Hydrocortisone

IMPAVIDO ® is the first and only FDA-approved oral therapy for visceral, cutaneous, and mucosal leishmaniasis caused by specific Leishmania species Leishmaniasis is a rare but potentially life-threatening parasitic disease that can cause severe skin lesions or systemic infection involving internal organs Exclusive U.S. commercialization rights to IMPAVIDO ® take effect September 26, 2026 DEER PARK, Ill., May 19, 2026 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or “the Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced it has entered into a supply and distribution agreement for the United States commercialization rights to IMPAVIDO ® (miltefosine) from an affiliate of Knight Therapeutics, Inc (“Knight”). IMPAVIDO ® is an orphan drug indicated for the treatment of visceral, cutaneous, and mucosal leishmaniasis caused by specific Leishmania species in adults and adolescents over the age of 12 and weighing more than 30 kilograms. Please see indications and important safety information below. “We are excited to add yet another 2026 product launch to our portfolio. IMPAVIDO ® is a critical, life-saving medication and a strong fit with Eton’s orphan-focused commercial model. We look forward to partnering with Knight to ensure reliable, high-touch access to the medication for patients across the United States and serving this important community,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. Leishmaniasis is a rare, potentially life-threatening parasitic disease caused by Leishmania parasites transmitted through the bite of infected sand flies. The disease can present in several forms, including cutaneous leishmaniasis, which commonly causes skin sores; mucosal leishmaniasis, which can affect the nose, mouth, or throat; and visceral leishmaniasis, the most severe form, which affects internal organs and can be life threatening if untreated. IMPAVIDO ® has been commercially available in the United States since 2016. The transaction further expands Eton’s growing portfolio of orphan therapies serving highly specialized patient populations and reflects the Company’s continued focus on expanding differentiated rare disease commercialization and patient support capabilities across multiple therapeutic areas. Important Safety Information for IMPAVIDO ® Indication for IMPAVIDO® (miltefosine) IMPAVIDO ® capsules contain the active ingredient miltefosine, an antileishmanial agent. IMPAVIDO ® is an antileishmanial drug indicated in adults and pediatric 12 years of age and