Research Update

Positive Results from Phase 3 Study of Kalydeco in Select CF Patient Groups By
by Andrea Flynn

Kalydeco™ was approved by the Food and Drug Administration in 2012 to treat individuals 6 years or older with the G551D gating mutation. Gating mutations like G551D allow the CF protein to reach its proper place in the cell membrane, but prevent it from opening and allowing for proper chloride transport. While G551D is the most common gating mutation, there are approximately 400 additional individuals with other kinds of CF gating mutations. Thus, an additional clinical trial was initiated to examine the efficacy of Kalydeco in individuals with three alternate gating mutations.

Vertex announced the initial promising results from this Phase 3 study earlier this summer. Study participants age 6 or older with non-G551D gating mutations who received Kalydeco showed statistically significant improvements in lung function and weight gain compared to placebo. Vertex plans to submit a new drug application to the FDA for the use of Kalydeco in individuals 6 and older with at least one copy of a CF gene with a non-G551D gating mutation.

Vertex is also currently enrolling two large-scale, international studies to examine the effectiveness of Kalydeco in combination with VX-809 in individuals with two copies of the most common CF mutation, Delta F508.

North American CF Conference Highlights

Researchers, physicians, and caregivers recently met in Salt Lake City, Utah for the 27th annual North American Cystic Fibrosis Conference.  The conference, sponsored by the Cystic Fibrosis Foundation, is the leading forum for 4,000 of the world’s cystic fibrosis experts to present cutting-edge scientific data, exchange ideas and discuss innovative approaches to CF research and care.

“This annual meeting reflects the dedication and cooperation of a world-class group of researchers and physicians and is integral in continuing to bring highly effective and innovative treatments to all individuals with CF,” said Martine Denis, Executive Director Greater New York Chapter.

This year’s meeting focused on current therapies in development to treat the basic genetic defect of CF, including all identified CF mutations, and the progress made in addressing CF pulmonary symptoms. Topics also included early detection and treatment for CF-related diabetes and how to best treat the emerging adult CF population.

N6022 Phase 2 Study Now Recruiting

S-nitrosoglutathione or GSNO is an important signaling molecule that is decreased in individuals with CF. N30 Pharmaceuticals, Inc. is currently developing N6022, an injectable compound that modulates GSNO levels to increase and stabilize the amount of CFTR at the cell membrane. A multi-center Phase 1b trial is currently enrolling volunteers to examine the effectiveness of N6022 in people with two copies of the F508del-CFTR mutation. For more information visit cff.org or clinicaltrials.gov.