VX-522, for CF Patients Who Can’t Use Modulators, to Enter Trial

Potential inhaled Vertex treatment for functional CFTR protein in lung cells

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Vertex Pharmaceuticals is launching a clinical trial to test VX-522, its inhaled messenger RNA (mRNA) therapy for lung disease in cystic fibrosis (CF) patients who are not eligible for treatment with an existing CFTR modulator.

The study is expected to open soon, and it will assess VX-522’s safety and tolerability at single ascending doses in adults with CF, ages 18 and older, according to a company press release.

A Vertex request to open the trial, in the form of an investigational new drug (IND) application, was recently cleared by the U.S. Food and Drug Administration.

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“It has been our longstanding goal to bring highly effective therapies to all people with CF. Clearance of the IND represents a pivotal turning point in reaching the remaining [approximately] 5,000 people with CF who are still waiting for a medicine to treat the underlying cause of their disease,” said Reshma Kewalramani, MD, Vertex’s CEO and president.

VX-522 is being developed with Moderna, a company that specializes in messenger RNA (mRNA) therapeutics, under a research collaboration established in 2016. Of note, mRNA is the DNA-derived template cells use to make the proteins.

“This partnership brings together Vertex’s scientific expertise and decades of experience in developing cystic fibrosis medicines with Moderna’s proven leadership in mRNA technologies,” said Stéphane Bancel, Moderna’s CEO.

CF is caused by mutations in the CFTR gene, which codes for a protein of the same name. A faulty CFTR protein, which normally acts like a gate in regulating the transport of chloride ions and water in and out of cells, leads to the accumulation of a thick and sticky mucus that clogs the airways, digestive tract, and reproductive system.

Such mucus buildup in the airways is known to cause chronic lung infections and progressive lung damage.

CFTR modulators help to correct the protein’s functionality in people with specific CF-causing mutations. However, about 1 in 10 patients are ineligible for these medications because of underlying mutations.

As an inhaled treatment, VX-522 is intended to deliver full-length CFTR mRNA directly to cells in the lungs.

The mRNA is enclosed in lipid (fat) nanoparticles and, once inside the cells, is expected to give rise to the production of a working CFTR protein.

“Moderna’s development of a proprietary inhalable lipid nanoparticle to deliver a functional cystic fibrosis treatment to the lungs could lead to a transformational medical achievement,” Bancel said. “We are excited by the progress that has been made.”

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