，截至2024年6月底，不同批次国谈药在全国各省的进院数量差距较大，其中2023批次药品配备率显著较低。 该研究选取了2017~2023年谈判药品中从上市到医保谈判时间≤5年的药品作为样本开展分析，共计涵盖国谈新药322种。结果显示，2023批次药品的进院数量中值仅为136家，低于2022批次药品的233家，更显著落后于2017~2021批次药品进院数据。这意味着，创新药在“纳保”的第一年，“进院”情况不容乐观。 值得一提的是，上海这一比例达到18.9%，为全国最高；除上海外，其他各省份配备率没有超过8%，如江苏、北京、浙江等比较好的省份配备率在6%~7%左右。 据悉，2023年，上海市医保单列支付创新药81.6亿元，较2021年增长164.9%。在上海“28条”措施出台的一年后，近期，上海医保局对484种纳入该市单列支付名单中的国谈药展开调研，结果显示新政效果初显。 具体来看，今年1-7月，上海市医药机构国谈创新药结算金额70.6亿元，同比增长100.1%，上海创新药“进院”使用情况整体较好。医保结算金额普遍实现快速增长，部分创新药在进目录当年实现10倍以上增长。 据IQVIA最新统计数据，2024年上半年，上海三级医院2023批次国谈药品的配备率和头部医院国谈药配备平均品种数两项统计遥遥领先，均列全国第一。 02 70万变3万的“天价”药品 “入保”后销售额大涨300% 随着对国谈药的支持性政策逐渐显效，在上海，国谈药在“总量快速增长”的同时，也出现“结构分化”。根据上海医保局数据，进一步拆解国谈药在当地医药机构的结算金额时可以发现：创新药比非创新药销售情况好，本市创新药比外省市新药销售情况好，跨国新药比国内创新药销售情况好。 具体来看，今年1~7月，上海市进入国家医保目录的1类新药（2018年以来共15个）销售额同比增长124.6%；外省市1类新药（共67个，统计前15个）同比增长94.1%；跨国新药（共8个）同比增长181.3%；本市非1类新药（共15个）同比增长73.9%。 据估算，15个纳入国家医保目录的上海1类新药，纳保以来医保结算金额的年复合增长率为86.53%，8个跨国新药的这一数据则为242.89%。 此前，首次入选的创新药品，绝大多数进入医保之后实现了以量换价。从全国来看，国家医保局数据显示，2019年-2023年，75个创新药进入医保目录后，总体的销售额和销售量在进入医保的次年都得到了大幅的提高。 销售量方面，生物药平均销量增幅最低，增长646.3%；化药平均销量增幅最高，增长39137.1%；中药销量的平均增幅处于中间水平，增长750%，三种类别平均增幅13511%。 销售额方面，化药的销售额提升幅度最大，平均增幅中位值近24155%；生物药的增幅最低，平均增幅162%，悬殊较大；中药平均增幅则约318%，三种类别平均增幅8212%。 来源：公开资料，新康界整理 从近五年来看，中药创新药单价平均降幅最大，为73.1%；化药创新药单价平均降幅最低，为57.8%，生物药处于中间水平，降幅65.1%。 来源：公开资料，新康界整理 以2018年医保谈判品种来看，价格平均降幅57%，销售额全部实现了正增长，第一年销售额平均增幅高达1024%。再以2019年医保谈判品种来看，新增品种价格平均降幅61%，销售额几乎全部实现了正增长，第一年销售额平均增幅高达2692%。 2019-2023年，进入医保后的创新药单价平均降幅为60.3%，其中，2023年与整体水平相比，创新药谈判成功率高7.4%，平均降幅低4.4%，通过谈判新增进入医保目录的105个药品，9月的药品销售额是1月的七倍。 聚焦具体品种，目前我国一共进行了8轮针对创新药的医保谈判，首次入选的创新药品，平均降幅大约在40%至62%之间，绝大多数创新药医保谈判“以价换量”的预期兑现。 如广受关注的“70万元一针”天价药诺西那生便以低于3.3万元每针的“地板价”进入2021版医保目录。在此之前，该药在2020年和2021年的国内销售额分别为5千万元和1.55亿元，而被纳入医保目录之后，2022年全年销售额大增至6.10亿元，增长率近300%‌。 部分创新药纳入医保前后 全国等级医院市场销售情况 来源：中康开思系统，新康界整理 此外，以国产PD-1抗肿瘤药物信迪利单抗为例，据中康开思系统显示，其于2019年被纳入医保，2020年等级医院销售额实现1099%的增长，达到8.02亿元，并于2023年冲至25.62亿元，同比上涨74.7%，2024年上半年达17.03亿元，全年销售额有望再破新高。 信迪利单抗 全国等级医院销售额 来源：中康开思系统 进口特应性皮炎新药度普利尤单抗注射液于2020年纳入医保，次年等级医院销售额实现77182%的增长，并于2023年突破18亿元，同比上涨74.6%，2024上半年销售额达10.67亿元，年底有望冲刺20亿元的销售规模。 还有替雷利珠单抗，在2020年首次纳入医保后，根据中康开思系统数据，2021年其医院销售额相比2020年同比增加了1326.4%，达到7.73亿元；2022年持续放量，一举突破21亿元。 再以艾力斯的伏美替尼为例，在2021年医保谈判降价79.4%后实现以价换量，2022年产品销售额同比增长4477.7%，达2.21亿元。 药渡媒体商务合作 媒体公关 | 新闻&会议发稿 张经理：18600036371（微信同号） 点击下方“药渡“，关注更多精彩内容 免责声明 “药渡”公众号所转载该篇文章来源于其他公众号平台，主要目的在于分享行业相关知识，传递当前最新资讯。图片、文章版权均属于原作者所有，如有侵权，请及时告知，我们会在24小时内删除相关信息。 微信公众号的推送规则又双叒叕改啦，如果您不点个“在看”或者没设为"星标"，我们可能就消散在茫茫文海之中~点这里，千万不要错过药渡的最新消息哦！👇👇👇

CAMBRIDGE, Mass.--( BUSINESS WIRE )--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today reported financial results and corporate updates for the third quarter ended September 30, 2024. “ We were thrilled to announce positive and statistically significant topline data from the pivotal Phase 3 SAPPHIRE trial, a major achievement that underscores apitegromab’s potential to redefine the standard of care for patients with SMA,” said Jay Backstrom, M.D., MPH, President and Chief Executive Officer of Scholar Rock. “ The positive SAPPHIRE trial validates our approach to targeting pro-and latent myostatin and demonstrates apitegromab’s potentially transformative benefit. Based on these results, we plan to submit a BLA and MAA in the first quarter of 2025 and are working diligently to prepare for the commercial launch of apitegromab, and if approved, to deliver the potentially transformative benefits of apitegromab to children and adults living with SMA.” Dr. Backstrom continued, “ The robust results from SAPPHIRE also reinforce the broader potential of our selective myostatin inhibition strategy across our pipeline, with potential readthrough to our cardiometabolic program as we evaluate apitegromab in obesity in our Phase 2 EMBRAZE proof-of-concept trial. We were pleased to announce in September that the EMBRAZE trial is fully enrolled, and we expect to report initial data in the second quarter of next year.” Company Highlights and Upcoming Milestones SMA Program Apitegromab is an investigational, fully human monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle and is being developed as a potential first muscle-targeted therapy for the treatment of SMA. Apitegromab is the first muscle-targeted therapy to show