北海康成的新一代基因技术研发中心实验室正在开发针对罕见遗传病,包括庞贝氏症、法布雷病、杜氏 肌肉营养不良症(DMD)和其他神经肌肉疾病的新型及潜在治愈性的基因疗法,并与世界领先的研究人员 和生物技术公司合作。公司的全球合作伙伴包括不限于Apogenix、GCPharma、Mirum、药明生物、 Privus、华盛顿大学医学部和ScriptrGlobal等。 7月3日，截至港股收盘，恒生指数下跌0.63%，报24069.94点。北海康成-B（01228.HK）收报0.365港 元/股，上涨17.74%，成交量728.1万股，成交额241.9万港元，振幅33.87%。 最近一个月来，北海康成-B累计涨幅91.36%，今年来累计涨幅133.08%，跑赢恒生指数20.75%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。 行业估值方面，药品及生物科技行业市盈率（TTM）平均值为3.78倍，行业中值6.5 ...

新京报讯（记者王卡拉）6月18日，1类新药芦沃美替尼片（商品名：复迈宁）在上海、北京、广东、山 东、江苏、湖南等多个省市医院开出首批处方，上海首位患者完成购药。该药是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。 芦沃美替尼片是复星医药自主研发的创新型小分子靶向药物，通过高选择性抑制MEK1/2蛋白活性，阻 断MAPK信号通路的异常激活，从而抑制肿瘤细胞增殖并诱导其凋亡。 1型神经纤维瘤病(NF1)是一种罕见病，是常染色体显性遗传疾病。NF1疾病负担较重，可致疼痛、运动 障碍、毁容，病情严重者甚至可危及生命。NF1呈现出家族遗传性特点，是最常见的神经纤维瘤病类 型，占罕见病神经纤维瘤病(NF)的96%。临床数据显示，约30%-50%的NF1患者经全身影像学检查可发 现患有丛状神经纤维瘤（PN），在儿童期生长迅速，可造成多种并发症，且手术治疗难以完全切除、 复发率高，迫切需要开发相关靶向药物，帮助患者以及家庭摆脱疾病痛苦。 从临床数据表现来看，芦沃美替尼疗效确切，安全性可控，起效快，为临床治疗提供新选择。Ⅱ ...

北海康成的新一代基因技术研发中心实验室正在开发针对罕见遗传病,包括庞贝氏症、法布雷病、杜氏 肌肉营养不良症(DMD)和其他神经肌肉疾病的新型及潜在治愈性的基因疗法,并与世界领先的研究人员 和生物技术公司合作。公司的全球合作伙伴包括不限于Apogenix、GCPharma、Mirum、药明生物、 Privus、华盛顿大学医学部和ScriptrGlobal等。 6月12日，截至港股收盘，恒生指数下跌1.36%，报24035.38点。北海康成-B（01228.HK）收报0.205港 元/股，上涨26.54%，成交量1907.9万股，成交额373.87万港元，振幅37.65%。 最近一个月来，北海康成-B累计涨幅20%，今年来累计涨幅21.8%，跑赢恒生指数21.47%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。 行业估值方面，药品及生物科技行业市盈率（TTM）平均值为4.2倍，行业中值6.01倍。 ...

Amicus Therapeutics (FOLD) FY Conference June 10, 2025 02:00 PM ET Speaker0 Bradley Campbell, the President and CEO of Anicus Therapeutics. Thank you so much for being here. Speaker1 Thank you for having us. Thanks to Goldman for hosting the conference. Nice to see everybody here today. Speaker0 Thank you. And to start off with a big picture question, can you give us a snapshot of your business today and your strategy with regard to commercial execution in the half of the year? Speaker1 Sure. Yes. At Amicus ...

I型神经纤维瘤病是一种常染色体显性遗传病，呈现出家族遗传性的特点，是最常见的神经纤维瘤病类 型，占罕见病神经纤维瘤病(NF)的96%，临床表现多样，影响皮肤、眼部、神经等系统。丛状神经纤维 瘤作为I型神经纤维瘤病的一种表现形式，发病率为30%-50%，在儿童期生长迅速，可造成多种并发 症，且手术治疗难以完全切除、复发率高，迫切需要新的治疗手段。 复星医药方面介绍称，芦沃美替尼片目前还在拓展新的适应证中，其中用于治疗成人Ⅰ型神经纤维瘤病 于中国境内处于Ⅲ期临床试验阶段；用于低级别脑胶质瘤、颅外动静脉畸形、儿童朗格汉斯细胞组织细 胞增生症的治疗于中国境内均处于Ⅱ期临床试验阶段。 作为一款高选择性MEK1/2抑制剂，芦沃美替尼片是通过高选择性抑制MEK1/2蛋白活性，阻断MAPK信 号通路的异常激活，从而抑制肿瘤细胞增殖并诱导其凋亡。 此次芦沃美替尼片获批上市的适应证有两个，除了可用于朗格汉斯细胞组织细胞增生症和组织细胞肿瘤 成人患者治疗外，还可用于2岁及2岁以上伴有症状、无法手术的丛状神经纤维瘤（PN）的I型神经纤维 瘤病（NF1）儿童及青少年患者治疗，后者同样也属于罕见病。 这是国内首款可用于朗格汉斯细胞组织细 ...

