Tenacia Biotechnology (Shanghai) Co. Ltd.

RADNOR, Pa.--( BUSINESS WIRE )-- Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today reported business highlights and financial results for the second quarter ended June 30, 2024. “ Since launching ZTALMY in the U.S. two years ago, we have seen significant growth and adoption with strong revenue for the second quarter, underscoring the appreciation physicians, patients and caregivers have for ZTALMY and its role in the treatment of CDKL5 deficiency disorder,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus. “ We continue to grow the brand globally with ZTALMY now approved in the U.S., EU, UK and China. We look forward to continuing to expand access to this important treatment option while generating revenue from markets outside the U.S.” Dr. Braunstein continued, “ Our proven commercial infrastructure and success in CDD provides us with a solid foundation to expand into TSC, which represents a significant unmet need in a larger refractory epilepsy population. With enrollment complete in the Phase 3 TrustTSC trial and data upcoming, we continue to make the appropriate investments and plan for a potential launch in the fall of 2025. We will be hosting an investor and analyst event on September 20 th , where senior management and key opinion leaders will discuss the treatment landscape, market potential and commercial opportunity for ZTALMY in TSC.” ZTALMY ® (ganaxolone) Oral Suspension CV Generated net product revenue of $8.0 million for the second quarter of 2024 representing 87% growth versus the second quarter of 2023 On track to achieve full year 2024 ZTALMY net product revenue guidance of between $33 and $35 million Continue to expand global access: Activated managed access programs for named patients in the Middle East and North Africa (MENA), Canada and Russia with ex-U.S. revenue expected in the third quarter of 2024 In July, ZTALMY was approved in China for patients with CDKL5 deficiency disorder (CDD); Tenacia Biotechnology is anticipating commercial launch in early 2025 Orion Corporation continues to prepare for commercial launches of ZTALMY in select European countries in the second half of 2024 with regulatory approvals secured in the European Union and United Kingdom for the CDD indication Clinical Pipeline Tuberous Sclerosis Complex Enrollment in the global Phase 3 TrustTSC trial of oral ganaxolone in tuberous sclerosis complex (TSC) is complete with last patient visit expected in September; topline data is on track for the first half of the fourth quarter of 2024 Targeting submission of a supplemental New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in April 2025 with a request for priority review TrustTSC maintains a low discontinuation rate of less than 7% with greater than 85% of patients to date continuing to the open-label extension Launch planning and market access landscape assessment underway in preparation of anticipated commercial launch in the second half of 2025 Other Rare Genetic Epilepsies Expect to initiate a proof-of-concept