clinical proof-of-concept in SMA. Reported positive topline data from Phase 3 SAPPHIRE clinical trial . The study achieved its primary endpoint demonstrating a statistically significant and clinically meaningful improvement for apitegromab versus placebo in motor function as measured by the gold standard HFMSE in SMA patients on chronic dosing of standard of care therapies (either nusinersen or risdiplam). In the main efficacy population (ages 2-12), the mean difference in change from baseline in HFMSE was 1.8 points (p=0.0192) for all patients receiving apitegromab 10 mg/kg and 20 mg/kg (n=106) compared to placebo (n=50). Patients receiving 20 mg/kg of apitegromab (n=53) showed a 1.4 point mean difference compared to placebo (p=0.1149). The prespecified analysis of the 10 mg/kg dose showed that patients receiving 10 mg/kg of apitegromab (n=53) showed a mean difference in change from baseline in HFMSE of 2.2 points compared to placebo (nominal p=0.0121). Based upon PK/PD data from the SAPPHIRE trial, similar levels of target engagement were observed for the 10 mg/kg and 20 mg/kg dose groups. Scholar Rock plans to submit a U.S. BLA and a European Union MAA in 1Q 2025. Presented preliminary baseline characteristics from SAPPHIRE during a poster presentation at the 29 th Annual Congress of the World Muscle Society in October in Prague, Czech Republic. Analyses of the full Phase 3 SAPPHIRE data are ongoing, and Scholar Rock plans to present detailed results at an upcoming medical conference in early 2025. The ONYX open-label, multi-center extension study remains ongoing. The study is evaluating the long-term safety and efficacy of apitegromab in patients who completed the TOPAZ or SAPPHIRE trials. More than 90 percent of patients on combination therapy in the TOPAZ trial have completed 4 years of apitegromab treatment and enrolled into ONYX. Following trial completion, 98 percent of SAPPHIRE patients (185/188) enrolled in the ongoing ONYX open-label expansion study. On track to initiate OPAL clinical trial in SMA patients under two years old in mid-2025. Cardiometabolic Program SRK-439 is a novel, preclinical, investigational myostatin inhibitor that binds to pro- and latent myostatin with high affinity and is selective for myostatin (i.e., no GDF11 or Activin A binding), and is initially being developed for the treatment of obesity. Based on preclinical data, SRK-439 has the potential to support healthier weight management by preserving lean mass during weight loss. Completed enrollment of Phase 2 EMBRAZE proof-of-concept trial with apitegromab in combination with a GLP-1 receptor agonist (GLP-1 RA) in obesity in September. The Phase 2 trial is a randomized, double-blind, placebo-controlled, multi-center study evaluating the safety and efficacy of apitegromab, a highly selective investigational myostatin inhibitor, to preserve muscle mass as an adjunctive therapy in overweight and obese adults who are taking a GLP-1 RA. Data are expected in the second quarter of 2025 and will be used to guide clinical development of SRK-439. The Company plans to file an IND for SRK-439 for the treatment of obesity in 2025. Immuno-Oncology Program SRK-181 is an investigational selective inhibitor of latent TGFβ1 activation and is being developed with the aim of overcoming resistance to checkpoint therapy, such as anti-PD-(L)1 antibodies, in patients with advanced cancer. New SRK-181 data from the Phase 1 DRAGON proof-of-concept trial presented at the SITC 39 th Annual Meeting in November. The presentation, “ DRAGON Trial: Durable remission rate with the latent TGFβ1 inhibitor linavonkibart (SRK-181) and pembrolizumab in patients with immune checkpoint inhibitor resistant advanced cancers,” highlighted new clinical and biomarker results. Highlights since the Company’s last update on the DRAGON trial include one additional complete response, seen in the clear cell renal cell (ccRCC) cohort (ORR=23.3%) and durable responses in multiple cohorts. Additionally, biomarker results continue to support proof of mechanism; new data shows suppressed granulocytic myeloid-derived suppressor cells (gMDSC) levels in the tumor microenvironment of