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美国的有钱人看上了中国的医药生意，赌了一把，赚了700个亿。这可不是个小数目哦！ 一个美国人通过卖一种中国的药赚了700亿。 10月6日，纳斯达克上市公司Summit Therapeutics Inc.（SMMT）的市值达到约140亿美元，两年前还只有数亿美元。 重大变化的转折点出现在2022年12月。 SMMT的负责人罗伯特·杜根与中国康方生物达成协议，获得了药物依沃西（AK112）在主要西方国家的销售权。 罗伯特·杜根 9月中旬，依沃西的临床研究结果出来，显示它比全球知名的K药效果更好。这个不怎么出名的SMMT因此受到追捧，年初至今，它的涨幅一度超过10倍。 杜根本人持有SMMT大约78%的股份，按照最新的市场估价，价值约110亿美元，扣除投入成本后，账面增值超过100亿美元，相当于人民币大约700亿元。 相信中国创新的力量，大胆投资，这位美国企业家赚了个盆满钵满。 1 财富迅速增加 杜根财富在9月16日达到了一个阶段性高峰。 那天，SMMT的股票价格猛冲到了33.89美元，杜根的持股财富，差点就到了200亿美元的关口。 事情是因为康方公布了一项研究数据才爆发的。 其研发的PD-1/VEGF双特异性抗体新药依沃西单抗，在一项III期临床研究中，直接与K药（帕博利珠单抗）进行“头对头”对比，针对的是局部晚期或转移性非小细胞肺癌患者，这些患者的一线治疗中PD-L1表达为阳性。 依沃西的效果更好。 这个新疗法让疾病进展或死亡的风险减少了大约一半。在试验中，目标患者的客观缓解率达到了50.0%，而对照组为38.5%。疾病控制率也从89.9%提升到了70.5%。此外，严重的不良反应事件也减少了。 默沙东的明星产品K药，2023年是全球销售额最高的药物，全年销售额达到了251亿美元，约合人民币1800亿元。 结果一出来，依沃西自然水涨船高。 在最近这短短7天里，康方生物这个项目涨势惊人，最高涨幅竟然达到了50%！而杜根持有的SMMT股权，涨势更是迅猛，最高涨幅达到了约176%！真是让人惊叹不已的市场表现！ 为什么SMMT的涨幅更大？ 在2022年12月，杜根和康方达成了一项协议，SMMT买断了其主要发达市场的权益。 简单来说，SMMT赚的钱，可能会比康方多很多，从这个新药里。 依沃西的势头越来越好，早就引起市场的注意了。 5月30日，股价突然从2.85元飙升至11.25元。初步的头对头研究显示，这项成果具有统计学上的显著意义，并且在临床应用中带来了重大益处。 从那个时间点算起，大约过了四个月，SMMT的股价猛涨了约十一倍，这使得杜根所持有的股份价值飞速攀升到了一百亿美金。 在22个月以前，当SMMT还没有与康方公司签下交易之前，它的股价最低跌到了0.66美元这个位置，整个公司的价值还不到5亿美元呢。 哇，杜根的股票就像变魔术一样，从投入的10亿美金，变出了110亿美元的财富！这简直就像是用魔法棒轻轻一挥，财富就翻了十倍！ 重注对赌，这句术语在商业投资中挺常见的，其实就是一种赌注较大的赌局。打个比方，就像你和朋友玩扑克，但这次的赌注不是小数目，而是大笔的钱。在商业上，这意味着公司或者投资者拿出了相当多的资金去押宝某个项目或决策，赌它能成功。如果赌赢了，回报可能非常丰厚；但如果输了，损失也会很惨重。这种情况下，决策的精准度和风险评估就显得尤为重要了。 杜根的成功，是因为他找到了一个好伙伴。 2022年12月初，他与康方生物的CEO夏瑜达成了一项交易。他以50亿美元的出价和销售净额低双位数的比例提成，获得了依沃西在北美、欧洲及日本等地区的独家销售权。 接着，杜根飞快地掏出了5亿美元的预付款，这部分钱里头，4.749亿美元是现金，还有价值2510万美元的1000万股股票。另外，他还让夏瑜在公司里多了一个董事的位子。 康方生物达成交易时，SMMT家底非常薄。到当年9月，账上只有1.22亿美元的现金，净资产大概1亿。 幸运的是，杜根本人是个非常富有的人。那一年，福布斯估计他的财富大约有20亿美元。 作为公司的主要股东兼CEO，他向SMMT提供了5亿美元的现金，基本解决了预付款的问题，并将持股比例提高到了78.2%。 杜根选在了最合适的时候出手。 那时，夏瑜团队正在和K药进行“头对头”的研究，这项研究很有商业价值。但是，康方生物自身的财力不够，无法支持后续的研究支出。 2022年上半年，康方的营收是1.63亿元人民币，但亏损了6.3亿元。他们的现金储备只有22亿元。同年11月，康方刚从广州高新区集团那里融资，只有5亿元资金，资金非常紧张。 康方生物，一个在生物医药领域里深耕的公司，致力于创新药物的研发，以期为全球患者提供更有效的治疗方案。 同时，于康方来说，也需要找到商业合作的伙伴。 在这种情况下，杜根提供的条件符合康方当时的需求，他的生物制药传奇经历也是一个很好的背书。 在生物制药领域，有很多不确定的事情。对于杜根和SMMT来说，拿出5亿美元是一场豪赌。如果依沃西表现不好，他最多可能损失1/4的家当。 万幸，大家都很开心。2023年春天，夏瑜团队收到预付款后，得以将依沃西顺利推进到全球3期临床。 根据花旗的分析，依沃西在北美和欧洲的年销售高峰预计能达到78亿美元。如果把其他市场也考虑进去，这个数字完全有可能突破100亿美元大关。 康方生物现在还有两款药物——卡度尼利AK104和莱法利AK117，看起来很有商业前景。 有人认为，当依沃西取得了巨大成功后，杜根仍然有机会，可以与夏瑜团队合作，共同研发这两款药物。 三 医药行业的发财机会 杜根的眼光非常独到，成功预测了康方和依沃西的潜力，并非完全靠运气。 他1954年出生，一开始什么都没有，但聪明能干。在大学里，他就开始投资股票，赚了点钱。后来，他开了缝纫工具店和连锁饼干公司，还搞了个计算机网络公司，赚了数千万美元。 在上世纪九零年代，他踏足了医疗健康领域的大门，当时他投资了一家叫做Computer Motion的公司，专门做手术机器人的。就像是在电脑和手术之间架起了一座桥梁。到了2003年，他把这家公司的掌控权以6800万美元的价格交给了后来更出名的达芬奇手术机器人公司。 达芬奇手术机器人，这可是现代医学界的一大神器！它就像是医生的高科技助手，能够进行极其精细的手术操作。和传统的手术刀不同，达芬奇机器人通过高精度的机械臂和先进的3D视图系统，让医生能够在极小的切口处完成复杂的手术，减少了对患者的创伤，加速了恢复过程。这玩意儿，简直就是给医生和患者带来福音的科技奇迹！ 