study with ZTALMY to treat a range of developmental and epileptic encephalopathies, including Lennox-Gastaut syndrome, in the first half of 2025, pending the TSC topline data Targeting submission of an Investigational New Drug application for a novel oral ganaxolone prodrug in the fourth quarter of 2025 Status Epilepticus Announced topline results from the Phase 3 RAISE trial of intravenous (IV) ganaxolone in refractory status epilepticus Planning to submit a request for a Type C meeting with the FDA to discuss the RAISE trial results and next steps for the IV ganaxolone program Continue to offer IV ganaxolone for patients with super refractory status epilepticus under emergency investigational new drug applications with 31 patients treated to date Ganaxolone development in the RAISE trial has been supported in part by the Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority (BARDA) under contract number 75A50120C00159. General Business and Financial Update On August 1, 2024, the Patent Trial and Appeal Board issued its final written decision in the post-grant review of Ovid Therapeutics’ U.S. Patent No. 11,395,817 for IV ganaxolone in the treatment of status epilepticus, determining that all remaining claims—claims 22 and 25-31—were unpatentable as obvious. The decision is publicly available on the Patent Office’s website here . In June 2024, Marinus restructured its agreements with Oaktree Capital Management, L.P. and Sagard Healthcare Partners. Under the restructuring, the $15 million minimum liquidity requirement was removed from both the Oaktree and Sagard agreements, and amortization payments due to Oaktree in 2024 have been reduced by 50%. In return, Marinus made a one-time principal payment of $15 million to Oaktree in the second quarter of 2024. Cost reduction plans initiated in the second quarter of 2024 are expected to reduce combined selling, general and administrative (SG&A) and R&D expenses by approximately 30% from $80.3 million in the first half of 2024 to between $55 and $60 million in the second half of 2024. The full on-going impact of cost savings is expected to be achieved in the third quarter of 2024. Full year 2024 guidance remains unchanged with projected ZTALMY net product revenue between $33 and $35 million and combined SG&A and R&D expenses in the range of approximately $135 to $140 million, including stock-based compensation expense of approximately $20 million. Through the execution of the cost reduction plans and restructured financing agreements, the Company expects that cash and cash equivalents of $64.7 million as of June 30, 2024, will be sufficient to fund the Company’s operating expenses and capital expenditure requirements into the second quarter of 2025. The Company continues to make investments to expand ZTALMY manufacturing capacity necessary for the global launch of the CDD indication and potential TSC expansion. Financial Results Recognized $8.0 million and $15.5 million in net product revenue for the three and six months ended June 30, 2024, respectively, as