responders. Notably new biomarker data in the ccRCC cohort supports a potential patient selection strategy with positive correlations between clinical response and baseline CD8+ infiltration status, elevated baseline Treg levels, and elevated baseline TGFβ1 levels in the tumor compartment. Enrollment of the DRAGON trial was completed in December 2023, and patients who remain on the study continue to be treated. Other Corporate Updates Appointed Beth Shafer, Ph.D., to the newly created role of Chief Business Officer. Ms. Shafer brings over 20 years of biopharmaceutical industry leadership and expertise to Scholar Rock, where she will drive the Company’s long-term corporate and business development strategy. Raised $345 million in upsized follow-on offering. In October, the Company completed an upsized follow-on offering raising aggregate gross proceeds of $345 million. Third Quarter 2024 Financial Results For the quarter ended September 30, 2024, net loss was $64.5 million or $0.66 per share compared to a net loss of $42.4 million or $0.53 per share for the quarter ended September 30, 2023. The Company did not record any revenue for the quarter ended September 30, 2024, or for the quarter ended September 30, 2023. Research and development expense was $48.7 million for the quarter ended September 30, 2024, compared to $30.3 million for the quarter ended September 30, 2023. The increase was primarily attributable to costs associated with the ONYX and EMBRAZE trials, and the development of SRK-439. General and administrative expenses were $16.1 million for the quarter ended September 30, 2024, compared to $13.3 million for the quarter ended September 30, 2023. The increase was due to employee-related costs. As of September 30, 2024, Scholar Rock had cash, cash equivalents and marketable securities of approximately $139.1 million ($463.5 million, on a pro forma basis, including the approximately $324.4 million raised in the October 2024 equity offering). Along with cash available to the Company, runway is extended into 4Q 2026. “ Supported by our recent upsized public offering on the heels of successful SAPPHIRE data, Scholar Rock is advancing our mission from a position of strength as we move forward with preparations for our first commercial launch in the fourth quarter of 2025,” said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock. “ We are working tirelessly to obtain approval for apitegromab and deliver it to patients with SMA as soon as possible, as well as continuing to advance our portfolio of highly selective myostatin inhibition programs.” Conference Call Information Management will provide an update on the Company and discuss third quarter 2024 results via conference call on Tuesday, November 12 at 8:15 am ET. To access the live conference call, participants may register here . The live audio webcast of the call will be available under “Events and Presentations” in the Investor Relations section of the Scholar Rock website at http://investors.scholarrock.com . To participate via telephone, please register in advance here . Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. An archived replay of the webcast will be available on the Company’s website for approximately 90 days. About Scholar Rock Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need. As a global leader in the biology of the transforming growth factor beta (TGFβ) superfamily of cell proteins and named for the visual resemblance of a scholar rock to protein structures, the clinical-stage company is focused on advancing innovative treatments where protein growth factors are fundamental. Over the past decade, Scholar Rock has created a pipeline with the potential to advance the standard of care for neuromuscular disease, cardiometabolic disorders, cancer, and other conditions where growth factor-targeted drugs can play a transformational role. This commitment to unlocking fundamentally different therapeutic approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. By harnessing cutting-edge science in disease spaces that are historically under-addressed through traditional therapies, Scholar Rock works every day to create new possibilities