Moderna 2022 Priorities and Capital Allocation
Propionic acidemia (PA) ongoing in Phase 1/2 study
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mRNA-3927 Phase 1/2 study
Propionic Acidemia (PA) is a rare metabolic disorder
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PA is characterized by a deficiency of propionyl-CoA
carboxylase (PCC), an enzyme involved in the
breakdown (catabolism) of several chemical
"building blocks" (amino acids) of proteins
As a result, harmful compounds can build up to toxic.
levels in the body
Leads to serious health problems, including recurrent
episodes of life-threatening metabolic
decompensation events (MDES)
Moderna's mRNA therapy for PA (mRNA-3927) encodes
for two protein subunits (PCCA and PCCB) that form the
deficient enzyme (PCC)
Slide 27
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Adaptive trial design; enrolling participants >1 years
old with PA in the US, UK and Canada
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Patients receive 1 dose of mRNA-3927 every 2 or 3
weeks for 10 doses
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First cohort is fully enrolled and we are enrolling
patients into additional cohorts
All five patients eligible for the Open Label Extension
(OLE) study have elected to participate
Total of 75 doses have been administered across
the Phase 1/2 study and OLE study
Study is evaluating safety, PK/PD, clinical events
(incl. MDES) and biomarkers
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