compared to $4.2 million and $7.6 million for the same periods in the prior year, respectively. Recognized $0.1 million and $0.2 million in Biomedical Advanced Research and Development Authority (BARDA) federal contract revenue for the three and six months ended June 30, 2024, respectively, as compared to $1.8 million and $8.9 million for the same periods in the prior year, respectively. The decrease was primarily driven by activity associated with the start-up of the API onshoring initiative in the first quarter of 2023 and completion of the base period funding in the fourth quarter of 2023. Research and development (R&D) expenses were $20.9 million and $45.0 million for the three and six months ended June 30, 2024, respectively, as compared to $21.4 million and $49.3 million for the same periods in the prior year, respectively. The three month decrease was due primarily to reduced personnel costs in 2024, partially offset by increased costs associated with the RAISE Trial. The six month decrease was also driven by costs associated with start-up of the API onshoring effort in the first quarter of 2023. Selling, general and administrative (SG&A) expenses were $16.7 million and $35.3 million for the three and six months ended June 30, 2024, respectively, as compared to $15.7 million and $30.9 million for the same periods in the prior year, respectively; the primary drivers of the increases were increased commercial spending and headcount costs in 2024. Restructuring costs were $2.0 million for the three months ended June 30, 2024, resulting from cost saving initiatives implemented in the second quarter of 2024. The Company had net losses of $35.8 million and $74.5 million for the three and six months ended June 30, 2024, respectively; cash used in operating activities increased to $68.3 million for the six months ended June 30, 2024, compared to $65.8 million for the same period a year ago. At June 30, 2024, the Company had cash and cash equivalents of $64.7 million, compared to cash, cash equivalents and short-term investments of $150.3 million at December 31, 2023. Readers are referred to, and encouraged to read in its entirety, the Company’s Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2024, to be filed with the Securities and Exchange Commission, which includes further detail on the Company’s business plans, operations, financial condition, and results of operations. Selected Financial Data (in thousands, except share and per share amounts) June 30, 2024 (unaudited) December 31, 2023 ASSETS Cash and cash equivalents $ 64,676 $ 120,572 Short-term investments - 29,716 Other assets 22,407 20,620 Total assets $ 87,083 $ 170,908 LIABILITIES AND STOCKHOLDERS’ (DEFICIT) EQUITY Current liabilities $ 35,867 $ 40,624 Long term debt, net 45,075 61,423 Revenue interest financing payable, net 35,431 33,766 Other long-term liabilities 18,055 18,330 Total liabilities 134,428 154,143 Total stockholders’ (deficit) equity (47,345) 16,765 Total liabilities and stockholders’ (deficit) equity $ 87,083 $ 170,908 