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Moderna is partnering with Italy’s Recordati for up to $160 million to develop a rare disease mRNA therapy that will deliver pivotal results later this year.
The deal,announced Friday, involved Moderna’s mRNA-3927, which is under development for propionic acidemia (PA). People with the rare metabolic disorder are unable to process certain proteins and fats, leading to a buildup of acids in the body that can cause life-threatening attacks. The condition is usually revealed within days of birth, causing poor feeding, weak muscle tone and vomiting, and in more serious cases, seizures, coma and death.
To address the disease, Moderna has been advancing mRNA-3927, an mRNA-based therapy that seeks to encode two enzymes that are deficient in people with the disorder. In a Phase I/II trial published inNaturein 2024, the therapy showed a 70% reduction in the risk of metabolic decompensation in eight participants during the 12-month treatment period. However, 94% of patients reported adverse events related to treatment. Moderna said that interim data revealed early signs of clinical benefit and infrequent treatment-limiting side effects.
Now, Moderna is teaming up with Recordati for support in advancing mRNA-3927. 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.
The deal comes amid tough times for Moderna, driven by declining COVID-19 vaccine sales and slow uptake of other products. The company has been on an aggressive cost-cutting push in an effort tobreak even by 2028. While Moderna reported early Q4 results in the upper end of target revenue, analysts said investors still want to see more reductions to support the break-even goal.