Meningococcal vaccine groups A C Y W-135 conjugate (second generation)(Sanofi)

Taylor Tieden for BioSpace On the FDA’s docket this month are two expansion bids, one for GSK’s asthma drug Nucala into COPD and another for Merck’s oral cancer drug for a pair of rare tumors. Amid the growing macro-level headwinds in the vaccine space, the FDA this month is expected to decide on Moderna’s mRNA COVID-19 shot, alongside a handful of other big verdicts. Read below for more. GSK Asks FDA to Approve Asthma Injection Nucala for COPD GSK is proposing its subcutaneous biologic Nucala to treat adult patients with chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype. The FDA’s decision is due on May 7. A pulmonary disease, COPD is characterized by inflammation in the lungs, manifesting as breathlessness, coughs and airflow obstruction. According to GSK, some 40% of patients suffer from disease exacerbations caused by type 2 inflammation, which in turn is linked to high eosinophil counts in the blood. Nucala , a monoclonal antibody, targets and blocks the IL-5 cytokine, which is crucial to maintain the activity of eosinophils. GSK is supporting Nucala’s COPD application with data from the Phase III MATINEE trial. The pharma released a topline readout from the study in September 2024, noting that Nucala elicited a “statistically significant and clinically meaningful reduction” in yearly rates of moderate or severe disease exacerbations as opposed to placebo. Nucala is currently approved for eosinophilic granulomatosis with polyangiitis and hypereosinophilic syndrome, and as an add-on maintenance treatment for asthma and chronic rhinosinusitis with nasal polyps. Arcutis Seeks Zoryve Expansion into Body and Scalp Psoriasis By May 22, the FDA will release its verdict on Arcutis Biotherapeutics’ proposed expansion for Zoryve into body and scalp psoriasis. Arcutis is backing its expansion bid with data from the Phase III ARRECTOR trial, which found that 66.4% of Zoryve-treated patients achieved clear or almost-clear skin, as opposed to 27.8% in placebo patients. Results also showed that 65.3% of patients in the Zoryve arm saw significant improvements in itch, versus 30.3% in the placebo group. Arcutis also filed findings from a Phase IIb study as well as long-term efficacy and safety data for Zoryve in plaque psoriasis. Zoryve is a topical phosphodiesterase 4 inhibitor that works by helping to suppress inflammation in the skin. The drug won FDA approval in 2011 for the treatment of plaque psoriasis in patients aged 12 years and up. If approved for body and scalp psoriasis, Zoryve would be a “truly meaningful innovation” for patients who suffer from inadequately controlled symptoms, Arcutis CEO Frank Watanabe said in a September 2024 release. Sanofi Eyes Extension of Meningococcal Disease Shot to Infants, Toddlers Sanofi’s meningococcal disease vaccine MenQuadfi is currently approved for the primary immunization of children aged two years and up. As a booster, it can be used in those 13 years and older who continue to be at risk of meningococcal disease. Sanofi is seeking to expand MenQuadfi’s label to also include infants and toddlers from six weeks to 23 months of age. The FDA is currently reviewing this proposal and is due to release its decision on May 23. If approved, MenQuadfi could protect this younger population from the A, C, W and Y serogroups of Neisseria meningitidis . In an April presentation before the CDC’s Advisory Committee for Immunization Practices, Sanofi presented data from several trials, including the Phase III MET42 study. Results showed that MenQuadfi was safe to use in infants and toddlers, with most side effects being mild or moderate in severity. Efficacy-wise, MenQuadfi was able to match a commercially approved vaccine in immune response and seroprotection. Liquidia Awaits Final Yutrepia Approval in Interstitial Lung Disease On May 23, Liquidia is expecting to win final approval of its inhalation powder Yutrepia for the treatment of patients with pulmonary arterial hypertension associated with interstitial lung disease (PH-ILD). The FDA gave the drug tentative approval in August 2024, indicating