mRNA-1010

Taylor Tieden for BioSpace Many of the FDA’s decisions this quarter involve applications that have previously been delayed, declined or outright rejected, including one for an mRNA vaccine that became the center of controversy earlier this year. The third quarter of 2026 is looking like a busy one for the FDA, with seven noteworthy decisions on its docket, including one that could mark a first in Alzheimer’s disease care and another that could justify a recent high-value buyout. Read below for more. Vera aims to challenge big names in IgAN arena In one of Q3’s earliest decisions, Vera Therapeutics is looking to join the IgA nephropathy game with its investigational fusion protein atacicept. The drug is currently under FDA review with a target action date of July 7 . Backing atacicept’s application are data from the Phase 3 ORIGIN 3 study, in which the drug elicited a 46% decrease in proteinuria at 36 weeks versus baseline, according to a November 2025 press release . Atacicept also eliminated blood in the urine in 81% of patients. These data paint a “compelling profile” for atacicept, Guggenheim Partners said in a Nov. 6 note to investors, strengthened even further by the drug’s “excellent safety pro is comparable to placebo.” If approved, atacicept would compete with Otsuka Pharmaceuticals’ APRIL inhibitor Voyxact, which won the FDA’s greenlight in November last year, and Novartis’ complement inhibitor Fabhalta . Vertex Pharmaceuticals, meanwhile, is close on Vera’s heels with its own fusion protein povetacicept, which has an FDA action date of Nov. 30 . Moderna’s mRNA flu shot nears D-day after sparking controversy Moderna’s investigational flu vaccine made headlines in February when the FDA hit the company’s application with a refusal-to- , declining even to give the submission the time of day. The agency at the time said that Moderna’s study used a comparator group that “does not reflect the best-available standard of care.” A few days later, after strong blowback and a hastily arranged type A meeting with the biotech, the FDA reversed course and accepted an adjusted application for review. This time, Moderna is seeking full approval of its mRNA vaccine, dubbed mRNA-1010, in adults 50 to 64 years, and accelerated approval in people 65 and older. A decision is expected on or before Aug. 5. Moderna is supporting the vaccine with many late-stage studies establishing its efficacy. One readout in June 2025 showed the mRNA shot was 26.6% more effective than a licensed comparator flu vaccine in adults 50 years and up. Last month, the FDA’s Vaccines and Related Biological Products Advisory Committee unanimously recommended the mRNA flu shot’s full approval for adults aged 50 through 64, and accelerated approval for those 65 and up. Vaccines Moderna’s mRNA Tech Could Boost Flu Shot Efficacy. Americans Will Have To Wait After the FDA’s decision to reject a review of Moderna’s mRNA-1010 flu shot, executives explain what Americans will miss out on as other nations embrace the technology. February 16, 2026 · 3 min read · Annalee Armstrong Read more Capricor hopes for cell therapy comeback in Duchenne By Aug. 22, the FDA is expected to release its decision on deramiocel, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy. An approval here would serve as a vindication for deramiocel, which was initially rejected by the FDA last summer. At the time, the regulator said Capricor’s data package for the product failed to satisfy the “statutory requirement for substantial evidence of effectiveness.” After deramiocel aced a Phase 3 trial, the FDA lifted its complete response letter in March, allowing the application to once again move forward. Capricor is seeking full approval of the candidate. Deramiocel uses a rare subset of allogeneic cells drawn from the heart that can modulate the immune response and reduce fibrosis. The treatment works by helping maintain the function of cardiac and skeletal muscles. Data from the Phase 2 HOPE-2 study, which supported deramiocel’s initial application, demonstrated significant improvements in upper-limb performance and cardiac function over placebo. Capricor’s resubmission now also includes data from the Phase 3 HOPE-3 study , which in December hit both its primary and secondary endpoints, showing statistically significant benefits in upper-limb function and a slowed decline in cardiac function as measured by left ventricular ejection fraction. Deramiocel also slowed disease progression and boosted overall functional performance, according to further data revealed in March. Duchenne muscular dystrophy Capricor Heads Back to FDA With Pivotal Results for DMD Cardiomyopathy Therapy Six months after receiving a surprise rejection due to what the FDA called “lack of substantial evidence of effectiveness,” Capricor’s cell therapy deramiocel showed significant benefits in upper-limb function and slowed decline in cardiac function in a Phase III trial. December 3, 2025 · 3 min read · Heather McKenzie Read more Ultragenyx times two: Pair of gene therapies up for approval Q3 is shaping up to be crucial for Ultragenyx, with two gene therapies lined up for FDA verdicts: DTX401 for glycogen storage disease type 1a (GSD1a), set for Aug. 23 , and UX111 for Sanfilippo syndrome type A, due Sept. 19 . The upcoming decision date is especially pressing for UX111, which has already been rejected once. The FDA declined to approve the gene therapy in July 2025, citing manufacturing issues. Ultragenyx emphasized at the time that the denial