Translate Bio (NASDAQ:TBIO) reported interim results from its Phase 1/2 clinical trial of MRT5005 in patients with cystic fibrosis (CF).
MRT5005 is inhaled to deliver the gene that encodes functional CF transmembrane conductance regulator (CFTR) protein to cells in the lungs. The study is evaluating single and multiple ascending doses (SAD and MAD) of the drug candidate.
In the SAD portion of the clinical trial, 12 patients were assessed one month after a single dose of treatment. MRT5005 was well-tolerated and patients demonstrated improved lung function, measured using percent predicted forced expiratory volume in one second (ppFEV1).
“Effectively delivering the desired mRNA sequence is an essential step to producing functional CFTR proteins,” Dr. Steven Rowe, the trial’s principal investigator, said in a statement.
“While some variability in ppFEV1 from day-to-day in CF patients is expected in a trial of this size, we believe that the observed improvements in ppFEV1 from baseline and the time course of the effect support a CFTR-related mechanism and may suggest that MRT5005 can enable the production of functional protein,” he added.
Data from the MAD portion of the trial are expected in 2020.