Three Months Ended June 30, Six Months Ended June 30, 2024 (Unaudited) 2023 (Unaudited) 2024 (Unaudited) 2023 (Unaudited) Revenue: Product revenue, net $ 7,951 $ 4,249 $ 15,460 $ 7,581 Federal contract revenue 87 1,814 239 8,862 Collaboration revenue 18 18 36 18 Total revenue 8,056 6,081 15,735 16,461 Expenses: Research and development 20,897 21,412 45,015 49,345 Selling, general and administrative 16,710 15,722 35,336 30,926 Restructuring Costs 1,950 - 1,950 - Cost of product revenue 735 386 1,491 592 Total expenses: 40,292 37,520 83,792 80,863 Loss from operations (32,236 ) (31,439 ) (68,057 ) (64,402 ) Interest income 1,109 2,128 2,571 4,471 Interest expense (4,617 ) (4,208 ) (8,963 ) (8,355 ) Other (expense) income, net (84 ) 47 (48 ) 84 Loss before income taxes (35,828 ) (33,472 ) (74,497 ) (68,202 ) Benefit for income taxes - 1,538 - 1,538 Net loss applicable to common shareholders $ (35,828 ) $ (31,934 ) $ (74,497 ) $ (66,664 ) Per share information: Net loss per share of common stock—basic and diluted $ (0.63 ) $ (0.61 ) $ (1.31 ) $ (1.28 ) Basic and diluted weighted average shares outstanding 57,064,095 52,551,918 56,957,953 52,162,962 Other comprehensive loss Unrealized (loss) gain on available-for-sale securities - (188 ) 20 (114 ) Total comprehensive loss $ (35,828 ) $ (32,122 ) $ (74,477 ) $ (66,778 ) Conference Call Information Tuesday, August 13, 8:30 a.m. ET Domestic: (877) 405-1242 International: (201) 389-0852 Webcast Registration: Click Here An archived version of the call will be available approximately two hours after the completion of the event on the Marinus website at ir.marinuspharma.com/events-and-presentations . About Marinus Pharmaceuticals Marinus is a commercial-stage pharmaceutical company dedicated to the development of innovative therapeutics for seizure disorders. The Company’s product, ZTALMY ® (ganaxolone) oral suspension CV, is an FDA-approved prescription medication introduced in the U.S. in 2022. For more information, please visit www.marinuspharma.com and follow us on LinkedIn , X and Facebook . Forward-Looking Statements To the extent that statements contained in this press release are not descriptions of historical facts regarding Marinus, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Words such as "may", "will", "expect", "anticipate", "estimate", "intend", "believe", and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Examples of forward-looking statements contained in this press release include, among others, statements regarding our commercialization and marketing plans for ZTALMY; our net product revenue and other financial guidance and projections; the potential benefits ZTALMY will provide for physicians and patients; statements regarding our expected clinical development plans, enrollment in our clinical trials, regulatory communications and submissions for ganaxolone, and the timing thereof; our expected data readouts; our expected cash runway; our expectations and beliefs regarding the FDA and EMA with respect to our product candidates; our expectations