Bioverativ, Inc.

本届大会以“共建创新生态 共赢产业未来”为主题，聚焦生物医药出海与国际化创新、创新药投融资、医疗器械等产业发展风向标，打造“高端化、国际化、专业化、立体化”的年度盛会。 美东时间2024年10月4日，意大利公司Recordati宣布与赛诺菲达成一项协议，获得Enjaymo（sutimlimab）的全球销售权，这是目前唯一获准用于治疗冷凝集素病（CAD）的靶向生物制剂，冷凝集素病是一种罕见B细胞淋巴细胞增生性疾病。 根据协议条款，Recordati将支付给赛诺菲8.25亿美元的预付款，如果净销售额达到或超过高峰年预期销售额的上限，还将支付最高2.5亿美元的额外商业里程碑付款。 过去12个月（截至2024年8月），Enjaymo带来的收入约为1亿欧元，预计2025财年的收入将超过1.5亿欧元，销售峰值可能达到2.5-3亿欧元，是目前水平的两倍多。 Recordati预计，该交易对2024年的收入贡献很小，但将立即增加息税折旧摊销前利润（EBITDA），到2025年，其利润率将高于目前罕见病公司的平均水平。 1 Enjaymo背后的战略转变 Enjaymo（sutimlimab）是一种人源化单克隆抗体，用于治疗成人CAD溶血。2022年，它获得了FDA、欧盟委员会（EC）和日本厚生劳动省的批准。这种慢性静脉注射治疗药物满足了CAD患者尚未得到满足的医疗需求。 从疾病市场来看，CAD在美国、日本和欧洲大约仅有11000名患者。 这是一种罕见病，B细胞产生的自身抗体会攻击并破坏红细胞。该病理过程在寒冷条件下触发，通常在3°C至4°C之间，患者会出现严重的衰弱性疲劳、虚弱和其他典型的贫血症状。 针对CAD，Enjaymo选择性地靶向C1补体蛋白，阻止补体级联的激活。这种作用机制使它能够有效阻断补体介导的红细胞破坏，且不会抑制凝集素和替代补体途径。 Enjaymo一直被视作赛诺菲快速增长的资产，但其销售额一直处于中等水平。2023 年，该疗法产生了近8000万美元的收入，按固定汇率计算，比前一年增长了200%以上。2024年上半年，Enjaymo同比增长近73%，带来约6000万美元的收入。 对于赛诺菲来说，Enjaymo的出售似乎是更广泛战略转变的一部分，该公司放弃了之前到2025年达到32%营业利润率的目标后，首席执行官Paul Hudson正在努力恢复投资者的信心。 对于罕见病药物公司Recordati来说，这笔交易为该公司的产品组合增加了第九个上市产品，Enjaymo的交易符合其公司在全球广泛的战略布局，扩大了其在美国、日本和欧洲的市场。 2 在巨头之间辗转 Enjaymo曾经历过多次易主。 2014年，总部位于旧金山的iPerian公司还在研发Enjaymo这款药物。就在那一年，BMS因其在神经退行性疾病领域的工作而收购了iPerian公司。 iPerian针对免疫系统的项目被剥离出来，单独成立了一家新的生物技术公司True North Therapeutics。 不过，True North公司并没有独立存活太久。2017年，它被渤健分拆出来的专注于血友病的Bioverativ公司收购。当时的Bioverativ首席执行官John Cox说，Enjaymo是这笔交易中的核心资产，填补了公司研究工作中的一个重要空白。 然而，2018年赛诺菲以110亿美元收购Bioverativ后，Enjaymo又易主了。 赛诺菲多次转向交易，以进一步扩大其罕见病业务。在过去一年里，这家法国制药巨头就获得一种治疗肌肉萎缩症的实验性药物的权利，并斥资22亿美元收购了一家生物技术公司及其治疗罕见肺病的潜在治疗方法。 赛诺菲还试图从初创公司Maze Therapeutics那里获得治疗庞贝病的药物许可，但在面临联邦贸易委员会的诉讼后放弃了该交易。 通过收Bioverativ，赛诺菲获得了Enjaymo以及另一种现在上市的血友病药物Altuviiio。 参考资料： 1.RECORDATI ANNOUNCES AGREEMENT TO ACQUIRE THE GLOBAL RIGHTS TO ENJAYMO®, STRENGTHENING ITS RARE DISEASES FRANCHISE 2.Sanofi’s rare disease drug finds yet another home；BioPharma Dive 3.Sanofi Offloads Rare Autoimmune Drug to Recordati in Potential $1B Deal；Biospace 同写意媒体矩阵，欢迎关注↓↓↓

