Acetyl-L-carnitine

「  本文共：16条资讯，阅读时长约：3分钟 」今日头条齐鲁ALK/ROS1抑制剂肺癌III期临床见刊。齐鲁制药新型口服ALK/ROS1抑制剂伊鲁阿克（WX-0593）在《胸部肿瘤学杂志》上公布一线治疗局部晚期或转移性ALK阳性、非小细胞肺癌（NSCLC）的III期INSPIRE研究积极结果。IRC评估的结果显示，与克唑替尼相比，WX-0593显著提高患者无进展生存期（中位PFS：27.7个月vs14.6个月），使疾病进展或者死亡风险降低了66％。两组重度不良反应的发生率相当（51.7%vs49.7%）。国内药讯1.恒瑞「他克莫司缓释胶囊」获FDA批准上市。恒瑞医药宣布「他克莫司缓释胶囊」的简略新药申请（ANDA）已获得FDA批准。他克莫司是一种强效的免疫抑制剂，能够抑制造成移植排斥反应之细胞毒淋巴细胞的生成、T细胞活化及T辅助细胞依赖型B细胞的增殖、淋巴因子的生成（如白细胞介素2、白细胞介素3及γ干扰素）以及白细胞介素2受体的表达。该产品原研产品由安斯泰来开发，美国商品名为Astagraf XL。2.亘喜双靶点FasTCAR-T获批新临床。亘喜生物靶向BCMA/CD19双靶点自体FasTCAR-T产品GC012F获FDA临床许可，即将在美国开展早线治疗多发性骨髓瘤(ELMM)Ⅰ期临床。去年2月和11月，FDA已批准GC012F在美国针对复发/难治性多发性骨髓瘤（R/R MM）和难治性系统性红斑狼疮（SLE）开展Ⅰ/Ⅱ期临床试验。在治疗R/R MM的临床试验（IIT）中，GC012F的总体应答率（ORR）达到100%。3.恒瑞PD-L1+CTLA-4单抗联合用药获批临床。恒瑞医药PD-L1单抗阿得贝利单抗注射液获国家药监局临床试验默示许可，拟联合CTLA-4单抗SHR-8068及含铂双药用于携带丝氨酸-苏氨酸激酶11（STK11）基因突变，或Kelch样环氧氯丙烷相关蛋白1（KEAP1）基因突变，或鼠类肉瘤病毒癌基因（KRAS）突变的晚期或转移性非鳞状非小细胞肺癌患者的一线治疗。4.浙江普洛终止索法地尔脑卒中III期临床。普洛药业宣布终止其1类化药注射用索法地尔治疗急性缺血性脑卒中的III期临床试验。临床试验终止后，已入组的受试者将继续按照临床试验方案进行随访。2009年，普洛药业旗下家园药业通过韩国Neurotech（GNT Pharma）授权，获得该产品在大中华区的自主知识产权。截至目前，索法地尔研发投入共计1.22亿元（未经审计）。5.疫苗研发公司康乐卫士申请港交所IPO。1月29日，康乐卫士在港交所递交IPO申请并获得受理。招股书显示，该公司拥有丰富的HPV疫苗产品组合。核心产品三价HPV疫苗将于2024年递交上市申请，九价HPV疫苗将于2025年递交上市申请，十五价HPV疫苗将于2024年启动Ⅰ期临床。康乐卫士还正在开发RSV疫苗、带状疱疹疫苗、七价诺如病毒疫苗、HFMD疫苗、脊髓灰质炎疫苗和二价治疗性HPV疫苗。国际药讯1.FDA发布基因编辑疗法和CAR-T开发指南。FDA日前针对使用基因组编辑的基因疗法和体外制造的CAR-T产品发布最终行业指南。FDA支持对基因组编辑产品使用加速批准通道，并鼓励开发商尽早讨论在研产品使用这一途径的可能性，包括在临床开发早期进行讨论，提供替代终点或中间临床终点。CAR-T开发指南除了提供关于CMC、药理学和毒理学以及临床试验设计的具体建议外，还新增了对癌症适应症的关注。2.Celltrion托珠单抗生物类似药在美报BLA。Celltrion旗下Celltrion USA公司开发的托珠单抗生物类似药CT-P47已向FDA递交生物制品许可申请（BLA）。托珠单抗原研产品（商品名：Actemra）是罗氏子公司中外制药开发的一款抗白介素-6受体（IL-6R）单抗，目前已获批用于治疗类风湿关节炎、活动性多关节幼年特发性关节炎、巨细胞动脉炎、细胞因子释放综合征、系统性硬化症相关间质性肺疾病（SSc-ILD）和新型冠状病毒感染等6项适应症。3.K药辅助治疗肾癌最新III期临床积极。默沙东PD-1抑制剂Keytruda辅助治疗肾切除术后或肾切除术联合转移灶切除术后有中高/高复发风险的肾细胞癌（RCC）患者的III期KEYNOTE-564研究结果积极。中位随访为57.2个月时，K药辅助治疗可使疾病复发或死亡风险降低28%（HR=0.72 [95% CI, 0.59-0.87]）；显著改善患者总生存期，死亡风险降低38%；研究中未出现新的安全性信号。4.Locus公司CRISPR噬菌体疗法上Ⅱ/Ⅲ期临床。Locus公司宣布获得美国生物医学高级研究与发展管理局（BARDA）2390万美元资助，将启动一项Ⅱ/Ⅲ期临床试验，评估CRISPR增强型噬菌体疗法LBP-EC01用于治疗由耐药性大肠杆菌引起的尿路感染（UTIs）患者的潜力。LBP-EC01具有独特的双重作用机制：噬菌体的天然裂解活性和CRISPR-Cas3的DNA靶向活性。该产品在杀死致病菌的同时保留有益细菌。5.体内RNA编辑疗法获批眼科Ⅰ/Ⅱ期临床。Ascidian公司RNA外显子编辑疗法ACDN-01获FDA批准开展Ⅰ/Ⅱ期临床STELLAR，评估一次性经视网膜下注射ACDN-01在患有Stargardt病和其他ABCA4相关视网膜病变的受试者中的安全性与初步疗效。Stargardt病是由ABCA4基因变异导致的一种遗传性黄斑变性。ACDN-01能够重新编辑RNA序列，将导致疾病的RNA序列替换为功能正常的RNA序列。6.吉利德与Arcus公司修订合作协议。吉利德宣布修订与Arcus公司的合作协议，并向Arcus公司追加3.2亿美元投资，将持股比例提高至33%。根据修订协议，两家公司重新确定了TIGIT抗体多凡纳利单抗（Domvanalimab）联合开发项目的优先次序，专注于推进并可能加速STAR-121（肺癌）和STAR-221（胃肠道癌）的Ⅲ期研究；注册启动一项Ⅲ期肺癌研究STAR-131（包括domvanalimab和PD-1抗体zimberelimab方案）。2020年，吉利德已与Arcus达成为期10年的合作。医药热点1.马斯克称已完成首例人类大脑设备植入手术。当地时间29日，马斯克在社交媒体上表示，其脑机接口公司Neuralink已完成首例人类大脑设备植入手术，接入者“恢复良好”。Neuralink公司的首款产品被命名为Telepathy(心灵感应)。马斯克表示，“只需要思考，就可以控制手机或电脑，并通过它们控制几乎任何设备”。首批使用者将是失去肢体功能的人。2.山东省立医院临床心理科成立。1月26日，山东第一医科大学附属省立医院（山东省立医院）临床心理科正式揭牌成立。临床心理学科在心理咨询门诊的基础上增设了病房服务。临床心理科病房的成立将助力增强专业科室的临床诊疗和科研教学水平，进一步提升医院综合服务能力和水平。3.去年跨省异地就医直接结算1.29亿人次。国家医保局29日公布2023年全国医疗保障跨省异地就医直接结算公共服务信息，信息显示，2023年1—12月，跨省异地就医直接结算运行基本平稳，跨省异地就医直接结算1.29亿人次，减少参保群众垫付1536.74亿元，分别较2022年增长238.67%、89.91%。截至2023年12月底，全国住院费用跨省联网定点医疗机构达8.23万家，较2022年底增加1.96万家。2023年1—12月，全国住院费用跨省直接结算1125.48万人次，减少个人垫付1351.26亿元，分别较2022年增长97.87%、77.25%。评审动态  1. CDE新药受理情况（01月30日) 2. FDA新药获批情况（北美01月29日）股市资讯上个交易日 A 股医药板块 -2.79%涨幅前三     跌幅前三广 誉 远 +9.98%  常山药业-11.67%通化东宝+4.78%  金凯生科-11.00%安图生物+4.21%  华熙生物-10.63%【透景生命]全资子公司上海透最申报的乙酷左旋肉碱、氯化胆碱、y-丁基甜菜碱、左旋肉碱和氧化三甲胺质控品、乙酰左旋肉碱、氯化胆碱、y-丁基甜菜碱、左旋肉碱和氧化三甲胺校准品医疗器械注册申请于近期获得了上海市药品监督管理局的受理。【明德生物]发布业绩预告，预计2023年年度实现归属于上市公司股东的净利润盈利5500万元--7500万元，同比增长98.69%至-98.22%;扣除非经常性损益后的净利润盈利-1.2亿元至-9000亿元。【君实生物]发布业绩预告，预计2023年年度实现营业收入15.41亿元左右。同比增长6.02%左右;归属于母公司所有者的净利润-22.5亿元左右，同比亏损减少5.78%左右;归属于母公司所有者扣除非经常性损益后的净利润-22.79亿元左右，同比亏损减少6.99%左右。- The End -戳“阅读原文”，了解更多医药研发及股市资讯。

