Vertex Moves Its Alpha-1 Antitrypsin Deficiency Program Forward

Vertex Pharmaceuticals Inc VRTX has advanced its investigational program targeting alpha-1 antitrypsin deficiency (AATD), a rare genetic disease characterized by a protein folding defect that can lead to liver and lung disease.
Vertex announced that the FDA had cleared the Investigational New Drug (IND) Application for VX-634, enabling the company to initiate a first-in-human clinical trial for this small molecule AAT corrector in healthy volunteers.
Also see: Vertex Pharma's Flagship Cystic Fibrosis Drug Scores FDA Approval For Infants.
Additionally, Vertex will initiate a 48-week Phase 2 study of VX-864, a first-generation AAT corrector, to assess the impact of longer-term treatment on polymer clearance from the liver and the resultant levels of functional AAT (fAAT) in the plasma.
Consistent with its portfolio approach for all programs, Vertex is bringing forward additional next-wave AAT correctors, with the next molecules expected to enter the clinic starting in 2023.
Price Action: VRTX shares are up 1.33% at $298.47 on the last check Tuesday.