regarding the development of new formulations and prodrug candidates; our expectations regarding our strategic partners; our expectations regarding our cost reduction plans; our plans to continue to expand global access; the potential safety and efficacy of ganaxolone, as well as its therapeutic potential in a number of indications; and other statements regarding the company's future operations, financial performance, financial position, prospects, objectives and other future event. Forward-looking statements in this press release involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the Company’s ability to continue as a going concern; unexpected market acceptance, payor coverage or future prescriptions and revenue generated by ZTALMY; unexpected actions by the FDA or other regulatory agencies with respect to our products; competitive conditions and unexpected adverse events or patient outcomes from being treated with ZTALMY, uncertainties and delays relating to the design, enrollment, completion, and results of clinical trials; unanticipated costs and expenses; the company’s cash and cash equivalents may not be sufficient to support our operating plan for as long as anticipated; our ability to comply with the FDA’s requirement for additional post-marketing studies in the required time frames; the timing of regulatory filings for our other product candidates; clinical trial results may not support regulatory approval or further development in a specified indication or at all; actions or advice of the FDA or EMA may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional clinical trials; the size and growth potential of the markets for the company’s product candidates, and the company’s ability to service those markets; our ability to develop new formulations of ganaxolone or prodrugs; our ability to obtain, maintain, protect and defend intellectual property for our product candidates; the potential negative impact of third party patents on our or our collaborators’ ability to commercialize ganaxolone; delays, interruptions or failures in the manufacture and supply of our product candidate; uncertainties related to the company’s expectations, projections and estimates regarding expenses, future revenue, capital requirements, and the availability of and the need for additional financing; the company’s ability to obtain additional funding to support its clinical development and commercial programs; the potential for our ex-US partners to breach their obligations under their respective agreements with us or terminate such agreements in accordance with their respective terms; the risk that drug product quality requirements may not support continued clinical investigation of our product candidates and result in delays or termination of such clinical trials and product approvals; and the availability or potential availability of alternative products or treatments for conditions targeted by us that could affect the availability or commercial potential of our product candidates. This list is not exhaustive and these and other risks are described in our periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the Securities and Exchange Commission and available at www.sec.gov . Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

