Moderna has out-licensed a rare disease drug candidate to Recordati, accepting $50 million upfront for an asset that could deliver registrational data this year.
Recordati, an Italian company that sells specialty and rare disease drugs around the world, has secured global rights to commercialize mRNA-3927. The candidate consists of two mRNA molecules encoding for subunits of an enzyme that malfunctions in people with propionic acidemia (PA), a rare disease driven by the buildup of toxic metabolites that trigger life-threatening metabolic decompensation events (MDEs).
Moderna began a phase 1/2 trial of the candidate in 2021. After seeing a 76% relative reduction in the risk of MDEs in the first part of the study, the biopharma started the pivotal portion of the study. The pivotal trial is now fully enrolled, putting Moderna on track to report data this year.
Earlier this month, Moderna named (PDF) PA as a growth driver that could come online in 2028. Yet the biopharma has decided to cash in now rather than commercialize mRNA-3927 itself. Moderna will continue to lead development of mRNA-3927, but Recordati will lead global commercialization efforts for the drug candidate if it wins approval.
In addition to the upfront fee, Moderna could receive up to $110 million in near-term development and regulatory milestones as mRNA-3927 advances to market. The deal also includes undisclosed commercial and sales milestones plus tiered royalties on annual net sales.
Recordati could become the first company with an approved PA treatment. Currently, management of the disease involves severe restriction of dietary protein intake. Liver transplants can allow patients to eat a less restricted diet without suffering MDEs, but better care options are needed.
Moderna has continued to invest in mRNA-3927 and another rare disease asset, methylmalonic acidemia (MMA) prospect mRNA-3705, as it has pulled back from other programs to cut costs. The assets were advanced by the time Moderna reconsidered its strategy in 2024, when President Stephen Hoge, M.D., told investors the company would defer new pivotal rare disease trials until completing its active projects.
With Moderna out-licensing the PA program, MMA candidate mRNA-3705 is now an outlier in a pipeline and portfolio dominated by oncology and infectious disease assets. The MMA program is less advanced than the PA candidate, with Moderna naming it among the early-stage pipeline investments it expects to mature in 2029 and beyond.