中新网北京新闻6月26日电 由复星医药自主研发的1类新药芦沃美替尼片（商品名：复迈宁®）在首都 医科大学附属北京天坛医院正式投入临床使用，标志着复迈宁®作为中国首个且目前唯一同时获批成人 朗格汉斯细胞组织细胞增生症（LCH）及组织细胞肿瘤、2岁及以上儿童青少年 I型神经纤维瘤病 （NF1）双适应症的靶向药物惠及当地患者，为相关领域肿瘤罕见病患者带来高品质的治疗新选择。 北京天坛医院投入临床使用，专家寄语新药价值 国产创新药芦沃美替尼片与5月29日正式获得国家药品监督管理局（NMPA）批准上市。获批不到一个 月在北京等多地投入临床使用，破除了罕见病从"无药可医"和"有药难及"的双重困境，切实提升了在罕 见病患者中的药物可及性，填补了国内相关领域的治疗空白，具有里程碑意义。 首都医科大学附属北京天坛医院肿瘤综合治疗中心主任李文斌表示，该药在北京投入临床使用，对于首 都的NF1相关丛状神经纤维瘤患者群体而言，是一个重要的好消息。这种疾病往往给患者，尤其是儿童 青少年，带来沉重的身心负担，手术干预存在局限。芦沃美替尼片作为国内首个获批用于此适应症的靶 向药物，其扎实的临床数据展现了良好的疗效和可控的安全性，为无法手术 ...

今年年初至今，创新药一跃成为港市涨幅最大的板块，超过30家创新药企股价实现翻倍，而北海康成-B(01228)以半个月2.5倍的股价涨幅名列前茅。 乘市场东风，流动性回暖 6月以来，北海康成在二级市场最大的变化除了高涨的股价外，便是显著放大的成交量，而这与今年港市整体流动性回暖息息相关。 据智通财经APP了解，今年以来，港股市场无论是从表现还是成交活跃度方面都明显提升，Wind数据显示，截止今年5月，恒生与恒生科技年内涨幅均超过 了15%，在全球主要指数中位列前茅。成交活跃度上，截至5月，年初以来港股主板日均成交额近2500亿港元，明显高于2024年全年日均1318亿和2023年日 均1049亿港元。在此背景下，一些港股所独有的行业结构如新消费、互联网、创新药在结构性行情下更是凸显优势。 智通财经APP观察到，6月以来，北海康成股价出现"旱地拔葱"式拉升，在6月的前11个交易日内，公司股价有5个交易日的单日涨幅超过15%，其中6月12日 和13日，北海康成股价分别大幅收涨26.54%和58.54%。得益于此，北海康成在今年6月月度股价最高涨幅达到252.94%。 北海康成之所以能在今年6月掀起一波大涨行情，离不 ...

| | 北海康成-B[01228] 2025-06-16 16:00 | | | | | 5PMA = | 10PMA = 20PMA = 30PMA = | | --- | --- | --- | --- | --- | --- | --- | --- | | 0.519 | | | | | | | | | 0.465 | | | | | | | | | 0.410 | | | | | | | | | 0.356 | | | | | | | | | 0.301 | | | | | | | | | 0.247 | | | | | | | | | 0.193 | | | | | | | | | 0.138 | | | | | | | | | 0.084 | | | | | | | | | 6296万 | 成交量:52063190 | | | | | | | | 4722万 | | | | | | | | | 3148万 | | | | | | | | | 1574万 | | | | | | | | | | 03-21 | 04-02 | 04-15 | 04-29 | 05-13 | 05-23 | 06-04 ...

"我们不是在乞求药物，而是在等待一个合理的解决方案。"在与罕见病患者沟通时，这样的声音反复响 起。这也是由于，这个世界上存在一些疾病，它们不仅鲜为人知，甚至连大多数普通人都未曾耳闻，即 便是医生，一生中也难得遇到几例，它们被统称为"罕见病"。 由于罕见病患者数量稀少，导致治疗药物研发成本难以分摊，这可能是罕见病治疗药物年费用高昂，相 当于普通家庭数十年收入的原因。根据最新数据，我国约有2000万罕见病患者，其中只有5%的患者能 够获得有效治疗。尽管近年来快速审评审批通道的建立使得更多罕见病药物得以在我国上市，然而，高 昂的研发成本、漫长的研发周期以及患者人数的稀少，共同导致了这些特效药对患者个体而言仍然价格 昂贵。 与此同时，尽管我国在罕见病用药保障方面取得了一定进展，例如将部分罕见病药物纳入医保目录，但 由于保障体系尚未完善，部分高值罕见病药物的医保报销仍面临挑战。在家庭支付能力有限的情况下， 患者可能面临无法承担高昂治疗费用的困境。 但，这丝毫不影响本土药企投入这一赛道的研发热情。眼下，中国头部创新药企正在推动罕见病药物研 发从"跟随式创新"向"全球领跑"跃迁，填补空白的新药背后，推动实现政策、资本与技术 ...