▎药明康德内容团队编辑 7月进入尾声。根据中国国家药监局（NMPA）官网批件信息，截至7月29日，本月共计有16款新药*获批，其中6款为首次获批，10款为获批新适应症或新剂型。其中，首次在中国获批上市的6款新药获批的适应症包括2型糖尿病、“糖痛”、真性红细胞增多症、血友病A、癫痫、强直性脊柱炎。获批新适应症或新剂型的10款新药，则为弥漫性大B细胞淋巴瘤、非小细胞肺癌、重症肌无力、体重管理、类风湿关节炎等患者带来了新的治疗选择。（*新药定义：根据NMPA受理号类型为X的获批药物，不含生物类似药） 图片来源：123RF 信立泰：苯甲酸福格列汀片 适应症：2型糖尿病 7月4日，NMPA官网宣布批准信立泰1类创新药苯甲酸福格列汀片上市，适用于改善成人2型糖尿病患者的血糖控制。苯甲酸福格列汀片是一款DPP-4抑制剂，具有口服吸收迅速、半衰期长、作用持久等特点。研究结果显示，无论是单药治疗还是联合二甲双胍治疗2型糖尿病患者，在给药24周后，福格列汀对比安慰剂都能显著地降低HbA1c，具有明显的降糖效果。 第一三共：苯磺酸美洛加巴林片 适应症：成人糖尿病性周围神经病理性疼痛 7月5日，NMPA官网公示，第一三共（Daiichi Sankyo）苯磺酸美洛加巴林片获批上市，用于治疗成人糖尿病性周围神经病理性疼痛（俗称“糖痛”）。美洛加巴林片为第三代钙离子通道调节剂类药物，其作用位点是钙离子通道的α2δ亚基，与钙离子通道α2δ亚基亲和力高，但与α2δ-1亚基（更多与镇痛作用相关）解离慢，与α2δ-2亚基（更多与中枢不良反应相关）解离快，可更好地平衡疗效和安全性，镇痛作用更强且更持久。 药华生物：罗培干扰素α-2b注射液 适应症：真性红细胞增多症 7月5日，NMPA官网公示，药华医药自主研发的罗培干扰素正式获得NMPA批准上市，用于治疗真性红细胞增多症（PV），这是一种造血干细胞恶性肿瘤。罗培干扰素是单一异构体超长效干扰素，可2~4周注射1次。经多项临床研究显示，该产品可快速有效控制血细胞的过度增殖、并显著降低致病基因的突变负荷，从而有效降低血栓栓塞并发症及其所导致的死亡风险。 诺和诺德：注射用培妥罗凝血素α 适应症：血友病A 7月5日，NMPA官网公示，诺和诺德（Novo Nordisk）注射用培妥罗凝血素α在华获批，用于12岁及以上成人和儿童血友病A患者按需治疗及控制出血事件、围手术期管理、常规预防治疗以防止或减少出血事件的发生。根据诺和诺德新闻稿，这是中国获批的首个长效重组凝血因子VIII（FVIII），开启了血友病长效治疗的新时代。注射用培图罗凝血素α（N8-GP）经创结构修饰后可将半衰期延长1.6倍，以长效治疗帮助血友病患者减少输液次数，减轻治疗负担。 元羿生物：加那索龙口服混悬剂 适应症：CDKL5缺乏症患者癫痫发作 7月16日，NMPA官网公示，元羿生物引进自Marinus公司的的加那索龙口服混悬剂在中国获批上市，用于治疗2岁及以上细胞周期蛋白依赖性激酶5（CDKL5）缺乏症患者癫痫发作。这是一款靶向GABAA受体的阳性别构调节剂。CDKL5缺乏症（或称为“希舞综合征”）是一种罕见、严重且快速进展的遗传性神经发育性疾病，严重影响患者及其家庭的健康和生活质量。根据元羿生物新闻稿介绍，一项2年长期开放性研究结果显示，使用加那索龙的患者中，30~50%患者癫痫发作频次下降≥50%，近10%的患者实现了3个月内无发作。 优时比：比奇珠单抗注射液 适应症：强直性脊柱炎 7月19日，NMPA官网公示，优时比（UCB）公司白介素-17A/F（IL-17A/F）抑制剂比奇珠单抗注射液上市申请已获得批准。根据业内公开资料，该药本次获批的适应症为常规治疗疗效不佳或不耐受的活动性强直性脊柱炎（AS，放射学阳性中轴型脊柱关节炎）成人患者。IL-17是AS研究的热门靶点之一，其是AS发病机制中关键的炎症细胞因子。根据优时比公布的一项3期临床结果，比奇珠单抗在广泛的脊柱关节炎患者具有较好的治疗潜力。 除了上述新药，还有10款新药在中国获批新适应症或获批新剂型： 海思科：苯磺酸克利加巴林胶囊 获批适应症：带状疱疹后神经痛（PHN） 继今年5月获批首个适应症（成人糖尿病性周围神经病理性疼痛）后，海思科口服GABA类似物1类新药苯磺酸克利加巴林胶囊在7月初迎来第二个适应症获批，用于治疗带状疱疹后神经痛（PHN）。在针对该适应症的3期临床试验中，克利加巴林胶囊40mg/日、80mg/日均在统计意义上显著优于安慰剂，研究达到主要疗效终点。 阿斯利康：布地奈德福莫特罗吸入气雾剂 (III) 适应症：慢性阻塞性肺疾病（推测） 阿斯利康（AstraZeneca）布地奈德福莫特罗吸入气雾剂 (III)获批新适应症，这是一款吸入糖皮质激素/长效β2受体激动剂联合疗法，此前已于2021年在中国获批治疗哮喘。其已经在多个国家和地区获批用于治疗哮喘或慢性阻塞性肺疾病。 德琪医药：塞利尼索 适应症：弥漫性大B细胞淋巴瘤 德琪医药引进的口服XPO1抑制剂塞利尼索获批弥漫性大B细胞淋巴瘤（DLBCL）新适应症。中国注册性临床试验SEARCH研究的试验数据显示，60例接受塞利尼索治疗的中国受试者的总体缓解率（ORR）达到试验预设的主要终点。 亿腾医药：二十碳五烯酸乙酯（IPE）软胶囊 适应症：降低心血管事件风险 亿腾医药引进的心血管创新药二十碳五烯酸乙酯（IPE）软胶囊获批新适应症，用于降低心血管事件风险（CRR）。这是亿腾医药引进自Amarin公司开发的一款含高度纯化活性成分IPE的单分子处方药。临床研究表明，与安慰剂组相比，IPE可显著减少25%的主要心血管事件（MACE），包括心血管死亡、非致死性心梗、非致死性卒中、冠状动脉血运重建和需要住院的不稳定心绞痛。 罗氏：盐酸阿来替尼胶囊 适应症：IB期至IIIA期非小细胞肺癌 罗氏（Roche）盐酸阿来替尼胶囊获批新适应症，用于ALK阳性的IB期至IIIA期非小细胞肺癌患者术后辅助治疗。此次获批标志着阿来替尼成为了同时覆盖早期和晚期的ALK酪氨酸激酶抑制剂（ALK-TKI）。ALINA研究结果显示，与以铂为基础的化疗组相比，使用阿来替尼口服辅助治疗2年，可以使患者疾病复发或死亡风险降低76%，使患者中枢神经系统复发或死亡风险降低78%。 再鼎医药：艾加莫德α注射液 获批新剂型：皮下注射，治疗重症肌无力 再鼎医药和argenx公司艾加莫德α注射液（皮下注射）在中国获批，用于治疗乙酰胆碱受体（AChR）抗体阳性的成人全身型重症肌无力（gMG）患者。艾加莫德是一款靶向FcRn的抗体片段，其静脉注射剂型此前已经在中国获批治疗重症肌无力。根据再鼎医药新闻稿介绍，皮下注射剂型将在30-90秒内以单次皮下注射的方式给药，连续4周每周注射一次为一治疗周期。 