- Vertex Pharmaceuticals Inc VRTX has progressed its testing program targets alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder characterized by a protein folding defect that can lead to liver and lung disease.
- Vertex announced that the FDA has approved an Investigational New Drug (IND) application for VX-634, allowing the company to initiate a first-in-human clinical trial of this small molecule AAT corrector in healthy volunteers.
- See also: Vertex Pharma’s Flagship Cystic Fibrosis Drug Receives FDA Approval for Infants.
- In addition, Vertex will initiate a 48-week Phase 2 study of VX-864, a first-generation AAT corrector, to determine the effects of long-term treatment on polymer clearance from the liver and resulting levels of functional AAT (fAAT) assess the plasma.
- Consistent with its portfolio approach to all programs, Vertex is advancing additional next-generation AAT correctors, with the next molecules expected to be in the clinic beginning in 2023.
- Price promotion: VRTX shares are up 1.33% at $298.47 as of the latest check Tuesday.
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