The U.S. Food and Drug Administration has given the go-ahead for MRT5005, Translate Bio’s lead candidate to treat the underlying cause of cystic fibrosis (CF), to be tested in a Phase 1/2 clinical trial. The company expects to begin dosing enrolled patients by mid-year.
MRT5005 is designed to address the underlying cause of CF, namely defects in the gene that produces the cystic fibrosis transmembrane conductance regulator (CFTR) protein. MRT5005 is a messenger RNA molecule that contains the blueprint, or instructions, for the proper production of a workng CFTR protein. It is also the first potential mRNA treatment to specifically target the lungs.
The therapy is delivered directly to the lungs in nebulized form, and intended to treat all CF patients regardless of the underlying mutation in their CFTR gene.
The FDA designated MRT5005 an orphan drug in 2015, a move that provides incentives to a company to develop therapeutics for rare diseases, or those affecting less than 200,000 people in the U.S.
Not yet open for patient enrollment, the Phase 1/2 clinical trial (NCT03375047) will be a randomized, double-blind, and placebo-controlled study in about 32 adults with CF. The safety and tolerability of single and multiple escalating doses of nebulized MRT5005 will be evaluated, and compared to each other and a placebo.
The trial’s main goal is to measure the treatment’s safety and tolerability, and a second goal or endpoint is measurements of mRNA delivery to bronchial epithelial cells, or cells that line the airways, after the fifth dose. Forced expiratory volume in one second (FEV1), a measure of lung function, will also be assessed at several points.
“As our … first clinical trial to evaluate a product candidate derived from our mRNA technology (MRT) platform, this is a significant milestone for the Company,” Ronald Renaud, its chief executive officer, said in a press release. “It also represents a pivotal time in the mRNA field as it will be the first mRNA therapeutic to enter the clinic with targeted delivery to the lung.”
Translate Bio, based in Lexington, Massachusetts, is working with the Cystic Fibrosis Foundation Therapeutics Development Network in conducting this trial.
“We are excited to get this study underway because it represents a new approach to treating CF. It is designed to treat the underlying cause of the disease regardless of an individual patient’s mutation by giving the body instructions to produce its own functional protein,” Renaud said.
More information about this trial is available by clicking here or on the trial’s identification number, given above.