that it had cleared all efficacy and safety hurdles and satisfied regulatory standards of product quality. However, Liquidia still needed to wait out regulatory exclusivity for a competing product. Yutrepia is Liquidia’s inhalable version of treprostinil, a vasodilator that has known activity against pulmonary hypertension. Yutrepia is backed by data from the Phase III INSPIRE study, which found that the drug was both safe and well-tolerated in patients who were naïve to treprostinil or who were switching to Yutrepia from nebulized treprostinil. The INSPIRE trial found that patients treated with Yutrepia saw symptoms stabilize or improve over one year of dosing. Merck Hopes to Tack Two Rare Cancers Onto Welireg’s Label Merck is hoping to expand its oral cancer drug Welireg into advanced pheochromocytoma and paraganglioma (PPGL). The FDA’s decision deadline is May 26. PPGL refers to two types of rare tumors of the adrenal gland. Diagnosed in some 2,000 patients per year, they are typically caused by specific genetic mutations. Symptoms include high blood pressure, palpitations and headaches. According to Merck, up to 25% of PPGL cases are already at the metastatic stage at diagnosis. Merck is supporting Welireg’s PPGL bid with data from Cohort A1 of the Phase II LITESPARK-015 study. The pharma did not provide specific data in a January news release , revealing only that the application is based on objective response rate and duration of response in PPGL patients after Welireg treatment. The FDA has granted the drug priority review in this indication. Welireg is an orally available inhibitor of the HIF-2α transcription factor, which is involved in oxygen sensing and in the body’s response to hypoxia. The drug is indicated for patients with renal cell carcinoma (RCC) and those with von Hippel-Lindau disease—a rare genetic disease involving the formation of tumors across various organs—who also require treatment for associated RCC. If approved, Welireg will become the only drug for advanced PPGL in the U.S., Merck said. After Delay, Eton Nears Approval for Oral Hydrocortisone By May 28, the FDA will release its verdict on Eton Pharmaceuticals’ ET-400, an oral formulation of hydrocortisone, for the treatment of adrenal insufficiency in infants. This new target action date comes after the FDA in February announced that it needed more time to review Eton’s data package for ET-400. Eton submitted supplemental information in December last year, in response to a request from the regulator. ET-400 is a proprietary oral formulation of hydrocortisone that, according to the biotech, is stable at room temperature. If approved, ET-400 will follow in the footsteps of Eton’s Alkindi Sprinkle, which also has hydrocortisone as an active ingredient, but is formulated as oral granules. Alkindi Sprinkle, approved in 2020 , is designed to rapidly deliver glucocorticoids to children with adrenocortical insufficiency. In a July 2024 announcement , Eton CEO Sean Brynjelsen said that the approval of ET-400 would allow the biotech to “capture a greater percentage of the oral hydrocortisone market” and, alongside Alkindi Sprinkle, would open up a peak opportunity of “more than $50 million annually.” Moderna Seeks Approval for Next-Generation COVID-19 Shot The FDA’s last big milestone for the month belongs to Moderna and its investigational next-generation COVID-19 vaccine mRNA-1283. The regulator is expected to release its decision on May 31. In June 2024, the company released Phase III data for the experimental shot, touting better efficacy than Spikevax, its currently approved COVID-19 vaccine. Moderna did not provide specific data, only claiming that mRNA-1283 elicited a “higher immune response” against SARS-CoV-2 than Spikevax. Of note, Moderna emphasized at the time that mRNA-1283 could induce robust responses against Omicron and original strains of the virus—a benefit the company claimed was most evident in seniors over 65 years of age. The CDC’s vaccine advisors were supposed to convene in late February to discuss various vaccines, including mRNA-1283, before the meeting was postponed . The panel finally convened last month.