杜根进军生物制药领域，是因为他有一个儿子，得了脑瘤，非常痛苦地离开了。 "我比大多数人在脑肿瘤这事儿上懂的多，因为得想办法帮我家孩子治病。" 杜根讲述，他决定把钱投到一家名叫Pharmacyclis的公司，这是一家在搞新药的家伙。他们手头有一个药，叫莫特沙芬钆（Xcytrin），这药专门对付脑癌。 当Xcytrin在向美国食品药品监督管理局（FDA）申请上市的道路上屡次受挫，其他投资者对它失去信心的时候，杜根出手了。他大量购买了Xcytrin的股权，用自己的钱为公司筹措了5000万美元。他的目标是坚持到底，希望能等到Xcytrin成功的一天。然而，最终，Xcytrin还是没有等到它应有的成功。 结果没想到，2006年，他们以660万美元引进了3款在研药。 在这些药物中，伊布替尼（Imbruvica）的表现格外引人注目。它不仅对慢性淋巴细胞性白血病有很好的治疗效果，还能显著改善罕见疾病套细胞淋巴瘤的病情。 杜根憋着一股劲，按照专家团的提议，把精力和资源都投到了潜力更大的伊布替尼项目上。他在2011年和强生签了个合作，拿到了1.5亿美元的启动款，这下子药物研究有资金了。聪明的是，他还保留了在美国市场的控制权。 接着，伊布替尼这药就上市了，它在最火的时候，全球一年能赚到的钱，接近100亿美金。 2015年，杜根主导了一次谈判，以210亿美元的高价将Pharmacyclics出售，成功退出市场。

TEL AVIV, Israel--(BUSINESS WIRE)--Azura Ophthalmics Ltd., a clinical-stage biopharmaceutical company developing AZR-MD-001, an investigational, potentially first-in-class, Ophthalmic Keratolytic targeting Meibomian Gland Dysfunction (MGD), today announced that it has received positive feedback from a pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) confirming that the company’s existing clinical development program for AZR-MD-001 is sufficient to support its planned NDA submission. “We are very encouraged by the FDA’s feedback and pleased by their determination that our existing clinical data package satisfies the requirements for a sign and symptom in two adequate and well-controlled confirmatory efficacy studies,” said Marc Gleeson, CEO of Azura Ophthalmics. “We believe AZR-MD-001 has the potential to redefine how patients with Meibomian Gland Dysfunction and Dry Eye Disease are treated by directly targeting gland function with a novel, easy-to-administer, twice-weekly ophthalmic ointment. As a company, we are focused on submitting our NDA package in the second half of 2026.” The FDA confirmed the CELESTIAL and ARIES study data fulfill the regulatory efficacy requirements for approvability of AZR-MD-001 for the treatment of the signs and symptoms of Dry Eye Disease (DED). The results from these two studies demonstrate statistically significant improvement in a sign, Meibomian Glands Yielding Liquid Secretion (MGYLS), and symptomatic benefit in Ocular Surface Disease Index (OSDI©) in CELESTIAL and increased comfortable contact lens wear time in ARIES. The FDA indicated that the safety database across CELESTIAL, ARIES, and ASTRO is sufficient for submission, with long-term safety data from the ongoing ASTRO study expected to be shared in Q1 2026, further supporting the application. The company believes that AZR-MD-001’s robust product profile, including its unique triple mechanism of action, keratolytic, keratostatic, and lipogenic, supports its differentiation from existing DED therapies. AZR-MD-001 is administered directly to the lower eyelid margin twice weekly at bedtime. It is designed to restore meibomian gland function, improve