Moderna recently confirmed that its Phase I/II study examining candidate mRNA-3927 is progressing. It also shared that data showed a decrease in the number of metabolic decompensation events (MDEs) in trials of mRNA-3927, and regulators seemed to be supportive of MDE as a primary endpoint for the study. Additionally, Moderna stated that all eligible participants in its study elected to continue to the Open Label Extension study. If approved, the drug candidate is set to generate $278 million in 2028, estimates GlobalData.
mRNA-3927 targets a rare genetic disorder, propionic acidemia, in which patients are unable to process certain parts of proteins and lipids properly. According to GlobalData’s Pharma Intelligence Center, mRNA-3927 is one of five non-vaccine mRNA therapeutics currently in clinical trial development. The other four non-vaccine mRNA therapeutics are as follows: BNT-141 by BioNTech, targeting ovarian cancer, bile duct cancer, adenocarcinoma of the gastroesophageal junction, colorectal cancer, pancreatic cancer, gastric cancer, and solid tumors; LUNARGSD3/UX053 by Ultragenyx, targeting glycogen storage disorder type III, also known as Cori’s Disease; mRNA-3705, Moderna’s second candidate, for methylmalonic acidemia; and OTX-2002 by Omega Therapeutics for hepatocellular carcinoma and solid tumors.
According to GlobalData’s analyst consensus forecast, OTX-2002 is projected to have the highest revenue by 2028, with sales of $1.6 billion during that year. The sales of all five candidates are forecast to reach over $2 billion by 2028.
Sarah Bundra, Analyst at GlobalData, comments, “mRNA therapeutics have received increased amounts of attention and funding ever since mRNA vaccines proved effective in curbing the spread of the COVID-19 pandemic. Now, researchers are exploring mRNA therapeutics’ use in other disease spaces.”
mRNA is a single-stranded ribonucleic acid that is transcribed from a strand of DNA. It carries the coding information for protein synthesis. mRNA therapeutics involve the delivery of in vitro transcription of mRNA into a target cell, where cellular machinery is then able to translate the mRNA into a functional protein. These proteins can help the immune system prevent or treat certain diseases. Propionic acidemia is caused by the deficiency of propionyl-CoA carboxylase, which converts propionyl-CoA to methylmalonyl-CoA. Moderna’s candidate alleviates the condition by activating propionyl-coenzyme A carboxylase.
Bundra concludes, “Propionic acidemia is a rare but devastating genetic disorder. If Moderna’s mRNA-3927 proves to be successful, the therapy will provide a potential solution for a patient population facing a scarcity of options.”