Moderna, Inc., a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for mRNA-3927, its investigational mRNA therapeutic for propionic acidemia (PA). The Company announced the open IND for mRNA-3927 on September 30, 2019.
“Propioic acidemia is caused by the inability of the body to breakdown certain proteins and fats which leads to the build-up of toxic chemicals. The disease is characterized by life-threatening illnesses in response to minor stressors, neurological dysfunction and cardiomyopathy,” said George Diaz, M.D., Ph.D., chief, division of medical genetics, Icahn School of Medicine at Mount Sinai Hospital. “Currently there are no approved therapies available that treat the underlying cause of this debilitating disease.”
“Fast Track designation underscores the urgent need for a therapy that treats the underlying cause of propionic acidemia,” said Tal Zaks, M.D., Ph.D., chief medical officer at Moderna. “We are preparing to initiate a Phase 1/2 clinical study of mRNA-3927 to continue learning about the potential for this investigational therapy to restore enzyme activity in patients with propionic acidemia.”
Fast Track is designed to facilitate the development and expedite the review of therapies and vaccines for serious conditions and fill an unmet medical need. Programs with Fast Track designation may benefit from early and frequent communication with the FDA, in addition to a rolling submission of the marketing application. The Company previously received Fast Track designation for its investigational Zika vaccine (mRNA-1893) and methylmalonic acidemia (MMA) (mRNA-3704) programs.
Moderna plans to initiate an open-label, multi-center, dose escalation Phase 1/2 study of multiple ascending doses of mRNA-3927 in primarily pediatric patients with PA in the United States and Europe. The objectives of this study are to evaluate the safety and tolerability of mRNA-3927 administered via IV infusion, characterize the pharmacokinetic profile of mRNA-3927 and assess the pharmacodynamic response as assessed by changes in plasma biomarkers.
PA and MMA are rare diseases that share similar disease pathology and are both typically treated by metabolic specialists. In order to characterize and describe the natural history of these disorders and identify potential clinical and biomarker endpoints, Moderna is conducting a global, multi-center, non-interventional observational study for patients with confirmed diagnosis of PA or MMA. More information about this study can be found at ClinicalTrials.gov. Moderna is currently recruiting patients with MMA for a Phase 1/2 study of mRNA-3704.