礼来：替尔泊肽注射液 适应症：长期体重管理 继今年5月在中国获批首个适应症（2型糖尿病）之后，礼来（Eli Lilly and Company）替尔泊肽注射液在中国获批新适应症，用于成人肥胖或超重患者长期体重管理。替尔泊肽是一款每周一次注射的GIP和GLP-1受体激动剂。在纳入中国受试者的3期SURMOUNT-CN研究中，第52周时，替尔泊肽10mg组、15mg组平均体重自基线分别降低14.4%、19.9%，均优效于安慰剂组（降低2.4%）。 康哲药业、Dr. Falk Pharma：熊去氧胆酸口服混悬液 获批新剂型：口服混悬液 康哲药业与Dr. Falk Pharma联合申请的熊去氧胆酸口服混悬液的两项新药上市许可申请（NDA）接连获批，分别用于治疗1月龄至18岁患者的囊性纤维化相关肝病；治疗胆囊胆固醇结石（必须是X射线能穿透的结石，同时胆囊收缩功能须正常）、胆汁淤积性肝病（如原发性胆汁性肝硬化）和胆汁反流性胃炎。根据康哲药业新闻稿，熊去氧胆酸是一种亲水、非细胞毒性的胆汁酸，包含了片剂、胶囊、口服混悬剂等剂型。康哲药业于1998年开始独家代理熊去氧胆酸胶囊（商品名为优思弗）。本次获批产品为口服混悬液剂型，相较于片剂和胶囊剂型，更适用于儿童或吞咽困难的患者。 荣昌生物：泰它西普 适应症：类风湿关节炎（RA） 荣昌生物BLyS/APRIL双靶点融合蛋白创新药泰它西普获批新适应症，用于对甲氨蝶呤疗效不佳的中、重度活动性类风湿关节炎（RA）成人患者。这是继系统性红斑狼疮之后的第二项适应症。根据荣昌生物新闻稿，最新研究数据显示，自第4周起，泰它西普显著提升RA患者ACR20应答率，24周ACR20应答率67.4%，48周ACR应答率持续提高，达72.2%；显著延缓RA患者的关节结构损害，92.8%患者24周无影像学进展，89.9%患者48周无影像学进展；显著改善RA患者的炎症水平和患者身体机能，缓解疼痛；达标（DAS28-ESR≤3.2）患者比例持续增加至48周，且安全可耐受。 微芯生物：西格列他钠 适应症：联合二甲双胍治疗2型糖尿病 继2021年10月获批单药治疗2型糖尿病后，微芯生物全新机制的PPAR全激动剂西格列他钠在今年7月获批新适应症，联合二甲双胍用于治疗2型糖尿病。根据微芯生物新闻稿介绍，西格列他钠是首个获批治疗2型糖尿病的PPAR全激动剂，其全新作用机制打破了胰岛素抵抗难以对因治疗的长期困境，有效针对代谢紊乱所引发的代谢综合症，为2型糖尿病的治疗开辟了新路径。 期待这些新药能够早日来到患者身边，为遭受疾病困扰的患者群体提供更多更好的治疗选择。 参考资料： [1]中国国家药监局官网.Retrieved July 30，2024, From https://www.nmpa.gov.cn/zwfw/zwfwpjfbzs/index.html [2]各公司官网及公开资料 本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权及其他合作需求，请联系wuxi_media@wuxiapptec.com。 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。

全球药物批准/研发动态 01 全球新药批准情况 根据药渡数据统计分析，本次统计周期（2024.07.13-07.19）全球（不含中国）共有2个新药获批上市。其中，BLA批准1个，新适应症批准1个。本次批准新药数量与上个统计周期相同。 7月15日，Daewoong Pharmaceutical宣布其肉毒杆菌毒素产品Nabota获得了阿根廷ANMAT的批准，剂量为100个单位和200个单位，商品名为Clodew。Daewoong计划于今年第四季度通过其合作伙伴Oxapharma S.A.在阿根廷推出Crodew。这是第一个也是唯一一个在阿根廷获得批准的韩国肉毒杆菌毒素，利用Clodew的批准，未来可扩大Daewoong在拉丁美洲BTX市场的影响力。 7月18日，Phathom Pharmaceuticals宣布，FDA已批准VOQUEZNA（Vonoprazan）10毫克片剂，用于缓解与成人非糜烂性胃食管反流病相关的胃灼热。该药物也被批准用于治疗所有严重程度的糜烂性食管炎（EE），并与抗生素联合用于根除幽门螺杆菌（H. pylori）感染。研究结果显示，服用VOQUEZNA的患者无胃灼热天数的平均百分比为45%，安慰剂组为28%（p<0.001），24小时无胃灼热天数的中位数百分比分别为48%和17%。 全球（不含中国）新药批准情况（部分） 02 全球新药申报进展 根据药渡数据统计分析，本次统计周期（2024.07.13-07.19）全球（不含中国）共有6个新药申报上市。其中，NDA申报1个，BLA申报5个。与上次统计周期相比，本次增加4个NDA/BLA申报。 7月19日，百奥泰宣布BAT2206乌司奴单抗注射液的生物制品上市申请获FDA和EMA受理。BAT2206是IL-12和IL-23共有的p40亚基的全人源单克隆抗体，IL-12和IL-23可与p40亚基结合，破坏IL-12和IL-23介导的信号传导和细胞因子的效应。 NDA/BLA申报（部分） 根据药渡数据统计分析，本次统计周期（2024.07.13-07.19）全球（不含中国）共有5个药物获监管机构特殊资格认定。其中，化学药2个，生物药1个，细胞疗法2个。与上次统计周期相比，本次增加1个获监管机构特殊资格认定的药物。 7月15日，SMPA宣布FDA授予DSP-5336快速通道资格，用于治疗患有KMT2A重排[也称为混合谱系白血病重排（MLLr）或核磷蛋白突变（NPM1m）]的复发或难治性急性髓系白血病（AML）患者。DSP-5336是一种研究性小分子menin和混合谱系白血病（MLL）蛋白相互作用抑制剂，I/II期研究临床结果显示57%的患者观察到客观反应，其中包括具有核磷蛋白1（NPM1）突变和KMT2A（MLL）重排的患者的反应，完全缓解或完全缓解伴部分血液学恢复（CR/CRh）的比例为24%。 7月16日，迈威生物宣布FDA授予7MW3711（B7-H3 ADC）孤儿药资格认定，用于治疗小细胞肺癌。7MW3711注射人体后，可与肿瘤细胞表面的抗原B7-H3结合内吞进入肿瘤细胞，通过特定酶解作用，定向释放小分子，从而实现对肿瘤的精准杀伤。7MW3711具有结构稳定，组分均一，纯度高，易于产业化放大等特点，同时具有良好的药物安全性及药代特性。 特殊资格认定情况 03 全球新药研发进展 根据药渡数据统计分析，本次统计周期（2024.07.13-07.19）全球（不含中国）新药临床研发状态更新共计34条，涉及肿瘤、血液和淋巴疾病、遗传代谢病、神经疾病以及皮肤病等共计13个领域。 其中，眼部疾病领域临床进展更新居各领域之首，为5条：涉及化学药1条，生物药4条。 7月16日，邦耀生物宣布，其与华东师范大学、上海长征医院合作的CAR-T疗法（TyU19）治疗自身免疫疾病研究成果于7月15日正式在Cell上发表，也是顶级期刊Cell首次发表CAR-T治疗自免疾病的研究成果。 研究团队使用TyU19治疗了3例难治性自身免疫性疾病患者：1例为免疫介导的坏死性肌炎（IMNM）、2例为系统性硬化症（SSc）。