Moderna has reported interim data from a Phase I/II clinical trial of its investigational mRNA therapy, mRNA-3927, for the treatment of propionic acidemia (PA), a rare, serious, inherited metabolic disorder.
Designed to assess the pharmacodynamics, pharmacokinetics, and safety of mRNA-3927, the multicenter, open-label study has enrolled a total of 16 participants aged one year and older with genetically confirmed PA.
An initial dose of 0.3mg per kilogram of mRNA-3927 was administered intravenously every three weeks to the participants with subsequent doses given every two weeks thereafter.
Determining the safety and tolerability are the primary outcomes while evaluation of potential plasma biomarkers, pharmacology, and the frequency and duration of metabolic decompensation events are considered as secondary and exploratory outcomes of the study.
Encouraging early signs of dose-dependent pharmacology and potential clinical benefit were observed during the study.
Additionally, mRNA-3927 was well-tolerated at the doses administered to the participants with PA.
Out of the total, 11 participants have completed the study and enrolled in the open-label extension study while five participants for a period of one year were treated with mRNA-3927.
So far more than 280 mRNA-3927 doses were administered across both trials.
Moderna senior vice-president and therapeutics and oncology development head Kyle Holen said: “We continue to observe encouraging results with mRNA-3927 as we enter the dose-expansion phase, where we will further assess safety, efficacy, and determine the recommended dose for future clinical studies.
“This is the first clinical trial reporting results of an mRNA therapeutic for intracellular protein replacement, and we currently have more than 13 patient-years of experience to date.”