older weighing greater than or equal to 30 kg (66 lbs) for treatment of: Visceral leishmaniasis due to Leishmania donovani Cutaneous leishmaniasis due to Leishmania braziliensis , Leishmania guyanensis , and Leishmania panamensis Mucosal leishmaniasis due to Leishmania braziliensis Limitations of use: Leishmania species evaluated in clinical trials were based on epidemiologic data. There may be geographic variation in the response of the same Leishmania species to IMPAVIDO ® . The efficacy of IMPAVIDO ® in the treatment of other Leishmania species has not been evaluated. Important Safety Information for IMPAVIDO® (miltefosine): IMPAVIDO ® may cause serious risks to pregnancy: Do not take IMPAVIDO ® if you are pregnant. If you take IMPAVIDO ® during pregnancy, your baby is at risk for death or serious birth defects. If you become pregnant while taking IMPAVIDO ® , stop taking IMPAVIDO ® and talk to your healthcare provider right away if you become pregnant during treatment with IMPAVIDO ® . You should have a pregnancy test before you start taking IMPAVIDO ® . Women who can become pregnant should use effective birth control (contraception) during IMPAVIDO ® treatment and for 5 months after their last dose of IMPAVIDO ® . Talk to your healthcare provider about which birth control method is right for you. Pregnancy Registry: There is a registry for women who become pregnant during treatment with IMPAVIDO ® . If you become pregnant or think you may be pregnant while taking IMPAVIDO ® , tell your healthcare provider right away. Talk to your healthcare provider about registering with the IMPAVIDO ® Pregnancy Registry. The purpose of this registry is to collect information about your health and your baby’s health. Your healthcare provider can enroll you in this registry by calling 1-866-588-5405 Do not take IMPAVIDO ® if you: are pregnant, plan to become pregnant, or become pregnant during treatment with IMPAVIDO ® . have Sjögren-Larsson-Syndrome are allergic to miltefosine or any of the ingredients in IMPAVIDO ® . are a woman who can become pregnant and have not had a pregnancy test. Women who can get pregnant must have a urine or blood pregnancy test before taking IMPAVIDO ® . The most common side effects associated with IMPAVIDO® include nausea, vomiting, diarrhea, stomach pain, decreased appetite, dizziness, headache, sleepiness, skin itching, abnormal liver tests, abnormal kidney tests, motion sickness, fever, tiredness, weakness, and enlarged lymph nodes. Tell your healthcare provider if you have