Moderna has entered into a partnership with Italian pharmaceutical company Recordati to develop an mRNA therapy for propionic acidemia, a rare metabolic disorder. The agreement, announced Friday, is valued at up to $160 million and centers on Moderna’s investigational therapy mRNA-3927.
Propionic acidemia prevents individuals from processing certain proteins and fats, resulting in a dangerous buildup of acids in the body. Symptoms typically appear within days of birth and can include poor feeding, weak muscle tone, vomiting, seizures, coma, or even death.
Moderna’s mRNA-3927 aims to address the condition by encoding two enzymes that are lacking in people with the disorder. According to results from a Phase I/II trial published in Nature in 2024, the therapy led to a 70% reduction in the risk of metabolic decompensation among eight participants over 12 months. However, adverse events related to treatment were reported by 94% of patients. Moderna stated that interim data indicated early signs of clinical benefit and infrequent side effects severe enough to stop treatment.
Under the terms of the deal, Moderna will continue leading clinical development and manufacturing for mRNA-3927. Recordati will pay $50 million upfront and could make up to $110 million more in milestone payments tied to development and regulatory progress. The Italian company will also take charge of commercialization efforts and pay royalties on future sales.
"The American biotech will continue to lead clinical development and manufacturing of the asset. Recordati will pay $50 million upfront plus up to $110 million in near-term development and regulatory milestone payments, plus royalties on future sales. The Italian company will lead commercialization."
This collaboration comes as Moderna faces challenges due to declining COVID-19 vaccine sales and slow uptake of other products. The company has implemented cost-cutting measures as it works toward breaking even by 2028.
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