tear stability, and reduce symptoms associated with DED, including Contact Lens Discomfort (CLD). “We continue to see growing recognition among physicians that AZR-MD-001 addresses meibomian gland obstruction, an important and previously untreated root cause of Dry Eye Disease,” said Dr. Francis Mah, Director of Cornea and External Disease and Co-Director, Refractive Surgery at Scripps Clinic Medical Group, La Jolla, Calif. “A product that can open and restore gland function with a convenient, twice-weekly dose could be practice-changing for millions of patients.” AZR-MD-001 is an investigational drug candidate and has not been approved by the U.S. FDA. To date, over 500 patients have been treated with AZR-MD-001 in clinical trials, and the product appears to be safe and well-tolerated. About Meibomian Gland Dysfunction (MGD) Meibomian Gland Dysfunction (MGD) is a chronic and progressive condition that affects the quality and flow of meibum from the meibomian glands. MGD is a leading cause of Dry Eye Disease (DED). Without treatment, MGD may lead to glandular dropout and increased ocular surface damage, which reduces quality of vision and decreases quality of life. Current therapies for DED primarily focus on inflammation or tear production and do not address the underlying glandular dysfunction that characterizes MGD. About CELESTIAL1,2 CELESTIAL, a multicenter, vehicle-controlled, randomized study to evaluate the safety, tolerability, systemic pharmacokinetics, and pharmacodynamics of AZR-MD-001 in patients with Meibomian Gland Dysfunction (MGD) and evaporative Dry Eye Disease (DED) was a parallel group study of AZR-MD-001 (0.5% and 1.0%) and vehicle in a 1:1:1 ratio, dosed twice-weekly in the evening (at bedtime). Patients were followed for a total of 6 months. The pre-specified primary efficacy endpoints were Meibomian Glands Yielding Liquid Secretion (MGYLS) and total Ocular Surface Disease Index (OSDI©) change from baseline to Month 3. About ARIES3 ARIES was a multi-center, vehicle-controlled study to evaluate the safety, tolerability, and pharmacodynamics of AZR-MD-001 and to determine common symptoms in patients with Dry Eye Disease (DED) characterized by signs of Meibomian Gland Dysfunction (MGD) and an inability to wear their contact lenses as desired. Patients were treated with either AZR-MD-001 0.5% and vehicle in a 1:1 ratio, dosed twice-weekly in the evening (at bedtime). Patients with DED were challenged with Contact Lens Wear and were followed for a total of 3 months. The primary efficacy sign endpoint was Meibomian Glands Yielding Liquid Secretion (MGYLS), and the symptom endpoint was Comfortable Contact Lens Wear Time in hours. About ASTRO ASTRO, a phase 3, multicenter, vehicle-controlled, randomized