Cystic fibrosis
The company’s three-drug combination met all primary and key secondary endpoints in two randomized controlled trials in cystic fibrosis patients, paving the way for an application to regulators.
The biopharma company reported sales of $2.24 billion in the second quarter for the cystic fibrosis treatment, beating analysts’ estimate, after it won FDA approval in children ages two to five in April.
Drugs that act on the CFTR protein only work in patients who produce the protein in the first place. That leaves 6% of patients hanging.
Vertex is kicking off a Phase I trial assessing VX-522, an mRNA therapy designed to treat the underlying cause of cystic fibrosis lung disease, while SpliSense is targeting a specific mutation.
Sionna hit the ground with a pipeline of first-in-class small molecules designed to fully restore the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein in CF patients.
Vertex has made a point over the past decade to make research and development a priority, investing over 70% of its expenses back into it.
Suliman anticipates filing an Investigational New Drug application with the FDA for the PCD asset this year, and next year filing one for the cystic fibrosis asset.
Avilar Therapeutics officially joined the game with $60 million in seed financing from RA Capital Management.
This first-of-its-kind collaboration will leverage the talent and innovation at companies such as Tessera Therapeutics to advance multiple candidates to human proof of concept.
Mammoth Biosciences just announced a collaboration with Vertex to develop in vivo gene-editing therapeutics for patients with either of two serious, but not-yet-disclosed diseases.
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