Interim data from Phase 1/1b clinical trial of CPI-818, including new evidence supporting the recently implemented minimum absolute lymphocyte count (ALC) predictive biomarker Data presented at the 10th Whistler Global Summit on Hematologic Malignancies BURLINGAME, Calif., April 03, 2023 (GLOBE NEWSWIRE) -- Corvus Pharmaceuticals, Inc (Nasdaq: CRVS), a clinical-stage biopharmaceutical company, today announced interim data demonstrating the potential of CPI-818, the Company’s ITK inhibitor, for the treatment of T cell lymphoma (TCL) at the 10th Whistler Global Summit on Hematologic Malignancies, which took place March 29 to April 2, 2023 in Whistler British Columbia, Canada. The CPI-818 data was presented by John Reneau, MD, PhD, from The Ohio State University Comprehensive Cancer Center. Dr. Reneau is a hematologist who specializes in treating patients with lymphoma and an investigator for the Phase 1/1b clinical trial of CPI-818 for TCL. “T cell lymphoma is a challenging disease to treat and there is significant need for new approaches given the limited efficacy of current therapeutic options,” said Dr. Reneau. “CPI-818 has a novel mechanism of action that includes the stimulation of normal T cells to infiltrate and destroy tumors. Our recent work indicates that the peripheral blood absolute lymphocyte count is a biomarker that may predict patients most likely to benefit from CPI-818. This positions our trial to build on the results CPI-818 has already demonstrated - monotherapy activity in advanced, relapsed patients, with potential for higher response rates driven by this new biomarker.” CPI-818 Phase 1/1b T Cell Lymphoma Data CPI-818 is currently being studied in a Phase 1/1b clinical trial as a single agent therapy in patients with relapsed TCL. As of February 23, 2023, 20 patients were enrolled in the 200 mg cohort (optimal dose), including 13 evaluable for tumor response. There have been 1 complete response (CR) of 24 months duration, 1 equivocal CR awaiting confirmatory PET scan of 13+ months duration (a previous partial response (PR)), 1 nodal CR of 21 months duration and 1 PR of 7 months duration. Ten patients continue on therapy, including seven who have not yet been evaluated for tumor response. Corvus has identified minimum absolute lymphocyte count (ALC) above 900 per cubic milliliter of blood as a biomarker associated with response to CPI-818. Interim data from the 200 mg cohort in the Phase 1/1b clinical trial indicate that this minimum ALC level is required for tumor response and disease control. As of February 23, 2023, four of eight patients with ALC above 900 had objective responses (those four patients are described above), all eight had disease control (stable disease, PR, CR) and the median progression free survival (PFS) was 28.1 months. No objective responses were seen in five patients (0 of 5) with ALC below 900 and the PFS was 2.1 months. New interim data, as of February 23, 2023, presented by Dr. Reneau evaluated the effect of ALC on PFS of patients based on their last prior chemotherapy treatment before receiving CPI-818. There was no significant difference in the PFS achieved in patients with ALC less than 900 per cubic milliliter (PFS = 3.2 months) compared to patients with ALC greater than or equal to 900 per cubic milliliter (PFS = 3.4 