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Vertex Provides Update on its Clinical Programs Targeting Alpha-1 Antitrypsin Deficiency
- Phase 2 study of VX-814 in patients with alpha-1 antitrypsin deficiency discontinued based upon safety and pharmacokinetic data - - Phase 2 study of VX-864
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n- Phase 2 study of VX-814 in patients with alpha-1 antitrypsin deficiency discontinued based upon safety and pharmacokinetic data -\n\n- Phase 2 study of VX-864 continues to enroll and dose patients; data expected in H1 2021 -\n\n- AATD research program continues to progress -\n\n BOSTON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today provided an update on its clinical programs targeting the small molecule correction of alpha-1 antitrypsin deficiency (AATD).\n\nVX-814 Phase 2 Proof-of-Concept Study \n\nBased on the safety and pharmacokinetic (PK) profile of VX-814 observed to date in a Phase 2 trial in AATD, Vertex has decided to stop dosing in the trial and will discontinue development of VX-814. The randomized, double-blind, placebo-controlled Phase 2 study of approximately 50 patients was designed to evaluate the safety and PK of VX-814, and the ability of VX-814 to increase functional levels of alpha-1 antitrypsin over 28 days of dosing. Elevated liver enzymes (AST/ALT) were observed in several patients. In four patients, across different doses studied, elevations greater than 8 times the upper limit of normal were noted; the elevated liver enzymes have either resolved or are resolving. No patients had concomitant elevation of bilirubin levels and all patients were asymptomatic. Analysis of the PK data from the study indicated that exposures achieved were low. Based on these data, Vertex concluded that it would not be feasible to safely reach targeted exposure levels, and thus meaningful increases in AAT levels, with VX-814, and the study was stopped.\n\n“Based on the liver enzyme elevations observed, along with the determination that we would not be able to safely achieve targeted exposure levels with VX-814, we are discontinuing further development of this molecule,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “We are grateful to the AATD patients and investigators who participated in the VX-814 studies and we remain committed to transforming the treatment of this disease. We look forward to continuing clinical study of VX-864 and other molecules targeting the underlying cause of AATD.”\n\nVX-864 Phase 2 Proof-of-Concept Study \n\nA Phase 2 trial of VX-864, which is structurally distinct from VX-814,...