Elexacaftor/Ivacaftor/Tezacaftor

Vertex's momentum for its two new launches were partly overshadowed by a loss of Trikafta sales in Russia. For Vertex Pharmaceuticals, the first quarter of 2025 brought good news and bad news. On the bright side, early momentum for newly launched cystic fibrosis (CF) med Alyftrek and pain treatment Journavx contributed to a U.S. revenue boost and the company's increased 2025 outlook. But, on the other hand, a unique generics situation in Russia weighed on the biopharma's international sales. The mixed bag evened out to a 3% year-over-year revenue gain to $2.77 billion, with $1.66 billion coming from the U.S., where Vertex grew sales by 9%.Outside of the U.S., the company's $1.11 billion in first-quarter sales represented a 5% slide from the same period last year.Despite “strong patient demand” in markets outside of the U.S., Vertex's international revenue was impacted by an “expected” decline in Russia, where the company is “experiencing violation of its intellectual property rights,” Vertex explained in its earnings release.“Vertex has strong intellectual property protection for its medicines around the world and believes this violation in Russia is a limited and isolated matter,” the company said.The situation boils down to an unauthorized generic of Vertex’s top-selling CF med Trikafta marketed in Russia. Since January, the Russian Ministry of Health has been switching children who take Trikafta to an Argentina-made generic called Trilexa, independent Russian news publication Okho reported in March, citing stories from parents of patients. The drug has not been tested in clinical studies, and the move has prompted protests within the CF community, according to Okho. As that situation plays out, Vertex’s quarterly revenues came in below consensus expectations of of $2.83 billion, Evercore ISI analysts wrote in a note to clients. BMO analyst Evan Seigerman also cited the “onetime headwinds” from Russia, plus “new product launch noise,” as reasons for the revenue miss.Still, given the trajectory of its two new launches—plus “contributed uptake” of CRISPR Therapeutics-partnered sickle cell and beta thalassemia gene therapy Casgevy—Vertex opted to slightly boost its full-year revenue guidance. Now, the company is eyeing 2025 revenues of $11.85 billion to $12 billion, up from prior guidance of $11.75 billion to $12 billion. Journavx's journey Vertex’s non-opioid Journavx was approved in January to much fanfare considering its unique position as a nonaddictive pain management treatment. The company quickly set out to begin shipments and has since racked up more than 20,000 prescriptions, Vertex reported, although revenue from the drug was “insignificant” during the first quarter because it's still so early in the launch. In his note to clients, Seigerman praised the company's access efforts and said he expects more revenue contributions from the med in the second half of the year.Ensuring patients have access to Journavx is especially key given the cheap generic opioids that currently dominate the pain market.  Alyftrek, meanwhile, won a December FDA approval as a once-daily triple combination for CF patients that have certain mutations. The drug has “potential to set a new standard of CF care,” according to Vertex, which looks to transition patients currently taking established Trikafta over to Alyftrek. So far, the company is seeing “enthusiastic feedback” from physicians and patients, it said in its earnings presentation. The company racked up $53.9 million in Alyftrek revenues in the first quarter. 

近日，根据外媒报道，罕见病巨头Vertex Pharmaceuticals将停止使用常用于基因治疗药物封装和递送的腺相关病毒（AAV）载体技术。这一决定的做出早有先兆，在今年2月末，Vertex已经终止了与Verve Therapeutics的一项最初于2022年展开的合作，合作为期四年，原本包括预付款和里程碑付款在内的交易总额达到了4.66亿美元。而彻底放弃AAV技术平台则意味着至少有两家AAV相关合作企业可能受到波及。涉及项目首先，2020年Vertex与另外一家AAV基因治疗公司Affinia Therapeutics签订的合作协议将就此作废。项目涉及AAV靶向肌肉疾病，如杜氏肌营养不良症（DMD）和强直性肌营养不良1型（DM1），而这次涉及的合作金额规模更大，潜在总额高达16亿美元。其次，tRNA治疗开发公司Tevard Biosciences也因为使用AAV作为载体而遭到了波及，双方最初于2023年2月达成了数额不明的合作协议，合作项目主要涉及无义突变的DMD，合作为期四年，有希望扩展到第二适应症。较为可惜的是，由于这两家都是未上市企业，目前尚未能清楚合作终止对于这两家企业的具体影响，不过从早先的采访中可以得知，Affinia至少收到了里程碑付款。另外，2019年Vertex收购过一家AAV基因治疗开发公司Exonics，这家公司的管线也是以DM1/DMD为核心的，公司计划利用SingleCut CRISPR对这些疾病进行基因编辑，然而收购之后，公司管线也没什么消息。推测在此次裁撤管线风波中，Exonics留存下的管线也会遭到抛弃。虽然放弃了AAV的相关研究，Vertex并没有放弃基因细胞治疗，公司发言人表示公司对细胞和基因疗法的承诺依然坚定，并将在DMD和DM1领域继续投资。然而，实际情况是Vertex自身的业绩并不容乐观。在该公司今年第一季度财报中展现出的数据让人大跌眼镜，在5月5日财报发布后，Vertex股价更在盘后交易中跌去了3%。基本盘或因为俄罗斯有失？Vertex的基本盘一直以来都是囊性纤维化药物。然而现在这个基本盘正在遭受知识产权问题挑战。从数值来看，虽然说整体收入相比于2024年第一季度增加了3%，但是如果将对象设定为2024年第四季度，那就会发现，Vertex的收入是在呈现出下滑趋势的。2024年第四季度的总收入为29.12亿，而2025年仅为27.7亿美元。Vertex在其财报中将收入下降原因归因于俄罗斯，财报指责俄罗斯目前正在侵犯Vertex的知识产权。虽然美国国内的收入增加了9%，但是在美国之外的市场，由于俄罗斯侵犯知识产权的原因总收入下降了5%。由于囊性纤维化高发于德系犹太人，具有一定德系犹太人人口的俄罗斯市场其实不容小觑。至于专利侵犯问题，则可以从2023年俄罗斯法院的判决看出。根据俄罗斯第九上诉商业法院2023年9月25日就第A40-185112/2022号案件作出的裁决，法院依据《俄罗斯民法典》第1362条第1款（专利使用不足）之规定，责令Trikafta药物系列专利权人Vertex制药公司向俄罗斯制药企业MIK公司授予普通（非独占）许可。考虑到Vertex本身是依靠高额囊性纤维化药价来维持公司收入的，俄罗斯药企MIK如果采用低价大规模生产囊性纤维化药物，就有可能导致Vertex的商业利益受到严重损失。在这种情况下，Vertex做出大规模裁去AAV等高价管线的行动也是可以理解了。参考来源：https://www.lidings.com/media/legalupdates/lackofuse/https://investors.vrtx.com/news-releases/news-release-details/vertex-reports-first-quarter-2025-financial-results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········