Sanofi is offloading its rare autoimmune disease medicine Enjaymo to an Italian pharmaceutical company for $825 million upfront. Recordati, based in Milan, said Friday it will pay up to an additional $250 million over time for the monoclonal antibody, which was greenlit in the US , Europe and Japan in 2022 for hemolysis in adults with cold agglutinin disease (CAD). In patients with the rare condition, their immune system prematurely demolishes red blood cells, and it can lead to severe anemia. Sanofi recorded €55 million ($60 million) in Enjaymo sales in the first six months of 2024, it said in July. Recordati predicts the drug could reach annual peak sales of €250 million to €300 million (about $275 million to $330 million). It marks a winding road for Enjaymo, which has gone through the full biotech-to-pharma journey. A Bay Area biotech called True North Therapeutics developed the drug and then was bought by Bioverativ for $400 million upfront in 2017. Less than 12 months later, Sanofi acquired Bioverativ for almost $11.6 billion. Two years after that, Sanofi was handed a rejection for the drug before it was approved in 2022. For Recordati, the deal pushes it further into the rare disease market. The company bought EUSA Pharma and its four rare disease drugs for nearly $850 million in December 2021. It’s also struck deals with GSK , Almirall and others in recent years. With “a strong clinical profile and as the only product approved for the treatment of CAD, Enjaymo addresses a serious unmet medical need for patients living with this debilitating disease,” Recordati CEO Rob Koremans said in a press release. Earlier this year, Sobi and Apellis Pharmaceuticals stopped a Phase 3 in CAD, citing “a decreased medical need in CAD and therefore a limited number of patients eligible for the study.” CAD affects about one person per million every year, according to the National Organization for Rare Disorders. The Recordati deal comes as Sanofi adjusts its portfolio. It now plans to hone in on immunology and inflammation under new R&D head Houman Ashrafian and new CSO Mike Quigley , who joined Sept. 30. It’s also planning to divest its consumer health group Opella. Earlier this year, Sanofi sold some early-stage cancer projects to Vir Biotechnology and shuttered a cell therapy unit, though it’s still making a few bets in oncology, including its first foray into radiopharma . The French drugmaker also has invested in several companies, including Ventyx, Vicore and Graviton.

Over the past decade, the medicine Enjaymo has been passed around no less than five times by developers large and small. Now its trading hands again, through a deal announced Friday.Sanofi is selling global rights to Enjaymo to the Italy-based drugmaker Recordati, in exchange for an upfront payment of $825 million. And if the medicine hits certain sales goals, Sanofi could take home up to $250 million more.For Recordati, which specializes in rare diseases, the deal adds a ninth marketed product to the companys portfolio. Enjaymo is approved in the U.S., Europe and Japan as a treatment for an uncommon type of anemia. In this condition, known as cold agglutinin disease or CAD, the bodys immune system mistakenly attacks and destroys some red blood cells. Enjaymo is designed to tamp down that immune response and spare the cells, thereby decreasing the need for red blood cell transfusions.Enjaymo won U.S. approval in early 2022 after a winding journey. In 2014, the medicine was in development at San Francisco-based iPerian. But that year, Bristol Myers Squibb bought the company for its work in neurodegenerative diseases. iPerians programs targeting the immune system were spun out into a new biotechnology firm, True North Therapeutics.True North wasnt around for very long, though. By 2017, it had been snapped up by Biogens hemophilia-focused spin off Bioverativ. John Cox, the then-CEO of Bioverativ, said at the time that Enjaymo was a central asset in the deal and filled an important gap in the companys research efforts.Enjaymo, however, would move to yet another owner in 2018, when Sanofi bought Bioverativ for $11 billion.Sanofi has turned to dealmaking several times since to further build out its rare disease business. Over the past year alone, the French pharmaceutical giant nabbed rights to an experimental drug for muscular dystrophy and spent $2.2 billion to acquire a biotech and its potential treatment for a rare lung disease.Sanofi also tried to license a drug for Pompe disease from the startup Maze Therapeutics, but gave up on that deal after facing a lawsuit from the Federal Trade Commission.From the Bioverativ acquisition, Sanofi got Enjaymo as well as another now-marketed hemophilia medicine, Altuviiio. According to Recordati, Enjaymo generated around 100 million euros, or roughly $108 million, over the 12-month period between September 2023 and August 2024. The company also expects revenue from the drug to exceed 150 million euros in the 2025 fiscal year, and reach peak annual sales of 250 million to 300 million euros.In a statement, Recordatis CEO Rob Koremans said the Enjaymo deal aligns with his companys broader strategy, complements its portfolio of cancer therapies, and expands its footprint in the U.S., Japan and Europe.Recordati says it will use its existing cash as well as new committed bank debt facilities to finance the deal, which is slated to close by the end of the year. '