any side effect that bothers you or that does not go away. These are not all the possible side effects of IMPAVIDO ® . For more information, ask your healthcare provider. You may report side effects to FDA at 1-800-FDA-1088. Tell your healthcare provider about all of the medicines you're taking, including prescription and over-the-counter medications, vitamins and herbal supplements. Tell your healthcare provider about all your medical conditions, including if you have or have had eye problems, have kidney or liver problems. Your healthcare provider should do blood tests to check your kidneys and liver before you start, during and after your treatment with IMPAVIDO ® . If you are breastfeeding or plan to breastfeed. It is not known if IMPAVIDO ® passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby if you take IMPAVIDO ® . You should not breastfeed while you take IMPAVIDO ® and for 5 months after your last dose of IMPAVIDO ® . IMPAVIDO® has not been studied in children under 12. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. IMPAVIDO® is available by prescription only. The information on this website should not take the place of talking with your doctor or healthcare professional. If you have any questions about your condition, or if you would like more information about IMPAVIDO®, talk to your doctor or healthcare professional and see the full prescribing information About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has ten commercial rare disease products: KHINDIVI™, INCRELEX ® , ALKINDI SPRINKLE ® , DESMODA™, GALZIN ® , HEMANGEOL ® , PKU GOLIKE ® , Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has four additional product candidates in late-stage development: Amglidia ® , ET-700, ET-800 and ZENEO ® hydrocortisone autoinjector. For more information, please visit our website at . Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Eton to undertake certain activities and accomplish certain goals and objectives. These statements include but are not limited to statements regarding Eton’s business strategy, Eton’s plans to develop and commercialize its product candidates, the safety and efficacy of Eton’s product candidates, Eton’s plans and expected timing with respect to regulatory filings and approvals, and the size and growth potential of the markets for Eton’s product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Eton’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Eton’s development programs and financial position are described in additional detail in Eton’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Eton undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Investor Relations: Lisa M. Wilson, In-Site Communications, Inc. T: 212-452-2793 E: lwilson@insitecony.com