study to evaluate the efficacy, safety, and tolerability of AZR-MD-001 in patients with abnormal meibomian gland function and associated symptoms of Dry Eye Disease (DED), is an ongoing parallel group study of AZR-MD-001 0.5% and vehicle randomized in a 1:1 ratio. The study drug is dosed twice-weekly to the lower eyelid in the evening (at bedtime). The study objective supporting the New Drug Application (NDA) is to evaluate the safety and tolerability of AZR-MD-001 sterile ophthalmic ointment 0.5% or vehicle dosed twice-weekly through Month 12. About AZR-MD-001 AZR-MD-001 is the first and only Ophthalmic Keratolytic investigational therapy in development as a foundational treatment for Meibomian Gland Dysfunction (MGD), a leading cause of Dry Eye Disease (DED). Delivered as a twice-weekly selenium sulfide ointment applied to the lower eyelid margin, AZR-MD-001 is designed to restore meibomian gland function by improving the quality and quantity of meibum, rejuvenating the lid margins, and alleviating evaporative dry eye symptoms. The therapy’s unique triple mechanism of action, keratolytic, keratostatic, and lipogenic, targets the root cause of MGD. About Azura Ophthalmics Azura Ophthalmics (“Azura”) is utilizing its deep understanding of ocular surface diseases and drug development to deliver a potential new therapeutic class of Ophthalmic Keratolytics to treat underserved ophthalmic conditions. The company’s differentiated approach combines ophthalmologic and dermatologic science to harness the unique properties of keratolytics for the treatment of Dry Eye Disease (DED) by targeting Meibomian Gland Dysfunction (MGD) and other ocular diseases. Azura’s pipeline of internally discovered new chemical entities represents a portfolio of potentially first-in-class ophthalmic therapeutics for significant unmet needs. For more information, visit www.azuraophthalmics.com and follow Azura on LinkedIn. References Watson SL, Jones LW, Stapleton F, Hinds M, Ng A, Tan J, et al. Efficacy and safety of AZR-MD-001 selenium sulfide ophthalmic ointment in adults with meibomian gland dysfunction: A vehicle-controlled, randomized clinical trial. Ocul Surf. 2023 Jul;29:537-46. Downie LE, Craig JP, Stapleton F, Tan J, Jones LW, Ng A, et al. Efficacy and safety of AZR-MD-001 selenium sulfide ophthalmic ointment in adults with meibomian gland dysfunction over six months of treatment: A Phase 2, vehicle-controlled, randomized extension trial. Ocul Surf. 2025 Jan:35:15-24. Stapleton F, Hinds M, Tan J, Jones L, Chalmers R, Bosworth C, Alster Y. AZR-MD-001 0.5% selenium sulfide ophthalmic ointment for the treatment of contact lens discomfort: A vehicle-controlled, randomized, clinical trial. Ocul Surf. 2024 Dec 28:S1542-0124(24)00144-7.