Press release
Phase 1/2 Interim Data on Moderna's mRNA-3927, an Investigational mRNA Therapy for Propionic Acidemia (PA), Presented at the 2023 ASGCT Annual Meeting
First clinical trial reporting results of an mRNA therapeutic for intracellular protein replacementTo date, mRNA-3927 has been generally well-tolerated at the
About this update from Moderna, Inc.
[{"type":"text","content":"First clinical trial reporting results of an mRNA therapeutic for intracellular protein replacementTo date, mRNA-3927 has been generally well-tolerated at the doses administered, with encouraging early signs of dose-dependent pharmacology and potential clinical benefitsNo occurrence of dose-limiting toxicities or study discontinuations due to drug-related treatment-emergent adverse events (TEAEs)More than 280 doses of mRNA-3927 were administered. Five patients have more than a year of dosingPhase 1/2 trial advances to the dose-expansion phaseCAMBRIDGE, MA / ACCESSWIRE / May 19, 2023 / Moderna,Inc. (NASDAQ:MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, reported on interim data from the Phase 1/2 trial of mRNA-3927, an investigational mRNA therapy for propionic acidemia (PA), presented at the 2023 American Society of Gene + Cell Therapy (ASGCT) Annual Meeting.The ongoing global Phase 1/2 clinical trial (ClinicalTrials.gov Identifier: NCT04159103) is a multicenter, open-label study designed to assess the safety, pharmacodynamics, and pharmacokinetics of mRNA-3927 in participants aged 1 year and older with genetically confirmed PA. The trial utilized a dose-escalation approach to evaluate the intravenous administration of mRNA-3927.The initial dosing regimen was 0.3 mg/kg administered intravenously every three weeks; subsequent doses were administered every two weeks. Participants who complete the dose optimization trial (10 doses) are eligible to continue treatment in an open-label extension study (NCT05130437). The primary outcomes of the trial are safety and tolerability, while secondary and exploratory outcomes include pharmacology, evaluation of potential plasma biomarkers, and the frequency and duration of metabolic decompensation events (MDEs). mRNA-3927 has been well-tolerated at the doses administered, with encouraging early signs of dose-dependent pharmacology and potential clinical benefit.To date, a total of 16 participants have received doses of mRNA-3927 across five dose cohorts. Of these, 11 participants completed the study and enrolled in the open-label extension study, and five participants were treated with mRNA-3927 for over one year. Following treatment initiation with mRNA-3927, most participants who had reported MDEs in the 12 months prior to dosing had either a lower ...