医道社：传承和发展民间中医 来源：海外网站 作者：医道社整理  版权归原作者所有，如有违规、侵权请联系我们删除！ 👆关注不迷路，更多创新药前沿资讯 一个值得所有临床医生和研发人认真看待的信号来了。 2026年5月18日，阿斯利康的 Baxfendy（baxdrostat） 获 FDA 批准，用于成人控制不佳的高血压，且需与其他降压药联合使用。 它不是又一个“me-too 降压药”，而是美国首个获批的醛固酮合成酶抑制剂（aldosterone synthase inhibitor, ASI）。  对很多人来说，这只是又一条审批新闻。 但对临床和研发来说，这可能意味着：高血压治疗在沉寂多年之后，终于再次出现了“机制级创新”。01｜为什么 Baxfendy 的获批，不只是“多了一个降压药”？ 高血压市场很大，大到大家都习以为常；也很“旧”，旧到几十年来真正让人眼前一亮的机制创新并不多。 临床现实是什么？ ·全球约14亿人患有高血压（WHO） ·美国有大量患者已经用了2种甚至3种以上降压药，但血压仍不达标 ·AstraZeneca 在新闻稿中引用的数据指出：美国约有2300万人属于“用了≥2种药仍控制不佳”的人群 ·这类患者面临的不是简单的“数字偏高”，而是卒中、心衰、CKD、心血管死亡风险持续累积 过去这些年，临床上对难治性/控制不佳高血压并不是没有办法。利尿剂、ACEI/ARB、CCB、β受体阻滞剂、MRA（如螺内酯、依普利酮、非奈利酮）都能用。 但问题在于：很多患者的病理驱动因素，并没有被真正“打到源头”。而这个源头之一，就是醛固酮失调。 这正是 Baxfendy 出场的意义。 02｜Baxfendy 真正的技术优势：不是“拦截效果”，而是“掐断源头” 先说结论： Baxfendy 最大的技术价值，不在于它也能降压，而在于它选择了比 MRA 更靠上的靶点：直接抑制醛固酮生成。2.1 MRA 的逻辑：堵受体 传统矿皮质激素受体拮抗剂（MRA）——如螺内酯、依普利酮、非奈利酮——是在下游阻断醛固酮与受体结合。这类药有效，PATHWAY-2 早已证明螺内酯是难治性高血压的重要选择。 但 MRA 的局限也很清楚： ·高钾风险限制使用 ·肾功能差患者更难用 ·螺内酯存在性激素相关不良反应 ·还会出现反射性肾素和醛固酮升高 ·不能完全覆盖醛固酮的非受体介导效应2.2 ASI 的逻辑：断供醛固酮 而 Baxfendy 所属的 ASI 路径，是直接抑制 aldosterone synthase（CYP11B2），也就是醛固酮合成最后关键步骤的酶。 换句话说，它不是在下游“拦人”，而是在上游“断货”。 这带来几个非常重要的技术优势：优势一：机制更靠前，理论上更接近病因干预 对于醛固酮驱动明显的患者——尤其是难治性高血压、原醛谱系异常、肥胖相关高血压、盐敏感性高血压——          直接抑制醛固酮生成，理论上比受体拮抗更彻底。优势二：有望避免传统 MRA 的部分局限 MRA 的一个麻烦在于，它虽然阻断了受体，却可能促使系统性反馈上调，使循环醛固酮更高。而 ASI 是直接压低醛固酮本身，这在生理学上更“顺手”。优势三：靶点可外溢到心肾代谢疾病 醛固酮从来不只是“升血压”这么简单。它还与： ·心肌重构 ·血管纤维化 ·肾小球高压与肾损伤 ·心衰进展 ·原发性醛固酮增多症 ·肥胖、盐敏感和交感兴奋相关通路 密切相关。 这意味着 Baxfendy 的商业和研发价值，可能根本不只在高血压本身，而在“高醛固酮驱动的心肾疾病谱系”。03｜最关键的数据，究竟硬不硬？ 答案是：对一个高血压创新药来说，这组数据是相当有冲击力的。 FDA 此次批准主要基于 BaxHTN Ⅲ期研究（NEJM 2025正式发表）。3.1 BaxHTN 研究设计 ·III期、随机、双盲、安慰剂对照 ·共 794名实际接受治疗患者 ·入组人群： ·未控制高血压：已用2种药仍未达标 ·难治性高血压：已用≥3种药仍未达标 ·所有人背景治疗中都包含利尿剂 ·随机分配至： ·baxdrostat 1 mg ·baxdrostat 2 mg ·placebo ·主要终点：12周坐位收缩压（seated SBP）变化3.2 核心结果 12周时： ·2 mg 组：绝对降幅 -15.7 mmHg ·1 mg 组：绝对降幅 -14.5 mmHg ·安慰剂组：-5.8 mmHg 安慰剂校正后： ·2 mg：-9.8 mmHg ·1 mg：-8.7 mmHg ·均 P<0.0001 这是什么概念？ 很多人看到 9.8 mmHg 可能无感，但做临床的人知道： 在一个已经接受多药背景治疗、而且包含利尿剂的人群中，再额外打出接近 10 mmHg 的 placebo-adjusted SBP 降幅，是非常有分量的。 而且不只是平均值漂亮，研究中还有两个更关键的信号：3.3 难治性亚组同样稳定 在resistant hypertension 亚组中： ·1 mg：安慰剂校正 -9.1 mmHg ·2 mg：安慰剂校正 -9.8 mmHg 这说明它不是只在“稍微控制不好”的人群里有效，而是在真正难治的患者里也有穿透力。3.4 达标率明显提升 达到 SBP <130 mmHg 的比例： ·Placebo：18.7% ·Baxdrostat 1 mg：39.4% ·Baxdrostat 2 mg：40.0% 也就是说，达标率差不多翻倍。 对于临床而言，这比平均降压值更有现实意义。因为医生最终面对的，不是统计学，而是“病人的血压能不能进目标区间”。