Moderna Announces First Patient Dosed in Phase 1/2 Study of mRNA-3705 for Methylmalonic Acidemia
mRNA-3705 is Moderna’s second rare disease program to enter clinical studies
mRNA-3705 granted Orphan Drug and Rare Pediatric Disease designation by the
MMA associated with significant mortality and morbidity; there are no approved therapies
“We would like to thank
“We are delighted to have been able to treat the first patient in the world with this new medicine here in Birmingham,”
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.
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.
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
In 10 years since its inception,
Moderna’s mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Today, 23 development programs are underway across these therapeutic areas, with 15 programs having entered the clinic.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: the Company’s development of an investigational therapeutic for methylmalonic acidemia (MMA) (mRNA-3705); the conduct of clinical trials for mRNA-3705; and the Company’s efforts to develop therapies for other rare diseases, including propionic acidemia, glycogen storage disease type 1a and phenylketonuria. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those other risks and uncertainties described under the heading “Risk Factors” in Moderna’s most recent Annual Report on Form 10-K filed with the
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