Preliminary results from a Phase 1/2 clinical trial, RESTORE-CF, evaluating single and ascending doses of Translate Bio’s (NASDAQ:TBIO) inhaled mRNA therapeutic MRT5005 in cystic fibrosis (CF) patients showed a positive effect at certain doses.
Data from the single ascending dose (SAD) portion in 12 CF patients (participants received either 8 mg, 16 mg, 24 mg or placebo) showed that the 16 mg dose was the most effective in terms of improved lung function (ppFEV1) with a mean maximum increase from baseline of 15.7%. All three subjects in this arm experienced maximum increases of 11.1 – 22.2%. The treatment effect in the 8 mg arm was not much better than placebo while the 24 mg arm was better than placebo but not as significant as the 16 mg arm.
On the safety front, the number of treatment-related adverse events in the three arms were 28, 25 and 33, respectively, compared to 11 in the control arm. All were mild or moderate. The most common were cough and headache.
The company plans to add a 20 mg dose cohort to the SAD portion. Preliminary data from this arm and the multiple ascending dose portion should be available in 2020.
Additional results from this initial dataset will be presented at the North American Cystic Fibrosis Conference in Nashville, TN, October 31 – November 2.
Orphan Drug-tagged MRT5005 is designed to address the underlying cause of CF by delivering mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to the lung epithelial cells through nebulization.
Management will host a conference call today at 8:00 am ET to discuss the results.
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