04｜真正让人震撼的，不只是降压，而是它背后的“研发判断” 如果说数据解决了“它有没有效”，那更值得行业反复咀嚼的是：为什么 Baxdrostat 能成，而很多高血压创新项目常常沉掉？ 我的判断是，Baxfendy 的成功，至少验证了3个研发认知。认知一：高血压不是“低技术含量赛道”，而是被长期低估的机制病理战场 这些年创新药行业常有一种偏见：高血压太成熟、太便宜、太卷，不适合做创新。 这话只说对了一半。 确实，高血压有大量仿制药，支付环境严格，商业化门槛高。但另一半事实是： 高血压并不是“没有创新空间”，而是过去缺少真正能抓住病理核心的人。 RAAS、盐敏感、交感激活、醛固酮逃逸、原醛谱系异常、肥胖相关机制、肾脏钠处理异常…… 这些都不是“老掉牙的问题”，而是今天依旧未被充分解决的真实临床难题。 Baxfendy 的价值就在于，它不是换壳，而是对病理驱动轴重新下注。认知二：高血压创新，未来不再只比“降压幅度”，而是比“能否进入心肾共病主航道” 单看高血压适应症，Baxfendy 已经够亮眼。但真正让阿斯利康重仓它的原因，绝不只是为了卖一个降压药。 从公开信息看，baxdrostat 正在拓展： ·原发性醛固酮增多症 ·CKD 合并高血压 ·心衰预防 ·与 dapagliflozin（达格列净） 联合开发 这背后的逻辑很清楚： 未来最值钱的高血压药，不是只能把血压从150打到140，而是能够切入心肾保护的疾病网络。 这也是 Baxfendy 与传统高血压新药最大的不同。它不是在单病种里找缝隙，而是在CVRM（心血管-肾脏-代谢）一体化疾病图谱里找平台价值。认知三：真正的 first-in-class，不是概念新，而是“临床能落地” 创新药行业很爱讲 first-in-class。但很多 first-in-class 到最后只剩“靶点故事”，落不到真实处方场景。 Baxfendy 这次更难得的一点在于：它面对的不是罕见病小样本，也不是 surrogate endpoint 边缘获批，而是极其主流、极其现实的高血压临床场景。 临床场景非常清晰： ·患者已经在吃药 ·甚至吃了不止一种 ·还包括利尿剂 ·但就是控制不住 ·医生缺一个“机制不同、可叠加、效果明确”的选项 这就让 Baxfendy 的产品定义很明确：不是替代一线，而是切入未满足需求最明确的 add-on 人群。 这对市场准入、指南渗透、临床教育都更友好。05｜但别急着神化：Baxfendy 也有必须正视的短板 如果只写成一篇“审批喜报”，那就太浅了。 任何认真做临床和研发的人，都必须看到 Baxfendy 的另一面。5.1 最大挑战：电解质安全性 这类药物的 on-target 风险非常明确，就是： ·高钾 ·低钠 ·以及一定程度的血压过低、eGFR 下降 在 BaxHTN 中：血钾 >5.0 mmol/L ·placebo：11.3% ·1 mg：23.8% ·2 mg：35.9%血钾 >5.5 mmol/L ·placebo：0.4% ·1 mg：6.1% ·2 mg：11.1%血钾 >6.0 mmol/L ·placebo：0.4% ·1 mg：2.3% ·2 mg：3.0% 这不是“小问题”。 如果放到真实世界，尤其是： ·CKD 患者 ·糖尿病患者 ·老年患者 ·合并 ACEI/ARB、SGLT2、利尿剂多重用药者 监测成本和使用门槛会直接影响渗透率。5.2 eGFR 早期下降也值得高度关注 研究中 12 周 eGFR 平均变化： ·baxdrostat 1 mg：-7.0 ml/min/1.73m² ·baxdrostat 2 mg：-6.9 ml/min/1.73m² ·placebo：-0.1 研究者倾向认为这是血压下降导致的功能性变化，且停药/撤药后可回归。但对于真实世界医生来说，看到肌酐变化，是否愿意继续开？这会极大影响其一线接受度。5.3 它还没有回答最关键的长期问题：心血管结局 今天 Baxfendy 获批，依据是降压终点，而不是 MACE、HF hospitalization、renal outcome。 这意味着它眼下是“有机制创新的降压药”，但要真正成为大品种，必须回答： ·长期用药是否持续有效？ ·是否改善心血管结局？ ·是否对肾脏结局有净获益？ ·与 MRA 比，谁在什么人群更优？ ·与螺内酯、非奈利酮相比，定位如何？ 这决定了它是“创新产品”，还是“重磅平台”。06｜横向看同赛道：Baxfendy 为什么领先于 lorundrostat？ 既然都是 ASI，行业自然会问：为什么 baxdrostat 率先上岸？ 另一位重要玩家是 Mineralys 的 lorundrostat。公开研究显示，lorundrostat 在 uncontrolled / treatment-resistant hypertension 中也表现出可观的降压效果，2025年 NEJM/JAMA 先后有重磅发表，办公室 SBP placebo-adjusted 降幅大致也在 ~9 mmHg 量级。 所以，baxdrostat 的领先并不只是“数据比别人高很多”，而可能是以下几方面的综合结果：6.1 临床开发路径更完整 AstraZeneca 在 CinCor 收购后，对 baxdrostat 做的不只是单点验证，而是形成了： ·BrigHTN ·HALO ·BaxHTN ·Bax24 ·原醛、CKD、高血压联合适应症探索 这种完整开发路径让 FDA、医生、投资人都更容易建立信心。6.2 选择性与“不过度碰 cortisol”是关键壁垒 历史上 ASI 路径并非没人碰，但早期项目常被 CYP11B1 / cortisol 相关问题 拖累。而 baxdrostat 的一大技术卖点，就是高选择性抑制 CYP11B2，在临床研究中未见明显皮质醇抑制信号。 这点非常关键。因为一旦影响 cortisol，整个安全性和长期可用性就会迅速变差。6.3 阿斯利康的商业化整合能力更强 这个现实也不能回避。