Moderna has designed mRNA-3927 to instruct the body to restore the missing or dysfunctional proteins, which cause PA.
PA is a rare, life-threatening and inherited a metabolic disorder that may result due to the deficiency in PCC, an enzyme crucial for metabolism. The deficiency may lead to a toxic buildup of acids in the body.
According to the company, the only better treatment for severely affected individuals is a liver transplant, which replaces the deficient PCC enzyme.
The company intends to commence an open-label, multi-centre and dose-escalation Phase 1/2 trial of multiple ascending doses of mRNA-3927 in primarily paediatric patients with PA in the US and Europe.
The assessment of safety and tolerability of mRNA-3927 administered via IV infusion, characterize the pharmacokinetic profile of mRNA-3927 and assess the pharmacodynamic response as evaluated by changes in plasma biomarkers are the objectives of the study.
It includes two mRNAs that encode for the alpha and beta subunits of the mitochondrial enzyme propionyl-CoA carboxylase (PCC), encapsulated within Moderna’s lipid nanoparticle (LNP).
The company already secured granted orphan drug and rare paediatric disease designations from the FDA and orphan designation from the European Medicines Agency (EMA) for the mRNA-3927.
Moderna chief medical officer Dr Tal Zaks said: “Fast Track designation underscores the urgent need for a therapy that treats the underlying cause of propionic academia.
“We are preparing to initiate a Phase 1/2 clinical study of mRNA-3927 to continue learning about the potential for this investigational therapy to restore enzyme activity in patients with propionic acidemia.”
Moderna is involved in the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases and cardiovascular diseases.
In August this year, Moderna secured fast track designation from the US Food and Drug Administration (FDA) for its investigational Zika vaccine, mRNA-1893.
At present, the mRNA-1893 vaccine is being assessed in a phase 1 study to prevent Zika virus infection in healthy adults.