Summary of Acadia Pharmaceuticals (ACAD) FY Conference Call - June 09, 2025 Company Overview - **Company**: Acadia Pharmaceuticals (ACAD) - **Industry**: Biotechnology, specifically focusing on neurology and rare diseases Key Points and Arguments Management Changes and Strategic Priorities - Katherine Owen Adams has been with Acadia for eight months, implementing changes in the commercial structure, including the appointment of Tom Garner as CCO and Alison McMillan as head of the debut franchise [2][3] - Focus on accelerating clinical trial programs, with notable progress in the Prada Woolley trial, which is expected to complete earlier than anticipated [4] - Emphasis on both organic and inorganic growth strategies, including a deal signed with San Arena in December [5] Intellectual Property and Market Exclusivity - Acadia won a recent appeal on its composition of matter patent, extending exclusivity for NUPLAZID until October 2030, with total exclusivity lasting until February 2038 [8] - The company is prepared to compete with any potential generic formulations that may enter the market [10] Sales and Marketing Strategies - A new strategy to raise awareness of Parkinson's disease symptoms has been successful, leading to increased patient inquiries and the highest number of new prescriptions (NBRxs) since 2020 [14][15] - Direct-to-consumer campaigns have also contributed to patient growth, with a current market share of 20% for NUPLAZID [16] Patient Retention and Compliance - Over 50% of patients remain on NUPLAZID therapy after one year, with a reported 35% discontinuation rate in Q1, showing improvement in patient retention [30][32] - Management strategies have been enhanced to address gastrointestinal side effects, leading to better patient compliance [33] Pipeline Developments - ACP 204, a new five HT2A inverse agonist, is being developed to address limitations of NUPLAZID, with ongoing studies in Alzheimer's disease psychosis and Lewy body dementia psychosis [22][24][55] - Upcoming phase two data for ACP 204 is expected mid-next year, with a focus on efficacy and safety in a complex patient population [46][51] Regulatory and Geographic Expansion - Acadia has submitted for regulatory approval of debut in the EU, with potential approval expected in Q1 next year [34] - Plans for a phase III study in Japan are underway, indicating a strategic focus on international markets [35] Financial Position and Business Development Strategy - The company has a strong financial position with over $680 million in cash, allowing for continued investment in business development and pipeline funding [60][61] - Acadia aims to expand its focus from neurology to other rare diseases, leveraging management's experience in rare disease markets [59] Upcoming Events - An R&D Day is scheduled to showcase early data from pipeline molecules, indicating a commitment to transparency and stakeholder engagement [57] Additional Important Insights - The company is optimistic about the future growth of NUPLAZID, citing potential for increased market penetration and awareness among healthcare providers and caregivers [17][18] - The unmet need in the Alzheimer's and Lewy body dementia populations is significant, with no approved therapies currently available, highlighting the potential impact of Acadia's pipeline [53][54]

6月2日，截至港股收盘，恒生指数下跌0.57%，报23157.97点。北海康成-B（01228.HK）收报0.162港 元/股，上涨19.12%，成交量1651.3万股，成交额257.89万港元，振幅21.32%。 最近一个月来，北海康成-B累计跌幅8.72%，今年来累计涨幅2.26%，跑输恒生指数16.1%的涨幅。 财务数据显示，截至2024年12月31日，北海康成-B实现营业总收入8510.3万元，同比减少17.27%；归母 净利润-4.43亿元，同比减少16.84%；毛利率63.81%，资产负债率506.19%。 机构评级方面，目前暂无机构对该股做出投资评级建议。 （以上内容为金融界基于公开消息，由程序或算法智能生成，不作为投资建议或交易依据。） 本文源自：金融界 作者：行情君 行业估值方面，药品及生物科技行业市盈率（TTM）平均值为5.92倍，行业中值5.86倍。北海康成-B市 盈率-0.12倍，行业排名第153位；其他精优药业（00858.HK）为0.72倍、金斯瑞生物科技（01548.HK） 为1.21倍、东瑞制药（02348.HK）为3倍、正大企业国际（03839.HK）为3.24倍、大健康国际 ...

在全球资本市场，"机构评级"始终是衡量企业价值的重要标尺，这在研发周期长、专业门槛高的生物医 药领域尤为显著。专业投资机构的深度研判往往能提前捕捉到具有突破潜力的创新企业。 近一个月来，深耕内分泌治疗领域的维昇药业持续在资本市场引发关注，先后获得了Morgan Stanley、 Jefferies，以及中信证券三家机构"增持"及"买入"评级。 | 研报发布时间 | 投行机构 | 语学级 | 目标价 | | --- | --- | --- | --- | | 2025年4月28日 | Morgan Stanley | 増持 | 80 港元 | | 2025年5月2日 | Jefferies | 买入 | 82 港元 | | 2025年5月20日 | 中信证券 | 买人 | 82 港酒 開 | （资料来源： Morgan Stanley 、 Jefferies 、中信证券研报） 在当前医药政策暖风与市场迎来估值修复的关键节点，三家顶级投行集体看好维昇药业的现象值得深 究。这家2018年成立的创新药企，如何凭借聚焦内分泌赛道的差异化战略，在跨国药企长期垄断的细分 领域打开突破口？ 从三家机构给出的目标价来看，当前维 ...

