Focus on Cystic Fibrosis Planned for Vertex’s 2016 R&D Program

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Cystic fibrosis research and development

Vertex Pharmaceuticals, a biotech company that discovers, develops, and commercializes medicines for a series of diseases, currently with a clinical development program focused on cystic fibrosis (CF), recently announced its key business priorities for 2016.

Vertex currently has two medicines approved for cystic fibrosis treatment: KALYDECO (ivafactor) and ORKAMBI (lumacaftor/ivacaftor).

ORKAMBI was approved in 2015 in both the United States and the European Union, for the treatment of patients with cystic fibrosis age 12 and older with two copies of the F508del mutation in the CFTR gene.

At the moment, Vertex is conducting two Phase 3 clinical trials to obtain ORKAMBI’s approval for the treatment of children 6 to 11 years old. The first study is evaluating the drug in 50 children and is directed for U.S. FDA approval, whereas the second study is evaluating the treatment in 200 children, for approval in the European Union.

As for KALYDECO, the drug has been approved for the treatment of cystic fibrosis patients age 2 and older with one of 10 specific CF gene mutations. Vertex aims to continue their label-expansion efforts in order to increase the eligible population for this medicine, currently with a supplemental new drug application under review by the FDA for its use in people age 2 and older with one of 23 functional mutations, covering about 1,500 patients in the U.S.

Vertex also plans to initiate a clinical study on KALYDECO for the treatment of children younger than 2 with one of the 10 mutations for which the drug is currently approved. The study is expected to begin in the first quarter of 2016.

In addition, Vertex highlighted a pipeline of several investigational medicines for cystic fibrosis. These include four Phase 3 studies to investigate the therapeutic potential of the combination VX-661 plus ivacaftor in CF patients who have at least one copy of the F508del mutation.

Besides data on efficacy, the scientists expect to obtain promising safety results in order to support a planned development of a triple combination regimen with a next-generation corrector, such as VX-152 and VX-440, which are also being evaluated alone.

VX-371, an investigational epithelial sodium channel (ENaC) inhibitor, is being developed by Vertex in collaboration with Parion Sciences as a treatment for cystic fibrosis regardless of the CFTR mutation present. The CLEAN-CF clinical trial is currently recruiting participants with a confirmed diagnosis of cystic fibrosis and any CFTR mutation. The primary endpoint of the trial is to determine the safety profile of the drug.

Also, through its collaboration with CRISPR Therapeutics, announced in October 2015, Vertex will evaluate the use of CRISPR’s gene editing technology, known as CRISPR-Cas9, to discover and develop treatments that target and correct the underlying genetic cause of CF.

Vertex’s priorities, which lie mostly on the continuing development of the two approved CF medicines and the exploration of other therapies to treat the disease, have been presented by Vertex CEO, Chairman and President Jeffrey Leiden, M.D., at the 34th Annual J.P. Morgan Healthcare Conference in San Francisco.

Vertex also disclosed information concerning other research and development programs in areas such as oncology, pain, and influenza. For more information, read the complete press release here.