for patients. The efficacy and safety of apitegromab, SRK-181, and SRK-439 have not been established and apitegromab, SRK-181, and SRK-439 have not been approved for any use by the FDA or any other regulatory agency. Scholar Rock ® is a registered trademark of Scholar Rock, Inc. Availability of Other Information About Scholar Rock Investors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com , including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on Twitter and LinkedIn. The information we post on our website, Twitter or LinkedIn could be deemed material information. As a result, we encourage investors, the media and others interested to review the information we post there regularly. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding its growth, strategy, progress and timing of its clinical trials for apitegromab and SRK-181 and its preclinical programs, including SRK-439, and indication selection and development timing, including the timing of any regulatory submissions, the therapeutic potential, clinical benefits and safety of any product candidates, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, its cash runway, expectations relating to commercial launch in the US in the fourth quarter of 2025, expectations regarding the achievement of important milestones, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, and the potential of its product candidates and proprietary platform. The use of words such as “may,” “might,” “could,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, without limitation, that preclinical and clinical data, including the results from the Phase 3 SAPPHIRE trial will be sufficient to support regulatory approval, that the full results from the Phase 3 SAPPHIRE trial may differ from the topline data, that preclinical and clinical data, including the results from the Phase 2 or Phase 3 clinical trial of apitegromab, or Part A or Part B of the Phase 1 clinical trial of SRK-181, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidates; the data generated from Scholar Rock’s nonclinical and preclinical studies and clinical trials; Scholar Rock’s ability to provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline; the data generated from Scholar Rock’s nonclinical and preclinical studies and clinical trials; information provided or decisions made by regulatory authorities; competition from third parties that are developing products for similar uses; Scholar Rock’s ability to obtain, maintain and protect its intellectual property; Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials; and Scholar Rock’s ability to manage expenses and to obtain additional funding when needed to support its business activities and establish and maintain strategic business alliances and new business initiatives, and as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law. Scholar Rock Holding Corporation Condensed Consolidated Statements of Operations (unaudited) (in thousands, except share and per share data) Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Operating expenses Research and development $ 48,719 $ 30,337 $ 134,185 $ 86,939 General and administrative 16,061 13,335 48,512 36,324 Total operating expenses 64,780 43,672 182,697 123,263 Loss from operations (64,780 ) (43,672 ) (182,697 ) (123,263 ) Other income (expense), net 301 1,313 2,857 3,600 Net loss $ (64,479 ) $ (42,359 ) $ (179,840 ) $ (119,663 ) Net loss per share, basic and diluted $ (0,66 ) $ (0,53 ) $ (1,86 ) $ (1,49 ) Weighted average common shares outstanding, basic and diluted 97,050,637 80,606,438 96,587,149 80,115,143 Scholar Rock Holding Corporation Condensed Consolidated Balance Sheets (unaudited) (in thousands) September 30, 2024 December 31, 2023 Assets Cash, cash equivalents and marketable securities $ 139,065 $ 279,938 Other current assets 13,862 8,256 Total current assets 152,927 288,194 Other assets 26,206 22,841 Total assets $ 179,133 $ 311,035 Liabilities and Stockholders' Equity Current liabilities $ 39,372 $ 32,741 Long-term liabilities 60,565 53,076 Total liabilities 99,937 85,817 Total stockholders' equity 79,196 225,218 Total liabilities and stockholders' equity $ 179,133 $ 311,035