Moderna Enters Strategic Collaboration with Recordati for Rare Metabolic Disorder

Rare Daily Staff

Moderna said it entered into a strategic collaboration with Italy-based Recordati to advance Moderna’s experimental therapeutic for the metabolic disorder propionic acidemia, mRNA-3927.

Under the agreement, Moderna will continue to lead the clinical development of mRNA-3927 through approval, and Recordati will lead commercialization. Moderna will receive an upfront payment of $50 million and up to an additional $110 million in near-term development and regulatory milestones, as well as commercial and sales milestones, and tiered royalties on net sales.

“Recordati brings deep rare disease commercial expertise and an established global commercial infrastructure in propionic acidemia that will help us accelerate the benefit of mRNA-3927 upon approval,” said Stéphane Bancel, CEO of Moderna.

Propionic acidemia is a rare, serious, inherited metabolic disorder associated with significant morbidity and mortality. The condition is caused by pathogenic variants in the propionyl-coenzyme A carboxylase (PCC) alpha or beta subunits (PCCA and PCCB genes, respectively), leading to PCC deficiency and accumulation of toxic metabolites. It is characterized by recurrent, life-threatening metabolic decompensation events and multisystem complications. There are currently no effective therapies for the disorder that target the underlying root cause.

mRNA-3927 is an experimental, novel mRNA-based therapeutic composed of two mRNAs encoding normal human PCCA and PCCB subunits. Intravenous administration of mRNA-3927 is intended to restore functional PCC enzymes in patients with propionic acidemia.

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 show that the therapy has infrequent treatment-limiting side effects.

“Propionic acidemia is a serious rare disease with a significant unmet medical need due to the lack of disease-modifying treatment options to date,” said Rob Koremans, CEO of Recordati. “Their experience in applying innovative mRNA technology, combined with our experience in rare metabolic disorders and strong established commercial infrastructure, positions us well to advance this potential therapy together to serve patients.”