was due to reasons “not directly related to the quality of the product.” UX111 uses an adeno-associated virus (AAV) vector to deliver a functioning copy of the SGSH gene, which in patients with Sanfilippo syndrome type A is mutated, giving rise to a broad array of neurological and developmental symptoms. Around 1 in every 50,000 to 250,000 people globally have one of the four types of Sanfilippo syndrome, with A being the most common. Meanwhile, GSD1a is caused by pathogenic mutations to the G6PC gene, produces an enzyme, G6Pase, that plays a role in the release of glucose from its stores. A deficiency in this activity can lead to severe hypoglycemia in between meals, as well as other complications that make the condition life-threatening. Patients with GSD1a are treated with cornstarch, which serves as an exogenous source of glucose. DTX401 works by stabilizing the expression and activity of G6Pase. In Phase 3 development, the gene therapy led to a “significant and clinically meaningful” decrease in patients’ need for daily cornstarch intake, while also minimizing hypoglycemia episodes, according a company news release in February . Biogen, Eisai ride out 3-month delay for starter dose of Leqembi Iqlik After extending its review for 3 months , the FDA set a decision date of Aug. 24 for Eisai and Biogen’s application for the use of an under-the-skin formulation of their Alzheimer’s disease therapy Leqembi to initiate patients onto the treatment. The FDA has already signed off on a subcutaneous version of Leqembi, now marketed as Leqembi Iqlik. However, the September 2025 approval only allows the use of the injection for maintenance treatment—that is, patients should first have completed 18 months of intravenous Leqembi. A nod for Leqembi Iqlik in the induction setting would make it the first anti-amyloid therapy for at-home dosing throughout the entire treatment journey, the companies said in a January news release . At the J.P. Morgan Healthcare Conference that same month, CEO Chris Viehbacher acknowledged that a nod is “going to be extremely important” and would help Leqembi compete with Eli Lilly’s Kisunla, which is infused monthly, versus Leqembi’s biweekly schedule. Kisunla’s current convenience edge “is going to go away once we have a subcutaneous formulation,” Viehbacher said. Nuvalent nears NSCLC ruling for drug that attracted GSK buyout Fresh off its $10.6 billion acquisition agreement with GSK, Nuvalent is nearing a potential approval for its ROS1 inhibitor zidesamtinib for the treatment of non-small cell lung cancer (NSCLC). The FDA’s verdict is expected on or before Sept. 18. Nuvalent is backing its application with data from the Phase 1/2 ARROS-1 study. A readout in September 2025 showed that zidesamtinib elicited a 44% objective response rate in patients who had undergone prior treatment with a tyrosine kinase inhibitor. This figure improved to 51% when focusing on those who had only one previous line of treatment. Zidesamtinib was one of the two stars of last month’s GSK takeover, alongside the ALK blocker neladalkib, also being proposed for NSCLC. The pharma believes both molecules have “multi-blockbuster potential,” according to GSK’s June 9 announcement . Truist Securities in a note the same day projected combined peak revenues for the two drugs to hit $3.5 billion. Mergers & acquisitions GSK pays $10.6B for cancer biotech Nuvalent in industry’s second-biggest buy of the year Nuvalent Bio is GSK’s third big-ticket purchase this year, after the pharma dropped $2.2 billion in January for RAPT Therapeutics and $950 million in February for 35Pharma. June 9, 2026 · 2 min read · Tristan Manalac Read more Scholar Rock gives muscle atrophy drug another try Capping off this list is Scholar Rock’s myostatin blocker apitegromab, which the biotech is proposing for the treatment of spinal muscular atrophy. The FDA’s target action date is September 30. Apitegromab is a monoclonal antibody that inhibits the activation of myostatin, a protein that works to negatively regulate the growth of muscles. By blocking myostatin, apitegromab improves muscle mass and strength, as well as overall patient function and quality of life, according to Scholar Rock’s website . Scholar Rock bore this out in the Phase 3 SAPPHIRE study, which in October 2024 showed significant motor improvements in patients treated with the antibody versus placebo. In September 2025, however, the FDA handed Scholar Rock a rejection , flagging violations at a Novo Nordisk–owned manufacturing site where apitegromab was supposed to be produced. Other applications have also been tripped up by this particular plant, including Regeneron’s high-dose Eylea . In March, Scholar Rock refiled its package for apitegromab, noting that while it will continue to engage the Novo facility, the biotech will also enlist a second fill-finish facility in the U.S. to “strengthen the apitegromab supply chain.”

莫德纳举办投资者日活动后股价异动，股价单日暴涨15%报68.85美元，创2024年9月以来最高收盘价，同期标普500指数仅上涨0.3%，股价后续累计翻倍有余。 莫德纳在投资者日披露三大核心动作：公布围绕三大商业板块的发展规划、计划扩建德国生产基地拓展欧洲市场，提出要转型为拥有多元产品线的生物制药企业，摆脱对新冠时期业绩的依赖。 公司公布的核心管线进展： 1. 流感疫苗mRNA-1010获FDA疫苗咨询委员会全票支持，PDUFA审批目标日为2026年8月5日，三期临床数据显示其在50岁以上人群防护效果优于现有上市流感疫苗； 2. 规划2027年启动首个体内原位CAR-T项目mRNA-6007的临床开发，首批适应症为B细胞介导的自身免疫疾病如系统性红斑狼疮，该技术可替代传统体外制备CAR-T方案，有望降本提效，帮助公司切入高价值治疗药物领域。 当前华尔街态度偏谨慎，杰富瑞维持该股持有评级，分析师共识评级并存持有、中性、卖出观点，本轮上涨属于市场对公司估值的重新定价博弈，尚未完全认可其转型逻辑，多数乐观预期已被部分定价。 后续核心关注节点： 2026年8月5日前FDA对mRNA-1010的审批结果 验证流感疫苗能否形成可持续商业增长曲线 2027年mRNA-6007按规划启动临床的相关进展 当前风险点为当前股价已提前透支尚未落地的业绩验证，所有新业务均无法短期贡献营收，流感疫苗可观营收预计2027年才会显现。