- Novel, inhaled mRNA therapy intended for the ~5,000 people with CF who cannot benefit from CFTR modulators -
- VX-522 clinical trial in people with CF to initiate in coming weeks -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration has cleared its Investigational New Drug (IND) application for VX-522, a messenger ribonucleic acid (mRNA) therapy targeted at treating the underlying cause of cystic fibrosis (CF) lung disease for the approximately 5,000 people with CF who cannot benefit from a cystic fibrosis transmembrane conductance regulator (CFTR) modulator. Vertex plans to initiate a single ascending dose clinical trial for VX-522 in people with CF in the coming weeks.
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VX-522 is delivered to the lung through inhalation of a CFTR mRNA encapsulated by a lipid nanoparticle. Once delivered to the target lung cells, the mRNA is designed to produce functional copies of the CFTR protein. VX-522 is the result of an exclusive research collaboration established with Moderna in 2016.
“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,” said Reshma Kewalramani, M.D., FASN, Chief Executive Officer and President, Vertex. “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.”
“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 Moderna Chief Executive Officer Stéphane Bancel. “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. We are excited by the progress that has been made with the upcoming advancement of VX-522 to the clinic and look forward to our ongoing collaboration to develop treatments for the underlying cause of cystic fibrosis.”About VX-522
This investigational messenger ribonucleic acid (mRNA) therapy aims to address the underlying cause of cystic fibrosis (CF). It is being evaluated by Vertex to treat lung disease for people living with CF who cannot benefit from cystic fibrosis transmembrane conductance regulator (CFTR) modulator treatments because they do not make any CFTR protein that responds to a CFTR modulator therapy. Globally, this represents a population of approximately 5,000 people living with this very rare form of the disease. The therapy is designed to deliver full length CFTR mRNA encapsulated in a lipid nanoparticle via inhalation directly to the lungs. Once delivered to target airway cells, the mRNA enables the production of functional CFTR protein. Improvements in CFTR quantity and function can lead to transformative benefits for people living with CF.
About the VX-522 Clinical Trial
Vertex plans to initiate a single dose escalation study in the coming weeks evaluating the safety and tolerability of VX-522 in people 18 years of age and the older with cystic fibrosis and a CFTR genotype not responsive to CFTR modulator therapy.
About the Vertex and Moderna Collaboration
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 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.
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