Vertex Pharmaceuticals said that it plans to initiate a Phase 1 SAD trial of its VX-522 inhaled mRNA therapy after the FDA cleared the company’s IND. VX-522 contains lipid nanoparticle-encapsulated mRNA that is designed to treat cystic fibrosis in patients who do not produce cystic fibrosis transmembrane conductance regulator (CFTR) and are therefore not helped by a CFTR modulator. Vertex is partnered with Moderna on development of VX-522.
Vertex President and CEO Reshma Kewalramani said, “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 ~5,000 people with CF who are still waiting for a medicine to treat the underlying cause of their disease. The partnership with Moderna that began more than five years ago has been instrumental in achieving this milestone, and we look forward to continuing our work together.”
VX-522 is the result of an exclusive research collaboration Vertex established with Moderna in 2016 to discover and develop mRNA therapeutics for CF. Under the terms of the collaboration, Moderna leads asset identification efforts, combining its leading mRNA platform technology and mRNA delivery expertise together with Vertex’s scientific experience in CF biology, functional understanding of CFTR, and Vertex’s proprietary assay platform that utilizes human bronchial epithelial (HBE) cells of multiple different CF gene mutations from people with CF. Vertex leads preclinical and clinical development, regulatory and commercialization activities associated with the advancement of mRNA therapeutics that result from this collaboration and will fund all expenses related to the collaboration. Moderna is responsible for mRNA and lipid nanoparticle (LNP) process development and manufacturing. Per Vertex’s agreement with Moderna, Moderna will receive certain milestone payments, as well as royalty payments from this collaboration.
Read the Vertex press release.