国家药监局官网截图 来源：环球网 近日，药明生物合作伙伴北海康成创新罕见病治疗产品注射用维拉苷酶β（商品名：戈芮宁）获国家药 品监督管理局（NMPA）批准上市，用于治疗12岁及以上青少年和成人I型和III型戈谢病患者。这是中 国首个也是目前唯一的本土自主开发的戈谢病酶替代疗法药物，也是目前覆盖适应症最广泛的戈谢病治 疗产品。 戈谢病是一种罕见的遗传性溶酶体贮积症，患者临床表现包括肝脾肿大、贫血、骨痛和神经系统病变， 严重时甚至可能因并发症危及生命。据统计，全球平均每10万人中戈谢病患病人数为0.7~1.75。目前， 戈谢病已被纳入中国《第一批罕见病目录》。重组人源葡萄糖脑苷脂酶替代疗法 (ERT) 作为戈谢病的标 准治疗方案，经过30年的临床应用，在改善患者的主要非神经系统症状以及提高生活质量方面具有显著 疗效。 北海康成创始人、董事长兼首席执行官薛群博士表示，得益于药明生物等合作伙伴的共同努力，戈芮宁 利用平台化技术将大大降低开发成本，让患者用得上、用得起安全、有效的国产酶替代药物。据药明生 物首席执行官陈智胜博士介绍，戈芮宁开发采用了第二代创新技术，提高了酶的活性，并通过公司超高 效连续生物工艺平台WuX ...

Financial Data and Key Metrics Changes - At the end of Q1 2025, the company had over $172 million in cash and short-term investments [23] - A $200 million debt facility was executed with Hercules, providing a cash runway into the second half of 2027 [23] Business Line Data and Key Metrics Changes - The company is focused on a single development program for a rare disease called autoimmune pulmonary alveolar proteinosis (APAP) [2] - The Phase III pivotal clinical trial IMPALA two showed statistically significant improvement in DLCO compared to placebo at week 24, with sustained improvement at week 48 [7][8] - 97% of patients completed the double-blind treatment period, with no withdrawals due to drug-related adverse events [8] Market Data and Key Metrics Changes - The diagnosed prevalence of APAP in the U.S. is estimated at approximately 3,600 patients, with an additional 3,700 likely undiagnosed patients [13][14] - The potential market opportunity in the U.S. is significant, with a total of over 7,000 patients identified [15] Company Strategy and Development Direction - The company aims to establish relationships with pulmonologists and treatment centers to gain visibility into the patient population before the launch of Molbrevi [17] - A U.S. commercial team is being built prior to approval, consisting of 25 to 30 individuals responsible for patient profiling and disease awareness campaigns [22] - The pricing power for Molbrevi is projected between $300,000 and $500,000 per patient per year, aligning with other orphan drug analogues [24] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of Molbrevi, highlighting the lack of approved medicines for APAP in the U.S. and Europe [4][11] - The company anticipates a potential PDUFA date around November if priority review is granted by the FDA [11] - There is a strong interest from U.S. pulmonologists and payers regarding Molbrevi, with 83% of pulmonologists likely to prescribe it [18][19] Other Important Information - The company launched a free blood antibody test called APAP ClearPath to facilitate quicker diagnosis of APAP [20] - The test has been piloted at an interstitial lung disease clinic, aiming to identify undiagnosed APAP patients [21] Q&A Session Summary Question: What is the current status of the regulatory submission for Molbrevi? - The company completed the submission of the BLA to the FDA and is awaiting feedback within a 60-day window [11] Question: How many patients does the company aim to reach by launch? - The company aims to have line of sight to 1,000 known APAP patients by launch, with a goal to confirm the total of 3,600 patients [15][17] Question: What are the expectations regarding payer coverage for Molbrevi? - 87% of payers indicated they intend to provide coverage with simple pre-authorization criteria, recognizing the disease burden of APAP [19]

