Granuloma Annulare

为打造全球ADC/RDC未来产业高地，成都医学城拟于2024年4月18日-19日举办首届未来XDC新药大会！大会以“共创偶联药物产业未来”为主题，由同写意策划，安排一场主论坛和九场主题论坛，邀请“政、产、学、研、金、媒”各领域专家，通过“成果发布、主题演讲、热点对话、表彰颁奖、展览展示”五位一体的多元化的组织形式，迸发更多XDC产业新科技、新业态、新模式，构建XDC产业全新生态，触摸医药产业未来脉动。昨天筛了一下市值排名前20的，有产品收入的Biotech公司。然后扒了一下各家2023年的收入（其中传奇的收入是一致性预期）。最后再翻了一下各家有多少员工。想综合看看谁家有Smart Drug的属性。具体数据如下：其实Viatris不算Biotech先看一下这TOP20有产品收入的公司市值：再看看各家的收入情况：众人皆爱看的PS：而我独爱看市值/员工比。各家员工人数：笼统的说，如果单个员工贡献的市值高，大多情况下可以理解为“公司只用很少的人力就能做出大家觉得很有前景的产品”。再换句话说：“产品足够牛逼，以后可以轻轻松松卖很大”。我喜欢管这类产品叫做：a drug that sells itself，“不用推销，我自己卖自己”！当然这只是笼统的说，每家公司的实际情况还是不一样的。比如有些基因疗法实际上是因为单价特别高，或者是公司将临床开发全部外包，自己临床团队人数很少等等....1）排名第一的Argenx主要是靠一款Vyvgart，这款药能给公司带来很高的市值/员工比主要是占了几大优势：gMG适应症的先发优势+高定价（一年22w+）+患者需求高会主动找药 = 销售不需要靠堆人头战术。2）Vertex：因为核心品种Trikafta是治疗罕见病的药物，患者不多，自然销售也不需要靠人海战术。且他在CF市场没有竞争对手。而且也是高定价（30w+）。也是患者会主动找上门的那种，因为背后有CFF基金会帮他们找患者，给患者宣传。第一梯队的ARGX和VRTX都是主攻罕见病市场，市值/员工比已经和第二梯队拉开了很大的距离。3）Roivant这个公司我还没仔细看过，只能大概说一说。这公司主打免疫新疗法，管线里有5个品种。其中关注度最高的就是下一代FcRn，市场对这个品种的预期是比着Argenx去的，也是贡献市值的主要因素。公司有上市产品，但暂时没有进入高速放量的成熟阶段。上市产品VTAMA® (tapinarof) cream在2023年的最后一个季度卖了2000多万，年化8000多万。4）Apellis要不是因为Syfovre眼药安全性问题还拖着，市值能比现在更高。这款药的定价不算特别高，年费用大概2.7~5.5w。但它竞争格局好，是第一个获批专门给GA AMD的药，是该适应症唯一靶向C3的药。竞争少，销售团队也就相应比较小。5）United Therapeutics的肺病药Tyvaso上量很猛的，有多个适应，去年8/7才获批，Q4销售额就3.5亿了（肺动脉高压单适应症）。2022年市场预期这个药峰值至少30亿+，总体产品价值150亿。市场最关注的肺纤维化适应症还早，今年年底才入组完。就是不知道3月马上要获批的sotatercept是不是会影响它。全球新药峰值estimate TOP10中也有它的一席之位：（下图没算减肥药）在市值TOP20的公司中，单个员工贡献市值800~1000万的公司最多，有9个，其中还包括：5）Regeneron这种Biopharma，产品销售的主力其实是MNC大药企合作伙伴。6）Alnylam：大家很看好的公司，但研发进展一直很尴尬。目前收入还是靠罕见病支撑，且不少还是ultra rare罕见病，这类疾病患病人数太少了，同时药物定价也没有达到几百万的那种水平。因此总体收入还是比较有限。最吃香的罕见病药物特点是：1）主要发病地区是欧美这类支付能力比较强的国家，2）患病率大约是5~10万人，3）定价在20~40万之间。注：罕见病大概率=高单价+小团队销售。再往后看7）Moderna和Biontech排名比较靠后主要是因为之前卖疫苗留下的人，疫苗是典型的低单价，高普及率的产品，需要人手。8）百济排名比较靠后，他们家员工人数比较多，比Biogen多，仅次于Regeneron。平均每个员工贡献收入20万。相比之下，另一个极端的UTHR和VRTX单个员工分别可贡献200、180万美元收入。注：这是总员工数，包括销售、研发、行政等，收入是公司总收入，除了产品收入外，可能还包括一些其他的合作收入等等。客观数据，没有不看好的意思。最后再看看这些公司2024年年初至今的涨跌幅：更多优质内容，欢迎关注↓↓↓

