Moderna has announced it is entering a strategic collaboration with Recordati to advance Moderna's investigational propionic acidemia (PA) therapeutic, mRNA-3927, through the final stages of clinical development and, upon approval, global commercialisation.
Recordati, based in Milan, Italy, is an international pharmaceutical group that provides treatments across speciality and primary care and rare diseases, including 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 (PCC) α or β subunits (PCCA and PCCB genes, respectively), leading to PCC deficiency and subsequent accumulation of toxic metabolites.
PA is characterised by recurrent life-threatening metabolic decompensation events (MDEs) and multisystemic complications.
Currently, there are no effective therapies for PA that target the underlying root cause of the disease.
Through this agreement, Moderna will continue to lead the clinical development of mRNA-3927 through approval and Recordati will lead commercialisation.
"We are proud to partner with Recordati in a joint mission to improve the lives of people living with propionic acidemia," said Stéphane Bancel, CEO of Moderna.
"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."
"Propionic acidemia is a serious, rare disease with a significant unmet medical need due to the lack of disease-modifying treatment options to date. We look forward to partnering with Moderna," 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."
"We are encouraged by the clinical data and look forward to the pivotal readout expected in 2026. This deal strengthens our development portfolio and builds on our heritage in the metabolic field."
Under the terms of the agreement, Moderna will receive an upfront payment of $50m and up to an additional $110m in near-term development and regulatory milestones, in addition to commercial and sales milestones and tiered royalties on net sales.
The transaction is subject to customary closing conditions, including US 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 enrolment, with an expected data readout in 2026.