VX-864 / Vertex - LARVOL DELTA

A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype (clinicaltrials.gov) - P2 | N=14 | Completed | Sponsor: Vertex Pharmaceuticals Incorporated | Active, not recruiting ➔ Completed | N=20 ➔ 14 | Trial completion date: Dec 2024 ➔ Aug 2024

Enrollment change • Trial completion • Trial completion date • Alpha-1 Antitrypsin Deficiency • Genetic Disorders • Hepatology • Pulmonary Disease • Respiratory Diseases

A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype (clinicaltrials.gov) - P2 | N=20 | Active, not recruiting | Sponsor: Vertex Pharmaceuticals Incorporated | Recruiting ➔ Active, not recruiting

Enrollment closed • Alpha-1 Antitrypsin Deficiency • Genetic Disorders • Hepatology • Pulmonary Disease • Respiratory Diseases

A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype (clinicaltrials.gov) - P2 | N=20 | Recruiting | Sponsor: Vertex Pharmaceuticals Incorporated | Not yet recruiting ➔ Recruiting

Enrollment open • Alpha-1 Antitrypsin Deficiency • Genetic Disorders • Hepatology • Pulmonary Disease • Respiratory Diseases

A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype (clinicaltrials.gov) - P2 | N=20 | Not yet recruiting | Sponsor: Vertex Pharmaceuticals Incorporated

New P2 trial • Alpha-1 Antitrypsin Deficiency • Genetic Disorders • Hepatology • Pulmonary Disease • Respiratory Diseases

Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype (clinicaltrials.gov) - P2; N=44; Completed; Sponsor: Vertex Pharmaceuticals Incorporated; Active, not recruiting ➔ Completed

Trial completion • Alpha-1 Antitrypsin Deficiency • Hepatology • Pulmonary Disease • Respiratory Diseases • AAT

"VX-864 will *not* advance into late-stage development" (@skathire)

Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype (clinicaltrials.gov) - P2; N=40; Active, not recruiting; Sponsor: Vertex Pharmaceuticals Incorporated; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • Alpha-1 Antitrypsin Deficiency • Hepatology • Respiratory Diseases • AAT

Vertex Reports Full-Year and Fourth-Quarter 2020 Financial Results (Vertex Press Release) - "Enrollment is ongoing in a Phase 2 proof-of-concept study for the Z-AAT corrector, VX-864. Data from this study is expected in the first half of 2021."

P2 data • Alpha-1 Antitrypsin Deficiency

Vertex Reports Third-Quarter 2020 Financial Results (Businesswire) - "Vertex is evaluating multiple compounds with the potential to correct the misfolding of Z-AAT protein in the liver, in order to increase the levels of functional AAT in the blood. Misfolded Z-AAT protein is the root cause of AAT deficiency; Enrollment is ongoing in a Phase 2 proof-of-concept study for the Z-AAT corrector, VX-864. Data from this study is expected in the first half of 2021....Enrollment is ongoing in a Phase 2 proof-of-concept study designed to evaluate the reduction in proteinuria in people with APOL1-mediated FSGS after treatment with VX-147. Data from this study is expected in 2021."

Enrollment status • P2 data • Alpha-1 Antitrypsin Deficiency • Focal Segmental Glomerulosclerosis

Vertex Provides Update on its Clinical Programs Targeting Alpha-1 Antitrypsin Deficiency (Businesswire) - P2, N=50; NCT04167345; Sponsor: Vertex Pharmaceuticals Incorporated; "Phase 2 study of VX-864 continues to enroll and dose patients; data expected in H1 2021; Based 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....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....Analysis of the PK data from the study indicated that exposures achieved were low....AATD Research Program: Vertex also continues to advance multiple small molecule correctors in late stage research with a goal of advancing at least one additional molecule into development in 2021."

Clinical • Discontinued • P2 data • PK/PD data • Trial status • Alpha-1 Antitrypsin Deficiency

Vertex Reports Second-Quarter 2020 Financial Results (StockHouse) - "Alpha-1 antitrypsin (AAT) deficiency....Enrollment and dosing have been re-initiated at some but not all sites following a temporary COVID-19-related pause in a phase 2 proof-of-concept study designed to evaluate the levels of circulating, functional AAT protein after treatment with VX-814...A phase 2 proof-of-concept study for a second Z-AAT corrector, VX-864, was initiated in July."

Enrollment open • Enrollment status • Alpha-1 Antitrypsin Deficiency

Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype (clinicaltrials.gov) - P2; N=40; Recruiting; Sponsor: Vertex Pharmaceuticals Incorporated; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Alpha-1 Antitrypsin Deficiency • AAT

Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype (clinicaltrials.gov) - P2; N=40; Not yet recruiting; Sponsor: Vertex Pharmaceuticals Incorporated

Clinical • New P2 trial • Alpha-1 Antitrypsin Deficiency

Edited transcript of VRTX earnings conference call or presentation 29-Apr-20 (Yahoo Finance) - "...VX-814 study in the AATD disease area. This is a Phase II dose-ranging study, proof-of-concept study....we had temporarily paused screening and enrollment, and where we are today is that we are in the process of reinitiating screening and enrollment on a site-by-site, country-by-country, region-by-region basis in those areas that are opening and are now able to resume clinical trial activity.....VX-864 is the next molecule in our AATD program. That one has completed its Phase I study. We've had a chance to look at the safety and the PK. That one is going to be ready to go into its Phase II proof-of-concept dose-ranging study, and I anticipate that that's going to happen in this year, the second half of this year."

New P2 trial • Trial completion • Trial status • Alpha-1 Antitrypsin Deficiency • Genetic Disorders

Vertex reports full-year and fourth-quarter 2019 financial results (Businesswire) - "VX-814: Enrollment is ongoing in the Phase 2 proof-of-concept study evaluating VX-814...for the treatment of alpha-1 antitrypsin (AAT) deficiency. Vertex expects to obtain data from this study in 2020. VX-864: A Phase 1 study is ongoing in healthy volunteers evaluating VX-864...investigational small molecule corrector for the treatment of AAT deficiency."

P2 data • Trial status