国内戈谢病患者迎来国产疗法。 5月16日，北海康成制药有限公司（以下简称"北海康成"）宣布，其全资子公司——北海康成（上海） 生物科技有限公司为持有人的注射用维拉苷酶β（商品名：戈芮宁）获国家药品监督管理局批准上市。 戈芮宁是国内首个本土自主研发适用于12岁及以上青少年和成人Ⅰ型和Ⅲ型戈谢病患者的长期酶替代治 疗，可完全替代同类进口产品。 北海康成创始人、董事长兼首席执行官薛群接受《每日经济新闻》记者采访时表示，相较高昂的进口药 物，戈芮宁可大幅降低患者用药成本。 值得注意的是，据统计，国内戈谢病患者仅约3000人。如何平衡社会意义与企业盈利，是罕见病研发企 业面对的一大难题。为解决这一问题，戈芮宁的CRDMO（合同研究、开发和生产组织）服务公司药明 生物从源头控制罕见病药物的开发成本和生产成本。 药明生物首席执行官陈智胜表示，"在中国开发罕见病药物，一定要通过技术手段，更好地控制成本。 让更多的罕见病药物在比进口产品定价低不少的情况下，仍然具有一定的盈利空间"。 又一罕见病国产疗法获批上市有望大幅降低患者年治疗费用 据悉，戈谢病是最常见的溶酶体贮积症之一，为位于1q22号染色体长臂上的葡萄糖脑苷脂酶基因突变 ...

Summary of PTC Therapeutics Conference Call Company Overview - **Company**: PTC Therapeutics (PTCT) - **Industry**: Biotechnology, focusing on rare diseases and gene therapies Key Points and Arguments Executive Order and Market Position - The recent executive order has unclear ramifications for companies, but PTC Therapeutics expects minimal impact due to its revenue being more international than domestic [4][5] - The company is preparing for the launch of its PKU product, emphasizing a global pricing corridor to maintain product value [5][6] FDA Interactions - PTC has three applications pending with the FDA, with no significant impact from recent agency changes noted [10][12] - The PKU New Drug Application (NDA) is progressing well, with confidence in approval by the PDUFA date [11][12] Translarna Sales in Europe - Despite a negative opinion from the CHMP, PTC is pursuing a country-by-country strategy to commercialize Translarna, with about 50% of countries showing interest [16][17] - The company anticipates achieving 25-30% of European revenue for the year from Translarna [18][20] PKU Launch Strategy - PTC is ready for a global launch of its PKU product, with an early access program initiated in Germany [33][34] - The company has built a robust infrastructure to support the launch, including a patient services team to assist with prior authorizations and patient engagement [38][39] Competitive Landscape for PKU - There are approximately 17,000 individuals with PKU in the US, with a significant unmet need as 85-90% are not on current therapies [44][45] - PTC's PKU therapy is positioned as a better option compared to existing treatments, with a focus on diet liberalization and improved tolerability [47][50] Pricing Strategy - PTC plans to price its PKU therapy at a premium to Palynziq, justified by strong efficacy data and a favorable safety profile [54][55] - The company believes it could achieve a billion-dollar opportunity in the US alone with low double-digit market penetration [57][58] PTC 518 and Huntington's Disease - PTC 518 is positioned as a potential first-ever disease-modifying therapy for Huntington's disease, with promising early data [63][64] - The company is focused on understanding the optimal patient population for this therapy, emphasizing the importance of early-stage patients [66][68] Competitive Landscape for Huntington's Disease - PTC believes that its oral therapy will be more attractive than gene therapies due to its safety, tolerability, and ability to be administered early in the disease process [78][79] - The partnership with Novartis is expected to enhance commercialization efforts for PTC 518 [76][79] Additional Important Content - The company is optimistic about the global opportunity for its therapies, with plans for launches in Japan and other markets [59][61] - PTC is committed to addressing unmet needs in rare diseases, which is a key focus for its product development strategy [32][33]

Mirum Pharmaceuticals Inc (MIRM) Q1 2025 Earnings Call May 07, 2025 04:30 PM ET Company Participants Andrew McKibben - Vice President of Investor Relations and FinanceChris Peetz - President and Chief Executive OfficerPeter Radovich - COO & PresidentEric Bjerkholt - Chief Financial OfficerSelena Zhang - Global capital marketsRyan Mcelroy - Equity Research AssociateRyan Deschner - Vice President - Equity Research Conference Call Participants Gavin Clark-Gartner - AnalystJessica Fye - Managing Director & Equi ...