Moderna Advances Rare Disease Pipeline Amid FDA Vaccine Debate and Cost Reductions
Moderna partners with Recordati on propionic acidemia therapy for up to $160 million, while FDA Commissioner defends mRNA vaccine support following funding cuts and regulatory challenges.
Moderna announced a strategic collaboration with Recordati to advance its investigational propionic acidemia therapeutic, mRNA-3927, through final clinical development stages and global commercialization upon approval. The company will receive up to $160 million in upfront and near-term development and regulatory milestones, in addition to commercial and sales milestones and tiered royalties on net sales.
Under the terms of the agreement, Moderna will receive an upfront payment of $50 million and up to an additional $110 million in near-term development and regulatory milestones. Moderna will continue to lead clinical development of mRNA-3927 through approval and manufacturing, while Recordati, based in Milan, Italy, will lead commercialization. The transaction is subject to customary closing conditions, including U.S. antitrust clearance which is expected within 30 days from the relevant filing.
mRNA-3927 is currently being evaluated in a registrational study that has reached target enrollment. The Company expects a potential data readout in 2026. The investigational novel mRNA-based therapeutic agent is composed of two mRNAs encoding for normal human PCCA and PCCB subunits. Intravenous administration of mRNA-3927 is intended to restore functional PCC enzymes in patients with PA.
Propionic acidemia is a rare, serious, inherited metabolic disorder with significant morbidity and mortality, affecting 1 in 100,000-150,000 individuals worldwide. PA is caused by pathogenic variants in the propionyl-coenzyme A carboxylase α or β subunits, leading to PCC deficiency and subsequent accumulation of toxic metabolites. PA is characterized by recurrent life-threatening metabolic decompensation events and multisystemic complications. Currently, there are no effective therapies for PA that target the underlying root cause of the disease.
Interim data from a first-in-human, phase 1/2, open-label, dose optimization study and extension study evaluating the safety and efficacy of mRNA-3927 indicate early signs of potential clinical benefit and demonstrate that mRNA-3927 has infrequent treatment-limiting side effects.
The U.S. Food and Drug Administration Commissioner defended the agency's position and record on mRNA vaccines on February 23, stating that the FDA supports mRNA vaccines but taxpayers should not foot the bill. This referred to several Health Department moves last year to cut funding for mRNA vaccine research. The commissioner said the department cut funding for the development of mRNA vaccines because companies that make the drugs can afford their own research, noting that companies that made mRNA vaccines made over $50 billion.
The FDA has approved two messenger RNA vaccines against RSV, one from Moderna and the other from GSK. The commissioner said the government should fund research in areas that are underfunded, such as bespoke conditions and rare diseases, conditions that have been in the blind spots of modern medicine.
The U.S. Department of Health and Human Services made an August decision to wind down mRNA vaccine development activities under its biomedical research unit. That followed the HHS's termination in May of more than $700 million in funding for Moderna to develop an mRNA vaccine to protect against pathogens with pandemic potential including bird flu, which had touched off a major outbreak in dairy cows that was infecting workers.
Moderna reported fourth quarter 2025 revenue of $678 million, on the higher end of the Company's prior expectations, driven primarily by COVID vaccine sales. Product sales were $264 million in the U.S. and $381 million in international markets. Fourth quarter revenue decreased 30% compared to the same period in 2024, primarily reflecting lower COVID vaccine sales volume compared to the prior-year period.
Total revenue for the full year 2025 was $1.9 billion, a 40% decrease compared to 2024, with the majority generated from COVID vaccine sales, along with $126 million of other revenue. U.S. revenue totaled $1.2 billion, while revenue from international markets was $745 million.
Research and development expenses for the fourth quarter of 2025 were $775 million, a 31% decrease compared to the same period in 2024. The decrease was driven primarily by lower clinical development and manufacturing costs, reflecting the wind-down of large Phase 3 respiratory programs, continued portfolio prioritization and cost discipline across the organization. The company lowered its annual operating expenses by approximately $2.2 billion in 2025, significantly surpassing its cost-reduction targets.
Net loss was $826 million for the fourth quarter of 2025, compared to net loss of $1.1 billion for the fourth quarter of 2024. Loss per share was $2.11 for the fourth quarter of 2025, compared to loss per share of $2.91 for the fourth quarter of 2024. Full-year net loss was $2.8 billion with diluted loss per share of $7.26.
The Company announced its influenza vaccine filing was accepted for regulatory review in the EU, Canada and Australia. The Company received a Refusal-to-File letter from the U.S. FDA and has requested a Type A meeting to understand the path forward. The Company also announced its Norovirus Phase 3 trial is fully enrolled with a data readout expected in 2026, and announced full enrollment of Phase 2 intismeran autogene trial in muscle invasive bladder cancer.
Moderna is entering the year with three approved products, Spikevax, mNEXSPIKE and mRESVIA, with seasonal vaccines expected to deliver up to 10% revenue growth in 2026. The Company recently announced long-term agreements with Mexico and Taiwan for respiratory vaccines, received regulatory approvals in Canada and Australia for mNEXSPIKE, and the strain-updated Spikevax vaccine was authorized in the UK for use in the spring vaccination campaign.