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Moderna Launches Trial of mRNA Vaccine for Methylmalonic Acidemia

Moderna Launches Trial of mRNA Vaccine for Methylmalonic Acidemia content piece image
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Moderna, Inc., (Nasdaq: MRNA) a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, has announced the first patient has been dosed in the Phase 1/2 study evaluating the safety and tolerability of mRNA-3705, its investigational mRNA therapeutic for methylmalonic acidemia (MMA), administered via intravenous infusion in patients with isolated MMA due to MUT deficiency.

“We would like to thank Dr. Santra and the whole team in Birmingham for their efforts and collaboration to achieve this milestone moment of dosing the first MMA patient with our mRNA therapeutic,” said Ruchira Glaser, M.D., Sr. Vice President and Therapeutic Area Head, Rare Disease, Autoimmune & Cardiovascular. “This is another step forward in Moderna’s mission to deliver on the promise of mRNA science to create a new generation of transformative medicines for patients.”

“We are delighted to have been able to treat the first patient in the world with this new medicine here in Birmingham,” Saikat Santra, M.D., Pediatric Inherited Metabolic Medicine Consultant, Clinical Inherited Metabolic Disorders at Birmingham Women’s and Children’s NHS Foundation Trust. “We sincerely hope that it brings this brave patient, and many more like them, a brighter future free of the restrictions of this terrible disease.”

Methylmalonic acidemia is a rare, life-threatening, inherited metabolic disorder that is most commonly (approximately 60% of cases) caused by a deficiency in the mitochondrial enzyme methylmalonic-CoA mutase (MUT). This deficiency can lead to metabolic crises due to a toxic buildup of acids in the body and progresses into multi-organ disease. As a result, MMA is associated with significant mortality and morbidity, and there are no approved therapies. Standard of care includes dietary and palliative measures. Currently, liver or combined liver and kidney transplant is the only effective treatment.

Moderna recognizes the impact of rare diseases on patients and their families, particularly when the disease lacks effective treatment options. In addition to MMA, Moderna is working to advance mRNA therapeutics that restore the activity of missing enzymes responsible for other rare diseases, such as propionic acidemia (PA), glycogen storage disease type 1a (GSD1a) and phenylketonuria (PKU). Moderna has active clinic programs in five different therapeutic areas: infectious disease, oncology, cardiovascular, rare disease and autoimmune disease.

About the Phase 1/2 Study

The Phase 1/2 study, called the “Landmark study,” is an adaptive, open-label study is designed to evaluate the safety and tolerability of up to five different dosing regimens of mRNA-3705 administered via intravenous infusion in patients one year and older with isolated methylmalonic acidemia due to methylmalonyl-CoA mutase (hMUT). Upon establishment of an optimized dose based on safety and pharmacological data, additional patients maybe enrolled in an optional expansion cohort.

About mRNA-3705

mRNA-3705 is designed to instruct the body to restore the missing or dysfunctional proteins that cause MMA and consists of mRNA encoding human MUT, the mitochondrial enzyme commonly deficient in MMA, encapsulated within Moderna’s proprietary lipid nanoparticle (LNP). mRNA-3705 uses the same proprietary LNP formulation as the Company’s antibody against chikungunya virus (mRNA-1944) and propionic acidemia (mRNA-3927) programs. mRNA-3705 has been granted Orphan Drug and Rare Pediatric Disease designation by the U.S. Food and Drug Administration (FDA). Moderna owns worldwide commercial rights to mRNA-3705.

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