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“Despite significant advances in the treatment of patients with CF, there remains a critical unmet need for patients whose genetic mutations are considerednon-amenable to CFTR modulators,” said Ann Barbier, M.D., Ph.D., chief medical officer of Translate Bio. “mRNA therapy represents a potential new approach to treating CF, and we look forward to sharing the results from the multiple-ascending dose part of the trial next year.”
Dr. Barbier continued, “On behalf of the team at Translate Bio, I would also like to extend our most sincere gratitude to the investigators, the Cystic Fibrosis Foundation, and most importantly, the patients who are participating and have participated in this clinical trial.”
“These encouraging interim results represent a milestone in the mRNA development landscape as this is the first time an mRNA therapeutic has been evaluated for its potential to treat a genetic disease,” said Ronald Renaud, chief executive officer of Translate Bio.“Also, as the first inhaled mRNA therapeutic, these data indicate the potential of mRNA therapeutics for the treatment of lung diseases, and support the planned expansion of our earlier-stage programs based on this pulmonary delivery platform.”
Phase 1/2 Clinical Trial Results
Study Design and Baseline Characteristics Summary
The interim results summarize data from 12 adult patients with CF who received a single dose of either MRT5005 or placebo (3:1 randomization). Patients who received MRT5005 were assigned to one of three dose groups (8, 16 or 24 mg). Of the 12 patients, 11 had at least one copy of the F508del mutation and one patient did not have a F508del mutation and was considerednon-amenable to CFTR modulator treatment. Seven of the 12 patients were taking an approved CFTR modulator through screening, dosing andfollow-up.
Safety, Tolerability and Pharmacokinetic Summary
The most common adverse events through Day 29 were cough and headache. There were no treatment-emergent serious adverse events (SAEs). All treatment-emergent adverse events (TEAEs) were considered mild to moderate. Five patients, 3 of whom were in the 24 mg dose group, experienced transient, mild to moderate febrile reactions deemed related to study drug. These events occurred approximately4-10 hours post dosing, and were characterized by fever and symptoms such as headache, fatigue, chills or nausea, which were treated with medicines such as acetaminophen and nonsteroidal anti-inflammatory drugs (NSAIDs), or an anti-emetic. Symptoms resolved within 24 hours, and all patients were discharged from the study center on Day 2 as planned. In these five patients, low levels of mRNA and/or lipid were detected in the blood.