months), which suggests the ALC biomarker is not predictive for standard therapies. This result is consistent with CPI-818’s proposed mechanism of action, which involves the enhancement of normal lymphocyte function, and indicates that the improved results seen with CPI-818 in patients with ALC above 900 is not due to more favorable patient characteristics. The swimmer and waterfall tumor plots for these patients, along with the data summarizing the ALC biomarker data, are included in Dr. Reneau’s presentation slides, which are available on the Publications and Presentations page of the Corvus website. “The data from our Phase 1/1b trial of CPI-818 for T cell lymphoma continues to be strong, with durable and deep tumor responses observed to date,” said Richard A. Miller, M.D., co-founder, president and chief executive officer of Corvus. “We are encouraged that enrollment has accelerated and that we have implemented a biomarker that we believe will enable us to enroll patients most likely to benefit from CPI-818, which is of particular importance as we plan for a registration Phase 3 randomized clinical trial. I want to thank Dr. Reneau and all our investigators for their participation in the trial and for the work they do in support of patients.” About Corvus Pharmaceuticals Corvus Pharmaceuticals is a clinical-stage biopharmaceutical company. Corvus’ lead product candidate is CPI-818, an investigational, oral, small molecule drug that selectively inhibited ITK in preclinical studies and is in a multicenter Phase 1/1b clinical trial in patients with several types of T cell lymphomas. The Company’s second clinical program, ciforadenant (CPI-444), is an oral, small molecule inhibitor of the A2A receptor that is in an open-label Phase 1b/2 clinical trial. Its third clinical program, mupadolimab (CPI-006), is a humanized monoclonal antibody directed against CD73 that has exhibited immunomodulatory activity and activation of immune cells in preclinical and clinical studies. For more information, visit . About CPI-818 CPI-818 is an investigational small molecule drug given orally that has selectively inhibited ITK (interleukin-2-inducible T cell kinase) in preclinical studies. It was designed to block malignant T cell growth and to modulate immune responses. ITK, an enzyme, is expressed predominantly in T cells and plays a role in T cell and natural killer (NK) cell lymphomas and leukemias, as well as in normal immune function. Recent clinical data in T cell lymphomas suggests that CPI-818 has the potential to control differentiation of T helper cells and enhance immune responses to tumors. Interference with ITK signaling also can modulate immune responses to various antigens. Optimal doses of CPI-818 have been shown to affect T cell differentiation and induce the generation of Th1 helper cells while blocking the development of both Th2 and Th17 cells and production of Th2 related cytokines. Th1 T cells are required for immunity to tumors, viral infections and other infectious diseases. Th2 and Th17 helper T cells are involved in the pathogenesis of many autoimmune and allergic diseases. The immunologic effects of CPI-818 lead to what is known as Th1 skewing and is made possible by the high selectivity of CPI-818 for ITK. The Company believes the inhibition of specific molecular targets in T cells may be of therapeutic benefit for patients with T cell lymphomas, solid tumors, and in patients with autoimmune and allergic diseases. The Company is conducting a