IMNM受试者接受治疗后，未出现任何发热以及细胞因子风暴的症状，且总改善评分迅速从基线的72.5上升至100。2名患有严重SSc的受试者在接受治疗后，CRISS评分显著提高，修改后的Rodnan皮肤评分（mRSS）显著下降。此外，在经TyU19治疗后所有患者的B细胞均被完全清除，IMNM患者的自身抗体被彻底消除；2名SSc患者的重要器官纤维化损伤均得到了逆转，并在6个月的监测期内持续改善。 全球新药研发进展详情（部分） 04 全球医药交易事件 本次统计周期（2024.07.13-07.19）全球（含中国）医药交易时间共计43起，涉及药物权益转让、公司并购等多起交易事件。 全球医药交易时间汇总表（部分） 国内药物批准/研发动态 01 国内新药批准情况 根据药渡数据统计分析，本次统计周期（2024.07.13-07.19）国内共有5个新药获NMPA批准上市。其中，NDA批准3个，BLA批准2个。与上次统计周期相比，本次增加3个NMPA批准新药。 7月16日，元羿生物宣布，泽元安®加那索龙（Ganaxolone）口服混悬剂已获得NMPA批准，用于治疗2岁及以上细胞周期蛋白依赖性激酶5（CDKL5）缺乏症患者癫痫发作。其全球III期关键试验主要有效性分析的结果显示，17周的随机双盲安慰剂对照研究中加那索龙组28天主要运动性癫痫发作频率相对于基线的下降百分比为30.7%。一项2年长期开放性研究结果显示，使用加那索龙的患者中，30~50%患者癫痫发作频次下降≥50%，近10%的患者实现了3个月内无发作。 7月19日，微芯生物收到NMPA正式批准PPAR全激动剂西格列他钠联合二甲双胍用于治疗2型糖尿病的上市申请。西格列他钠通过提高糖尿病患者对胰岛素的敏感性、调节血糖，改善其所并发的脂质代谢紊乱，潜在降低心血管并发症的发生及其危害程度，带来更好的疗效和安全性，实现对糖尿病及其并发症的综合治疗，联合二甲双胍可更好地实现持续降糖、调节血脂和降低心血管风险等多方面临床获益。 中国新药批准情况（部分） 02 国内新药临床默示许可进展 根据药渡数据统计分析，本次统计周期（2024.07.13-07.16）国内共有18个新药获临床默示许可，涉及28个受理号。其中，化学药8个，治疗用生物制品9个，预防用生物制品1个。与上个统计周期相比，本次减少2个临床默示许可获批受理号。 本周国内新药临床默示许可进展（部分） 03 国内新药申报进展 根据药渡数据统计分析，本次统计周期（2024.07.13-07.19）国内共有17个新药申报上市，涉及29个受理号。其中，化药7个，治疗用生物制品10个。与上个统计周期相比，本次增加24个新药申报上市受理号。 国内新药申报上市情况（部分） 根据药渡数据统计分析，本次统计周期（2024.07.13-07.19）国内共有35个新药申报临床，涉及47个受理号。其中，化学药14个，治疗用生物制品18个，预防用生物制品3个。与上个统计周期相比，本次减少48个临床申报受理号。 国内新药临床申报情况（部分） 根据药渡数据统计分析，本次统计周期（2024.07.13-07.19）国内无药物获NMPA特殊资格认定。 04 国内新药研发进展 根据药渡数据统计分析，本次统计周期（2024.07.13-07.19）国内新药临床研发状态无更新。 05 国内新药研发领域政策法规动态 关于公开征求《疫苗佐剂非临床研究技术指导原则（征求意见稿）》意见的通知 为更好地指导疫苗佐剂非临床研究和评价，促进新型佐剂以及创新佐剂疫苗的研发，经广泛调研和讨论，我中心组织起草了《疫苗佐剂非临床研究技术指导原则（征求意见稿）》。 我们诚挚地欢迎社会各界对征求意见稿提出宝贵意见和建议，并及时反馈给我们，以便后续完善。征求意见时限为自发布之日起1个月。 06 国内新药研发领域热点新闻 逆势上涨的医药“黑马” 行情再差，也有“逆行者”。 今年上半年，医药板块下跌了21.09%，严重跑输大盘（沪深300上涨了0.89%）。但也不乏逆势上涨的药企，比如占据涨幅榜前三的诺泰生物、川宁生物、艾力斯。 值得一提的是，这些“逆行者”都有一个共同点，就是基本面靓丽。 这说明，在行情不好时，资本市场更看重企业的基本面，也就是公司的内在价值、财务状况、业务模式、市场竞争力等核心要素。这也是投资者在市场波动中寻求稳定回报和长期价值的必然选择。更多资讯，请阅读原文 下半年，国产创新药将有哪些“爆点”？ 上半年的中国制药业，不客气地讲，十分令人失望，CXO全线溃败、创新药半死不活、中药苦苦支撑。反应在资本市场方面，便是股价没有最低只有更低，一路向下头也不回。造成这一局面有多方面的因素，行业周期、新冠后遗症、地缘政治的影响等等。但归根结底，还是中国制药业的内生动力不足。 下半年，能否有惊喜？更多资讯，请阅读原文 自免领域：口服小分子创新药崭露头角 目前，治疗自身免疫疾病的核心药物包括三大类：改善病情抗风湿药、非甾体抗炎药、甾体抗炎药等传统小分子药物；大分子生物制剂和小分子靶向药。生物制剂正在取代传统抗炎药物，成为自免疾病领域的主流治疗方案。 然而，自免生物制剂仍存在诸多不足：生物制剂多采用静脉或皮下注射方式递送，降低了患者对治疗的顺应性和依从性；长期用药经济负担较大。在此背景下，口服小分子靶向药应用而生。目前，已有一批自免小分子候选药在临床后期阶段取得了积极的疗效数据，未来，口服小分子自免创新药有望凭借用药便利性和差异化疗效获得可观的市场份额。更多资讯，请阅读原文 先声药业第四代抗失眠药国内申报上市 今日（7月16日），国家药品监督管理局药品审评中心（CDE）官网公示，先声药业与Idorsia公司联合申报的抗失眠药物盐酸达利雷生（Daridorexant）片的上市申请获CDE受理。 从药渡数据检索可知，达利雷生是一款双食欲素受体（OX1和OX2）拮抗剂，2022年1月获FDA批准上市。食欲素受体拮抗剂是第四代抗失眠药物，与传统催眠药相比，食欲素拮抗剂具有较少的副作用，记忆障碍和依赖潜力较小。研究表明白天给予食欲素激动剂能够促进清醒，而夜间给予食欲素受体拮抗剂可以促进睡眠。 2022年11月先声药业与Idorsia公司就达利雷生在中国的开发签订独家许可协议，根据协议……更多资讯，请阅读原文 国内首款IL-17A/IL-17F抗体药物获批上市 7月19日，NMPA官网公示优时比公司申报的白细胞介素17A（IL-17A）和白细胞介素17F（IL-17F）生物制剂比奇珠单抗（Bimekizumab）获批上市，这也是国内首款获批上市的IL-17A/IL-17F单抗。 白细胞介素-17（IL-17）家族包括六个成员（IL-17A-17F），其中IL-17A和IL-17F在氨基酸序列上相似度高达50%，可以形成异二聚体。IL-17A/IL-17F由Th17、CD8+ T、γδT、NKT、NK、ILC3等细胞产生，IL-17A/IL-17F与疾病的研究目前集中在自身免疫病与炎症性疾病，如银屑病、多发性硬化、炎症性肠病、肿瘤等领域。 比奇珠单抗是一款人源化单克隆IgG1抗体，可选择性抑制IL-17A、IL-17F和IL-17A/F异二聚体……更多资讯，请阅读原文 咨询、合作请联系作者 小D有话说 为方便读者阅读及保存，我们将周报原文整理成了PDF版本，如需获取全文，可点击最上方蓝字，在药渡Daily公众号后台回复“0726创新药周报”，即可下载。