2025年落幕，FDA旗下药物评价与研究中心（CDER）已批准44款新药，其中超过半数为"first-in-class"药物。这一数据不仅彰显了全球新药研发的创新能 力，更揭示出治疗范式正在从"改良"走向"颠覆"。小分子药物依然占据主流，但多肽、核酸疗法、ADC等新型治疗模式正加速进入临床，靶点创新、机制 突破成为年度关键词。 | 药物名 | 开发机构 | 治疗疾病 | | | --- | --- | --- | --- | | Voyxact (sibeprenlimab) | 大冢制药 | 成人原发性免疫球蛋白 A 肾病 | 靶向细胞因子 A 增殖诱导配体(APRIL)的创新疗 | | Redemplo (plozasiran) | Arrowhead Pharmaceuticals | 家族性乳糜微粒血症综合征(FCS) | 持久降低载脂蛋白 C-III 蛋白表达的 siRNA 药物 | | Kygevi (doxecitine 和 doxribitime ) | 优时比 | 胸苷激酶 2 缺乏症(TK2d) | 通过提供 DNA 合成原料恢复线粒体 DNA 水平 | | Jascayd ( neran ...

Industry Overview - The drug and biotech sector regained momentum in the latter part of 2025 after a weak first half, setting the stage for a strong year for select stocks [1] - Improved policy clarity following drug pricing agreements with the Trump administration reduced uncertainty, while a rebound in mergers and acquisitions revived investor appetite for risk [1] - Innovation accelerated across high-growth areas such as obesity, gene therapy, inflammation, and neuroscience, with the FDA approving 43 novel therapies as of December 22, 2025 [2] Company Highlights Ionis Pharmaceuticals - Ionis Pharmaceuticals' shares surged 127.6% in 2025, significantly outperforming the industry growth of 5% [5][6] - The company advanced its wholly-owned portfolio, highlighted by the FDA approval of Tryngolza for familial chylomicronemia syndrome, generating $57.4 million in sales in the first nine months of 2025 [9] - Ionis is evaluating Tryngolza in late-stage studies for severe hypertriglyceridemia, with plans to file for FDA label expansion soon [10] - The FDA approved Ionis' second drug, Dawnzera, for hereditary angioedema, with a regulatory filing in the EU expected in early 2026 [11] Structure Therapeutics - Structure Therapeutics' shares surged 127.2% in the past year, compared to the industry's 15.6% growth [13] - The stock rally was driven by positive data from the ACCESS clinical program for aleniglipron, showing up to 15.3% placebo-adjusted weight loss [15][16] - The company plans to initiate phase III development for aleniglipron in mid-2026 and is also developing other candidates for obesity treatment [18] Monopar Therapeutics - Monopar Therapeutics' shares rallied 185.9% in the past year, significantly outperforming the industry [20] - The stock price increase was fueled by positive expectations regarding its lead candidate ALXN-1840 for Wilson disease, following a licensing agreement with AstraZeneca [22] - Monopar plans to submit a regulatory filing with the FDA in early 2026 based on favorable long-term data for ALXN-1840 [26] Kodiak Sciences - Kodiak Sciences' shares rallied 181.1% in the past year, contrasting with the industry's 15.7% decline [27] - The stock's performance reflects growing investor confidence in its late-stage pipeline assets, particularly tarcocimab and KSI-501 [29] - Kodiak plans to submit a regulatory filing for tarcocimab for multiple indications, with top-line data from pivotal studies expected in 2026 [31][33]

Core Insights - Pharvaris N.V. announced topline data from the RAPIDe-3 Phase 3 trial of deucrictibant for treating hereditary angioedema (HAE) attacks, indicating positive results for the on-demand treatment [1][3] Study Results - The RAPIDe-3 trial enrolled 134 participants aged 12 years and older, evaluating the oral deucrictibant immediate-release (IR) capsule for HAE attacks [2] - Deucrictibant showed a faster median time to treatment response compared to placebo, with shorter median times for substantial symptom relief and complete symptom resolution [3] - The data indicated fewer attacks required a second dose or rescue medication within 12 hours [4] - 83.0% of attacks were treated with a single capsule of deucrictibant IR, and 93.2% of treated attacks did not require rescue medication [9] Safety and Tolerability - Deucrictibant was well tolerated, with no serious treatment-related adverse events reported, and no participants discontinued treatment due to adverse events [5] Future Plans - The data from the RAPIDe-3 study will support marketing authorization applications planned for submission in the first half of 2026 [3] Competitive Landscape - The FDA approved Ionis Pharmaceuticals' Dawnzera for HAE prophylaxis, which significantly reduced the monthly attack rate by 81% compared to placebo over 24 weeks [6] - KalVista Pharmaceuticals' Ekterly was also approved for acute HAE attacks, being the first oral on-demand treatment for HAE [7] Market Reaction - Pharvaris shares increased by 12.32% to $27.09 following the announcement of the trial results [7]

On Friday, Takeda Pharmaceutical Co Ltd's TAK stock wobbled after touching as low as $14.53 during the premarket session, before bouncing back to about $15.47 at last check Friday.On Thursday, the U.S. Food and Drug Administration (FDA) approved Ionis Pharmaceuticals, Inc.'s IONS Dawnzera (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older.Investors should note that Takeda also has a hereditary angioedema prevention drug, ...