Moderna, a US-based biopharmaceutical company, reportedly announced that the first patient in its Phase 1/2 trial has been enrolled to help evaluate the tolerability and safety of intensifying doses of mRNA-3704 given via intravenous infusion for patients suffering from isolated MMA (methylmalonic acidemia) due to MUT deficiency. This is the company's first rare disease program to start clinical study enrollment.

The enrolled patient has entered in an observational period before the treatment, which evaluates the baseline disease of the patient before initiating the therapy period. The LNP (lipid nanoparticle) formulation utilized for mRNA-3704 is also used in mRNA-1944 (the company's chikungunya antibody program), which recently showed positive interim Phase 1 outcomes.

The Phase 1/2 open-label trial is created to assess the tolerability and safety of approximately four scaling dose levels of mRNA-3704 given via intravenous infusion among patients one year or older with isolated MMA caused due to hMUT (methylmalonyl-CoA mutase) deficiency with increased plasma methylmalonic acid, a key metabolite which accumulates in disorder.

Chief Medical Officer, Moderna, Tal Zaks, M.D., Ph.D., stated that this trial would provide vital information about the capability of mRNA approach for replacement of the deficient enzyme in patients suffering from MMA, which presently has no authorized medical treatment or investigational medicines that can address the underlying cause.

Zaks further added that as the first of the company's rare disease programs to start clinical trial enrollment, the company looks forward to hearing from the enrolled patients and families in the company's clinical study as it investigates and advances an mRNA platform that might help treat this challenging condition.



The U.S. FDA (Food and Drug Administration) has granted mRNA-3704 with Orphan Drug, Fast Track, and Rare Pediatric Disease designation. mRNA-3704 has also received orphan designation by the EMA (European Medicines Agency). Moderna is presently planning to start its several sites outside the United States and thus far got MHRA (Medicines and Healthcare products Regulatory Agency) approval in U.K.



 



Source credit: https://investors.modernatx.com/news-releases/news-release-details/moderna-announces-first-patient-enrolled-phase-12-study-mrna