Italian drugmaker Recordati has reached a strategic partnership with Moderna to jointly develop and commercialize mRNA-3927, an investigational mRNA-based therapy aimed at treating propionic acidemia (PA), a rare inherited metabolic disorder.
Under the terms of the agreement, Recordati will make an upfront payment of $50 million to Moderna. The companies also agreed contingently on up to $110 million in additional near-term payments tied to development and regulatory milestones, in addition to a framework for tiered royalties on any future product sales.
The collaboration seeks to deliver what the partners describe as the first disease-modifying therapy for PA, a condition that affects roughly 1 in 100,000 to 150,000 people worldwide and is associated with life-threatening metabolic crises and multisystem complications.
mRNA-3927 is designed to restore activity of the propionyl-CoA carboxylase enzyme in patients with PA. The program has produced interim clinical results published in Nature that the partners say demonstrate early signs of clinical improvement and show few treatment-limiting adverse effects to date.
Target enrollment for the registrational study has already been reached, the companies noted, which could allow progression toward regulatory submission and a commercial launch if pivotal results and regulatory reviews proceed positively.
Pivotal clinical data for mRNA-3927 are expected by the end of 2026. Recordati has indicated it does not expect the collaboration to have a meaningful impact on EBITDA prior to a potential product launch.
The agreement pairs Moderna’s mRNA technology platform and development capabilities with Recordati’s global commercial infrastructure focused on rare diseases and its experience in metabolic disorder markets.
Summary
Recordati will pay $50 million up front to Moderna and may pay up to $110 million more in near-term development and regulatory milestones. The companies will share profits through tiered royalties if mRNA-3927 is approved and commercialized. Interim data indicate early clinical benefit with limited treatment-limiting safety signals, and pivotal data are anticipated by the end of 2026.