Phase 1/1b trial in patients with refractory T cell lymphomas that was designed to select the optimal dose of CPI-818 and evaluate its safety, PK, target occupancy, immunologic effects, biomarkers and efficacy. Interim data from the Phase 1/1b clinical trial of CPI-818 for T cell lymphoma demonstrated tumor responses in very advanced, refractory, difficult to treat T cell malignancies, and identified a dose that maximally affects T helper cell differentiation. Forward-Looking Statements This press release contains forward-looking statements, including statements related to the potential safety and efficacy of CPI-818, ciforadenant and mupadolimab; the Company’s ability to develop and advance product candidates into and successfully complete preclinical studies and clinical trials, including the Company Phase 1/1b clinical trial of CPI-818 and the Company’s planned Phase 3 registration clinical trial with CPI-818 for T cell lymphoma; the design of clinical trials, including the target number of patients to be enrolled and whether identifying certain biomarkers will help accelerate enrollment; and the timing of the availability and announcement of clinical data and certain other product development milestones. All statements other than statements of historical fact contained in this press release are forward-looking statements. These statements often include words such as “believe,” “expect,” “anticipate,” “intend,” “plan,” “estimate,” “seek,” “will,” “may” or similar expressions. Forward-looking statements are subject to a number of risks and uncertainties, many of which involve factors or circumstances that are beyond the Company’s control. The Company’s actual results could differ materially from those stated or implied in forward-looking statements due to a number of factors, including but not limited to, risks detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the Securities and Exchange Commission on March 28, 2023, as well as other documents that may be filed by the Company from time to time with the Securities and Exchange Commission. In particular, the following factors, among others, could cause results to differ materially from those expressed or implied by such forward-looking statements: the Company’s ability to demonstrate sufficient evidence of efficacy and safety in its clinical trials of CPI-818, ciforadenant and mupadolimab; the accuracy of the Company’s estimates relating to its ability to initiate and/or complete preclinical studies and clinical trials; the results of preclinical studies and interim data from clinical trials not being predictive of future results; the unpredictability of the regulatory process; regulatory developments in the United States, and other foreign countries; the costs of clinical trials may exceed expectations; and the Company’s ability to raise additional capital. Although the Company believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the events and circumstances reflected in the forward-looking statements will be achieved or occur, and the timing of events and circumstances and actual results could differ materially from those projected in the forward-looking statements. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and the Company undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise. INVESTOR CONTACT: Leiv Lea Chief Financial Officer Corvus Pharmaceuticals, Inc. +1-650-900-4522 llea@corvuspharma.com MEDIA CONTACT: Sheryl Seapy Real Chemistry +1-949-903-4750 sseapy@realchemistry.com