声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。 近日，法国制药巨头赛诺菲（Sanofi）公开了2024 Q3最新管线进展和业绩情况。 业绩概要 财报显示，2024 Q3公司总销售额达到134亿欧元，同比增长15.7%，主要得益于流感和呼吸道合胞病毒疫苗销售的提前。得益于强劲的业务表现，赛诺菲已将2024年的每股业务收益指引上调至至少实现低个位数百分比增长。 根据板块来看，2024 Q3制药业务收入83.65亿欧元，同比增长13%；疫苗收入38.02亿欧元，同比增长25.5%，主要得益于流感疫苗的分阶段销售，以及 Beyfortus因获批增加产能而带来的增长；消费者保健业务收入12.71亿欧元，同比增长7.9%。赛诺菲预计最早将于2025年Q2完成拆分消费者保健业务，专注于创新药业务和疫苗。 根据产品来看，Dupixent（度普利尤单抗）为最吸金的产品，2024 Q3的销售额34.76亿欧元，同比增长23.8%。Dupixent2024年前三季度总销售额已经达到96.14亿欧元，2024年销售额有望达到130亿欧元。 管线调整 2024 Q3，赛诺菲移除了两项III期研究和一项II期研究。 1、losmapimod用于治疗面肩肱型肌营养不良（FSHD）的III期临床研究 Losmapimod是一种口服选择性p38α/β丝裂原活化蛋白激酶（MAPK）抑制剂，最早由葛兰素史克开发，2019年4月，Fulcrum从GSK手中收购了losmapimod的所有权益。今年5月13日，赛诺菲从Fulcrum Therapeutics引进Losmapimod（洛吡莫德）的开发和商业化权益。 根据协议条款，Fulcrum将获得8000万美元首付款，并有资格在达到特定的监管和销售里程碑后获得高达9.75亿美元的额外付款，以及从Losmapimod在美国以外的年度净销售额中获得从低十位数开始递增的分层特许权使用费。 2024年9月12日，Fulcrum Therapeutics宣布losmapimod治疗面肩肱型肌营养不良症（FSHD）的III期REACH试验未能达到主要终点。此外，次要终点也没有统计学意义。losmapimod的安全性和耐受性与之前报告的研究结果一致。公司将完成对所获数据的全面评估，并计划在即将召开的医学会议上分享评估结果。该消息发出后，Fulcrum的股价也下跌了约61%。 2、流行性脑脊髓膜炎疫苗MenQuadfi III期研究 MenQuadfi是赛诺菲的一款创新性脑膜炎球菌疫苗，MenQuadfi是美国FDA批准的唯一一种适用于2岁至56岁及以上人群的四价脑膜炎球菌疫苗。该疫苗也是美国第一种也是唯一一种使用破伤风类毒素作为蛋白质载体的四价脑膜炎球菌疫苗。此次终止的是一项MenQuadfi的六周+ III期研究。 3、oditrasertib治疗多发性硬化症（MS）的II期研究 Oditrasertib是一款RIPK1抑制剂。赛诺菲和 Denali 于2018 年达成合作开发了 RIPK1 抑制剂用于治疗一系列神经系统和全身炎症性疾病。根据协议条款，赛诺菲将向Denali支付1.25亿美元的预付款，以及未来可能超过10亿美元的开发和推广里程碑付款。 赛诺菲正在停止其在研 RIPK1 阻断剂oditrasertib 的多发性硬化症II期研究，因为该候选药物未能达到其主要和关键次要终点。这不是该药物今年的第一次失败。2024年4月，赛诺菲宣布终止oditrasertib 在肌萎缩侧索硬化症 (ALS) 中的II期试验，因为中期结果令人失望；2024 年2月，oditrasertib用于治疗ALS的II期HIMALAYA研究未达主要研究终点。 赛诺菲Q3的管线进展里程碑包括4项监管批准和4个III期研究取得了阳性结果。度普利尤单抗在美国和中国获批COPD适应症，以及该药在美国的青少年人群中获批用于慢性鼻窦炎伴鼻息肉适应症，另一项监管批准是Sarclisa的多发性骨髓瘤适应症。III期研究中，度普利尤单抗的BP、CSU研究达主要终点；Tolebrutinib的nrSPMS研究和Sarclisa的NDMM、TE研究也达到了主要终点。 赛诺菲预计2024 Q4将迎来几个管线里程碑，包括治疗IgG4介导疾病的口服BTK抑制剂ralzabrutinib和治疗炎症性肠病的抗TL1A生物制剂duvakitug的中期临床试验。 参考资料：公司公告 -END- 2025CMC-CHINA药博会 • 展位火热预定中！