高血压不是一个光靠论文就能打透的市场，它需要： ·大规模医学教育 ·与心内、肾内、全科联动 ·支付和准入谈判能力 ·在 CVRM 组合中的定位能力 而 AZ 在这一点上，显然远强于多数 biotech。尤其是它已有 Farxiga 在心肾代谢的深厚临床基础，Baxfendy 更容易被纳入同一个学术叙事中。07｜Baxfendy 对中国创新药研发，有哪些真正的启发？ 这部分我想说得更直接一点。 过去国内很多团队做心血管，常常有两个误区：误区一：心血管不性感，做不过肿瘤和自免 错。心血管不是不性感，而是门槛更高、评价更严、临床价值更真实。 肿瘤可以靠 biomarker 和人群切割迅速起量，但高血压、CKD、HF 这种领域，你必须真正理解疾病生理，真正跑出可复制、可监测、可长期使用的数据。 Baxfendy 的成功再次说明：只要靶点抓得准，心血管领域依然可以诞生 first-in-class。误区二：成熟病种没必要做创新 也错。越成熟的病种，越说明存在大规模未满足需求；越是“老病种”，越缺真正改变格局的新机制。 高血压就是典型。患者多、临床路径清楚、结局明确、指南成熟。一旦有真正有效的新机制，反而更容易形成行业级影响。08｜临床上，Baxfendy 最可能先在哪类患者中打开局面？ 从当前证据看，我认为它最有机会先切入以下几类人群：1）已经用了 2–3 种药仍不达标的患者 这是标签最直接覆盖的人群，也是最现实的起量入口。2）疑似“醛固酮驱动型”高血压患者 如： ·低肾素状态 ·肥胖相关高血压 ·盐敏感明显 ·夜间血压高 ·晨峰血压突出 ·对传统 RAAS 抑制反应一般3）不耐受 MRA 的患者 尤其是： ·螺内酯副作用不能接受 ·既往 MRA 使用受限 ·需要寻找不同机制 add-on 的人群4）心肾高风险但尚未进入明确结局适应症的患者 如果未来 CV/renal outcome 数据进一步强化，这一块可能才是 Baxfendy 真正的爆发点。09｜最强烈的观点：Baxfendy 不是高血压创新的终点，而是“醛固酮时代”的起点 我想把这篇文章最核心的判断写得再明确一些： Baxfendy 的真正意义，不只是批准了一个药，而是证明“醛固酮失调”可以被作为高血压创新的核心轴重新定义。 它让行业重新看到： ·高血压依然存在重大的机制创新机会 ·醛固酮不只是 RAAS 的附属变量，而是独立的病理驱动轴 ·上游抑制醛固酮生成，可能比下游拮抗受体更有延展性 ·未来的降压药研发，必须从“数值控制”转向“病理通路控制” ·高血压产品的终极竞争，不在单次审批，而在能否打通 CVRM 全链条 这也是为什么，阿斯利康会把 baxdrostat 放在其 2030 战略版图里。因为它看中的不是一个高血压单品，而是一个可能连接高血压、原醛、CKD、心衰预防的机制平台。10｜写在最后：我们为什么必须重视这次批准？ 因为这次 FDA 批准，至少释放了三个清晰信号：信号一：高血压创新并未终结，只是长期缺少真正的机制突破 Baxfendy 证明，沉寂多年之后，这个赛道依旧能跑出有说服力的 first-in-class。信号二：醛固酮通路正在从“辅助概念”升级为核心研发阵地 未来几年，围绕 ASI、原醛谱系、心肾联动的研究会明显升温。信号三：真正优秀的心血管创新药，必须同时满足三件事 ·机制有新意 ·数据有硬度 ·临床路径可落地 Baxfendy 至少已经完成了前两步，并且第三步也已经看到了轮廓。一句话总结 Baxfendy（baxdrostat）的技术优势，不只是“降压有效”，而是它以首个获批 ASI 的身份，把高血压治疗从“对抗症状”往“切断醛固酮源头”推进了一大步。如果后续心肾结局数据成立，它很可能不只是一个新药，而是下一代 CVRM 平台药物的起点。参考依据（公开资料整理） 1.AstraZeneca / BusinessWire, May 18, 2026: BAXFENDY approved in the US as the first and only aldosterone synthase inhibitor treatment for adults with hypertension. 2.Flack JM, et al. Efficacy and Safety of Baxdrostat in Uncontrolled and Resistant Hypertension. N Engl J Med. 2025;393(14):1363–1374. doi:10.1056/NEJMoa2507109 3.Freeman MW, et al. Phase 2 Trial of Baxdrostat for Treatment-Resistant Hypertension. N Engl J Med. 2023;388:395–405. 4.Saxena M, et al. Lorundrostat in Participants With Uncontrolled Hypertension and Treatment-Resistant Hypertension. JAMA. 2025. 5.Laffin LJ, et al. Lorundrostat Efficacy and Safety in Patients with Uncontrolled Hypertension. N Engl J Med. 2025. 6.WHO. Global report on hypertension 2025. 7.Carey RM, et al. Prevalence of apparent treatment-resistant hypertension in the United States. Hypertension. 2019;73(2):424-431. 8.Williams B, et al. PATHWAY-2 related studies, Lancet / Lancet Diabetes Endocrinol.