Clinical-stage biotechnology firm Moderna has enrolled the first patient in the Phase I/II trial of mRNA-3704 in methylmalonic acidemia (MMA) patients.
The mRNA-3704, which is the company’s first rare disease programme to enter clinical trials, will be administered through intravenous infusion in patients with isolated MMA due to mitochondrial enzyme methylmalonic-CoA mutase (MUT) deficiency.
The open-label study will evaluate the safety and tolerability of up to four mRNA-3704 ascending dose levels.
Moderna noted that the patients will take part in a pre-dosing observational period before moving to treatment period and a follow-up period after treatment withdrawal. the observational period helps analyse the patient’s baseline disease.
According to the company, the lipid nanoparticle (LNP) formulation used for mRNA-3704 is also used in Moderna’s chikungunya antibody programme (mRNA-1944) and propionic acidemia (mRNA-3927). Moderna’s chikungunya antibody programme has reported positive results for the interim Phase I study.
Moderna chief medical officer Tal Zaks said: “This study will provide important information about the potential of an mRNA approach to replace the deficient enzyme in patients with MMA, which currently has no approved medical therapy or investigational medicines that address the underlying cause.
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“As the first of our rare disease programmes to begin clinical trial enrolment, we look forward to learning from the patients and families in our clinical trial as we investigate and advance an mRNA platform that may help treat this challenging condition.”
Moderna’s mRNA-3704 has received fast track, orphan drug, and rare paediatric disease designation from the US Food and Drug Administration (FDA).
The drug has also received orphan designation by the European Medicines Agency (EMA).
Moderna plans to start several sites outside the US. The company has secured approval from the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK so far.
In September 2019, Moderna reported positive interim results from the Phase I clinical trial of its mRNA-1647 prophylactic vaccine for cytomegalovirus (CMV).