不到半年时间，FDA批准了第二款用于眼科疾病的治疗补体药物。昨日（2023年8月7日），VERIC bio公司宣布，其补体因子C5抑制剂Zimura治疗年龄相关性黄斑变性(AMD)相关的继发性地图样萎缩(GA)获得FDA批准上市，成为首款用于GA的补体C5抑制剂，商品名为Izervay。AMD分为干性AMD和湿性AMD。地图状萎缩（GA）是干性AMD的晚期形式，这类患者已经存在明显的视力损害且有继续恶化的风险。2023年2月17日，FDA批准了首个GA疗法--Syfovre，Apellis 公司的补体C3抑制剂，通过靶向 C3，Syfovre旨在提供对补体级联反应的全面控制。基于GA领域先前空白，Syfovre批准以来快速打开市场，2023Q2 Syfovre的销售额达6730万美元，占公司2023Q2总收入(9497万美元)的71%。不过，Syfovre也报告了令人担忧的严重的副作用事件--视网膜血管炎，Syfovre获批以来Apellis已确认总共7例出现了视网膜血管炎事件，包括4例闭塞性，3例非闭塞性。该事件发生最终可能带来失明风险，公司正在视网膜血管炎病例的可能原因进行全面调查。鉴于Syfovre目前原因不明的并发症事件，Apellis的股价因此大跌37%。并且可能会限制使用。Zimura的获批时间也很关键，Zimura是Iveric bio研发的一款特异性补体因子C5抑制剂，其可阻断C5的切割和末端片段C5a及C5b的形成，以减少炎性小体的激活和膜攻击复合物（MAX）的形成，从而抑制或减缓视网膜色素上皮细胞（RPE）变性，缓解GA-AMD疾病进展。GATHER1与GATHER2两项Ⅲ期临床试验均宣告成功。与对照组相比，12个月内，Zimura可大幅降低地图状萎缩病变速度，并且耐受性良好。总结在罕见病之外，补体药物在眼科的常见病中已彰显应用前景，特别在干性AMD的眼科疾病中，补体理论是当下最主流的治疗机理。除了C5，C3之外，补体系统中的D因子，FB等在眼科的开发应用逐渐活跃起来。本周好文推荐如需转载请联系佰傲谷并在醒目位置注明出处﹀ ·········点亮在看，传递信息♥

Company and Yale University Hospital seeking participants with GA for Phase 1 trials of medicine FDA-approved for other ailments NEW HAVEN, Conn., June 2, 2023 /PRNewswire/ -- TWi Biotechnology (TWiB), a clinical stage biopharmaceutical company specializing in development of repositioned drugs for unmet medical needs, is currently seeking participants for a Phase 1 clinical trial of a topical gel called tofacitinib for the treatment of granuloma annulare (GA). The TWiB team and collaborating physicians invite eligible individuals to participate in this groundbreaking study at Yale University Hospital. Individuals with GA or friends and family members can find study information at: . For further information, please contact: Nicole Olszewski Study Coordinator +1-203-785-5505 [email protected] GA is a rare skin condition affecting approximately 330,000 cases in the US, predominantly in women. It is characterized by raised bumps in a ring pattern, most commonly on the hands and feet, and while it is neither contagious nor causes pain, it can cause a high degree of self-consciousness. This can be particularly detrimental to patients with long-term cases or a highly visible rash, who can suffer from emotional distress. The disease is poorly understood and has no effective treatment, Dr. William Damsky, MD, PhD of Yale School of Medicine — the clinical trial's principal investigator — has already conducted a small-scale study showing that oral tofacitinib, a type of medicine called a JAK inhibitor, shows promise for GA — treatment with tofacitinib both improved skin disease and reduced circulating inflammatory biomarkers in all GA patients. The current trial, using a topical treatment already approved for oral use to treat other, more common ailments, represents a significant step towards addressing this unmet medical need. Repurposing an FDA-approved medicine The oral formulation of tofacitinib has already been FDA approved to treat such ailments as rheumatoid arthritis, psoriatic arthritis, ulcerative colitis, polyarticular course juvenile idiopathic arthritis, and ankylosing spondylitis. The topical gel formulation of tofacitinib (AC-1101), which is being tested in this clinical trial, is applied topically to the skin and tends to have lower systemic exposure compared to the oral route. Addressing gaps in treatment Channeling science and integrity to alleviate the suffering of patients, TWi Biotechnology specializes in rare diseases and dermatological diseases associated with inflammation or inflammatory conditions — typically helping patients that have been left behind by the larger pharmaceutical industry. TWi Biotechnology's specialty of developing drugs for unmet medical needs focuses on drug repositioning — repurposing already-approved treatments for a different ailment. In particular, the company has demonstrated leadership in developing treatments for rare skin diseases, including epidermolysis bullosa simplex and GA. About TWi Biotechnology TWi Biotechnology, Inc (TWi Biotechnology) is a clinical stage biopharmaceutical company based in Taipei, Taiwan specializing in the development of repositioned drugs for unmet medical needs, especially in rare diseases associated with inflammation or inflammatory conditions. Established in July 2010, TWi Biotechnology is expanding its product pipeline through in-licensing and internal research. TWi Biotechnology's product development pipeline includes two drug candidates for treating epidermolysis bullosa simplex and immunodermatology diseases. With its extensive experience in clinical, pre-clinical, animal pharmacology/toxicology studies, domestic and international regulatory standards, and deep knowledge in international patent protection and the specialty drug market in the US, TWi Biotechnology is in a leading position to develop new treatments in these therapeutic areas. For more information, please visit: . Media Contact: Michelle Chen, [email protected] SOURCE TWi Biotechnology,Inc