Translate Bio targets Q2 data drop on nebulized mRNA therapy

Lung model
The phase 1/2 study is the first trial to administer a mRNA therapeutic to the lung using nebulizers(Robina Weermeijer / Unsplash)

Translate Bio is aiming to deliver data on the first clinical-stage nebulized mRNA therapy early in the second quarter. The biotech set the target after the phase 1/2 cystic fibrosis clinical trial completed enrollment and dosing for the interim analysis.

COVID-19 enabled BioNTech and Moderna to validate the effectiveness of mRNA in the prevention of infectious diseases last year but other applications of the technology remain unproven. Translate Bio is seeking to push the field forward in another area through a phase 1/2 clinical trial that is testing a drug, MRT5005, that encodes for the CFTR gene that underpins cystic fibrosis.

The phase 1/2 study is the first trial to administer an mRNA therapeutic to the lung using nebulizers and the first to give multiple doses to treat a chronic genetic disease. Translate Bio will share a look at how participants are performing early in the second quarter.

The interim analysis will cover participants in the 8, 12 and 16 mg multiple-ascending dose groups and the 20 mg single-ascending dose cohort. Translate Bio is continuing to recruit participants in other dose cohorts as it seeks to show the effect of MRT5005 on the lung function of adult cystic fibrosis patients who have protein production or processing mutations. The other arms include a 24 mg group and a cohort that will receive five consecutive daily doses of 4 mg. 

MRT5005 will need to excel to make an impact. Vertex has transformed cystic fibrosis care in recent years but there remain opportunities to improve on the available options. Translate Bio is pitching MRT5005 as a way to treat cystic fibrosis regardless of the underlying genetic mutation. Vertex’s triple combination therapy cystic fibrosis drug Trikafta is limited to the 90% of patients with at least one F508del mutation in the CFTR gene.