Financial Data and Key Metrics Changes - In Q1 2025, the company reported net revenue of $20.4 million, with $17.1 million from MyPlifa, $0.1 million from Olpruva, and $2.3 million in net reimbursements from the French EAP for Aramcholamol [29][30] - Operating expenses for Q1 were $22.8 million, a decrease of $0.6 million compared to the same quarter last year [30] - The net loss for Q1 2025 was $3.1 million, compared to a net loss of $16.6 million in the same quarter a year ago [30][31] - Total cash, cash equivalents, and investments as of March 31, 2025, were $68.7 million, a decrease of $6.8 million from December 31, 2024 [30][31] Business Line Data and Key Metrics Changes - MyPlifa's launch has exceeded expectations, with 122 prescription enrollment forms received since launch, indicating that approximately one-third of diagnosed NPC patients in the U.S. have been enrolled [20][21] - Olpruva received a total of 28 prescription enrollment forms since its initial availability, with five forms received in Q1 2025 [12][27] - The company has achieved 38% of covered lives for MyPlifa, which is in line with expectations at this stage of the launch [21][22] Market Data and Key Metrics Changes - The company is focusing on expanding MyPlifa's availability outside the U.S., with plans for a marketing authorization application in Europe in the second half of the year [11] - The European market for NPC is well established, with approximately 1,100 people estimated to be living with the disease [11] Company Strategy and Development Direction - The company is executing on four strategic pillars: commercial excellence, pipeline and innovation, talent and culture, and corporate foundation [5][8] - The focus is on bringing innovative therapies to patients with rare diseases, with a strong emphasis on MyPlifa as a cornerstone treatment for NPC [17][33] - The company is also assessing strategic alternatives for its Phase III ready asset KP1077 for rare sleep disorders [6] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the early success of MyPlifa and the refined marketing strategy for Olpruva, indicating a strong position for future growth [33] - The company believes it is well positioned to support its strategic priorities and execute on its long-term vision, independent from capital markets [31][32] Other Important Information - The company has successfully monetized its Pediatric Rare Disease Priority Review Voucher, adding $148.3 million of non-dilutive capital to its balance sheet [8][31] - The company is committed to patient access and comprehensive patient services through its in-house program, Amplify Assist [27] Q&A Session Summary Question: Details on the number of patients on MyPlifa and reimbursement status - The company received 122 enrollments since launch, with the majority of patients actively on the drug, while some are in the benefits investigation process [36] Question: Reasons for reimbursement denials for MyPlifa - Initial denials are common for rare disease products, primarily due to prior authorization requirements, but the company has been successful in securing coverage through medical exception processes [39][40] Question: Visibility on patient cohorts and enrollment cadence - The company estimates around 600 undiagnosed patients and 300 diagnosed patients, with ongoing efforts to raise awareness and facilitate treatment [47][59] Question: Inventory levels at the end of the quarter - The company is managing inventory closely and maintaining target levels as it supports patient needs during the early launch phase [50] Question: Qualitative metrics on refill rates and patient retention - The majority of patients receiving active drug have continued to refill, reflecting the clinical benefit and durability of MyPlifa [54] Question: Coverage details and impact on patient access - 38% of covered lives have access to MyPlifa, with ongoing efforts to increase this percentage as more plans evaluate the product [63][64]

Zevra Therapeutics (ZVRA) 2025 Conference May 07, 2025 11:30 AM ET Speaker0 We're gonna go ahead and get started. Thanks again everybody for joining us this morning at the Citizens Life Sciences Conference. Really pleased to be joined next by Zevra Therapeutics, CEO Neil McFarlane, CFO LeDuane Clifton. Zevra is a company focused on, developing and commercializing therapies in in the rare disease space. The company has a portfolio now of approved products. The the the one we're focused on most near term is i ...

Spero Therapeutics, Inc. (NASDAQ:SPRO) Q4 2024 Earnings Conference Call March 27, 2025 4:30 PM ET Company Participants Shai Biran - Senior Director Investor Relations Esther Rajavelu - Interim Chief Executive Officer, Chief Financial Officer Tim Keutzer - Chief Operating Officer Conference Call Participants Gavin Clarke-Gartner - Evercore ISI Athena Chin - TD Cowen Operator Good afternoon, and welcome to the Spero Therapeutics Fourth Quarter and Full Year 2024 Earnings Conference Call. At this time, all par ...