Clinical Trials

Date: 2014-10-09

Type of information: Presentation of results at a congress

phase: 2

Announcement: presentation of results at the 28th Annual North American Cystic Fibrosis Conference (NACFC), Atlanta, Georgia, USA, October 9-11, 2014.

Company: Vertex Pharmaceuticals (USA - MA)

Product: VX-661 ( tezacaftor) in combination with ivacaftor

Action mechanism:

• CFTR potentiator. Kalydeco^® (ivacaftor) is a potentiator of the CFTR protein. The CFTR protein is a chloride channel present at the surface of epithelial cells in multiple organs. Ivacaftor facilitates increased chloride transport by potentiating the channel-open probability (or gating) of the G551D-CFTR protein.This oral medicine aims to help the CFTR protein function more normally once it reaches the cell surface, to help hydrate and clear mucus from the airways. VX-661 is another CFTR potentiator.

Disease: cystic fibrosis

Therapeutic area: Rare diseases - Genetic diseases

Country:

Trial details:

Latest news:

• • On October 9, 2014, Vertex Pharmaceuticals reviewed recent progress and announced upcoming milestones in its efforts to develop multiple combinations of medicines that treat the underlying cause of cystic fibrosis for the majority of people with the disease. These updates were made in conjunction with the 28th Annual North American Cystic Fibrosis Conference (NACFC).
• VX-661 in Combination with Ivacaftor VX-661 is Vertex's second CFTR corrector and is being developed to play a role in multiple combinations of CFTR modulators aimed at treating people with CF who have one or two copies of the F508del mutation. Enrollment Complete in 12-week Phase 2 Study: VX-661 is currently being evaluated in combination with ivacaftor as part of a 12-week Phase 2b study in people ages 18 and older who have two copies of the F508del mutation. Vertex announced that this study is fully enrolled and that data will be available in early 2015.
• Pivotal Phase 3 Program of VX-661 Planned for First Half of 2015: Vertex announced plans to initiate a pivotal Phase 3 clinical program of VX-661 in combination with ivacaftor in the first half of 2015, pending regulatory discussions and data from the ongoing 12-week Phase 2b study in people with two copies of the F508del mutation.
• This Phase 3 program is expected to evaluate efficacy and safety of VX-661 in combination with ivacaftor in people with the following CFTR mutations:
• • Two Copies of the F508del Mutation, based on previously announced data from a Phase 2 study of VX-661 in combination with ivacaftor in people with two copies of the F508del mutation to be presented at NACFC ("Phase 2 studies reveal additive effects of VX-661, an investigational CFTR corrector, and ivacaftor, a CFTR potentiator, in patients with CF who carry the F508del-CFTR mutation."
• • One Copy of the F508del Mutation and a Second Mutation That Results in a Gating Defect in the CFTR Protein, based on data announced in mid-2014 from a Phase 2 proof-of-concept study in people with the F508del mutation and G551D mutation to be presented at NACFC .
• • One Copy of the F508del Mutation and a Second Mutation That Results in Residual CFTR Function, based on data announced in mid-2014 from a Phase 2 proof-of-concept study of ivacaftor in people with a residual function mutation to be presented at NACFC.Additionally, based on data from the Phase 2 study of ivacaftor in people with a residual function mutation, referenced above, Vertex plans to evaluate ivacaftor used as monotherapy in people with these mutations as part of the pivotal program.
• • One Copy of the F508del Mutation and A Second Mutation That Results in Minimal CFTR Function - The evaluation of people with one copy of the F508del mutation and one copy of a CFTR mutation that results in minimal CFTR function as part of the pivotal Phase 3 program would be the first evaluation of VX-661 in combination with ivacaftor in people with these mutations. To date, treatment with a combination of lumacaftor and ivacaftor has not resulted in a significant improvement in lung function for people with these mutations.
• Planning for the pivotal Phase 3 program is underway to support the initiation of enrollment in the first half of 2015, pending regulatory discussions planned for later this year and the data from the ongoing Phase 2b study, which are expected in early 2015. Triple Combination of VX-661, Ivacaftor and a Next-Generation Corrector: Vertex has multiple next-generation correctors in the lead-optimization stage of research and expects to begin clinical development of a next-generation corrector in 2015. In vitro data showed that a triple combination of VX-661, ivacaftor and a next-generation corrector resulted in increased chloride transport in human bronchial epithelial cells with one or two copies of the F508del mutation, as compared to the use of a single corrector in combination with ivacaftor.

Is general: Yes